https://repositorio.ufba.br/handle/ufba/523 2026-05-19T22:52:43Z https://repositorio.ufba.br/handle/ri/44502 Farmacoepidemiologia do uso de medicamentos biológicos para doenças inflamatórias intestinais. Castro, Caroline Tianeze de Santos, Carlos Antonio de Souza Teles Background: Inflammatory bowel diseases (IBD) are chronic conditions with rising incidence, and their management has been revolutionized by biological therapies. Despite their high cost, these medications are provided free of charge by the Brazilian Unified Health System (SUS). However, gaps remain in the understanding of usage patterns for these drugs, particularly in contexts of social inequalities and among vulnerable populations such as pregnant women.This study aimed to analyze the global prevalence and geographic variations in the use of biologics for IBD. Population-based studies and/or studies using administrative databases published up to July 2024 were identified in the PubMed/MEDLINE, Web of Science, Scopus, Embase, IBECS, WPRIM, BRISA/RedETSA, and LILACS databases. Among 68 included studies, the estimated global prevalence of biologic use in IBD was 15.06% (95% CI 11.84–18.28%), being higher in Crohn's disease (21.41%; 95% CI 16.31–26.50%) than in ulcerative colitis (9.70%; 95% CI 6.20–13.18%). Most studies were concentrated in high-income countries, highlighting a data gap in other regions. Article 2: Using data from the Brazilian Outpatient Information System (SIA) (2008–2022), an ecological study was conducted to evaluate geographic and temporal patterns of biologic prescriptions for IBD within the SUS. Prais-Winsten regression was used to estimate trends, and the global Moran's index (GMI) and Local Indicators of Spatial Autocorrelation (LISA) were applied to assess spatial autocorrelation and identify spatial clusters of biologic prescriptions, respectively. Biologic prescribing increased from 3.0% to 16.7% during the study period, with infliximab predominating until 2012 and adalimumab from 2013 onward. Biologic prescriptions were more common in Crohn's disease (40.5%) than in ulcerative colitis (3.2%). Prescriptions were concentrated in the Southeast and South regions; however, the Center-West and North regions showed the greatest changes in prescription rates over time. Article 3: This article investigated the effects of material deprivation, income inequality, and human development on biologic prescriptions for IBD in the SUS, using data from SIA (2008-2022) in a fixed-effects negative binomial regression model for panel data. The Brazilian Deprivation Index (BDI), the Gini Index, and the Municipal Human Development Index (MHDI) were evaluated, adjusting for demographic, social, and health variables. Lower biologic prescription rates were found in municipalities in the third (RR 0.75; 95% CI 0.67-0.85), fourth (RR 0.69; 95% CI 0.62-0.78), and fifth (RR 0.65; 95% CI 0.58-0.73) BDI quintiles compared with those in the first quintile. Biologic prescription rates were 14% lower in municipalities with lower income equality (95% CI 0.81-0.92) compared with those with higher equality. In municipalities with a medium MHDI biologic prescribing was 30% lower (95% CI 0.56-0.88), while those with very high MHDI had a 37% higher prescribing (95% CI 1.01-1.86) compared with municipalities with low MHDI. Article 4: Following PRISMA guidelines, a systematic review and meta-analysis was conducted to assess the global prevalence of biologic use among pregnant women with IBD. Searches were conducted in PubMed/MEDLINE, Web of Science, Scopus, Embase, IBECS, WPRIM, BRISA/RedETSA, and LILACS in April 2025 to identify population-based studies and/or studies using administrative data on this topic. Seventeen cohort studies were included. The prevalence of biologic use among pregnant women was 12.18% (95% CI: 8.74– 15.61%), higher in Crohn's disease (21.07%; 95% CI 10.77–31.38%) than in ulcerative colitis (5.41%; 95% CI 3.66–7.16%), with notable geographic variations. Article 5: A retrospective cohort study was conducted using SIA data (2008–2022), including 248 pregnant women aged 12–55 years with Crohn’s disease or ulcerative colitis, with the objective of describing the profile and treatment patterns in this population. Sociodemographic, clinical, and treatment data were analyzed, including medications and discontinuation rates. Most pregnant women had ulcerative colitis (64.92%), received conventional therapies (88.31%), and were treated with monotherapy (97.98%), with mesalazine being the most common drug (72.58%), especially in ulcerative colitis (92.59%). Biologic use was higher in Crohn's disease (38.37%) than in ulcerative colitis (2.47%), with infliximab being the predominant agent. The discontinuation rates at 90 and 180 days were 14.92% and 35.89%, respectively. Conclusions: The use of biological medicines for IBD is expanding globally and in Brazil, yet it remains marked by socioeconomic and regional inequities. Global prevalence was 15.06% in the general population and 12.18% in pregnant women, with a lack of data from low- and middle-income countries. In Brazil, the increase in biologic use is influenced by socioeconomic factors and regional disparities. Among pregnant women, treatment generally follows guideline recommendations, but the high discontinuation rate underscores the need for health education strategies for patients and professionals. The findings highlight the value of administraadministrative data for pharmacoepidemiology and for supporting more equitable health policies. Universidade Federal da Bahia Tese 2025-10-03T00:00:00Z https://repositorio.ufba.br/handle/ri/44501 Fatores associados à funcionalidade de crianças com Síndrome Congênita do Zika Vírus participantes da coorte dica. Pereira, Ana Paula Medeiros Santos, Darci Neves dos This thesis aimed to update knowledge about aspects of the functionality of children with congenital changes and compare the functionality profile between those with and without Congenital Zika Virus Syndrome (CZS), verifying the contribution of environmental factors to functional performance, being constructed in the format of three articles. The first is a systematic review that sought to investigate the scientific evidence on the functionality of children with Congenital Zika Virus Syndrome and identify the environmental factors that are related to the functionality outcome in these children. 16 studies were identified that showed a diversity of deficiencies in body structures and functions in children with CZS that impact the performance of activities and social participation, the latter being little explored. The environmental factors identified as facilitators and barriers to functionality were concentrated on the following themes: products and technologies; health services, systems and policies; public policies; accessibility; social support; attitudes; impacts due to the COVID-19 pandemic. This was followed by a cross-sectional study that compared the functional performance of 113 children with CZS and 68 children with typical development. It was found that the percentage of children performing below expectations was higher in the group affected by CZS (daily activity: 64.6%; mobility: 76.5%; social-cognitive: 69.9%) compared to the group with typical development (daily activity: 1.5%; mobility: 2.0%; social-cognitive: 1.5%). Intermediate social position, worse housing conditions, receipt of both social benefits (BPC and bolsa familia program) or only BPC, suspicion of CMD in caregivers and better quality of domestic stimulation were associated with a higher frequency of below-expected functional performance among children with CZS for all domains of functionality. Finally, in the third article, the relationship between environmental factors and the functionality of children with CZS was investigated. To this end, the hierarchical theoretical model was used. It was found that children without access to education had lower functional performance in the three components: mobility (OR=8.99; 95% CI=2.40-32.59); daily activity (OR =4.58; 95% CI=1.78-11.81) and social-cognitive (OR=3.87; 95% CI=1.41-10.60). Receiving a family grant was associated with better functional performance, with the greatest association being for the social-cognitive component (OR=0.02; CI95%=0.02-0.22), followed by mobility (OR=0.06; CI95%=0.00-0.51) and daily activity (OR=0.10; CI95%=0.01-0.61). A lowquality domestic stimulus showed an association with better functional performance in the mobility component (OR=0.22; 95%CI=0.06-0.82). Social participation and environmental factors are still little investigated in the scientific literature, as functionality is described with emphasis on the function and structure of the body and activity of children with CZS. An association was observed between functional performance and receipt of benefits, access to education and quality of domestic stimulation, which highlights the importance of public policies on access to income and education for children with CZS and their families. Universidade Federal da Bahia Tese 2025-04-30T00:00:00Z https://repositorio.ufba.br/handle/ri/44500 Regulação Sanitária de medicamentos considerados inovadores no Brasil, Estados Unidos e União Europeia. Cardoso, Amanda dos Santos Teles Costa, Ediná Alves Medicines are complex objects involved both health and market interests. They are subject to health regulation, a social mechanism that mediates between public and private interests, aiming to protect health and guide pharmaceutical innovation. Health regulation is a state function, not politically neutral, and in relation to medicines, it articulates technical-scientific knowledge, administrative practices, and political action. Pharmaceutical regulation has become increasingly flexible in establishing rules regarding aspects of health protection in order to accelerate access to medicines considered innovative. Based on a review of scientific literature and an empirical analysis of regulatory documents from Brazil, the United States, and the European Union, this study examined the current modes of health regulation for this category of medicines from the perspective of accelerated approval. The literature review followed the PRISMA methodology for scoping reviews and selected 51 articles for bibliometric analysis and thematic synthesis. Researchers have shown growing interest in the topic in recent years, although few studies have focused on Brazil. The studies addressed health regulation from the perspective of oncology drugs, orphan drugs, and regenerative medicine. They also discussed regulatory strategies and challenges in a faster-paced context for drug approval; the use of new sources of evidence, such as real-world data; and the therapeutic value of medicines considered innovative. The document analysis was based on Minayo’s hermeneutic-dialectical approach and the method of interpretation of meanings. The analysis of regulatory documents on medicines for rare diseases and accelerated approval showed both similarities and differences in the regulation of innovative medicines across the three jurisdictions. The regulatory instruments developed (1) sought to reduce the time required for regulatory review, and (2) modified the standards of scientific evidence. The United States and the European Union also established financial incentives to promote pharmaceutical innovation in the field of rare diseases. The United States was a pioneer in regulatory changes, and both Brazil and the European Union adapted their frameworks to align with regulatory objectives introduced by U.S. legislation. The European Union adopted a more conservative regulatory discourse, whereas Brazil demonstrated greater flexibility through its abbreviated regulatory pathway. The factors influencing the institutionalization of an abbreviated drug development pathway in Brazil included demands from the pharmaceutical market, efforts toward international alignment, Brazil’s entry into the International Council for Harmonisation, and the COVID-19 pandemic. The health regulation of innovative medicines is a complex and dynamic phenomenon, given its mission to protect public health and the set of competing interests within the pharmaceutical field. Current regulatory frameworks have integrated scientific and technological advances to reinforce a paradigm centered on innovation and access, which enhances the value of post-marketing regulatory assessment. New medicines have reached the market more quickly; however, the literature indicates that these changes have not revolutionized therapeutic options, nor led to the introduction of safer medicines or those aimed at neglected populations. This signals that the health regulation of medicines still needs to progress in order to prioritize the interests of patients and public health in the approval of innovative medicines. Universidade Federal da Bahia Tese 2025-05-30T00:00:00Z https://repositorio.ufba.br/handle/ri/44499 Por dentro do Poptrans: “quem” e “como” se produz conhecimento em um estudo sobre saúde e modos de vida de mulheres transexuais e travestis em Salvador, Bahia. Sacramento, Adriana Prates Silva, Luís Augusto Vasconcelos da This thesis takes as object of reflection a research known as Poptrans, highlighting the surroundings and the practical aspects related to the production of knowledge, in order to highlight the relationship between science and politics. To do this, were looked situations such as the composition of the sample, aspects of the field routine and the participation of the public of interest of Poptrans, a research that combined quantitative and qualitative methodologies to address aspects related to the health and lifestyles of transsexual women and transvestites. Were highlighted the numerous associations made up of humans and non-humans that were mobilized and brought together to obtain the Poptrans data , to trace the path of its production and showing how knowledge was constructed in practice, taking into account the most diverse elements that acted in the process, highlighting their condition as mediators, in the sense that they act idiosyncratically, without necessarily depending on a cause; highlighting, based on the mentioned, the situated, contingent and performative nature of knowledge production. The exercise of reflexivity was developed from the position of a cisgender researcher, without disregarding other markers and considering discussions on the themes of epistemicide, extractivism and representativeness. Along the way, the relationship between the dimension of reflexivity and ethics was highlighted in an ANT-inspired report, which sought to meet the demand posed by Ethnoepidemiology to broaden reflexivity in research by applying ethnographically-inspired methods to study scientific practice. Universidade Federal da Bahia Tese 2025-07-17T00:00:00Z