MIDD: Perspectives and Possibilities

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Presentation transcript:

MIDD: Perspectives and Possibilities Dionne L. Price PhD Director, Division of Biometrics IV Office of Biostatistics/OTS/FDA

Modeling and Simulation

Model-based Drug Development

Model-Informed Drug Development “quantitative framework for prediction and extrapolation, centered on knowledge and inference generated from integrated models of compound, mechanism, and disease level data and aimed at improving the quality, efficiency, and cost effectiveness of decision making” *EFPIA MID3 Workgroup, CPT Pharmacometrics Syst Pharamcol. (2016) 5, 93-122.

Organizational Structure

Modeling and Simulation A Challenge “It is a great challenge that in applying M&S, we should preferable be skilled in several scientific disciplines: statistics, mathematics, computations, pharmacology, medicine, economic, and others.” (Burman and Wiklund, 2011) Statistics Pharmacology Modeling and Simulation Medicine Computations

Some Areas of Potential Collaboration Biomarker qualification/validation Disease progression models Trial design Meta-analysis Sub-group analyses for confirmatory purposes Dose Selection

PDUFA VI Pilot Programs Enhancing regulatory decision tools to support drug development and review Complex Innovative Designs Pilot Program Led by the Office of Biostatistics Includes designs involving complex adaptations, Bayesian methods, or other features requiring simulations to determine operating characteristics Model Informed Drug Development Pilot Program Led by the Office of Clinical Pharmacology Excludes aforementioned designs

PDUFA VI Complex Innovative Design (CID) Develop staff capacity Conduct a pilot program Convene a public workshop Publish draft guidance Develop or revise relevant MAPPs, SOPPs, and/or review templates Objective: To facilitate the advancement and use of complex innovative designs

PDUFA VI Model Informed Drug Development (MIDD) Develop staff capacity Conduct a pilot program Convene a series of workshops Publish draft guidance Develop or revise relevant MAPPs, SOPPs, and/or review templates Objective: To facilitate the development and application of MIDD approaches

Model Informed Drug Development *Excludes designs involving complex adaptations, Bayesian methods, or other features requiring simulations to determine the operating characteristics

MIDD Federal Register Notice

MIDD Pilot Program: Priority areas 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 the 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 biomarker of interest)

MCP-Mod Fit for Purpose: A collaborative effort

Oncology dose finding: collaborative example

Summary There is a recognition of the utility and benefits of MIDD MIDD requires a multi-disciplinary effort and represents multiple opportunities/possibilities for collaboration Continued/increased dialogue and education are necessary Internal and external strategies will be needed to bridge the gaps