The Regulatory Pathway for Advanced Cell Therapy and Gene Therapy Products in Brazil: A Road to Be Built

Abstract

The regulation of cell therapy and gene therapy products is a major challenge for the Brazilian state. From a legal point of view, the legislative apparatus, including constitutional, prohibits the marketing and patent of human substances. From the point of view of the organization of the state bureaucracy, the responsibilities for the regulation of research and application of these technologies in humans may involve up to four different institutions. The National Agency for Health Surveillance (ANVISA) has been the protagonist in structuring the regulation of cell therapy and gene therapy in Brazil, and steps have been taken to ensure quality of these products. However, obstacles such as the commercialization of these therapies and the need to determine whether these products will be regulated following the assumptions adopted in Brazil for drugs and biological products or for human blood and tissues still remain.

Global Regulatory Developments for Clinical Stem Cell Research: Diversification and Challenges to Collaborations

“…The development of a regulatory framework for clinical stem cell research in Brazil has been challenging for two reasons: religious opposition to stem cell research, and a constitutional prohibition that bans the commercial use of human cells and tissues [44]. Religious protests first flared up in 2005, when the Brazilian Congress approved Law #11,105, which legitimized the use of human embryonic stem cells (hESC) for research, including in clinical trials.…”

“…Religious protests first flared up in 2005, when the Brazilian Congress approved Law #11,105, which legitimized the use of human embryonic stem cells (hESC) for research, including in clinical trials. According to this law, the regulation for the production and clinical use of hESC and other types of stem cells (with the exception of bone marrow transplants) fell under the responsibility of Brazil's National Agency for Health Surveillance (ANVISA), the country's national drug regulatory authority [44]. Yet, following a complaint by the Catholic Church at the Brazilian Supreme Court, the authorization of the use of embryos for research purposes was suspended for three years.…”

“…In 2008, a final verdict confirmed that the 2005 law was valid, and that hESC research could go ahead [45]. According to officials of ANVISA this threeyear deliberation delayed the development of effective regulation also for other types of stem cells [44]. A first regulatory step for the clinical use of stem cells was issued by ANVISA in March 2011, in the form of ANVISA Board Resolution #9.…”

“…It did not address standards for clinical trials and market authorization. The reason for this was that the Brazilian constitution prohibits the commercialization of human body materials, including human cells and their derivates [44]. As a result, market approval and commercial distribution have until to this moment not been permitted.…”

“…Regulatory debates on this issue within ANVISA and the Brazilian MOH are ongoing. However, because ANVISA has since 2013 worked on a draft regulation for clinical trials for advanced cell products, it is expected that commercialization of stem cell products will ultimately be approved in Brazil [44]. One consequence of this constitutional prohibition is that the number of for-profit providers of experimental stem cell interventions has been much lower than in other countries [5].…”