MHRA CONSULTS ON NEW REGULATORY FRAMEWORK TO MAKE UK A GLOBAL LEADER IN RARE DISEASE THERAPY DEVELOPMENT

LONDON, May 21 -- The government of the United Kingdom issued the following news:

The Medicines and Healthcare products Regulatory Agency (MHRA) has today launched apublic consultationon a proposed new Rare Disease TherapiesFrameworkthat would introduce significant regulatory innovation to the UK's rare disease landscape-and theMHRAis calling on the pharmaceutical and life sciences industries to play a central role in shaping the final framework.

The draftframeworksets out a technology-agnostic regulatory framework designed to address the fundamental scientific,evidentiaryand commercial barriers that prevent rare disease therapies from reaching patients under conventional development models.

It is designed for therapies targeting rare diseases with a prevalence of typicallyone in approximately 50,000 orfewerin the UK, where there are clear and measurable barriers to conventional development.

Theconsultationruns until30Julyand industry input iscriticaltoenable earlier access to potentially life-saving therapies for rare diseases.

Why industry input matters

The MHRA is clear that the detail of this framework must be shaped by those who will use it. Theconsultationprovides a direct opportunity for pharmaceutical companies, biotech developers, contract research organisations, academics, and clinicians to influence the final guidance - including the eligibility criteria foranInvestigationalMarketingAuthorisation (IMA)designation, the approach to real-world evidence generation, scientific advice processes, and the interface with existing routes such as orphan designation and the Innovative Licensing and Access Pathway (ILAP).

The framework has been developed with input from the Rare Disease Consortium - a cross-sector group includingtheMedicines and Healthcare products Regulatory Agency (MHRA), theHealthResearchAuthority (HRA),theNational Institute for Health and Care Excellence (NICE),theDepartment of Health and Social Care (DHSC), NHS England, patient advocacyorganisations, academia, and industry partners. Theconsultationnowopensthe processforwiderinput.

Respond to theconsultation:

Draft rare disease therapies regulatory framework

Early engagement meetings with the MHRAwill beencouraged.The MHRA will be arranginga series ofgeneralsessions over the summer whilst the consultation is underway andresponses are being considered.For more information, please contactourCustomerExperience Centre.

A new model for rare disease development

Traditional rare disease programmes typicallytake10-12 years to reach marketing authorisation, driven by linear phase progression andtraditionally limited opportunities forearlyregulatoryinvolvement. The proposed framework is designed to compress these timelines meaningfully, particularly in early phases and during regulatory decision-making.

At the heart of the proposal is a newIMA-a single authorisation that would combine clinical trial approval witha progressiveroute to marketing authorisation. Rather than requiring sponsors to transition from clinical trial approval to marketing authorisation as separate regulatory steps, the IMA provides a coherent lifecycle that supports rolling data submissions, modular assessments, and earlier patient access where there is limited but compelling evidence, supported by structured post-authorisation evidence generationfor safety, quality and efficacy.

The guidance explicitly supports adaptive and innovative trial designs-including basket trials, umbrella trials, and hybrid designs incorporating real-world evidence-and accepts that surrogate or patient-relevant endpoints may beappropriate whereconventional endpoints are notfeasible. It also sets outtheMHRA's openness to the use of computational modelling, digital twins, and non-animal methods where scientifically justified.

For larger pharmaceutical companies, the guidance creates new opportunities to diversify intovery rareindications with more iterative investment strategies and a more predictable regulatory environment. For smaller developers and academics, it provides earlier regulatory certainty and structured scientific advice from the outset.

The UK's competitive position

The UK has the foundations to be a global leader in rare disease innovation: a strong academic base, a single national genomics provider, and the unique scale and diversity of NHS datasets.

The new guidance is designed toutilisethese strengths within a coherent enabling framework, positioning the UK as the destination of choice for rare disease therapy development and clinical trials,while safeguarding patients andmaintainingconfidence in regulatory decision-making.

The framework is technology-agnostic and applicable across advanced therapy medicinal products (ATMPs), individualised medicines, gene-based therapies, digital-enabled medicinalproducts,innovativemanufacturing platformsand includes repurposed medicines.

Public Health Minister Sharon Hodgson said:

For the millions of people in the UK living with a rare disease, and for the families and carers who support them, the search for effective treatment can be long,exhaustingand deeply uncertain.

These landmark proposalsrepresentan important steptowards a more agile and compassionate system - one that recognises the unique challenges of rare disease research whilemaintainingthe highest standards of patient safety.

By helping innovative therapies reach patients faster, this framework has the potential to transform lives, strengthen the UK's position as a global leader in life sciences, and give renewed hope to families who have waited too long for progress.

I encourage patients, clinicians, researchers and carers to take part in this consultation and help shape the future of rare disease treatment in the UK.

Julian Beach, Executive Director of Healthcare Quality and Access at the MHRA, said:

Patients living with rare diseases often face significant barriers in accessing effective treatments. Thisconsultationmarksan important steptowards a more flexible and responsive regulatory system that reflects the challenges and realities of rare disease development.

For developers, the guidance provides a more streamlined and efficient process. A single authorisation removes the need fora discretetransition from clinical trial approval to marketing authorisation, supports more predictable and adaptable evidence requirements, and allows for rolling data submissions to accelerate decision-making. It also promotes better alignment between clinical development, regulatory approval, patient access, and reimbursement processes, thereby reducing complexity.

By working closely with patients, partners and industry, we're building a framework that supports innovation while maintaining the high standards of safety that patients expect.

Industry feedback is vital to thisconsultation,so please share your viewsat Draft rare disease therapies regulatory framework

HelenKnight, Director of Medicines Evaluation attheNationalInstitute for Health and Care Excellence, said:

The MHRA's proposed Rare Disease Therapies Regulatory Framework potentially compliments NICE's approach that enables NHS patients to receive innovative and promising medicines whileadditionalevidence is gathered on how well they work in practice. These proposals could help to address clinical uncertainty, with the MHRA continuing stringent patient safety monitoring while NICE ensures value for money for the taxpayer.

We support the ambition to improve timely access to rare disease therapies and look forward to engaging in more detail alongside wider system partners to ensure this delivers safely for patients, value for the NHS and aligns with broader government policy objectives.

Nick Meade, Chief Executive of Genetic Alliance UK, said:  

For many rare condition communities, innovative treatment development has been out of reach.That'schanging with this programme, as more treatment paradigms areopened upand lower prevalence levels become more commerciallyviable. Nowit'stime to make sure the detail is right, so that we can be sure the UK rare condition community can be the first tobenefitfrom this welcome and ambitious undertaking.

Dr Jacqueline Barry, Chief Clinical Officer, Cell and Gene Therapy Catapult, said:

For patients with rare diseases, regulatory timelines are not an abstract concern. Theyrepresentthe difference between accessing a potentially life-changing therapy and not. The proposed framework responds to that directly. The Investigational Marketing Authorisation has the potential to accelerate the development pathway for advanced therapies, enabling iterative, evidence-led progression that better reflects the science, while upholding the highest standards of safety,efficacyand quality. We welcome this consultation and strongly encourage developers across the advanced therapy sector to engage.

Sam Barrell, CEO ofLifeArc, said:

For families affected by rare diseases, time matters. But even when there's real promise in the science, treatments can take far too long to reach them because the regulatory pathways were not designed with rare diseases in mind.

This consultation is a chance to design a better way forward: one that enables therapies to reach patients faster without compromising safety.We'dencourage companies, researchers,cliniciansand patient groups to get involved so the final framework reflects theirexpertiseand experience.

Dr Rick Thompson, CEO of Beaconfor rare diseases, said:

There are millions of people in the UK living with a rare condition, struggling to secure research or treatment. This new regulatory framework has the chance to be transformative for rare diseases - inspiring more research and ensuring that more therapies have the chance to reach patients who need them. I encourage all stakeholders to engage with the MHRA consultation. Together, we can help to ensure these transformative ideas are implemented in a manner that works for developers and underscores their importance to those affected by rare diseases.

Professor Claire Booth, Consultant Paediatric Immunologist at Great Ormond Street Hospital, said:

This consultation is an important and hopeful step for families affected by rare diseases, where time and access to treatment are critical. Too often, patients face long diagnostic journeys with limited options, not because the scienceisn'tadvancing, but because the systemhasn'tkept pace.

Dr Harriet Holme,Drug Development Clinician atWeatherdenandExecutive Chair of PCD Researchsaid: 

This proposed regulatory frameworkrepresentsa transformative step forward for patients living with rare diseases. Itmaintainsthe UK'shigh standardsof safety while enabling earlier, more iterative development approaches to fundamentally shift the value inflection point forinvestment, anddrive meaningful patient impact.

At the current pace of developing treatments one disease at a time, with programmes taking over a decade, it would take centuries to meet the unmet need across rare conditions. By supporting scalable, platform-based approaches and more flexible development pathways, the UK has a clear opportunity to strengthen its position as a global leader in rare disease innovation for millions of patients.

ENDS

Notes to Editors

The Rare Disease TherapiesFramework, which wasannounced in November 2025, will introduce a more flexible, risk-proportionate regulatory approach, enabling promising treatments to reach patients more quickly whilemaintainingthe MHRA's robust standards of safety,qualityand efficacy.

This work would not have been possible without the support of the Rare Disease Consortium, which is made up of:

* Patient and advocacy groups:Genetic Alliance UK, Beacon, Unique, Mila's Miracle Foundation * Academia and research:University of Oxford, Newcastle University (Rare Diseases Research UK), Great Ormond Street Hospital, UC Berkeley (Innovative Genomics Institute) * Industry:LifeArc, Catapult Cell and Gene Therapy, AstraZeneca (Alexion), Biogen, Alnylam, Ipsen, Mereo BioPharma, BIA, ABPI,Weatherden, Vertex, BioMarin,Syncona, UCB.Additionalcontributors include the Rare Therapies Launchpad. * Government / regulators:Medicines andHealthcare productsRegulatoryAgency, NationalInstitute forHealthandCareExcellence, Department forHealth andSocialCare, NHS England

The Medicines and Healthcare products Regulatory Agency (MHRA)is responsible forregulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe. All our work is underpinned by robust and fact-based judgments to ensure that the benefits justify any risks.

The MHRA is an executive agency of the Department of Health and Social Care.

For the MHRA Customer Experience Centre, please contactinfo@mhra.gov.uk

For media enquiries, please contactnewscentre@mhra.gov.ukor call 020 3080 7651.

Disclaimer: Curated by HT Syndication.