Site selection in global clinical trials in patients hospitalized for heart failure: perceived problems and potential solutions.

Published

Journal Article

There are over 1 million hospitalizations for heart failure (HF) annually in the United States alone, and a similar number has been reported in Europe. Recent clinical trials investigating novel therapies in patients with hospitalized HF (HHF) have been negative, and the post-discharge event rate remains unacceptably high. The lack of success with HHF trials stem from problems with understanding the study drug, matching the drug to the appropriate HF subgroup, and study execution. Related to the concept of study execution is the importance of including appropriate study sites in HHF trials. Often overlooked issues include consideration of the geographic region and the number of patients enrolled at each study center. Marked differences in baseline patient co-morbidities, serum biomarkers, treatment utilization and outcomes have been demonstrated across geographic regions. Furthermore, patients from sites with low recruitment may have worse outcomes compared to sites with higher enrollment patterns. Consequently, sites with poor trial enrollment may influence key patient end points and likely do not justify the costs of site training and maintenance. Accordingly, there is an unmet need to develop strategies to identify the right study sites that have acceptable patient quantity and quality. Potential approaches include, but are not limited to, establishing a pre-trial registry, developing site performance metrics, identifying a local regionally involved leader and bolstering recruitment incentives. This manuscript summarizes the roundtable discussion hosted by the Food and Drug Administration between members of academia, the National Institutes of Health, industry partners, contract research organizations and academic research organizations on the importance of selecting optimal sites for successful trials in HHF.

Full Text

Duke Authors

Cited Authors

  • Gheorghiade, M; Vaduganathan, M; Greene, SJ; Mentz, RJ; Adams, KF; Anker, SD; Arnold, M; Baschiera, F; Cleland, JGF; Cotter, G; Fonarow, GC; Giordano, C; Metra, M; Misselwitz, F; Mühlhofer, E; Nodari, S; Frank Peacock, W; Pieske, BM; Sabbah, HN; Sato, N; Shah, MR; Stockbridge, NL; Teerlink, JR; van Veldhuisen, DJ; Zalewski, A; Zannad, F; Butler, J

Published Date

  • March 2014

Published In

Volume / Issue

  • 19 / 2

Start / End Page

  • 135 - 152

PubMed ID

  • 23099992

Pubmed Central ID

  • 23099992

Electronic International Standard Serial Number (EISSN)

  • 1573-7322

Digital Object Identifier (DOI)

  • 10.1007/s10741-012-9361-8

Language

  • eng

Conference Location

  • United States