The United Kingdom's healthcare system is grappling with mounting challenges in providing access to rare disease medicines, despite recent approvals of three new treatments in early 2024. A comprehensive analysis reveals systemic barriers threatening patient access to innovative therapies for rare conditions.
Declining Position in European Access Rankings
The UK has experienced a dramatic decline in its standing for rare disease medicine availability among European nations. Once ranked second behind Germany, the country has now fallen to tenth place for England and thirteenth for Scotland, according to EFPIA's Patients WAIT indicator data. This decline reflects growing concerns about the UK's ability to provide timely access to orphan medicines.
Rising Barriers to Medicine Approval
A troubling trend has emerged in the evaluation of new medicines through the National Institute for Health and Care Excellence (NICE). Data analysis by the Office for Health Economics shows the rate of terminated NICE appraisals has more than doubled, jumping from 9.8% between 2016-2019 to 20.1% in 2020-2023. These terminations occur when manufacturers either withdraw their submissions or choose not to submit at all, primarily due to anticipated rejections.
Industry Concerns and Future Pipeline Impact
A joint survey by the Association of the British Pharmaceutical Industry (ABPI) and BIA revealed that since 2018, companies decided against NICE evaluation for 16 out of 64 rare disease medicines approved by the MHRA. More concerning for future access, 11 out of 18 surveyed pharmaceutical companies indicated they plan to launch less than 75% of their rare disease pipeline in the UK over the next five years.
Commercial Environment Challenges
The increasingly challenging commercial landscape includes significant hurdles for pharmaceutical companies. The Voluntary Scheme payment rates have escalated to 22.9% of industry UK turnover in 2025, up from 15%. Companies cite this alongside strict entry criteria for NICE's Highly Specialised Technologies evaluation programme as major deterrents to bringing rare disease treatments to the UK market.
Impact on Patient Care
For patients with rare diseases, these systemic challenges translate to reduced access to potentially life-changing treatments. Even when medicines receive NICE approval, local NHS adoption often proves slow and inconsistent, creating inequitable access across regions. Cost-effectiveness thresholds in England, among the lowest globally, have declined approximately 50% in real terms over two decades.
Future Outlook and Proposed Solutions
The UK government has implemented Rare Disease Action Plans across England, Scotland, Wales, and Northern Ireland, demonstrating commitment to addressing these challenges. The ABPI advocates for extending the UK Rare Disease Framework beyond its current January 2026 endpoint, emphasizing the need for continued coordination and accountability across the four nations.
Industry stakeholders stress the importance of addressing fundamental issues, including the VPAG rates and NICE's evaluation methods, to prevent further deterioration of UK patient access to rare disease treatments. Without significant changes, patients with rare conditions may face increasing difficulties accessing innovative therapies that often represent their only treatment options.
