New medicinal products for chronic heart failure: advances in clinical trial design and efficacy assessment

Martin R. Cowie, Gerasimos S. Filippatos, Maria de los Angeles Alonso Garcia, Stefan D. Anker, Anna Baczynska, Daniel M. Bloomfield, Maria Borentain, Karsten Bruins Slot, Maureen Cronin, Pieter A. Doevendans, Amany El-Gazayerly, Claudio Gimpelewicz, Narimon Honarpour, Salim Janmohamed, Heidi Janssen, Albert M. Kim, Dominik Lautsch, Ian Laws, Martin Lefkowitz, Jose Lopez-SendonAlexander R. Lyon, Fady I. Malik, John J.V. McMurray, Marco Metra, Santiago Figueroa Perez, Marc A. Pfeffer, Stuart J. Pocock, Piotr Ponikowski, Krishna Prasad, Isabelle Richard-Lordereau, Lothar Roessig, Giuseppe M.C. Rosano, Warren Sherman, Wendy Gattis Stough, Karl Swedberg, Benoit Tyl, Faiez Zannad, Caroline Boulton, Pieter De Graeff

Research output: Contribution to journalReview article

Abstract

Despite the availability of a number of different classes of therapeutic agents with proven efficacy in heart failure, the clinical course of heart failure patients is characterized by a reduction in life expectancy, a progressive decline in health-related quality of life and functional status, as well as a high risk of hospitalization. New approaches are needed to address the unmet medical needs of this patient population. The European Medicines Agency (EMA) is undertaking a revision of its Guideline on Clinical Investigation of Medicinal Products for the Treatment of Chronic Heart Failure. The draft version of the Guideline was released for public consultation in January 2016. The Cardiovascular Round Table of the European Society of Cardiology (ESC), in partnership with the Heart Failure Association of the ESC, convened a dedicated two-day workshop to discuss three main topic areas of major interest in the field and addressed in this draft EMA guideline: (i) assessment of efficacy (i.e. endpoint selection and statistical analysis); (ii) clinical trial design (i.e. issues pertaining to patient population, optimal medical therapy, run-in period); and (iii) research approaches for testing novel therapeutic principles (i.e. cell therapy). This paper summarizes the key outputs from the workshop, reviews areas of expert consensus, and identifies gaps that require further research or discussion. Collaboration between regulators, industry, clinical trialists, cardiologists, health technology assessment bodies, payers, and patient organizations is critical to address the ongoing challenge of heart failure and to ensure the development and market access of new therapeutics in a scientifically robust, practical and safe way.

Original languageEnglish
Pages (from-to)718-727
Number of pages10
JournalEuropean Journal of Heart Failure
Volume19
Issue number6
DOIs
Publication statusPublished - Jun 1 2017

Keywords

  • Clinical trial
  • Drug approval
  • Heart failure

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine

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    Cowie, M. R., Filippatos, G. S., Alonso Garcia, M. D. L. A., Anker, S. D., Baczynska, A., Bloomfield, D. M., Borentain, M., Bruins Slot, K., Cronin, M., Doevendans, P. A., El-Gazayerly, A., Gimpelewicz, C., Honarpour, N., Janmohamed, S., Janssen, H., Kim, A. M., Lautsch, D., Laws, I., Lefkowitz, M., ... De Graeff, P. (2017). New medicinal products for chronic heart failure: advances in clinical trial design and efficacy assessment. European Journal of Heart Failure, 19(6), 718-727. https://doi.org/10.1002/ejhf.809