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

Cowie, MR; Filippatos, GS; Alonso Garcia, MDLA; Anker, SD; Baczynska, A; Bloomfield, DM; Borentain, M; Bruins Slot, K; Cronin, M; Doevendans, PA; et al. Cowie, MR; Filippatos, GS; Alonso Garcia, MDLA; Anker, SD; Baczynska, A; Bloomfield, DM; Borentain, M; Bruins Slot, K; Cronin, M; Doevendans, PA; El-Gazayerly, A; Gimpelewicz, C; Honarpour, N; Janmohamed, S; Janssen, H; Kim, AM; Lautsch, D; Laws, I; Lefkowitz, M; Lopez-Sendon, J; Lyon, AR; Malik, FI; McMurray, JJV; Metra, M; Figueroa Perez, S; Pfeffer, MA; Pocock, SJ; Ponikowski, P; Prasad, K; Richard-Lordereau, I; Roessig, L; Rosano, GMC; Sherman, W; Stough, WG; Swedberg, K; Tyl, B; Zannad, F; Boulton, C; De Graeff, P (2017) New medicinal products for chronic heart failure: advances in clinical trial design and efficacy assessment. Eur J Heart Fail, 19 (6). pp. 718-727. ISSN 1879-0844 https://doi.org/10.1002/ejhf.809
SGUL Authors: Rosano, Giuseppe Massimo Claudio

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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.

Item Type: Article
Additional Information: This is the peer reviewed version of the following article: Cowie, M. R., Filippatos, G. S., Alonso Garcia, M. d., 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. , Lopez‐Sendon, J. , Lyon, A. R., Malik, F. I., McMurray, J. J., Metra, M. , Figueroa Perez, S. , Pfeffer, M. A., Pocock, S. J., Ponikowski, P. , Prasad, K. , Richard‐Lordereau, I. , Roessig, L. , Rosano, G. M., Sherman, W. , Stough, W. G., Swedberg, K. , Tyl, B. , Zannad, F. , Boulton, C. and De Graeff, P. (2017), New medicinal products for chronic heart failure: advances in clinical trial design and efficacy assessment. Eur J Heart Fail, 19: 718-727, which has been published in final form at https://doi.org/10.1002/ejhf.809. This article may be used for non-commercial purposes in accordance with Wiley Terms and Conditions for Use of Self-Archived Versions.
Keywords: Clinical trial, Drug approval, Heart failure, Cardiovascular Agents, Clinical Trials as Topic, Consensus, Drug Approval, Heart Failure, Humans, Outcome Assessment (Health Care), Humans, Cardiovascular Agents, Drug Approval, Consensus, Outcome Assessment (Health Care), Clinical Trials as Topic, Heart Failure, 1102 Cardiovascular Medicine And Haematology, Cardiovascular System & Hematology
SGUL Research Institute / Research Centre: Academic Structure > Molecular and Clinical Sciences Research Institute (MCS)
Journal or Publication Title: Eur J Heart Fail
ISSN: 1879-0844
Language: eng
Dates:
DateEvent
6 June 2017Published
27 March 2017Published Online
7 February 2017Accepted
Publisher License: Publisher's own licence
PubMed ID: 28345190
Go to PubMed abstract
URI: https://openaccess.sgul.ac.uk/id/eprint/111206
Publisher's version: https://doi.org/10.1002/ejhf.809

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