Orphan Medicinal Products in the United Kingdom

Overview of the orphan medicinal product regime

In Great Britain (England, Scotland and Wales), orphan medicinal products are regulated to support development of treatments for rare diseases and ensure patients have access to innovative therapies. This regime includes provisions for market exclusivity, fee relief, and application procedures under the authority of the Medicines and Healthcare products Regulatory Agency (MHRA). Source: Orphan medicinal products (gov.uk) ([GOV.UK][1])

The orphan framework in Great Britain has evolved following Brexit, with the MHRA now responsible for reviewing orphan designation at the time of marketing authorisation applications, rather than as a separate pre-approval designation step (a departure from the EU model). Source: Orphan medicinal products (gov.uk) ([GOV.UK][1])

Criteria and eligibility for orphan designation

To qualify as an orphan medicinal product in Great Britain, a proposed medicine must meet criteria similar to the EU’s definition of orphan drugs: it must be intended to diagnose, prevent or treat a life-threatening or chronically debilitating disease, and either the prevalence of the condition must not exceed a specified rare threshold or it must be unlikely that the product would generate sufficient return on investment without incentives. Source: Orphan medicinal products (gov.uk) ([GOV.UK][1])

Under UK practice, because orphan designation is reviewed alongside a marketing authorisation application (MAA), the applicant must demonstrate that orphan criteria are met at the time of submitting the MAA. Source: Orphan medicinal products (gov.uk) ([GOV.UK][1])

Application process

In Great Britain, sponsors submit orphan designation requests concurrently with their marketing authorisation application (MAA), and the MHRA assesses the request as part of its overall evaluation of the marketing authorisation. There is no separate early orphan designation prior to the MAA. Source: Orphan medicinal products (gov.uk) ([GOV.UK][1])

The MHRA’s Commission on Human Medicines typically examines both the MAA and orphan designation in parallel, and a decision on orphan status is issued at the same time as the marketing authorisation decision. Source: Orphan Drug Designation in the US, EU & GB ([Scendea][2])

Market exclusivity and incentives

Products granted orphan status in Great Britain will benefit from market exclusivity for up to 10 years for that specific indication. During this period, no similar product can be authorised for the same therapeutic indication unless specific exceptions apply (e.g., consent from the original orphan authorisation holder). Source: Orphan medicinal products (gov.uk) ([GOV.UK][1])

In addition to exclusivity, incentives may include regulatory fee reductions or exemptions and potential scientific regulatory support to facilitate development of orphan medicines. Source: Orphan medicinal products (gov.uk) ([GOV.UK][1])

Certain jurisdictions also consider additional incentives for paediatric indications which may extend exclusivity if agreed paediatric investigation plans are completed, although the precise mechanisms differ under UK regulations. Source: Orphan drugs in the EU/EEA and UK ([J A Kemp][3])

Regulatory context and recent changes

The implementation of the Windsor Framework and amendments to the Human Medicines Regulations 2012 will affect the UK orphan regime, with significant changes coming into force from 1 January 2025. These changes may impact how orphan medicinal products are authorised and the scope of regulatory incentives. Source: Orphan medicinal products (gov.uk) ([GOV.UK][1])

Practical impact

The UK orphan medicinal products framework aims to balance incentives for developers with patient access and public health safeguards, ensuring that rare disease treatments can be developed and authorised despite small patient populations and commercial challenges. Source: Orphan medicinal products (gov.uk) ([GOV.UK][1])

Conclusion

Great Britain’s orphan medicinal products regime continues to evolve post-Brexit, retaining core incentives such as market exclusivity while aligning procedural requirements with the UK’s regulatory framework under the MHRA. Source: Orphan medicinal products (gov.uk) ([GOV.UK][1])