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FDA Urges More Valid HCT/P Research with Agency Assistance

September 30, 2019

To help increase the number of effective trials of regenerative medicine therapies, the FDA will work with researchers and sponsors to focus on the populations, endpoints and designs best suited to prove success.

Approximately 5,000 regenerative medicine trials are being conducted under the watch of the Center for Biologics Evaluation and Research (CBER) and Center for Device and Radiologic Health, but the agency is working to encourage more legitimate research in the area to combat the rise of untested treatments that put the public
at risk.

The agency’s overall goal is to protect the public against bogus therapies that have not been proven in a clinical setting, such as the accidental blinding of two patients after an untested HCT/P was injected into their eyes in March 2017.

To help allay HCT/P manufacturers’ fear of rejection by the FDA, the agency in June started the Rapid Inquiry Program for HCT/Ps, which gives manufacturers a “rapid, preliminary, informal, non-binding assessment” of how their research could be regulated.

To further encourage clinical research, the agency issued a draft guidance in February that promotes the use of innovative regenerative medicine trial designs (CenterWatch Weekly, Feb. 18, 2019).

Despite the encouragement, the agency has seen only modest progress among sponsors, says Barbara Blumenfeld of Buchanan Ingersoll & Rooney PC.

“There’s understandably been some hesitation from what I have heard from some entities to come forward to FDA,” she said, because “sometimes you might be afraid to hear the answer.” Sponsors should view the FDA as a partner in their development plans, rather than see the agency as a watchdog.

Clinics and physicians using regenerative medicine therapies should think about starting their own investigator-initiated research projects, applying to the FDA for an investigational drug or biologics license and actually conducting a clinical trial, Blumenfeld says. “I think really what it boils down to is that anyone involved, including physicians, have to take an honest look at the practices that they’re engaged in.”

In addition to promoting valid regenerative medicine trials, regulators are concerned that the term “clinical research” may be misused to market untested treatments that don’t meet regulatory standards, Blumenfeld says. The federal database ClinicalTrials.gov is considering changing the way it characterizes its listings to clarify the difference between listings of legitimate trials that are part of an FDA-granted investigational new drug or biologics license application and listings by for-profit stem cell clinics that aren’t conducting sanctioned research.

Some clinics may submit listings to the database just to say that “Hey, we have a trial listed here, and therefore, it’s great,” Blumenfeld says. The concern is that such listings on a federally sponsored database could imply federal government endorsement or approval.

By Colin Stoecker