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.
Title : What does a technology-enabled patient concierge mean to the orphan drugs industry?
Harsha Rajasimha, Jeeva Informatics Solutions Inc, United States
Title : Improving regulatory confidence for formal early phase clinical development.
Candida Fratazzi, BBCR Consulting - Massachusetts, United States
Title :
Victor Shengkan jin, Rutgers University, United States
Title : Scleritis and systemic diseases: What should know the internist/rheumatologist ?
Philip Bielefeld, Avignon Hospital, France
Title : Progress related in genetic research on Kawasaki disease
Jiao Fuyong, Children’s Hospital of Shaanxi Provincial People’s Hospital, China
Title : Lumevoq Gene Therapy in Leber Hereditary Optic Neuropathy
Magali Taiel, GenSight Biologics, France