PDUFA VI Public Meeting August 15, 2016

The FDA is holding a public meeting to discuss the reauthorization of PDUFA and the proposed commitment letter on Aug 15, 2016. Part of that meeting involves some important new proposals that impact the Pharmacometrics Community.

To facilitate the development and application of exposure-based, biological, and statistical models derived from preclinical and clinical data sources, herein referred to as “model-informed drug development” (MIDD) approaches, FDA will conduct the following activities during PDUFA VI:

a. FDA will develop its regulatory science and review expertise and capacity in MIDD approaches. This staff will support the highly-specialized evaluation of model-based strategies and development efforts.

b. FDA will convene a series of workshops to identify best practices for MIDD. Topics will include:
(1) physiologically-based pharmacokinetic modeling; (2) design analysis and inferences from dose-exposure-response studies; (3) disease progression model development, including natural history and trial simulation; and (4) immunogenicity and correlates of protection for evaluating 31 biological products, including vaccines and blood products. Each workshop will focus on current and emerging scientific approaches, including methodological limitations. FDA will produce a written summary of the topics discussed in each workshop.

c. Starting in FY 2018, FDA will conduct a pilot program for MIDD approaches. For sponsors participating in the pilot program, FDA will grant a pair of meetings specifically designed for this pilot program, consisting of an initial and a follow-up meeting on the same drug development issues, to occur within a span of approximately 120 days. These meetings will be led by the clinical pharmacology or biostatistical review components within CDER or CBER.

i. FDA will publish a Federal Register Notice announcing the pilot program and outlining the eligibility criteria and process for submitting to FDA requests to participate in the pilot program.
ii. FDA will select 2-4 proposals (e.g., 1-2 per Center) quarterly each year. FDA will convene an internal review group to review proposals on a quarterly basis and provide recommendations on prioritization and selection of proposals and share knowledge and experience. Program selection will take into account development programs where clinical data are limited such that integration across non-traditional sources may be needed, and for which MIDD can assess uncertainties about issues such as dosing, duration, and patient selection in a way that can inform regulatory decision-making.
iii. Sponsors who do not participate in the pilot will have an opportunity to interact with the Agency through traditional channels.

d. By end of FY 2019, FDA will publish draft guidance, or revise relevant existing guidance, on model-informed drug development.

e. By end of FY 2021, FDA will develop or revise, as appropriate, relevant MAPPs or SOPPs, and/or review templates and training, to incorporate guidelines for the evaluation of MIDD approaches.

Meeting Link

The registration is free. You can attend remotely.

The model-informed drug development (MIDD) section starts on page 30 of the letter at the link above.

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