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 medicines regulatory system at supporting novel clinical trials for rare disease treatments.

No specific assessment has been made. However, the Medicines and Healthcare products Regulatory Agency (MHRA) offers a 10-year period of market exclusivity for orphan designated products, which can encourage the pharmaceutical industry to develop medicines for rare diseases. There are flexibilities in the licensing system which consider novel clinical trials and data generation aspects for small population research, such as a Conditional Marketing Authorisation.

The Innovative Licensing and Access Pathway (ILAP) supports bespoke product development programmes and aims to accelerate the time to market, facilitating more rapid and efficient patient access to medicines. The entry criteria for the ILAP include a rare disease aspect and a toolkit encourages the use of novel approaches to the clinical trial design and development programme. The MHRA supports the design of novel clinical trials through formal scientific advice, where the methodology, challenges and opportunities of the approach can be considered by a team of experts and the study sponsor.