tives and regulatory requirements for orphan drugs and Humanitarian Use Devices are not likely to have much practical impact on the development or use of the product. In addition, if a device is one that qualifies for clearance through the 510(k) process, then the HDE process and conditions likely will not be considered.20

It is difficult to assess the extent to which the relatively low number of HDE approvals is influenced by the profit disincentive and the limit on yearly shipment or is, rather, a function of limited opportunities for devices—compared to drugs—to meet substantial unmet health needs for small populations. As noted at the beginning of this chapter, the emphasis in discussions of rare diseases is overwhelmingly on drugs.

For devices covered by an HDE, information on the number of device units shipped is not readily available nor are the estimates submitted by companies (in support of their HDE application) of the number of affected individuals. Although indication-specific information is not available, a recent press release by Medtronic recently reported that a cumulative 75,000 patients worldwide had been treated with its implanted deep-brain stimulation technology, including for the four indications described earlier (Medtronic, 2010).


Based on information presented to the committee by representatives of companies and FDA and provided by a review of past proposals for policy change, the committee concluded that the development of reasonable and effective incentives specific to device development for small populations has proven difficult. The incentives relevant for drug development, particularly the protections from market competition, are not well matched to the realities of device development. Although recent initiatives to promote the development of pediatric medical devices modify the HDE process by allowing profits, they do not move toward either the marketing protections or the stricter approval requirements applicable to orphan drugs.

In contrast to pediatric medical devices, relatively little attention has been directed to needs for medical devices for people with rare conditions. (Even the statement to the committee from the Advanced Medical Technology Association focused as much or more on pediatric devices as on devices


The committee found one rare condition for which FDA has approved an orphan drug and an HDE, although the approved indication for the device appears considerably more restrictive. In 2000, the agency approved botulinum toxin type A as an orphan drug to decrease the severity of abnormal head position and neck pain associated with cervical dystonia (BLA 103000/1004). In 2003, it approved an implanted deep-brain stimulation device under an HDE for the management of chronic, severe, drug-refractory dystonia, including cervical dystonia (H020007).

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