STORY
The Medicines and Healthcare products Regulatory Agency has launched a consultation on a proposed framework intended to make it quicker and less costly to develop therapies for rare diseases. The regulator said the new approach could help patients with rare conditions access treatments earlier where conventional development routes are difficult.
The proposed Rare Disease Therapies Regulatory Framework includes plans for an Investigational Marketing Authorisation and compressed development pathways. The MHRA said the framework is designed to address scientific, evidentiary and commercial barriers that can prevent rare disease therapies from reaching patients.
The Government said rare diseases affect up to 3.5 million people in the UK. The consultation will allow the pharmaceutical and life sciences sectors, patient groups and other stakeholders to comment before the framework is finalised.