Banca de DEFESA: Camile Giaretta Sachetti

Uma banca de DEFESA de DOUTORADO foi cadastrada pelo programa.
STUDENT : Camile Giaretta Sachetti
DATE: 15/12/2023
TIME: 09:00
LOCAL: plataforma teams - link disponível na página do programa
TITLE:

ADVANCED THERAPY MEDICINAL PRODUCTS IN BRAZIL: CHALLENGES AND OPPORTUNITIES FROM RESEARCH TO ACCESS

KEY WORDS:

Advanced Therapy Medicinal Products; ATMPs; Brazil; R&D; public funding; Unified Health System; regulatory

PAGES: 100
BIG AREA: Ciências da Saúde
AREA: Saúde Coletiva
SUMMARY:

Introduction: Advanced Therapy Medicinal Products (ATMPs) are innovative biologics that holds the promise of revolutionizing treatments, particularly for rare diseases and unmet medical needs. However, they represent the most challenging category of new technologies, both due to their complex development and high prices, which impose specific regulatory demands. Therefore, promoting access has become a major challenge for governments and industries, especially in countries with universal and comprehensive health care. Goals: The first aim of this study was to analyze the evolution of Research and Development (R&D) investments made by the Ministry of Health (MoH) of Brazil and partners in the ATMPs. Second, it was identify the industry's experience in clinical development, marketing authorization and access to ATMPs through the Unified Health System (SUS, acronym in Portuguese), from a regulatory perspective. Methods: A descriptive analysis was performed based on secondary data to achieve the first aim. Additionally, a survey containing structured questions was conducted among research participants who work at companies that commercialize ATMPs, and a descriptive analysis was performed. Results: The investments coordinated by the MoH (61.5%) in partnership with S&T, education, and economy sectors (38.5%) consisted of Int$ 137.35 million in 282 ATMPs projects. Funding included scientific, technological, and innovation research (67% of the total amount) and projects to implement or maintain infrastructure in selected research centers (32.98%). On the global convergence, cell therapy was the ATMP that most benefited from public investment, especially basic and preclinical research. Cardiology and Neurology were the focus of clinical trials. The survey included 10 foreign pharmaceutical companies. Overall, participants assessed that Brazil has a well-established regulatory system, especially the sanitary registration by the National Health Surveillance Agency (Anvisa), which ensures the quality, safety, and efficacy of the products. The Agency's good interaction with the regulated sector, the harmonization of sanitary and ethical assessment systems with other countries, and the analysis time in the biosafety assessment of Genetically Modified Organisms (GMOs) stand out as positive in industry’s evaluation. On the other hand, it is important to advance the pricing regulation for these products since Brazilian regulations do not establish specific criteria for ATMP. Despite the need to implement improvements in the Brazilian investment policy of R&D of ATMPs, as well as to advance regulatory improvements (especially in pricing and HTA), Brazil has made important progress and can serve as a benchmark for other countries with socio-economic similarities. To provide the population with universal, equitable and sustainable access to ATMP in the SUS, it is recommended to promote continuous financing of R&D and create effective public policies to promote the national Health Economic-Industrial Complex, based on a mission-oriented that considers value for public health. Considering the increasing number of approvals of cell and gene therapies in Brazil in the coming years and the financial impact on the SUS, it is suggested to consider explicit priority-setting approaches to support HTA, as well implement encourages innovative models of financing, especially those that consider risk-sharing and co-financing between combinations of interested parties, such as public-public and/or public-private participation (Blended Finance).”

COMMITTEE MEMBERS:
Externa à Instituição - LEONOR MARIA PACHECO SANTOS - UnB
Presidente - 1681340 - EVERTON NUNES DA SILVA
Interna - ***.356.791-** - MARIA SUELI SOARES FELIPE - USP
Externo à Instituição - MARTIN HERNAN BONAMINO - INCA