Characterizing Uncertainty in the Assessment of Benefits and Risks of Pharmaceutical Products Workshop in Brief (2014) / Chapter Skim
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Characterizing Uncertainty in the Assessment of Benefits and Risks of Pharmaceutical Products-Workshop in Brief
Characterizing Uncertainty in the Assessment of Benefits and Risks of Pharmaceutical Products-Workshop in Brief
Pages 1-8
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From page 1... ...
The FDA PDUFA Plan notes that identifying and evaluating sources of uncertainty in a regulatory application is an important part of an FDA new drug application reviewers' work; however, drawing conclusions in the face of uncertainty can be a complex and challenging task. Effectively communicating regulatory decisions necessarily includes explanation of the impact of uncertainty on decision making.
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Limitations include reliance on volunteers as clinical trial participants, Statement of Task An ad hoc planning committee will plan two 1-day public workshops that will address the need to advance the development of more systematic and structured approaches to characterize and communicate: (a) the sources of uncertainty in the assessment of benefits and risks, and (b)
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An ongoing weight loss study at SPRC involves educating potential participants prior to randomization about research methods, trial design, control conditions, random assignments, and the impact of dropouts. The FDA PDUFA Plan identifies the following two areas of uncertainty as warranting additional attention: 1.
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Decisions Made Under Uncertainty: Tysabri and Anoro Ellipta Case Studies Patrick Frey, FDA CDER, provided an overview of FDA's approach to evaluating benefits and risks of pharmaceutical products, with specific focus on how these approaches take sources of uncertainty into consideration. The FDA PDUFA Plan notes that systematic approaches to evaluating uncertainty is an area worthy of further consideration to inform the drawing of conclusions in the context of uncertainty.
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tells you how much uncertainty you have … in the available evidence about the question of interest." Applying decision theory approaches to regulatory decision making Several speaker presentations generally addressed decision theory techniques and the scientific basis for incorporating patient and other stakeholder preferences. Several speakers suggested that scientific methodologies for the incorporation of expert deliberation and stakeholder perspectives can help to improve certainty of forecasts, place what is known and what is unknown in a practical context, address uncertainties in the context of patient preferences, reveal new uncertainties that otherwise may have been overlooked, and provide important information on values for regulatory determinations.
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Individual workshop participants noted a number of FDA attributes and processes that currently incorporate, or could be enhanced to incorporate, the scientific methods and approaches discussed to enhance decision making under or with respect to uncertainty. • FDA structure, authorities, and processes.
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Although the focus of the workshop was on consideration of scientific methods that could be applied to the existing regulatory authorities and processes currently available to FDA, there was some discussion among individual workshop participants about regulatory approval pathways that do not currently exist but have been proposed that could address certain aspects of the uncertainty issues. For example, a workshop participant noted that the Special Medical Use (SMU)
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Jack D Keene Advancing Translational Sciences, NIH; Patrick Frey, Office of Amgen Inc., Thousand Oaks, CA Duke University Medical Center, Program and Strategic Analysis, Center for Drug Evaluation and Durham, NC Research, FDA; Tarek Hammad, Merck & Co., Inc.; Gavin HuntleyRuss Biagio Altman Stanford University, CA Rusty Kelley Fenner, Huntley-Fenner Advisors; Charles Manski, Northwestern Burroughs Wellcome Fund, Research University; Paul Seligman, Amgen Inc.; Lana Skirboll, Sanofi; Brian Margaret Anderson Triangle Park, NC FasterCures, Washington, DC Strom, Rutgers Biomedical and Health Sciences; Myrl Weinberg, Ronald L
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