P114: Improving Patient Subgroup Representation with Real-World Data
Poster Presenter
Nora Emmott
Senior Policy Analyst
Duke Margolis Institute For Health Policy United States
Objectives
RWD has enabled the development and use of RWE that is generated and increasingly used in pragmatic trials, externally controlled trials, and observational studies. Studies using RWD/E can enable inclusion of underrepresented subgroups as there are fewer barriers to research participation.
Method
Review of opportunities for and challenges to leveraging RWD in order to estimate and measure treatment effects among and across patient subgroups.
Results
Underrepresentation of these subgroups limits the generalizability of research findings, reinforces treatment inequities, perpetuates data bias resulting from overestimations, and further excludes subgroups from benefits of the knowledge and innovation derived from research. Understanding the individual and/or combined influence of clinical and demographic characteristics among patient subgroups holds measurable value, especially in cases where such characteristics can be a proxy for observable variations in treatment effects.
Conclusion
Challenges and opportunities exist to leverage RWD to observe and measure real-world efficacy in patient subgroups. The concept of race as a social construct adds to the difficulty of collecting race and/or ethnicity, as racial categories might look different in other countries. The origin of race data is often unclear and may conflate behavioral/environmental risk factors with genetic/risk factors. A lack of consensus on a set of diversity variables often exists; researchers are unsure which variables to use to define and bring out variability. The lack of agreement on regulatory applications for ex-U.S. data in U.S. subpopulations can create challenges, in addition to difficulty in accessing RWD/E in underdeveloped countries. The lack of global racial/ethnic standardized categories; variability in race/ethnicity and sex, gender, gender identity, and sexual orientation data collection methods; and the difficulty in accessing RWD/E in underdeveloped countries all serve as barriers to engaging underrepresented groups in clinical trials, specifically groups in different countries. By considering each of these unique characteristics among patients and treatment options available to them in the real world, clinicians can make more informed decisions and provide more personalized care. Policy plays a crucial role in ensuring that patient subgroups are appropriately served by clinical research. Therefore, policies that are related to the research and development of treatments that prioritize the equitable inclusion of patient subgroups, which have previously been underrepresented in clinical research, are essential in order to ensure that medical advances are informed by all people who may need them.
