Impact and Significance
In recent years, there has been an emergence of real-world data (RWD) use to support drug development processes and overall healthcare decisions. This development was further accelerated after the passage of the 21st Century Cures Act in 2016 by the FDA, which endeavored to develop guidelines on leveraging real-world evidence to support regulatory decisions, post-marketing requirements and review of new indications for approved drugs.
As the adoption rate of using real-world evidence (RWE) continues to evolve, it has become evident that the data needs to be “fit for purpose” and fit for partners’ needs. In essence, the evidence should support answering research questions, even if it was never intended for the purpose of research. Furthermore, the use and applicability of RWD continues to expand beyond the pharmaceutical realm, with payors now leveraging RWE to support making reimbursement and coverage decisions.
Target RWE strives to remain at the forefront of this paradigm shift and aims to provide fit for purpose data-driven insight to partners across the healthcare spectrum to support their RWE initiatives.
Real-World Data vs. Real-World Evidence
In 2016, the Food and Drug Administration (FDA) passed the 21st Century Cures Act in effort to incorporate more patient perspectives and expand the approved types of data that can be used to support regulatory drug development decisions. The Cures Act is a step towards improving the FDA’s capability to streamline clinical trial designs and clinical outcome assessments, which includes the use of real-world data and evidence.
The FDA defines real-world data* as “the data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources.” This can include data from the following sources:
- Electronic health records
- Insurance billing and claims
- Product and disease registries
- Patient-generated data (e.g. patient-reported outcome measures or surveys)
- Biometric monitoring devices (e.g. mobile phones or watches)
The Cures Act additionally provides guidance on the use of real-world evidence*, which states it is “the clinical evidence regarding the usage and potential benefits/risks of a medical product derived from the analysis of RWD.” RWE is essentially the analytical data which details the use, potential benefits, or risks of a drug obtained from non-traditional clinical trials such as:
- Randomized controlled trials, or RCTs (including large simple trials and pragmatic trials)
- Observational studies (prospective and/or retrospective)
With the use of RWE, the FDA has better understandings on how to best design and operate clinical trials/studies in healthcare settings to resolve unanswered questions and improve treatments.
*Source: https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence