2nd Annual Orphan Drug Congress 2012

This year’s conference will discuss the major concerns, newest challenges and the best practices in the development of orphan drugs. The use of patient registries, open innovation in rare diseases, designation, the importance of communication between pharma, patients and HTA bodies, use of social media and many more will be covered by the au fait pharmaceutical representatives.


  • Successful development plan for Orphan Drug
  • Regulatory challenges surrounding Orphan drug development
  • Developing patient registries
  • Commercial aspects of orphan drug development
  • Patient perspective


  • Ms Sharon Terry (President and CEO of Genetic Alliance) will open the first day of the conference with keynote presentation “A team of teams: Orphan drug development”.

President of OrphaNet, Ms Segolene Ayme will present the International Rare Disease Research Initiative

Treatments of rare diseases has long represented market niche in the pharmaceutical industry. Recently the importance of developing medicines for rare and obscure diseases have forced traditional pharma companies to re-evaluate their position on orphan and ultra orphan drugs. Despite the incentives relating to marketing exclusivity, taxes and fees, protocol design and research grants for the development of orphan drugs, there remain significant challenges.