Endpoints in Heart Failure Drug Development

Aliza Hussain, Arunima Misra, Biykem Bozkurt, Aliza Hussain, Arunima Misra, Biykem Bozkurt

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.

Keywords: Endpoints; clinical trials; heart failure.

Conflict of interest statement

Disclosure: BB has received consulting fees from Bristol Myers Squibb, scPharmaceuticals, Baxter Healthcare, Sanofi-Aventis and Relypsa, and serves on the Clinical Event Committee for the GUIDE HF trial sponsored by Abbott Vascular and the Data Safety Monitoring Committee of the ANTHEM trial sponsored by Liva Nova. All other authors have no conflicts of interest to disclose.

Copyright © 2022, Radcliffe Cardiology.

Figures

Figure 1:. Development and Implementation Steps to…
Figure 1:. Development and Implementation Steps to Bring New Developments to Patients

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