The role of early-phase trials and real-world evidence in drug development

Harriette G.C. Van Spall, Arnaud Bastien, Bernard Gersh, Barry Greenberg, Reza Mohebi, James Min, Karsten Strauss, Steffen Thirstrup, Faiez Zannad

Research output: Contribution to journalArticlepeer-review


Phase 3 randomized controlled trials (RCTs), while the gold standard for treatment efficacy and safety, are not always feasible, are expensive, can be prolonged and can be limited in generalizability. Other under-recognized sources of evidence can also help advance drug development. Basic science, proof-of-concept studies and early-phase RCTs can provide evidence regarding the potential for clinical benefit. Real-world evidence generated from registries or observational datasets can provide insights into the treatment of rare diseases that often pose a challenge for trial recruitment. Pragmatic trials embedded in healthcare systems can assess the treatment effects in clinical settings among patient populations sometimes excluded from trials. This Perspective discusses potential sources of evidence that may be used to complement explanatory phase 3 RCTs and to speed the development of new cardiovascular medications. Content is derived from the 19th Global Cardiovascular Clinical Trialists meeting (December 2022), involving clinical trialists, patients, clinicians, regulators, funders and industry representatives.

Original languageEnglish
Pages (from-to)110-117
Number of pages8
JournalNature Cardiovascular Research
Issue number2
StatePublished - Feb 2024
Externally publishedYes


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