Challenges in Rare Diseases Treatment

There are around 7,000 rare diseases, which from a regulatory outlook are defined as those diseases where there are less than 200,000 patients in the US or that affect no more than five in 10,000 of the general population in the EU. Orphan drugs are medicinal products envisioned for diagnosis, prevention and treatment of life-threatening rare diseases. They are "orphans" because the pharmaceutical industry has little interest under normal market conditions in developing and marketing drugs intended for only a small number of patients suffering from very rare conditions.

  • Different types of rare diseases
  • Misperceptions about rare diseases
  • Role of Patient Registries
  • Regulatory Approaches and Alternative Methodologies
  • Rising Patient Costs
  • Reimbursement Strategies

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