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.
Title : The foundation for rare disease and its role in the european rare disease research landscape
Daniel Scherman, Foundation for Rare Diseases, France
Title : The effect of Eupalinolide B on amyotrophic lateral sclerosis: A case report
Wang Huaixiu, Shanxi Provincial Hospital, China
Title : Progress related in genetic research on kawasaki disease
Jiao Fuyong, Shaanxi Provincial People’s Hospital, China
Title : Covid-19 seems to be Initiated by the heparan-sulfate dysregulation by coronavirus: The use of low-molecular- weight heparin (LMWH) can prevent and treat covid-19 when it Is used in early stages, as a heparan-sulfate-regulating medicine
Fereshteh Sedaghat, Sedaghat Memory Clinic, Iran (Islamic Republic of)
Title : Lumevoq gene therapy in leber hereditary optic neuropathy
Magali Taiel, GenSight Biologics, France
Title : Drug recommendation system using a collaborative filtering in machine learning
J. Somasekar, Jain University, India