MPNErare2017 session 4 the rare cancer challenge

MPNErare2017 Session 4 The rare Cancer Challenge 7th October 2017 Leiden

The Rare Cancer Challenge- introduction

The need for treatments in rare melanoma • Less efficient or NO treatments • Slow drug research & development How we can better research ?

How we can get faster access?

How we can help to support the above?

The rare cancer challenge: the clinical perspective Sophie Piperno-Neumann Dept of medical oncology Institut Curie, Paris-France MPNErare2017, 6th- 8th- October , Leiden

Definition of Rare Cancer

I I: French NCI (INCa) definition - Incidence (new cases) < 6/100 000 inhabitants per year - Cancers requiring for optimal management access to highly specialized centres, due to heterogeneity of presentation, complexity in diagnosis and treatment

MPNErare2017, 6th- 8th- October , Leiden

Rare Cancers major issues

â&#x20AC;˘ Rare cancers are a challenge for Patients, Doctors, Scientists and Health authorities. â&#x20AC;˘ Definition is multiple, regarding Epidemiology, Physician perspective, Patient perspective, Health Authorities or regulatory perspective

MPNErare2017, 6th- 8th- October , Leiden

Rare Cancers major issues

• Late diagnosis or misdiagnosis • Non-expert management • High rate of morbidity • High potential of recurrences • Lack of effective treatment in the metastatic setting

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Basic requirements for optimal management Of Rare Cancers 1. Early diagnosis 2. Referral to a specialist centre 3. Therapeutic strategy discussed by a specialist multidisciplinary team (Multidisciplinary Tumor Board) 4. Access to appropriate treatment MPNErare2017, 6th- 8th- October , Leiden

Situation in Europe

• • •

Rare Cancers care is heterogeneous

• •

Major concerns with Eastern European Countries

27 countries 500 million humans in different healthcare systems Unequal access to treatment and care

Discrepancies between patients over Europe in terms of access to:

• • • • •

information diagnosis expert medical opinion (initial and second) appropriate management, treatments & care clinical trials

MPNErare2017, 6th- 8th- October , Leiden

Issues in UM Background:

• Lack of published data/insufficient knowledge and shared experience: Risk/chance to be managed for a patient by non experts in UM UM is a rare “melanoma” → BRAF mutation status, PET imaging and immunotherapy … frequently used for patients managed by dermatologists or non expert oncologists • Lack of clinical/preclinical research: animal models and translational research are crucial before clinical trials • Lack of formation (students, nurses) and information (patients, general practitioners) • Lack of communication tools • Not enough interest/money dedicated to UM from pharmas/HA

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Ideas to avoid these problems/limits, compared to frequent tumors Background:

• Communication: “savoir faire et faire savoir” in French expertise and information: let know about it • Groups of experts in the field gathered in national and international networks: collaborative efforts to understand biology and develop innovative therapies i.e. Melachonat in France, Euracan in Europe, IRCI worldwide • Patient Advocacy Groups are starters for research on rare tumors: tumor samples are rare, trials are rare..

• Preclinical models recapitulate patient tumors’ characteristics: test in these models single drugs and combinations before any clinical trial • Trial design in rare cancers: i.e. multi-arm multi-stage trials • Lobbying to involve industry and accelerate drug approval in such “niches” by HA MPNErare2017, 6th- 8th- October , Leiden

European policy response to rare cancers: the case of sarcoma European Parliament, 8th February 2017 Policy debate 14.30-16.30

Hosted by Marlene Mizzi, MEP, on behalf of the Group of Experts of the Sarcoma Policy Checklist

Wi-Fi login details: visit0208 8qnU2Ctg

The Group of Experts of the Sarcoma Policy Checklist is an independent multi-stakeholder group including leading patient representatives, clinical experts, politicians and industry representatives set in 2016 with the aim to develop a Policy Checklist for Sarcoma. The group of experts maintains full editorial control of the final document. Eli Lilly & company (Lilly) is providing th th financial support for meeting costs and materials produced by the group.

MPNErare2017, 6 - 8 - October , Leiden

The Sarcoma Policy Checklist • Developed by sarcoma experts including patient organisations, clinical experts, politicians and industry representatives • Five key areas to focus policy change to improve care and outcomes for sarcoma patients • To see how we are doing in France, Germany, Italy, Spain, Sweden and United Kingdom

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Group of experts of the Sarcoma Policy Checklist

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What is most needed to improve sarcoma care?

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The Sarcoma Policy Checklist • Sarcoma patients still report some of the poorest experiences of care among cancer patients.

• This report proposes five key areas where policy advances are needed to help redress this situation.

• These recommendations are, to a large extent, also applicable to other rare cancers.

• As rare cancer patients represent 22% of all cancer cases in Europe, the urgency to improve care and outcomes for these patients should be a key priority for all European health care systems.

The Sarcoma Policy Checklist is available online, on the SPAEN website MPNErare2017, 6th-

8th- October , Leiden

Clinical trials in rare cancers: the patient perspective

Estelle LECOINTE

AND MEMBER OFâ&#x20AC;¦

MPNErare2017, 6th- 8th- October , Leiden

+ Clinical trials in oncology : crucial tools To improve medical practice: Multi-disciplinary approach Teams coordination Optimal patient therapeutic pathway

To evaluate: Treatment relevance Survival Quality of life

MPNErare2017, 6th- 8th- October , Leiden

+

n

n

When the therapeutic armamentarium has been drained n

Access to innovative therapies

n

Increasing of survival:10.6% of overall response in phase I trials in Cancer)*

1st application in l’humain

Phase 2

Phase 3 Benefit, toxicity comparison vs standard

Early detection of resistance and/or progression

Contributing to the “general need” n

Phase 1

Efficacy, toxicity, dosage

Optimal patient follow-up n

n

PARTICIPATION IN CTs: 3 “good” reasons

Lab

Will to contribute to the development of research

Market Authorization ?

---------------------------------

Phase 4 Long term risk assessment, optimization

(*) Horstmann: Risks and benefits of phase I oncology trials. NEJM 2005, 352:895-904)

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+

STILLâ&#x20AC;Ś Some patients do hesitate to participate ; have no access to clinical trialsâ&#x20AC;Ś

WHY ???

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+

PATIENTS NEED INFORMATION TOâ&#x20AC;¦.

UNDERSTAND THE DISEASE BETTER

BE PART OF THE THERAPEUTIC DECISION MAKING PROCESS

BE FULLY COMPLIANT MPNErare2017, 6th- 8th- October , Leiden

+

EXPECTED INFORMATION DISEASE

PROTOCOL

THERAPEUTIC DECISION MAKING PROCESS

TREATMENT

SIDE-EFFECTS

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+

Impact of information on patient n The

more and the clearer the patient is informedâ&#x20AC;Ś

n

The better he/she will contribute to the maintenance of his/her QOL ;

n

The better he/she will comply to the therapies;

n

The better he/she will increase his/her chance to surviveâ&#x20AC;Ś

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+

INCREASING LENGTH OF INFORM CONSENT FORMS

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BMS Seminar â&#x20AC;&#x201C; Le Touquet , September 23th 2015

+

COMPLEX DESCRIPTION OF THERAPEUTIC REGIMENS

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+

SHORT TIME REFLECTION PERIOD

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+

INCOMPREHENSIBLE WORDING HISTOLOGY PLACEBO

SYSTEMIC THERAPY

RANDOMISATION TREATMENT ARM

MULTICENTRIC COHORT

DOUBLE-BLIND CROSS-OVER PHENOTYPE

CHEMOSENSITIVE

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+

ADVANTAGES AND DRAWBACKS n

Individual advantages: n

n

Collective advantages: n

n

Access to innovative therapies, new diagnostic technics, optimal monitoring

Scientific and medical progress

Drawbacks : n

Risks of inefficacy, progression under placebo, toxicity, surveillance burden

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+

PAG/PHARMA/EXPERTS COLLABORATION n

IMPROVE PATIENTS UNDERSTANDING ABOUT CLINICAL RESEARCH

n

INCREASE THE VISIBILITY OF THE AVAILABLE CLINICAL TRIALS

n

IMPROVE INFORM CONSENT FORMS

n

DESIGN RESEARCH PROTOCOLS MEETING PATIENTS NEEDS

n

INCREASE PATIENTS PARTICIPATION IN CLINICAL TRIALS

MPNErare2017, 6th- 8th- October , Leiden

Trial Design in rare cancers • Rare disease (6/million/year): difficult to enroll a large number of patients with conventional trial designs within a reasonable timeframe • Multicenter worldwide studies • Need for well-designed scientific-based clinical trials - Use existing data to define the population of patients - Choose appropriate endpoints (clinical, biological) - Choose appropriate design: innovative methodologies, such Multi-Arm Multi-Stage design (MAMS) and Bayesian statistics • Aim: maximise the potential for answering research questions (i.e. reduce the study duration and the sample size, by using interim analyses and predefined stopping rules) MPNErare2017, 6th- 8th- October , Leiden

Precision medicine Background:

• AIM: To identify patient subgroups who could benefit from existing drugs outside their registered indication •DNA sequencing = Look for DNA mistakes •Classification of cancer has moved from anatomic/histopathologic classification to molecular/genetic classification→ many rare entities in frequent cancers.

•New approach to personalizing medicine, using compounds targeting molecular aberrations in a tumor in multi-drug multi-tumor trials • Applicable to rare cancers and patients for whom there is no standard of care.

• ESMO 2017 Dr Voest (NKI Amsterdam) Drug Rediscovery Protocol - 19 different drugs, 10 companies - 250 patients screened in 1 year, 70 started treatment - Analysis of response to treatment and clinical benefit (OR or SD>16 wks) - 2 stages: 1* 8 pts, same tumor type, same genetic mutation 2* 24 pts expansion cohort if at least 1 patient benefits from the treatment in stage 1 - Results: 20 cohorts, clinical benefit in 37% of patients, 6 cohorts/20 to second stage MPNErare2017, 6th- 8th- October , Leiden

Examples of networks for Uveal melanoma

•

Melachonat in France

• UMCure2020 and EURACAN in Europe • The International Rare Cancer Initiative (IRCI) worldwide

MPNErare2017, 6th- 8th- October , Leiden

Melachonat Background: • French Uveal Melanoma Network • Supported by the French NCI • Launched in 2013 and coordinated by Dr Desjardins, ophthalmologist at Institut Curie • National database (>4500 registered patients) • Tumor bank coupled with research projects • Dedicated website: - information for patients on existing centers inside the network (contacts and modalities of access) - educational tools (web pages, newsletters..)

- specific access for physicians, including access to the weekly Multidisciplinary Tumor Board (MTB) • National Clinical Practice Guidelines (CPGs) • BUT No french UM PAG !

MPNErare2017, 6th- 8th- October , Leiden

UM Cure 2020

• A Consortium of European experts in Uveal Melanoma • Funded by the European Union’s Horizon 2020 research and innovation programme • Project duration: January 2016 – December 2020 • Aim: To combine the efforts of European Centres of Excellence in Uveal Melanoma oncology and research (from basic to translational and clinical) with patient organisations to develop new therapeutic approaches to treat metastatic UM patients, and share this new knowledge efficiently with the patient and the medical community www.umcure2020.org

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UM Cure 2020 â&#x20AC;&#x201C; 12 European Partners

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A patient-centered approach

WP: work package

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WP1: European UM biobank network

Harmonisation of existing biobank procedures and creation of a European UM virtual biosample registry.

4 UM referral centres - (Comprehensive Cancer Centre or University Hospital): • • • •

Royal Liverpool University Hospital NHS Trust/University of Liverpool, UK Institut Curie, Paris, France Leiden University Medical Centre, The Netherlands Jagiellonian University, Krakow, Poland MPNErare2017, 6th- 8th- October , Leiden

WP1: European UM biobank network

Harmonisation of existing biobank procedures and creation of a European UM virtual biosample registry.

Tasks and deliverables: • • •

Agreement on minimal datasets to be available for each sample/patient Extraction of data from each centre database to build a common virtual UM Cure 2020 registry Prospective collection of metastatic and matched samples with common SOPs for collection, storage transport and processing

MPNErare2017, 6th- 8th- October , Leiden

WP2: Preclinical validation of biology driven therapeutic approaches

Evaluate single drugs/drug combinations in preclinical models to identify novel therapeutic options for metastatic UM patients First phase: test available drugs in available models

Second phase: New targets (WP4) and disease models (WP3)

Tasks and deliverables:

Drugs/drug combinations (Drugs include innovative peptidic approach with biotech PEP-Therapy)

Cell lines

Mouse model/Patientderived Xenograft (PDX)

Zebrafish PDX MPNErare2017, 6th- 8th- October , Leiden

WP3: Development of next generation pre-clinical models

Patient-derived (PDX/CDX) and genetically-modified (GEM) mouse and zebrafish models

Further develop relevant in vitro and in vivo UM models from metastatic samples to better understand the mechanisms of UM oncogenesis and dissemination and to evaluate the most promising therapies for success in the clinic

MPNErare2017, 6th- 8th- October , Leiden

WP4: Increased understanding of molecular Characterisation changes in metastatic UM and generation of UM metastases of new target hypotheses for WP2 Decipher the: • genetic alterations • dysregulated signalling pathways

• characteristics of the UM immune landscape

MPNErare2017, 6th- 8th- October , Leiden

WP5: Dissemination and implementation through clinical trials initiation

Communication tools, UM patient network and interaction with academia and/or Pharma for initiation of clinical trials

â&#x20AC;˘ Ensure widespread dissemination and maximal exploitation of results, in particular through the initiation of UMdedicated clinical trials sponsored by academia or pharma. â&#x20AC;˘ Increase patient information and involvement through a dedicated UM patient and UM caregivers portal associated with the project website, as well as European-wide initiatives for UM patients to meet, network and get involved.

http://www.umcure2020.org MPNErare2017, 6th- 8th- October , Leiden

Main achievements in the first 18 months of the project Ø Already 533 patient samples from 101 patients, among which 278 metastatic UM samples are registered in a common virtual database, available for an in depth characterisation of the disease. Ø 22 combinations of drugs, chosen on the basis of our current knowledge in UM, have been tested in vitro on a panel of patient-derived UM cell lines. Some promising results will now be validated in patient-derived animal models. Ø A chemical screen of FDA- and EMA-approved drugs was also performed in a zebrafish GEM model of UM. Hits will now be further studied. Ø Our panel of preclinical models representative of the metastatic disease is growing, with some models still under development or validation. Ø A total of 115 patient samples have been analysed by whole exome sequencing, and the on going analysis will reveal the first genetic landscape of liver mets. Ø We are intensively characterising the immune landscape of mets in order to evaluate the potential for immunotherapies. Ø The European patient network MPNE Ocular/Rare already counts 214 members from 18 countries.

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MPNErare2017, 6th- 8th- October , Leiden

EURACAN

EURACAN aims to establish a world-leading, patient-centric and sustainable network of multidisciplinary researchintensive clinical centres focused on RARE ADULT CANCERS (RACs).

EURACAN gathers 66 Health Care Providers in 17 European countries, and 22 Associate partners (PAGs, rare disease stakeholders).

MPNErare2017, 6th- 8th- October , Leiden

Melanoma Patient Network Europe

RARE SOLID ADULT CANCERS MPNErare2017, 6th- 8th- October , Leiden

DISTRIBUTION OF MEMBERS BY COUNTRY COUNTRIES/Towns BELGIUM (Antwerp, Brussels, Leuven, Liège)

CZECH REPUBLIC (Brno, Prague) DENMARK (Aarhus) GERMANY (Berlin, Essen, Mannheim, Hamburg-Eppendorf, Marburg, Würzburg)

FINLAND (Turku) FRANCE (Lyon, Paris, Villejuif) HUNGARY (Budapest) ITALY (Aviano, Bologna, Candiolo, Firenze, Genoa, Meldola, Milan, Naples, Rome, Siena, Torino, Treviso)

LITHUANIA (Kaunas) NETHERLANDS (Amsterdam, Leiden, Maastricht, Njimegen, Rotterdam, Gronigen)

NORWAY (Oslo) POLAND (Warsaw) PORTUGAL (Coimbra, Lisboa, Porto) SPAIN (Sevilla, Barcelona) SWEDEN (Karolinska, Uppsala) SLOVENIA (Ljubljana) UNITED KINGDOM (Coventry, London, Oxford, Sheffield)

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OBJECTIVES At 5 years Objective 1: To increase and facilitate the access of RAC patients to expert centers/disease information/treatment options and better and safer healthcare Objective 2: To ensure optimal diagnosis & treatment options and delivery in a timely manner (including access to latest diagnostic and therapeutic innovation) Objective 3: To promote optimized quality and care of RAC patients Objective 4: To secure EURACAN sustainability

MPNErare2017, 6th- 8th- October , Leiden

TARGETED RACs

Rare adult solid cancers are grouped in 10 domains corresponding to the RARECARE classification and the ICD10.

These domains are also based on pre-existing successful collaborations, in particular for clinical research and expert networks active in the last 10-20 years

MPNErare2017, 6th- 8th- October , Leiden

The International Rare Cancer Initiative (IRCI) • • •

22% of new cancers diagnosed annually are rare cancers; 4 300 000 patients are living today in the European Union with a diagnosis of a rare cancer The outcome for patients with a rare cancer is inferior to those with more common tumors Joint initiative (2011)

-

Cancer Research UK (CRUK) National Institute of Health Research Clinical Research Network: Cancer (NIHR CRN: Cancer) National Cancer Institute (NCI) European Organisation for research and Treatment of Cancer (EORTC) Institut National Du Cancer (INCa) National Cancer Institute of Canada Clinical Trials Group (NCIC CTG).

• •

AIM: facilitate the development of clinical trials for patients with rare cancers 11 groups, including a Rare Melanoma Group

MPNErare2017, 6th- 8th- October , Leiden

MPNErare2017, 6th- 8th- October , Leiden

MPNErare2017, 6th- 8th- October , Leiden

Background:

THANK YOU !

sophie.piperno-neumann@curie.fr

MPNErare2017, 6th- 8th- October , Leiden

4.2 Fast access, better data.

MAPPs- Medicinesâ&#x20AC;&#x2122; Adaptive Pathways to patients Bettina Ryll, MD/ PhD MPNE

MPNErare2017, 6th- 8th- October , Leiden

why care? a few comments why regulatory processes are important for ALL Melanoma patients

MPNErare2017, 6th- 8th- October , Leiden

The 4 years that made all the difference in Melanoma OS 2yr 15%

OS 2yr 45%

OS 2yr 59%

OS 2yr 64%

IMMUNO-THERAPIES

slide modified after G. Spurrier

MPNErare2017, 6th- 8th- October , Leiden

For several years, promising therapies were only accessible in clinical trials This is what one of our Melanoma patients said after being randomized to DTIC versus PD1:

Loriâ&#x20AC;&#x2122;s experience on a clinical trial https://www.youtube.com/watch?v=H03vz24JhgM The trials we want https://youtu.be/wilTXvFN2NU

MPNErare2017, 6th- 8th- October , Leiden

WMA Helsinki Declaration Ethical Principles for Medical Research involving Human Subjects- 1964, last amendment 2013

3. The declaration of Geneva of the WMA binds the physicians with the words ‘The health of my patient will be my first consideration’ and the International Code of Medical Ethics declares that, ‘A physician shall act in the patient’s best interest when providing medical care’. …

8. While the primary purpose of medical research is to generate new knowledge, this goal can never take precedence over the rights and interests of individual research subjects. ….

26. …The potential subject must be informed of the right to refuse to participate in the study or to withdraw consent to participate at any time without reprisal. ….

full version: http://www.wma.net/en/30publications/10policies/b3/

MPNErare2017, 6th- 8th- October , Leiden

Need to reduce (unavoidable) uncertainties – fast

‘Access vs evidence’: an ethical and scientific conundrum

Adaptive Pathways a solution to inevitable problems?

Sustainability of costs slide courtesy by F. Pignatti

Development of nonconventional products (e.g. ATMPs)

Need to enlarge the toolbox for evidence generation

(where RCTs cannot answer the questions) th

MPNErare2017, 6 - 8th- October , Leiden

Adaptive Pathways - component parts

Focus on high unmet need (sub-)population first, and on products likely to have major impact for patients Reduce uncertainty as fast as possible; react to incoming data (iterative development; rapid cycle analysis)

Leverage multi-stakeholder collaboration Manage on-market utilisation slide courtesy by F. Pignatti

Pre-plan, across entire life span (incl. postmarketing) Use entire tool box for knowledge generation MPNErare2017, 6th- 8th- October , Leiden

MAPPsMedicine’s Adaptive Pathways to Patients

Advantages for patients: • •

early access to innovative therapies continuous evaluation and systematic learning -‘no evidence wasted’ = faster progress

Source: H.G. Eichler http://www.ema.europa.eu/docs/en_GB/document_library/Prese ntation/2012/04/WC500124930.pdf MPNErare2017, 6th- 8th- October , Leiden

Current scenario:

wasted opportunity

Post-licensing, treatment population grows rapidly; treatment experience does not contribute to evidence generation MAPPs:

our cure

wasted opportunity

original slide courtesy F. Pignatti

after initial license, number of treated patients grows more slowly, due to restrictions; patient experience is captured to contribute to real-world Eichler HG et al. Clin Pharmacol Ther. 2012 information

MPNErare2017, 6th- 8th9 - October , Leiden

From invention to healthcare innovation.

Accessing innovative medicines in Europe.

patient access

product

decision level

mode of access

Early Access Programs

clinical trials

impacted by

attractiveness of regional market

black hole

attractiveness of national market

structural properties and capacities

MPNE, B. Ryll, 05- 2017

• • • •

•

Unrestricted access

•

Restricted access

cost-effectiveness budget impact local preferences political will

• • • • •

systematic, e.g. MEAs arbitrary

centralised vs de-centralised healthcare system budget holder centres of excellence/ certification Luck guidelines educational level of oncologists

MPNErare2017, 6th- 8th- October , Leiden

“Well, if that plane was heading towards a cliff, then yes, I would”.

The risk of not taking risks when you are diagnosed with a life-threatening disease.

And there is no such a thing as ‘the’ patient preference.

MPNErare2017, 6th- 8th- October , Leiden

MPNE2015 documentary www.youtube.com/watch?v=VIreDdQG4kc

“Would you jump out of a plane if you knew that there was a 1 in 10 chance that your parachute would not open and you would die?”

quote from a patient workshop, kindly provided by M. Longley, WIHSC

a comment on risk

Summary • •

1. Patients in desperate situations need ACCESS AND ONGOING LEARNING 2. No interest can take precedence over the interests of the single research individual’s interest

•

3. Regulatory approval is just the first step towards access. Clinical trials need to anticipate also later needs for evidence- HTA bodies and payers.

•

4. What is risky depends on your situation and it is ultimately patients who risk their lives on clinical trials and who die because of lack of access

•

5. We need new ways to gather evidence as the old models are no longer fit for purpose.

MPNErare2017, 6th- 8th- October , Leiden

Thank you www.melanomapatientnetworkEU.org

MPNErare2017, 6th- 8th- October , Leiden

Interacting with your national HTA body ERIC LOW

FORMER CEO MYELOMA UK INDEPENDENT HEALTH CARE CONSULTANT / THE AMYLOIDOSIS RESEARCH CONSORTIUM ERIC@ERICLOWCONSULTING.COM ELOW@ARCI.ORG @ERICLOW71

MPNErare2017, 6th- 8th- October , Leiden

Content - What health technology appraisal is - Where it fits in the drug discovery, development approval continuum - What are the issues - Get involved - Summary

MPNErare2017, 6th- 8th- October , Leiden

What health technology appraisal is Some countries have specific â&#x20AC;&#x153;health technology assessmentâ&#x20AC;? (HTA) bodies which make decisions on new medicines HTA is a formal health economic process which looks at whether or not a drug is/how valuable to a health service â&#x20AC;&#x201C; there are a range of different formula used to calculate the value (e.g. QALY) Other countries have bodies that conduct a less formal assessment of a new drug, however, still looking at the cost-benefit of a new medicine Different ways of engaging patients

MPNErare2017, 6th- 8th- October , Leiden

Which medicines to approve Key questions: • How well does the medicine work?

• Which patients will benefit from it?

• Is it equal to or better than medicines the NHS already uses to treat the particular condition?

• Is it good value for money?

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Where it fits in the system â&#x20AC;¢

Source: Geissler, Ryll, Leto, Uhlenhopp EPALCO/EUPATI (2016)

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Helping to address tensions in the system Limited scarce resources

Unlimited wants and needs

The economic problem is to match limited resources to unlimited wants and needsâ&#x20AC;Ś MPNErare2017, 6th- 8th- October , Leiden

What are the issues

MPNErare2017, 6th- 8th- October , Leiden

Get involved • Influence research • Find out about methods and processes used • Ask about patient and public involvement • Horizon scan • Engage early • Represent the patient perspective • Be the honest broker MPNErare2017, 6th- 8th- October , Leiden

SMC Process

MPNErare2017, 6th- 8th- October , Leiden

Case study • First novel treatment in myeloma • Received its European licence in 2004 as a monotherapy in relapsed myeloma patients • Myeloma patients in England and Wales did not get access to Velcade until 2007 • Major problem with the trial design and high price, so NICE issued negative guidance • Risk management scheme negotiated MPNErare2017, 6th- 8th- October , Leiden

Summary 1. HTA has an important role to play and is here to stay 2. Important to think about influence research to ensure better HTA inputs 3. Horizon scan and engage early 4. Be an honest broker 5. Important to work on access more broadly i.e. named patient programmes 6. Lots of resources out there that MPNE can give you

MPNErare2017, 6th- 8th- October , Leiden

MPNE LEAN PATIENT REGISTRY EFFORTS MPNE Andrew Evans

MPNErare2017, 6th- 8th- October , Leiden

THERE IS POWER IN NUMBERS WHEN WE HAVE NUMBERS, WE HAVE A VOICE MPNErare2017, 6th- 8th- October , Leiden

PATIENT REGISTRIES ARE A COMPLEX TOPIC

IAIN COVERED THIS AT AN MPNE MEETING LAST YEAR AND IT WAS A WHOLE SESSION TO ITSELF THERE ARE MANY PURPOSES FOR REGISTRIES AND IMPORTANT CONSIDERATIONS IF THE DATA WILL BE USED FOR RESEARCH, ETC.

MPNErare2017, 6th- 8th- October , Leiden

BUT WE DON’T NEED A FULLY-REALIZED REGISTRY TO GAIN LEVERAGE FOR PATIENTS WE JUST NEED PATIENTS TO START RAISING THEIR HANDS AND SAYING “I’M HERE AND I WANT TO PARTICIPATE”

MPNErare2017, 6th- 8th- October , Leiden

USING LEAN STARTUP PRINCIPLES, WE CAN CREATE VALUE FOR THE PATIENT COMMUNITY CHEAPLY AND QUICKLY

THE REST CAN FOLLOW WE CAN ITERATE AND IMPROVE, AND FEED INTO BIGGER REGISTRY EFFORTS LATER - CAN MOBILIZE THESE PATIENTS TO JOIN THOSE AT THE LAST MPNE MEETING IN KREUSENBERG, WE DID A LEAN EXPERIMENT USING SOCIAL MEDIA AND MAILCHIMP - WE BUILT A

MPNErare2017, 6th- 8th- October , Leiden

THANK YOU!

MPNErare2017, 6th- 8th- October , Leiden