Orphan Drugs

Drugs and biologics intended to treat rare and neglected diseases have been given the name, ‘ Orphan Drugs’  by the Orphan drug designation program for the safe and effective treatment and proper diagnosis or prevention of rare diseases that affect 1:20000 people but are not expected to recover the costs of developing and marketing a treatment drug. In addition to orphan drugs, there are orphan vaccines for disease control and the prevention of spread of contagious rare diseases. Vaccines are expensive to develop for a small number of rare infectious diseases and hence only eight vaccines have been registered with the orphan status. Some of the Orphan drugs are, Rituximab, Bortezomib, Sorafenib, Sacrosidase, Clofezimine, and Nilotinib, etc. The nature of Orphan Drugs can create a difference in the amount of safety information for approval needed for limited number of patients in clinical trials and quality of the clinical trials.

  • Rituxan
  • Soliris
  • Tasigna
  • Avonex
  • Yervoy
  • Rebif
  • Jakavi
  • Kyprolis
  • Novoseven
  • Revlimid
  • Sprycel

Related Conference of Orphan Drugs

Orphan Drugs Conference Speakers