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Rare Diseases and Orphan Products: Accelerating Research and Development
primary human epithelial cells harvested from lung transplants, a purified CFTR (cystic fibrosis transmembrane conductance regulator) protein supply, and validated assay services. These efforts of CFF and others are summarized in Chapter 5. NIH has also created new internal capacities and new partnership mechanisms for facilitating drug discovery, which are described in this section.
Public-Private Partnerships and Other Coordinating Strategies
Public-private partnerships have been a standard approach when the needs of the public sector converge with goals of the private sector, prompting the joint provision and management of resources for targeted projects. Examples include the delivery of services or facilities in the energy, transportation, education, or urban development sectors. NIH defines a public-private partnership as an agreement for the agency “to work in concert with a nonfederal party or parties to advance mutual interests to improve health” (NIH, 2007, p. 2). Although gifts, clinical research contracts and other contracts, and technology transfer agreements involve relationships with a nonfederal party or parties, NIH does not consider these arrangements to be partnerships. Other groups may have more expansive interpretations of the concept.
The formation of public-private partnerships involving government, industry, and nonprofit organizations has been a successful model for the infrastructure gaps in the area of neglected tropical diseases, which share with rare diseases the lack of commercial incentives for product development. For example, the multilateral Special Programme for Research and Training in Tropical Diseases (Morel, 2000; Ridley, 2003) and, more recently, the Medicines for Malaria Venture (Ridley, 2002) combine government, philanthropic, and industry funding2 and enlist the expertise of an external scientific advisory board to select projects for support. These initiatives coordinate activities between industry and academic centers (e.g., sharing of compound libraries) to discover new molecules for the treatment of tropical diseases and shepherd them through the subsequent stages along the discovery-development pipeline, thereby acting as “virtual biotech” companies. Projects not meeting specified milestones are dropped and replaced with others, such that each organization manages a portfolio
The malaria venture was, for example, initially cosponsored by the World Health Organization, the International Federation of Pharmaceutical Manufacturers Associations (IFPMA), the World Bank, the Dutch government, the Department for International Development in the United Kingdom, the Swiss Agency for Development and Cooperation, the Global Forum for Health Research, the Rockefeller Foundation, and the Roll Back Malaria Partnership.