PARLIAMENTARY WRITTEN QUESTION
(9 December 2024)

The Government is committed to improving the lives of those living with rare diseases. The UK Rare Diseases Framework sets out four priorities collaboratively developed with the rare disease community, including improving access to specialist care, treatments, and drugs. We remain committed to delivering under the framework, and will publish an England action plan in 2025. Delivery partners have committed to reviewing the effectiveness of treatment access pathways like the Early Access to Medicines Scheme (EAMs), the Innovative Licensing and Access Pathway (ILAP), and the Innovative Medicines Fund for rare disease therapies.

NHS England commissions over 80 highly specialised services provided to small numbers of patients, which includes services for rare diseases. The services are delivered and co-ordinated nationally through a limited number of expert centres, to develop appropriate clinical quality, expertise, and experience.

The Medicines and Healthcare products Regulatory Agency supports development and access to treatments for rare diseases through regulating the medicines supplied in the United Kingdom. These include the Orphan Medicinal Products Regulation, and Marketing Authorisations under Exceptional Circumstances for treatments where there is limited data, such as rare diseases. Additionally, initiatives like Project Orbis, the ILAP, the EAMS, and the Regulatory Advice Service for Regenerative Medicines accelerate access to treatments.

The National Institute for Health and Care Excellence (NICE) makes recommendations for the National Health Service on whether all licensed new medicines should be routinely funded by the NHS, based on an assessment of their costs and benefits. The NHS in England is legally required to fund medicines recommended by the NICE, normally within three months of the publication of final guidance. The NICE operates a separate Highly Specialised Technologies (HST) programme for very rare diseases, with significantly higher cost-effectiveness thresholds than those evaluated under the NICE’s standard technology appraisal processes. Decisions on whether new medicines should be evaluated through the HST programme are taken by the NICE against published routing criteria.