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Rare Diseases: South East

Answer

The Government is committed to improving the lives of those living with rare diseases, and we acknowledge unmet need remains, with just 5% of rare diseases having an approved and effective treatment. The UK Rare Diseases Framework sets out four priorities collaboratively developed with the rare disease community, such as improving access to specialist care, treatments, and drugs, including in Slough. We published the fifth annual England action plan in February 2026, where we report on the steps we have taken to advance these priorities. These include:

• the Medicines and Healthcare products Regulatory Agency (MHRA) developing a framework to enable innovation in rare disease therapies;
• new clinical trial regulations being fully implemented from April 2026;
• the MHRA and the National Institute for Health and Care Excellence (NICE) aligning a pathway for parallel decision making to reduce the gap between marketing authorisation and NICE guidance decisions;
• continuing to review the effectiveness of the Early Access to Medicines Scheme, the Innovative Licensing and Access Pathway and the Innovative Medicines Fund, access pathways for rare therapies; and
• NHS England progressing work to develop an operational framework for individualised therapies.

Secondary information

Type

Written question

Reference

120352

Session

2024-26

Subjects

Medical treatments South East Rare diseases

Link

View this Written question on www.parliament.uk