New FDA Pilot Program to Support Complex Trial Designs in Drug Development

The FDA launched a pilot program designed to advance innovative clinical trial designs. The program is part of the agency’s larger efforts to reform drug development processes and promote innovation in drugs that target unmet needs in the cancer treatment space.

FDA Commissioner Scott Gottlieb, MD, described the pilot as an “idea incubator” that brings together representatives from select drug and biologics companies with FDA staff from across disciplines to discuss more effective and efficient practices for complex clinical trials. The project is called the Complex Innovative Designs Pilot Meeting Program.

“The adoption of novel clinical trial designs and methods for analyzing data are a key to advancing innovation in the development of drug and biologics for hard to treat medical conditions,” Gottlieb said in a statement issued by the FDA.

According to an FDA press release, complex innovative trial designs (CIDs) include

• the use of seamless trial designs,
• modeling and simulations to assess trial operating characteristics,
• the use of biomarker enriched populations,
• complex adaptive designs,
• Bayesian models and other benefit-risk determinations, and
• other novel designs.

In the proposed meetings, which are scheduled to take place over a period of 5 years, FDA staff participants will meet with select pharmaceutical and biologics companies’ representatives twice each to provide advice and direction on how a particular complex innovative trial design approach could be used to develop specific drugs, and how to comply with regulations.

By design, only a limited number of companies can participate in the pilot; however, the FDA plans to share public presentations on the approaches to trial designs discussed in the meetings with the science community at large. Also included in this program are trial designs for drugs that have not yet been approved.

Lastly, Gottlieb highlighted 2 draft guidance documents that were released by the FDA earlier in August that demonstrated the agency’s commitment to innovation and reform. The documents encouraged the use of seamless clinical trials to expedite development of new treatments and alternatives to placebo designs for new cancer therapies.

“These proposals, as well as others in development, are part of a broader program to create a new framework for how innovators can modernize their approaches to clinical development, to make it more effective and efficient,” Gottlieb said.