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  5. Premarket Submissions: Selecting and Preparing the Correct Submission
  6. Humanitarian Device Exemption
  1. Premarket Submissions: Selecting and Preparing the Correct Submission

Humanitarian Device Exemption

Send Medical Device eSTAR and eCopy Premarket Submissions Online

October 3, 2022 - The FDA is announcing that you may now send electronic copy (eCopy) or electronic Submission Template And Resource (eSTAR) premarket submissions online through the CDRH Customer Collaboration Portal ("CDRH Portal").

Building on the progress tracker for 510(k) submissions launched in 2021 and the trial process of electronic uploads launched in July 2022, the CDRH Portal now allows anyone to register for a CDRH Portal account to send CDRH eCopy or eSTAR premarket submissions online.

Starting October 1, 2023, all 510(k) submissions, unless exempted*, must be submitted as electronic submissions using eSTAR.

In accordance with the Orphan Drug Act (ODA) of 1984, a rare disease is defined as a disease or condition that affects fewer than 200,000 people in the United States. Currently, in the United States, only a portion of the 7,000 known rare diseases have approved treatments. By definition, rare diseases or conditions occur in a small number of patients. As a result, it has been difficult to gather enough clinical evidence to meet the FDA standard of reasonable assurance of safety and effectiveness.

In order to address this challenge, Congress included a provision in the Safe Medical Devices Act of 1990 to create a new regulatory pathway for products intended for diseases or conditions that affect small (rare) populations. This is the Humanitarian Device Exemption (HDE) Program.

Definitions

Humanitarian Use Device (HUD): 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 (Section 3052 of the 21st Century Cures Act (Pub. L. No. 114-255).

Humanitarian Device Exemption (HDE): a marketing application for an HUD (Section 520(m) of the Federal Food, Drug, and Cosmetic Act (FD&C Act)). An HDE is exempt from the effectiveness requirements of Sections 514 and 515 of the FD&C Act and is subject to certain profit and use restrictions.

Prohibition on Profit

The Food and Drug Administration Amendments Act of 2007 (FDAAA) contained incentives to facilitate development of medical devices for pediatric populations (defined as patients who are younger than 22 years of age).

Under section 520(m)(6)(A)(i) of the FD&C Act, an HUD is only eligible to be sold for profit after receiving an HDE approval if the device is intended for the treatment or diagnosis of a disease or condition that either:

  1. occurs in pediatric patients or in a pediatric subpopulation, and such device is labeled for use in pediatric patients or in a pediatric subpopulation in which the disease or condition occurs; OR
  2. occurs in adult patients and does not occur in pediatric patients or occurs in pediatric patients in such numbers that the development of the device for such patients is impossible, highly impracticable, or unsafe.

HDE applicants whose devices meet one of the eligibility criteria and wish to sell their HUD for profit should provide adequate supporting documentation to FDA in the original HDE application. HDE holders who wish to sell their devices for profit and who did not submit the request in the original HDE application may submit a supplement and provide adequate supporting documentation to demonstrate that the HUD meets the eligibility criteria.

Annual Distribution Number

The number of HDE devices that may be sold for profit is limited to a quantity known as the Annual Distribution Number (ADN). If the FDA determines that an HDE holder is eligible to sell the device for profit, FDA will determine the ADN and notify the HDE holder.

The ADN is calculated by taking the number of devices reasonably necessary to treat or diagnose an individual per year and multiplying it by 8000. For example, if the typical course of treatment using an HDE device, in accordance with its intended use, requires the use of two devices per patient per year, then the ADN for that HDE device would be 16,000 (i.e., 2 x 8000).

If the number of devices distributed in a year exceeds the ADN, the sponsor can continue to sell the device but cannot earn a profit for the remainder of the year.

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