FDA plans on issuing updated guidance on humanitarian device exemptions "soon" to reflect provisions in the FDA Amendments Act of 2007, says Debra Lewis, associate director of the agency's Office of Orphan Products Development.

The new law includes an incentive for companies to validate and label their products for pediatric use. Specifically, it allows manufacturers to record a profit for devices granted humanitarian device exemptions by FDA specifically for use in patients 21 years and younger.

HDEs can be awarded to devices that are intended to treat or diagnose a condition that affects fewer than 4,000 patients in the United States per year based on a lower threshold of evidence than PMAs. Typically, companies can't charge more for an HDE device than the total cost of research and development, fabrication and distribution for each device.

The Amendments Act offers an exemption to the profit restriction for devices labeled for a qualifying pediatric indication, but there is a catch. Instead of a 4,000-per- year patient limit, for pediatric HDEs, FDA will need to specify an annual number of devices that can be distributed per year on a device-by-device basis. The number cannot exceed 4,000, but it can be a lot lower.

The number "will be based upon the number of individuals with that condition and how likely they are to use the product and how many devices they will need," Lewis said on a Jan. 16 conference call sponsored by the Regulatory Affairs Professionals Society. "So, a number will be assigned during the HDE review not to exceed 4,000 and if the sponsor goes over that number it must report that."

The agency is in the final stages of updating a July 2006 HDE "questions and answers" guidance document to include instruction on how the annual distribution number will be set and what will happen if it is exceeded, Lewis said.

If a company goes over the limit, "it is not a requirement that FDA work to withdraw [the product], but we certainly would need to consider it and look at the public health ramifications and what the situation is," she explained.

The guidance will also explain the procedure for petitioning the agency to increase the distribution number for a particular device, Lewis said.

The profit provision is explicitly not retroactive to already approved HDEs. But, Lewis says, the guidance writers and the FDA general counsel's office are still hashing out whether a manufacturer of an existing HDE product can add on a pediatric indication that would tip the total annual distribution number above 4,000 and still be allowed to profit from the pediatric use.

"It's possible that there could be a situation where you're going to amend an HDE that previously did not have pediatrics in the label's indication and now is ... meeting the pediatric need. Perhaps, that might qualify," she said.

"So that will be something that needs to be spelled out in that guidance."

Institutional Review Board Guidelines Expected

The updated guidance will also include additional information for institutional review boards.

The Amendments Act (signed into law Sept. 27) calls for FDA to issue guidance within 180 days for IRBs "on how to evaluate requests for the approval of medical devices for which a humanitarian device exemption ... has been granted."

The July 2006 HDE guidance explains the role of IRBs, noting that approval by the hospital-based groups is required for use of a humanitarian-use device at a facility, but that the IRB has discretion on whether to issue patient-by-patient approval or issue a blanket decision for the institution.

The updated guidance, Lewis suggests, will offer more instructions to IRBs on how they can use HDE information in evaluating the products.

- Ingrid Mezo (i.mezo@elsevier.com)

Updated Guidance To Address New Humanitarian Device Exemption Provisions

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