ICN Invites Feedback on MHRA Rare Disease Therapies Consultation

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has launched a public consultation on its proposed Rare Disease Therapies Regulatory Framework. The draft framework aims to introduce a more flexible regulatory pathway for rare disease therapies, recognising that conventional approaches to evidence generation are often not feasible for very small patient populations.

These proposals are highly relevant to N-of-1 trials and single-case designs, which have the potential to support evidence generation for rare diseases and other settings involving very small patient populations.

The International Collaborative Network for N-of-1 Trials and Single-Case Designs (ICN) will be preparing a submission to the consultation. We invite members and the wider community to share comments or perspectives that may help inform our response.

Please submit your feedback by 28 July 2026 5pm UK time to allow sufficient time for us to prepare and finalise the ICN submission ahead of the MHRA consultation deadline.