MHRA set to overhaul the UK’s rare disease drug regulatory pathway
The medicines regulator will aim to take a more flexible licensing approach for the research and manufacture of rare disease therapies in the UK.
The Medicines and Healthcare products Regulatory Agency (MHRA) is aiming to make sweeping changes to the UK’s regulatory pathway for rare disease drugs to speed up and ease their testing, manufacture and approval.
Central to the changes outlined in the regulator’s new policy paper is addressing the rare disease sector’s unique barriers getting therapies to patients. These obstacles include conducting clinical trials with a limited patient population, and the subsequent issues with recruitment and overall expense.
the MHRA aims to create flexible, science-led regulatory pathways that speed up clinical trials and product approvals for rare disease therapies, while upholding the highest standards of safety, efficacy, and quality”
Dr Jacqeline Barry, Chief Clinical Officer at the Cell and Gene Therapy Catapult, said: “Through this initiative, the MHRA aims to create flexible, science-led regulatory pathways that speed up clinical trials and product approvals for rare disease therapies, while upholding the highest standards of safety, efficacy, and quality.”
Other key recommendations in the MHRA’s policy paper includes issuing an early, single approval for both a clinical trial application and marketing authorisation based on compelling but limited evidence. Such an approval would be granted with a strict safety monitoring plan in place and a timetable for review of its real-world evidence base.
The paper also proposes measures to:
- Better share evidence in the UK and globally to pool scarce data
- Strengthen post-market surveillance
- Improve health system alignment in the UK and internationally.
Julian Beach, MHRA Executive Director, Healthcare Quality and Access, said: “The UK has the ingredients to be a global leader in rare disease therapies, with a rich academic base, a single provider of genomics and the unique, diverse datasets of the NHS. The challenge is bringing all these elements together, which our new framework will do.”
MHRA’s proposed framework is supported by the new Rare Disease Consortium, of which members include pharma companies such as AstraZeneca (Alexion), Biogen and Ipsen, regulators like the MHRA and NICE, and industry bodies including Catapult Cell and Gene Therapy, BIA and ABPI.
Full publication of the MHRA’s proposed policy framework is scheduled for next year, when it will add further weight to the UK Government’s life sciences strategy. Published in July, the strategy aims to support the sector’s growth, innovation, attraction for investment.
