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

Clinical Research and Developments

Clinical Research and Developments

A rare disease, often known as an orphan disease, is a medical disease that only affects a small percentage of the population. A pharmacological agent developed specifically to treat an orphan disease is known as an orphan drug. The journey from the discovery of a new drug to its commercialization is lengthy (10 years on average), costly (tens of millions of euros), and fraught with risk (among ten molecules tested, only one may have a therapeutic effect). The development of a drug to treat a rare condition does not allow for the recovery of the money spent on research. Over the previous two decades, the percentage of new medicines approved for orphan drugs has risen considerably. Approximately 500 orphan designated medications have been approved for the treatment of a variety of oncological, metabolic, hematologic, immunologic, infectious, and neurological disorders. Patients with rare diseases must be kept up to date on scientific and treatment developments.

  • Clinical Trial Transparency
  • Data Sharing
  • Global Market Opportunity
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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