Major change for rare disease treatments on way, signals MHRA

New paper published today commits UK to major reform in rare therapies

Bold new rulebook for rare therapies is being written, for publication next year

≈3.5 million people in UK affected by rare disease but only 5% of rare diseases have approved treatment

MHRA reforms will speed up path for rare therapies from discovery to delivery

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