Orphan drugs refer to medicines for rare diseases. This name is justified because there are only few drugs known for rare disease. People with a rare disease have to live with the problem, that although they receive a diagnosis, there will be no medicine available for their illness.
To date, there are approximately 530 different Orphan Drugs (June 2009), some of which are approved for the same disease, although some are not available in all European countries or Switzerland. Orphan drugs have different regulatory procedures; however, pharmaceutical companies have barely invested in research for these drugs, due to the lack of lucrative profit expectations. To enforce this research, special regulatory procedures were established by the USA (FDA), EU (EMA) and Switzerland (Swiss Medic). Research on orphan should be promoted, thanks to these special procedures, but unfortunately too little has been done since the establishment of this legislation 8 years ago.
Some small pharmaceutical companies, working on developing new drugs, are a good example to show that it is worthwhile to invest in development. However, there are so many different diseases that these efforts are merely a drop in the ocean.
Your support for our research and development projects will help achieve our objective and together we can do more for those affected by a rare disease.