UK clinical trial regulation may support haemophilia treatment access

The Medicines and Healthcare products Regulatory Agency (MHRA) new Notification Scheme for lower-risk clinical trials has enabled an application for a Phase IV haemophilia A clinical trial to be accepted by day 14.

The treatment, Nuwiq, is a fourth-generation recombinant FVIII is indicated for severe haemophilia A in patients undergoing major surgery while receiving emicizumab prophylaxis, MHRA explained.

Initially announced in late 2023, the agency’s new Notification scheme aims to reduce the time taken to approve the lowest-risk clinical trials by more than 50 percent. While approximately 20 percent of UK initial clinical trial applications are anticipated to be eligible, the agency urged that all clinical trial sponsors should use the new scheme for all eligible trials.

Vitally, the Notification scheme “enables a more streamlined and risk-proportionate approach to processing Clinical Trial Authorisations (CTAs) for initial applications in Phase III and IV trials”, according to MHRA’s Deputy Director of Clinical Investigations and Trials, Andrea Manfrin. 

“This case study [on the new Notification scheme] demonstrates the effective operation of the MHRA’s new, streamlined clinical trials system, in reducing the time taken to get the lowest-risk clinical trials up-and-running without undermining patient safety. Clinical trials regulation should be flexible and risk-proportionate so that the regulatory requirements are geared to the risk that a trial presents. This will help give UK patients quicker access to the potentially life-saving medicines being studied,” explained Andrea Manfrin, the MHRA’s Deputy Director, Clinical Investigations and Trials.

MHRA regulation overhaul: new scheme for lowest-risk UK clinical trials

Accessing the new Notification scheme

Clinical trial sponsors can access the scheme by applying through the Integrated Research Application System (IRAS) – the single system for applying for permissions and approvals in the UK.

MHRA highlighted that if a trial is not accepted under the scheme, the application goes through the full combined review clinical trial application (CTA) assessment route.

Full details of the new Notification scheme are available in the MHRA’s online guidance on clinical trials for medicines.