UK urged to enhance access to rare disease treatment

Report champions international collaboration and best practices to boost patients' access

A new report highlights the UK's potential to lead the global effort in enhancing patient access to new treatment for rare diseases. Getty Images

The UK trails behind Germany and France in providing patients with access to approved rare disease treatments, a report has revealed.

The findings call for the UK to spearhead greater international co-operation to enhance the availability of critical medicine.

The report, backed by the Bio Industry Association (BIA), was conducted with research from global consultancy firm PwC.

It found a comparative lag in the UK in the availability of treatment for rare diseases, with only 59 per cent of rare disease drugs approved by the European Medicines Agency (EMA) being dished out in England between 2018-2021, behind Germany's 86 per cent and France's 77 per cent

The UK has recently announced projects to enhance collaborative efforts, highlighted by the new International Recognition Procedure.

The report identifies successful international processes and schemes that could inform future practice in the UK.

Steve Bates, chief executive of the BIA, pointed out the UK's potential in leading the global effort: “The UK has an incredibly strong rare disease community.

“By working collaboratively to build on the strong foundations this community has developed, the UK has an opportunity to demonstrate international leadership in improving the lives of people affected by rare diseases.”

Stephen Aherne, pharmaceuticals and life sciences leader at PwC UK, noted the importance of sustained efforts in this area: “Many rare disease patients remain underserved with respect to gaining timely access to life-changing and life-saving medicines.

“While significant progress has been made in recent years, it’s important that this momentum is maintained as stakeholders continue to collaborate to address remaining challenges.”

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  A group of mothers with their children wait outside Middlesex County Council Clinic for the first polio vaccinations in May 1956. All photos: Getty Images

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  A polio vaccine being tested at the Glaxo Laboratories in Sefton Park, Buckinghamshire, in 1956.

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  A child is injected with a vaccine against polio in 1956, the first stage of the biggest vaccination programme ever undertaken in Britain at the time.

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  Four young children wait for injections against polio at the Fairfield Welfare Clinic in Wandsworth, south London, in 1956.

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  Health minister Dennis Vosper examining the polio virus in 1957, during the production of Polivirin, Britain's anti-poliomyletis vaccine, at Glaxo's Virus Research Laboratories in Stoke Poges.

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  A queue of factory workers waiting to receive polio vaccinations in one of the new London Mobile Immunisation Clinics at Highbury Grove in 1959.

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  Junior members of staff at the Ministry of Health on Saville Row, London, line up to receive their inoculation against polio in 1959.

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  Nurses in one of the many emergency polio vaccination clinics in Hull read the latest reports on the health scare in 1961.

The report provides an evidence base to inform future reforms that could ameliorate the situation in the UK.

Despite the progress in treatment access over the recent years, the report notes that substantial challenges still impede reaching the goals set out in the Rare Diseases Framework.

While the UK has introduced changes to its market access ecosystems to better address the access challenges for orphan drugs, real challenges limiting access to these medicines remain, the report suggests.

The report also evaluates the effectiveness of recent UK regulatory and access pathways, highlighting areas where further work is needed.

Among these are the lack of consideration for the quality of life impacts on families or carers in health economic evaluations and the need for greater clarity and effectiveness in new systems like ILAP (Innovative Licensing and Access Pathway) and IMF (International Monetary Fund).

The report encapsulates insights from interviews and focus groups involving key UK stakeholders in the rare disease sector, including NHS England, NICE, patient groups, clinicians, and industry representatives.