Roughly one out of every 10 Americans lives with at least one of more than 7,000 rare diseases. As explained at rarediseaseday.org, 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:
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.
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.
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The draft guidance document titled “Humanitarian Device Exemption (HDE) Program” was released by FDA this week in order to answer questions about the HDE program.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.