Real-World Evidence

rwe pohoto

Real-World Evidence

As thought leaders in RWE, Duke-Margolis engages public and private stakeholders in this burgeoning field. Read on to find the publications, projects, news, and contact information of our real-world evidence team.

Randomized controlled trials (RCTs) are traditionally used by the US Food and Drug Administration (FDA) to evaluate a product’s safety and effectiveness. While they are the gold-standard from a research perspective, RCTs are resource-intensive and require investing significant financial, time, and human capital. Coupled with unmet clinical needs and drug-access challenges, the demands of RCTs have fueled a call to action from patients and legislators. The regulatory system needs new ways to optimize the drug development process, so the delivery of effective drugs to the marketplace accelerates—while public safety remains ensured.

A large amount of patient information is captured at the point of care. Health care stakeholders are enthusiastic to explore whether that information can inform and hasten new drug development. Called real-world data (RWD), analyzing this burgeoning data source has the potential to provide real-world evidence (RWE). In turn, that RWE can tell us about the use of drugs, drug outcomes, and it can help us understand whether drugs are performing as intended in routine care.

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 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.
To inform FDA as it explores RWE and regulatory decision-making, the Duke-Margolis Center for Health Policy RWE Collaborative engages stakeholders to guide high priority efforts aimed at improving the development and use of RWE.