FDA marks progress in implementing real-time clinical trials

The US Food and Drug Administration (FDA) has taken two key steps to implement real-time clinical trials (RTCTs), part of its efforts to modernise of the drug development process.

AstraZeneca and Amgen have commenced separate proof-of-concept RTCTs. They will report endpoints and data signals to the FDA in real time, supported by improvements in AI and data science. These milestones enhance safety monitoring and significantly increase the efficiency of drug development.

AstraZeneca’s Phase II multi-site trial TRAVERSE is evaluating participants with treatment-naïve mantle cell lymphoma. Amgen is conducting a Phase Ib trial, STREAM-SCLC, in limited-stage small cell lung carcinoma.

With a real-time approach, the FDA met with the sponsors regarding establishing criteria for reporting real-time signals. This contrasts to the traditional model where trial data is reported from sites to sponsors, then submitted to the FDA.

The agency has received and validated signals from AstraZeneca’s trial, confirming the feasibility of the technical framework required for real-time signal sharing. Final site selection for Amgen’s study is ongoing.

“[These steps] will help us accelerate promising therapies, and build toward our ultimate goal of running real-time, continuous trials across all phases of drug development”

FDA Commissioner, Marty Makary, said: “For 60 years, we’ve been conducting clinical trials in the same way, where key data signals can take years to reach the FDA. The lag time can delay regulatory decisions unnecessarily and slow down the drug development timeline.

“We are boldly advancing a modern approach whereby FDA scientists can view safety signals and endpoints in real time as a trial progresses. This will help us accelerate promising therapies, and build toward our ultimate goal of running real-time, continuous trials across all phases of drug development.”

To build on these proofs-of-concept trials, the FDA plans to launch a pilot programme for RTCT in the summer. The agency has issued a request for information (RFI) on the programme design and implementation, alongside evaluation metrics and success criteria.

Public comment on the RFI is open until 29 May. Pilot selection is anticipated to be completed in August.

This development follows a recent US approval win for AstraZeneca. The company’s Breztri Aerosphere was authorised as the first triple-therapy inhaler for asthma by the FDA. While in February, Amgen achieved EU approval for its biologic Uplizna (inebilizumab) as a new first-in-class approach for generalised myasthenia gravis.