MHRA to announce guidance for faster approval of rare disease therapies

The rulebook for rare disease therapies 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 published today supporting the ambitions of the Government’s life sciences strategy.  

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

For details, see:

• Press release: Major change for rare disease treatments on way, signals MHRA - GOV.UK
• Full policy paper: https://www.gov.uk/government/publications/rare-therapies-and-uk-regulatory-considerations
• Coverage in the Sunday Times: https://www.thetimes.com/uk/healthcare/article/new-drugs-to-be-approved-faster-so-patients-dont-wait-years-k6qtfp839