HYBRID EVENT: You can participate in person at Rome, Italy or Virtually from your home or work.

2nd International Conference on Orphan Drugs and Rare Diseases

March 17-19, 2025

March 17 -19, 2025 | Rome, Italy

Orphan Drug Therapeutics

Orphan Drug Therapeutics

Orphan therapeutics are pharmaceuticals used to diagnose, prevent, or treat life-threatening or life-altering diseases or disorders that are rare. Because of the small patient groups required for clinical trials, orphan drug development is particularly difficult. By definition, rare diseases affect fewer than 200,000 Americans, with some affecting only a few hundred. There are about 7000 circumstances like this. Because the pharmaceutical business has little interest in developing and promoting treatments for a tiny number of patients, these drugs are referred to as "orphan" drugs. The exceptionally high cost of bringing a therapeutic product to market would not be covered by predicted sales, according to drug makers. As a result, the prospective market for novel pharmacological treatments is minimal, and the pharmaceutical industry would suffer a financial loss as a result. The FDA has provided several perks and incentives to pharmaceutical and research businesses to help with medication development and treatment choices for orphan diseases.

  • Challenges in Orphan Drug Development
  • Drug Repurposing/Repositioning
  • Regulatory Considerations for Drug Repurposing
  • Future Perspectives

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