The so-called Advanced Therapies comprise a new therapeutic class that includes advanced cell therapy, tissue engineering and gene therapy products. The products resulting from advanced therapies are biological products, used for therapeutic purposes, obtained from human cells and tissues that have undergone a manufacturing process; or products consisting of recombinant nucleic acids and whose purpose is to regulate, repair, replace, add or delete a genetic sequence or modify the expression of a gene.
These products are a great therapeutic promise for complex diseases and without medical alternatives available, but also a challenge to the development of control mechanisms that guarantee their quality, safety and effectiveness.
In the States and in Europe, there are already records of advanced therapy products. It is possible to highlight the patient’s own culture of chondrocytes (cells present in the cartilage tissue) with indication for repairing symptomatic cartilage defects caused by trauma (type of advanced cell therapy); corneal epithelial cells also from the patient himself, cultured in a fibrin matrix, indicated for the repair of corneal damage (type of tissue engineering); and genetically engineered autologous T cells for the treatment of patients with lymphoblastic leukemia (gene therapy).
In Brazil, the regulatory framework developed by the National Health Surveillance Agency (Anvisa), aims to stimulate national and transnational investments in Research and Development, in addition to promoting the development of advanced therapy products in the country. Cryopraxis, through its co-sister Cellpraxis, has been investing in this issue for more than two decades.
Currently, Anvisa is based on RDC 214/2018, which provides for the adoption of good practices in the use of human cells for therapeutic use and clinical research, by RDC 260/2018, which establishes criteria for conducting clinical research in the area of cellular and gene therapies and by RDC 338/2020, which provides for the registration of advanced therapy products. Provided that in accordance with its rules, the new standard allows therapy with blood cells and umbilical cord tissue in patients with rare diseases, with no chance of cure or with strictly limited therapeutic options.