August 29, 2018
“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,” said FDA Commissioner Scott Gottlieb, M.D. “We’ve already taken steps this month to advance policies to make the development process more modern and efficient, including issuing draft guidance on the use of seamless clinical trials to expedite development and the use of alternatives to placebo designs for new cancer therapies.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. The aim is to develop more efficient strategies to assess the safety and efficacy of medical products earlier in the development process and to adopt innovative techniques that help make clinical trials more cost efficient and flexible, enabling innovators to advance new approaches to care. For example, adaptive clinical trial designs can allow us to more quickly learn which patients are most likely to benefit from a treatment, or experience a side effect. They can make it easier to target drugs to patients who are more likely to experience a benefit, based on the biological characteristics of their disease. Other types of innovative trial designs may involve simulations or multiple types of adaptations to help optimize product development even in smaller populations, or the use of natural history models to supplement or replace the use of placebo arms. The FDA has invested, for example, in the development of natural history models for a number of rare diseases; and we’re seeking additional resources for these efforts as part of the President’s budget. The pilot program we’re announcing today is an idea incubator to support another new effort aimed at these same goals. Through interaction between FDA and pilot participants, this program will help accelerate the development of products in areas of unmet need, and ensure knowledge exchange to help advance the scientific community’s approach to designing clinical trials overall, while maintaining our gold standard for conducting high quality, well-controlled clinical trials that produce reliable results to assess safety and efficacy.”
Today, the U.S. Food and Drug Administration announced a new pilot program (“Complex Innovative Designs Pilot Meeting Program”) in which drug and biologic companies who participate in the pilot program will have additional opportunities to meet with agency staff to discuss the use of novel complex innovative trial designs (CID) for their clinical development programs. Complex innovative trial designs includes 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 determiniations, and other novel designs. The new program is aimed at helping to solidify the science used to support these novel approaches, and promote their adoption in drug development programs where these trial constructs can advance innovation.
These meetings are intended to help stimulate and inform individual product development programs and propose innovative trial designs to inform regulatory decision making. It is often the case that the use of novel approaches to clinical trial designs requires earlier and more intensive feedback from the agency to help advance these opportunities. In meetings granted under this pilot program, FDA staff across disciplines within the Center for Drug Evaluation and Research (CDER) and Center for Biologic Evaluation and Research (CBER) will provide advice and direction on how a proposed CID approach can be used in a specific drug development program.
To maximize the lessons learned through the program, the FDA is committed to sharing innovative CID approaches considered through this pilot program with the broader scientififc community through public presentations, including trial designs for drugs that have not yet been approved by FDA. The hope is that these pilot trials will inform future trial designs by companies who did not participate in a pilot program meeting. Meetings under the pilot program will be conducted through the fall of 2023.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products..