The FDA on Tuesday firmed up its position that in some limited circumstances, clinical trial data may be obtained from routine clinical practice. The use of such trials could potentially reduce the need for dedicated trial sites.
The 13-page draft guidance outlines the roles of each of the primary partners working on such a trial, and points to one recent example of such data serving as one of the biggest clinical trial success stories of the pandemic: the UK-based RECOVERY trial that used existing infrastructure and local healthcare providers in hospitals across the island to test the efficacy of more than a dozen Covid interventions. The results of the trial supported the FDA’s additional indication for Roche’s tocilizumab, for the treatment of some hospitalized adults with Covid-19.
But the agency notes that the draft doesn’t apply to observational studies. It does apply to studies involving FDA-approved drugs up for new indications, populations or doses, as well as safety studies for FDA-approved drugs or other postmarketing studies like comparative effectiveness. It’s unclear how many sponsors may use this approach. The draft also includes “trials of unapproved drugs in later development” when the safety profile is “sufficiently characterized” and the drug can be used in routine clinical practice.
In terms of these well-understood safety profiles, the agency points to members of an existing drug class, or drugs where safety is already well-characterized from prior trials. Sponsors should consult with the review division to determine if a particular environment is suitable for trials with these unapproved drugs, FDA says.
The draft also discusses using a “quality-by-design” approach, which includes remote or on-site monitoring by the sponsors to ensure reliable results and that participants remain safe.
The FDA on Tuesday also finalized guidance from May 2023 on clinical trials with decentralized elements.
“To help ensure the appropriate oversight trials with decentralized elements, the integrity of trial data, and the safety of trial participants, this guidance covers the responsibilities of sponsors and investigators,” FDA said, noting that changes from the draft to the final guidance include removing language regarding a requirement to maintain a task log of local healthcare providers and clarification about challenges related to data variability.