Rare Diseases: Drugs
The question
To ask the Secretary of State for Health and Social Care, if his Department will include a target in a future Rare Disease Action Plan on proportionate access to multi-indication medicines for (a) rare and (b) ultra-rare disease patients.
Answered by Ashley Dalton
Improving access to specialist care, treatments, and drugs is a priority under the UK Rare Diseases Framework. The 2025 England Rare Diseases Action Plan was published in February 2025 and reports on progress against existing actions under this priority. This included reviewing the effectiveness of the Early Access to Medicines Scheme, the Innovative Licensing and Access Pathway, and the Innovative Medicines Fund, which are key access initiatives aimed at facilitating earlier availability of innovative treatments. Our review specifically assessed how effective these pathways are in supporting timely access to therapies for individuals living with rare diseases.
As set out in the Life Sciences Sector plan, we will be introducing a new and proportionate approach to National Institute for Health and Care Excellence (NICE) appraisals and NHS indication-specific based pricing agreements for medicines with large numbers of indications, strong long-term outcome data, and low affordability risk. This will support treatments for rare diseases. There are currently no plans to introduce an access target, as it is essential to review the available evidence on a case-by-case basis. However, the Government remains committed to improving access to medicines for rare and ultra-rare diseases.
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