Encouraging researchers and sponsors to simplify clinical trials and employ a fit-for-purpose protocol is one way that the US Food and Drug Administration (FDA) can promote recruitment of trial populations that are racially and ethnically diverse, according to Patrizia Cavazzoni, director of the Center for Drug Evaluation and Research (CDER).
FDA has been focused on increasing clinical trial diversity, issuing draft guidance in April 2022 that calls on sponsors to develop a Race and Ethnicity Diversity Plan to enroll adequate numbers of trial participants from underrepresented racial and ethnic populations in the United States (RELATED: FDA recommends sponsors plan to include race, ethnicity in clinical trial design, Regulatory Focus 13 April 2022). While guidance is one of the agency’s tools to increase diversity, Cavazzoni said FDA can also encourage the use of innovative trial designs.
“I think that FDA [and] other agencies, including [the National Institutes of Health], have a role in striving to simplify clinical trials and getting to a point where clinical trials protocol and execution are fit-for-purpose for the questions that are being asked and not complicated by a whole bunch of things that may be intellectually interesting but do not necessarily add to what the trial is really supposed to answer,” Cavazzoni said at the 2023 Innovations in Regulatory Science Summit sponsored by the UCSF-Stanford Center of Excellence in Regulatory Science and Innovation (CERSI).
Cavazzoni said FDA has a significant role when it comes to working with researchers and companies on trial designs, statistical analysis planning, and Quality by Design (QbD) approaches.
“We need to strike the right balance when it comes to collection of information and the need to make clinical trials feasible,” Cavazzoni said. “Appropriate simplification of clinical trials’ protocols and execution is very much on the to-do list.”
Technology and legislative tools
Encouraging the use of technology that can move trials closer to patients is another focus for FDA, Cavazzoni said. In January 2022, the agency issued guidance on the use of digital health technologies for remote data acquisition in clinical investigations (RELATED: Digital health tech in clinical investigations: FDA issues draft guidance, Regulatory Focus 05 January 2022).
FDA has also sponsored research to look at developing a telemedicine version of a scale used to measure function for patients with ALS. This example in ALS “is something that really should apply to all therapeutic areas,” she said.
The passage of the Diverse and Equitable Participation in Clinical Trials (DEPICT) Act, which was rolled into the Consolidated Appropriations Act at the end of 2022, will allow FDA to “use even more muscle when it comes to improving clinical trial design and enhancing the diversity of clinical trials,” Cavazzoni said.
The DEPICT Act, co-sponsored by Rep. Anna Eshoo (D-CA), requires drug and device manufacturers to submit a diversity action plan for enrolling trial participants that reflects the populations impacted by the disease. While similar to the FDA’s draft guidance, the DEPICT Act creates a requirement for diversity planning.
“This will be the first time there’s ever been a standard for diversity in clinical trials in law. In the past, the FDA has sought to improve clinical trial diversity through voluntary guidance,” Eshoo, a member of the Health Subcommittee of the House Energy and Commerce Committee, said at the CERSI Summit.
The legislation also requires the FDA to issue guidance to promote decentralized clinical trials. “These types of trials use digital health technology and telehealth to allow for a really meaningful diverse clinical population,” Eshoo said.
Eshoo said she’s also considering legislation in the current Congress that would promote more representative trials by diversifying the clinical staff that recruits trial participants.