The ISPOR RWE Summit 2023 offers attendees an in-depth examination of key issues surrounding real-world evidence (RWE) in regulatory decisions and health technology assessment. The scientifically rigorous program will be centered around the theme of data quality and fit-for-purpose use and will delve into studies that showcase the utilization and effectiveness of RWE in healthcare decision-making. Specific topics will include developments in data quality frameworks, validation, determination of fit-for-purpose, study tools and transparency, and analytic issues affecting decision-making.

Duke-Margolis Center staff, including Rachele Hendricks Sturrup and Adam Aten, will be presenting at this year's summit.

More Background
The RWE Transparency Initiative collaboration of ISPOR, The International Society for Pharmacoepidemiology (ISPE), Duke and the National Pharmaceutical Council, started in 2018, resulted in the creation of a registry for study protocols for RWE “hypothesis-evaluating treatment effectiveness” studies, which was piloted in 2020 and, after some revisions, formally launched in fall 2021. It is akin to clinicaltrials.gov but is designed to be much more fit-for-purpose for retrospective database studies that are meant to test a treatment effectiveness hypothesis. Its goal is to help engender trust in such studies by having their protocols posted prospectively.

Another aspect of concern about RWE studies is data quality, and there has been much discussion – including in FDA and EMA draft guidances – about what constitutes fit-for-purpose real world data for regulatory use and whose responsibility it is to validate those data (i.e., the sponsor or the data provider). Duke-Margolis has pursued significant work in this area, and a number of ISPE and ISPOR members have been involved in their advisory committees.

Establishing credibility and use of RWE for healthcare decision-making, especially at regulatory/HTA levels, and getting payers to pay attention to external studies are long-term goals for our societies. It takes not only internal efforts but also substantial communication, discussion, and prodding/incentivizing of stakeholders to make progress in such efforts. Creating an event to present and discuss our work in these areas – with some “influencers” like journal editors and government officials, potentially including both US and EU – taking part, is part of the way we can “accelerate” the take-up in this area. A public event also allows for press coverage to broaden its reach.