#HEORInsights

The world today is observing an exponential growth in the volume and variety of the real-world data (RWD). Thanks to the technological advancements and the rise in the use of integrated electronic medical records (EMRs), RWD is ever more accessible and applicable in the regulatory domain as well as outcomes research. The evidence from randomized controlled trials (RCTs) is still undoubtedly the gold standard for assessing treatment efficacy; however, the interest and potential for adapting RWD into real-world evidence (RWE) is on the rise. This can prove extremely beneficial to make informed healthcare decisions. (1)

RWE has several advantages over RCT findings, particularly in research to aid decision making for healthcare delivery. These advantages include the availability of well-timed data at reasonable cost, large sample sizes enabling analyses of subgroups and less common effects, and the overall better representation of the real-world practices and behaviours. (1) Nonetheless, RWE has several concerns questioning its credibility, including data quality, biases – thanks to lack of randomization, and possibly false results owing to data mining. Some other major challenges, as highlighted by the USFDA, include inconsistency in sources and formats, different nature of source data captured by different regions, differences in terminology and exchange, different methods used to build datasets for aggregation, and differences in overall data quality. (2) These are the challenges that have haltered the progress of RWE in healthcare despite its significant data capabilities. (1) In the same context, USFDA has acknowledged the need for standardizing RWD for healthcare decision making. As a result, a draft guidance has been recently released for the industry, outlining USFDA’s requirements from the sponsors for submission of drug and biological product study data by RWD sources. (2)

Acknowledging similar concerns over RWD quality, the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), in collaboration with the International Society for Pharmacoepidemiology, the Duke-Margolis Center for Health Policy, and the National Pharmaceutical Council, has recently launched the Real-World Evidence Transparency Initiative with the purpose to promote health economics and outcomes research (HEOR) excellence to improve global healthcare decision making. Additionally, it will help instituting a culture of transparency for analysis and reporting of hypotheses to evaluate RWE studies on treatment effects. (3)

In order to further improve transparency and credibility, the Real-World Evidence Transparency Initiative, on October 26^th 2021, launched the Real-World Evidence Registry. (4, 5) The registry will offer a fit-for-purpose platform to the researchers to prospectively register their study designs before starting data collection. (4) The registry will implement open, centralized workflows that will enhance collaboration and facilitate the transparency needed to promote the trust in the study results. (4, 5)

The RWE registry is a streamlined registration website, especially for RWE studies conducting the hypothesis evaluation of treatment effects (HETE studies) using secondary data. This searchable platform will provide a place for pre-registration of studies that may not need registration for regulatory purposes, but benefit from the accuracy of transparent study methods and also provide a reference (such as a URL or doi) to share with the involved stakeholders, such as peer reviewers, assessors, or other decision makers. (4)

With the growing adaptation of RWE studies alongside RCTs, the launch of the registry could not have happened at a more opportune time. We hope that researchers optimize this resource and this move helps improving the transparency and credibility of RWD and thus, RWE studies. At Marksman Healthcare, we are well equipped to provide services in this domain, including RWE study protocol development, study/protocol registration in the RWE registry, RWD analysis, and RWE publication support, among others.

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References

1. Real World Evidence. ISPOR Strategic Initiatives. Available at: https://www.ispor.org/strategic-initiatives/real-world-evidence
2. FDA drafts data standards guidance for RWD. October 2021. Available at: https://www.raps.org/news-and-articles/news-articles/2021/10/fda-drafts-data-standards-guidance-for-rwd
3. Real-World Evidence Transparency Initiative. ISPOR Strategic Initiatives. Available at: https://www.ispor.org/strategic-initiatives/real-world-evidence/real-world-evidence-transparency-initiative?utm_medium=press_release&utm_source=public&utm_campaign=general_ispor&utm_content=press_release_oct26&utm_term=rwe_registry
4. Real-World Evidence Registry. Available at: https://www.ispor.org/strategic-initiatives/real-world-evidence/real-world-evidence-registry
5. New Real-World Evidence Registry Launches. October 26 2021. Available at: https://www.newswise.com/articles/new-real-world-evidence-registry-launches