With increasing availability of large, real-world data (RWD) sources and advances in the application of RWD to support regulatory decision-making, professional experts and societies have developed varying guidance for data assessment, study design, and reporting. Most guidance has been explicitly or implicitly developed for “Hypothesis Evaluating Treatment Effectiveness” (HETE) studies using RWD, that are generally used for regulatory or coverage decision-making. Yet, HE&OR activities comprise numerous study types serving a variety of use cases. The ISPOR Institutional Council (IC) identified guidance for non-HETE studies such as exploratory or descriptive studies as a potential development need. To address this, the IC has convened a working group to assess how existing guidance developed to support HETE studies may be applicable to a broader range of study types, and identify gaps where additional guidance may be needed. This effort is intended to highlight considerations for research conduct, improve the quality of research, optimize resources, and increase transparency of all study types.

This forum will present the work of the IC through the perspectives of different stakeholders as well as solicit forum participant feedback on the initial findings. The forum will begin with a brief overview of the study types of interest, a summary of prominent guidance and best practice literature supporting research using RWD, and an overview of how current guidance is applicable to non-HETE studies. The process for determining if gaps exist will also be discussed. A panel discussion will follow to provide a range stakeholder experiences in conducting research using RWD. Finally, an opportunity for attendees to provide their own perspectives on the conduct of non-HETE RWD research will help inform next steps.