The Orphan Disease Center and Jim Wilson MD, PhD in São Paulo, Brazil, discussing the Pathway to Gene Therapy Approval in Latin America.
The Orphan Disease Center hosted the second “Pathway to Gene Therapy in Latin America” meeting in Sao Paolo, Brazil on November 17-18th 2022.
During this event, representatives from Brazil, Colombia and Argentina, as well as the ODC and the Gene Therapy Program at the University of Pennsylvania, came together to discuss the challenges of developing therapies for rare diseases in Latin America and the necessary global development programs in this challenging commercial landscape.
Over the 2 days, we heard from a panel of speaker representing Fiocruz, Casa dos Raros, EMS Pharmaceuticals, ANVISA, the Federal University of Rio Grande do Sul, Interfarma, the Asociacion Colombiana de Medicos Genetista, and the patient federations of Colombia and Brazil. Speakers and participants had the opportunity to discuss the state of clinical development of therapeutics in Latin America, as well as issues regarding the registration process, pricing, and reimbursement in these countries.
The overarching intent of the meeting addressed the collaboration between Latin American nations, and the potential pathway to approval from regulatory agencies in the different countries. We also discussed the need from key players worldwide to offer support for these goals.
The ODC will continue to work with all the attendees to pursue initiatives for regional collaboration and global access of life-saving therapeutics for rare diseases.