Immune checkpoint inhibitors for the treatment of non-small cell lung cancer: The regulatory journey in Brazil.

BACKGROUND: Recent therapeutic advances such as immune checkpoint inhibitors (ICIs) have impact on the care of non-small cell lung cancer (NSCLC) patients, however, they bring new setbacks for regulatory agencies. OBJECTIVE: To evaluate the regulatory journey of ICIs registered for NSCLC treatment in Brazil and to establish comparisons of Brazilian regulatory agency with the US regulatory agency. METHODS AND DATA SOURCE: Information for each ICI prescribing as well as the date of regulatory approval of the therapeutic indications of interest were collected from the Anvisa and the FDA websites. The search took place on October 2022. KEY FINDINGS: There are only 20 % disagreements on regulatory approvals between Anvisa and FDA. The prioritization review at Anvisa in 2008 has made the regulatory assessment faster. CONCLUSIONS: The results of this study identified a potential improvement in Anvisa's time performance to connect the target established by the legal framework of the sector.

Advanced Therapy Products in Brazil: Regulatory Aspects.

Advanced therapy products, considered special medications, require Anvisa approval for use and commercialization in Brazil. They include advanced cellular therapy products, tissue engineering products, and gene therapy products, which due to their complexity involve innovation and risks, optimized regulatory channels for their development and life cycle monitoring. The scientific elements and the compliance with applicable regulatory aspects are fundamental pillars for the advancement of clinical trials, the positive evidence of the benefit-risk profile, and the definition of the critical quality attributes, from the perspective of making safe, efficacy, and high-quality products available to the population. The approval models of these products in Brazil adapt to the specificities and characteristics of the technology and the patient target population, with accelerated regulatory analyses, use in emergency situations by risk controls and specific monitoring mechanisms, principally those related to rare diseases without other therapeutic alternatives. The opportune access to the advance therapy product with safety, efficacy, and quality involves innovative normative elements that include the long-term follow-up of the safety and efficacy and of the adaptive pharmacovigilance requisites, as well as the traceability mechanisms for starting materials, products, and patients.

Mapeamento de processo regulatório para aprovação de vacinas novas provenientes de desenvolvimento nacional

The availability of medicines to the population necessarily goes through a long regulatory path, where several steps must be fulfilled. In this scenario, ANVISA is the national regulatory agency responsible for establishing the regulations that companies, producers and developers must follow. The objective of this work is to map the different regulatory processes involved in the approval of a vaccine, a type of biological medicine, developed by companies or national research institutions. Therefore, there is a focus on local regulations that involves the regulatory approval of a vaccine. The steps from the initial stages of development of a vaccine are accompanied by processes that are guided by regulatory requirements, often complex and with different directions. Until final release to the public, the rules governing this release involve several approvals that must be strictly followed by developers. Thus, the scope of this work is to delineate the regulatory processes from the initial stages of development of a vaccine, through the distinct phases of clinical trials, to its final approval for distribution to the general public, contributing to a global comprehension by researchers of the regulatory process as a whole.

A disponibilização de medicamentos à população passa necessariamente por um longo caminho regulatório, onde várias etapas devem ser cumpridas. Neste cenário, a ANVISA é o órgão responsável pelo estabelecimento da regulamentação que as empresas, produtores e desenvolvedores devem seguir. O objetivo deste trabalho é mapear os diversos processos regulatórios envolvidos na aprovação de uma vacina, que é um tipo de medicamento biológico, desenvolvido por empresas ou instituições de pesquisa nacionais. Desta forma, foca-se na regulamentação local que envolve a aprovação regulatória de um produto biológico. Os passos desde as etapas iniciais de desenvolvimento de uma vacina são acompanhados de processos que são norteados por requisitos regulatórios, muitas vezes complexos e envolvendo diferentes direcionamentos. Até a disponibilização final ao público, as normativas que determinam essa liberação envolvem diversas aprovações que devem ser rigorosamente seguidas pelos desenvolvedores. Desta forma, o escopo deste trabalho é delinear os processos regulatórios desde as fases iniciais de desenvolvimento de uma vacina, passando pelas diferentes fases de ensaios clínicos, até a sua aprovação final para a disponibilização à população, contribuindo para fornecer uma compreensão global aos pesquisadores do processo regulatório como um todo.

Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular Consensus on genetically modified cells. Special Article: Advanced therapy medicinal products in Brazil: regulatory panorama.

Advanced therapy medicinal products, considered special medications, requires Anvisa approval for use and commercialization in Brazil. They include the advanced cellular therapy products, tissue engineering products and gene therapy products, which due to their complexity involve innovation and risks, optimized regulatory channels for their development and life cycle monitoring. The scientific elements and the compliance with applicable regulatory aspects are fundamental pillars for the advancement of clinical trials, the positive evidence of the benefit-risk profile and the definition of the critical quality attributes, from the perspective of making safe, effective and high-quality products available to the population. The approval models of these products in Brazil adapt to the specificities and characteristics of the technology and the patient target population, with accelerated regulatory analyses, use in emergency situations by risk controls and specific monitoring mechanisms, principally those related to rare diseases without other therapeutic alternatives. The opportune access to the advance therapy product with safety, efficacy and quality involves innovative normative elements that include the long-term follow-up of the safety and efficacy and of the adaptive pharmacovigilance requisites, as well as the traceability mechanisms for the start-off materials, products and patients.

Current clinical research environment: focus on psychiatry

The introduction of international guidelines on Good Clinical Practices (GCP) in 1996, immediately followed by the publication of Resolution CNS 196/96 in Brazil, created a great opportunity for Brazilian research centers to participate in international trials. Such studies must be strictly monitored in order to assure compliance with the regulations, as well as with the standards of patient safety. Clear agreement among the investigator, the sponsor and the institution carrying out the study must be previously defined in order to avoid any conflicts of interest during or after the study. Operational aspects, such as the time needed to gain regulatory approval of the study design, strategies for patient recruitment/retention and appropriate logistics, are also important. In 2005, the Brazilian National Clinical Research Network was established, bringing together a number of research centers in teaching hospitals. The objective was to subsidize public clinical research with state-of-the-art practices and appropriate technical/scientific training programs. The development of research protocols that prioritize public health care needs in Brazil is other fundamental goal of this network. This article addresses general aspects of clinical research, as well as some specific issues in psychiatry. Improving the health and quality of life of the global population is certainly the major objective of all of the work done in this area.

A introdução de diretrizes internacionais de Boas Práticas Clínicas, em 1996, imediatamente seguida pela publicação da Resolução do Conselho Nacional de Saúde 196/96, abriu uma grande oportunidade para a participação de centros de pesquisa brasileiros em estudos internacionais. Tais estudos devem ser estritamente monitorados, a fim de assegurar a adesão às legislações, assim como garantir a segurança dos pacientes envolvidos. A fim de evitar possíveis conflitos de interesse durante e após o estudo, todos os aspectos relacionados devem ser claramente definidos previamente entre o pesquisador, o patrocinador e a instituição. Aspectos operacionais, tais como tempo para aprovação regulatória do estudo, métodos de recrutamento e retenção de pacientes e a logística em geral, também são importantes. Em 2005, a Rede Nacional de Pesquisa Clínica foi criada no Brasil, somando a experiência de vários centros de pesquisa ligados a hospitais de ensino. Seu objetivo é reforçar a atividade de pesquisa clínica no país, com práticas atualizadas e adequado treinamento técnico-científico. O desenvolvimento de protocolos de pesquisa que foquem as prioridades de saúde do país é outro objetivo fundamental da Rede. Aspectos gerais da pesquisa clínica e algumas particularidades em Psiquiatria são discutidos no artigo. O objetivo final de todo o trabalho nesta área de pesquisa é, sem dúvida, a busca de melhores condições de saúde e qualidade de vida da população.