Innovative Clinical Trial Designs for Precision Medicine in Heart Failure with Preserved Ejection Fraction

Sanjiv J. Shah*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

35 Scopus citations


A major challenge in the care of patients with heart failure and preserved ejection fraction (HFpEF) is the lack of proven therapies due to disappointing results from randomized controlled trials (RCTs). The heterogeneity of the HFpEF syndrome and the use of conventional RCT designs are possible reasons underlying the failure of these trials. There are several factors—including the widespread adoption of electronic health records, decreasing costs of obtaining high-dimensional data, and the availability of a wide variety of potential therapeutics—that have evolved to enable more innovative clinical trial designs in HFpEF. Here, we review the current landscape of HFpEF RCTs and present several innovative RCT designs that could be implemented in HFpEF, including enrichment trials, adaptive trials, umbrella trials, basket trials, and machine learning-based trials (including examples for each). Our hope is that the description of the aforementioned innovative trial designs will stimulate new approaches to clinical trials in HFpEF.

Original languageEnglish (US)
Pages (from-to)322-336
Number of pages15
JournalJournal of Cardiovascular Translational Research
Issue number3
StatePublished - Jun 1 2017


  • Clinical trials
  • Heart failure with preserved ejection fraction
  • Personalized medicine
  • Precision medicine
  • Treatment

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine
  • Genetics(clinical)
  • Genetics
  • Molecular Medicine
  • Pharmaceutical Science


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