(a) This subpart H implements sections 515A and 520(m) of the act.

(b) The purpose of section 520(m) is, to the extent consistent with the protection of the public health and safety and with ethical standards, to encourage the discovery and use of devices intended to benefit patients in the treatment or diagnosis of diseases or conditions that affect or are manifested in not more than 8,000 individuals in the United States per year. This subpart provides procedures for obtaining:

(1) HUD designation of a medical device; and

(2) Marketing approval for the HUD notwithstanding the absence of reasonable assurance of effectiveness that would otherwise be required under sections 514 and 515 of the act.

(c) Section 515A of the act is intended to ensure the submission of readily available information concerning:

(1) Any pediatric subpopulations (neonates, infants, children, adolescents) that suffer from the disease or condition that the device is intended to treat, diagnose, or cure; and

(2) The number of affected pediatric patients.

(d) Although a HUD may also have uses that differ from the humanitarian use, applicants seeking approval of any non-HUD use shall submit a PMA as required under § 814.20, or a premarket notification as required under part 807 of this chapter.

(e) Obtaining marketing approval for a HUD involves two steps:

(1) Obtaining designation of the device as a HUD from FDA's Office of Orphan Products Development, and

(2) Submitting an HDE to the Office of Product Evaluation and Quality (OPEQ), Center for Devices and Radiological Health (CDRH), the Center for Biologics Evaluation and Research (CBER), or the Center for Drug Evaluation and Research (CDER), as applicable.

(f) A person granted an exemption under section 520(m) of the act shall submit periodic reports as described in § 814.126(b).

(g) FDA may suspend or withdraw approval of an HDE after providing notice and an opportunity for an informal hearing.