DCTs ideal for confirmatory trials for fast-tracked biologics, says ACRO

The Association of Clinical Research Organizations (ACRO) has proposed that the US FDA consider decentralized trials (DCTs) in guidelines for firms seeking accelerated approval for biologics for serious or life-threatening conditions.

The US industry group suggested the inclusion this month in a written response to the draft guidance document, “Expedited Program for Serious Conditions Accelerated Approval of Drugs and Biologics,” which the FDA released in December.

ACRO wrote, “We ask the agency to consider inclusion of a discussion of decentralized clinical trials in the final guidance … Confirmatory trials may be amenable to a decentralized trial design due to the nature of the participants. The accelerated approval pathway is limited to drug development programs for the treatment of a serious or life-threatening disease or condition.

“The flexibilities that trials with decentralized elements offer to patients may be of particular value for patients with serious or life-threatening diseases and conditions who may have limited mobility and other challenges that impact trial participation.”

The organization added, “These flexibilities include the moving of trial assessments from the central investigator site to the patient’s home, local community facility, or mobile unit.”

Accelerated approval

Under the proposed rules, biopharmaceuticals granted accelerated approval must be re-evaluated in confirmatory clinical trials.

Developers that do not conduct a confirmatory study are at risk of having the approval revoked. Likewise, if the drug fails to show the anticipated therapeutic effect, the FDA can withdraw the approval.

The draft guidelines give the FDA the right to impose specific conditions for the confirmatory study related to protocol design and completion date.

In addition, the guideline covers how sponsors should deal with any impact that accelerated approval has on efforts to enrol patients in confirmatory trials.

The FDA stated, “Sponsors should mitigate the anticipated impact of accelerated approval on participant enrolment and retention by completing all or a significant portion of enrolment prior to accelerated approval.”

And this is where ACRO thinks decentralized studies could have a significant impact.

“The recommendation to ‘complete all or a significant portion of enrollment prior to accelerated approval’ may be more easily achieved in a trial with decentralized elements that offers participants locational flexibilities for assessment procedures,” the organization wrote.

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