Finally, our panel will interact with the audience on various protocol influences of believability and how larger effect sizes, fit-for-purpose data for the specific research question, and transparent analytic techniques minimize the risk of various biases, thereby maximizing believability and robustness of results. Pre-specified replicability approaches may also enhance believability. Special note will be made on using confidence intervals over p-values for interpretation, as the latter are driven by larger sample sizes typical in RWE.
A pre-specified statistical analysis plan, including statistical assumptions verification, sensitivity analyses, and bias quantification techniques, are essential to ensure believability. Techniques to prevent or minimize information bias will be discussed, such as ensuring that data are complete, reliable and relevant for the specific research objective (i.e., fit-for-purpose). Designs and methods that minimize confounding are preferred (e.g., new-user active comparator design, propensity score approaches). For the RWE & Regulatory Decisions workgroup of ISPE RWE Task force, we will review approaches to improve believability of RWE to regulators and health authorities.ĭescription: Foundational to believability are well-articulated pre-specified objectives, study designs and analysis plans detailed in registered protocols. Attendees should expect to learn more about what health authorities need to evaluate robustness and believability of RWE results. Objectives: To discuss various study designs and methods fostering believability of RWE to guide informed regulatory decisions. However, what makes RWE results believable to health authorities is not widely understood among pharmacoepidemiologists. The FDA recently issued a data quality framework and regulators globally are working on guidelines to use RWE for regulatory purposes. SYMPOSIA Believability of real-world evidence (RWE) for informed regulatory decisions Jacinthe Leclerc 1 Kimberly G Brodovicz 2 Stephen Chang 3 Magdalene M Assimon 4 Hisashi Urushihara 5 Cynthia J Girman 6 1Universite du Quebec a Trois-Rivieres 2Boehringer Ingelheim 3FDA 4University of North Carolina School of Medicine 5Keio University 6CERobs Consulting, LLCīackground: Regulators are increasingly considering RWE of medical products to support new indications or expand labeling.
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