Appendix
The development of orphan drugs has been financially incentivised through US law via the Orphan Drug Act of 1983. The National Organization for Rare Disorders (NORD), which was instrumental in establishing the Act, currently estimates that there are more than 10,000 rare diseases affecting over 30 million Americans. (In the US, ‘rare’ is defined as affecting fewer than 200,000 people; in Europe it is fewer than 5 in 10,000 people.)
Prior to the 1983 Act, 38 orphan drugs were approved in the United States. The success of the original Orphan Drug Act in the US led to it being adopted in other key markets, most notably in Japan in 1993 and in the European Union in 2000. Orphan drug classification methodology Using publicly available sources, EvaluatePharma® identify products that have been granted orphan drug designations in the US, EU or Japan. These products are then classified as ‘EvaluatePharma® orphan drugs’ if:
The product is approved only for use in the indication/s for which it was awarded orphan designation and these indications are covered by Evaluate Pharma®.
Approximately one-fifth of products have designations in orphan and non-orphan indications. Products that have orphan and non-orphan drug designations and are expected to generate less than 50% of their sales in 2015 and 2028 from their orphan-designated indication/s, are excluded from this analysis. This has led to the exclusion of therapies such as Avastin, Opdivo, Enbrel, Herceptin, Humira and Remicade.
EvaluatePharma® may also classify R&D products as ‘orphan drugs – prior to the products receiving this status from regulatory bodies – in the following cases: - The product is being developed in an indication that is classified by regulatory bodies as an orphan disease, and other products for this disease were granted orphan drug designation. - The company developing the product states it is seeking orphan drug designation for the product’s lead indication.