As any regulatory professional will know for the marketing department “It’s always the submission we don’t have” that will really get products selling. One potentially appealing strategy, particularly for people that have a product that is staring at a long PMA process, is the possibility of getting the product to market quicker via an Humanitarian Device Exemption (HDE) process. What folks need to know is that FDA is particularly concerned about industry “slicing and dicing the law”.

Basically to obtain an approved HDE there is a two-step process:

• HUD designation request submitted to OOPD (Orphan Office of Product Development). Upon receipt of the application, an answer from FDA is issued in 45 days. Submit two copies of your application request to the Office of Orphan Products Development (OOPD)
• After HUD designation, an HDE application may be submitted to CDRH Office of Device Evaluation. FDA will grant or deny HDE application within 75 days from receipt. This includes a 30-day filing period for substantial review determination (similar to an IDE process, etc.)

The Orphan Drug Office handles the Humanitarian Use Device (HUD) application and then designation (the first step towards HDE approval). This office wants to ensure that the intended population or subset of a larger population is really being intended for the HUD designation which is defined as “…medical device intended to benefit patients in diagnosis and/or treatment of disease/ condition affecting/ manifested in fewer than 4,000 patients/ year in the United States”. What precludes the device from being used in the larger population? This is a question that most certainly the Orphan Drug Office will ask. In other words why is your device better suited for this subset population rather than the larger population (that you may eventually trying to go after?} The dilemma of course is often the manufacturer is purposely, iteratively pursuing other, possibly more lucrative indications for their particular device(s) and likely do not want FDA from narrowing the company’s true long-term desired indication. This is the crux of what needs to be considered is “letting the cat out of the bag” so to speak. The company basically has to present why their technology is better suited for a smaller, subset population (>4000 patients/ year) but would not be necessarily useful for a larger population because of the company’s design as an example. This is exactly why if the company is ultimately pursuing a larger, more profitable population to market its device, pursuing the HDE may not (long term) be in its best interest.

FDA will consider HDE application only when no comparable, approved device is available to treat. What is a comparable device? One must consider intended use and technological characteristics, patient’s population to be treated and whether the device meets the needs of the identified patient population.

In FDA’s Premarket Notification Database Search for HDE current approvals there are approximately 40 approved HDEs presently (see http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfHDE/HDEInformation.cfm).

If you are interested in pursuing an HDE, your first task should be to look at a rather good guidance document (http://www.fda.gov/cdrh/ode/guidance/1381.pdf ) from FDA’s website. The other immediate task is to obtain the “HDE checklist for Filing Decision” of which the guidance document helps direct you to.

The guidance document noted above also discusses several other questions relating to Informed Consent, Investigational Review Board approval, annual report filing (similar to PMA annual report), and other important regulatory considerations.