Patient Advocacy Groups – Key Support System When Planning the Launch of an Orphan Drug in Brazil

Patient advocacy groups (PAGs) for orphan diseases in Brazil (e.g., Instituto Vidas Raras, Casa Hunter) support manufacturers during all phases of launching an orphan drug. Specifically, they help navigate the fast-track regulatory process with the Brazil National Health Surveillance Agency (ANVISA), share local epidemiology information, and engage with Brazil’s HTA body, the National Commission for the Incorporation of Technologies (CONITEC), to advocate reimbursement in the public setting. Seeking support from PAGs early on in the planning process is a key step to a successful launch.

PAGs for orphan diseases are in regular contact with ANVISA and offer assistance with submitting the regulatory dossier and registering local clinical trials. On December 29, 2017, ANVISA published Resolution RDC No. 205/2017 (effective on February 26, 2018), which established fast-track procedures for new drugs designed for the diagnosis, prevention, and treatment of rare…