We have responded to the Medicines and Healthcare products Regulatory Agency's (MHRA) consultation on aproposed regulatory framework for rare disease therapies. The proposals aim to support the development of treatments for very rare conditions by creating a regulated pathway that enables earlier patient access while continuing to generate evidence on safety and effectiveness. In our response, we support the principle of bringing more rare disease treatments into a regulated, evidence-generating pathway rather than relying on off-label use. We highlight that a regulated route can provide stronger safeguards for patients through ethical review, safety monitoring, transparency and structured evidence generation.
UK Health Research Authority · August 03, 2026
Health Research Authority response to MHRA rare disease therapies regulatory framework consultation
Original title: Our response to the MHRA rare disease therapies regulatory framework consultation
HRA supports MHRA's proposed regulated pathway for rare disease therapies to enable earlier patient access while generating safety and effectiveness evidence.
Published by UK Health Research Authority. HealthSignal links to the original document; the summary above is extracted from the source text.