UK Regulator Unveils Major Reforms to Speed Approval of Rare Disease Treatments
Medicines and Healthcare products Regulatory Agency (MHRA) sets out framework to address the 95 % of rare conditions lacking approved therapies
The United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) has announced a sweeping overhaul of its regulatory approach to therapies for rare diseases, following the release of a position paper on 2 November 2025. The reforms aim to accelerate access for the estimated 3.5 million people in the UK living with rare conditions while only around 5 % currently have approved treatments.
The agency acknowledged that traditional regulatory pathways—designed for common conditions with large patient populations and multiple confirmatory trials—are ill-suited to rare diseases, which often involve small, heterogeneous patient groups, unclear clinical endpoints and urgent unmet needs.
It noted that advanced therapies such as gene and cell-based treatments raise particular manufacturing and long-term safety challenges.
Under the new framework, the MHRA proposes a more flexible licensing and registration regime tailored to rare-disease therapies.
Measures include the use of real-world evidence, adaptive trial designs, expedited review processes and the possibility of a single marketing authorisation covering individually tailored treatments.
A public consultation is scheduled for 2026, with an expected draft regulatory guidance by spring of that year and potential legislative steps to follow.
The reform builds on earlier initiatives such as the UK Rare Diseases Framework and the Clinical Trials Amendment Regulations 2024, which eased trial-approval procedures.
Proponents say the latest changes signal the UK’s ambition to become a global hub for rare-disease innovation and reassure patients that the system recognises their needs.
However, the pharmaceutical industry emphasises that regulatory reform must be matched by improved access and reimbursement mechanisms, noting that only one in twenty rare conditions has an approved therapy and that the UK’s ranking for access to orphan medicines has slipped in recent years.
With the MHRA’s new plan, the UK government is actively aligning regulatory innovation with patient-focused outcomes, aiming to reduce the years-long delays many rare-disease patients currently endure.
The agency acknowledged that traditional regulatory pathways—designed for common conditions with large patient populations and multiple confirmatory trials—are ill-suited to rare diseases, which often involve small, heterogeneous patient groups, unclear clinical endpoints and urgent unmet needs.
It noted that advanced therapies such as gene and cell-based treatments raise particular manufacturing and long-term safety challenges.
Under the new framework, the MHRA proposes a more flexible licensing and registration regime tailored to rare-disease therapies.
Measures include the use of real-world evidence, adaptive trial designs, expedited review processes and the possibility of a single marketing authorisation covering individually tailored treatments.
A public consultation is scheduled for 2026, with an expected draft regulatory guidance by spring of that year and potential legislative steps to follow.
The reform builds on earlier initiatives such as the UK Rare Diseases Framework and the Clinical Trials Amendment Regulations 2024, which eased trial-approval procedures.
Proponents say the latest changes signal the UK’s ambition to become a global hub for rare-disease innovation and reassure patients that the system recognises their needs.
However, the pharmaceutical industry emphasises that regulatory reform must be matched by improved access and reimbursement mechanisms, noting that only one in twenty rare conditions has an approved therapy and that the UK’s ranking for access to orphan medicines has slipped in recent years.
With the MHRA’s new plan, the UK government is actively aligning regulatory innovation with patient-focused outcomes, aiming to reduce the years-long delays many rare-disease patients currently endure.
