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 13-15, 2025

March 13 -15, 2025 | Rome, Italy

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 2025 - Sergey Suchkov

Sergey Suchkov

The Russian University of Medicine & Russian Academy of Natural Sciences, Russian Federation
Speaker at Orphan Drugs and Rare Diseases 2025 - Vladlen Slepak

Vladlen Slepak

University of Miami, United States
Speaker at Orphan Drugs and Rare Diseases 2025 - Harsha Rajasimha

Harsha Rajasimha

Jeeva Clinical Trials, Inc, United States
Orphan Drugs 2025 Speakers
Speaker at Orphan Drugs and Rare Diseases 2025 - Vladlen Slepak

Vladlen Slepak

University of Miami, United States
Speaker at Orphan Drugs and Rare Diseases 2025 - Harsha Rajasimha

Harsha Rajasimha

Jeeva Clinical Trials, Inc, United States
Speaker at Orphan Drugs and Rare Diseases 2025 - Sergey Suchkov

Sergey Suchkov

The Russian University of Medicine & Russian Academy of Natural Sciences, Russian Federation
Speaker at Orphan Drugs and Rare Diseases 2025 - Melissa Keenan

Melissa Keenan

La Jolla Labs, United States

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