The Medicines and Healthcare products Regulatory Agency (MHRA) has launched a public consultation on a proposed Rare Disease Therapies Framework a regulatory proposal that would introduce a new development and authorisation pathway for therapies targeting rare diseases in the United Kingdom. The consultation, published on 21 May 2026, is open until 30 July 2026, and the MHRA is calling on pharmaceutical companies, biotechnology developers, contract research organisations, academics, and clinicians to contribute to the shaping of the final framework.
The proposed framework represents a structured regulatory response to the well-documented barriers that prevent rare disease therapies from reaching patients under conventional development models. It is specifically designed for therapies targeting conditions with a prevalence of typically one in approximately 50,000 or fewer in the UK, where the limitations of standard development pathways are most pronounced.
At the centre of the Rare Disease Therapies Framework is a newly proposed instrument called the Investigational Marketing Authorisation (IMA) a single authorisation that combines clinical trial approval with a progressive route to marketing authorisation. The IMA pathway is designed to remove the need for sponsors to manage a separate, sequential transition from clinical trial approval to marketing authorisation, replacing it with a coherent lifecycle that supports rolling data submissions, modular assessments, and earlier patient access where limited but compelling evidence exists.
The Investigational Marketing Authorisation also incorporates structured post-authorisation evidence generation requirements for safety, quality, and efficacy, ensuring that earlier access does not come at the cost of regulatory rigour.
The framework explicitly supports adaptive and innovative trial designs, including basket trials, umbrella trials, and hybrid designs that incorporate real-world evidence. It acknowledges that surrogate or patient-relevant endpoints may be appropriate where conventional endpoints are not feasible. The MHRA has also confirmed its openness to the use of computational modelling, digital twins, and non-animal methods where scientifically justified.
Traditional rare disease development programmes typically take between 10 and 12 years to reach marketing authorisation a timeline driven by linear phase progression and limited early regulatory engagement. The proposed Rare Disease Therapies Framework is designed to compress these timelines meaningfully, particularly during early phases and regulatory decision-making processes.
For larger pharmaceutical organisations, the framework creates new opportunities to engage with very rare indications through more iterative investment strategies within a more predictable regulatory environment. For smaller developers and academic institutions, the framework is intended to provide earlier regulatory certainty and structured scientific advice from the outset of development.
The Rare Disease Therapies Framework is technology-agnostic and applicable across a broad range of therapy types, including advanced therapy medicinal products, individualised medicines, gene-based therapies, digital-enabled medicinal products, innovative manufacturing platforms, and repurposed medicines. This scope is designed to ensure the framework remains fit for purpose as science and technology continue to evolve.
The framework has been developed with input from the Rare Disease Consortium a cross-sector group that includes the MHRA, the Health Research Authority, the National Institute for Health and Care Excellence, the Department of Health and Social Care, NHS England, patient advocacy organisations, academic institutions, and industry partners.
Public Health Minister Sharon Hodgson stated that the proposals represent an important step towards a more agile and compassionate system one that recognises the unique challenges of rare disease research while maintaining the highest standards of patient safety.
The MHRA has highlighted that the UK possesses distinctive strengths for rare disease innovation: a strong academic base, a single national genomics provider, and the scale and diversity of NHS datasets. The Rare Disease Therapies Framework is designed to utilise these strengths within a coherent enabling structure, positioning the UK as a destination for rare disease therapy development and clinical trials.
The MHRA consultation on the draft Rare Disease Therapies Framework runs until 30 July 2026. The MHRA will be arranging a series of general engagement sessions over the summer while the consultation is underway and responses are being considered. Industry input is described as critical to enabling earlier access to potentially life-saving therapies for patients living with rare diseases, and the MHRA is encouraging early engagement meetings throughout the consultation period. The agency has also confirmed that the Rare Disease Therapies Framework was first announced in November 2025.
