NorthWest Biotherapeutics Inc (NWBO)

[Lykiri]

On 11 April 2025, the UK Parliament approved the Medicines for Human Use (Clinical Trials) (Amendment) Regulations 2025, marking the most significant update to the UK’s clinical trials framework in over two decades. A 12-month implementation period began on that date, with the new regulations scheduled to come into force on 28 April 2026, following a minor technical delay during the final processing of the legislation.
The deadline for stakeholder feedback on the new legislation is 30th July 2025.

At the time of publication, the MHRA announced that further draft guidance documents would be issued in the months ahead to help sponsors and researchers prepare for the new regulatory environment. As stated in the agency’s communication:

“Work to bring in the new rules will continue over the coming months, backed by updated guidance and ongoing engagement with trial sponsors and researchers.”

In that context, I believe the MHRA draft guideline on the use of external control arms based on real-world data to support regulatory decisions, published on 20 May 2025, represents one of these supporting documents. The guidance offers detailed points to consider regarding protocol design, estimands, bias mitigation and the appropriate use of real-world external control arms, in line with the principles of the new framework.

Accordingly, I expect that this specific guidance will also come into force around 28 April 2026, alongside the broader implementation of the new clinical trial regulations.

Clinical Trials regulations signed into law.
https://www.gov.uk/government/news/clinical-trials-regulations-signed-into-law

Regulatory Round-up - June 2025
https://ct.catapult.org.uk/news/regulatory-round-up-june-2025

Euro Roundup: MHRA seeks feedback on using external control arms based on RWD.

The draft provides points to consider about the protocol, pre-specification and addressing bias that apply to both trial designs, although the focus is on fully external control arms. MHRA expects the protocols for the trials to “be of the same standard, style and level of detail” as for traditional randomized trials. The protocols should pre-specify the objectives, data to be collected, endpoints and analysis methods.

Adding an external control arm after the analysis of a single-arm trial “may inflate type I error if it is not clear which was the primary analysis,” MHRA said, but the agency is open to incorporating RWD after pre-specification in some cases. If an appropriate external dataset was not available when the trial was designed, a sponsor can amend the protocol, justify the switch and note the change in regulatory filings.

MHRA began the consultation on International Clinical Trials Day. The agency and UK government used the day to highlight work to create a faster, more effective clinical trials system, notably the move to a new regulatory framework that began last month.

https://www.raps.org/news-and-articles/news-articles/2025/5/euro-roundup-mhra-seeks-feedback-on-using-external

NHS 10 Year Plan commits to early MedTech adoption and roll-out.

To make sure patients in England are able to access medicines faster, by April 2026 MHRA and NICE will launch a new joint process, supported by information sharing and joint scientific advice, that will boost the speed of decisions and cut administrative burdens for the system and industry. The challenge for the sector will be to engage to ensure that evidential hurdles are not set unrealistically high, and that real world data and evidence is taken fully into account.

https://med-techinsights.com/2025/07/03/nhs-10-year-plan-commits-to-early-medtech-adoption-and-roll-out/