Endpoints in Heart Failure Drug Development

Authors

Aliza Hussain
Arunima Misra
Biykem Bozkurt

Publication Date

1-1-2022

Journal

Cardiac Failure Review

DOI

10.15420/cfr.2021.13

PMID

35111335

PMCID

PMC8790723

PubMedCentral® Posted Date

1-18-2022

PubMedCentral® Full Text Version

Post-print

Published Open-Access

yes

Keywords

Endpoints, heart failure, clinical trials

Abstract

Heart failure (HF) is a major health problem worldwide. The development of effective drug and/or device therapy is crucial to mitigate the significant morbidity, mortality and healthcare costs associated with HF. The choice of endpoint in clinical trials has important practical and clinical implications. Outcomes of interest including mortality and HF hospitalisations provide robust evidence for regulatory approval granted there is sufficiency of safety data. At the same time, it is important to recognise that HF patients experience significant impairments in functional capacity and quality of life, underscoring the need to incorporate parameters of symptoms and patient-reported outcomes in clinical trials. In this review, the authors summarise the evolution and definition of cardiovascular endpoints used in clinical trials, discuss approaches to study design to allow the incorporation of mortality, morbidity and functional endpoints and, finally, examine the current challenges and suggest steps for the development of cardiovascular endpoints that are effective, meaningful and meet the needs of all relevant stakeholders, including patients, physicians regulators and sponsors.