netic tests that are necessary for the diagnosis and treatment of certain rare conditions are, in certain cases, regulated as medical devices. In addition, people with rare conditions benefit from a large number of medical devices that are used generally in connection with complex surgery, anesthesia, respiratory support, nonsurgical cardiac procedures, administration of certain medications, diagnostic and therapeutic imaging of various kinds, laboratory testing, and other services.

Clinical studies of the titanium rib were supported under the orphan products grants program described in Chapter 3. Earlier, the National Organization for Rare Disorders provided a seed grant from its donated research funds. The two companies that were involved in manufacturing the device for research use participated out of interest in children’s health rather than expectations of profit (Campbell, 2007). After years of investigation and adaptation, the device was approved by the Food and Drug Administration (FDA) in 2004 through a Humanitarian Device Exemption (HDE). This process was established in the Safe Medical Devices Act of 1990 (P.L. 101-629) to provide incentives for the development of medical devices for small populations. Although medical devices for small populations are grouped under the label orphan products in the grants program created by the Orphan Drug Act and are within the charge of the Office of Orphan Product Development (OOPD), the term orphan medical device does not appear in legislative or regulatory language.

Regulatory requirements and product development pathways differ significantly for medical devices compared to drugs and biologics. Thus, this report devotes a separate chapter to medical device development, regulation, and reimbursement.

This chapter begins with a brief overview of important differences between devices and drugs. It then reviews device regulation and reimbursement with an emphasis on the HDE process and other policies or procedures that are potentially most relevant to complex, high-risk devices intended for small populations. This discussion is followed by an overview of the research and development process for complex devices and a discussion of barriers and opportunities for the development of devices for small populations. As this chapter highlights, the stringency of government regulation of devices is related to the risk presented by the device.


Compared to pharmaceuticals, medical devices are an extremely diverse group of products. Some are as simple as adhesive bandages, tongue depressors, and plastic tubing. Others are complex, for example, various implanted cardiac and neurological devices, stair-walking wheelchairs, robotic surgical systems, and magnetic resonance imaging devices. In contrast

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