Government Response

The Government responded to this petition on Tuesday 12th October 2021

The Government recognises the challenges faced by people affected by rare disease, and funds research into rare conditions – including fibrodysplasia ossificans progressiva – via NIHR and UKRI.

Although rare diseases are individually rare, they are collectively common, affecting 1 in 17 people at some point in their lifetime. The Government recognises the challenges faced by people affected by rare diseases, including ultra-rare conditions such as fibrodysplasia ossificans progressiva (FOP).

In January 2020, the UK Government published the UK Rare Diseases Framework, outlining our vision to improve the lives of those living with rare diseases over the next five years. The Framework describes four key priorities: helping patients get a final diagnosis faster; increasing awareness of rare diseases among healthcare professionals; better coordination of care; and improving access to specialist care, treatments, and drugs. The Framework additionally describes five underpinning themes: patient voice; national and international collaboration; digital, data, and technology; wider policy alignment; and pioneering research.

While the Framework describes our ambitions for rare disease, our forthcoming Action Plan will ensure delivery. Each of the four UK nations have committed to publishing an Action Plan within two years of the Framework’s publication. England’s Action Plan describes work underway across the Department of Health and Social Care (DHSC) and our arm’s-length bodies to address the challenges posed by rare conditions and is expected in early 2022.

Scientific advancements have underpinned many breakthroughs in rare disease. The Government’s 2021 UK Research and Development Roadmap sets out the UK’s vision and ambition for science, innovation, and research.

Alongside other charities and organisations, the Government primarily funds research into rare diseases such as FOP via the National Institute for Health Research (NIHR) and UK Research and Innovation (UKRI).

NIHR is the nation’s largest funder of health and care research, spending £1 billion from DHSC on research every year. In the past five years, the Institute has supported one study into FOP via NIHR Infrastructure and seven studies via NIHR Clinical Research Networks. Bringing together the seven disciplinary research councils, including the Medical Research Council (MRC), UKRI convenes, catalyses, and invests in close collaboration with others to build a thriving, inclusive research and innovation system. Since 2010, MRC have contributed funding towards three projects of underpinning relevance to FOP and the biological processes that contribute towards the condition, totalling over £6.6 million. More broadly, UKRI holds a diverse and dynamic portfolio of research directed towards musculoskeletal health and disease more generally. Findings from this broader pool of projects could stimulate future activity and discovery in FOP research.

NIHR welcomes funding applications for research into any aspect of human health, including FOP. Research proposals in all areas compete for the funding available. These applications are subject to peer review and judged in open competition, with awards being made on the basis of the importance of the topic to patients and health and care services, value for money, and scientific quality. Within NIHR, for all disease areas the amount of funding depends on the volume and quality of scientific activity. The usual practice of NIHR is not to ring-fence funds for expenditure on particular topics. In addition, recognising the impact of rare diseases such as FOP, the MRC Population and Systems Medicine Board supports the UK Rare Diseases Framework by encouraging the scientific community to come forward with their best ideas aimed at addressing research questions related to rare conditions.

Outside of research, the National Institute for Health and Care Excellence (NICE) is in the early stages of developing highly specialised technologies guidance (which make recommendations on the use of new and existing highly specialised medicines and treatments within the NHS in England) on ‘palovarotene for preventing heterotopic ossification associated with FOP (ID3739)’. NICE aims to align publication of its highly specialised technologies guidance as closely as possible with a product receiving UK regulatory approval to ensure patients in the NHS can access new medicines and treatments in a timely manner. NICE cannot publish final guidance until this has been granted. For further information, including timelines for development, please visit www.nice.org.uk/.

Department of Health and Social Care

Parliamentary Debate

This petition was debated in Parliament on Monday 6th December 2021

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