Research Australia - INSPIRE Issue 21: The consumer's role in Health & Medical Research by Research Australia

2021 | 021

A FOCUS ON THE CONSUMER'S ROLE IN HEALTH AND MEDICAL RESEARCH

FOREWORD This issue of INSPIRE is all about keeping the consumer in focus, and I think the overwhelming response from the membership to this theme speaks volumes. If not for the patient, then who?

s the peak body for health and medical research we are aware of the central importance of the consumer. The researchers themselves are very aware, the charities who fundraise fo r r e s e a r c h a r e awa r e, a n d certainly the commercial entities in our sector are aware. But do we tell the story well enough? England’s former Chief Medical Officer, Professor Dame Sally Davies once famously said, “No matter how complicated the research or how brilliant the researcher, the patients and the public always offer unique, invaluable insight.” In this issue we bring together real-life examples of how consulting with the consumer makes for better research. The myriad benefits include greater accountability, improved quality and outcomes, decreased costs, more effective research translation and improved public confidence in research. Increasingly, research funding bodies are demanding a demonstration of consumer involvement as a condition of funding, and it is great to hear how some of our charity members, in particular, are collaborating with consumers. In this is sue a nd the nex t, we specifically asked our members to highlight how they involve the consumers in their research or

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in their decision-making. I was delighted to read just how integrated consumers are across the pipeline, and in a diversity of areas, including guiding advisory panels, sitting on strategic review panels, all manner of volunteering, participation in clinical trials, and much more. The Red Cross's Lifeblood team includes social researchers who help to understand what keeps donors coming back and create a better experience for their volunteers. L ikew ise, S yd n ey U n i ve r s i t y is putting user experience research at the forefront of its research into m o b i l e h e a l th (m H e a l th) a p p s, telehealth plat forms, immersive technologies and or clinical-decision support systems a comprehensive understanding of a user’s specific needs and attitude toward digital health interventions. Rare Voices Australia is seeking to move beyond the conventional, sometimes tokenistic, approaches to incorporating consumer expertise. They believe consumer engagement in research should extend beyond participation, and that the consumer perspective and expertise can help to identify gaps and research priorities. In The Last Word, one of our most popular regular features, Dr Andrew Nash, Chief Scientific Officer, shares CSL’s views on these themes. We are lucky to have had Andrew as a

Board Director of Research Australia for a number of years and his views from the commercial end of the pipeline are invaluable. The call for abstracts on this topic clearly hit the mark for you and we received a massive response. So much so that we are dedicating two issues of INSPIRE to this significant topic. Thank you for sharing your work with us so we can amplify your efforts across our broad sector and to demonstrate the importance of health and medical research in our lives, and to keep in mind the all-important why. Healthy wishes Nadia and the Research Australia Team CEO – Research Australia

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CONTENTS

Australian Health & Medical Research & Innovation

Events overview

Turning up the volume on gynaecological cancer

Roles and Impacts of Consumer Engagement in Research

RESEARCH AUSTRALIA

UNIVERSITY OF QUEENSLAND

26 Strengthening consumer engagement in rare disease research RARE VOICES AUSTRALIA

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Publisher Research Australia Ltd Art Direction Matthew Ware p +61 403 844 763 or e matt@objktive.com For Advertising enquiries please contact the Research Australia office on p 02 9295 8546 or e admin@researchaustralia.org researchaustralia.org

INSPIRE ONLINE issuu.com/researchaustralia

22 World-first research revolutionising heart transplant surgery VICTOR CHANG CARDIAC RESEARCH INSTITUTE

CHRISTOPHE KEREBEL

24 Survival but not at any cost THE KIDS CANCER PROJECT

CHRISTOPHE KEREBELINSPIRE My : @chriskereof is Twitter aKEREBEL publication CHRISTOPHE

My Twitter : @chriskere

Research Australia Ltd ABN 28 095 324 379 384 Victoria Street Darlinghurst NSW 2010 Who can submit articles? Any current member of Research Australia who would like to share a relevant story that affects their organisation including, philanthropic donations and their outcomes, research findings, and any other related health and medical research topic that affects the Australian population. Submission guidelines & deadlines For information regarding how to submit and publishing deadlines visit the Research Australia website. Disclaimer The opinions expressed in INSPIRE do not necessarily represent the views of Research Australia. Whilst every effort has been made to ensure accuracy, no responsibility can be accepted by Research Australia for omissions, typographical or inaccuracies that may have taken place after publication. All rights reserved. The editorial material published in INSPIRE is copyright. No part of the editorial contents may be reproduced or copied in any form without the prior permission from Research Australia. © Research Australia 2020.

34 How one mother used her voice to open the door for thousands TELETHON KIDS INSTITUTE

36 Participation First UNIVERSITY OF WESTERN AUSTRALIA

CONTENTS

Australian Health & Medical Research & Innovation

A journey of mutual discovery

Collaborating with people around the globe in Glycome Research

Collaborating with parents of children with long-term conditions

EDITH COWAN UNIVERSITY

56 Guiding drug development with patient insight CSL

54 Patient-empowered research in prostate cancer UNIVERSITY OF MELBOURNE

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62 Designing with Donors UNIVERSITY OF SYDNEY

CHRISTOPHE KEREBEL

My Twitter : @chriskere CHRISTOPHE KEREBEL

44 Community and researchers unite for cerebral palsy research CPA RESEARCH INSTITUTE

58 Understanding the consumer experience to enhance quality care in the digital age UNIVERSITY OF SYDNEY

50 The Power of the Consumer Voice UNIVERSITY OF MELBOURNE

THE LAST WORD

66 Involving the consumer at the outset of research MURDOCH CHILDREN'S RESEARCH INSTITUTE

Putting consumer leadership in the spotlight

Building the unbroken pipeline

THE AUSTRALIAN NATIONAL UNIVERSITY

CSL

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RESEARCH AUSTRALIA AT OTHER EVENTS! NOVO NORDISK OCEANIA ON THE MOVE Research Australia’s CEO Nadia Levin was proud to be a guest of honour at the official opening of Novo Nordisk’s new head office in North Sydney on June 7. The move follows over two decades being based in Baulkham Hills and supports a significant expansion of the Oceania organisation and an expanding product pipeline beyond diabetes and obesity. “As Novo Nordisk innovates and transforms into the wider chronic disease space, we are also supporting our workforce with innovative and transformative work environments. “Novo Nordisk will be continuing the search for innovative, growth-focused individuals in Australia to support both our growth into these new areas, and capitalising on our current expertise.” Says Jeppe Theisen, Vice President and Managing Director. Left Nadia Levin | Research Australia Middle Elie Antoun | Novo Nordisk Right Jodie Thomas | Novo Nordisk

Left Jeppe Theisen, Novo Nordisk & Nadia Levin, Research Australia

Novo Nordisk is a leading global healthcare company, founded in 1923 and headquartered in Denmark. Its purpose is to drive change to defeat diabetes and other serious chronic diseases by pioneering scientific breakthroughs, expanding access to medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 45,800 people in 80 countries and markets its products in around 170 countries

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ARCS Dr Buzz Palmer from MedTech Actuator invited Research Australia's CEO to be a panelist at the ARCS Australia Annual Conference on June 8 at ICC Darling Harbour. The sessions were focused around science innovation, entrepreneurship and commercialisation. The key subject was ‘Funding models for early-stage research and innovation and the panel explored how founders and entrepreneurs should be thinking about funding in early stage research. What models need to be considered when approaching funding organisations? How do you prepare for these meetings and articulate your value proposition?

ARCS Australia is a national, membership-based organisation focused on the development and growth of the healthcare sector. ARCS provides education, career pathways, professional development and advocacy to the healthcare sector.

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TELSTRA HEALTH LAUNCHES KYRA On 20 April, Telstra Health announced the launch of Kyra, its suite of integrated digital solutions for hospitals, bringing together customers, health and hospital sector representatives and Telstra health employees, attending both in person and virtually at the Sydney event. Compered by CEO and Managing Director of Research Australia, Nadia Levin, event attendees listened to an insightful introduction from Telstra Health’s Managing Director, Professor Mary Foley AM. This was followed by the launch of Kyra by Telstra Health’s Graeme Osborne, Head of Hospitals and Connected Health, including a high-level overview of the suite of sophisticated digital health solutions that make up the Kyra Platform. Titled the ‘Future of Digital Hospitals’, the event also hosted a panel discussion between a number of notable digital health leaders and experts including Jeff Woods, Chief Nursing Officer at Healthscope, Melanie Gates , Head of Product, Solutions and Virtual Care Solutions at Telstra Health, Peter Crome, General Manager, Customer Solutions, Hospitals and Connected Health at Telstra Health, and Tricia Liebke, Digital Health Strategic Consultant. The panellists opened a discussion about the journey digital hospitals have experienced in recent years, the digital challenges they have faced and currently face, and the vital role digital health technologies can play in supporting the delivery of care in hospitals. Kyra connects hospitals to the health ecosystem and supports clinical teams to deliver safe, high quality care and positive patient experiences. The new digital hospital platform uses data visualisation to optimise models of care and patient experiences as well as decision support and predictive analytics to help support and enhance the role of clinicians and clinical teams. Research Australia CEO Nadia Levin facilitated a panel including Graeme Osborne, Head of Hospitals and Connected Health, Telstra Health, Jeff Woods, Chief Nursing Office, Healthscope, Melanie Gates, Peter Crome and Tricia Liebke from Telstra Health.

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AUSTRALIAN WOMEN IN SCIENCE SEMINAR Our CEO, Nadia Levin was invited by Dr Lana McClements from the University of Technology Sydney to present at one of their Australian Women In Science Seminars. Nadia spoke on work-life balance from the perspective of a CEO. ‘We have to be willing to sometimes blur the lines between work life and home life...to show that it’s not always perfect...but it is doable. This openness will enable the women who come after us to look at how they structure work around family. It will enable organisations, to look at new, more flexible models for the vexed question of parental leave. This openness will help us shift with purpose, the workplace that is undergoing a redesign to inclusivity, in

some places faster than others and there will be pockets of resistance for sure, however there is an onus on all of us to call out inequality, support those who do and know that you can pretend to be one of the boys as much as you like but it doesn’t change the need for an equitable society where gender is not a divide, it is not an absence, it is not a quota or a target – it just is. It’s important that we talk about how we enable women to participate in paid work. Not just because it’s nice to do. Not just because it’s the right thing to do. Not even just because it’s what women want. But because it makes good economic sense,ʼ said Nadia to a very engaged audience.

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RESEARCH AUSTRALIA AT OTHER EVENTS! ABC RADIO’S SCIENCE IN PUBLIC Each year five researchers are selected to undertake a two-week residency at ABC Radio National with the aim of giving a voice to early-career researchers.

Over two weeks they learn the craft of communicating with a wide audience from some of Australia’s best broadcasters and developing content across radio and digital platforms. The program is designed to nurture the communication skills and media awareness of emerging thinkers to help them share their knowledge and expertise with audiences seeking credible material and informed debate. As a researcher, when they step into the media spotlight, they become a spokesperson for science. By joining the conversation, they start to influence the decision makers in civil society, government, and industry. As they are getting an intensive insight into the media, we want to open their eyes to other forms of communication and how, what they have learnt can be used to assist them in their careers. As an ex ABC News reader and CEO of Research Australia, Nadia was invited to join the panel to help get the future leaders thinking about what impact they want to have, who they want to influence and why.

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Other Panellists included Cathy Foley, Australia’s Chief Scientist, Darren Saunders, a biomedical researcher and communicator and was moderated by Sarah Brooker, Managing Director of Science in Public.

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TURNING

THE VOLUME ON GYNAECOLOGICAL

CANCER

Forgotten, unnoticed, muted – these are common themes and words used by women diagnosed with gynaecological cancer (GC) to describe how they feel during their cancer journey. 14 INSPIRE 021 | 2021

he Queensland Centre for Gynaecological Cancer Research (QCGC Research) is an agile research team based at The University of Queensland’s Centre for Clinical research (UQCCR) that is using its research mission to give women diagnosed with gynaecological cancer a voice and much-needed encouragement to speak loudly. Gynaecological cancer patients are at the core of all planning, decisions and actions made by the research unit. This consumer-centric approach to research promotes and delivers much-needed positivity to patients

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recruited onto trials by helping women feel engaged and valued from early days of diagnosis, through their treatment program and beyond.

There have been some lonely and alone times where I have got quite frustrated with myself. There is plenty of support for breast cancer, but I found the network was not there for gynae girls.” Mia, vaginal cancer The early phase of patient engagement at QCGC Research is managed by clinician-researchers who play an integral role in laying a strong foundation for a consumer centric approach. Most of the ideas for future research projects are sparked by questions, experiences and outcomes that are communicated through patient and doctors’ interactions. These early interactions highlight issues and gaps that require further attention and investigation in order to find better treatments for women with gynaecological cancer.

I had no idea what the warning signs of this disease were. There is nowhere near enough education for women in regards to gynae cancer.” Kim, ovarian cancer The second critical phase of patient engagement at QCGC Research is the recruitment of patients to trials and gathering of data. There are multiple touch-points during the course of a study where research trial staff make contact with patients. The touch-points are in person wherever possible but more likely to be over the phone (for geographical reasons or at the request of the patient). These interactions are led by researchers but driven by patients. The women being interviewed are encouraged to talk about their health, wellbeing, milestones and other issues key to the research program involved. The QCGC Research also implemented and hosted the Annual Patient and Community Symposium. This event invites patients and members of their support network to attend an information session to learn more about the impact that research is having on the diagnosis, treatment and quality of life of women diagnosed with gynaecological cancer. A number of guest speakers are invited to present on a range of topics ranging from survivorship, health policy, new treatments, prevention and everything in between. Participants have the opportunity to pose questions and are encouraged to provide input into topics, themes and speakers for future symposiums.

This feedback has continued to strengthen attendance and support for the event each year.

It keeps cancer patients and their f ami l ie s i nf o r me d wit h al l t he research results so far undertaken. I also feel knowledge equates to hope when life seems bleak. I appreciate all that you all do and your devotion to, hopefully, give cancer p a t i e n t s a b e t t e r l i f e .” S y m p o s i u m participant 2019 CONSUMER ENGAGEMENT IN RESEARCH DOES NOT END ONCE A TRIAL CONCLUDES.

Patients who have participated in QCGC Research trials continue to receive invitations to patient symposiums and newsletters that outline research progress and other topics that will be of interest to women whose lives have been affected by gynaecological cancer. Finally, the establishment of consumer research panels for each of the gynaecological cancers creates an important engagement opportunity for both patients and QCGC Research. These panels represent the needs of cancer consumers and help set directions/priorities for future projects. It gives women and/or their carers an opportunity to provide significant input into finding better treatments. QCGC Research is incredibly grateful to every single woman who enrols as a participant on a research trial. The research team listens, records and reviews every comment, thought, suggestion offered. One voice added to others is too powerful to ignore or mute. Only together we will create change. “The QCGC Research team is humbled by the trust shown to us by every single woman who enrols on a research trial. We repay that trust by continuing to engage with our consumers well after each trial concludes. QCGC Research wouldn’t exist without them.” Professor Andreas Obermair, Director of QCGC Research. Gynaecological cancer patients and survivors are not forgotten or unnoticed. Through research, their voices are collectively making a positive difference to the lives of women now and in the future.

Author: Lisa Harrold Business Development Manager Queensland Centre for Gynaecological Cancer Research UQCCR. The University of Queensland

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ROLES AND IMPACTS OF CONSUMER ENGAGEMENT IN RESEARCH

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CHRISTOPHE KEREBEL

My Twitter : @chriskere CHRISTOPHE KEREBEL

Consumer engagement (CE) in health research refers to the active involvement of stakeholders throughout the research process including prioritisation of research efforts, study design and implementation, interpretation of results, and dissemination of findings.1 Recognition for the value of CE in health research is increasing.2

n fact, many institutions internationally have mandated that CE is used to inform research efforts. 3,4 Despite the increasing appreciation for CE, outcomes regarding the impact of CE on health research are rarely reported. Therefore, there is limited evidence to support its impact and value.5

This is potentially due to lack of reporting standards and standardised tools to evaluate the impact of CE on health research.5 This article will provide an overview of what dimensions of CE could be evaluated when introducing a CE program in a research environment, what tools, if any, have been created to capture this impact, and recommendations for successful CE. A 2015 review 6 reported on aspects of CE programs which should be considered in the evaluation process. The findings of this paper, in combination with subsequent literature has led us to the following summary: 2021 | INSPIRE 021 17

DIMENSION

RESEARCH QUESTION: DOES CE AFFECT…

Quality of research6-9

…the credibility and validity of the research?

Research focus/relevance6,9,10

…research alignment with interests and values of the wider community?

Study design/protocols6,8-10

… the review of ethical considerations and the appropriateness study designs?

Research Transparency6,7

…the transparency and/or reporting of methods?

Funding opportunities6,10

…funding opportunities?

REcruitment6,10,11

…amount of enrolment and retention rates, and the diversity/ representativeness of participants?

Results Interpretation 6-8,11

…interpretation of the research findings?

Research Dissemination

…the quality of research dissemination?

6,9

Consumer experience6-8

…consumer satisfaction, trust, and empowerment?

Scope OF ENGAGeMENT6,7,11

Have the researchers consulted with appropriate and diverse stakeholders throughout all stages of the research process?

TOOLS FOR EVALUATING CE IN RESEARCH

However, methods for evaluating the outcomes of CE in research are still lacking, and to our knowledge, none exist. Most commonly, studies evaluated the success of CE based on the consumer experience and diversity of the consumer group. Research evaluations to date can be summarised as follows: • Evaluation of consumer experience. We identified one qualitative12, and one quantitative13 survey, developed to evaluate consumer experience of engagement programs. • Consumer diversity: A 7-item Stakeholder Engagement Reporting Questionnaire measured consumer diversity.7 • Impact of CE on the quality, the ethics and relevance, and understanding of the research process has been anecdotally measured.6 • Impact on enrolment. Increased enrolment and retention rates were associated with CE programs in randomised control trials.10,11 A recently reported evidence-based tool, “GRIPP2”, is useful when reporting on CE programs in a standardized manner. 14

CONSIDERATIONS FOR NEW CE PROGRAMS

There are a diverse group of stakeholders involved in the CE programs including the institution, the researchers and the patient/public/community. We reviewed the literature to identify what unique barriers and facilitators for each stakeholder group, should be considered prior to establishing a new CE program.

INSTITUTIONAL LEVEL

The literature highlighted two major barriers to successful CE programs: lack of organisational policies; and lack of funding/resources allocated to CE programs.2,15,16 CE program success is strongest when institutional policies 18 INSPIRE 021 | 2021

require it as part of research, and in environments that are inclusive and culturally safe.2 Funding and resources to remunerate consumer for their expertise, and employing a CE coordinator, optimise the probability of program success.2

RESEARCHERS

Meaningful CE for researchers incurs time and fiscal costs7,10, and both should be budgeted for when planning new research projects.2,10,16 Outcomes are optimised when consumers are involved as early in the research process as possible, thereby positively impacting on study and recruitment design.2,10 Some reviews highlight the need for researchers improve communication skills with consumers by using lay language and developing educational materials to optimise consumers’ understanding of the research. 2,16 Furthermore, many reviews suggest that researchers benefit from training courses to empower them to optimally engage with consumers/consumer groups.2,15,16

PATIENT/PUBLIC/COMMUNITY

To enhance communication and empower patient/public/ community stakeholders, consumers should be offered training and resources.3,11 Ideally, it is recommended that CE be conducted in a face-to-face setting, with a diverse group of individuals, to ensure consumers feel comfortable and that they represent a wide range of perspectives.11 Training should include enhancing understanding of the research and scope of engagement.11,15 Ongoing communication facilitates a higher level of research engagement while ensuring that consumers feel valued.2,16

CONCLUSIONS

There are limited tools available for evaluating the impact of CE on health research. However, several domains have been identified which should be considered in the evaluation process. The development of further standardised tools for CE are necessary. Furthermore, we have summarised important elements of CE frameworks which facilitate successful CE programs.

Consumer Engagement Group

1. Sarrami-Foroushani P, Travaglia J, Debono D, et al. BMC Health Serv Res. 2014;14:250. 2. Anderst A, Conroy K, Fairbrother G, et al. Aust Health Rev. 2020;44(5):806-813. 3. Synnot AJ, Cherry CL, Summers MP, et al. Aust J Prim Health. 2018;24(3):197-203. 4. Rendalls S, Spigelman AD, Goodwin C, et al. International Journal of Health Governance. 2019;24(4):274-283. 5. Jacobs LM, Brindis CD, Hughes D, et al. J Healthc Qual. 2018;40(3):139146. 6. Esmail L, Moore E, Rein A. J Comp Eff Res. 2015;4(2):133-145. 7. Concannon TW, Fuster M, Saunders T, et al. J Gen Intern Med. 2014;29(12):1692-1701. 8. Tindana P, de Vries J, Campbell M, et al. BMC Med Ethics. 2015;16:24. 9. Warren NT, Gaudino JA, Jr., Likumahuwa-Ackman S, et al. Med Care. 2018;56 Suppl 10 Suppl 1:S58-S63. 10. Domecq JP, Prutsky G, Elraiyah T, et al. BMC Health Serv Res. 2014;14:89. 11. Oldfield BJ, Harrison MA, Genao I, et al. J Gen Intern Med. 2019;34(7):1292-1303. 12. National Partnership for Women and Families. Aligning Forces for Quality & National Partnership for Women and Families. Consumer Engagement Survey. 2014; http://forces4quality.org/node/3050.html. Accessed July 30, 2021. 13. Stocks SJ, Giles SJ, Cheraghi-Sohi S, et al. BMJ open. 2015;5(3):e006390. 14. Staniszewska S, Brett J, Simera I, et al. BMJ. 2017;358:j3453. 15. Miller CL, Mott K, Cousins M, et al. Health Res Policy Syst. 2017;15(1):9. 16. Brunton G, Thomas J, O’Mara-Eves A, et al. BMC Public Health. 2017;17(1):9 44.

Authors: Top Courtney Wallingford, PHD Student, Dermatology Research Centre and Bottom Dr Aideen McInerney-Leo, Senior Research Fellow – the University of Queensland

September 7 is World Duchenne Awareness Day. It is held on the 7th day of the 9th month because the DMD gene, encoding for the dystrophin protein, is the longest human gene known. It consists of 79 exons. Every year the World Duchenne Organisation announces a theme for the day and this year the theme is “Adult life & Duchenne”. Save Our Sons Duchenne Foundation was founded in 2008 and is the peak body for those living with Duchenne and Becker muscular dystrophy (around 1,000 young people) across Australia. Our vision is to find a cure for Duchenne and Becker muscular dystrophy whilst actively working to ensure enhanced quality of life for those young people and their families affected by these life altering conditions. Duchenne is an X-linked genetic condition that affects 1 in 3500 boys and in very rare cases 1 in 50 million girls. It is the most common and most severe form of muscular dystrophy and is also known as dystrophinopathy, which refers to a mutation in the dystrophin gene, with a similar milder form of the condition called Becker muscular dystrophy. Our quest has always been to empower oneself with information, support and community. Save Our Sons has worked to ensure no family or child is alone when faced with this devastating diagnosis. We continually

work on updating our programs to meet the needs of our community. Fundraising, advocacy work and community engagement are crucial to achieving our vision as we continually develop and evolve our programs, resources, and helpful service delivery pathways for those affected by the conditions and their families. Along with funding of research and clinical trials, Save Our Sons funds a critical nurses program in some of our major children’s hospitals across Australia with the aim of enhancing the quality of life of those affected with the conditions. Currently, we also deliver a telehealth nursing service, scholarship programs, and resources to help our families navigate the journey of a Duchenne or Becker diagnosis. We invite you to join us in raising awareness of Duchenne on the 7th of September and throughout the month. To find out more and for ways to get involved, visit www.saveoursons.org.au

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WORLD-FIRST RESEARCH REVOLUTIONISING HEART TRANSPLANT SURGERY Professor Peter Macdonald and his research team at the Cardiac Transplantation Laboratory at the Victor Chang Cardiac Research Institute have now made two incredible discoveries that will dramatically expand the available pool of heart donors, providing hope to patients on heart transplant waiting lists around the world.

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CHRISTOPHE KEREBEL

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rofessor Macdonald and the Institute team have already discovered how to successfully transplant a heart that has stopped beating. This was achieved by developing a preservation fluid in the Institute’s laboratory in combination with a machine that allows the heart to beat outside the body. Known as ‘heart in a box’ it extends the amount of time a donor transplant heart can spend in transit from four to 14 hours. This means donor hearts can be transported further and so be better matched with a recipient. It not only improves the outcomes for patients, it also dramatically expands the available pool of donor hearts. This has already saved hundreds of lives. The results of the world-first breakthrough were published in the Lancet in 2015 and focused on three patients who received a heart transplant with the new technique. Whilst their operations took place at St Vincent’s in Sydney, the hearts came from donors from other hospitals. All three recipients had normal cardiac function within just a week of transplantation, and all made a good recovery.

REPAIRING HEARTS WITH DEADLY SPIDER VENOM

Professor Macdonald is now working closely with the University of Queensland on the use of minute quantities of funnel web spider venom to further extend the life of donor hearts. In research published in Circulation in July 2021, the team identified a potential new drug developed from a molecule found in the venom of the Fraser Island (K’gari) funnel web spider that can not only extend the life of donor hearts used for organ transplants but also repair hearts damaged by a heart attack. The drug candidate works by stopping a ‘death signal’ sent from the heart in the wake of an attack.

The Hi1a protein from spider venom blocks acid-sensing ion channels in the heart, so the death message is blocked, cell death is reduced and heart cell survival is improved. This is the first time that ion channels have been identified as a target for treating the fallout after heart attacks, called ischemia-reperfusion injury (IRI). There are currently no drugs in clinical use that prevent the damage that IRI causes. Professor Peter Macdonald, who is also a senior cardiologist at St Vincent’s Hospital in Sydney, said this result had been decades in the making.

Professor Peter Macdonald

“This could not only help the hundreds of thousands of people who have a heart attack every year around the world, it could also increase the number and quality of donor hearts, which will give hope to those waiting on the transplant list,” Professor Macdonald said. “The survival of heart cells is vital in heart transplants — treating hearts with Hi1a and reducing cell death will increase how far the heart can be transported and improve the likelihood of a successful transplant. The protein has been tested in human heart cells as well as in pre-clinical models. It is hoped human clinical trials for both stroke and heart disease could begin within a couple of years. T his research was funded by T he Universit y of Queensland, the Australian National Health and Medical Research Council and the National Heart Foundation of Australia.

IMPACT

There are around 5,000 heart transplants performed per year around the world. At St Vincent’s in Sydney almost a third of transplants now use the heart in the box procedure. It is also now being used by surgeons in the US, the UK and Europe. If the new drug candidate’s potential is realised, Professor Macdonald expects to see a further rise in the number of transplants by around a third. Author: Julia Timms, Media & Communications Manager, Victor Chang Cardiac Research Institute

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SURVIVAL BUT NOT AT ANY COST After a devastating cancer diagnosis compounded by late-effects of treatment James and his family are supporting medical research in every way possible. “We don’t just support research, we take part in it.”

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The Bloor Family

CHRISTOPHE KEREBEL

My Twitter : @chriskere CHRISTOPHE KEREBEL

oni vividly remembers rushing her five-year-old son James to the emergency room on Good Friday in 2014. Despite giving him two different types of over-the-counter pain relief, the pain in little James’s head was causing him to scream in agony. For the previous two months, baffling symptoms had medical experts including his GP, an ENT specialist, his dentist and more unable to figure out what was causing the pain, the swollen glands and a lump on the side of his head. But scans that night in the hospital revealed the full extent of his terrible condition. “A tumour was below his brain and filling every other space and cavity in his head,” Toni says. “It was all through his nasal cavity, it was backing onto the top of his brain, it was pushing out the side of his head and going down his throat. It had even wrapped around the nerves that ran through his ear.” What the scans showed was there was simply no space left inside his head, in fact, James had only a millimetre of airway left in his throat. It was Burkitt’s lymphoma, a particularly aggressive form of lymphoma that accounts for up to 30 percent of childhood non-Hodgkin lymphomas. Without treatment in the next few hours, he would die. James began chemotherapy almost immediately. “The first shot of chemo broke the tumour up,” Toni says. “The doctors were blown away. We knew how effective it was because James had always snored loudly. But since that first night in hospital and after one shot of chemo, James has never snored. The treatment was amazing.” Chemotherapy continued for six months and by the end of treatment, the cancer was gone. The tumour, once so aggressively and painfully invading every available space in James’s head was now defeated. However, a new medical battle was only just beginning. During his treatment regimen, James was t wice administered anthracyclines and this has led to cardiomyopathy, a thickening of the heart muscle. The drug that helped save the boy’s life has caused debilitating heart disease. James, who is now 11, takes heart medications to manage this, what is known as a late-effect of cancer treatment. He has learned to rest when he needs to. He’s happy to ride a bicycle or jump on a trampoline, for instance, but only for ten minutes at a time. The experience has led James and his family to do all they can to support research into new drugs and therapies that cure, but not at any cost.

Read more: Understanding the genetic basis of chemotherapy-induced cardiomyopathy “For us, as a family, if something good can come out of what we’ve been through, if we can help other children and families to not have to go through these side-effects, it would be amazing,” Toni says.

In the trial we’re on at the moment, James has had some blood taken. They’re growing a baby heart to see if the gene for cardiomyopathy is in there, then they’ll remove the gene to see if the heart will heal itself. That will explain if this has anything to do with genetics or not,” Toni explains. “That’s one of the parts of what we’re doing at the moment. James also goes to the Baker Heart and Diabetes Institute and they do lots of exercise MRIs and different bits and pieces, with a few other children too, so they can get some good information on how the condition is affecting his heart and what they can do to help. So, we don’t just support research, we take part in it.” This is a four-year study, funded by The Kids’ Cancer Project in partnership with the RACP Foundation, looking at anthracycline-induced cardiotoxicity (ACT). ACT can result in life-threatening impairment of heart function, with 70 per cent of severely affected patients dying from the complication. Around the world, every year over 300,000 kids receive anthracyclines during their cancer treatments and in around 60,000 survivors, the major long-term side-effect is cardiac damage. Researchers Dr Rachel Conyers and Dr David Elliot have discovered that children who suffer this heart damage have genetic markers, potentially predisposing them to ACT. Read more: Behind the science | Dr Rachel Conyers “We want a world in which life-saving medicine isn’t life threatening,” Toni says. “We hope that one day a prevention or cure for heart damage from cancer treatment can be found so that other children can have better outcomes. We want James to beat this one too.”

Author: Jennie Smiedt - Head of Marketing & Communications | The Kids Cancer Project

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STRENGTHENING CONSUMER ENGAGEMENT IN RARE DISEASE RESEARCH

Innovative mechanisms to progress research co-design.

ngaging consumers in research is invaluable, even more so in rare disease. Rare Voices Australia (RVA) is seeking to move away from conventional, somewhat tokenistic, approaches to incorporating consumer expertise. Consumer engagement in research should extend beyond participation, and consumer groups should not solely be an endpoint to project delivery but a mechanism for research co-design. The consumer perspective

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and expertise can help to identify gaps and research priorities; it can inform research objectives, design, and the implementation of outcomes. Consumers also have important roles to play in informing the best way forward around governance of patient data. With the recognition that consumer engagement in research should traverse conventional boundaries, there is a new impetus for more strategic inclusion of consumer expertise in research. To that account, RVA has been reframing its program delivery, actively identifying, trialling, and sharing innovative and tangible ways to build the capacity of rare disease organisations to engage in all levels of research. Through training and mentorship, RVA is empowering rare disease support group leaders to contribute to research

CHRISTOPHE KEREBEL

in effective and meaningful ways, with the goal to build evidence for policy reform that improves people’s lives.

One of the difficulties that consumers face is how to engage in research meaningfully. Support group leaders can feel disempowered. In a survey of leaders from Australian rare disease organisations, one respondent shared: ‘Walking the walk is not our problem but being volunteers is perceived by some as incongruous with having skill, expertise and deep knowledge’6. One of our goals is to change this narrative by magnifying and strengthening the voices of advocates across the rare disease research continuum, from fundamental discovery or basic research to clinical translation. RVA’s Research Partnership Guidelines, developed in line with the Action Plan by RVA’s Scientific and Medical Advisory Committee (SMAC), highlight our research priorities and have informed aspects of RVA’s formal education program that relate to research. Through RVA’s Rare Disease Research in Australia threepart webinar series, experts shared information about the multitude of channels and tangible opportunities where rare disease advocates and organisations can contribute to the research continuum. This education is tailored to the needs of rare disease organisations across the areas of: • Eth i c a l re s e a rc h pa r ti c i pati o n a n d re s e a rc h governance. • Effective and strategic consumer engagement with researchers. • Developing an organisational strategy or guidelines for research partnerships. • Research for Health Technology Assessment (HTA) and advocacy purposes. • Consumer engagement in registries and the role of registries in advocacy. The knowledgebase for this component of RVA’s education program is born out of our ongoing experience building our credibility as active participants in rare disease research. Innovative ways that RVA has been or is planning to contribute to research co-design include: • Influencing government investment in research through consumer-centred systemic advocacy. For example, RVA motivated broader and ongoing investment in the Medical Research Future Fund Rare Cancers, Rare Diseases and Unmet Need Grant opportunity. • Commissioning research projects or white papers to build evidence and address gaps. • Commissioning/co-partnering (as a Chief or Associate Investigator) on projects with relevant academic researchers that advance implementation of the Action Plan. • Providing letters of support for grant applications on relevant projects. • Participating in peer review processes on relevant grant review panels.

My Twitter : @chriskere

CHRISTOPHE KEREBEL • Leading the collaborative development of the Rare Awareness Rare Education (R.A.R.E) Portal, a living website of rare disease information and resources for people living with a rare disease and their families and carers, researchers, clinicians and allied health professionals. • Participating as an independent patient advocate on a steering committee or patient advisory committee for clinical trials or other relevant projects. • Providing researchers with in-kind support around rare disease advocacy, policy and HTA processes. • Providing researchers with in-kind suppor t to convene focus groups or acting as a conduit to relevant organisations for meaningful consumer engagement. • Co-writing grant proposals. • Co-authoring academic research papers.

RVA seeks to inspire this level of research engagement at a systemic level through our education and mentorship programs. We are working hard to uncover and share more innovative ways to contribute to rare disease research, and we encourage rare disease researchers and advocates to do the same. RVA’s SMAC has provided invaluable guidance for this exponential growth in RVA’s level of research engagement. Having advice at arm’s length from researchers and clinicians is vital. RVA encourages all rare disease group leaders, researchers and clinicians alike to seek out mutually beneficial and relevant relationships with one another. These relationships build the networks we need for collaborative, person-centred research co-design to ensure the best outcomes for Australians living with a rare disease. 1. European Commission (n.d.). Rare Diseases. https://ec.europa.eu/ health/non_communicable_diseases/rare_diseases_en 2. Orphanet (2012). About Rare Diseases. https://www.orpha.net/ consor/cgi-bin/Education_AboutRareDiseases.php?lng=EN 3. United States Department of Health & Human Services (2019). FAQs About Rare Diseases. https://rarediseases.info.nih.gov/ diseases/pages/31/faqs-about-rare-diseases 4. EURORDIS Rare Diseases Europe (2019). What is a rare disease? https://rarediseases.info.nih.gov/diseases/pages/31/faqs-aboutrare-diseases 5. Australian Government Department of Health. (2020). National Strategic Action Plan for Rare Diseases. https://www.health.gov. au/resources/publications/national-strategic-action-plan-for-rarediseases 6. Pinto, D., Martin, D., & Chanall, R. (2016). The involvement of patient organisations in rare disease research: A missed methods study in Australia. Orphanet Journal of Rare Diseases, 11. https:// doi.org/10.1186/s13023-016-0382-6

Authors: Falak Helwani, Research and Evaluation Officer Rare Voices Australia and Nicole Millis, Chief Executive Officer, Rare Voices Australia

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Celebrating a selection of 2020-21 Award finalists

PROFESSOR DAVID HUNTER

Award: Health Services Research Award Sponsored by the NSW Government Primary Nominator: Prof Manuela Ferreira | University of Sydney Secondary Nominator: Prof Robyn Ward | University of Sydney BIO: Professor Hunter is a rheumatology clinician researcher whose main research focus is clinical and translational research in osteoarthritis (OA). He is the Florance and Cope Chair of Rheumatology and Professor of Medicine at University of Sydney and Royal North Shore Hospital. He holds a medical degree and Master of Sports Medicine from UNSW Sydney, a fellowship in Rheumatology at the Royal Australian College of Physicians and earned a Masters of Medical Science (Clinical Epidemiology) from the University of Newcastle and received his PhD from the University of Sydney in 2001. He is an editor for leading international journals in his field and leads a team dedicated to improving our understanding of osteoarthritis and the quality of life for those who suffer from this prevalent disabling disease. 28 INSPIRE 021 | 2021

PROJECT

In the last 10 years, Hunter has led 14 RCTs and been an investigator on a further 6, which have provided high quality new knowledge supporting non pharmacologic (e.g. bracing, weight loss) and pharmacologic interventions (e.g. stem cells, long acting steroid injections). His research has challenged paradigms in the management of osteoarthritis and shifted clinical practice from a fragmented model based on the use of high cost, high risk and low value treatment approaches to an evidence-based, patient-driven care model. The Osteoarthritis Chronic Care Program he developed promotes coordinated, high quality care for hip and knee osteoarthritis. Due to the health and economic impact of this program, the State government mandated the program be run at every NSW local health district and to date it has reached more than 20,000 people with OA. Internationally, David leads the OA management consortium titled “Joint effort” that comprises more than ten international OA chronic disease management programs.

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My Twitter : @chriskere CHRISTOPHE KEREBEL

PROFESSOR JULIE BYLES

Award: Health Services Research Award Sponsored by the NSW Government Primary Nominator: Professor Kichu Nair | University of Newcastle Secondary Nominator: Professor Brian Kelly | HCF Research Foundation BIO: Professor Byles is Global Innovations Chair in Responsive Transitions in Health and Ageing at the University of Newcastle, co-Director of the Centre for Women’s Health Research, and co-lead of the Public Health program of the Hunter Medical Research Institute. She is also a founding investigator and Director of the Australian Longitudinal Study on Women’s Health. Professor Byles’ research interests focus on the role of health services, preventive activities and treatments, and aged care services in maintaining quality of life for older people. Professor Byles is also Head of the International Longevity Centre – Australia, a Fellow and Life Member of the Australian Association of Gerontology and Chair of the International Association of Gerontology (Asia Oceania) Social Research and Planning sub-committee.

PROJECT

Professor Byles researches health services, preventive activities, and treatments for maintaining quality of life for older people, and physical, psychological and social factors associated with optimal physical and mental health of men and women as they age. She led the development and evaluation of health assessments for older people, and provided assessment tools that have been adopted nationally and internationally. Her work led to the establishment of the Medicare item for health assessments, and informed the assessment framework. She has continued to evaluate the uptake and impact of the assessments through the Australian Longitudinal Study on Women’s health (ALSWH). Analysis of linked survey, Medicare, hospital and aged care data show the widespread use of assessments and their potential effects on overall quality of health care, reduction in admissions to residential care, and improvements in physical function and mortality. Complementing this work, and working with scholars under her supervision, she has also developed and evaluated methods for falls safety assessment, medication review, urinary incontinence, prevention of falls in residential care, and improvement of nutrition in aged care. 2021 | INSPIRE 021 29

NATIONAL BREAST CANCER FOUNDATION

PROJECT

Award: Advocacy Award Primary Nominator: Professor David Currow | Cancer Institute of NSW Secondary Nominator: Professor Roger Reddel | MCRI

NBCF is pioneering an ongoing series of collaborative funding opportunities that aim to leverage synergies, opportunities and research expertise across cancer types. NBCF has now delivered three linkage grants by partnering with other cancer research charities. These grants have enabled innovative large-scale research collaborations that would otherwise have struggled to secure funding in more traditional grant schemes.

BIO: The National Breast Cancer Foundation (NBCF) is the largest national funding body for breast cancer research in Australia. We’ve invested $181 Million in 557 research projects since 1994, supporting over 745 individual Australian breast cancer researchers. This investment has been made possible with money raised entirely by the Australian public as they receive no government funding.

In 2017 NBCF launched the Endowed Chairs program, another Australian-first. This program offers 10-year research grants for the brightest stars in breast cancer research, designed to keep emerging leaders based in Australia and focused on innovative research that could lead to the next major breakthrough by providing stable, long term support for their salaries and research programs.

NBCF works collaboratively with its researchers, ambassadors, volunteers and donors to raise awareness about the crucial impact NBCF-funded research has on breast cancer. Since NBCF’s inception in 1994, the fiveyear survival rate for breast cancer has increased from 76% to 91%. That’s over 47,000 lives that have been saved thanks to research. 30 INSPIRE 021 | 2021

NBCF has established a world-class research impact tracking system for funded projects, becoming the first Australian organisation to use ResearchFish. NBCF has now accumulated a major database of research impact from 2012 through to the present day, covering publications, patents, further funding, changes to policy and practice, new collaborations and more.

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HOW BIONEER TRANSITIONED TO DELTEK ERP IN THE CLOUD – AND THREE LESSONS THEY LEARNED ON THE WAY In this article, Deltek interviewed leading biotech consultancy firm, Bioneer, to look at how their company reduced manual data entry, improved forecasting and reporting, and accelerated business insights with Deltek’s cloud-based Enterprise Resource Planning (ERP) system. Plus, three tips for successful ERP implementation from Bioneer’s CEO, Lars Pedersen.

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CHRISTOPHE KEREBEL

My Twitter : @chriskere CHRISTOPHE KEREBEL

MANAGING COMPLEX ASSIGNMENTS OF EVERY SHAPE AND SIZE

With a contract research unit and a manufacturing unit, Bioneer is well-positioned to develop pharmaceutical drug candidates, produce proteins, and test such candidates in advanced human cell models. Today, it provides research, development and consultancy services to nearly 100 companies across Europe and US and Asia. Bioneer works on assignments of all shapes and sizes – and in an industry with set ways of doing things, managing diverse project types and sizes is a complex task. It was even more challenging with a limited ERP system that left teams reliant on manual data entry. “We’re selling laboratory-based services, which requires registering every hour worked, and we need to generate intuitive reports each month for Bioneer management and our board of directors,” says Lars Pedersen, Bioneer’s CEO. “And as our projects are often split across compartmentalised business units, we need detailed insights into forecasted timelines and financials to keep projects on track.” “With our old ERP, we needed to run many operations in Excel,” says Lars. “There’s always a risk of error when you take something out of a system and process it manually. Even the smallest mistakes can have a big impact on reporting and forecasting, especially as we were updating balance sheets manually. It also makes it a lot harder to extract meaningful insights from our data.”

THE END OF AN ERA – AND A NEW OPPORTUNITY

Bioneer saw an opportunity to solve these challenges, when its ERP licence ended. The need to adopt a new cloud-based ERP solution gave Bioneer a chance to digitalise more of its project and resource management processes – and accelerate business agility with a solution specifically designed for project-based businesses. “After having examined many different ERP solutions, we knew we wanted a system that had the necessary capabilities to let us move quickly – and didn’t require any extra module programming. A person from an organisation with the same parent company as us had worked with Deltek in the past, and suggested Deltek’s ERP solution.” With a new cloud-based ERP, Bioneer gets all the benefits of modern reporting and data analysis. Since implementing the solution, the burden of manual data handling has been reduced significantly, with Excel usage still dropping. “It’s already made life at Bioneer a lot easier,” says Lars. “Crucially, we can plan ahead better – and get faster, more accurate reporting. Within a few minutes, I can get an

overview of our monthly financials and forecasts. Invoices can be generated straight from the system, reducing the amount of paper processes. And we can extract more meaningful insights from our data, which is great for our clients, as it makes it much easier to communicate a project progress.” Bioneer are already looking at how they realise further benefits – including digitalised client collaboration, people management planning, and, later down the line, AI-driven data extraction.

THREE TIPS TO SUCCESSFUL ERP IMPLEMENTATION

We asked Lars about the challenges Bioneer faced on its road to cloud ERP. He offers three pieces of advice for other organisations in a similar position: 1. GOOD THINGS TAKE TIME “While we were looking at ERP solutions, a CFO told me: ‘You’ll hate the new system, and love the old one’. People get comfortable with what they know – so converting from a well-known system to a new is overwhelming. The transition had to happen fast and we were fully operational ERP- wise after 10 to 12 months” 2. GET EXPERTS INVOLVED FROM THE OFF “Hiring experts, especially bookkeepers who were wellversed in using Deltek ERP, was a big part of our change management strategy. For my accountants, who had been using Axapta system, getting a feel for the new system took some resources, as they also had to do annual reporting in the beginning using the old system. So, I hired experts to champion the system – and it made the whole process a lot smoother. I’ve even kept one of them on.” 3. DON’T JUST STOP WITH YOUR PEOPLE “Everyone who interacts with the new solution will benefit from being educated on it. We had to teach our external accountants and auditing firm, who wanted to know how we had implemented the solution and how we’re using it to manage financial processes. With a little effort on our part, and from Deltek consultants, they understand the system and transaction processes better, and can do their due diligence – which helps us a lot.”

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HOW ONE MOTHER USED HER VOICE TO OPEN THE DOOR FOR THOUSANDS

Thirty years ago, Anne McKenzie had to push her way into meetings to get clinicians and researchers to even listen to health consumers, let alone involve them in decisions. Today, largely thanks to her tireless efforts, consumer involvement is second nature to thousands of researchers across Australia – and once closed doors swing open before her.

hen I first dipped my toes into the waters of consumer advocacy in the early 1990s, I didn’t even know what that phrase meant. In my eyes, I was just Anne from Morley in Perth, Western Australia, doing my job as a mum in trying to get the best possible care for my youngest daughter, who had been born with spina bifida. I soon learnt that if I wanted to have a say and get answers from clinicians and an unwieldy health system, I would have to be persistent. If that meant being seen as pushy, then so be it. In 1994 I established the Parent Advocate role at Princess Margaret Hospital and became involved with the WA Health Consumers Council – roles which advocated for the engagement of families in decisions around service delivery and the utilisation of complaint data to improve 34 INSPIRE 021 | 2021

Anne McKenzie AM Telethon Kids Institute

these services. It was during this time that I first became aware of how research informed healthcare. In 2004 I was offered a wonderful opportunity to work for the Telethon Kids Institute and The University of Western Australia’s School of Population and Global Health as the Consumer Advocate – a first in an Australian university setting. At that point there was still considerable resistance within the research sector to the idea of consumer involvement, but senior champions like Telethon Kids Institute Founding Director and 2003 Australian of the Year Professor Fiona Stanley, UWA Emeritus Professor D’Arcy Holman, and Telethon Kids Founding Researcher Carol Bower supported me every step of the way. Ignoring sometimes pointed criticism from yet-to-beconverted colleagues, they provided dedicated funding for

CHRISTOPHE KEREBEL

CHRISTOPHE KEREBEL My Twitter positions, resources and training, ensured organisational Their work has been recognised internationally as: a@chriskere best strategic plans included a stated commitment to consumer practice model forCHRISTOPHE effectivelyKEREBEL and rapidly engaging the and community involvement, and constantly reinforced our community in research during a pandemic and will be guiding refrain: that consumers add value to research. used as a case study in the NHMRC’s Ethical Guidelines for future pandemics. I am immensely proud of this group’s Their faith, for which I remain eternally grateful, paid off. The work – they personify the community’s genuine interest in consumer and community involvement program I established having input into the important research happening across in this role is now delivered across WA and is recognised the globe. nationally and internationally as the gold standard for consumer involvement in research. The program’s success Consumer and community has since influenced many other research organisations engagement is now second nature across the country to undertake activities to increase across the organisation at Telethon Kids, consumer and community involvement.

One outstanding example of consumer involvement in practice under this program was the collaborative development by consumers and Telethon Kids Fetal Alcohol Spectrum Disorder (FASD) researchers of resources to raise FASD awareness among health professionals and improve identification and care for patients with FASD. In addition, we facilitated community events to discuss information available on alcohol consumption during pregnancy, established national community reference groups, and undertook a project to identify the top ten priorities for future research from both community members and service providers. The more I worked to convince researchers of the value of consumer involvement – not always an easy task – the more I realised the need for formal training. In 2007 I developed and nervously delivered my first training workshop. Since then, my colleagues and I have delivered 154 workshops to more than 3,000 attendees across Australia. I no longer have to twist arms to get people to attend – more and more, these valuable sessions are in high demand as researchers increasingly understand that consumer involvement simply has to be a fundamental pillar of their work if they want to design, undertake and translate sensitive, efficient and intelligently targeted research into real-world outcomes that genuinely make a difference to people’s lives. When COVID-19 disrupted our ability to deliver face-toface-workshops, we successfully transitioned to online and virtual workshops. At the same time, we cemented plans to ensure the training program’s long-term sustainability – working with the University of Queensland Australasian Cerebral Palsy Clinical Trials Network’s Centre of Research Excellence to develop a team of 11 trainers to deliver our workshops into the future. In April 2020 – as research teams mobilised in response to the pandemic – we quickly established a national Community Advisory Group to provide input into rapidly emerging research projects. This group’s 22 senior consumer advocates, drawn from across Australia, have so far contributed to 22 local, national and international research projects, conferences and webinars, as well as the development of Federal Government priorities for future pandemic-related research.

with our researchers able to draw on more than 450 consumers and community members across dozens of reference groups and committees, including a Youth Advisory Group.

In 2020, as Manager, Consumer Engagement at the Institute, I launched Telethon Kids CONNECT – an Australia-first program to recognise and reward community members’ contributions to research. I am excited to see how the program will expand and develop new initiatives and directions. In 2015 I was appointed to the Order of Australia for ‘Significant service to community health through consumer advocacy roles and strategic policy research and development’; and this year I was honoured to receive the National Health and Medical Research Council’s Biennial Consumer Engagement Award. It was extremely humbling to be recognised in these ways – but also deeply encouraging to reflect on how far consumer involvement has come since those first nervous days when I struggled to find my own voice when fighting for my daughter’s needs. More and more hospitals have advisory councils or committees, there are consumers in health departments, and researchers across the country are actively seeking to form community reference groups and genuinely engage with health consumers on the ‘what, why and how’ of research. It’s no longer about pushing to be heard, but about being effective and contributing meaningfully to research and, ultimately, to clinical services. I feel as passionate about consumer involvement in healthcare and research today as I did when I first started and am excited about the fast-growing interest from all sectors across the country to increase opportunities for the community to have a ‘voice’ in research. Author: Anne McKenzie AM is a nationally and internationally recognised health consumer advocate. Until recently she was Manager of Consumer Engagement at the Telethon Kids Institute. She has been a member of numerous influential state and national groups and committees. 2021 | INSPIRE 021 35

PARTICIPATI The active participation of consumers in health systems and processes has increasingly been recognised both as a right, and as s good practice in healthcare in Australia (NHMRC 2018) and internationally.

his applies across the continuum of healthcare, from the co-design of health ser vices, to consumer feedback on existing services and service-gaps, and the imperative for a strong lived experience voice in health evaluation and research. For people who are struggling to survive day to day on the street, and who often have had past experiences of ‘not feeling listened to’ or worse, rejected by the health system, different approaches to consumer involvement are needed.

KEY FINDINGS Benefits of lived experience participation

The Home2Health Team at the University of Western Australia (UWA) has for the last 3 years, been trialling and learning ‘what works’ to ensure that the voice of people experiencing homelessness is heard in our UWA research, and in our evidence-led advocacy. Conventional mechanisms such as consumer reference groups can be challenging for this cohort, and it is imperative that people with a lived experience of homelessness have choice and agency in the ways that they might be involved.

Challenges

This article shares some findings from a recent project that explored the benefits and challenges associated with the participation of people with lived experience of homelessness in the design, development, or delivery of services. Data for this study was collected through twelve faceto-face interviews – with people with lived experience of homelessness (n=8), as well as staff (n=4) who had facilitated participation in their organisation by people with experience of homelessness.

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The value that participation in and of itself had on the lives of participants with lived experience was a strong theme. Importantly, this value was not seen to be contingent on whether participation yielded benefits to the organisation or its practice. Key benefits of involvement included feelings of inclusion, acceptance and respect for engagement in lived experience work. Power and power differentials People with experience of homelessness frequently discussed facing stigma, discrimination, and exclusion, when accessing homelessness and health services, as well as when participating in lived experience work. Organisations that are facilitating lived experience work need to have an intentional understanding of and response to the role of power differentials in their work and to reflect on power in roles and relationships where it might not currently be considered to be an issue. People with lived experience of homelessness need to be included at all levels of decision-making in the design and delivery of services and policy, in order to have the power to effect change on the services, systems, and processes that are relevant to their lives. Meaningful participation The question of whether people with lived experience were engaged to share their expertise or simply to tell their

ION FIRST

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CHRISTOPHE KEREBEL

stories about homelessness was discussed throughout the interviews undertaken. A number of participants advocated for lived experience work having more meaningful engagement with participants’ range of knowledge, experience, and skills, beyond people with lived experience just retelling their stories – an experience described by some participants as reliving and retelling a horror story. Ensuring that this participation is not tokenistic is critical. Trauma informed imperative While this research did not set out to overtly explore issues of trauma, this emerged as a recurrent theme. Experiences of childhood and adult trauma are pervasive among people experiencing homelessness, compounded by the trauma of homelessness itself. Whilst participation in homelessness services or research can be rewarding and empowering, it can also be retraumatising (for example where people find themselves retelling their own stories without adequate support). Experiences of personal trauma and of vicarious trauma were described by lived experience participants as having a powerful impact on their participation in lived experience work. The effects of repeatedly reliving trauma resulted in some participants questioning their capacity to continue engaging in lived experience work.

I do worry about that. If I continue this work, then that would be just kind of reliving my trauma every day. I think … you either move on and you just distance yourself from it or you stay with it and you try and make it better for everyone else. But then you’re stuck in that cycle of every day being like “When I was homeless, this happened…” (Person with lived experience) However, experiences of trauma were also identified as a powerful motivator for people with lived experience wanting to participate in designing and delivering services and policy, in order to better support people who were still experiencing homelessness.

CONCLUSION

There is considerable scope for people with lived experience of homelessness to transition from being recipients of services, to being active participants in the design and delivery of services. Factors that are central to this transition are that people with lived experience of homelessness are supported to participate safely and ethically, and that decision-making and power is genuinely shared.

Authors: Shannen Vallesi Donna Quinn, & Lisa Wood, University of Western Australia

2021 | INSPIRE 021 37

A JOURNEY OF MUTUAL DISCOVERY The newly formed Edith Cowan University Strategic Research Centre for Precision Health (CPH) recognises that central to their activities in genomics, and other -omics, is the engagement and involvement of people affected by the conditions it researches.

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CHRISTOPHE KEREBEL

My Twitter : @chriskere CHRISTOPHE KEREBEL

recision Health is an emerging and evolving field whereby increasing biological information is used to shape prevention, prediction, diagnosis and treatment at the population, patient group and individual level. Since the 2002 Statement on consumer involvement by the NHMRC, the impor tance of consumer and community involvement in research has become widely publicised and adopted by charitable and government research funders. Much has been said about the benefit of community engagement to research, from increased awareness and confidence, to more effective ways of using research to inform policy and practice. But it is in the actual conversations between researchers and consumers where we experience powerful growth.

As researchers, we just look at test tubes and clinical data - but interacting with people makes us realise there is someone’s child, parent or grandparent at the end of all our work. And these people are telling us: “Don’t give up; keep going.” This power ful motivation engages the team to be more productive and find real-world applications our discoveries.

MELANOMA RESEARCH GROUP

ECU’s Melanoma Research Group (which is now part of CPH) has been working for more than a decade with consumer representatives. Some of them have significant experience in dealing with researchers and institutions, others have a living experience of melanoma and want to share their stories and those of others like them. Their concerns and queries guide the direction our research pursues. Since the formation of our group by Professor Mel Ziman, engagement by those affected by melanoma brought to the forefront of our activities the development of blood test that can aid the diagnosis, prognostication and monitoring of melanoma. From this pursuit emerged the MelDx® test for early detection of melanoma now undergoing clinical testing. Since then, many life-saving therapies have entered the clinic, significantly improving the outcome of melanoma patients. In this new context, consumers have highlighted the need to determine what treatment is the best fit for a patient. Other concerns included, the need for regular monitoring in case of the disease returning, exposure to scans, and the likelihood of harmful side effects from treatments, especially from immunotherapies.

‘As scientists trying to communicate scientific terms in plain language, we were surprised how much those affected by melanoma and their families know about genes and mutations.’

The idea of a blood test is very appealing for consumers and many advocates for their speedy translation into clinical practice. Guided by these discussions, we have pursued developing and testing different liquid biopsies modalities for melanoma. ‘Liquid Biopsy is a test done on a sample of blood to look for cancer cells from a tumour that are circulating in the blood or for pieces of DNA from tumour cells that are in the blood.’ Our research has shown that liquid biopsies can predict which patients are more likely to respond to therapy, show early evidence of response and disease progression, providing complementary information to imaging. In addition, liquid biopsy can provide an alternative for long term monitoring of patients after therapy cessation and patients in rural areas where access to imaging facilities is challenging.

COMMUNITY SUPPORT GROUP

Engagement with melanomaWA (part of Melanoma Institute Australia), a community support group for people living with melanoma and their carers in Western Australia, has provided fur ther oppor tunities for community engagement. Community forums with their support groups have guided on the concerns of patients, attitudes towards research and engagement in research and clinical trials. We have participated with them in melanoma awareness campaigns, lab visits and fundraising activities. Above all, we have formed strong bridges on mutual understanding. Research priorities should be led by those affected by the research and those whose data is being used. This is the only way to build trust with patients and the public.

Author: Associate Professor Elin S. Gray, PhD. Deputy Director and Cancer Stream Lead, Centre for Precision Health. Lead, Melanoma Research Group, Course Coordinator, Masters by Research Course (J90), Chair, Radiation, Biosafety and Hazardous Substances Committee (RBHSC), School of Medical and Health Sciences, Edith Cowan University

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COLLABORATING WITH PEOPLE AROUND THE GLOBE IN GLYCOME RESEARCH Human IgG Glycome and Its Global Variability

mmunoglobulin G (IgG) is the most abundant serum antibody with which structural characteristics and effector functions are modulated through the attachment of various sugar moieties called glycans. Composition of the IgG N-glycome changes with the age of an individual and in different diseases. As a result, peoples’ profiles of IgG-N Glycan can be used as a tool for screening for chronic disease and its relationship to lifestyle and environmental factors. For example, it can be used to monitor the progression of metabolic syndrome towards hypertension, type 2 diabetes mellitus and cardiovascular diseases. Variability of IgG glycosylation within a population is well studied and is known to be affected by both genetic and environmental factors. However, global inter-population differences in IgG glycosylation have never been properly addressed before now.

A GLOBAL STUDY

Forty-two scientists from 24 research institutions in 13 countries made this global effort to analyse the total IgG glycans and subclass specific Fc glycopeptides from 27 ethnic groups worldwide. 40 INSPIRE 021 | 2021

The study presented ethnic group-specific N-glycosylation patterns of IgG, firstly analysed in five different populations of China, Croatia, Estonia and two cohorts from the United Kingdom (population of Scotland from Orkney Islands and England from the TwinsUK cohort), totalling 10,482 IgG glycomes. The study then further investigated the IgG’s fragment crystallizable region (Fc) by analysing 2,579 individuals from 27 populations globally. The samples were primarily collected specifically for the studies of IgG N glycome diversity and its association with diseases susceptibility. This study was conducted following the Declaration of Helsinki and was approved by the respective local Ethics Committees with the consent of participants. Countr y of residence was associated with many N-glycan features and the strongest association was with monogalactosylation where it explained 38% of variability. IgG monogalactosylation strongly correlated with the development level of a country, defined by United Nations (UN) health and socioeconomic development indicators, and with the expected lifespan. Par ticipants from developing countries had low levels of IgG galactosylation, characteristic for inflammation and biological ageing.

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Professor Wei Wang, MD, PhD, FFPH, FRSB, FRSM

This international collaborative study reached two conclusions: 1. significant changes in IgG glycosylation were associated with age, sex and country of residence, where age and country of residence were able to explain a significant portion of glycosylation variability; and 2. IgG glycans also strongly correlated with the development level of a countr y and with specific development indicators as well. This study for the first time suggests that citizens of developing countries may be exposed to environmental factors that can cause low-grade chronic inflammation and the apparent increase in biological age.

STUDIED POPULATIONS:

Tatars and Yakuts; Orkney and Shetland islanders, Croatian, German, Kosovo, Italy, Russian, Sweden; Slavs; Turkish 1. Štambuk J et al: Global variability of the human IgG glycome. Aging, (2020) 12(15), 1-13. 2. Y u X et al. Profiling IgG N-glycans as potential biomarker of chronological and biological ages A community-based study in a Han Chinese population. Medicine (2016) 95:28(e4112) 3. Yu, X., Wang, W. A rapidly aging world in the 21st Century: Hopes from glycomics and unraveling the biomarkers of aging with the sugar code. OMICS: A Journal of Integrative Biology, (2021). 25(4), 7p. 4. Zhang X et al. Association of dementia with immunoglobulin G N-glycans in a Chinese Han Population. npj Aging and Mechanisms of Disease (2021) 7:3

AFRICA: Uganda

AMERICA: Latin American: Jamaican; Native American: Trinidad and Tobago ASIA: Han Chinese Han, Evenks, Kazak, Kyrgyz and Uyghur; Indian; Papua New Guinean; Thai EUROPE: Bayash (Vlax) and Balkan Roma; Kazakhs,

Author: Professor Wei Wang is a Professor in Global Health, Centre for Precision Health at Edith Cowan University, Australia. His current research interests lie in Suboptimal Health, Genomics and Glycomics.

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COLLABORATING WITH PARENTS OF CHILDREN WITH LONGTERM CONDITIONS

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Long-term conditions among children are increasing. Parents of children with long-term conditions are at risk of experiencing higher degrees of parental stress due to the substantial social, emotional, and personal demands associated with caregiving.

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Professor Evalotte Mörelius

ur research involved interviewing parents caring for children with long-term conditions on their lived experience. Our aims included identifying factors that may increase or decrease stress for families in Western Australia. We first met Mrs Lauren Jones at a Consumer Advisory Meeting when we presented our idea for this study. We wanted to find out what parents caring for children with long-term conditions thought about our research proposal. Lauren put her hand up to collaborate with us on the study and she has been involved ever since throughout the whole research process. Lauren provided her views on the research study including the recruitment strategy and collaborated on the ethics application and reviewed the study documentation including the parent information sheet, consent form, and the interview questions. Lauren’s involvement ensured that the wording on the study documentation was suitable and relevant interview questions were asked. Lauren was also an investigator on the Perth Children’s Hospital Foundation Seeding Grant which we were successful in receiving support for the study and was involved in reviewing the application and writing the lay summary. Lauren’s contribution provided essential, valuable, and meaningful expertise from the parent’s viewpoint. We are privileged to have Lauren as a co-author on the research manuscript which we are shortly submitting for publication, for her extensive contribution including her input on the findings of the research, and the recommendations. Mrs Lauren Jones’ experience: “It has been a privilege to be involved in the ‘Parental Stress’ study as a health consumer. As a parent of two children with chronic health conditions, and previously a facilitator of a community group supporting families caring for children with medical complexities, it has been a pleasure to be asked for my input on this important topic.

Dr Stephanie Smith

The process has been rewarding. From preparing for the ethics application, up until reporting and dissemination of the findings, I have felt valued and included. I believe that by involving consumers in research, this ensures the reliability and validity in the research and makes the process collaborative and transparent.

I am truly honoured to have been invited to become co-author of the final paper and believe that it reflects the experiences of parents caring appropriately. Thanks again for the opportunity.” Including consumer engagement is paramount in research. Our experience highlights that important research priorities for families are addressed and further research enquiries meet consumer needs. The collaboration ensures that the translation of our findings into practice remains targeted.

Authors: Dr Stephanie Smith is a Postdoctoral Research Fellow (Children and Young People) at Edith Cowan University and Perth Children’s Hospital. Stephanie is a Health Psychologist with research interests in consumer engagement, experiences of health and illness, and the evaluation of health services. Professor Evalotte Mörelius is Professor of Nursing (Children and Young People) at Edith Cowan University and Perth Children’s Hospital. Evalotte’s specialties are in Paediatric and Neonatal Nursing, stress within the family, and parent-child interaction.

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COMMUNITY AND RESEARCHERS UNITE FOR CEREBRAL PALSY RESEARCH

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In 2005 the Cerebral Palsy Alliance established a Research Foundation and a Research Institute to support and conduct Australian and international research into the prevention of CP, potential cures, and best practice interventions to improve quality of life for people with CP.

P Quest is a collaborative initiative instigated in 2015 by parents of children with CP and researchers from the Research Institute. It works as a mechanism to involve families and people with CP (Research Partners), in research activities so that lived experience can be incorporated in all research activities to ensure that 1) valid high-quality research is conducted and 2) ideas from the CP community are integrated into current and future research, and 3) the results are disseminated back to the community in easy to understand formats and in a timely manner. One of our first activities was to identify people with CP and their families’ research priorities. As a CP Quest member, Natasha Garrity was part of the team that led the project. Research Partners (n=30) were asked to identify their top three research priorities for CP research. Natasha’s lived experience was invaluable when categorising the responses, resulting in a more sensitive interpretation of the data which also improved the dissemination of the results. These finding and priorities were disseminated to families, clinicians and researchers at international conferences and are used to guide the Research Institute’s projects, and the Research Foundation’s agenda for its grant program. CP Quest has provided training for its community members and researchers via workshops facilitated by Anne McKenzie (Telethon Kids Institute). Training is a priority for this group and will ensure that Research Partners have confidence that their lived experience is an important and equal contribution to the research team. Two CP Quest members are now participating in a Train the Trainer program through Telethon Kids Institute and the Australasian Cerebral Palsy Clinical Trials Network, which will enable us to offer regular training opportunities 2021 | INSPIRE 021 45

Members of the Original CP Quest committee, developing processes and Terms of Reference. (Gino Mammoliti, Kerry West, Margaret Wallen, Isabelle Balde, Maria Jerez, Shannon Clough, Hayley Smithers-Sheedy, Lucy Jacka, Sophie Marmont, Alex Goodwin, Rob Lowe).

for both researchers and community members. CP Quest is also working to develop written training materials to offer greater flexibility for training. Shannon Clough, our Community Chair of CP Quest was involved in the Australian and NZ strategy steering committee from the day it started, as a consumer representative having lived experience of caring for her son Ethan. Shannon contributed to the vision, goals and objectives that are now captured in the CP Strategy. Her passion to see even more research into CP, and helping people living with CP getting the support they need to live a fulfilling life, has led her to develop CP Quest and be involved in many projects and initiatives. Research Partners participate in reference and advisory groups to provide expert advice, be informed of research and assist with implementation and policy initiatives. An example is the Stem Cell reference group. During these meetings, the nature of different stem cells and potential treatments are explained and discussed and updates are given on the status of any ongoing trials or projects. The reference group allows the members to learn about the research process while the researchers consider the opinions and values of the CP community on ethical issues and sometimes controversial treatments. The information collected from these meetings is being used to shape research and policies regarding future stem cell treatments and research. Research Partners have become co-investigators and coauthors on projects, contributing to the research process from design to dissemination. These projects range from surveys concerning out of pocket costs of CP and emotion regulation in adults through to developing studies of genomic testing for families with multiple children with neurological conditions, new communication technologies for children, and virtual group meditation interventions for adults. 46 INSPIRE 021 | 2021

The main impediment for people with CP and their family members to both volunteer as Research Partners and to access training has been lack of time due to multiple commitments including work, study, medical/therapy appointments and caring responsibilities. This has highlighted the need for more Research Partners to share the load (we now have 80+ members) and to provide as much flexibility as possible.

We are committed to paying people for their time and expertise, and are encouraging researchers to build this into their budgets when applying for grants. We have a small budget to pay for people’s time or expenses if researchers are yet to receive their grants. It is important for researchers to involve people with CP in research as it ensures relevance to the community and can improve translation and implementation of results. People with CP and their families make significant contributions to CP research, are eager to be involved in research, but need formal support and flexible arrangements to facilitate their involvement. Authors: Natasha Garrity CP Quest Research Partner who has CP. She is also a Science student at Macquarie University. Shannon Clough, Community Chair CP Quest CPA Research Institute, and mum to Ethan and Hayley. Isabelle Balde, Project Co-ordinator CP Quest, CPA Research Institute Sarah McIntyre: Senior Research Fellow and Research Chair CP Quest, CPA Research Institute

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RESEARCH AUSTRALIA AWARD SPONSOR FEATURE

GSK Australia takes a look back at 40 years of the GSK Award for Research Excellence (ARE) and where some of its notable winners are now.

The Australian professor helping end malaria with his award-winning discovery

Professor Brendan Crabb AC won the 2019 GSK Award for Research Excellence (ARE) for his discovery of a ‘weak spot’ in the parasite that causes malaria. The work of this Australian team has kicked started a major research direction ultimately aimed at developing desperately-needed novel therapies.

rofessor Brendan Crabb is an infectious disease expert, and together with his research group at the Burnet Institute, has made significant discoveries into the DNA of malaria parasites. The $80,000 grant awarded by GSK to Prof Crabb has enabled him and his team to further their research into malaria treatments over the past two years. Their work has transformed how scientists explore malaria prevention and treatments globally. It is now helping identify vaccine and drug targets for the disease. “My team discovered something we refer to as the ‘export machine’. It’s an Achilles’ heel in the parasite, as so many different processes rely on this machine,” Prof Crabb says. Targeting previously unexplored pathways of parasites is Prof Crabb’s special area of interest. “I am striving to understand what is it about the parasite that makes it infectious to people,” says Prof Crabb. “The ‘export machine’ is a Holy Grail, if you will, for exploring this. We can look to make drugs that interfere with many different functions crucial to the parasite that haven’t been targeted before.” Malaria has long been one of the world’s deadliest infectious diseases, and it remains one of the worst in the world. Each year, more than 200 million people are infected with malaria. In 2019, the disease claimed 409,000 lives according to the World Health Organisation (WHO). Children are the highest risk group, with two thirds of these deaths are in kids aged under five years. “Malaria is the world’s first great pandemic having jumped into humans about 50,000 years ago,” says Prof Crabb. “Death rates have almost halved over the past decade, but the numbers are still shocking. A staggering 48 INSPIRE 021 | 2021

1,200 kids die from malaria every day. And COVID-19 is a major threat to malaria control efforts.” Prof Crabb became interested in the world’s vulnerable communities healthcare, having lived in Papua New Guinea as a child. He is currently the CEO at the Burnet Institute, an Australian not-for-profit research institute whose work aims to improve the health of underprivileged people. According to Prof Crabb, social and socioeconomic status can have a big impact on the spread of infectious disease. “As seen with COVID, people from lower socioeconomic backgrounds make infection prevention and control a huge challenge, as, for example, their living conditions require them to go to work, regardless of their health, and live in crammed spaces due to unaffordability of rent. People who are poor are the most at-risk of infectious diseases and their consequences, it’s an awful double whammy. Focusing infection control on those most in need is not just a moral imperative, it is also the best thing to do for society as a whole.” The Burnet Institute’s work emphasises how early treatment reduces deaths, and disease transmission. Prof Crabb’s research could help reduce the annual death toll even further, which is already on a downward trajectory. “Currently there are lots of drugs under development, but these collectively target only a small number of processes in the malaria parasite,” says Prof Crabb. According to the WHO, resistance to antimalarial drugs is an ongoing problem. “Malaria parasites become resistant to some drugs, rendering these medicines less effective,” says Prof Crabb. “During the past year, we have been testing

Journey through time: GSK ARE winners 2019 Winner: Professor Brendan Crabb AC, Director and CEO of Burnet Institute

GSK Australia is proud of over 40 years of the award and support for home-grown research that helps to improve outcomes for patients globally.

2008

existing compounds for their capacity to block new and essential parasite pathways, and now we have some very strong prospects to progress with.” he says.

2009

Malaria invades the red blood cells, and Prof Crabb’s team is working on a way to block this process.

2010

The grant awarded with the GSK ARE enabled the Burnet Institute to continue their research, helping bridge the gap in funding shortages that the WHO says poses a critical threat to controlling malaria.

2011

“We used the GSK ARE grant money to screen and test a panel of compounds to understand how they work and whether they could help to target some of those processes of the parasite to block the invasion of the red blood cell,” says Prof Crabb. The fight against malaria has made good progress in the past decade, with declining rates of infection and mortality. Between 2010 and 2019, the mortality rate decreased by 44%, due to increased global intervention. “This is a very exciting time for us, as we are at the most advanced point we have ever been at this lab. It is a particularly urgent time in malaria.” Prof Crabb says the global spotlight on COVID-19 has helped shine a light on infectious diseases more generally. “COVID-19 has made clear that science always has to be front and centre of the response,” he says. Prof Crabb was the 38th recipient of the GSK ARE, and another example of a great Australian scientist making globally-significant discoveries and impact via his research.

2012 2013 2014 2015 2016 2017 2018

Associate Professors Jane Visvader and Geoffrey Lindeman - Walter & Eliza Hall Institute of Medical Research Professors Joseph Trapani and Mark Smyth – Peter MacCallum Cancer Centre Professor Charles Mackay – Monash University

Professor Kathryn North – Sydney Medical School, University of Sydney Professor Chris Goodnow – Australian National University Professor Ingrid Scheffer – University of Melbourne Professor David Craik – University of Queensland Professors James McCluskey and Jamie Rossjohn – University of Melbourne and Monash University Professors Jamie Sexton and Arthur Christopoulos – Monash University Professor Timothy Hughes – South Australian Health and Medical Research Institute Professors Georgina Long and Richard Scolyer – Melanoma Institute Australia

2019

Professor Brendan Crabb – Burnet Institute

2020

Professor Mark Febbraio – Monash University

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THE POWER OF THE CONSUMER VOICE Embedding the consumer perspective in large collaborative cancer research initiatives

ustr alian cancer patients need timely and affordable access to the best cancer treatments available. Yet, deciding which new cancer treatments to subsidise on the PBS and MBS is a complex undertaking, with rapid growth in the number, type, combinations, and cost of cancer treatments creating a dynamic landscape. This variation increasingly leads to gaps in the supporting evidence about the new treatments, introducing uncertainty for decision makers and patients. PRIMCAT, a large consumer-informed multi-institutional MRFF funded research program (MRF1199701), is underway to address some uncertainties by forecasting changes in cancer patient populations eligible for treatment. This will better inform government decision making and improve patient access to needed treatments. Led by Professor Maarten IJzerman (University of Melbourne), and in collaboration with the University of NSW: Centre for Big Data Research in Health, The Walter and Eliza Hall Institute, The Melbourne Institute and The Sir Peter Mac Callum Department of Oncology, PRIMCAT has partnered with consumers from inception. A key

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member of the team, consumer leader and Associate Investigator Ms Sophy Athan drives the strategic intent to broaden and strengthen the role of consumers over the course of the research program.

CONSUMERS IMPROVE RESEARCH

Research impacts consumers and consumers should therefore play an active role in research. When we partner with consumers and understand their perspective, it can influence the research undertaken, making it more relevant for consumers and highlighting priority areas and research gaps. Consumer advice can reframe research and change the way it is approached or designed, bringing insights into the potential impacts of research and transforming the translation of results.

PARTNERSHIP MODEL OF CONSUMER ENGAGEMENT

To achieve meaningful consumer engagement in PRIMCAT, it was important to adopt a partnership model. This meant finding a diverse group of consumer leaders who could bring an informed consumer perspective to the research team and creating an environment where consumers and researchers could actively engage as peers in a professional and supportive environment. Consumer leader Sophy Athan convened the team to discuss the consumer impact of our research and the areas that would benefit from consumer input. We reflected on the value that consumers could provide, what we might learn from consumers and the opportunities created from a strong consumer partnership with our researchers.

The PRIMCAT Consumer Panel (L-R): Ms Sophy Athan (Chair), Mr David Attwood, Mrs Carolyn Rowan, Ms Catherine Bressanutti, Mrs Wendy Benson, Mr Paul Baden and Mr George Jiang.

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We discussed different approaches that we could take to engage consumers meaningfully in our work, acknowledging the planning and preparation to ensure a well-executed process that supported consumers and researchers and lay the groundwork for success.

A HYBRID MODEL TO SUPPORT DIVERSE RESEARCH NEEDS

We determined that a hybrid approach was needed. We sought to establish a panel of highly competent and experienced consumer leaders who would come together over the course of the research program, developing a detailed understanding of the research and providing consumer perspective at a strategic level across different aspects of the research program. Diversity was a recruitment priority, driven by a need to have many voices around the table to strengthen the contribution. We also recognised a need to periodically form single-topic consumer focus groups to provide deep-dive input on very specific research areas based on individual consumer experiences. With the approach established, we set to work to bring it to fruition. This involved careful planning

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and clear documentation to ensure transparency in requirements, recruitment and operation, and a system of monitoring and feedback to support both consumers and researchers.

MEET THE PRIMCAT CONSUMER PANEL

The PRIMCAT consumer panel is now operational. We have a diverse group of highly experienced and committed consumers who are bringing a powerful voice to our research. The panel brings learnings developed from their previous roles on consumer committees and in other consumer engagements, allowing transcendence of individual experiences to identify with groups of consumers. Established health issue literacy and the ability to professionally advocate for important patient issues lays a strong foundation. The panel will continue to meet regularly over the course of the research program and interact directly with the research team.

FUTURE POSSIBILITIES

An active collaboration across three MRFF funded research programs ( The Daf fodil Centre at the

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University of Sydney, a partnership with Cancer Council NSW: Cancer Patient Population Projections; Monash University: Epidemiological modelling to deliver better care for Australian patients with myeloma ‘EpiMAP Myeloma’ and PRIMCAT) provides a potential opportunity to leverage or expand the PRIMCAT consumer panel and relationships and to engage with other groups on their consumer consultation processes. We anticipate opportunities for the dissemination of research results through consumer channels and for broad aspirational consumer collaboration on new areas of consumer-led research.

Author: Karen Trapani (University of Melbourne: Cancer Health Services Research) Sophy Athan (PRIMCAT Associate Investigator & PRIMCAT Consumer Panel Chair)

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PATIENTEMPOWERED RESEARCH IN PROSTATE CANCER A consumer-led initiative within the Prostate Cancer Prognosis and Treatment Study (PRECEPT).

e a r n i n g t h a t yo u h ave c a n c e r c a n b e a confronting and frightening experience, and the variation in prostate cancer treatment options can be particularly confusing for men. How do men that were diagnosed with localized prostate cancer decide on a treatment? And how would biomarker information influence the decision-making process? These are questions consumers from the Victorian Comprehensive Cancer Centre (VCCC) and researchers from the University of Melbourne are tackling together as part of the PRostatE CancEr Prognosis and Treatment Study (PRECEPT).

Sophy Athan (top left), Dr Riccarda Peters (top right), Les Leckie (lower left), Keith Donohoe (lower right). 54 INSPIRE 021 | 2021

The PRostatE CancEr Prognosis and Treatment Study (PRECEPT), which was funded by the Prostate Cancer Research Alliance, a joint initiative between the Movember Foundation and the Australian Government, aims to improve care for patients with prostate cancer. PRECEPT

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involves genomics, immunotherapy, and health services research to establish genomic biomarker-based tests for shared decision-making in treatment. This research program brings together a multi-institutional, transdisciplinary and international team to make significant improvements in the outcomes of men with localised prostate cancer and involves several research projects. While there is ongoing consumer involvement in programs within the PRECEPT study, there is one that stands out as great example of consumer-led practice, which we describe here in more detail. Conversation with the Cancer Consumer Advisory Committee (CCAC) at the Victorian Comprehensive Cancer Centre ( VCCC) about the program started well before it got underway. This consultation process identified a knowledge gap around treatment decision making from the perspective of men. Sophy Athan, Keith Donohoe and Les Leckie are the consumer advisors who have been involved in this project since its inception. They are respectively Chair and members of the Cancer Consumer Advisory Committee (CCAC) at the Victorian Comprehensive Cancer Centre (VCCC), and importantly bring their consumer experience with prostate cancer as patients and carer, their skills and their consumer advisory expertise, as well as their lived experience as an asset to the team.

The involvement of consumer advisors in research has so many benefits, particularly in a project like this one with a focus on understanding the decision-making process of patients. Consumer engagement can help identify areas of need and priorities to understand what drives value from a consumer perspective, which ensures that the work is relevant and therefore likely to have a real-world impact. And of course, patients and carers who are impacted by the research bring a unique perspective.

HOW DOES THIS COLLABORATION WORK IN PRACTICE?

Since starting to work together, Sophy, Les and Keith have been meeting regularly with the researchers to develop the project together and to discuss progress and priorities. The study involves the development of a discrete choice experiment (DCE) to understand what matters to men in

the decision-making process for treatments for localised prostate cancer. Discrete choice experiments are a type of survey that allows researchers to elicit preferences for goods or services. DCEs are increasingly used in healthcare and to elicit preferences for various healthcare interventions and services, they allow understanding of peoples’ preferences, and the trade-offs people are willing to make when considering, for example, a new treatment. The development of such a DCE survey involves several stages following a mixed-method approach and requires a thorough understanding of the decision problem. The first stage typically involves a review of the literature, stakeholder consultation and qualitative research. In the next phase an experimental design is established, the survey is constructed, and data collected using the survey. Having the consumer advisors involved in these stages has been invaluable. From the conceptualisation of the research question to the preparation of focus groups with patients, to the actual experimental design and data collection. This discrete choice experiment design has been greatly enhanced throughout by the lived experience of both the patient and carer view of the collaborators. They have provided a unique focus on the decision-making process of diagnosed men which has informed the deployment of a high-quality questionnaire. The consumers have reported great pleasure and satisfaction in being involved in shaping the course of this discrete choice project. Quote from one of our consumer advisors: “Being involved as a consumer carer has helped me better understand how complex the choice process is for both the patient and the partner/carer.” If you would like to know more about the PRECEPT project visit www.prostatecancer-research.org/index.html Author: Dr Riccarda Peters is a Research Fellow in the Cancer Health Services Research Unit, The University of Melbourne, and the PRECEPT consumer team: Sophy Athan is the Chair of the Cancer Consumer Advisory Committee (CCAC) at the Victorian Comprehensive Cancer Centre (VCCC), Keith Donohoe and Les Leckie are members of the Cancer Consumer Advisory Committee (CCAC) at the Victorian Comprehensive Cancer Centre (VCCC).

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GUIDING DRUG DEVELOPMENT WITH PATIENT INSIGHT At the core of CSL’s values and strategy is our promise to meet the current and future needs of patients and better human health.

n rare disease communities, patients have often battled for years to receive an accurate diagnosis. Many patients with rare diseases accept that they may not see a ‘cure’ in their lifetime, so their focus and that of their healthcare professional may be on life-saving medicines which will deliver them a better quality of life. Naturally, they have strong and considered opinions about what they want and need to help manage their condition and what specifies a tangible and measurable increase in their quality of life. For CSL, the end goal is always translating basic science into life-saving, life-changing medicines but the reality is that bringing new products to market is not just about commercialisation. The foundation of CSL’s whole business is our promise to patients – meeting their needs in new and evolving ways. The accurate and deep understanding of a patient’s disease symptoms, the impact their disease has on quality of life and the outcomes that are important to them as individuals is pivotal in developing medicines to meet their needs. 56 INSPIRE 021 | 2021

CSL recognises the need to position itself as a partner with patients throughout the entire product development lifecycle – from laborator y innovation to bedside administration. Listening to people who live with the diseases our therapies treat begins in the earliest stages of drug research.

A FORMAL APPROACH TO PATIENT INSIGHT GATHERING

Throughout the entire drug research and development process, CSL employs numerous strategies to create ongoing conversations with patients where insights are captured and brought back to influence the work we do. In addition to utilising patient surveys, interviews and focus groups, we engage patient advisory boards, exercises to map the patient journey and studies to surface patient preferences for their care. These valuable interactions not only deeply motivate us at CSL, they have inspired better strategic decision-making for research, clinical development and study execution teams.

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PATIENT INSIGHTS FOR SJOGRENS SYNDROME TO INFORM EARLY STAGE RESEARCH: A CASE STUDY

To gain vital insights and inform research, CSL invited an advisory board of 10 people living with Sjogren’s syndrome to meet with our scientists and explore how current treatment options for this autoimmune disorder could be improved or enhanced. Hearing directly from people living with this disease was invaluable in helping our scientists design research programs to address their real unmet needs and improve quality of life – and the earlier in the process the better. In addition to providing critical insights for the research process, the patients found value in sharing their stories with each other. Our CSL employees embraced the opportunity to learn from the patients and were hugely motivated by this interactive engagement. To support patient focused work and engagement, CSL is building an internal online platform which will serve as a central resource for teams seeking inspiration, guidance, connection and wanting to learn more about colleagues’ patient engagements. The aim of the portal is to support employees as they embed patient focus into their ways of working and create a continuous learning environment. Additionally, the formalisation of the process in which patient insights are captured and stored reflects our belief that patients are experts in their condition, and when we partner with them and actively listen to their experience, we can better reflect their needs and perspectives when developing and delivering new therapies. Current components of our internal patient focus infrastructure include an employee forum to share information across the business to further develop concepts and ideas. Through a global network, CSL employees subjects for discussion and participate to foster cultural connections through conversations about work and experiences patients.

can suggest in sessions meaningful with, and for

In addition, senior leaders act as patient focus champions within a dedicated peer network. Set up to leverage combined experiences, it serves as a guiding forum for CSL teams seeking advice and strategic input about patient-focused activities.

CLINICAL TRIAL DESIGN AND IMPLEMENTATION

In our approach to clinical trials, we make every effort to ensure that they are designed in collaboration with patients, caregivers and healthcare professionals. Clinical trial design is one of the areas where partnering with

patients is most crucial, as a successful study design translates into a positive experience for study participants throughout the clinical trial lifecycle.

To that end, we work closely with patients and patient groups to help design the way we conduct our clinical trials and optimise the clinical trial experience. Our commitment to partnering with patients doesn’t stop at clinical trial design. CSL remains engaged with patients from the point of initial interest in a clinical trial all the way through to the end of their study participation. In fact, the next evolution of our patient engagement experience is the implementation of an electronic patient engagement exchange (EPEX), which will provide a digital platform for prospective and randomised study participants to have an even better clinical trial experience. The platform is designed to provide easy access to information, study transparency and resources that will make life easier for our study participants. Through the secure EPEX platform, we will continue to ensure that patients and caregivers have access to information that will help them make informed decisions about study participation, all while maintaining patient anonymity. Our development teams listen closely to patients in a further effort to gain their perspective to inform design decisions as the development process moves forward. It’s a continuing conversation that creates a mutually beneficial experience for all involved. As one clinical trial participant recently acknowledged, the experience can change the healthcare paradigm for patients. Going into the clinical trial, she saw herself simply as a taker of medicine. Afterwards, she described herself as “part of the team.”

AT THE CORE OF CSL’S VALUES IS OUR PROMISE TO MEET THE CURRENT AND FUTURE NEEDS OF PATIENTS

Patient focus is deeply engrained into our research and development efforts, and our partnership with patients doesn’t end when therapies make it to market. At CSL, relationships with patient groups are among the most important collaborations we have. In all of our efforts, we aim to uncover new ways in which we may be able to serve patient needs and apply their insights to new treatment solutions.

Author: Deirdre BeVard, Senior Vice President, CSL R&D Strategic Operations.

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UNDERSTANDING THE CONSUMER EXPERIENCE TO ENHANCE QUALITY CARE IN THE DIGITAL AGE In 2018, the WHO published ‘Delivering Quality Health Services – a Global Imperative for Universal Health Coverage’, urging governments to focus on quality of healthcare service provision, rather than quantity. This involves “the right care, at the right time, in the right place, and by the right care provider” (WHO, 2018). High-quality care is associated with safe, equitable, effective and efficient health delivery, leading to improved patient satisfaction and health outcomes, thus decreasing societal and economic burdens.

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r g u a b l y, d i g i t a l h e a l t h i s a n e s s e n t i a l component of high-quality care delivery in chronic diseases such as melanoma. Indeed, the transformative digital advancement in healthcare unlocked a more effective and collaborative approach to health, enabling enhanced experiences of care for patients. Nevertheless, to enhance the impact of health services and effectively implement digital solutions for end-users, it is essential to have a comprehensive understanding of the consumer needs and individual user characteristics. In other words, employing a user-centred approach to design new products and services will enhance iterative adoption and growth of digital health. A systematic review by Rollin, Ridout & Campbell (2018) provided a documented basis for how melanoma survivors use technology to manage their illness. The findings suggested that tailored digital health can promote high-quality care.

However, of utmost importance, it highlighted extensive research gaps found from prior data captured and analysed in this field of research, which primarily focused on product functionalities and outputs, rather than on user experience and outcomes. Many of the studies reviewed demonstrated digital health opportunities by highlighting its overall benefits - being cost-effective, time-effective or convenient - instead of focusing on the users’ actual needs and demands. In fact, none of the studies considered individual characteristics and needs when assessing survivors’ experience of digital interventions to manage their health. To provide users with the best solutions, it is essential to collect person-specific information to create actionable insights and understand the social, cultural and environmental drivers of digital health adoptions. To achieve this, researchers must look at not only practical needs related to existing management plans and associated comorbidity, but also individual sociodemographics, psychological needs, IT capabilities, and current perceptions and use of technology. In a recent study (Rollin, 2021), we adopted a userexperience research approach to develop a digital health framework that can benefit both melanoma survivors and clinicians. During the design process, the study surveyed patients to identify gaps in the current model of care and identified digital tools that are likely to respond to their preferences and needs. Results indicated that melanoma survivors are willing to integrate technology into their medical routine if it makes their life better and easier. However, attitudes toward digital health varied by technology/system. These differences between users’ preferences for certain technologies were explained by their differing 60 INSPIRE 021 | 2021

individual needs (e.g., their communication preferences, informational needs, social or psychological needs, or practical needs and wants). In summary, to achieve the greatest impact, it is essential to keep the consumer in focus when researching, designing and implementing digital health solutions. However, establishing a user-centric model is not without its challenges. The rapid pace of technological change generates difficulties in measuring the benefits of digital health in mid- to long-term adoption. Indeed, the misalignment between the cadence of traditional research and fast-paced innovations gives rise to the digital health paradox of “no evidence, no implementation—no implementation, no evidence” (Guo

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et al., 2020). While it adds to the difficulties of providing timely and robust evidence of digital health best practices, researchers must make the effort to properly investigate and understand the behaviour, motivations and requirements of end-users if they are to identify what tools, systems and mechanisms will actually amplify the adoption of digital health innovations. Delivering quality health services: a global imperative for universal health coverage. Geneva: World Health Organization, Organisation for Economic Co-operation and Development, and The World Bank; 2018. Rollin, A., Ridout, B., & Campbell, A. (2018). Digital Health in Melanoma Posttreatment Care in Rural and Remote Australia: Systematic Review. Journal of Medical Internet Research, 20(9), Article e11547. Rollin, A. (2021). Digital health to improve Australians’ experience of

care: The case of melanoma posttreatment care [Unpublished doctoral dissertation]. The University of Sydney. Guo, C., Ashrafian, H., Ghafur, S., Fontana, G., Gardner, C., & Prime, M. (2020). Challenges for the evaluation of digital health solutions-A call for innovative evidence generation approaches. NPJ Digit Med, 3, Article 110.

Authors: Audrey Rollin, Member of the Cyberpsychology Research Group. Associate Professor Andrew Campbell, Chair and Cofounder of the Cyberpsychology Research Group Dr Brad Ridout, Deputy Chair and Co-founder of the Cyberpsychology Research Group. University of Sydney 2021 | INSPIRE 021 61

Lehi Poppe 62 INSPIRE 021 | 2021

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DESIGNING WITH DONORS At Australian Red Cross Lifeblood, we manufacture life-giving medical products from materials given freely by donors, and we’re working with donors from all walks of life to make sure we have blood products available for everyone who needs them.

ife blo o d’s r e s e a r c h e r s do n’t a ll wo r k in laboratories, looking down microscopes and testing tubes of blood. Our team includes social researchers who help us understand what keeps our donors coming back, and make sure they have the best experience when they come in to donate blood or plasma.

FINDING RARE BLOOD DONORS IN THE PACIFIC

Blood types are molecular markers on the outside of our red blood cells, and like our hair, eyes and skin colours, there are many variations and combinations that vary depending on someone’s ethnic ancestry. The commonly known A, B and O blood types are just the tip of the blood-group iceberg. Some patients need blood that is matched much more closely to their own than just the A, B and O groups, and if their type is rare, we need to find a rare type to match. In particular, we often have difficulties locating a rare blood type called “JkNull” which is found more frequently among people with Polynesian ancestry – although it is also rare among them too (0.1% - 1.4%). To give us a better chance

of finding it for patients who need it, Lifeblood needs to increase the number of people from those communities who donate blood. In the past we appealed for donors of Polynesian ancestry through TV, radio, and newspapers. While this has sometimes helped, it hasn’t been as successful as we need. The message just didn’t seem to get to the people who need to hear it. We decided to try something different and use co-design, where the communities are the experts, guide how we conduct the research, and play a major role in developing the things that come from the research. One of the things our participants created with us was a video for social media – their preferred form of media. It was done in the voice of the community that we worked with, in their style, their music, their humour. The message was presented in a way that they believed would actually be received and acted on. They were right. In one month, over 82,000 people viewed the video on Lifeblood’s Facebook page and thousands more on the community members’ own posts. Over 231 people signed up to donate by June 30 and 174 donations were made by community members. Most importantly, we’ve found a record number of donors with the rare blood type and it’s already helping patients.

DONORS SHARING EXPERTISE

O ur donors are ce ntral to our wor k, and we’re incorporating their input into our research program. In 2017 we first called out for blood donors in Victoria to join our Donor Research Consultation Committee. We were overwhelmed with offers-over 400 applicants for 20 positions, all eager to contribute their opinions and expertise to Lifeblood. Our committee combines people with a range of donation experience and with diverse characteristics including gender, age and location. We also wanted to make sure 2021 | INSPIRE 021 63

we selected people who brought a range of perspectives to the group. The committee has provided us with great feedback that’s helped us improve our communications. Sometimes when a donor comes in for their appointment, they may not be able to donate blood for a range of reasons, and their donation is postponed until a later date. It’s important that we communicate the reasons clearly, and let them know when they can return. The committee provided input on the first drafts of the postponement brochure which is now being used in donor centres. Their feedback supported the idea of a brochure that focused on empathising with the donor rather than reminding donors of recipients (who they have been unable to help) at this time. Members of our committee have been enthusiastic about the consultation process.

My involvement as a volunteer donor has taught me a huge amount about what really makes Lifeblood tick and highlighted just how much happens behind the scenes” said Pete Ransom “I can honestly say that I feel I have taken a lot more from this process, than I’ve given.” Jocelyn McMillan, another long-term committee member said “I’m a huge fan of service users being given the opportunity to give their perspective. It gave me some insight into the complexities of blood, plasma and platelet donations, something I had never considered before. The COVID-19 pandemic has had unexpected benefits for this committee. Since the restrictions on meetings and travel during the pandemic, we’ve moved our meetings from face-to-face to virtual, so we’re able to extend the invitation to donors from outside of Victoria and to those in regional Australia. Author: Dr Rachel Thorpe, Research Fellow, Australian Red Cross Lifeblood. Dr Luke Gahan, Research Fellow, Australian Red Cross Lifeblood. Dr Alison Gould, Scientific Communications Specialist, Australian Red Cross Lifeblood.

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INVOLVING THE CONSUMER AT THE OUTSET OF RESEARCH

enomic medicine is transforming the healthcare sector, but at unequal rates. Sharing data, tools and knowledge, and critically, involving consumers at the outset of research, to create an international learning health system is essential if we are to effectively accelerate and sustain the integration of genomics into healthcare in Australia and globally.

had just got him home and as far as we knew, he was a perfect, healthy baby,” Karsha said.

FROM THE WORST NEWS POSSIBLE, TO THE BEST, IN JUST THREE DAYS.

Within the week Levi was transferred to The Royal Children’s Hospital in Melbourne, under the care of Dr Katherine Howell, a paediatric neurologist and the epilepsy research team leader at the Murdoch Children’s Research Institute (MCRI).

Levi Trowbridge was just three days old when he had his first seizure. His parents, Karsha and Steve, were left “shocked and confused” by Levi’s experience. “We 66 INSPIRE 021 | 2021

After experiencing a second seizure, his parents rushed him to hospital where he had another four seizures. Despite being treated with multiple anti-seizure drugs, Levi didn’t improve. “It was a terrifying time, watching your baby have multiple seizures a day and, at times, turning blue and holding his breath,” Karsha said.

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“We had a result just days later,” Dr Howell said.

CHRISTOPHEGenomics, KEREBEL Investigator of Australian a collaborative network launched in 2016 to help drive the integration of genomics into mainstream healthcare. With more than 80 partner organisations and over 440 investigators, the network comprises consumer support groups, clinical geneticists, genetic counsellors, clinical laboratories, hospitals, academic research institutions, and federal and state governments.

The testing uncovered a rare genetic condition known as KCNQ2 encephalopathy, caused by disrupted potassium regulation in the brain.

This broad spectrum of research will enable a nationally consistent approach to the implementation of genomics in healthcare.

Armed with that knowledge, Dr Howell changed Levi’s anticonvulsant medication, weaning him off the other drugs that weren’t working. Levi was seizure-free within weeks.

Australian Genomics, is a ‘Driver Project’ of GA4GH, helping develop tools and policies for data sharing, testing them under real-world conditions, and disseminating best practice.

INTERNATIONAL ACTION

IMPLEMENTING NEW KNOWLEDGE

Levi’s initial diagnosis was early infantile epileptic encephalopathy.

Dr Howell arranged “ultrarapid” genomic testing of Levi through an Australian Genomics national study of critically ill children, led in Victoria by the MCRI’s not-for-profit subsidiary, the Victorian Clinical Genetics Service.

As Levi’s story shows, genomic medicine is transforming the global healthcare landscape and MCRI has been at the forefront of this transformation nationally and internationally. A new global collaboration — the International Precision Child Health Partnership (IPCHiP) — will see four major paediatric centres on three continents join forces to analyse medical and genomic data to speed up our understanding of genetic disease and accelerate development of new therapies. IPCHiP participants include MCRI, the Royal Children’s Hospital and the University of Melbourne in Australia, the Boston Children’s Hospital in the US, the Hospital for Sick Children in Canada and the UCL Great Ormond Street Institute for Child Health and Great Ormond Street Hospital in England. IPCHiP’s initial effort will focus on two key areas of genomic health: infant genetic epilepsies, and improving acute diagnostic rates – and increasing treatment options – of rare genetic disease. The team plans to recruit up to 450 families across the four sites over three years in the rare genetic epilsepy project. Sharing data, tools and knowledge to create a global learning health system Professor North is Vice-Chair of the Global Alliance for Genomics and Health (GA4GH), a collaborative network of more than 600 organisations across 100 countries, supported by the US National Institutes of Health (NIH), the UK Medical Research Council (MRC) and the Wellcome Trust.

The goal of GA4GH is to develop and implement technical and regulatory standards for the responsible sharing of clinical and genomic data to benefit human health. AUSTRALIAN GENOMICS

At the national level, Professor Nor th is the Lead

In partnership with the GA4GH, Australian Genomics and Genomics England established the Genomics in Health Implementation Forum (GHIF) to strengthen international collaboration between large-scale precision health initiatives. Co-Chaired by Professor North and Sir Mark Caulfield from Genomics England, GHIF supports the development and implementation of GA4GH standards, and provides a platform for large-scale national genomics initiatives to identify potential opportunities to collaborate and share resources and expertise. The group is developing an online toolkit to facilitate collective learning and data sharing, which will help medical professionals provide targeted precision medicine treatments for consumers.

AN INCREDIBLE FUTURE AWAITS

Eight years ago, genomic sequencing was only used for research. Now, it’s increasingly used in clinical practice, and with over 60 million patients providing genomic data over the next five years it’s only a matter of time until genomic and molecular data have been collected for over a billion people. This ultimate example of consumer engagement will deliver both personalised and population-based benefits in acute healthcare, preventative medicine, and other unforeseen ways. Currently we can only diagnose around half of all kids with rare disease. When researchers everywhere can access secure data for secondary analysis, discoveries will accelerate in both health and fundamental biology. This ability to provide a fast and accurate diagnosis, and remove the trial and error from treatment, will benefit kids like Levi, everywhere. Author: Tom Keeble BSC PhD, Communications Manager, Marketing and Communications Murdoch Children's Research Institute 2021 | INSPIRE 021 67

PUTTING CONSUMER

LEADERSHIP IN THE SPOTLIGHT

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Dr Brett Scholz’s approach to his research is putting consumer leadership in the spotlight, recently winning him the Australian Capital Territory 2021 Young Tall Poppy Science Award, and being a finalist, along with his colleague, Professor Imogen Mitchell, in the Research Australia Health and Medical Awards, with winners to be announced later this year.

ecisions about health care services, policies and systems have traditionally been the domain of medical professionals, allied health workers, researchers and policy makers. But there is one voice consistently missing from these discussions. The consumer - the people who engage with health services. That is now changing as the consumer leadership in health movement gains momentum. The movement consists of consumer organisations, individual consumers working to change systems, and includes some health care professionals who want to support the movement’s goals. Dr Brett Scholz, an early career researcher from the Australian National University (ANU), is one such proponent for the inclusion of consumers in health care decisions. “A consumer knows the health system in a completely different way to a medical professional. They have a holistic understanding of what it might be like to sit in a noisy waiting room as they anticipate the results of their cancer screening, or how it might feel to find out they are ineligible for a particular treatment.” “Consumer expertise often goes unrecognised but it has implications for how health systems and organisations make decisions about everything from how services can remain comfortable, safe, and relevant, to how potentially life-saving care should be allocated in a pandemic.” “Their experience provides a much needed balance in decision making processes,” Dr Scholz says. “When I started my research career some years ago, I was lucky enough to work in an organisation that had

an identified role for a consumer researcher. It helped me become aware of how oblivious I had been to the expertise and invaluable knowledge consumers have.” He found that diversity of views within a research group significantly enhanced the breadth and quality of all aspects of the research, including an increased awareness and mutual learning, a stronger ethics review process, a deepened data analysis, expanded recruitment and data collection methods, and an overall relevancy to the research. “In the case of mental health research, there was no level of awareness, or mindfulness, or formally learned knowledge in higher education or clinical experience that could replace the direct lived experience, particularly of those who had been marginalised by society and further stigmatised in their mental health service use.” This was the start of Dr Scholz including consumers within his own research and advocating for them to be included in health care decision-making more broadly. “Over the past six years I’ve co-conceptualised and coauthored about two-thirds of my research with those who have a lived experience of illness. I also try to collaborate with consumers in the delivery of my teaching so that medical students can learn first-hand about consumer experiences and see me modelling what a partnership between a consumer and another health professional might look like.” As for the future, Dr Scholz would like to see a wider variety of consumer groups included in decision making across the health care sector, such as people with disabilities, Aboriginal and Torres Strait Islander people, older adults and younger consumers.

I’m focussed on continuing to support the expansion and extension of consumer led work so that we end up with a health system that truly partners with consumers in policy, service, education and research.” “When I think about research work that wins awards, qualitative research that advocates for social change is not the type of work that typically springs to mind so I’m very thankful and happy that the work I love to do has been recognised and I’m able to raise further awareness about consumer leadership.”

Author: Kierra-Jade Maciver, Strategic Communications & Marketing Manager, College of Health and Medicine, The Australian National University.

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THE LAST WORD

Building the unbroken pipeline The future of patient focused medicine in Australia requires a well-established and sustainable biotech ecosystem which encourages end to end, on-shore research translation, development and commercialisation to make medicines available to the people who need them, sooner.

SL is a global specialty biotechnology company, headquar tered in Melbourne, Au s t r a li a . O u r pu r p os e i s to d eve lo p innovative medicines which protect human health and help people with rare and life-threatening medical conditions live full lives. As the world’s third largest biotech we have a dedicated team of over 1,700 research scientists working across multiple sites in Australia and around the world. These teams are forging discoveries across six therapeutic areas of research focus: immunology, haematology, cardiovascular and metabolic, transplant, respiratory and influenza. Australia is home to CSL’s global hub for research and translational medicine and we are Australia’s largest private sector investor in medical research. Over the past five years we have invested US$3.7 billion into R&D globally. Through early stage research discovery and partnerships, we aim to deliver innovative development opportunities to meet the clinical needs of patients with rare diseases and protect human health. Melbourne, our home, is a world-renowned centre for original biotechnology-focused research and is one of only four cities with two universities in the global top-40 biomedical rankings - in good company with London, New York and Cambridge Massachusetts. The Parkville precinct alone contains numerous world-class hospitals, medical research institutes and universities and attracts almost 25% of Australia’s competitive biotech research funding.1

However, Australia lags similar global regions in the translation of early stage medical research into spinouts and commercial outcomes. In fact, our universities and public research organisations commercialise far fewer start-ups per $100 million research expenditure 70 INSPIRE 021 | 2021

than our UK, Canadian and US peers. 2 Australia is known for early stage innovation to address the world’s unmet medical needs but there is a gap in our collective ability to translate early stage innovation into commercial outcomes. Research translation, the process of moving innovative medical discoveries through the research pipeline to become new products, is an integral component of a thriving biomedical ecosystem and is vital to the ongoing health of patients worldwide. Translation is critical. Ground-breaking research which stays within laboratories or stalls in the transition to becoming a commercial treatment for those most in need, is not just a lost opportunity but time that patients often don’t have. A healthy biomedical ecosystem with strong end-toend capabilities, functioning within a policy environment that is conducive to translation will benefit us all universities, research institutions, pharmaceutical companies, and most importantly, patients. It means innovative discoveries born out of the laboratories of our universities and smaller biotechs may make a difference in patients’ lives sooner through a streamlined R&D pipeline, access to clinical trials and the ability for patients input into the drug development process. An established and attractive biotech sector requires numerous components to work in synergy: world class research; cutting edge infrastructure and facilities; investment support for innovations across the development lifecycle; the ability to translate the research into commercial outcomes, and above all, collaboration. To strengthen the existing network requires ongoing collaboration between industry, research institutions, universities, investors and bespoke Government initiatives.

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Fundamental to shaping our biotech environment are Government implemented policies which increase the attractiveness of Australia’s business environment and even the playing field with international peer nations. This includes incentives across the pipeline which are becoming standard across the industry. The recently announced Australian Patent Box tax incentive, which offers concessional tax treatments to Australian IP derived profits, is a good example. Tax incentives like the patent box, when implemented appropriately create a stronger, more competitive business environment when decisionmakers of emerging and established biotech’s consider where to locate and commercialise their intellectual property. As Australia’s largest biotech, CSL is committed to helping foster a thriving biomedical ecosystem. CSL is currently undergoing large scale investment to expand current endto-end biomedical development through to manufacturing capacity. These investments will ensure we can sustain our operations here in Australia by further integrating them into our global network and responding to an increased demand for our products.

addition, patients who are waiting on life changing or saving medicines born out of discoveries in Australian research institutes will benefit sooner as IP won’t need to be shipped around the world to progress to the next development stage. Under the spotlight of a global pandemic and great patient need for fast tracked innovation from bench to bedside, now is the time to explore options to establish and future proof Australia’s end to end biotech capabilities for patients and the future of research and development in Australia. References 1. Melbourne Biomedical Precinct. https://www.melbournebiomed. com/the-melbourne-biomedical-precinct-office/faqs/ (accessed August 2021) 2. Commonwealth of Australia. (2017). Australia 2030: Prosperity Through Innovation; Australian Government Department of Industry, Innovation and Science, National Survey of Research Commercialisation, DIIS, Canberra https://www.industry.gov.au/dataand-publications/australia-2030-prosperity-through-innovation

In addition to a new base fractionation facility at our Broadmeadows site and a new state-of-the-art, cellbased influenza vaccine facility for Seqirus at Tullamarine, we are currently building our new global headquarters, located in the heart of Melbourne’s biomedical precinct – Parkville. Our presence here is no accident. The building will be home to nine levels of cutting edge, world-class laboratories, and facilities. Further, we have plans to include a biotech incubator hub within the new building to foster greater commercialisation of research in the biotech precinct. Biotech incubators have been shown to accelerate the pathway from research to commercialisation by providing affordable infrastructure and access to capabilities, capital, and communities that are essential to start-up company success. For patients, who are collectively, the reason many scientists and researchers in the biomedical sector decide on their chosen career path, it means more opportunities and time saved. Building our local biotech end-to-end capabilities and creating an environment rich in support for local research translation means, in the simplest terms, a streamlined and expedited pipeline of Australian therapies and technologies to address unmet medical needs for patients. It will open opportunities to Australian patients who will have greater access to clinical trials as research is translated into lifesaving medicines, locally. In

Author: Dr Andrew Nash, CSL’s Chief Scientific Officer & Senior Vice President, Head of Research and Research Australia Board Member.

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