for rare diseases [AdvaMed, 2009].) CDRH recently held a meeting to discuss unmet devices needs, but it did not specifically address the needs of people with rare conditions.

Without the time for a very focused examination, the committee found it difficult to assess the possible extent of unmet device needs for adults with rare conditions and the extent to which changes in FDA policies (e.g., an increase in the criterion of 4,000 patients per year for HDE approval) might promote innovation to meet these needs). A first step in understanding the potential areas for device innovation is a needs assessment for adults with rare conditions. Such an assessment, which should involve patient groups, clinicians, biomedical engineers, and device developers, can also illuminate impediments to innovations to meet those needs.

RECOMMENDATION 7-1: FDA and NIH should collaborate on an assessment of unmet device needs and priorities relevant to rare diseases. That assessment should focus on the most plausible areas of unmet need, identify impediments to meeting these needs, and examine options for overcoming impediments and stimulating high priority innovations.

The identification of needs, priorities, and impediments should help inform the consideration by government, private foundations, and others of additional incentives and supports for medical device development for small populations. Beyond simplifying some aspects of the HDE process as suggested in Recommendation 7-4, options to encourage device innovation for rare diseases include

  • the provision of additional orphan products grants and NIH awards for the development of devices to meet priority needs;

  • the authorization of tax credits for certain research and development costs, similar to those available for companies developing orphan drugs; and

  • the creation of inducement prizes for the design and initial testing of novel devices in areas of unmet need.

In addition, the changes in the HDE incentives for pediatric devices, including the relaxation of the restriction on profits, provide an opportunity to examine the case for similar changes to encourage innovative devices for adults with rare conditions. Also, as experience with the revised incentives for pediatric devices is gained, including through the GAO evaluation due in 2010, that knowledge should be applied to the encouragement of devices for adults with rare conditions. One alternative to eliminating the profit restriction altogether would be the development of a cost-plus option that

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