Children's Healthcare - Q4 2022

Children’s Healthcare Q4 2022 | A promotional supplement distributed on behalf of Mediaplanet, which takes sole responsibility for its content

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“Children continue to struggle in getting an accurate and timely diagnosis and in accessing health and social services.”

“They hope to remove the difficulty for young children who have already gone through gruelling treatments .”

Flaminia Macchi and Dolores Cvitičanin, Rare Diseases International

Christiana Ogunbote, Head of Research, Children with Cancer UK

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IN THIS ISSUE

“Around 70% of rare diseases have a childhood onset.”

María Cavaller Bellaubi Gulcin Gumus EURODIS

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Ensuring children with a rare disease are no longer left behind There are over 300 million people living with a rare disease worldwide. Many rare diseases are present at birth, and an estimated 70% of rare genetic conditions start in childhood, making children a significant proportion of the global community of people living with a rare disease.

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are diseases are often chronic, progressive, life-threatening, and highly debilitating. Their impact is felt throughout a lifetime and in every aspect of life by both children with a rare disease themselves and their families.

“25 million infants missed out on basic routine vaccines in 2021 alone.”

Laetitia Bigger Director Vaccines Policy, IFPMA

Barriers children face The adoption of a UN General Assembly Resolution on ‘Addressing the Challenges of Persons Living with a Rare Disease and their Families’ on 16 December 2021 improved the visibility and recognition of the specific challenges faced by children and families impacted by rare diseases.

The United Nations and its Member States recognised that children living with a rare disease encounter barriers to their education and multiple forms of discrimination, stigma and exclusion.

Page 06 When entering the school system, children with a rare disease often deal with a lack of awareness of rare diseases.

“Take your child to the dentist as soon as the first tooth appears.”

Dr Nigel Carter OBE Chief Executive, The Oral Health Foundation

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Access to education and equal opportunities The global rare disease community encourages policymakers to tackle rare diseases, not solely as a health priority but also as a social justice issue that affects children’s access to education — which in turn leads to lifelong negative consequences.

When entering the school system, children with a rare disease often deal with a lack of awareness of rare diseases.

They navigate schooling facilities and programmes that are not adapted to welcome learners with uncommon health conditions.

As a global alliance, RDI increasingly engages UN agencies, including the United Nations Educational, Scientific and Cultural Organization (UNESCO) and the Office of the High Commissioner for Human Rights (OHCHR) to address the social challenges and barriers linked to going through life with a rare disease.

This year, on UHC Day (12 December), RDI and the OHCHR will host a public event calling for national measures to strengthen health systems for all people living with a rare disease and ensure no child is left behind.

The right to health for all The UN Convention on the Rights of the Child affirms that access to health is a fundamental right — not a privilege. No child should be deprived of the highest attainable standard of physical and mental health, regardless of where they live.

However, due to a lack of awareness, knowledge and expertise on rare diseases, children continue to struggle in getting an accurate and timely diagnosis and in accessing health and social services. In addition, families are at greater risk of impoverishment due to significant out-of-pocket expenses, disruption of work and reduction in income.

Rare Disease International (RDI) is working with civil society groups across the globe to advocate for the World Health Organisation (WHO) to adopt a resolution on Universal Health Coverage (UHC) for all people living with rare diseases.

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WRITTEN BY Flaminia Macchia Executive Director, Rare Diseases International (RDI)

WRITTEN BY Dolores Cvitičanin Public Affairs Manager, Rare Diseases International (RDI)

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How the life sciences industry can improve the lives of rare disease patients Access to rare disease medicines in the UK is slower than in comparable EU countries. But there are opportunities to transform this environment¹ — and the lives of patients.

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ou can’t talk about children’s health without discussing the field of rare diseases, explains Ben Whitehouse, Head of Rare Diseases UK at global healthcare company, Sanofi. That’s because 75% of rare diseases affect children — 30% of whom, tragically, will die before their fifth birthday.²

INTERVIEW WITH Ben Whitehouse Head of Rare Diseases, Sanofi

WRITTEN BY Tony Greenway

Limited resources and treatment It’s also impossible to talk about rare disease science without looking at the broader UK life sciences industry. This, currently, is in a perilous position.

“At present, UK life sciences faces enormous challenges,” says Whitehouse. “Unfortunately, these challenges impact patients of every kind.”

The first challenge Whitehouse notes is specific to the area of rare diseases, and that’s the lack of treatments as 95% of rare diseases have no licensed treatment available,³ which is worrying when you consider that science in the UK is declining across the industry. “Since 2017, the UK has seen a 41% reduction of patients in clinical trials,”4 he admits. “Reasons include diversity and limited understanding of each disease, difficulties identifying and treating small populations, plus a decrease in the Government’s R&D reimbursement incentives over time.”

Only 61% of orphan medicines approved in the EU between 2017–2020 are reimbursed for use in England, compared to over 70% in France and Italy and over 95% in Germany.

Document No: MAT-XU-2205445 (V1.0) Date of Prep: December 2022

Paid for by Sanofi

Issues around investment, access and reimbursement More widely, there is the problem of investment.

Most major developed countries average around 15% of their healthcare spend on drugs. However, the UK’s investment only averages around 9%.5 Then there’s the issue of the Voluntary Scheme for Branded Medicines Pricing and Access (VPAS) — a repayment mechanism requiring companies to pay back a percentage of sales of most branded medicines to the Government. Before Covid-19, the yearly payment percentage was around 5%–10%. Next year, however, this will soar up to 30% and have a bruising impact on the UK life sciences industry — particularly pharma companies.6 Whitehouse welcomes the Government’s UK Rare Diseases Framework with its commitment to improving the lives of those living with rare diseases and the Innovative Medicines Fund designed to enable patients to benefit from early access to promising, new medicines. Yet, he points out that there are big challenges around reimbursement — the process where bodies such as The National Institute

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for Health and Clinical Excellence (NICE) assess if a drug is cost-effective enough to go to market.

Only 61% of orphan medicines approved in the EU between 2017–2020 are reimbursed for use in England, compared to over 70% in France and Italy and over 95% in Germany.7 Access in the UK is also lower and slower. For new medicines licensed and launched between 2015–2019, UK uptake was just over half (56%) of comparable countries, rising to only 75% after four years.¹ UK to be a world leader in innovation and health outcomes Whitehouse is optimistic that — with good stakeholder collaboration — this bleak picture can change. “The UK is home to great scientists, physicians and academics,” he notes. “We have world-class treatment centres and some of the most passionate and informed patient groups. We have business units dedicated to rare diseases, and we’re prepared to invest — and, indeed, are investing — in areas of the science where there are no therapies at present. So, UK life sciences has all the ingredients to be a success, but we need to call out the challenges it faces and then work together to solve them. That includes transforming certain policy levers, building on reforms to the regulatory and access environment and creating a pricing and reimbursement system that incentivises innovation in healthcare. After all, treating people — however rare their disease — is part of what we stand for as a company and society.”

References: 1. Life Science Competitiveness Indicators 2021 (publishing.service.gov.uk) 2. https://cypmedtech.nihr.ac.uk/rare-diseases/ 3. https://www.efpia.eu/about-medicines/development-of-medicines/ intellectual-property/help-us-make-rare-disease-even-rarer/ 4. https://www.bmj.com/content/379/bmj.o2540 5. https://www.iqvia.com/insights/the-iqvia-institute/reports/drug-expendituredynamics 6. https://www.britishgenerics.co.uk/uploads/BGMA-OHE-synopsis-reportfinal-oct.pdf 7. https://www.efpia.eu/media/676539/efpia-patient-wait-indicator_updatejuly-2022_final.pdf

Find out more at sanofi.com

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Why you shouldn’t just correct your child’s short-sightedness with glasses A new parent-focused portal is launched to increase awareness of childhood myopia, what it really is and what you should be doing about it.

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yopia (short-sightedness) affects one in three people in the UK; however, it is now twice as common as in the 1960s. Of the 5 billion people globally predicted to be myopic by 2050, 1 billion will have ‘high myopia,’ which can cause serious eye problems later in life including complications that could lead to blindness. The latest research highlights an association between screen time and the progression of myopia, with optometrists seeing a sharp rise in the number of children struggling to see the board at school.

Raising awareness among parents The crisis has led Optometrist Jason Higginbotham to launch MyopiaFocus.org, a parent-focused education portal to raise awareness of the myopia epidemic, highlight the risks and treatment options and, ultimately, connect parents with local myopia management specialists.

Higginbotham says, “I’ve launched this campaign because I am concerned at the lack of understanding and support for what is going on with children’s eyes right now.

Increased use of screens, close work and lack of outdoor time have been proven to have led to higher rates of myopia.”

Long-term management “For too long now, myopia has just been dealt with by prescribing glasses. The problem is, this does nothing to control the increased risks of eye health problems associated with ‘axial-length myopia’ — caused when children’s eyeballs grow too long — and is known to account for the majority of all myopia. The good news is there are myopia management treatments available — unfortunately, not enough parents know about them,” Higginbotham continues.

“We want to highlight the urgent need for proactive myopia management. A personalised care plan can significantly decrease the progression of myopia in children by as much as 50%. As people are unaware that eye tests in schools are limited — unless parents act and have their child’s eyes tested — myopia may go undetected.”

He concludes: “If you or your child are myopic, visit your local optician and ask them about myopia management for your child.”

Myopia Focus has launched a change.org campaign to get the NHS to recognise myopia as an ocular disease and improve myopia management funding. Scan the QR code to join the campaign – sign the petition.

INTERVIEW WITH

Jason Higginbotham, BSc (Hons) MCOptom Optometrist & Managing Editor, Myopia Focus

Ensuring paediatric medicines are tailored to young people and children Patient engagement is key. Young patients and their families involved in paediatric drug development contribute to faster and more efficient medicine development.

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atient engagement in the process of drug development can lead to active collaboration between developers, researchers, clinicians and patients who come together and share their knowledge and expertise throughout a medicine’s lifecycle. This stretches from driving research to co-creating regulatory protocols for health technology assessments and cost reimbursements.

Consider children in medicine development Unfortunately, only a few patients are engaged in medicine development due to a lack of collaboration between patients and developers and geographical barriers, among other reasons. The reality is more dramatic when it comes to paediatric medicines, even though around 70% of rare diseases have a childhood onset.

Today, many medicines prescribed to children are being used ‘off-label,’ which means they may have not been tested specifically on children. This is mostly because the administration of drugs in the paediatric population is commonly and erroneously assumed.

Most medicines exhibit different pharmacokinetics in children compared to adults, so we must ensure that the medicines developed are right for them.

Clinical trials for young people It wasn’t until the European Union’s Paediatric Regulation came into force in 2007 that clinical trials with children started to become more widespread. But there’s still work to be done.

The research infrastructure supporting the delivery of large clinical trials hasn’t been developed equally across Europe. Clinical trials are often delayed, and the rollout of medicines approved for children is painstakingly slow.

Across Europe, 35 academic and 10 industry partners — including EURORDIS-Rare Diseases Europe — have joined forces and created the panEuropean network ‘Conect4Children’ (C4C) to accelerate the development of innovative medicines for the entire paediatric population. EURORDIS aims to amplify the voices of children and young people living with a rare disease and bring their needs to the attention of researchers, clinicians and developers.

As part of this project, we have discussed the establishment of a framework for clinical trials. We have also launched educational videos for patients and parents about paediatric clinical research, and we have organised events empowering patient organisations.

Address young patients’ needs Through C4C and other initiatives, we aim to create a common framework that enables structured, effective, meaningful, ethical and sustainable patient engagement — specifically for young patients, similar to that which already exists for adults.

Only by engaging the young population in medicines development can we safely provide them with treatments based on actual evidence, which address their specific needs.

WRITTEN BY

Richard Kadri-Langford

Paid for by Myopia Focus

WRITTEN BY María Cavaller Bellaubi Patient Engagement & Therapeutic Development Senior Manager, EURODIS

WRITTEN BY Gulcin Gumus Research and Policy Project Manager, EURODIS

Find out more at myopiafocus.org

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Thousands of UK children and young people diagnosed with cancer every year

toxic treatments. Pioneering research, funded by Children with Cancer UK, demonstrated that by genetically modifying immune cells and injecting them back into the immune system, children were in improved clinical remission in comparison to chemotherapy treatment.

Today, experts are improving on this immunotherapy discovery. They hope to remove the difficulty for young children who have already gone through gruelling treatments by offering a less harsh process. They hope to be ready for clinical trials in three years.

In the UK, 12 children and young people will be diagnosed with cancer every day with two of those young patients not surviving.

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WRITTEN BY Christiana Ogunbote Head of Research, Children with Cancer UK

ancer is caused by genetic changes within a cell that divides and causes uncontrollable growth.

Symptoms of childhood cancer can present in a variety of ways with the most common being sudden changes to vision, excessive bruising, headaches with early morning sickness and severe pain in joints.

There are 12 main diagnostic groups, with leukaemia accounting for 30% of diagnoses, brain and spinal tumours for 20% and lymphomas for 11%.

Childhood cancer vs adult cancer Treatment options for adults and children, at times, share similarities.

With some exceptions, children tend to respond better because it can be more intense, and they generally

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have fewer pre-existing health issues.

However, due to their growing bodies, it can be expected that two in three childhood cancer survivors will go on to experience long-term side effects from treatment such as heart failure, mobility issues, deafness, growth issues and infertility. Only 2% of national cancer research funding is spent on children.

Treatment innovations: CAR-T Chimeric antigen receptor T-cell therapy (CAR-T) — a form of immunotherapy — is a relatively new treatment which has revolutionised care for children. As a targeted therapy, immunotherapy teaches the immune system to recognise and fight cancerous cells by itself, reducing the need for exposure to

Only 2% of national cancer research funding is spent on children.

Looking forwards Fifty years ago, 75% of children with cancer died; today, more than 84% of children diagnosed with cancer survive their disease for five years or more.

Unfortunately, cancer remains one of the leading causes of death for under-15s which is why researchers and charities continue to work for higher survival rates, identification of genetic causes and limiting the number of long-term side effects for young patients.

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Ultra-rare genetic disease often confused with cancer in children FOP turns patients’ muscles and tendons into bone and is often misdiagnosed. Know the signs and how to get more information on this rare, disabling condition.

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ibrodysplasia ossificans progressiva (FOP) is one of the rarest, most disabling conditions known to medicine. With only 1,000 confirmed cases worldwide, the disease exhibits no ethnic, racial or gender patterns. FOP causes bone to form in muscles, tendons and connective tissues. Eventually, these bridges of bone restrict movement and form a second skeleton, imprisoning the body.

Signs of FOP for clinical diagnosis Telltale signs of FOP include: • Bilateral deformity of the great toes (and thumbs in some individuals) • Soft tissue swelling • Progressive heterotopic ossification (HO) The exact rate of progression for FOP is unpredictable and varies from person to person. However, it appears symptoms in the upper body are more prevalent in childhood.

Lower body symptoms tend to present during adolescence.

While the initial diagnosis is clinical, a definitive FOP diagnosis requires genetic confirmation of the ACVR1 gene mutation.

It’s important to note FOP is accelerated by trauma. This includes intramuscular injections and other procedures. If FOP is suspected, all elective procedures such as surgeries, biopsies and immunisations should be deferred. Patients should be referred to genetics and/or an FOP expert.

Treating FOP patients: supporting families FOP patients need a primary physician who is willing to collaborate with FOP experts to assemble and coordinate a local care team. The International FOP Association (IFOPA) was formed in 1988 by a woman with FOP and can assist healthcare providers.

The IFOPA also offers healthcare professionals access to FOP Treatment Guidelines and Covid-19 resources essential for FOP patients who often have restricted lung capacity.

International network of FOP expertise The International Clinical Council on FOP (ICC) is an autonomous and independent group of 21 internationally recognised physicians who are clinical experts in FOP from 14 nations and six continents. The ICC was established to coordinate and consolidate a global voice for the best practices for clinical care and clinical research for people who suffer from FOP.

WRITTEN BY Michelle Davis Executive Director, IFOPA

Paid for by IFOPA You can connect with the ICC and other helpful resources explicitly for healthcare providers caring for patients with FOP at ifopa.org/for_medical_professionals

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Improving child health around the world through immunisation Improving and expanding childhood vaccination against routine and new diseases is vital for our children’s health and global public health.

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ver since the smallpox vaccine discovery over two centuries ago, vaccines have successfully worked to eradicate, eliminate or control many infectious diseases.

immunisations back on track to avoid unnecessary outbreaks of debilitating diseases like measles, polio and pertussis. Furthermore, we need to raise awareness of the benefits of maternal immunisation against pertussis, tetanus or influenza, which can Red alert for child health provide early protection — not only for pregnant However, in the wake of the Covid-19 pandemic, women but also their newborns during the vulnerable global health organisations have sounded a ‘red first hours and weeks of their lives. alert’ for child health. Childhood vaccination rates New and improved vaccines against viruses like around the world have dropped for the first time in RSV (respiratory syncytial virus) — a leading cause of 30 years, threatening decades of progress for child infant hospitalisation and the most common cause health. Worryingly, 25 million infants missed out of bronchiolitis and pneumonia in infants — along on basic routine vaccines in 2021 with vaccines against malaria, alone, and most are ‘zero-dose pneumonia and diarrhoea-causing children’ who did not receive a diseases can also improve the vital first vaccine dose against health and wellbeing of children We must urgently diphtheria, tetanus and pertussis. and their families.

get routine childhood

Teenage HPV vaccine rates drop Lessons learned immunisations back on Another fallout from the Covid-19 The Covid-19 pandemic showed pandemic is the decrease in human track to avoid unnecessary us that when we work together, papillomavirus (HPV) vaccination we can achieve amazing things. outbreaks of debilitating rates which protects teenage girls By continuing to invest in vaccine against cervical cancer. As a result, diseases like measles, innovation while working with in over 100 countries that have global health partners to ensure polio and pertussis. introduced HPV vaccination to routine immunisation programmes date, approximately 1.6 million get back on track, encourage girls missed out in 2020. Globally, only 13% of girls life course immunisation and equitable access were vaccinated against HPV in 2020, falling from to vaccines, we can optimise the lifesaving and 15% in 2019. life-enhancing power of immunisation and protect the future of millions of children and their families.

Improving child health Significant progress has been made in child health and child survival rates over the past three decades WRITTEN BY with the global under-five mortality rate decreasing Laetitia Bigger by more than 60%. Childhood vaccination can Director Vaccines nurture our success in preventing infectious Policy, IFPMA diseases we now consider rare and controlled.

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How to take care of your children’s teeth It’s easy to feel overwhelmed when caring for your child’s teeth. The good news is that a healthy smile is achievable for every child by following just a few, simple steps.

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hether it’s their first tooth or first trip to the dentist, a child’s early experiences of looking after their mouth can be with them for the rest of their life. Giving them a great start to life, as far as their teeth are concerned, is relatively simple.

WRITTEN BY Dr Nigel Carter OBE Chief Executive, The Oral Health Foundation

Brushing a child’s teeth Brushing is one of the most important skills a child can learn. In their early years, they will need a helping hand to clean their little gnashers. When the first teeth start to come through, use a children’s toothbrush with a small smear of children’s fluoride toothpaste. The amount of fluoride it should contain is 1,000 ppm for under-3s and up to 1,350– 1,500 ppm for over-3s.

Once all the teeth have come through, use a small-headed, soft toothbrush in small, circular movements; and try to concentrate on one section at a time. Make sure to brush the inside and outside of each tooth, as well as the biting surfaces — and don’t forget the gum line. Cleaning should become part of their daily routine and be done twice a day.

Dental visits Take your child to the dentist as soon as the first tooth

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appears. Baby teeth are still important as they can impact how well their adult teeth come in. Trips to the dentist from an early age help the child get used to the sights, sounds and smells of the dental practice.

The earlier and more regularly you take them to the dentist, the less likely they will have anxiety or fear of the dentist as they grow older.

Healthy, balanced diet Nutrition plays a critical role in how a child grows and develops — this includes the health of their mouth. During snack time, get them used to having pieces of fresh fruit and raw vegetables. Any sweet foods should be limited and only allowed at mealtimes.

Still water and milk are the best drinks for any of us, especially good for young and developing bodies. Sugary drinks should be avoided, including fruit juices. Parents must also avoid getting into the habit of giving children sweets to reward them for good behaviour. The long-term effect of frequent sugar consumption can be very damaging to a child’s oral health, so help them avoid it as often as possible.

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In over 100 countries that have introduced HPV vaccination to date, approximately 1.6 million girls missed out in 2020. Globally, only 13% of girls were vaccinated against HPV in 2020, falling from 15% in 2019.

Laetitia Bigger, Director Vaccines Policy, IFPMA

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