Characterizing and Communicating Uncertainty in the Assessment of Benefits and Risks of Pharmaceutical Products Workshop Summary (2014) / Chapter Skim
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2 Identifying and Characterizing Uncertainty
2 Identifying and Characterizing Uncertainty
Pages 9-18
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From page 9... ...
• Several dimensions of uncertainty are inherent to population based benefit–risk assessments, and operational factors can exacerbate them. • Finding an approach that can combine results of studies with different designs and data sources might provide a clearer picture of benefits and risks and reduce uncertainty.
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These approaches included finding a scientifically acceptable method to bridge the gap between randomized trials, which focus on proving drug efficacy in a study population, and observational studies, which focus on risk and adverse events in the real world. KEY SOURCES OF UNCERTAINTY IN BENEFIT–RISK ASSESSMENT AND ASSOCIATED CHALLENGES1 Tarek A
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This reduces the value of RCT results outside the trial population. Also, the standard length of a clinical trial is generally too brief to anticipate adverse events with long latency periods, such as in drugs that treat chronic conditions.
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, Stanford University School of Medicine, presented one promising innovative retention approach that could help to optimize both high and non-differential retention of subgroups. An ongoing weight loss study at SPRC involves educating potential participants prior to randomization about research 3 This section is based on presentations by Deborah A
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a This section is based on the presentation by Tarek A Hammad, Executive Director, Epi demiology, Merck Research Laboratories, Merck & Co., Inc.
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RCTs are typically the source for information about benefit or efficacy, while large observational claims data studies are typically the source for information about adverse events in real-world populations. To collectively provide the most valid, comprehensive, and affordable information for decision makers, Schneeweiss said, a benefit–risk assessment should include evidence from multiple study types with different data sources, intelligently arranged to maximize information available to a decision maker while complementing each study's methodological weaknesses.
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The RCT conducted prior to market authorization would include health claims or electronic health record data for all trial participants prior to the start of the trial. Obtaining these data, with the permission of patients, facilitates the characterization of the RCT patient population via the same data source used in a later observational study, thereby maintaining the comparability of subgroups across study design.
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F Rehnborg Professor in Disease Prevention, Stanford University; and Professor of Health Research and Policy, and Director of SPRC, Stanford University School of Medicine, presented results from approaches he has used to assess the internal and external validity of RCTs.
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Subjective outcomes tend to be affected the most by the validity of study design, ranging from 20 to 30 percent. As some workshop participants had also noted, Ioannidis argued that to address external generalizability, a trial needs to be compared against other trials done in the same field on questions that are relevant.
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