Who Owns the Data?
The Benefits and Challenges of Real-World Data to Support Traditional Clinical Trial Initiatives
RWD’s Credibility Problem
Historically, people viewed medical research data from outside traditional clinical trials with a heavy dose of skepticism — often before anyone even evaluated the methodology. Real-world data (RWD) was widely used to satisfy safety monitoring and post-approval regulation requirements. But many clinicians saw RWD, and the real-world evidence (RWE) it generated, as unverified and unreliable data mining aimed at drug marketing goals.
By definition, RWD is unstructured compared to randomized controlled trials. Researchers can gather it through a variety of methods from multiple sources, which creates a credibility problem for this “real evidence.” Unfortunately, there are several examples of RWD that was exploited or misused, with methodologies tailored to produce data that filled messaging gaps. This has fostered mistrust and misunderstanding of RWD in the medical community.
Fortunately, there’s substantial evidence that RWD can produce high-quality data and essential observations. Still, a few bad examples created a credibility issue that’s hard to undo, and that creates a barrier to using RWD and RWE in clinical research.
Why Unstructured Data Has Real Value
The same unstructured, less precise nature of RWD that opens it up to misuse is exactly what makes the data and evidence valuable — it reflects the real world. Clinical trials, by design, run under controlled conditions that minimize factors that could influence the outcome. When researchers gather RWD with the right methodologies from credible sources, it complements clinical trial data well.
RWD and the RWE it generates can help assess safety and efficacy in patient populations not represented in clinical trials, determine whether a perceived drug risk is real, and identify gaps in research and areas of unmet need in standard of care. It’s also valuable for assessing how well clinical trial data generalizes, generating hypotheses for additional trials, and studying quality of life and changes in drug use patterns.
How the Cures Act Changed RWD’s Role
Over the past five years, RWE has become more valuable to the research community, thanks in part to the 21st Century Cures Act. The Cures Act created avenues to speed up drug development, so life-saving therapies can reach patients faster. One of these avenues lets RWD and RWE support drug approval applications — a role once reserved for post-approval and safety surveillance requirements.
This shift was driven largely by time-critical oncology research that couldn’t wait for survival endpoint data from clinical trials. Oncology Phase 1 and 2 trials have started integrating RWD extensions as part of the expedited approval process. In short, RWD and RWE now play a critical role in getting life-extending therapies to critically ill patients — helping put RWD’s credibility issues in the rearview.
FDA Guidance on Quality RWD
To support RWD and RWE’s place as credible research, the Food and Drug Administration (FDA) has issued guidance on proper RWD study design and data gathering from quality sources. This further solidifies RWD’s importance in the research paradigm. The guidance identifies three main challenges to quality RWD: the methodologies used, the reliability and relevance of the data, and the quality of the data sources.
Getting the Most From RWD/RWE
To navigate these challenges, researchers should consider partnering with research companies experienced in long-term, real-world, observational studies. These companies have the infrastructure to support a variety of study designs — large simple trials, hybrid or pragmatic designs, and long-term observational studies. A company that specializes in gathering and verifying data through in-home patient visits, medical health record reviews, and longitudinal biometrics analysis can help ensure quality RWE. Partnerships like these can raise both the quantity and quality of RWD, supporting traditional RCTs and drug approval pipelines.
REFERENCES
Corrigan-Curay J, Sacks L, and Woodcock J. Real-World Evidence and Real-World Data for Evaluating Drug Safety and Effectiveness. JAMA. 2018;320(9):867-868
Food and Drug Administration. Framework for FDA’s real-world evidence program. Available from: Framework for FDA’s real-world evidence program
Miseta E. Why all the talk about real-world evidence? Clinical Leader. July 2019. Available from: Why all the talk about real-world evidence?
Sherman RE, Anderson SA, Dal Pan GJ, et al. Real-World Evidence – What Is It and What Can It Tell Us? N Engl J Med. 2016;375(23):2293-2297
Shore C, Gee AW, Kahn B, et al., editors. Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series. Washington (DC): National Academies Press (US); 2019 Feb 6. Available from: https://www.ncbi.nlm.nih.gov/books/NBK540112/
