RARE Daily

Industry Group Says UK Needs to Do More to Support Access to New Rare Disease Therapies

November 6, 2023

Rare Daily Staff

The BioIndustry Association, a UK-trade organization, is calling for the United Kingdom to lead greater collaboration between stakeholders in the United Kingdom and internationally, to improve access to new treatments for rare disease patients.

The BIA’s Rare Disease Industry Group pointed to a new report by the BIA, with research provided by global consultancy firm PwC, that examined the progress that has been made in improving access to rare disease treatments in the United Kingdom and the challenges that remain.

The report, “Evaluating patient access to rare disease treatments’: Insights from the UK and beyond,” is based on a series of interviews and focus groups with key stakeholders in the United Kingdom rare disease space, including NHS England, NICE, patient groups, clinicians, and industry.

The report found that the United Kingdom lags comparable countries, including France and Germany, on the degree of availability of treatments for rare diseases. Data from the European Federation of Pharmaceutical Industries and Associations show that 59 percent of rare disease drugs approved by the European Medicines Agency had been reimbursed in England between 2018-2021, and 55 percent in Scotland. This compares to 86 percent in Germany and 77 percent in France, although it ranks above Spain with 52 percent.

Recently, both NICE and the MHRA announced new ways to increase their collaboration with other HTA bodies and regulators to speed up patients’ access to new medicines, including through the new International Recognition Procedure. To help inform this collaboration, the report identifies successful examples of processes and schemes that help to facilitate access to rare disease drugs in comparable OECD countries that could become international best practice, including in the United Kingdom.

“The UK has an incredibly strong rare disease community, including the close network of patient advocacy groups, the specialist NHS centers and clinicians, and the leading scientists and academics,” said Steve Bates, chief executive of the BIA. “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. Making sure that patients have timely and broad access to treatments is an essential part of this, and this report provides an important evidence base to inform future reforms in this space.”

Photo: Steve Bates, chief executive of the BioIndustry Association

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