Real-world data (RWD) and real-world evidence (RWE) are valuable across the entire pharmaceutical product lifecycle, from examining patient outcomes to supporting disease education and awareness, measuring patient adherence, to monitoring safety and efficacy, and beyond.
In October 2021, the Food and Drug Administration (FDA) issued guidance on the expanded use of real-world data and real-world evidence, acknowledging their increasing role in health care decisions beyond post-approval safety and efficacy studies.
The FDA concedes that the health care community uses real-world data to support expanded coverage decisions and to develop guidelines and decision support tools for use in clinical practice. In particular, the increased use of technology (computers, mobile devices, wearables, and other biosensors) to gather and store huge amounts of health-related data has been rapidly accelerating. The FDA has thus refined its definitions for the terms "real-world data" and "real-world evidence."
According to the FDA, real-world data are the data relating to patient health status and/or the delivery of health care that are routinely collected from a broad variety of sources, including:
- Electronic health records (EHRs)
- Claims and billing activities
- Product and disease-specific registries
- Patient-generated data, including in home-use settings
- Data gathered from other sources that can inform on health status, such as mobile devices
Real-world evidence is clinical evidence regarding the usage and potential benefits or risks of a medical product that is derived from the analysis of real-world data. Real-world evidence can be generated by multiple study designs or analyses, including, but not limited to, randomized trials, large simple trials, pragmatic trials, and observational studies (prospective and/or retrospective).1
"These data hold [sic] potential to allow us to better design and conduct clinical trials and studies in the health care setting to answer questions previously thought infeasible. In addition, …with the development of sophisticated, new analytical capabilities, we are better able to analyze these data and apply the results of our analyses to medical product development and approval," the FDA said in a statement.
Real-world use of real-world data: The challenges
Despite the FDA's efforts to expand considerations and delineate data sources for their applications to clinical trial design, the drug development industry still experiences key roadblocks with real-world data. For instance, applying the data to the needed evidence strategy can be challenging. Another issue is integrating siloed data and deriving relevant, usable insights from them.
Other hurdles can include:
- Access to and use of correct data subsets
- Leveraging unstructured data
- Simplistic infrastructure limiting the power of Big Data
- Understanding and abiding by data privacy regulations
This is but a sampling of the potential obstacles to applying real-world data to solving specific therapeutic questions.2-4
The tailored, science-first approach
Leveraging a science-first approach — understanding the true gaps in information and data — is the ideal way for sponsors to address and overcome any given real-world evidence challenge.
In Part 2 of our series, we will detail traditional and innovative registry models as valuable sources for RWE.
1) Real-World Evidence. https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence. Accessed 1/22/22.
2) The State of Real-World Evidence in Biopharma.acornai, 2020. https://www.medidata.com/en/acornai/whitepaper/real-world-evidence-in-biopharma. Accessed 1/22/22.
3) "Evidence Through Advanced Analytics." Perkin Elmer, 2016, https://www.perkinelmer.com/lab-solutions/resources/docs/WTP_Real-WorldEvidence-Through-Advanced-Analytics-012927_01.pdf 4. Accessed 1/22/22.
4) "Key Takeaways from FDA's Framework for Real-World Evidence for Pharmaceuticals." Covington Digital Health, 20 Feb. 2019, https://www. covingtondigitalhealth.com/2018/12/key-takeaways-from-fdas-framework-for-real-world-evidence-for-pharmaceuticals