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

Orphan Drugs Clinical Research

Orphan Drugs Clinical Research

Clinical trials funding for orphan products have been shown to be a viable technique of fostering and promoting the development of novel, safe, and effective medical products for rare diseases and conditions. Orphan drug clinical development is both challenging and exciting. There is no single aspect that is truly unique to it; rather, it encompasses the majority of the challenges: design, outcomes, recruiting, ethics, cost, probability, and predictability. To solve these challenges, academic institutions, small and major pharmaceutical firms, patient representatives, and health authorities must work together to give assistance and creative ideas. The ultimate goal is to provide medications with a good benefit-risk ratio to individuals with unmet medical needs. FDA is focusing their efforts with a new funding opportunity to facilitate and move new therapies along in drug development in a safe and efficient manner by encouraging innovative clinical trial methods such as adaptive and seamless trial designs, modelling and simulations, and basket and umbrella trials to address the remaining unmet need and lack of treatments for the majority of rare diseases. These techniques are critical for effective trials and data analysis, which can help speed up drug development.

  • Regulatory Considerations
  • Methodology of Clinical Trials
  • Ethics
  • Post Approval Clinical Studies
  • Costs and Financial Support
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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