MHRA proposes new framework to accelerate rare disease therapies in UK

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has launched its public consultation on a proposed Rare Disease Therapies Framework designed to accelerate development and approval of treatments for rare and ultra-rare conditions.

At the center of the proposal is a new Investigational Marketing Authorisation (IMA), which would combine clinical trial approval and marketing authorization into a single, continuously reviewed regulatory pathway. The MHRA said the approach is intended to remove the traditional divide between clinical development and commercial approval, enabling rolling data submissions, modular assessments and earlier patient access where evidence is limited but promising.