ANVISA breaks records of approved products for rare disease
Last February the Brazilian health regulatory agency - ANVISA celebrating the Mundial day of Rare Disease announced that during 2019, as a result of an important effort had approved: 21 medicines, 10 new treatments and 15 molecular biology tests/ citogenetic / immunoassays for rare disease.
According to WHO it's considered a rare disease the disease that affect 65 patients in 100 thousand people. In Brazil 13 million patients are affected by one among 8.000 listed diseases. It's estimated that 85% of rare disease have a genetic base and 20% are environmental.
Specific protocols were created for disease like: Multiple Sclerosis, Acromegaly and Spinal Muscular Atrophy and others for Fabry's Disease, Niemann-Pick Type C Disease, Pompe's Disease, Bullous Epidermolysis, Homocystinuria and Intellectual Disability are in preparation.
In addition to that, a recently resolution (RDC 338/2020) approved will comes into force in June for advanced technologies clinical trials define the necessary regulatory bases to develop and register products based on human cells and genes, becoming a reference in Latin America.
The rare diseases are very complex and it represents a big challenge for the whole world. Many of these diseases have no treatment or few scientific information and there is a need for trained professionals. Therefore, the Ministry of Health's permanent effort to incorporate new technologies and edit protocols that guide the diagnosis and treatment of these diseases. The early detection and an appropriate approach can reduce the complications and symptoms of rare diseases, improving the person's quality of life.