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

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 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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