Developing therapies for heart failure with preserved ejection fraction: current state and future directions

Area of Science:

• Cardiology
• Translational Medicine
• Regulatory Science

Background:

• Heart failure with preserved ejection fraction (HFpEF) presents a significant and growing health burden.
• Current therapeutic options for HFpEF are limited, with no approved treatments to reduce mortality or hospitalizations.
• The complex pathophysiology of HFpEF involves cardiac, vascular, and end-organ abnormalities, alongside comorbidities, with substantial knowledge gaps.

Purpose of the Study:

• To summarize the proceedings of a Food and Drug Administration (FDA)-facilitated meeting.
• To foster a collaborative discussion among academia, industry, and regulatory agencies on advancing HFpEF therapy development.
• To identify key challenges and propose strategies for future research and drug development in HFpEF.

Main Methods:

• A facilitated meeting involving diverse stakeholders including academic researchers, pharmaceutical industry representatives, and regulatory agency officials.
• Discussion and synthesis of current understanding of HFpEF pathophysiology.
• Identification of critical unmet needs and potential pathways for therapeutic intervention.

Main Results:

• The meeting highlighted the complexity of HFpEF pathophysiology and the urgent need for effective treatments.
• Key areas for future research were identified, focusing on a deeper understanding of underlying mechanisms.
• A consensus was reached on the importance of collaborative efforts to accelerate the development of novel HFpEF therapies.

Conclusions:

• Addressing the significant burden of HFpEF requires a concerted effort to unravel its complex pathophysiology.
• Future therapeutic development necessitates a multi-disciplinary approach and strategic alignment between research, industry, and regulatory bodies.
• This meeting serves as a foundation for advancing the treatment landscape for patients with HFpEF.