New proposal seeks to fast-track rare disease treatments in UK
Health officials invite patients and caregivers to voice thoughts on rolling reviews
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The Medicines and Healthcare products Regulatory Agency (MHRA) is seeking public feedback on a proposal to revamp how rare disease treatments are developed in the U.K. The regulatory body wants input on a new plan that would streamline the clinical testing and approval processes to help get therapies to patients much faster.
“For the millions of people in the U.K. living with a rare disease, and for the families and carers who support them, the search for effective treatment can be long, exhausting and deeply uncertain,” Sharon Hodgson, Public Health Minister, said in a press release. “These landmark proposals represent an important step towards a more agile and compassionate system — one that recognizes the unique challenges of rare disease research while maintaining the highest standards of patient safety.”
Developing new treatments for any disease is a time- and resource-intensive process. It often takes more than a decade from the time a therapy is first developed until it is approved for commercial use.
Traditionally, drug developers first conduct preclinical tests to establish that a therapy is safe enough to test in humans. Developers can then apply to begin human testing, which usually begins with small-scale tests in a limited number of volunteers to assess the experimental treatment’s safety and pharmacological profile, followed by larger trials aimed at proving the treatment is effective. Only after all that clinical testing is complete can developers apply to make a therapy available commercially.
Overcoming the challenges of rare disease trials
However, for rare diseases like AADC deficiency, this setup is often impractical or even impossible. Because these conditions affect only a small number of people, running the large clinical trials that are standard for more common conditions is extremely difficult. Given that companies typically invest millions in drug development, many pharmaceutical developers simply don’t allocate resources to treatments for rare diseases.
At the core of the new proposal is a framework to streamline the development process for rare diseases. Instead of requiring two separate applications — one to start clinical testing and another later for commercial approval — the new framework would combine everything into a single process. The new application would begin when developers request permission to start clinical testing, and trial results could be added to the application on a rolling basis, aiming to allow full approval without requiring a separate step in the regulatory process.
Members of the public can voice their thoughts on the proposal until July 30. The MHRA is especially hoping to garner feedback from people who are themselves affected by a rare disease, not just drug developers and people who already work within the system.
“By helping innovative therapies reach patients faster, this framework has the potential to transform lives, strengthen the UK’s position as a global leader in life sciences, and give renewed hope to families who have waited too long for progress,” Hodgson said. “I encourage patients, clinicians, researchers and carers to take part in this consultation and help shape the future of rare disease treatment in the U.K.”
The new proposal has received vocal support from a wide range of public health officials, lawmakers, researchers, and rare disease advocacy groups.
“For many rare condition communities, innovative treatment development has been out of reach. That’s changing with this programme, as more treatment paradigms are opened up and lower prevalence levels become more commercially viable,” said Nick Meade, chief executive of Genetic Alliance UK, a group that advocates for people in the U.K. with rare genetic diseases. “Now it’s time to make sure the detail is right, so that we can be sure the UK rare condition community can be the first to benefit from this welcome and ambitious undertaking.”
Other leaders within the medical research community emphasized that updating these outdated regulatory pathways is long overdue.
“For families affected by rare diseases, time matters. But even when there’s real promise in the science, treatments can take far too long to reach them because the regulatory pathways were not designed with rare diseases in mind,” added Sam Barrell, CEO of the life science medical research charity Lifearc. “This consultation is a chance to design a better way forward: one that enables therapies to reach patients faster without compromising safety. We’d encourage companies, researchers, clinicians and patient groups to get involved so the final framework reflects their expertise and experience.”
