The Present and Future
JACC State-of-the-Art Review
Optimal Background Pharmacological Therapy for Heart Failure Patients in Clinical Trials: JACC Review Topic of the Week

https://doi.org/10.1016/j.jacc.2021.11.033Get rights and content
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Highlights

  • Guideline-directed pharmacological therapy can improve functional status and reduce morbidity and mortality in patients with HFrEF, but in clinical practice are often suboptimally employed.

  • This creates a conundrum when new therapies for patients with HFrEF are evaluated in clinical trials, because uniform use of optimal background therapies may limit generalizability to patients managed in general practice.

  • Potential approaches include specific drug class recommendations, scoring systems, and other strategies that meet the needs of trialists, sponsors, regulators, payers, patients, and prescribers.

Abstract

With the current landscape of approved therapies for heart failure (HF), there is a need to determine the role of a standard background therapy against which novel therapies are studied. The Heart Failure Collaboratory convened a multistakeholder group of clinical investigators, clinicians, patients, government representatives including U.S. Food and Drug Administration and National Institutes of Health participants, payers, and industry in March 2021 to discuss whether standardization of background drug therapy is necessary in clinical trials in patients with HF. The current paper summarizes the discussion and provides potential conceptual approaches, with a focus on therapies indicated for HF with reduced ejection fraction.

Key Words

clinical trials
device therapy
drug therapy
FDA
guideline directed medical therapy
heart failure
HFrEF
medical therapy
medication

Abbreviations and Acronyms

FDA
U.S. Food and Drug Administration
EF
ejection fraction
GDMT
guideline-directed medical therapy
HF
heart failure
HFpEF
heart failure with preserved ejection fraction
HFrEF
heart failure with reduced ejection fraction

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Listen to this manuscript's audio summary by Editor-in-Chief Dr. Valentin Fuster on JACC.org.

Christie M. Ballantyne, MD, served as Guest Editor-in-Chief for this paper.

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