MHRA opens consultation for landmark rare disease therapy draft guidance
Agency’s proposal for a new model for rare disease therapy development in the UK compresses pathways, potentially enabling faster patient access.
Following its initial announcement in November 2025, the Medicines and Healthcare products Regulatory Agency (MHRA) is seeking feedback from the pharmaceutical industry on its draft Rare Disease Therapies Framework. It outlines a flexible, risk-proportionate approach that is designed to shorten timelines and reduce costs for developing rare disease therapies.
The technology-agnostic regulatory framework is intended to address
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