June 27, 2019

Media Inquiries

  Sandy Walsh
  301-796-4669

“One important aspect of the FDA’s efforts to modernize the agency’s new drug program is the continued focus on providing disease-specific drug development guidances to industry. An area of current focus is drugs to treat heart failure. Despite the availability of drugs and devices to treat heart failure, it remains a cause of significant disability, has major effects on physical function and quality of life, and is a leading cause of death. The FDA is hoping to stimulate drug development in this area as new drugs are needed for the treatment of this serious condition,” said Ellis Unger, M.D., director of the Office of Drug Evaluation I in the FDA’s Center for Drug Evaluation and Research. “Today, we are issuing, for public comment, draft guidance that provides recommendations and important clarifications for drug developers on the type of evidence of effectiveness needed to support the approval of drugs to treat heart failure. The draft guidance notes that an effect on symptoms or physical function, without favorable effects on survival or hospitalization, can be a basis for approval. The draft guidance also addresses the need to assess mortality in clinical trials of drugs under development to treat heart failure. Along with the draft guidance, today we’ve announced an upcoming public workshop that will bring together stakeholders, patients, drug developers and the FDA to address key questions around the development of drugs to treat heart failure.”

The draft guidance, Treatment for Heart Failure: Endpoints for Drug Development, when finalized, will describe the agency’s current thinking on efficacy endpoints related to how patients feel and function, noting that evidence of effectivess of a heart failure drug could be based on improvments in symptoms, such as fatigue, and/or function, such as walking. Additionally, the draft guidance describes how to assess hospitalization as a clinical outcome and discusses possible biomarkers and surrogate endpoints. It also highlights several specific areas that are in need of further discussion, for which the FDA is seeking public input.

The public workshop, Endpoints for Drug Development in Heart Failure, will be held on Friday, July 26, 2019 from 9 a.m. to 4 p.m. at the FDA’s White Oak Campus, 10903 New Hampshire Avenue, Bldg. 31, Room 1503 (the Great Room), Silver Spring, Maryland, 20993. The purpose of the workshop is to bring the stakeholder community together to discuss clinical endpoints for trials in heart failure that could be used to support FDA approval of drugs. The workshop will focus on endpoints related to symptoms and physical function. In addition, there will be discussion of the need to assess mortality effects of drugs under development for heart failure.

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The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.