Orphan drugs protocol assistance

In order to encourage pharmaceutical companies to invest in orphan drug development, legislation provides for a number of incentives. These include application fee waiver (the extent of reduction varies with the region of the world, and in the European Union it is 50% for all fees since 2002), market exclusivity and protocol assistance. In the European Union, there is 100% reduction in the fee applicable to the provision of any scientific advice. The fund made available by the Community for fee exemptions for orphan medicinal products amounts to= 3 700000 in 2005. 

Within the Act itself, the four major benefits are (1) the period of marketing exclusivity, which may be considered as a type of patent (2) the tax benefits on clinical trials between the date of orphan drug designation and NDA approval and (3) the FDA s Office of Orphan Products Development grants to support clinical trials on orphan drugs. A fourth benefit of protocol assistance, from the FDA, was always available for important new drugs (as well as others) and is important, but not necessarily new. Nonetheless, it is useful to call attention to this provision. 

Food and Drug Administration assistance to orphan drug developers in protocol design for new drug approval (NDA) or product license approval (PLA) applications 28... 

The clinical research tax credit (discussed in chapter 8), protocol assistance, and clinical research grants theoretically lower the cost of orphan drug R D the market exclusivity provision increases the expected revenues to such R D. In practice, the protocol assistance has had little effect, especially in recent years, and the tax credit and grants program represent, overall, a relatively small commitment of Federal funds to orphan products. This commitment may be critical for certain drugs, however, so it should not be discounted. 

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