Under the draft framework, adaptive and innovative trial designs would be supported including basket and umbrella trials, and hybrid designs incorporating real-world evidence. Surrogate or patient-relevant endpoints may also be appropriate where conventional endpoints are not feasible. There is also an openness to the use of computational modelling including digital twins and non-animal methods, where it can be scientifically justified.

The guidance also allows for rolling data submissions to accelerate decision making, and promotes better alignment between clinical development, regulatory approval, patient access and the reimbursement process to reduce complexity, the MHRA said.

Shaping the framework

Industry feedback is “vital” to this consultation, Julian added. “By working closely with patients, partners and industry, we’re building a framework that supports innovation while maintaining the high standards of safety that patients expect.”

The MHRA’s plans for reform “potentially” compliments NICE’s approach to enable NHS patients to receive innovative and promising medicines. That was the view shared by Helen Knight, director of medicines evaluation at the National Institute for Health and Care Excellence (NICE).

“The proposals could help to address clinical uncertainty, with the MHRA continuing stringent patient safety monitoring while NICE ensures value for money for the taxpayer,” she said.

For patients with rare diseases, regulatory timelines are not an abstract concern, explained Dr Jacqueline Barry, Cell and Gene Therapy Catapult’s chief clinical officer. “They represent the difference between accessing a potentially life-changing therapy and not. The proposed framework responds to that directly,” she said.

The introduction of single authorisation, known as the investigational marketing authorisation (IMA), has the potential to accelerate the development pathway for advanced therapies, she added. “This will enable iterative, evidence-led progression that better reflects the science, while upholding the highest standards of safety, efficacy and quality.”

Sharon Hodgson, public health minister, said that the proposals represent an important step towards a more “agile and compassionate” system. 

“One that recognises the unique challenges of rare disease research while maintaining the highest standards of patient safety.” She added that the framework has the “potential to transform lives and strengthen the UK’s position as a global leader in life sciences,” and urged patients, clinicians, researchers and parents to take part in the consultation.

The proposed regulatory framework represents a “transformative” step forward for people living with rare diseases. That is the view of Dr Harriet Holme, drug development clinician at Weatherden, and executive chair of PCD Research. It would maintain the UK’s high standards of safety while enabling earlier, more iterative development approaches to “fundamentally shift” the value inflection point for investment to drive meaningful patient impact, she said.

“At the current pace of developing treatments one disease at a time, with programmes taking over a decade, it would take centuries to meet the unmet need across rare conditions,” Harriet added.

The consultation offers an “important and hopeful step for families affected by rare diseases”, added Professor Claire Booth, a consultant paediatric immunologist at Great Ormond Street Hospital. “Too often, patients face long diagnostic journeys with limited options, not because the science isn’t advancing, but because the system hasn’t kept pace.”

Patient charities also supported calls for clinicians, academics and industry partners to contribute to the consultation to shape the new framework for rare disease therapies. 

“For many rare condition communities, innovative treatment development has been out of reach,” explained Nick Meade, chief executive of Genetic Alliance UK. “That’s changing with this programme, as more treatment paradigms are opened up, and lower prevalence levels become more commercially viable. Now, it’s time to make sure the detail is right, so we can be sure the UK rare condition community can be the first to benefit from this welcome and ambitious undertaking.”