Orphan criteria

As discussed earlier, the first step in orphan product development is the determination that the product is indeed entitled to orphan designation. The government gives one criterion as a prevalence of 200,000 individuals in the United States (not incidence). Extensive research into the disease must be conducted. The object of this chapter is not to discuss research techniques however there are numerous government sources or national foundations that may be enlisted to help research the disease with appropriate demographic data. All such data should be carefully compiled and tabulated for presentation to the government. 

Medical value of the drug. In the author s opinion, this is the most important criterion to judge (or classify) orphan drugs. If the drug does not have, or is not expected to have, high medical value, there are very few instances where its development would make sense. A classification of medical value may be as simple as high, medium and low. 

Since 1985, virtually all orphan designations have met the frost criterion. The exact interpretation of this provision has been subject to dispute. For example, the number of AIDS patients in this country has climbed above 200,000, but several AIDS drugs were designated as orphans early in the epidemic, when the prevalence of the disease was much lower (21). 

An orphan drug is used to treat rare diseases affecting fewer than 200 000 people (US criterion), or no more than five people in 10 000 (European criterion). These drugs are themselves very rare because their development costs are higher than the income from their marketing. 

---