International Conference On Orphan Drugs and Rare Diseases

August 17-19, 2023

August 17 -19, 2023 | Online Event

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
Orphan Drugs 2023 Speakers
Speaker at Orphan Drugs and Rare Diseases 2023 - Vicki Ratner

Vicki Ratner

ESSIC - International Society for the Study of BPS, United States
Speaker at Orphan Drugs and Rare Diseases 2023 - Kondakova O. B

Kondakova O. B

National Medical Research Center for Children's Health Federal state autonomous institution of the Russian Federation Ministry of Health, Russian Federation
Speaker at Orphan Drugs and Rare Diseases 2023 - Paul L Kaufman

Paul L Kaufman

University of Wisconsin-Madison, United States
Speaker at Orphan Drugs and Rare Diseases 2023 - Magali Taiel

Magali Taiel

GenSight Biologics, France

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