On 21 May 2026, the Medicines and Healthcare products Regulatory Agency (“MHRA”) published its draft regulatory framework for rare disease therapies (the “Framework”), marking a significant step in the UK’s efforts to accelerate development and improve access to rare disease treatments.
The Framework forms part of a larger regulatory and policy landscape in the UK focused on addressing the needs of those living with rare diseases (see our recent insight article R is for Rare Diseases in the UK: An Overview of the Regulatory and Policy Landscape).
The Framework has been deliberately designed as a technology‑agnostic regulatory framework for medicinal products. It seeks to strike a balance between a patient-centred, adaptive approach providing quicker access to therapies, while maintaining high standards of safety and effectiveness. It emphasises its aim to streamline, and meaningfully compress, the typical 10–12-year timeline for programmes to reach market authorisation. Recognising the challenges of research into rare diseases, such as small patient samples, the Framework suggests more flexible evidence requirements. It balances this with an emphasis on the importance of post-market data collection and monitoring after the approval of a therapy, ensuring continual maintenance of safety standards.
The introduction of a new Investigational Marketing Authorisation (“IMA”) is principal to the MHRA’s aims, offering a singular, centralised authorisation to provide a faster and easier access route to innovative medicines. The IMA’s compressed pathway aims to reduce development costs of rare disease therapies by combining clinical trial and market authorisation approvals.
Ambitions for the UK to be a global leader in rare disease innovation are evidenced in the MHRA’s continued support of innovative trial designs. The Framework takes a positive stance towards the use of technology, including the use of computational modelling, implying a willingness to embrace technological tools and support attempts to address the challenges of rare diseases.
The Framework is accompanied by an open call for public consultation, with the MHRA seeking input from pharmaceutical companies, biotech developers, contract research organisations, academics and clinicians, as well as patients, carers and families.
An area of focus for the consultation is the criteria for accelerated IMA approval, with the MHRA requesting views on the conditions or types of evidence that should qualify a therapy for this faster approval process.
Consultation responses are open until 30 July 2026. They can be provided online here.
With thanks to Lili Strachan for her contribution to this article
