Major Overhaul of UK Rare Disease Regulation: MHRA Proposes Enhanced Framework

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has announced its intention to introduce sweeping reforms for accelerating rare disease therapy development and approval, with the aim to position the UK as a global leader in the field. The new framework will put in place flexible licensing for rare disease therapies with adaptive trial design and a new “investigational marketing authorization.” It will also ensure a patient-centered approach with an emphasis on transparency,…
By: Morgan Lewis – As Prescribed