Clinical Pharmacology Corner: Model-Informed Drug Development Pilot Program

FDA Announces Availability of a Notice “Pilot Meetings Program for Model-Informed Drug Development Approaches”

On April 17, 2018, the U.S. Food and Drug Administration (FDA or Agency) announced the availability of a notice entitled “Pilot Meetings Program for Model-Informed Drug Development Approaches” that affords sponsors or applicants who are selected for participation the opportunity to meet with Agency staff to discuss model-informed drug development (MIDD) approaches in medical product development. For this pilot program, MIDD is defined as the application of exposure-based, biological, and/or statistical models derived from preclinical and clinical data sources to address drug development and/or regulatory issues. 

The Prescription Drug User Fee Act of 2017 (PDUFA VI), part of the FDA Reauthorization Act of 2017 (FDARA), highlights the goal of advancing MIDD. This pilot program is being conducted to facilitate the development and application of MIDD approaches. MIDD approaches use a variety of quantitative methods to help balance the risks and benefits of drug products in development. This excludes statistical designs involving complex adaptations, Bayesian methods, or other features requiring computer simulations to determine the operating characteristics of a confirmatory clinical trial.  When successfully applied, MIDD approaches can improve clinical trial efficiency, increase the probability of regulatory success, and optimize drug dosing/therapeutic individualization in the absence of dedicated trials. 

The pilot program will be conducted by FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) during fiscal years 2018 to 2022. It consists of two meetings between sponsors or applicants and the relevant center and will provide an opportunity for drug developers and FDA to discuss the application of MIDD approaches to the development and regulatory evaluation of medical products in development.

The Agency will initially prioritize selecting requests to participate in the pilot program that focus on:

• Dose selection or estimation (e.g., for dose/dosing regimen selection or refinement).
• Clinical trial simulation (e.g., based on drug-trial-disease models to inform the duration of a trial, select appropriate response measures, predict outcomes).
• Predictive or mechanistic safety evaluation (e.g., use of systems pharmacology/mechanistic models for predicting safety or identifying critical biomarkers of interest).

The Pilot Meetings Program for Model-Informed Drug Development Approaches notice is available athttps://go.usa.gov/xQjta. Please refer to the notice for more details. Additional information on the Pilot Meetings Program for Model-Informed Drug Development Approaches (including eligibility for the program, procedures and submission information) can be found at https://go.usa.gov/xQjtx.

You may submit your comments about the MIDD pilot meetings program by May 17, 2018 to the Docket No. FDA-2018-N-1203 available at https://www.regulations.gov. Your comments do make a difference and can impact the outcomes of FDA regulatory policy. Share your knowledge and experience, and make your voice count.