3/30/2020
The Brazilian health surveillance agency Anvisa issued a rule on the registration of advanced therapy medicinal products (RDC No. 338 of February 20, 2020) upon proof of efficacy, safety and quality for use and marketing in Brazil. The new rule will come into force on June 1.
Advanced therapy refers to medicinal products that use genes, tissues or cells to treat diseases such as cancer, thus offering excellent innovation prospects.
This rule has been subject to a 45 days’ public consultation (public consultation No. 706/2019) and also to a Sector Dialogue. In addition, Anvisa has put in place an Advanced Therapy Technical Committee, the CAT, which will assist Anvisa’s plenary board in regulating, reviewing, registering and conducting post-registration activities concerning advanced therapy products.
The rules in force before the new rule was enacted only addressed the development of clinical trials. They were: plenary board rule No. 214 of February 7, 2018, providing for the Good Practices in Human Cells and laying down minimum manufacturing criteria; and plenary board rule No. 260 of December 21, 2018, providing for the rules on clinical trials using advanced investigational trials in Brazil.
The new rule also lays down the criteria based on which companies may register advanced therapy products – a new product category created by the rule itself. This will make room for more research and treatment of rare diseases or diseases with no therapeutic alternative.
According to the new rule, products which can be registered as advanced therapy products are gene therapy products and tissue engineering products. Registrations will be valid for 5 years; after a second renewal, registrations will be valid for 10 years.
Advanced therapy products which are manufactured on a non-routine basis, for a specific patient under imminent risk of life, for treatment of diseases with no therapeutic alternative available in Brazil will not be eligible for registration. Instead, a prior authorization notice or communication will be required in such cases. A legally qualified professional will have to be in charge of such advanced therapy products and marketing of such products are expressly prohibited by the new rule.
Anvisa will have 365 days to reply to all requests of registration of advanced therapy medicinal products classified as ordinary and 120 days when the product is classified as high-priority.
A product will be classified as high-priority if any of the requirements below is fulfilled: (i) if it is used for rare, neglected, emerging or reemerging diseases or for public health emergencies or under severely debilitating conditions and whenever no therapeutic alternative is available; (ii) if it has a new therapeutic use or extended use for children; (iii) if stage I or II of clinical trials has been conducted in Brazil.
Advanced therapy medicinal products will only be registered after publication of the Good Manufacturing Practices certificates for all companies involved in the manufacturing of the active ingredient and the end product.
However, if the manufacturing chain involved in the manufacturing of the advanced therapy medicinal product is certified by an officially recognized international health surveillance authority, then Anvisa may grant the registration request after examining the inspection report and other related documents to be provided by the registration applicant.
Import and export requirements with respect to advanced therapy medicinal products will be set by specific regulation.
Anvisa made an announcement emphasizing that advanced therapy products have the potential to maximize benefits to patients suffering from complex diseases and without any medical alternatives.
Anvisa is currently monitoring eight clinical studies using advanced gene and cell therapies. There are three other clinical trials which are in the initial stages and will be analyzed by Anvisa.
