On 21 May 2026 the Medicines and Healthcare products Regulatory Agency opened a public consultation on a draft Rare Disease Therapies Regulatory Framework, with responses due by 11:59pm on 30 July 2026. According to the consultation and the draft guidance, the route is aimed at therapies for conditions typically affecting around 1 in 50,000 people or fewer in the UK where standard development or licensing routes are difficult to use. (gov.uk) The immediate policy question is how existing evidence standards should operate where patient numbers are too small for conventional development models. The MHRA says more than 3.5 million people in the UK will be affected by a rare disease during their lifetime, yet only around 5% of rare diseases have an approved medicinal product. (gov.uk)
The main structural change is a proposed Investigational Marketing Authorisation, or IMA. In the draft framework, the MHRA describes this as a single authorisation combining clinical trial approval with a continuously reviewed route towards marketing authorisation, so that controlled patient access, data generation and regulatory review can run in parallel rather than as separate stages. (gov.uk) That matters because the draft says traditional rare disease programmes usually take 10 to 12 years to reach marketing authorisation. The MHRA argues that earlier scientific advice, modular assessments and rolling submissions could shorten that timetable, but it also states that full implementation of the IMA would require legislative change or a new statutory power. (gov.uk)
Evidence standards would not disappear under the proposal, but they would become more flexible. The draft says the MHRA would consider adaptive and Bayesian designs, single-arm studies with external or real-world comparators, basket and umbrella trials, hybrid structures, surrogate endpoints where scientifically justified, and post-authorisation evidence plans built around real-world data and long-term follow-up. (gov.uk) The framework also signals openness to prior knowledge, patient video assessments, digital biomarkers, computational models including digital twins, and non-animal methods where these help answer questions that conventional trials cannot address in very small cohorts. For developers, the practical effect is that the consultation is not only about speed; it is also about what counts as usable evidence in rare disease regulation. (gov.uk)
Entry to the route would not be automatic. The draft says applicants would need rare disease designation and would be expected to show prevalence in the region of 1 in 50,000 in the UK, serious unmet need, measurable barriers to a standard development programme, and a credible plan for demonstrating efficacy. The framework is described as technology-agnostic and is intended to cover medicines ranging from advanced therapies and individualised products to repurposed medicines. (gov.uk) The MHRA is also clear about what the framework does not do. It does not replace existing licensing routes, it does not alter orphan designation rules, and it can sit alongside support mechanisms such as the Innovative Licensing and Access Pathway where a product meets the relevant tests. (gov.uk)
For patients, clinicians and ethics bodies, the consultation puts unusual weight on communication and ongoing consent. The draft says early access under the framework should be accompanied by clear explanation of uncertainty, regular updates as evidence changes, opportunities to revisit decisions, and long-term follow-up arrangements, with Research Ethics Committee review, Health Research Authority processes and proportionate Good Clinical Practice standards remaining in place. (gov.uk) That is an important design choice. The proposed framework is built on the assumption that some products may reach defined patient groups before a conventional evidence package exists, so the regulator is pairing earlier access with tighter monitoring and a more explicit consent model rather than treating consent as a single event at the start of treatment. (gov.uk)
For NICE, NHS England and commissioners, the consultation draws a firm boundary between regulation and funding. The draft states that an IMA would not in itself secure reimbursement or routine commissioning, and it says evidence plans should, where possible, support both MHRA decision-making and health technology assessment so that reimbursement questions are addressed earlier rather than at the end of the process. (gov.uk) That means the consultation is as much about system alignment as about licensing mechanics. The MHRA says managed access or conditional reimbursement may be appropriate for some products with immature evidence, and the agency is encouraging early engagement while the consultation runs, including further sessions over the summer. (gov.uk)
In policy terms, this consultation is the next formal step after the MHRA's rare therapies position paper, published on 2 November 2025, and it sits within the wider delivery of the England Rare Diseases Action Plan 2026. Those earlier documents framed rare disease regulation as part of a broader package covering clinical trial reform, MHRA-NICE co-ordination and the government's life sciences ambitions. (gov.uk) For industry, academics, contract research organisations and patient groups, this is the stage at which operational details can still be shaped. The draft says the full process for application, grant, conversion, restriction or withdrawal will be set out in the final framework after consultation, while the fee schedule is still to be developed and full implementation of the IMA would require legislative change or a new power for the MHRA. (gov.uk)