In 2018, the time to register medicines for rare diseases in Brazil was three times lower than for other drugs. In relation to rare diseases, Anvisa - Brazilian Health Surveillance Agency - took the average time of 170 days for registration of biological products and 196 days for new synthetic drugs. For other medicines, this period was 520 days for organic products and 515 days for synthetic drugs.

Rare diseases are usually chronic, progressive, degenerative, and often life threatening. There is usually no effective cure and therapeutic options are restricted. According to the Brazilian policy of care for people with rare diseases, they are defined as those that affect up to 65 people in every 100 thousand individuals, based on official Brazilian data or, when nonexistent, on data published in technical-scientific documentation.

Among the medicines for rare diseases registered in recent years are Spinraza (nusinersena), indicated for the treatment of patients with spinal muscular atrophy (SMA), and Translarna (atalureno), indicated for the treatment of pediatric male patients with dystrophy muscle of Duchenne. In addition, Soliris (eculizumab), indicated for the treatment of adults and children with nocturnal paroxysmal hemoglobinuria (PNH), stands out.

SOURCE: http://portal.anvisa.gov.br/noticias?p_p_id=101_INSTANCE_FXrpx9qY7FbU&p_p_col_id=column-2&p_p_col_pos=1&p_p_col_count=2&_101_INSTANCE_FXrpx9qY7FbU_groupId=219201&_101_INSTANCE_FXrpx9qY7FbU_urlTitle=doencas-raras-registro-e-tres-vezes-mais-rapido&_101_INSTANCE_FXrpx9qY7FbU_struts_action=%2Fasset_publisher%2Fview_content&_101_INSTANCE_FXrpx9qY7FbU_assetEntryId=5515470&_101_INSTANCE_FXrpx9qY7FbU_type=content