Sep 01, 2018 Volume 392 Number 10149 p711-794
Outrageous prices of orphan drugs: a call for collaboration
Lucio Luzzatto, Hanna I Hyry, Arrigo Schieppati, Enrico Costa, Steven Simoens, Franz Schaefer, Jonathan C P Roos, Giampaolo Merlini, Helena Kääriäinen, Silvio Garattini, Carla E Hollak, Giuseppe Remuzzi on behalf of the Second Workshop on Orphan Drugs participants
Few instances of a single act of legislation have shifted industrial policy in the pharmaceutical industry like the Orphan Drugs Act did when it was signed in the USA in 1983. The Act was written to facilitate the development of drugs for rare diseases and health conditions, and the incentives provided by the Act, such as 7 year exclusivity, tax credits of up to 50% of research and development costs, and access to research and development grants, resulted in the US Food and Drug Administration (FDA) approving 575 drugs and biological products for rare diseases between 1983 and 2017—a real success. In 2000, the European Commission passed similar legislation for orphan medicinal products (OMPs). As a matter of fact, the diseases, not the drugs, are the orphans because all drugs are very expensive, having marrying this success story (table).
Although we are dealing with rare diseases, the increasing number of new OMPs introduced each year is beginning to threaten the sustainability of health-care systems.
The socioeconomic, ethical, and legal implications of this state of affairs have been analysed extensively. We have previously discussed these implications, and here we concentrate on possible corrective actions. Although the focus here is on OMPs, our recommendations are applicable to other drugs…