academia. He also stressed the need for better target validation and safety at all levels. Tim Coté reviewed the history of the orphan drug law and its current status. He also presented ten strategies for moving orphan drug development forward more rapidly and efficiently (see Chapter 3).


Four models for drug development were described: a not-for-profit pharmaceutical company, a foundation that operates a virtual company linking investors with biopharmaceutical companies, a for-profit company with a vested interest in rare diseases, and a global private-equity fund dedicated to advancing drug discovery (see Chapter 4).

The Institute for OneWorld Health, the not-for-profit pharmaceutical company, eliminated the profit requirement from its business plan. Seed money was provided by the Gates Foundation. Acknowledging that a single funding source is not a sustainable model, the Institute is now seeking additional funders, with the ultimate goal of being able to support itself partially or fully with revenues from marketed products.

Cystic Fibrosis Foundation Therapeutics (CFFT) has shown how a disease-oriented foundation can become a virtual drug company. CFFT establishes business partnerships with pharmaceutical companies, lowering their risk in the development of drugs for rare disorders by providing financial support, access to leading cystic fibrosis experts and research tools, and access to the Cystic Fibrosis Therapeutic Development Network of Cystic Fibrosis Care Centers for facilitation of clinical trials.

Genzyme approaches the development of drugs for rare diseases as a for-profit venture. There are several keys to the sustainability of this model: the therapy must be effective and must address an unmet medical need, presumably one involving a condition that is life-threatening or causes severe morbidity; there must be a global market; and the price must be sustainable. Partnerships can allow for the development of a product that would not be possible for a single company, and less profitable drugs can be developed if they are part of a larger portfolio.

Celtic Therapeutics is a global private equity firm that functions as a virtual pharmaceutical company, acquiring or investing in novel therapeutic candidates to bridge the gap between discovery and preclinical development and late-phase clinical trials/approval. As well-financed pharmaceutical company pipelines dwindle, underfinanced biotechnology companies have drug candidates but lack the resources to develop them. This new venture management group plans to fill a portion of its portfolio with promising drug candidates for rare or neglected diseases that are in Phase II, develop the products to the point at which a large pharmaceutical partner would be interested, and sell them at auction to pharmaceutical companies. An

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