Rare Cancers
The question
To ask the Secretary of State for Health and Social Care, what progress his Department has made on reviewing regulations of treatments for rare diseases and orphan drugs as required by the Rare Cancers Act 2026.
Answered by Preet Kaur Gill
Following the launch of the new rare disease therapy framework, the Department has made significant progress in reviewing and proposing updates to the regulation of treatments for rare diseases and orphan drugs. Building on earlier policy commitments, the Medicines and Healthcare products Regulatory Agency has introduced a proposed Rare Disease Therapies Regulatory Framework, now subject to public consultation, which sets out a fundamentally new, flexible and risk‑proportionate approach to developing and licensing treatments for conditions affecting small patient populations. This includes measures such as an Investigational Marketing Authorisation to support earlier patient access to promising therapies based on limited but compelling evidence, alongside strengthened use of real‑world data and ongoing monitoring.
The framework has been developed in close partnership with patients, clinicians, and industry, and is intended to accelerate access to innovative treatments while maintaining robust safety standards. More information on the framework is available at the following link:
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