MHRA proposes pairing trial authorization with market access for very rare diseases

MHRA's new Rare Disease Therapies Framework introduces a dedicated marketing authorization route for therapies treating very rare diseases. It’s designed to build flexibility into premarket evidence requirements and reduce development risks and costs enough to make the U.K. a differentiated destination for rare disease drug development.

The proposed changes are innovative, and they could do more to encourage rare disease drug development than initiatives from other regulatory bodies. If they work as intended, diseases currently considered too rare to be commercially viable could come back into scope. But where the new line falls between rare and too rare for drug developers will likely depend on how much flexibility regulators accept in practice, and the path to reimbursement...