Humanitarian Device

FDA Friday: New Guidance for Humanitarian Use Devices



Roughly one out of every 10 Americans lives with at least one of more than 7,000 rare diseases. As explained at, a disease or disorder is defined as rare in the USA when it affects fewer than 200,000 Americans at any given time. These conditions often have genetic origins whilst others are the result of infections (bacterial or viral), allergies and environmental causes, or are degenerative and proliferative. Tragically, half of those affected by rare diseases are children.

FDA came up with a program after recognizing the need for a more streamlined process of approval for different types of medical devices that could potentially serve as a cure or relief to a rare disease. This program was formed in 1990, and is called the Humanitarian Device Exemption (HDE) Program, which creates new regulatory pathways for devices and products that are intended for rare diseases. Due to the fact that these rare diseases are so infrequent or uncommon, developers are unable to gather enough clinical evidence to support and meet the normal FDA standard of assurance and safety, which is why the HDE Program was created.


The devices which are submitted to the HDE Program for approval are called Humanitarian Use Devices (HUDs), and are defined as “medical devices that are 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” (Source). Each of the HUDs are submitted with an HDE, the marketing application that is exempt from the effectiveness requirements that pertain to normal applications. FDA may grant an HDE if the device meets all of the requirements, which are as follows:


  1. The device will not expose any patient to a high level of danger and/or illness, and the benefit from use of the device outweighs any risk that may come from using the device.

  2. The device is available only through the HDE, and there are no other similar devices that have already achieved approval that could potentially treat or diagnose such disease or condition.

  3. The device is designed to diagnose or treat a rare disease or condition, where “rare” means that not more than 8,000 individuals in the U.S. are affected each year.


The draft guidance document titled “Humanitarian Device Exemption (HDE) Program” was released by FDA this week in order to answer questions about the HDE program. FDA has provided this information in the hopes that sharing the information will help bring awareness to more manufacturers and companies, and therefore expand the number of medical devices that are geared towards helping rare diseases or conditions.  

Additional Reading:

Med Device Monday: Flourish Esophageal Atresia and Humanitarian Devices

1 in 2,500 babies in the United States are born with esophageal atresia, a congenital defect in which the upper and lower parts of the esophagus don't properly connect in utero. Typically, affected infants have problems with eating and require a feeding tube until they're large and stable enough to have surgery performed to connect the esophagus. 

A device approved in May of 2017, Flourish Pediatric Esophageal Atresia Device, offers a different, less invasive, and innovative new solution. Flourish uses two magnets to slowly connect the two parts of the esophagus. As they explain, "Over the course of several days, the magnets gradually stretch both ends of the esophagus, after which the tissue connects to form an intact esophagus. To date, 16 patients have been successfully treated with this device."

The magnets are inserted through catheters in the mouth and via the abdominal wall, as shown:

Photo from 

Photo from 

It's important to note that the device cannot be used in patients with tracheoesophageal fistula, a condition commonly found in those with esophageal atresia. However, if a fistula has been repaired, Flourish can be used. Additionally, of the 16 treated patients, 13 required a follow-up procedure to fix narrowing of the newly formed esophagus. Notably, this is a complication that is common with the traditional surgical repair as well. 

What makes this innovative device even more special is that was granted approval from FDA via the Humanitarian Use Device (HUD) pathway. A HUD is defined as 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 (thanks to the 21st Century Cures Act, the population estimate was changed from "fewer than 4,000" to "not more than 8,000" this past December). In order to pursue approval via this pathway, an application is filled out and submitted to FDA. It's somewhat similar to a Premarket Approval Application (PMA), but does not have the effectiveness requirement of a PMA. However, it "must contain sufficient information for FDA to determine that the device does not pose an unreasonable or significant risk of illness or injury, and that the probable benefit to health outweighs the risk of injury or illness from its use, taking into account the probable risks and benefits of currently available devices or alternative forms of treatment. Additionally, the applicant must demonstrate that no comparable devices are available to treat or diagnose the disease or condition, and that they could not otherwise bring the device to market."

In 2007, title VIII of the Food and Drug Administration Amendments Act (FDAAA) included a provision that all applications via the Humanitarian pathway are required to include certification that all applicable clinical trial information has been submitted to the data bank. 

You can check out the list of FDA's approved Humanitarian Devices on their website.



Further reading:

RAPS article about Flourish

FDA press release about Flourish