Proposed overhaul of UK’s rulebook for rare disease therapies

The rulebook for rare disease treatments will be overhauled to make it quicker and easier to get these therapies tested, manufactured and approved in the UK, the Medicines and Healthcare products Regulatory Agency (MHRA) has announced in a new paper supporting the ambitions of the government's life sciences strategy.

Central to this reform will be tackling the unique barriers that currently prevent rare disease therapies from reaching patients, like small patient numbers and difficult evidence generation, while maintaining safety. 

Huge unmet need