Overview
- The Medicines and Healthcare products Regulatory Agency published a draft Rare Disease Therapies Regulatory Framework and opened a public consultation on its proposals.
- The centrepiece is a new Investigational Marketing Authorisation that would combine trial approval and limited market access so patients can receive therapies while more evidence is gathered.
- The draft sets out specific evidence expectations for very small or mixed patient groups, including adaptive trial designs, real‑world data collection, use of prior knowledge, and predictive modelling.
- The framework requires sustained patient engagement and ongoing informed consent so individuals and families stay involved as safety and effectiveness data are collected.
- Adoption depends on consultation feedback and further legal and regulatory detail, and the change could affect more than 3.5 million people in the UK who live with rare diseases and often lack approved treatments.