FDA promoting innovative clinical trial designs as part of new pilot program

By Melissa Fassbender contact

- Last updated on GMT

(Image: Getty/Pinkypills)
(Image: Getty/Pinkypills)

Related tags: Fda, PDUFA, Clinical trials

The FDA has launched a new pilot program targeting complex, innovative clinical trials designs as part of its commitment under PDUFA VI.

The US Food and Drug Administration yesterday announced​ the Complex Innovative Designs Pilot Meeting Program.

According to the agency, participating drug and biologic companies will have the opportunity to meet with agency staff to discuss novel complex innovative trial design (CID) use in clinical development programs.

The use of such designs is highlighted in the sixth iteration of the Prescription Drug User Fee Act (PDUFA VI​), which was signed into law last August as part of the FDA Reauthorization Act of 2017 (FDARA).

Per the FDA, complex innovative trial designs include: 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, among others.

The meetings – running through the fall of 2023 – are intended to help “stimulate and inform individual product development programs”​ and “propose innovative trial designs to inform regulatory decision making.”

FDA Commissioner Scott Gottlieb, MD in a statement said, “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.”

Modernizing the drug development process

The pilot program builds on steps taken earlier this month​ by the agency to advance policies to modernize the drug development process.

The FDA on August 13, 2018 published a draft guidance on expansion cohort use in first-in-human (FIH) clinical trials with the goal to expedite development of oncology drugs and biologics.

“The use of expansion cohorts in Phase I clinical trials in oncology presents an opportunity to provide benefits to more patients for whom limited curative therapies exist,”​ said Elizabeth Gallagher, director logistics global accounts, ThermoFisher Scientific.

“However the potential for a breakthrough therapy needs to be carefully considered versus the risk to the patient,”​ she told us. “Mitigation of the risk and expediency needs to the highest priority in all aspects of trial conduct from patient screening and data accuracy to the delivery of clinical trial material.”

Gallagher noted that trials with expansion cohorts demand a high level of flexibility and accuracy.

The FDA’s proposals are part of a broader program aimed at creating a “new framework”​ for how the industry can modernize clinical development to make the process 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,”​ added Gottlieb.

The Commissioner noted the FDA’s investment in the development of natural history models for rare diseases. Gottlieb said the agency is seeking additional resources for these efforts as part of the President’s budget and described the pilot program as “an idea incubator to support another new effort aimed at these same goals.”

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