Cost-effectiveness analysis in healthcare: documentary study of theses and dissertations

Abstract

Objective  To characterize Brazilian theses and dissertations that used cost-effectiveness analysis methods in health technologies and the respective decision models.

Method  This documentary and quantitative study was carried out in February 2023 with a search in the Catalog of Theses and Dissertations (Coordination for the Improvement of Higher Education Persons) in the period 2011-2022 using the term “cost-effectiveness”. After applying the inclusion and exclusion criteria, 81 studies were included in the study and then a quantitative analysis of theses and dissertations was carried out.

Results  The majority of studies were doctoral theses (54.3%), defended in 2018 (22.2%) in southeastern Brazil (60.5%) and conducted in a hospital setting (42.0%). The Markov Decision Tree model (37.0%) (28.4%) was adopted, followed by other models. Furthermore, most theses followed the recommendations of good practices in economic cost-effectiveness analysis: they met a largely (55.6%), minimally (25.9%) and partially (18.5%) of the necessary items and recommendations.

Conclusion  The cost-effectiveness studies carried out in the theses analyzed used the Decision Tree model and followed best practice recommendations in their development.

Cost-effectiveness analysis; Health evaluation; Markov chains; Models, econometrics; Health postgraduate programs

Resumo

Objetivo  Caracterizar as teses e dissertações brasileiras que usaram métodos de análise de custo-efetividade em tecnologias de saúde e os respectivos modelos de decisão.

Métodos  Este estudo documental e quantitativo foi realizado em fevereiro de 2023 com busca no Catálogo de Teses e Dissertações (Coordenação de Aperfeiçoamento de Pessoas de Nível Superior) no período 2011-2022 usando o termo “custo-efetividade”. Após a aplicação dos critérios de inclusão e exclusão, 81 produções foram incluídas no estudo e então foi realizada a análise quantitativa das teses e dissertações.

Resultados  A maioria das produções era de teses de doutorado (54,3%) defendidas em 2018 (22,2%) na região sudeste do Brasil (60,5%) e conduzidas em ambiente hospitalar (42,0%). Foi adotado o modelo de Árvore de Decisão (37,0%) de Markov (28,4%), seguido de outros modelos. Além disso, a maioria das teses seguiu as recomendações de boas práticas na análise econômica de custo-efetividade: elas atenderam grande parte (55,6%), minimamente (25,9%) e parcialmente (18,5%) os itens necessários e recomendações.

Conclusão  Os estudos de custo-efetividade realizados nas teses analisadas usaram o modelo de Árvore de Decisão e seguiram as recomendações de boas práticas em seu desenvolvimento.

Análise de custo-efetividade; Avaliação em saúde; Cadeias de Markov; Modelos econométricos; Programa de pós-graduação em saúde

Resumen

Objetivo  Caracterizar las tesis de doctorado y de maestría brasileñas que utilizaron métodos de análisis de costo-efectividad en tecnologías de salud y los respectivos modelos de decisión.

Métodos  Este estudio documental y cuantitativo fue realizado en febrero de 2023 con búsqueda en el Catálogo de Tesis de Doctorado y de Maestría (Coordinación de Perfeccionamiento de Personas de Nivel Superior) durante el período 2011-2022 usando el término “costo-efectividad”. Después de aplicar los criterios de inclusión y exclusión, se incluyeron 81 producciones en el estudio y luego se realizó el análisis cuantitativo de las tesis de doctorado y de maestría.

Resultados  La mayoría de las producciones fue de tesis de doctorado (54,3 %) defendidas en 2018 (22,2 %) en la región Sudeste de Brasil (60,5 %) y llevadas a cabo en ambiente hospitalario (42,0 %). Se adoptó el modelo de Árbol de Decisión (37,0 %) de Markov (28,4 %), seguido por otros modelos. Además, la mayoría de las tesis siguió las recomendaciones de buenas prácticas en el análisis económico de costo-efectividad: cumplieron gran parte (55,6 %), mínimamente (25,9 %) y parcialmente (18,5 %) los ítems necesarios y recomendaciones.

Conclusión  Los estudios de costo-efectividad realizados en las tesis analizadas utilizaron el modelo de Árbol de Decisión y siguieron las recomendaciones de buenas prácticas en su desarrollo.

Análisis de costo-efectividad; Evaluación en salud; Cadenas de Markov; Modelos econométricos; Programas de posgrado en salud

Introduction

The escalation in healthcare spending has been presented as a problem within national financial constraints. Furthermore, there is a lack of knowledge about the real cost of providing healthcare services to users in a scenario of population aging with overlapping acute and chronic conditions.¹,² Thus, health systems, which have limited resources, are facing increasing pressure to meet growing population demand. To do this, they need to improve their efficiency, avoid wasting resources by applying economic assessment methods.³

In parallel with financial restrictions, the launch of new technologies and the pressure for their incorporation in the healthcare sector attract attention around the world. National health systems seek to protect the sector by reducing the negative economic impact and ensuring access to users. In Brazil, technology assessment methods have been developed to manage their introduction into the Brazilian Health System (SUS – Sistema Único de Saúde).⁴

Health technology assessment (HTA) is a comprehensive process through which the clinical, social and economic impacts of health technologies are assessed. Its objective is to help in making assertive decisions about the incorporation of new technologies into the system.⁵ Thus, the HTA process assessed safety, efficacy, effectiveness, clinical indications and the benefiting population considering the burden of the disease and its impact on society. HTA’s economic aspect consists of assessing costs with cost-effectiveness (CEA), cost-utility, cost-benefit, cost-minimization and budgetary impact analysis. As for patients, the following items are considered: characteristics and social impact of the disease, relevance and benefit of technologies already used, demands not yet addressed, including acceptability, convenience and ethical aspects regarding the incorporation of a new product or process.⁶HTA’s organizational aspect also includes assessment of dissemination feasibility, professional training, optimized use of resources, monitoring of results and sustainability of the new technology.⁷

Health technologies’ CEA allows to compare costs (in monetary units) and results (in non-monetary units). In other words, it deals with clinical impacts, the relationship between costs and benefits of a given health treatment.⁸ CEA-type economic assessment studies are relevant as they simultaneously assess the costs and effectiveness of the technology used. Furthermore, economic issues (such as cost-effectiveness) must be considered when choosing a technology, as this contributes to the most appropriate option for both patients (regarding the most effective technology) and institutions when choosing the most cost-effective technology.⁹

Therefore, it is important to know the academic production (theses and dissertations) on methodologies used in CEA of health technologies in the country. Furthermore, it is essential to assess how these studies were conducted in terms of best practice recommendations. Considering the academic role of generating scientific knowledge, studies must present specific items to be transparent, replicable, interpretable and understandable.⁹ More than that, such studies can identify gaps in existing knowledge, thus contributing to adopting the methodological rigor recommended by good practices and enabling more robust and quality approaches and results for more assertive health decisions.¹⁰,¹¹

Therefore, we questioned the following: what are the characteristics of Brazilian academic production that applied CEA methods in health technologies and what decision models were adopted? Therefore, this study aimed to characterize Brazilian theses and dissertations that used CEA methods in health technologies and the respective decision models.

Methods

This was a documentary study with a quantitative approach carried out after searching the Thesis and Dissertation Catalog (Coordination for the Improvement of Higher Education Personnel, CAPES CAPES (Coordenação de Aperfeiçoamento de Pessoal de Nível Superior); Ministry of Education). The search was carried out in February 2023 by the main researcher using the term “cost-effectiveness” (structured vocabulary from the Health Sciences Descriptors (DeCS)). Filters were applied in the large knowledge area “health sciences” and temporal fields to restrict the theses and dissertations defended in the period 2011-2022. The year 2011 was chosen because it was the year when the Brazilian National Commission for the Incorporation of Technologies into the Brazilian Health System (CONITEC - Comissão Nacional de Incorporação de Tecnologias no Sistema Único de Saúde) was established in Brazil (Decree 7,646; 12/21/2011).⁶

Theses and dissertations on CEA, linked to the area of health sciences, developed in Brazil and defended from 2011 onwards, were included. Theses and dissertations that did not present CEA with the respective decision model in the abstracts were excluded. Furthermore, those where the file was not available in full were excluded. To collect the data, an instrument was developed that included the following characterization items: year of defense; study design; geographic region of the university of origin; institution; graduate program; methodological approach; method; target population; study perspective; context; time horizon; technology (intervention and comparator); willingness to pay threshold; discount rate; currency; costs; effectiveness; sensitivity analysis; budgetary impact; model assumptions; and decision model used. The items listed in the instrument were based on the components recommended for preparing CEA according to the Consolidated Health Economic Evaluation Reporting Standards (CHEERS).⁹ Thus, to classify the level of quality of the findings based on an economic assessment study,¹² the following stratification parameters were adopted: meet the recommendations minimally (<40% of items: reasonable quality), partially (41-80% of items: moderate quality), and largely (>80% of items: good quality).

The initial search yielded 43,676 results, 4,229 of them were linked to the broad area of health sciences, resulting in a final sample of 932 studies (including studies from 2011 onwards). The selection of this material was carried out independently by two reviewers, and the result of their consensus is presented in a flowchart in Figure 1 (adapted from Preferred Reporting Items for Systematic Review and Meta-Analyses, PRISMA).¹³ To access full texts, database tools and Google searches were used.

Figure 1
Theses and dissertations selection process (adapted from PRISMA)

Data analysis consisted of extracting information from each thesis or dissertation. To describe the selected studies, a synoptic table prepared for this purpose was used with year of publication, institution, area of knowledge, study design, participants and research setting, level of academic training, as well as the model of decision used for CEA. Descriptive statistical analysis (absolute and relative frequencies) was performed using the Excel program (Microsoft Office, 2010).

As this study was documentary research, and all information is in the public domain, it was not necessary to be assessed by a Research Ethics Committee. However, ethical aspects related to authorship and insertion of citations were respected.

Results

The study included 81 academic productions (theses and dissertations) that applied the CEA methodology to assess technologies for the health sector (8.7% of the total studies in the period investigated). Most studies were presented in 2018 (22.2%); they were theses (44; 54.3%) and dissertations (37; 45.7%) from institutions in the Southeast (48; 60.5%), South (17; 19.8%), Midwest (10; 12.3%) and Northeast (6; 7.4%). The Universidade de São Paulo presented the highest production (20; 24.7%), followed by the Universidade Federal do Rio Grande do Sul (8; 9.8%), Universidade Federal do Estado do Rio de Janeiro (6; 7.4 %) and Universidade Federal do Paraná (5; 6.1%), these being the five most observed institutions. The five graduate programs with the highest production were health sciences (20; 24.7%), followed by public health (19; 23.4%), nursing (11; 13.6%), pharmaceutical sciences (9; 11.1%), dentistry (3; 3.8%), HTA (2; 2.5%) and health assessment (1; 1.2%). Concerning the method used, observational (21; 25.9%), hypothetical cohort (14; 17.2%), economic assessment (11; 13.6%), systematic review (9; 11.1%), clinical trial (4; 5.0%), followed by other method studies (one study for each method; 22; 27.2% predominated). Most of studies were carried out in a hospital setting (42.0%), involving patients undergoing treatment (42.0%). In relation to the types of decision model for CEA, the Decision Tree was the most used model (30; 37.0%), followed by the Markov model (23; 28.4%). The Markov Model and the Decision Tree were used together (3; 3.7%), and the Dynamic Model was used alone (1; 1.2%) in the analyses. In some studies, other methods were used to assess cost-effectiveness (4; 5.0%); in other studies, the model used was not specified (20; 24.7%). In terms of compliance, the studies largely (45; 55.6%), minimally (21; 25.9%) and partially (15; 18.5%) met the items recommended by CHEERS ⁹ (Chart 1).

Chart 1
Synthesis of studies included in the study and compliance with the items recommended in CHEERS

Author’s name	Year	Level	Title	Level of service
Gomes MP	2013	Dissertation	Análise de custo-efetividade do tratamento supervisionado e autoadministrado da tuberculose	Largely
Finkelsztejn A	2013	Thesis	Esclerose múltipla no Brasil: estudo de custo da doença e de custo-efetividade dos tratamentos disponíveis no país	Largely
Costa DNO	2013	Dissertation	Custo-efetividade da vacina oral monovalente contra rotavírus humano (VORH) no Brasil	Partially
Salgueiro MM	2013	Dissertation	Análise custo-efetividade através do método de análise envoltório de dados em ensaio clínico de tratamento com dose padrão e alternativa de antimoniato de meglumina em pacientes com leishmaniose tegumentar americana na forma cutânea	Minimally
Cintra MACT	2013	Thesis	Avaliação do custo efetividade da profilaxia do vírus sincicial respiratório em lactentes com cardiopatia congênita	Partially
Villafuerte SDR	2014	Dissertation	Análise de custo-efetividade da cintilografia óssea de corpo inteiro na avaliação pré-transplante hepático de pacientes com carcinoma hepatocelular em um centro de referência na região Sul do Brasil	Minimally
Fernandes RRA	2014	Dissertation	Custo-efetividade do uso do palivizumabe na prevenção de internações por doença respiratória grave em crianças de alto risco infectadas pelo vírus sincicial respiratório na perspectiva do Sistema Único de Saúde	Largely
Maia CR	2014	Thesis	Estudo para avaliação de custo-efetividade do tratamento do transtorno de déficit de atenção/hiperatividade com metilfenidato de liberação imediata no Brasil	Partially
Inoue KC	2014	Thesis	Custo-efetividade de duas coberturas na prevenção de úlcera por pressão sacral	Minimally
Stella SF	2014	Thesis	Custo efetividade de tecnologias contemporânea no manejo da doença arterial coronariana: stents recobertos e cateter de medida de fluxo (FFR)	Largely
Costa RN	2014	Thesis	Estudo comparativo entre os métodos percutâneo e cirúrgico no tratamento da comunicação interatrial do tipo ostium secundum em crianças e adolescentes: análise da segurança e eficácia clínica e do custo-efetividade incremental	Largely
Nunes SEA	2014	Thesis	Estimativa dos custos da doença pneumocócica e estudo de custo-efetividade da introdução universal da vacina antipneumocócica 10 valente no Brasil	Largely
Saccilotto IC	2014	Dissertation	Análise da efetividade do centro de referência estadual do Hospital de Clínicas de Porto Alegre para tratamento do mieloma múltiplo	Minimally
Cunha PDJ	2014	Dissertation	Análise de custo e efetividade do tratamento de diabéticos adultos atendidos no Centro Hiperdia de Juiz de Fora, Minas Gerais	Minimally
Chevrand WP	2015	Dissertation	Avaliação da custo-efetividade da dabigatrana na fibrilação atrial	Largely
Araújo RL	2015	Dissertation	Custo-efetividade da Terapia Tripla para pacientes adultos portadores do genótipo 1 da hepatite C crônica	Largely
Ungari AQ	2015	Thesis	Análise de custo-efetividade de esquemas quimioterápicos no tratamento do câncer colorretal metastático sob a perspectiva de um hospital público de ensino	Largely
Lopes ASA	2015	Dissertation	Custo efetividade do tratamento coletivo para esquistossomose no estado de Pernambuco, Brasil	Minimally
Borges LR	2015	Thesis	Custo-efetividade da prova tuberculínica versus QuantiFERON-TB Gold-In-Tube no diagnóstico e tratamento da infecção latente tuberculosa em profissionais de saúde da Atenção Básica no Brasil	Largely
Maia SCOM	2015	Dissertation	Análise de custo-efetividade do tratamento da hepatite C crônica genótipo 1: comparação da adição do boceprevir à terapia padrão (interferon-α peguilhado e ribavirina)	Largely
Rodrigues FA	2015	Thesis	Avaliação do controle pressórico de pacientes hipertensos em seguimento farmacoterapêutico de acordo com o índice de complexidade da farmacoterapia e análise de custo-efetividade	Partially
Munaretto JRS	2015	Dissertation	Avaliação do custo-efetividade da incorporação do Espectômetro de Massa em Tandem no Programa Nacional de Triagem Neonatal no Brasil para diagnóstico da Fenilcetonúria e MCAD	Largely
Steimbach LM	2016	Dissertation	Análise de custo-efetividade das formulações de anfotericina B	Partially
Cruz BA	2016	Dissertation	Análise de custo-efetividade da intervenção coronária percutânea guiada pelo fluxo fracionado de reserva do miocárdio	Largely
Luque	2016	Thesis	Uso de estatina em pacientes com doença isquêmica do coração: análise de custo-efetividade	Largely
Bastos CRG	201	Thesis	Custo-efetividade da tomografia de emissão de pósitron (PET scan) no diagnóstico diferencial do nódulo pulmonar solitário na perspectiva do Sistema Único de Saúde	Largely
Langsch LRS	2016	Dissertation	Avaliação do custo efetividade do tratamento da Doença de Alzheimer leve e moderada no Brasil	Largely
Moura ACMR	2016	Dissertation	Análise de custo e custo-efetividade do tratamento diretamente observado da tuberculose e sua expansão no estado do Rio de Janeiro	Largely
Santos AS	2016	Dissertation	Custo-efetividade dos medicamentos antipsicóticos utilizados para o tratamento da Esquizofrenia no Brasil	Largely
Júnior JRL	2016	Dissertation	Estudo do custo-efetividade do tratamento paliativo dos tumores da confluência biliopancreática mediante comparação entre as abordagens laparotômica e endoscópica	Minimally
Entringer AP	2017	Thesis	Análise de custo-efetividade e de impacto orçamentário do parto vaginal comparado à cesariana eletiva em gestantes de risco habitual	Largely
Zuccolotto VMR	2017	Dissertation	Avaliação do custo e da efetividade da radiofrequência na dor cervical crônica com componentes autonômicos simpática e somática e um hospital público	Minimally
Jarek NAA	2017	Dissertation	Análise de custo-efetividade do tratamento precoce da hepatite viral C crônica com simeprevir, daclatasvir e sofosbuvir sob a perspectiva do SUS	Largely
Souza TO	2017	Dissertation	Custo-efetividade da estimulação ovariana mínima versus estimulação ovariana convencional em pacientes más respondedoras submetidas à fertilização in vitro	Minimally
Laranjeira FO	2017	Thesis	Avaliação das tecnologias disponíveis para diabetes Tipo 1 no Brasil: efetividade, impacto orçamentário e custo-efetividade	Largely
Gomes RKS	2017	Thesis	Impacto dos medicamentos imunobiológicos e da doença coronária na evolução de pacientes com artrite reumatoide: uma análise de custo-efetividade	Largely
Moreira APA	2017	Thesis	Incorporação tecnológica no contexto da saúde: uma análise de custo efetividade das bombas de infusão na insulinoterapia intravenosa	Minimally
Rodrigues VO	2017	Dissertation	Aférese x centrifugação do sangue total: análise de custo-efetividade entre os distintos procedimentos para produção do concentrado de plaquetas	Minimally
Fernadez CRSHW	2017	Dissertation	Análise de custo-efetividade da técnica de IMRT para o tratamento do câncer de próstata localizado	Largely
Viscondi JYK	2017	Thesis	Análise de custo-efetividade de estratégias de rastreamento do câncer do colo do útero no Brasil	Largely
Pedrolo E	2017	Thesis	Custo-efetividade das coberturas para cateter venoso central de curta permanência	Largely
Marques LF	2017	Thesis	Custo-efetividade da utilização da central de mistura intravenosa em unidade de terapia intensiva neonatal	Largely
Carvalho RP	2018	Dissertation	Custo-efetividade da abordagem sistematizada do sangramento digestivo de origem obscura em hospital terciário e proposta de protocolo clínico e de regulação para rede assistencial hierarquizada e de complexidade crescente	Minimally
Magacho FLR	2018	Dissertation	Análise de custo-efetividade dos imunossupressores utilizados no tratamento de manutenção do transplante renal	Partially
Avelino NR	2018	Dissertation	Análise do custo-efetividade da implantação de uma unidade de prática integrada no cuidado de idosos com fratura do quadril	Minimally
Oliveira LS	2018	Dissertation	Custo-efetividade das ferramentas de captação	Minimally
Júnior LHB	2018	Thesis	Análise do custo efetividade de um Serviço de Atenção Domiciliar	Minimally
Pessoa JA	2018	Dissertation	Análise do custo-efetividade do uso do stent farmacológico vs. stent não farmacológico na intervenção coronariana percutânea uniarterial no Sistema Único de Saúde	Largely
Fernandes EG	2018	Thesis	Avaliação de custo-efetividade da introdução da vacina tríplice acelular do adulto (dTpa) no calendário de imunização de adultos do Programa Nacional de Imunização no Brasil	Largely
Brandão SMG	2018	Thesis	Custo-efetividade e custo-utilidade dos tratamentos clínicos, cirúrgicos e percutâneo em portadores de doença coronariana multiarterial estável	Partially
Silva OLO	2018	Thesis	Análise do custo-efetividade da Iniciativa Hospital Amigo da Criança na promoção da amamentação e redução da mortalidade infantil	Largely
Pontes SC	2018	Dissertation	Avaliação cardiológica pré-operatória: custo-efetividade do ecocardiograma com stress farmacológico comparado a angiografia coronariana em pacientes de alto risco cardiovascular assintomáticos, submetidos a transplante renal na santa Casa de Juiz de Fora	Partially
Scudeler TL	2018	Thesis	Custo-efetividade da cirurgia de revascularização do miocárdio com e sem circulação extracorpórea em pacientes portadores de doença coronariana multiarterial estável: resultados do estudo MASS III	Largely
Sousa DMN	2018	Thesis	Análise custo-efetividade de protocolo clínico para detecção de lesões precursoras do câncer de colo uterino (progyne)	Partially
Santos AS	2018	Thesis	Limiares de custo-efetividade e a precificação de medicamentos: aripiprazol para o tratamento da esquizofrenia no Brasil	Largely
Campos TM	2018	Dissertation	Avaliação do custo-efetividade da fotobiomodulação na mucosite oral em pacientes com câncer de cabeça e pescoço	Minimally
Ramos MCA	2018	Thesis	Análise de custo-efetividade de programa para diagnóstico de mutação germinativa em genes BRCA1/2 e de estratégias preventivas para pacientes com câncer de ovário e seus familiares de primeiro grau	Largely
Teodoro VB	2018	Dissertation	Análise custo-efetividade aplicada à avaliação do padrão alimentar de pacientes usuários da atenção básica do SUS no município de Bauru, SP	Minimally
Fonseca GGP	2018	Dissertation	Custo-efetividade da espuma multicamadas de poliuretano com silicone comparada ao filme transparente de poliuretano na prevenção de lesão por pressão	Largely
Kühr EM	2018	Thesis	Custo e efetividade da reabilitação cardíaca na cardiopatia isquêmica	Minimally
Oliveira KRS	2019	Dissertation	Custo-efetividade do serviço diagnóstico por imagem em policlínicas, comparando a emissão de laudos via medicina convencional x telemedicina: subsídio à tomada de decisão gerencial	Minimally
Sousa DF	2019	Dissertation	Custo-efetividade incremental da farinha do albedo do maracujá amarelo versus cúrcuma no controle glicêmico e lipídico de pessoas com diabetes mellitus tipo 2	Minimally
Brito NC	2019	Thesis	Infiltração intralesional de antimoniato de meglumina para tratamento de leishmaniose cutânea localizada: revisão sistemática, metanálise e análise de custo-efetividade	Largely
Souto MLS	2019	Thesis	Custo-efetividade da terapia de cessação do tabagismo para pacientes com periodontite	Largely
Garmatter LPL	2019	Thesis	Avaliação da eficácia, segurança e custo-efetividade dos medicamentos utilizados no tratamento da acromegalia	Largely
Hartmann R	2019	Thesis	Efetividade e custo-efetividade de três alternativas de tratamento para reabilitação do desdentado mandibular: ensaio clínico randomizado com acompanhamento de 1 ano	Partially
Ribeiro APL	2019	Thesis	Análise custo efetividade do plasma rico em plaquetas no tratamento de úlceras venosa	Largely
Oliveira RAR	2019	Thesis	Análise da custo-efetividade do rastreamento e das modalidades terapêuticas do câncer de próstata	Partially
Matuoka JY	2019	Thesis	Análise econômica do denosumabe e dos bisfosfonatos no tratamento de pacientes com metástase óssea	Largely
Lucchetta RC	2019	Thesis	Eficácia, efetividade, segurança e impacto orçamentário das terapias modificadoras da doença para esclerose múltipla recidivante-remitente	Partially
Di Giaimo CRM	2019	Thesis	Avaliação econômica do uso de ômega-3 na redução dos fatores de risco cardiovascular: análise de custo-efetividade	Partially
Neto MHM	2019	Dissertation	Custo-efetividade de opções reabilitadoras fixas para reposição de um único dente	Largely
Santos WM	2019	Thesis	Análise de custo-efetividade de antimicrobianos usados no tratamento da Klebsiella pneumoniae carbapenemase	Largely
Arnaldi AP	2019	Dissertation	Análise de custo-efetividade dos protocolos LLA 97 e GBTLI 99 para crianças em tratamento de leucemia linfocítica aguda	Minimally
Santos WM	2019	Thesis	Custo-efetividade de intervenções no aumento da adesão a terapia antirretroviral e redução da carga viral em pacientes com HIV	Largely
Farah DMAT	2020	Thesis	Avaliação da segurança, eficácia e custo-efetividade dos métodos contraceptivos de longa duração em comparação aos métodos convencionais em adolescentes	Largely
Carmo PB	2020	Dissertation	Angiotomografia de coronárias no SUS: Futuro ou Realidade? Análise da Custo-Efetividade dos métodos não invasivos para diagnóstico de DAC obstrutiva estável em pacientes sintomáticos na perspectiva do SUS	Partially
Gomes RM	2020	Thesis	Custo-efetividade dos regimes imunossupressores utilizados na manutenção do transplante renal: coorte de dezesseis anos no Sistema Único de Saúde	Largely
Miranda MC	2020	Thesis	Análise do custo-efetividade da triagem neonatal para a hiperplasia adrenal congênita: comparação de duas populações diagnosticadas com e sem triagem neonatal	Largely
Fantin SS	2021	Thesis	Análise econômica do acesso venoso central em adultos hospitalizados: resultados clínicos e comparação da custo-efetividade na perspectiva de um hospital público universitário	Largely
Torreglose CG	2021	Thesis	Análise de custo-efetividade do Programa Alimentar Brasileiro Cardioprotetor (BALANCE): um estudo randomizado, multicêntrico em indivíduos com doença cardiovascular	Partially

Table 1 presents a compilation of recommendations and/or items to prepare an economic analysis (including cost-effectiveness). The majority of productions presented the recommended items for preparing CEA as recommended by CHEERS.

Table 1
Items recommended by good practices for preparing cost-effectiveness analyzes presented in Brazilian theses and dissertations in health

Recommended items for preparing a cost-effectiveness analysis	n(%)
Target population
Patients undergoing treatment	34(42.0)
Patients in hospital	19(23.5)
Primary Care Users	10(12.3)
Not specified and/or not mentioned	18(22.2)
Perspective
Brazilian Health System	68(84.0)
Brazilian Health System and Supplementary System	2(2.5)
Supplementary System	1(1.2)
Society	1(1.2)
Not specified and/or not mentioned	9(11.1)
Context
Hospital	34(42.0)
Primary attention	15(18.6)
Other health institutions	14(17.2)
Not specified and/or not mentioned	18(22.2)
Time horizon
Less than one year	17(20.9)
1-4 years	20(24.7)
5-10 years	12(14.8)
11-20 years	7(8.6)
21-40 years	3(3.9)
Over 40 years	9(11.1)
Not specified and/or not mentioned	13(16.0)
Technology (intervention and comparators)
Pharmacological	38(46.9)
Non-pharmacological	35(43.2)
Pharmacological and non-pharmacological	8(9.9)
Willingness to pay threshold
Not specified and/or not mentioned	51(62.9)
Yes	30(37.1)
Discount rate
Not specified and/or not mentioned	45(55.6)
Yes	36(44.4)
Coin
Not specified and/or not mentioned	53(65.4)
Yes	28(34.6)
Costs
Yes	72(88.9)
Not specified and/or not mentioned	9(11.1)
Effectiveness
Yes	69(85.2)
Not specified and/or not mentioned	12(14.8)
Decision models
Yes	61(75.3)
Not specified and/or not mentioned	20(24.7)
Sensitivity analysis
Yes	52(64.2)
Not mentioned and/or not performed	29(35.8)
Budget impact
Not mentioned and/or not performed	73(90.1)
Yes	8(9.9)
Model assumptions
Not specified and/or not mentioned	56(69.1)
Yes	25(30.9)

Discussion

In Brazil, higher education institutions offer lato sensu, stricto sensu and short-term courses to improve knowledge in the area of economic assessment in health. They also offer courses that make it possible to assess technologies relevant to the healthcare system. Graduate programs in = health play a fundamental role in advancing technical and scientific knowledge, contributing to training qualified human resources. Thus, people can be trained in economic analysis and more assertive decision-making, positively influencing the implementation of SUS actions and services and promoting their sustainability.¹⁴

Brazilian researchers who carry out investigations in health economics are mostly doctors.¹⁵Our findings agree with this statement because theses are more available in the CAPES thesis and dissertation catalog. Furthermore, the large area of knowledge in which health assessment research groups operate (where economic assessments are inserted) is predominantly health sciences.¹⁶

The year 2018 was highlighted by a greater number of theses and dissertations defenses (18; 22.2%) that used CEA methodologies in health technologies. This number may be related to the economic crisis in the Brazilian healthcare sector that began in 2016 with the implementation of a spending cap. This ceiling limited spending in several health sectors and may have influenced the carrying out of studies on economic assessment. Furthermore, 2018 was marked by a significant increase in the incorporation of new health technologies into the Brazilian market. This reinforces the idea that production and questioning about health costs and effectiveness have expanded among researchers and professionals working in the area.¹⁷

The number of graduate programs and research groups on economic assessment in southeastern Brazil is significantly greater, mainly in São Paulo. This is seen as a disparity in the national distribution of programs due to their high concentration in this region of the country.¹⁶,¹⁸ This scenario can also be seen in the data from the present study, with the vast majority of studies developed in this region, and almost a quarter produced at the Universidade de São Paulo. Regarding the graduate program from which the studies originated, the area of health sciences was highlighted (24.7%). Our results agree with this highlight.

A quantitative methodological approach was adopted in thesis and dissertation analysis, reflecting the methodologies applied. This is because the quantitative approach is the most used statistical method to analyze results as well as to develop models and verify costs and consequences in economic studies.¹¹ In relation to the method adopted, observational studies (25.9%), followed by hypothetical cohort studies (17.2%), stood out. Furthermore, there are many possible data sources to develop economic assessments as primary source data from research and secondary data obtained from the literature can be used.¹¹

As for Decision Models, the majority of theses and dissertations (75.3%) adopted some type of model. It is recommended to describe the structure of the chosen model and justify the choice. Furthermore, it is advisable to consider time horizon and the nature of the technology assessed (whether linked to chronic or acute diseases). It is recommended to make a graphical presentation of the model structure and patient flow throughout the model.⁹In theses and dissertations, the decision model most used for CEA was the Decision Tree, followed by the Markov Model. The Decision Tree is a simple assessment method, appropriate for acute events and short time horizons, and is suitable for individuals with similar attributes. The model requires little data preparation (numerical or categorical) and can be validated through statistical tests.¹⁹ Furthermore, the Decision Tree can be easily interpreted and understood, helping to reduce the time needed to make a decision regarding the most cost-effective treatment.²⁰ Differently, the Markov method is used in chronic conditions or over a longer time horizon, and its elaboration, calculation and analysis are relatively accessible.¹⁹, ²¹

Most (45; 55.6%) studies followed good practices in conducting CEA, having presented the recommended items for these analyzes.⁹ Regarding recommended items, most studies had patients undergoing treatment as their target population (34; 42.0%). In economic assessments, the target population description is essential as its description can modify the economic impact of any intervention. Furthermore, costs and consequences can vary significantly depending on participant and research setting and/or context characteristics.⁹

As for the perspective, most studies adopted the SUS perspective, which refers to the point of view from which the problem is judged (i.e., the payer of the services described in the analysis). The objective of this study is directly linked to the perspective, as both will indicate the costs that will be measured.¹⁰ Brazil is a country that invests in health technologies (mainly within the scope of SUS) to ensure the constitutional right to universal access to health. Therefore, managers must engage with economic issues, a necessary aspect for health system sustainability.¹⁴ In 2018, the SUS presented an annual expenditure (on purchases of products and technologies) greater than 20 billion reais (Brazilian currency).²² Therefore, it is understandable that all these factors may have considered study development in this perspective.

This study took place predominantly at hospitals. This follows a review that revealed the existence of hospitals in Brazil with HTA centers. Furthermore, hospital institutions are partners in this type of research to help managers make assertive decisions.²³ Knowledge about hospital costs can help develop a more comprehensive and prepared approach to organizing and optimizing patient care and resource allocation.¹¹,²⁴,²⁵

In most studies, a time horizon of 1-4 years was adopted. In general, time horizon presents the relationship with the natural course of the health condition analyzed to assess the potential impact of interventions, thus representing the study duration.¹⁰

Most studies assessed pharmacological (38; 46.9%) technologies (intervention and comparators), reflecting a global scenario of significant increase in the production and incorporation of hard technologies (equipment, medicines, procedures, etc.)¹⁰ In relation to interventions and comparators, a detailed description of their complexity, forms of presentation, application and/or use, and duration of administration (in the case of medication dosage) is necessary as they are directly linked and influence costs and effectiveness.⁹

Willingness to pay threshold was not adopted in the majority of studies (51; 62.9%); it refers to the amount that the institution is willing to pay for incorporating the new technology. Care must be taken with this item as not all study designs apply to or reflect willingness to pay.⁹ As for discount rate (45; 55.6%), studies did not present this item in the study. Although its value varies depending on the country of origin of the research, its use is relevant as adjusting costs and the consequences of inflation help to reflect changes in prices over time.¹¹ As for currency, the vast majority (53; 65.4%) did not specify this in studies. In general, it is recommended that currency be the first piece of information when valuing cost items.¹⁰In addition, it is necessary to describe the year of conversion, dates of unit costs and estimates made as well as price adjustment methods (when used).⁹

Concerning costs, healthcare service production basically involves human, material, service and structural resources. In general, each assistance provided to a patient uses several resources that may vary and be repeated in other services; hence, the difficulty in determining the costs of a specific service and the need for methodologies to systematize the cost calculation process. Furthermore, cost is a relevant factor for study reproducibility; therefore, the items included in the analysis, selection criteria and data source must be described.¹⁰

Effectiveness is an essential factor in developing a health economic assessment (especially in CEA), as this method requires selecting one or more outcome measures that reflect harm and effectiveness. The studies analyzed (69; 85.2%) described this item as it is necessary to inform what will be considered effective and the methods for measuring effectiveness.⁹

The study identified sensitivity analysis (52; 64.2%) in studies. The method for analyzing this item may vary. Most studies use Monte Carlo simulation as it is one of the most traditionally used metrics. However, other methods (such as Tornado Diagram) are increasingly being reported.⁹ Monte Carlo simulation is a technique for assessing the uncertainty regarding any covariate included in the economic model. Uncertainty is an inherent factor in any economic assessment, and it is necessary to analyze its impact on the result.²⁶,²⁷ Budget impact refers to one of the final stages of HTA, being an assessment of the financial consequences caused by the adoption and/or incorporation of a health technology in a given scenario within up to five years. This type of analysis helps managers make decisions because it estimates the financial feasibility of a certain technology in a healthcare service or system.²⁸ However, only 8 (9.9%) of studies analyzed assessed the budgetary impact.

The model’s premises are based on the assumptions that will be adopted by the author and/or researcher in relation to the outcome of their analysis. This may cause concern as the choice of one parameter over another (or the formulation of a specific assumption) may induce bias in the model, influencing technology assessment in a favorable or unfavorable way.²⁶

The function of economic assessment is to provide clear information to support decision-making. Its role is to determine the investment necessary to obtain specific benefits in a group of patients with different health problems.²⁹ CEA has become an important tool for professionals and managers in the public health system,³⁰ as it helps institutions with sustainability. To achieve this, it is important to create partnerships with educational institutions to train qualified human resources.¹⁰ CEA applicability is relevant to providing assistance and managing health as it enables greater assertiveness in decision-making regarding health technology use.

As limitations of this study, it is important to consider that the search for studies was restricted to the CAPES portal, and some analyzes were hampered by the lack of clarity in the methodological path presentation in some studies. However, this survey provided a comprehensive view of CEA methodologies and the decision model most used in health research in the context of Brazilian graduate studies. Knowing this scenario is essential to improve research, clinical practice and health management. The results of this study allow to identify opportunities, challenges and trends, in addition to encouraging the adoption of best practices in the development of robust and quality CEA, thus contributing to a more efficient, effective and sustainable health system.

The findings presented contribute to identifying gaps in Brazilian academic production on the topic. For instance, there was a lack of production assessing the cost-effectiveness of non-pharmacological technologies. Therefore, conducting studies focused on this methodology is essential to train human resources to make good health decisions. Furthermore, it is important that researchers adopt best practice recommendations for carrying out health economic assessments. This will allow the development of more transparent studies, which are easy to understand, interpret and replicate.

Conclusion

Brazilian academic production in health analyzed the cost-effectiveness of health technologies, with a peak in 2018. Most studies were carried out at a doctoral level within graduate programs in southeastern Brazil, mainly at the Universidade de São Paulo. In the methodological design, observational studies stood out using the Decision Tree model to analyze the cost-effectiveness relationship (CEA). Most studies followed good practice recommendations for CEA, presenting the main items recommended for developing this type of study.

Acknowledgements

For financial support from the CAPES of the Ministry of Education (Code 001).

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Edited by

• Associate Editor
  Alexandre Pazetto Balsanelli (https://orcid.org/0000-0003-3757-1061) Escola Paulista de Enfermagem, Universidade Federal de São Paulo, São Paulo, SP, Brasil

Publication Dates

• Publication in this collection
  04 Oct 2024
• Date of issue
  2024

History

• Received
  10 Oct 2023
• Accepted
  6 May 2024