In addition to financing product development, commercial firms are almost always responsible for meeting the regulatory requirements necessary to obtain FDA approval to market drugs, biologics, and certain complex medical devices. They likewise manufacture and distribute products consistent with regulatory standards. Chapters 5 and 7 discuss the role of the private sector in product development in more depth.

Following the early initiatives of the Cystic Fibrosis Foundation and the Committee to Combat Huntington’s Disease, an increasing number of patient advocacy groups have become active in research. They have helped create innovative models for funding and organizing research and product development. The emphases of advocacy groups vary, depending in part on the state of the science within different disease areas and in part on other factors that may include the number of affected individuals, the interests and skills of organizational founders and leaders, and the success of fundraising strategies. If researchers have not yet identified the genetic or other cause of a condition or delineated how the disease develops, a group may concentrate its grants and other activities on closing these gaps in knowledge.

Policies of Other Countries and International Initiatives

The policies of the United States on orphan drugs and pharmaceuticals do not exist in isolation. The United States was the earliest adopter of formal incentives for orphan drug development, but a number of other nations have followed with policies that are broadly similar, although differing in some details. The European Union has developed a common policy for its member states on some issues (e.g., length of market exclusivity period) but not all (e.g., the availability of grants). Table 1-3 summarizes selected policies.

FDA, regulatory agencies in other countries, industry, and advocacy groups have engaged in discussions to harmonize various aspects of orphan product policies (see, e.g., Wechsler, 2008). One result is that the European Medicines Agency (EMEA) and the FDA have adopted a joint application form for orphan product designation. Each agency still makes its own decisions, but the two regularly communicate about application reviews. Work to harmonize views on what constitutes acceptable clinical trial design and analytic strategies is particularly important when patient populations are small, multi-nation studies are essential, and confirmatory trials are difficult or impossible.

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