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  1. FDA In Brief

FDA In Brief: FDA provides transparency and clarity on the Humanitarian Device Exemption pathway, helping bring more life-saving medical devices to patients in need

September 5, 2019

Media Inquiries

  Alison Hunt

“Spurring innovation of medical products, so patients have access to treatments and diagnostics that improve or save lives, is a fundamental part of the FDA’s mission. Our Humanitarian Device Exemption, or HDE, Program is one such example. This unique pathway is designed to help bring to market medical devices intended to treat or diagnose patients with rare diseases or conditions affecting no more than 8,000 people per year in the United States,” said Jeff Shuren, M.D., director of FDA’s Center for Devices and Radiological Health. “To date, 76 medical devices have been approved through this regulatory pathway, providing more options to patients in need—from a device that allows patients with certain spinal cord injuries to breathe without the assistance of a mechanical ventilator to our most recently approved HDE in August 2019 for a device indicated to correct idiopathic scoliosis, a sideways curvature of the spine in children and adolescents for which the cause is unknown. Prompted by Congress in the 21st Century Cures Act, the final guidance issued today advances our goal to provide more transparency to patients, physicians and the medical device industry on how the FDA determines whether to approve an HDE application. It is part of our larger commitment to spur the development of new technologies to meet the needs of small patient populations.”

Today, the U.S. Food and Drug Administration issued a final guidance, Humanitarian Device Exemption (HDE) Program, to provide updated information about the HDE application process and other considerations specific to the HDE Program.

An HDE application is the premarket submission for a Humanitarian Use Device (or HUD), which is a medical device intended to benefit patients in the treatment or diagnosis of a disease or condition that affects, or is manifested in, not more than 8,000 individuals in the United States per year. A device approved under an HDE application is exempt from the requirement to demonstrate a reasonable assurance of effectiveness. To be eligible for an HDE, the FDA must determine, among other things, that the device would not be available to a person with the disease or condition in question without the HDE approval and that there is no comparable device, other than another device approved under an HDE or Investigational Device Exemption (IDE), available to treat or diagnose the disease or condition.

As required by the 21st Century Cures Act, the final guidance explains the principal criteria that the FDA considers when determining if probable benefit(s) to health have been demonstrated for a HUD that is being reviewed through the HDE Program. The final guidance also discusses FDA’s assessment of the probable benefits and risks for a device as part of its HDE application review. When performing this probable benefit-risk assessment, the FDA considers relevant factors in the context of the intended use of device, including the target patient population and the size of the population. The FDA’s probable benefit-risk assessment also considers currently available alternative treatments or diagnostics. The final guidance includes a filing checklist to clarify the information needed for the FDA to consider the application sufficiently complete to allow substantive review, as well as tools to assist application review staff in consistent decision-making.

The guidance issued today finalizes the draft guidance on the HDE Program that was issued on June 12, 2018. This final guidance supersedes Guidance for HDE Holders, Institutional Review Boards (IRBs), Clinical Investigators, and Food and Drug Administration Staff, Humanitarian Device Exemptions (HDE) Regulation: Questions and Answers, issued July 8, 2010.

Related Information

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.

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