Will faster approvals unlock rare disease innovation?

A proposed new regulatory framework from the MHRA could help bring rare disease therapies to patients more quickly, but some biotech leaders believe faster approvals alone will not be enough to transform the sector.

The Rare Disease Therapies Regulatory Framework is designed to accelerate the journey from drug development to patient access through earlier engagement with developers and greater regulatory flexibility. Central to the proposal is an Investigational Marketing Authorisation (IMA), which would bridge the gap between clinical trials and full marketing approval.

For companies developing treatments for rare conditions, the changes are a welcome step. However, questions remain over whether the UK offers sufficient incentives to encourage investment in a field where scientific risk is high, patient populations are small and development costs can be substantial.

Around 3.5 million people in the UK are affected by a rare disease, yet only a small fraction of the approximately 10,000 known conditions have an approved treatment. Many are caused by mutations in a single gene, making them attractive targets for highly specialised approaches such as gene editing and antisense oligonucleotides.

Dan Williams, CEO of SynaptixBio, believes the proposed framework could reduce development costs and help smaller companies navigate the regulatory process more efficiently. However, he argues that the UK still lacks the financial and commercial incentives available elsewhere.

“The FDA’s Priority Review Voucher scheme has no equivalent here,” he said. “While the new framework could help reduce costs, it does not create the same opportunities for companies to recoup investment.”

The comparison highlights a longstanding challenge for the UK life sciences sector. In the US, orphan drug policies have helped stimulate investment through a combination of market exclusivity, tax incentives and transferable review vouchers. Supporters argue that such measures have played a significant role in driving rare disease innovation.

As the MHRA consultation continues, the debate is likely to extend beyond regulatory efficiency. The broader question is whether the UK can create an environment that not only accelerates approvals but also attracts the investment needed to develop therapies for some of the world’s most underserved patients. With the consultation due to conclude at the end of July, stakeholders now have an opportunity to shape a framework that could influence the future of rare disease drug development in the UK.

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