ABSTRACT
The predominant reporting of clinical trial results today is what we might call the ''regulator's analysis.'' This analysis meets requirements that regulators have set for their work. Shouldn't there also be an analysis for other stakeholders, for example, physician's analysis, a thirdparty payor's analysis, a patient's analysis, and other analyses? Different analyses for different stakeholders appearing in different journals would allow Bayesian analyses, simulation results, effectiveness data, realworld evidence, more sophisticated safety analyses, and so on to also be published. The need for analyses for different stakeholders has been heightened by the various stakeholders involved in the analysis of COVID-19 trials and the current attention to estimands and concern about the proportional hazards assumption in survival analysis. This session will present the case that ''persuasive evidence'' is defined differently by different clinical trial stakeholders and provide some examples of innovative clinical trials analyses. Chair and Org: Greg Ball, Merck [greg.ball@merck .com] Title: Is There Only One Analysis? Jay Herson, Johns Hopkins Bloomberg School of Public Health [jay.herson@ earthlink.net]. Summary: The main output of clinical trials is to yield persuasive evidence. However, our notion of persuasive evidence has changed over time. The intent-to-treat frequentist analysis preferred by regulatory agencies may no longer be considered persuasive to other stakeholders. This talk will motivate the need for and provide examples of additional analyses that are useful for other stakeholders such as physicians, patients, and insurers. Title: Safety Analysis: A Physician's Perspective Barbara Hendrickson, AbbVie [barbara.hendrickson@ abbvie.com] Summary: Clinical trial safety data presentations are typically confined to rates of different categories of adverse events (e.g. common, fatal, serious, and those leading to discontinuation). However, other types of questions are important to healthcare providers and patients. Specifically, information about time to onset, reversibility with and without intervention, and additional measures of severity by functional impairment or treatments required is frequently asked questions. Also, more meaningful risk factor analyses as well as information about the likelihood for key adverse reactions in different patient subpopulations (e.g. by age and renal impairment) are needed. Title: Efficacy Analysis: Beyond the Tyranny of the p-value Michael Gaffney, Pfizer [michael.gaffney@pfizer. com]. Summary: Clinical trials are usually large, expensive and not repeated. The adherence to statistical rules often impedes the interpretation and dissemination of the strength of clinical trial results. These rules serve the legitimate objectives of regulators and medical journal editors. However, the emergence of personalized and targeted therapy;the relatively new concept of estimands;the increasing use of adaptive designs;and real-world evidence all provide an opportunity for a new paradigm which should lead to a fuller and integrated understanding of clinical trial results. This new paradigm requires reducing the role of hypothesis testing/ decision-making and increasing the role of estimation and uncertainty. Discussant: Elizabeth Garrett-Mayer, American Society of Clinical Oncology [Liz.Garrett-Mayer@asco.org].