Orphan Drug legislation

Details about orphan drug legislation, its application and associated procedures and guidance notes are available at the EMEA website (see end of chapter). 

True incentives for the pharmaceutical industry are required in order for any orphan drug legislation to be successful. Without true incentives, the legislation will have little, if any, effect. The major incentive required is a period of exclusivity for marketing. Without this incentive, industry is not likely to modify its activities in this area. A 10-year period of marketing exclusivity ruther than seven at in the U.S. is appropriate... 

Promote "orphan drug" legislation to encourage development of compounds with insufficient economic potential for unsubsidized development. 

Orphan Drug legislation has been passed in Australia and Japan, and is currently under consideration in a number of other Asian countries. In general, prevalence criteria have trended towards that used by the EU, and that in the USA remains relatively liberal. However, the use of a fixed number of 200 000 cases causes a progressive decrease in the prevalence criteria when the denominator is a growing US population (Fox 2002). 

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. 

The chapter on orphan populations described what constitutes an orphan population and an orphan drug , the history of legislation, and the current inducements for industry. 

Legislation to amend the Orphan Drug Act has been slow. Early in 1994 a compromise proposal was introduced, which would reduce the period of marketing exclusivity from 7 to 4 years, allow more than one version of an orphan drug if they were developed simultaneously, and allow withdrawal of exclusivity for orphan products when the patient population exceeds 200 000. Orphan drugs of limited commercial potential could be granted an additional 3 years exclusivity if sales information and related data justified it. ... 

Second, abuses of the law by the pharmaceutical industry must be prevented if orphan drug privileges are to survive. Politicians may well wish to establish sales caps in the future, and legislate that market exclusivity must disappear when the cumulative sales of a drug reach a predetermined level. The sales cap should represent a fair incentive to the companies, yet protect the government or other groups from excessive payments. 

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