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  • Writer's picturePaul Andrews

Call For Better Collaboration To Improve Access To New Treatments

A group of companies developing innovative new medicines for rare diseases, working together through their trade association, is calling for the UK to lead greater collaboration between stakeholders, and uptake of best practice both in the UK and internationally, to improve access to new treatments for patients.

The call from the BioIndustry Association (BIA)’s Rare Disease Industry Group (RDIG) is informed by a new report by the BIA, with research provided by global consultancy firm PwC, which has highlighted the progress that has been made in improving access to rare disease treatments in the UK, 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 UK rare disease space, including NHS England, NICE, patient groups, clinicians, and industry.

The report identifies that the UK 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 (EFPIA) show that 59% of rare disease drugs approved by the European Medicines Agency (EMA) had been reimbursed in England between 2018-2021, and 55% in Scotland. This compares to 86% in Germany and 77% in France, although it ranks above Spain with 52%.

Recently, both NICE and the MHRA have 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 UK.

Steve Bates, Chief Executive of the BIA, said: “The UK has an incredibly strong rare disease community, including the close network of patient advocacy groups, the specialist NHS centres and clinicians, and the leading scientists and academics. 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.”

Stephen Aherne, Pharmaceuticals and Life Sciences Leader at PwC UK, said: “Over the past decade, the UK life sciences sector has made significant strides in the development of treatments for rare diseases. Yet 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.”

Read the full report here:

Download PDF • 2.64MB


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