CHALLENGES IN THE RESEARCH COMMUNITY:
Research in rare and orphan diseases faces two major hurdles:
- Epidemiology: low number of subjects/patients.
- Financial constraints: these diseases are considered economically uninteresting.
Therefore, research focuses on:
- Basic research
- Clinical research
- Therapeutic trials
Research on rare and orphan diseases is important. Rare diseases can serve as a model for more common diseases and consequently help a large population. For research to be optimal, it is necessary that it be developed in collaboration with specialists from around the world, thereby including the greatest number of patients. This combined effort allows for an exchange of multidisciplinary expertise and best practices.
To motivate members of the industry to enter this small market, EU Regulation No. 141/2000 provides the opportunity to develop market “niche”, something of particular interest for small and medium enterprises. Even though this policy has enabled the development of orphan drugs, most rare diseases are still without effective treatment.
It is imperative to strengthen links between universities and industry to capitalize on the results of academic research by using them to develop new diagnostic and therapeutic tools.