Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what assessment he has made of the effectiveness of the Early Access to Medicines Scheme in facilitating patient access to new treatments for neuromuscular diseases.

The Medicines and Healthcare products Regulatory Agency (MHRA) supports access to treatments for people living with neuromuscular diseases. The majority of neuromuscular diseases are defined as rare diseases. On the 2 November 2025, the MHRA published a policy paper on rare therapies and United Kingdom regulatory considerations, which is available at the following link:

https://www.gov.uk/government/publications/rare-therapies-and-uk-regulatory-considerations/rare-therapies-and-uk-regulatory-considerations

The Early Access to Medicines Scheme (EAMS), is an existing pathway across the regulatory and access system designed to support innovative treatments being available to patients earlier in the development cycle, outside of a clinical trial. This includes for those living with neuromuscular diseases. The Government is collaborating across the regulatory system to continuously review the effectiveness of these pathways for rare diseases. Further information on the EAMS is available at the following link:

https://www.gov.uk/guidance/apply-for-the-early-access-to-medicines-scheme-eams