FOI release

Evidence of Accelerated Regulatory Pathways for Innovative Rare Disease Therapies

Some or all of the information requested was not provided because we didn't hold it.

Case reference FOI2025/01272

Received 5 December 2025

Published 29 January 2026

Request

I am writing to submit a request under the Freedom of Information Act 2000 for specific, quantifiable records concerning the implementation and impact of the UK Rare Diseases Action Plan (2025) regarding accelerated regulatory pathways for innovative rare disease therapies. Please provide the following records: Pipeline Progress (ILAP Status): A list of all innovative rare disease products (including gene therapies) that have been granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) since January 1, 2024, including the current status of their Target Development Plan (TDP). Policy Interpretation (New UK-OD Guidance): Copies of the specific pages or sections from the internal 'Technical Guidance for Industry' draft (or latest working version) for the new UK Orphan Drug (UK-OD) pathway that use an ultra-rare disease product (e.g., a gene therapy) as a worked example or case study to illustrate the new pathway's requirements. Governance/Coordination (MoU/SLA & Alternatives): Copies of any formal Memoranda of Understanding (MoU), Service Level Agreements (SLA), or the Terms of Reference (ToR) for the primary Joint Steering Committee or Working Group tasked with coordinating the regulatory, appraisal, and funding aspects of the Rare Diseases Action Plan between the MHRA and NICE/NHS England since January 1, 2025. f any part of this request is deemed too broad, I ask that you clarify the refusal grounds and provide any records that can be separated, particularly focusing on requests 2 and 3.

Response

See attached

Documents

• FOI2025_01272 (redacted).pdf (990.6 KB)

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