Key Takeaways
· MHRA plans bold reforms to accelerate rare disease therapy approvals, aiming to streamline regulatory processes and reduce barriers for innovative treatments.
· Only 5% of rare diseases currently have approved therapies, leaving millions without options and highlighting the urgent need for change.
· Proposed framework introduces single-step approvals and enhanced data-sharing, supported by strict safety monitoring and real-world evidence reviews.
Context
A recent paper from the MHRA outlines the UK’s commitment to reforming regulatory guidelines for rare therapies, with the full publication expected to be released next year.
The Medicines and Healthcare products Regulatory Agency (MHRA) is seeking to make it quicker and easier for rare disease therapies to get tested, manufactured, and approved. The new paper detailed the ambitious strategies to support the Government’s plan.
The key component of this reform is to overcome the niche barriers that presently prevent life-changing rare disease therapies from reaching patients like small patient numbers, complex evidence generation, and maintaining safety. The principal reasoning behind the overhaul is that only 5% of rare diseases have approved treatment.
Challenges
Approximately 3.5 million people in the UK live with a rare disease and only 5% of rare diseases have an approved treatment. Furthermore, the average diagnostic journey takes 5.6 years and almost a third of affected children die before they reach five years old.
Given that patient populations are small and scattered it is difficult to recruit participants and organise and conduct clinical trials. These complexities contribute to a limited scientific understanding of the conditions because they make gathering evidence much harder. Alongside this, companies feel less inclined to invest due to a lack of financial incentive.
Currently, each new therapy requires a separate route through regulatory approval, including an approval to set up the clinical trial and an additional approval to market the therapy in the UK. This process is costly and inefficient, making it unfeasible to develop several highly targeted treatments.
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.”
He added, “There is still more work to be done, but I hope this paper reassures all those affected by rare disease that we are listening and are prepared to take bold action to speed the path from discovery to delivery, while maintaining strict standards of safety.”
Potential Solutions
To support the development of the reforms is the newly formed Rare Disease Consortium, which comprises patients and their representatives, academics, and industry.
The paper will put forward the idea of an early and single approval for both a clinical trial application and marketing authorisation. Approval will be dependent on compelling but limited evidence and granted with a thorough and strict safety monitoring plan and frequent real-world evidence reviews.
Additionally, the paper will also highlight better evidence-sharing practices in the UK to effectively pool scarce data, the significance of strengthened post-market monitoring, and better health system alignment in the UK and beyond.
