The “Coming of Age” of Real‐World Evidence in Drug Development and Regulation

Abstract

Real world evidence (RWE) has the potential to inform drug discovery, development, and regulatory decision making. The emergence and availability of large population‐based datasets around the globe have stoked enthusiasm in the research community. However, much work remains to refine research methodologies, evolve the science, and define the place of RWE as a complementary source of evidence to randomized controlled trials.

Real-world study of overall survival with palbociclib plus aromatase inhibitor in HR+/HER2− metastatic breast cancer

“…A key strength of this analysis is the inclusion of key variables that can affect treatment selection and survival, including the number of metastatic sites, ECOG performance status, visceral involvement, and the interval from initial breast cancer diagnosis to MBC diagnosis, improving the ability to balance patient cohorts and reduce the risk of confounding 34 , 35 . The opportunity for real-world evidence to be a component in regulatory decision making continues to evolve, and as standards in real-world study design and transparency in analysis and reporting are adhered to there remains an important opportunity to leverage this data for that purpose 36 . Finally, real-world data may also contain helpful information for international health technology assessment practices that play a role in patient access to innovative treatments 37 .…”

Trends in Clinical Pharmacology and Therapeutics

“…A search of PubMed for “real‐world data” also reflects a sharp growth (1,826 PubMed entries in 2015 vs. 8,336 entries in 2021). In May 2021 and January 2022, CPT devoted two special issues to this ever‐emerging area with overwhelming response 2,3 . The notation “real‐world data” sharply increased (2015: 0%, 2021: 7%).…”

“…In May 2021 and January 2022, CPT devoted two special issues to this ever-emerging area with overwhelming response. 2,3 The notation "real-world data" sharply increased (2015: 0%, 2021: 7%). In 2021, 43% of publications on real-world-like data used the terms "real-world data" or "realworld evidence."…”

External Control Arms: We're Not on Cruise Control Yet

“…Although the concept of historical controls as an external control was described in literature as early as 1976, 1 external controls have recently been receiving renewed attention in the drug development community. In addition to historical controls, the use of real‐world data (RWD) and real‐world evidence (RWE) are increasing in prominence 2 . In some ways, the coronavirus disease 2019 (COVID‐19) pandemic helped to accelerate the consideration and use of external controls 3 …”

“…In addition to historical controls, the use of real-world data (RWD) and real-world evidence (RWE) are increasing in prominence. 2 In some ways, the coronavirus disease 2019 (COVID-19) pandemic helped to accelerate the consideration and use of external controls. 3 The idea of external control arms can be appealing-they can decrease the time, effort, and cost for drug developers, and thus potentially accelerate access to new treatments for patients.…”