Banca de QUALIFICAÇÃO: Camile Giaretta Sachetti

Uma banca de QUALIFICAÇÃO de DOUTORADO foi cadastrada pelo programa.
STUDENT : Camile Giaretta Sachetti
DATE: 07/11/2022
TIME: 14:00
LOCAL: Plataforma Teams
TITLE:

Advanced therapy medicinal products in Brazil: from R&D to market access

KEY WORDS:

advanced therapy medicinal products; ATMPs; R&D; public funding; market access, pharmaceutical companies, Brazil

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

Introduction: The recent advances on biomedical research and biotechnology have promoted the development of personalized therapies for human use. Although Advanced Therapy Medicinal Products (ATMPs) are revolutionizing the way diseases are treated, they represent the most challenging category of new pharmaceutical products, both due to their complex clinical development and high prices. Thus, promoting access to these therapies have become a great challenge for governments and industries, especially in countries with universal healthcare systems. ATMPs have reached the forefront of biotechnological innovation, partly due to public funders' efforts in the early stages of research and development (R&D). In Brazil, the recent consolidation of the regulatory framework for authorizing the sanitary registration of ATMPs has been propelling the commercialization of these products by large, foreigncapitalized pharmaceutical companies. Objective: In this context, the primary objective of this study is to analyze the evolution of investments made by the Ministry of Health (MoH) of Brazil and partners in the ATMPs field between 2004 and 2020. Additionally, the secondary objective is to identify the challenges and opportunities from the research path to the market access to ATMPs in a national context from the perspective of national and foreign capital pharmaceutical industries. Method: A descriptive analysis was performed based on secondary data. The object of analysis of the study consisted of scientific, technological, innovation research and of projects to research infrastructure supported in the field. The database was stratified by: year of funding; ATMPs type; type of study or research infrastructure project; amount invested in the project; targeted disease for which clinical trials in ATPMs were developed; and the financing sector (health, education, science & technology – S&T, and economy). To reach the secondary objective, a descriptive study will be developed, based on semi-structured interviews organized in axes. The number of foreign and domestic pharmaceutical industries will be defined by non-probabilistic purposive sampling. All interviews will take place between November and December of 2022. For the statistical analysis, the simple frequency distribution of the variables set in the script of the interview will be carried out. Results of the primary objective: 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 type of ATMP that most benefited from public investment, totaling 82.23% of the total funding in the analyzed period. Cardiology (29%) and Neurology (21%) were the main focus of clinical trials. Following the global trend of public sector R&D funding, the number of basic and preclinical research represented 78.06% of the total number of projects. Conclusions: Despite the need to implement improvements in R&D of ATMPs financing policy in Brazil, the country has made important steps in the field and can serve as a benchmark for other countries with socio-economic similarities. Among the main lessons are the prioritization of researches aligned with the health needs of the population, the cross-sectoral articulation by the health policymaker to coordinate the R&D efforts of this sector in the country, and the formulation of a specific sectoral policy (Programa Genomas Brasil) to promote the knowledge translation. Considering the large number of ATMPs that may reach the Brazilian market in the coming years, as well as the scenario of worsening technological and production dependence, this project will enable the generation of new, original information that can subsidize the decision making process, especially in the context of the Brazilian National Health System.

BANKING MEMBERS:
Presidente - 1681340 - EVERTON NUNES DA SILVA
Interno - 549.640.403-72 - JORGE OTAVIO MAIA BARRETO - UFPI
Externa à Instituição - MARIA SUELI SOARES FELIPE - UCB
Externo à Instituição - MARTIN HERNAN BONAMINO - INCA