The Medicines and Healthcare products Regulatory Agency has published a policy paper setting out plans to redesign how rare disease therapies are assessed and licensed in the UK. The document, issued on 2 November 2025, signals a shift to a more flexible pathway intended to shorten the time from discovery to delivery while maintaining safety standards.
The scale of unmet need is explicit in the agency’s announcement. Around 3.5 million people in the UK live with a rare disease, fewer than 5% of rare diseases have an approved treatment, the average diagnostic journey is 5.6 years, and an estimated 30% of affected children die before age five. The press release also cites annual costs of £340 million linked to delayed diagnosis, £4.7 billion in health‑related disability, and £14.9 billion in wider economic loss.
The MHRA proposes a licensing model that can bridge clinical trial and marketing decisions, including the potential use of an Investigational Marketing Authorisation with modular data, iterative checkpoints and real‑world evidence collection. A mandatory pre‑designation discussion would set evidence expectations and lifecycle obligations from the outset, and the regulator is exploring the use of ‘source files’ so prior knowledge from related products can be reused to reduce burden.
Eligibility for the new route is described in consultation terms as therapies for conditions with a prevalence of no more than 5 in 10,000 where standard development is impractical. The paper flags flexibility for individualised medicines, including the possibility of a single authorisation even when a component is tailored to an individual’s characteristics, subject to proportionate controls.
Post‑market oversight is to be strengthened. Proposals include national rare disease registries to capture long‑term outcomes, risk management plans with mandated reviews triggered by time or patient numbers, and alignment-where feasible-between regulatory and health technology assessment evidence needs to support managed access. International data sharing is identified as necessary to supplement small UK cohorts.
The programme emphasises earlier, structured engagement with system partners so regulatory review runs in parallel with NHS access processes and NICE’s appraisal work. The Rare Disease Consortium, comprising patient groups, academia, industry and system bodies, will support development; the reforms are presented as contributing to the England Rare Diseases Action Plan published in February 2025.
Timelines are set out across 2026. The policy paper indicates a draft framework is anticipated by spring 2026, external review in the first half of the year, and a public consultation during 2026, with the aim of moving to a refined and implementable model by late 2026. The MHRA notes that legislative steps may be required as the pathway is finalised.
For developers and sponsors, the direction of travel points to designing adaptive studies, planning for real‑world evidence and registry participation from the start, and preparing manufacturing and centre accreditation plans for advanced modalities. Early dialogue on endpoints, prior knowledge, and platform definitions will be central to predictability and speed.
The paper situates the reforms alongside existing UK schemes such as the Innovative Licensing and Access Pathway rather than as a replacement. The intent is a rare‑therapy‑specific route that can handle small populations and individualised products, with safety anchored by enhanced pharmacovigilance.
Stakeholder responses published with the announcement-from patient organisations and research bodies-back the direction of travel, stressing faster access without compromising standards. The MHRA frames the work as a UK‑wide effort to pool scarce evidence while keeping regulatory decisions transparent and patient‑centred.