International Conference On Orphan Drugs and Rare Diseases

August 17-19, 2023

August 17 -19, 2023 | Online Event

Post-Marketing Safety and Treatment Outcomes

Post-Marketing Safety and Treatment Outcomes

To examine the safety and efficacy of new pharmaceuticals, a multi-stage process involving drug discovery, animal studies, and human clinical trials is used. Rare disease medication development entails a lower number of patients, a higher proportion of children, and a more convoluted clinical presentation. Post-approval studies are intended to address a lot of faults that have been identified in rare disease clinical trials. Observational studies, pragmatic trials, and randomized controlled studies are all examples of post-approval research for rare disorders. Original data collecting studies and the utilization of secondary data are both examples of observational studies (retrospective studies). Original data collection can help retrospective investigations overcome restrictions caused by inadequate information in secondary data sources. Disease registries focus on specific health care outcomes associated with a single product and may include a comparator of an alternative therapy or therapies, whereas product-related registries focus on specific health care outcomes associated with a single product and may include a comparator of an alternative therapy or therapies.

  • Drug Discovery
  • Animal Studies
  • Human Clinical Trials
  • Observational Studies
  • Pragmatic Trials
  • Randomized Controlled Studies
  • Post-Marketing Surveillance
  • Regulatory Requirements
  • Pharmacovigilance
  • Pharmacoepidemiology
  • Risk Evaluation
  • Mitigation Strategies
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

Submit your abstract Today

Watsapp