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

International Conference On Orphan Drugs and Rare Diseases

August 21-23, 2023

August 21 -23, 2023 | London, UK

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
Committee Members
Speaker at Orphan Drugs and Rare Diseases 2023 -  Daniel Scherman

Daniel Scherman

Fondation Maladies Rares, France
Speaker at Orphan Drugs and Rare Diseases 2023 -  Jiao Fuyong

Jiao Fuyong

Children’s Hospital of Shaanxi Provincial People’s Hospital, China
Speaker at Orphan Drugs and Rare Diseases 2023 -  Victor Shengkan jin

Victor Shengkan jin

Rutgers University, United States
Orphan Drugs 2023 Speakers
Speaker at Orphan Drugs and Rare Diseases 2023 - Candida Fratazzi

Candida Fratazzi

BBCR Consulting - Massachusetts, United States
Speaker at Orphan Drugs and Rare Diseases 2023 - Philip Bielefeld

Philip Bielefeld

Avignon Hospital, France
Speaker at Orphan Drugs and Rare Diseases 2023 - Harsha Rajasimha

Harsha Rajasimha

Jeeva Informatics Solutions Inc, United States

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