Endpoint selection is a critically important step in clinical trial design. It poses major challenges for investigators, regulators, and study sponsors, and it also has important clinical and practical implications for physicians and patients. Clinical outcomes of interest in heart failure trials include all-cause mortality, cause-specific mortality, relevant non-fatal morbidity (e.g., all-cause and cause-specific hospitalization), composites capturing both morbidity and mortality, safety, symptoms, functional capacity, and patient-reported outcomes. Each of these endpoints has strengths and weaknesses that create controversies regarding which is most appropriate in terms of clinical importance, sensitivity, reliability, and consistency. Not surprisingly, a lack of consensus exists within the scientific community regarding the optimal endpoint(s) for both acute and chronic heart failure trials. In an effort to address these issues, the Heart Failure Association of the European Society of Cardiology (HFA-ESC) convened a group of expert heart failure clinical investigators, biostatisticians, regulators, and pharmaceutical industry scientists (Nice, France, 12-13 February 2012) to evaluate the challenges of defining heart failure endpoints in clinical trials and to develop a consensus framework. This report summarizes the group's recommendations for achieving common views on heart failure endpoints in clinical trials.
AimTo evaluate the challenges of defining heart failure endpoints in clinical trials and to develop a consensus framework.
ContributorsZannad, Faiez Garcia, Angeles Alonso Anker, Stefan D. Armstrong, Paul W. Calvo, Gonzalo Cleland, John G. F. Cohn, Jay N. Dickstein, Kenneth Domanski, Michael J. Ekman, Inger Filippatos, Gerasimos S. Gheorghiade, Mihai Hernandez, Adrian F. Jaarsma, Tiny Koglin, Joerg Konstam, Marvin Kupfer, Stuart Maggioni, Aldo P. Mebazaa, Alexandre Metra, Marco Nowack, Christina Pieske, Burkert Pina, Ileana L. Pocock, Stuart J. Ponikowski, Piotr Rosano, Giuseppe Ruilope, Luis M. Ruschitzka, Frank Severin, Thomas Solomon, Scott Stein, Kenneth Stockbridge, Norman L. Stough, Wendy Gattis Swedberg, Karl Tavazzi, Luigi Voors, Adriaan A. Wasserman, Scott M. Woehrle, Holger Zalewski, Andrew McMurray, John J. V.
Disease Category: Heart & circulation
Disease Name: Chronic heart failure
Age Range: 18 - 100
Sex: Either
Nature of Intervention: Any
- Members of a clinical trial network.
- Pharmaceutical industry representatives
- Regulatory agency representatives
- Statisticians
- COS for clinical trials or clinical research
- Semi structured discussion
The Heart Failure Association of the European Society of Cardiology (HFA-ESC) convened a group of expert heart failure clinical trialists, biostatisticians, regulators, and pharmaceutical industry scientists (Nice, France, 12–13 February 2012) to evaluate the challenges of defining heart failure endpoints in clinical trials and to develop a consensus framework.