Protocols 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. 

Details of guidance for companies requesting scientific advice and protocol assistance can be accessed on the EMEA website (see end of chapter). 

Details of guidance for companies requesting sdentific advice and protocol assistance http //www.emea. eu.int/pdfs/human / sciadvice/426001 en.pdf... 

Advice to companies on drug development (scientific advice and protocol assistance)... 

European Medicines Agency. New framework for scientific advice and protocol assistance. EMEA/267/187/2005 Rev. 1,26 April 2006. http //www.emea.europa.eu/ pdfs/human/sciadvice/26718705en.pdf... 

If requested, the sponsor can have help with protocol assistance from the FDA during development. 

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. 

Once the OOPD determines that the proposed compound is for a disease or condition that is rare in the U.S., the request will be forwarded to the responsible division for formal review and direct response. The OOPD monitors the review process within the respective CDER/CBER reviewing division and, where possible, assists in resolving specific issues that may arise during the review process. It should be understood that protocol assistance provided under the Act does not waive the necessity for the submission of an Investigational New Drug Application (IND) by sponsors planning to conduct clinical trials with the product. 

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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