Written question asked by Andrew Lewer (Conservative) on Wednesday, 17 April 2024, in the House of Commons. It was due for an answer on Friday, 19 April 2024. It was answered by Andrew Stephenson (Conservative) on Monday, 22 April 2024 on behalf of the Department of Health and Social Care.

Rare Diseases: Drugs

Question: To ask the Secretary of State for Health and Social Care, what steps her Department is taking to support effective routes to market for precision medicines for (a) motor neurone disease and (b) other rare diseases in the UK.

Answer: Should the Medicines and Healthcare products Regulatory Agency (MHRA) receive a marketing authorisation application (MAA) or applications, the MHRA will consider the drug with regards to quality, safety, and efficacy. The MHRA has procedures in place to consider drugs as part of an accelerated pathway. This is possible as part of national access collaboration, and international recognition. The MHRA would be able to consider motor neurone disease and other rare disease treatment products, under any of the procedures for the benefit of patients.

Secondary information