Endpoints in Heart Failure Drug Development: History and Future

Area of Science:

• Cardiology
• Clinical Trials
• Patient-Reported Outcomes

Background:

• Heart failure (HF) significantly impacts patient quality of life and physical function.
• Current HF therapies primarily target reducing mortality and hospitalizations.
• Few HF drugs are approved for improving patient-reported outcomes like symptoms or function.

Observation:

• Historically, HF drug approvals in the US prioritized mortality and hospitalization reduction.
• Patient-reported outcomes (PROs) directly capture patient experiences with symptoms, function, and quality of life.
• PROs are increasingly recognized as valuable endpoints in clinical trials.

Findings:

• This paper reviews the evolution of endpoints in US HF drug approvals.
• It highlights the potential of PROs as approvable endpoints for HF therapies.
• Focus is placed on PROs measuring symptoms, physical function, and quality of life.

Implications:

• Incorporating PROs could lead to HF drug development that better addresses patient-centered goals.
• This shift may result in treatments that more effectively improve daily living for HF patients.
• Recognizing PROs expands the scope of successful drug development beyond survival metrics.