Rare Daily Staff

The U.S. Food and Drug Administration issued a final guidance document to provide clarity on its Humanitarian Device Exemption (HDE) program to conform with a requirement of  the 21st Century Cures Act.

The final guidance explains the principal criteria that the FDA considers when determining if probable benefits to health have been demonstrated for a humanitarian use device 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.

The HDE 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. To date, 76 medical devices have been approved through the HDE pathway.

Most recently, the FDA granted an HDE approval 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.

When performing this probable benefit-risk assessment, the FDA said it 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.

“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,” said Jeff Shuren, director of FDA’s Center for Devices and Radiological Health. It is part of our larger commitment to spur the development of new technologies to meet the needs of small patient populations.”

Photo: Jeff Shuren, director of FDA’s Center for Devices and Radiological Health

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