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ObjectiveTo establish the clinical comprehensive evaluation index system for Chinese patent medicine（CPM） based on Evidence and Value：Impact on DEcisionMaking（EVIDEM） framework， so as to promote the scientific， systematic and standardized implementation of clinical comprehensive evaluation of CPM. MethodThe clinical comprehensive evaluation index system was determined through literature review， semi-structured interview and Delphi method， and the weights of each index were clarified by analytic hierarchy process（AHP）. ResultThe recovery rates of both rounds of expert consultation were 100%， and the authority coefficient of experts was 0.90 and 0.905， respectively. Kendall's coordination coefficients（W） of the second- and third-level indicators in the first-round consultation were 0.320 and 0.283（P<0.001）， and in the second round were 0.411 and 0.351， respectively（P<0.001）. The finally constructed clinical comprehensive evaluation index system for CPM included 6 first-level indicators， 13 second-level indicators and 28 third-level indicators. Among the first-level indicators， the weights of effectiveness， safety， economy， innovation， suitability and accessibility were 37.34%， 32.68%， 11.85%， 5.87%， 5.79% and 6.47%， respectively. ConclusionThis study has constructed a universal clinical comprehensive evaluation index system for CPM， and the domain and criteria are introduced and interpreted in detail， which can provide reference and information for carrying out the clinical comprehensive evaluation of CPM， but it needs to be refined and improved in combination with the clinical practice of CPM for specialized diseases.
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OBJECTIVE To rapidly assess the efficacy， safety and cost-effectiveness of novel highly selective Bruton’s tyrosine kinase （BTK） inhibitor zanubrutinib in the treatment of chronic lymphocytic leukemia （CLL）， small lymphocytic lymphoma （SLL） and mantle cell lymphoma （MCL）. METHODS Retrieved from PubMed， Cochrane Library， CNKI， Wanfang database， VIP， and health technology assessment （HTA） websites， systematic reviews/meta-analyses， randomized controlled trials （RCTs）， pharmacoeconomic studies and HTA reports related to zanubrutinib were collected from the database/website establishment to July 2023. The literature was screened according to inclusion and exclusion criteria， and its quality was assessed by using relevant evaluation tools. Data extraction was presented by qualitative description. RESULTS A total of 5 literature were included， comprising of 3 RCTs and 2 cost-effectiveness analyses. In terms of efficacy， compared with the control group， zanubrutinib treatment resulted in significantly longer progression-free survival （P＜0.05） and a higher overall response rate （P＜0.05）. However， there was no statistical significance in overall survival between 2 groups （P＞0.05）. In terms of safety， zanubrutinib had lower incidence of cardiac adverse events， incidence of major bleeding events， and drug discontinuation rate due to adverse drug events， compared to first-generation BTK inhibitors ibrutinib； but the risk of bleeding events caused by zanubrutinib was still higher， compared to traditional chemoimmunotherapy （bendamotine+rituximab）. In terms of cost-effectiveness， zanubrutinib was found to be cost-effective in the treatment of recurrent or refractory MCL， compared to ibrutinib. CONCLUSIONS Zanubrutinib demonstrates sound efficacy and safety in patients with CLL/SLL and MCL patients. Furthermore， it exhibits economic advantages for patients with relapsed or refractory MCL.
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Objective To evaluate the efficacy,safety and economy of teriparatide in the treatment of osteoporosis.Methods PubMed,Embase,Cochrane Library,Web of Science,CNKI,WanFang Data,VIP databases and websites related to health technology evaluation were systematically searched to collect high-quality clinical evidence and economic evaluation literature of teriparatide in the treatment of osteoporosis from the inception to January 20,2023.Two researchers independently identified studies,extracted data,assessed the quality of included studies,and descriptive analyzed and summarised the results.Results A total of 25 literatures were included,involving 3 HTA reports,15 systematic review/Meta-analyses and 7 economic studies were included.In terms of effectiveness,the evaluation results showed that teriparatide could improve bone mineral density in patients with osteoporosis,reduce the incidence of vertebral/non-vertebral fractures in primary and secondary osteoporosis and prevent the fractures in postmenopausal osteoporosis compared to bisphosphonates and placebos.In terms of safety,teriparatide was proven to be safe with no elevated risk of adverse drug reactions.In terms of economic cost,teriparatide has a higher cost and economic disadvantage compared with bisphosphonates,however,for people with severe postmenopausal osteoporosis and high risk of fracture,teriparatide can be considered as a potential cost-effect treatment option.Conclusion Teriparatide is effective and safe in the treatment of osteoporosis,but it is not cost-effective advantages compared with the existing other anti-osteoporosis medications.
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AIM:To rapidly evaluate the effective-ness,safety and economy of dienogest in the treat-ment of endometriosis so as to provide evidence-based reference for clinical drug selection and deci-sion.METHODS:Retrieved from PubMed,Embase,Cochrane Library,CNKI,SinoMed,Wanfang and health technology assessment(HTA)organization websites,HTA report,systematic review/Meta-anal-ysis and pharmacoeconomic study were included during the inception to Sep 2023.Data extraction and quality evaluation were carried out for the lit-erature that met the inclusion and exclusion crite-ria,and the research results were summarized and analyzed qualitatively.RESULTS:Nine systematic re-view/Meta-analyses,and 2 pharmacoeconomic studies were included.The quality of Meta-analysis literatures was low,and the quality of economic re-search was good.In terms of effectiveness,com-pared with no treatment,dienogest can significant-ly reduce postoperative recurrence rate,VAS score,and improve pregnancy rate and effective rate;Di-enogest has similar therapeutic effects in terms of recurrence rate as GnRHa and COC;With regard to recurrence rate and pregnancy rate,dienogest is superior to danazol,gestrinone,and mifepristone.In terms of safety,compared with placebo,the inci-dence of vaginal bleeding and headache was signifi-cantly increased with dienogest,and there was no difference in bone loss;The incidence of vaginal bleeding caused by dienogest was significantly high-er than that of GnRHa,but the incidence of hot flashes and bone loss was lower.In terms of econo-my,dienogest has a more cost-effective advantage comparing with GnRHa,but does not have econom-ic benefits comparing with COC.CONCLUSION:Di-enogest has good effectiveness and safety in the treatment of endometriosis,and economically su-perior to GnRHa,but inferior to COC.
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Reducing low-value care is a key measure to optimize service model for cancer patients and improve the quality of and value of care.We summarizes the conceptual connotation of low-value medical services for cancer patients,compares the relevant measurement indicators at home and abroad,explains the logic of low-value medical services use for cancer patients at the three levels of"individual-institution-system",and proposes strategies for reducing low-value medical services for cancer patients in five aspects:construction of service value framework,dissemination of evaluation results,connection of drug access mechanisms,moni-toring of cancer care services,and empowering shared decision-making.
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Health technology assessment(HTA)is an important tool to inform health decision-making.Although highly related to ethical issues in the context of HTA,equity has attracted much attention from the academia,a consensus has not yet been reached on how to define and evaluate equity in China and abroad.It introduces the concept of equity,pointes out the necessity to realize health equity and the reflection of equity in healthcare sector,and further elaborates four ways to consider equity,and described the official practice of equity in HTA at home and abroad.It proposes several suggestions for China's HTA:considering equity in HTA and the discussion of equity should depend on specific decision-making scenarios;clarifying what health measurement perspective should be adopted before measuring health equity;paying attention to the value judgment of equity adopted by various stakeholders;conducting basic researches on the general population's preference for health measurement perspectives and value judgments of equity in China in a gesture to improve the evaluation system of equity in HTA.
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The pricing and reimbursement of orphan drugs are related to the accessibility of patients,and are of great significance to the prevention and guarantee of rare diseases.European countries have formed special standards and paths for health technology evaluation,and established special payment funds and diversified risk-sharing agreements,which have effectively improved the accessibility of orphan drugs.Based on this,it selected typical European countries to compare the orphan drug pricing and reimbursement methods.Then,it put forward some suggestions"building orphan drug health technology evaluation accelerated program,exploring the health of orphan drug classification security mechanism,and attaining supply incentives and development incentives through orphan drug pricing and adjustment",to optimize the basis for the orphan drug market access mechanism to provide reference.
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The access evaluation of new medical technology is an important part of the preclinical application of medical technology and plays a vital role in ensuring the quality and safety of medical services.However,in the con-crete practice of access evaluation,there are still some problems such as imperfect access theoretical framework,imperfect evaluation index system.With the strategic support of health policies,laws,and regulations,the theory and method of HB-HTA are used for reference,core elements such as assessment subject,assessment object,and assessment content are comprehensively considered,the index system is designed from the dimensions of tech-nical characteristics,safety,effectiveness,economy and applicability,and the access evaluation framework of im-ported medical new technologies is constructed.To offer a theoretical framework and evidence-based basis for medi-cal facility medical technology access management.
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ObjectiveThis study aims to understand the recognition of practitioners in traditional Chinese medicine （TCM） hospitals on hospital-based health technology assessment （HB-HTA）， assessment needs， challenges， and suggestions， so as to provide references for the future work. MethodThe convenient sampling method was adopted to survey the relevant practitioners in TCM hospitals. The questionnaire included 39 questions in 4 dimensions and was distributed through the online platform Weijuanxing. ResultA total of 244 questionnaires were recovered， and the obtained data were analyzed in SPSS. The results showed that 137 practitioners were very familiar with HB-HTA and there was no significant difference in the recognition of practitioners in different occupations （F=0.251； P=0.778）. The practitioners in Hong Kong， Macao， and Taiwan had lower recognition than those in other regions. In terms of the assessment needs， 127 practitioners believed that it was very necessary to carry out HB-HTA in TCM hospitals in the future. Chinese patent medicines/Chinese herbal medicine decoction pieces （5.91） and TCM appropriate technology （5.57） had higher assessment priority scores. The assessment needs were high for the effectiveness （235 practitioners） and safety （224 practitioners） of health technology. The lack of specialized organization and standardized evaluation process system and the shortage of talents were considered to be the major challenges for the future development in this field. ConclusionThe stakeholders carrying out the health technology assessment in TCM hospitals had certain awareness of HB-HTA. Most practitioners believed that it was necessary to carry out HB-HTA in TCM hospitals in the future， while the work might face challenges such as the lack of organizations and system and the shortage of talents， which requires policy support.
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A Avaliação de Tecnologias em Saúde (ATS) considera os domínios de benefícios clínicos, perfil epidemiológico, inovação, custo-efetividade, ética e de equidade no processo de decisão dos gestores em saúde. No contexto dos medicamentos para doenças raras, é desafiador o trabalho da ATS, dada a baixa disponibilidade de evidências robustas e o alto custo unitário das tecnologias. O objetivo da revisão foi analisar as estratégias disponíveis de avaliação das demandas de incorporação de medicamentos para o tratamento de doenças raras em sistemas de saúde. Foi realizada uma revisão rápida com busca estruturada na base de dados MEDLINE (via PubMed), Cochrane Library e Health Systems Evidence. Incluíram-se estudos sobre estratégias de avaliação de medicamentos utilizados para tratamento de doenças raras. Adicionalmente, foram realizadas buscas nas Agências de ATS do Brasil, Austrália, Nova Zelândia, Canadá, Reino Unido, França, Estados Unidos e Alemanha. A síntese dos resultados foi qualitativa com o agrupamento dos achados nos seguintes eixos temáticos: Segurança e efetividade, Custo-efetividade, Impacto orçamentário e Perspectiva da sociedade. Foram identificadas 267 publicações, sendo selecionadas 16 das bases de dados indexadas e 7 da literatura cinzenta. Com a análise dos documentos, pode-se concluir que a adoção de critérios específicos harmonizada com o atual modelo de ATS é um possível caminho a ser seguido no contexto dos medicamentos para doenças raras. Concomitante a isso, abordagens no sentido de incentivo a pesquisa e produção de dados de mundo real e a criação de comitês específicos para tratativa do tema nas agências de ATS apresentam-se como alternativa para lidar com as fragilidades no contexto de doenças raras.
The Health Technology Assessment (HTA) considers evidence regarding clinical benefits, epidemiological profile, innovation, cost-effectiveness, ethics and equity in its assessment process to support managers' decisions. In the context of drugs in rare diseases, the work of the ATS is challenging given the low availability of evidence and the high cost of technologies. The objective of the review was to analyze the available strategies for evaluating the demands for incorporating drugs for the treatment of rare diseases in health systems. A rapid review was performed with a structured search in the MEDLINE database (via PubMed), the Cochrane Library and Health Systems Evidence. Studies on strategies for evaluating drugs used to treat rare diseases were included and, additionally, searches were carried out in ATS Agencies in Brazil, Australia, New Zealand, Canada, United Kingdom, France, United States and Germany. The synthesis of the results was qualitative, grouping the major ones into thematic axes: Safety and effectiveness, Cost-effectiveness, Budgetary impact and Society's perspective. 267 publications were identified, 16 selected from indexed databases and 7 from gray literature. With the analysis of the documents, it can be concluded that the adoption of specific criteria harmonized with the current ATS model is a possible path to be followed in the context of drugs for rare diseases. At the same time, approaches to encourage research and the creation of specific committees to deal with the issue in HTA agencies would complement actions towards the consolidation of this work.
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Produção de Droga sem Interesse Comercial , Avaliação da Tecnologia Biomédica , Doenças RarasRESUMO
Introdução: O aumento da judicialização da saúde tem destacado a importância dos centros de avaliação judicial em decisões baseadas em evidências. A atrofia muscular espinhal (AME) é uma doença neurodegenerativa caracterizada principalmente por hipotonia muscular progressiva e alta mortalidade nos primeiros dois anos de vida. Embora o medicamento Zolgensma® (onasemnogeno abeparvoveque) seja indicado para o tratamento da AME, seu custo elevado e a indisponibilidade pelo sistema público de saúde brasileiro tornam o tratamento inviável, resultando em processos judiciais. Objetivo: Descrever o perfil das informações contidas nas notas técnicas (NT) disponíveis no Sistema Nacional de Pareceres e Notas Técnicas (e-NatJus), utilizadas como apoio nas decisões judiciais relacionadas ao Zolgensma®. Material e Métodos: Foi realizado um estudo descritivo com dados de notas técnicas disponíveis no e-NatJus no período entre setembro de 2021 a setembro de 2023. Resultados: Foram identificadas 63 notas técnicas, sendo que uma foi excluída por se tratar de nota complementar. Das 62 NTs consideradas elegíveis para análise neste estudo, a idade média dos participantes foi de 2,03 (± 1,54) anos, sendo a maioria do sexo masculino. A maioria das solicitações foi para tratamento de AME tipo 1 (N= 52; 82%). Destas, somente 23 atendiam a faixa etária inferior a 2 anos. Quanto à recomendação final, 14 NTs foram favoráveis ao fornecimento do Zolgensma®. Conclusão: Os resultados revelaram o perfil das NTs baseadas em evidências científicas, que subsidiam decisões judiciais visando minimizar impactos no orçamento da saúde. Iniciativas como o Sistema e-NatJus são fundamentais para acesso a esses subsídios, embora a disseminação de informações e capacitações continuadas sejam necessárias. Pesquisas adicionais sobre o impacto dos Núcleos de Avaliação de Tecnologias em Saúde (NATS) e Núcleos de Apoio Técnico ao Judiciário (NATJUS) na judicialização da saúde são importantes.
Introduction: The increasing judicialization of health has highlighted the importance of judicial evaluation centers in evidence-based decisions. Spinal Muscular Atrophy (SMA) is a neurodegenerative disease characterized mainly by progressive muscle hypotonia and high mortality in the first two years of life. Although the medication Zolgensma® (onasemnogene abeparvovec) is indicated for the treatment of SMA, its high cost and unavailability through the Brazilian public health system make the treatment unfeasible, resulting in legal proceedings. Objective: To describe the profile of the information contained in the Technical Notes (TN) available in the e-NatJus System used as support in Zolgensma® judicial decisions. Material and Methods: A descriptive study was conducted with data from technical notes available in the National System of Opinions and Technical Notes (e-NatJus) from September 2021 to September 2023. Results: 63 Technical Notes were identified, with one excluded as a Complementary Note. Of the 62 TNs considered eligible for analysis in this study, the mean age of the participants was 2.03 (± 1.54) years, with the majority being male. Most requests were for the treatment of SMA type 1 (N= 52; 82%). Of these, only 23 met the age criteria of less than 2 years. Regarding the final recommendation, 14 TNs were favorable to the provision of Zolgensma®. Conclusion: The results revealed the profile of technical notes based on scientific evidence, which support judicial decisions aimed at minimizing impacts on healthcare budgets. Initiatives like the e-NatJus System are essential for accessing these subsidies, although the dissemination of information and continuous training are necessary. Further research on the impact of NATS and NATJUS on healthcare judicialization is important.
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Preparações Farmacêuticas , Judicialização da Saúde , Avaliação da Tecnologia Biomédica , Atrofia Muscular Espinal , Prática Clínica Baseada em EvidênciasRESUMO
Introdução: A Empresa Brasileira de Serviços Hospitalares (Ebserh) gere 41 hospitais universitários federais (HUFs), atualmente com 36 Núcleos de Avaliação de Tecnologias em Saúde (NATS). No contexto hospitalar, o NATS desenvolve produtos de avaliação de tecnologias em saúde (ATS) para subsidiar o gestor na tomada de decisões, sendo essencial para a sustentabilidade do Sistema Único de Saúde. A implantação efetiva dos NATS ainda é desafiadora, devido à insuficiência de diretrizes específicas para ATS hospitalar. Objetivo: Relatar atuação do Grupo de Trabalho em ATS (GT-ATS) da Ebserh na elaboração de guia para orientar a estruturação e fortalecimento dos NATS dos HUFs.Relato de Experiência: O GT-ATS foi instituído por portaria, composto por representantes de NATS dos HUFs e da administração central da Ebserh. Inicialmente, fez-se levantamento dos processos envolvendo os NATS, da prática de ATS na Ebserh e foi realizado diagnóstico situacional dos NATS. Em seguida, elaborou-se o guia a partir da expertise dos membros do GT-ATS e das melhores práticas e recomendações sobre ATS hospitalar, identificadas em revisão da literatura. O "Guia para organização e funcionamento dos NATS na Rede Ebserh" foi publicado em março de 2023 no portal eletrônico da Ebserh e inclui contextualização da ATS hospitalar no Brasil, objetivos e escopo de atuação dos NATS da Ebserh, orientações para organização e funcionamento dos núcleos e modelo de regulamento interno. Também foram elaborados modelos de nota técnica, declaração de conflito de interesses, formulário de solicitação e termo de compromisso de confidencialidade. Conclusão: O GT-ATS, por meio do Guia e dos documentos elaborados, contribuiu para institucionalização, harmonização e implementação dos processos de ATS nos hospitais da Ebserh, respeitando a heterogeneidade dos contextos e níveis de maturidade dos NATS. O trabalho colaborativo dentro do GT-ATS permitiu articulação de saberes interdisciplinares e práticas interinstitucionais, ampliando a dimensão, alcance e efetividade das ações de ATS.
Introduction: The Brazilian Hospital Services Company (Ebserh) manages 41 federal university hospitals (HUFs), currently with 36 Health Technology Assessment Centers (NATS). In the hospital context, NATS develop health technology assessment (HTA) products to help managers make decisions and are essential for the sustainability of the Unified Health System. The effective implementation of NATS is still a challenge, due to the lack of specific guidelines for hospital HTA. Objective: To report on the work of Ebserh's HTA Working Group (GT-ATS) in drawing up a guide for structuring and strengthening NATS HUFs. Experience Report: The GT-ATS was set up by ordinance, made up of representatives from NATS of HUFs and from Ebserh's central administration. Initially, a survey about the processes involving the NATS and the practice of HTA at Ebserh was carried out to make a situational diagnosis. The guide was then drawn up based on the expertise of the GT-ATS members and the best practices and recommendations on hospital HTA identified in literature review. The "Guide for the organization and operation of NATS in the Ebserh network" was published in March 2023 on Ebserh's electronic portal and includes a contextualization of hospital HTA in Brazil, the objectives and scope of action of Ebserh's NATS, guidelines for the organization and operation of the centers and a model of internal regulations. Templates have also been drawn up for technical note, declaration of conflict of interests, application form and confidentiality agreement. Conclusion: Through the Guide and the documents produced, the GT-ATS contributed to the institutionalization, harmonization and implementation of HTA processes in Ebserh hospitals, while respecting the heterogeneous contexts and maturity levels of the NATS. The collaborative work within the GT-ATS has allowed interdisciplinary knowledge and inter-institutional practices to be brought together, expanding the dimension, scope and effectiveness of HTA actions.
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Avaliação da Tecnologia Biomédica , Sistema Único de Saúde , Redes de Informação de Ciência e Tecnologia , Promoção da Saúde , Hospitais Universitários/organização & administração , InstitucionalizaçãoRESUMO
RESUMO: Objetivo: descrever o desenvolvimento de um protótipo de software baseado na Caderneta de Saúde da Pessoa Idosa, utilizando a Avaliação Heurística para análise de sua usabilidade. Método: pesquisa aplicada de desenvolvimento tecnológico, utilizando a Avaliação Heurística de Nielsen como forma de realizar a análise de usabilidade, iniciada em novembro de 2021 em Juiz de Fora e São João Del Rei. Resultados: o protótipo inicial conta com 5 telas, que contêm alguns dados relevantes para o cuidado em saúde da pessoa idosa, como idade, comorbidades e histórico de alergias. Os idosos conseguirão fazer a edição dos dados pessoais, mas somente os profissionais conseguirão incluir os dados de saúde dos pacientes, com o objetivo de torná-los mais fidedignos. Conclusão: a utilização deste aplicativo contribuirá para atualizar e avançar com o uso de tecnologias voltadas para o cuidado em saúde, e que trará benefícios para os sistemas de saúde e para os usuários.
ABSTRACT Objective: to describe the development of a software prototype based on the Health Booklet for the Elderly, using Heuristic Evaluation to analyze its usability. Method: applied technological development research, using Nielsen's Heuristic Evaluation as a way of carrying out usability analysis, which began in November 2021 in Juiz de Fora and São João Del Rei. Results: the initial prototype has 5 screens, which contain some relevant data for the health care of the elderly, such as age, comorbidities, and history of allergies. The elderly will be able to edit their personal data, but only professionals will be able to include the patient's health data, with the aim of making it more reliable. Conclusion: The use of this application will help to update and advance the use of technologies aimed at health care and will bring benefits to health systems and users.
RESUMEN Objetivo: describir el desarrollo de un prototipo de software basado en la Cartilla de Salud de la Persona Mayor, utilizando la Evaluación Heurística para analizar su usabilidad. Método: investigación aplicada de desarrollo tecnológico, utilizando la Evaluación Heurística de Nielsen como medio para realizar el análisis de usabilidad, iniciada en noviembre de 2021 en Juiz de Fora y São João Del Rei. Resultados: el prototipo inicial cuenta con 5 pantallas, que contienen algunos datos relevantes para el cuidado de la salud de las personas mayores, como la edad, las comorbilidades y el historial de alergias. Los ancianos podrán editar sus datos personales, pero sólo los profesionales podrán incluir los datos de salud del paciente, con el objetivo de hacerlo más fiable. Conclusión: El uso de esta aplicación contribuirá a actualizar y avanzar en el uso de las tecnologías destinadas a la atención sanitaria, y aportará beneficios a los sistemas de salud y a los usuarios.
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Resumo O trabalho analisou o efeito das Consultas Públicas (CP) e suas contribuições nas recomendações da Comissão Nacional de Incorporação de Tecnologias (CONITEC). Trata-se de estudo descritivo e retrospectivo, com abordagem qualiquantitativa, com fonte de dados secundárias de acesso público, entre 2012 e 2017. Elaborou-se banco de dados para caracterizar as CP de medicamentos e suas contribuições, o que permitiu identificar casos de reversões entre a recomendação preliminar e final da CONITEC. Analisou-se as contribuições nos casos de reversão para caracterização de eixos argumentativos e tipo de embasamento. Das 307 demandas de incorporação, 205 destes passaram por CP, com 23.894 contribuições. A reversão das recomendações ocorreu em 9% das CP abertas (15 medicamentos), todas no sentido da não incorporação para incorporação. Principais eixos argumentativos trataram de benefícios clínicos e menores eventos adversos, prevalecendo o envio de experiências clínicas e opiniões. Evidencia-se avanços nos processos de incorporação de tecnologias no SUS pela realização da CP e ficou claro o desafio que os tomadores de decisão enfrentam nos espaços institucionais para o aprimoramento da participação social no sentido de fortalecer o benefício público.
Abstract The work analyzed the effect of Public Consultations (PC) and their contributions to the recommendations of the National Commission for the Incorporation of Technologies (CONITEC). This is a descriptive and retrospective study with a qualitative-quantitative approach using a secondary data source of public access, between 2012 and 2017. A database was developed to characterize the PC of medications and their contributions, which allowed the identification of cases of reversals between the preliminary and final recommendation of CONITEC. We analyzed the contributions in cases of reversal for characterization of argumentative axes and type of basis. Of the 307 demands for incorporation of medications, 205 went through PC, with 23,894 contributions. The reversal of the recommendations occurred in 9% of the open PC (15 medications), all in the sense of non-incorporation for incorporation. Main argumentative axes dealt with clinical benefits and minor adverse events, with prevalence of the submission of clinical experiences and opinions. Advances in the processes of incorporation of technologies in the SUS by performing PC were found and the challenge that decision makers face in institutional spaces for the improvement of social participation to strengthen the public benefit was clear.
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Abstract Screening newborns for genetic and other diseases is one of the most effective ways to improve health and reduce disease in a population. In developed countries, newborn screening has been a cornerstone of public health for decades. In many developing countries, however, newborn screening is still in its infancy. Many countries still lack screening programs. When a program is available, it generally lacks well-defined criteria on which decision-makers can justify the choice of diseases screened for and the methods used. One of the reasons put forward to understand this observation is the fact that little consideration is given by decision-makers to economic evaluations as a pillar of decision-making, as is the case in industrialized countries. This article provides a brief description of the challenges of using economic evaluation of newborn screening in developing countries. This will be illustrated by the example of the national newborn screening program in Vietnam.
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O termo "desinvestimento" se refere ao processo de retirada de recursos de intervenções que oferecem pouco ou nenhum ganho em saúde frente a seu custo. O intuito deste processo é reforçar práticas comprovadamente seguras, efetivas ou mais custo-efetivas, otimizando os resultados em saúde e a sustentabilidade econômica dos sistemas de saúde. O objetivo do trabalho foi caracterizar o processo de desinvestimento de medicamentos conduzido pela Comissão Nacional de Incorporação de Tecnologias (CONITEC) no Sistema Único de Saúde (SUS) entre 2012 e 2022, de forma exploratória através de análise documental dos seus relatórios técnicos de recomendações. Foram coletados nome do medicamento; sua classificação pelo sistema ATC; indicação clínica; demandante; realização de Consulta Pública; modalidade de desinvestimento recomendada e justificativa para o desinvestimento. Também foi avaliado o alinhamento das diretrizes de tratamento com as decisões de desinvestimento e o status de registro sanitário das tecnologias desinvestidas em diferentes ocasiões. Foram avaliados 30 relatórios de recomendação, correspondentes a 90 medicamentos. Três relatórios tiveram como recomendação a manutenção de sete tecnologias de perfil diversificado no SUS. Outros três relatórios eram referentes a tecnologias que foram incorporadas sob a modalidade ad experimentum e que, portanto, foram reavaliadas após três anos no SUS. Quanto às tecnologias efetivamente desinvestidas (80), elas se dividiram principalmente pelos grupos L (agentes antineoplásicos e imunomoduladores; 29,3%), J (anti-infecciosos de uso sistêmico; 21,3%) e A (aparelho digestivo e metabolismo; 20%). As principais indicações clínicas dos medicamentos desinvestidos foram: artrite reumatoide; HIV; hepatite C; e doença de Crohn. Justificativas mais mencionadas foram a indisponibilidade de registro ativo do medicamento no país (24,1%), seguida por problemas relacionados à segurança (20,6%) e efetividade (19,9%). Todas as demandas tiveram origem interna do Ministério da Saúde. Em 31,3%, houve exclusão do medicamento para indicação específica e, em 30%, exclusão total do sistema de saúde; em 27,5%, optou-se por excluir apenas determinada apresentação farmacêutica; em 10% as exclusões foram de apresentação para indicação específica; e em 1,2% ocorreu restrição de uso. Consulta Pública foi realizada em 36% dos casos. Após a publicação da Diretriz de Avaliação de Desempenho de Tecnologias em Saúde no final de 2016, o perfil de medicamentos desinvestidos por categoria ATC e por indicações clínicas adquiriram maior diversidade; as justificativas para o desinvestimento, que antes focavam em questões relacionadas a efetividade e segurança, passaram a se concentrar na indisponibilidade do medicamento no mercado; as modalidades de desinvestimento se acumularam mais em exclusões do SUS e exclusões de apresentação, e a Secretaria de Ciência, Tecnologia, Inovação e Insumos Estratégicos se tornou a principal demandante; submissão a consultas públicas subiu de 11,9% para 86,8%. O máximo de adequação estrutural identificado nos relatórios em relação aos tópicos preconizados pela Diretriz foi de 46,2%. Embora as iniciativas de desinvestimento tenham avançado nos últimos anos, o tema ainda enfrenta dificuldades para estabelecer uma agenda sólida no país.
Disinvestment refers to withdrawing resources from interventions that offer little or no health gain compared to their cost, seeking to reinforce practices proven to be safe, effective or more cost-effective and to optimize health outcomes and the economic sustainability of health systems. This study aimed to characterize the drug divestment process conducted by the National Commission for Incorporation of Technologies (CONITEC) in the Brazilian Unified Health System (SUS) between 2012 and 2022, in an exploratory way through their the technical recommendations reports. Drug name and ATC classification, clinical indication, proponents, occurrence of Public Consultation, recommended divestment modality and justifications for disinvestment were evaluated. We also evaluated the agreement of treatment guidelines with disinvestment decisions and the sanitary registration status of technologies disinvested at different times. We evaluated 30 recommendations reports corresponding to 90 drugs. Three reports recommended the maintenance of seven technologies in SUS. Another three reports referred to technologies that were incorporated under the ad experimentum modality and then were reassessed after three years in SUS. As for the technologies effectively disinvested (80), the drugs mainly belonged to the ATC classes L (29.3%), J (21.3%) and A (20%). The main clinical indications of the disinvested drugs were: rheumatoid arthritis, HIV, hepatitis C, and Crohn's disease. The main justifications were absence of market approval for the drug in Brazil (24.1%) and problems related to safety (20.6%) and effectiveness (19.9%). All requests were from the Brazilian Ministry of Health. Public Consultation was carried out in 36% of the situations. There were recommendations to exclude the drug for a specific indication in 31.3% of the cases and total exclusion from the SUS in 30%; exclusion of a particular pharmaceutical presentation and exclusion of presentation for a specific indication occurred in 27.5% and 10%, respectively. After the publication of the Methodological Guideline for Performance Avaliation of Health Technologies ate the end of 2016, the profile of drugs disinvested by ATC category and by clinical indications acquired bigger diversity; the justifications for disinvestment, which previously focused on issues related to effectiveness and safety, passed to focus on the unavailability of the drug on the market; disinvestment modalities concentrated more on SUS exclusions and presentation exclusions; the Secretaria of Science, Technology, Innovation and Strategic Insums became the main proponent of disinvestment demands; submission to public consultations grow up from 11.9% to 86.8%. The maximum structural adequacy identified of the reports in relation to the topics recommended by the Guideline was 46,2%. The lack of standardization and overly simplified reporting formats stood out. Although divestment initiatives have advanced in recent years, this theme still needs to improve in establishing a solid agenda in Brazil.
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Avaliação da Tecnologia Biomédica , Tomada de Decisões Gerenciais , Sistema Único de Saúde , Custos de Medicamentos , Gestão em Saúde , BrasilRESUMO
OBJECTIVE To evaluate the efficacy， safety and economical efficiency of Xuesaitong injection in the treatment of stroke by rapid health technology assessment，so as to provide evidence for clinical rational drug use. METHODS Retrieved from Wanfang database， CBM， CNKI，PubMed，Cochrane Library，Embase， INAHTA and HTAI databases or organization websites， health technology assessment （HTA） reports， meta-analysis/systematic reviews and pharmacoeconomic studies related to Xuesaitong injection in the treatment of stroke were summarized and analyzed. RESULTS A total of 29 pieces of literature were included. Among them， 14 studies were conducted on meta-analysis/systematic reviews，15 studies were conducted on pharmacoeconomics， HTA was not obtained. The results of meta-analysis/systematic reviews showed that Xuesaitong injection had certain advantages for stroke in improving the total effective rate， clinical symptoms and related scale scores compared with blank control group and some drug control groups. Safety studies had shown that the adverse reactions of Xuesaitong injection were mainly allergic-like reactions. The results of pharmacoeconomic evaluation are quite different， which may also be related to the long time span among various studies and the adjustment of some drug prices. CONCLUSIONS Xuesaitong injection in the treatment of stroke is helpful to improve the clinical efficacy and evaluation indexes， but there are some serious adverse reactions， and it is not economically superior to some chemical drugs.
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Cancer is one of the major fatal diseases that seriously threaten human health， and its burden needs to be solved urgently. Health technology assessment （HTA） can provide scientific evidence-based basis for cancer diagnosis， treatment， prevention and related policy formulation. Cost-utility analysis is the gold standard for economic evaluation in HTA， and the accurate measurement of its health utility is one of the key elements to determine the accuracy of its results. This article focuses on systematic introduction of direct measures， multi-attribute health utility scales， and mapping methods in the field of cancer measurement and reviews their applications in cancer patients. Among them， direct measures are complex， costly， and require a high level of subject knowledge； multi-attribute health utility measures are currently the preferred method for measuring health utility in cancer patients； with the continuous development and refinement of disease-specific utility measures in multi-attribute health utility instruments， the mapping method may gradually decrease in future applications. This paper can provide a reference for the selection of health utility measurement tools for HTA in the field of cancer， and provide evidence-based basis for optimizing resource allocation and policy formulation in the field of cancer.
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The articles involving Xiangju Capsules were retrieved, and qualitative research and quantitative research methods were combined to evaluate the evidence of the safety, effectiveness, economy, innovation, suitability, accessibility, and characteristics of traditional Chinese medicine( "6+1" dimensions) of this drug. Multi-criteria decision analysis(MCDA) model and CSC v2.0 software were used to comprehensively evaluate the clinical value of Xiangju Capsules in the treatment of rhinosinusitis and clarify the precise clinical positioning. The dimensions are graded A, B, C, or D. Multi-source safety evidence showed that the main adverse reactions were gastrointestinal reactions, rash, itching, dizziness, and headache. Based on the available studies, the risk is controllable and the safety is grade A. Meta-analysis showed that Xiangju Capsules + conventional western medicine could recover the Lund-Kennedy score, Lund-Mackay score, and CT score, relieve headache, nasal congestion, olfactory disturbance, and facial pain, with the effectiveness is grade B. The incremental cost-effectiveness ratio of Xiangju Capsules + conventional western medicine compared with conventional western medicine alone in the treatment of chronic rhinosinusitis was 263.71 yuan, about 0.82% of the per capita disposable income. The results of sensitivity analysis showed that the research results were relatively robust. Based on the assumption that the per capita disposable income in 2020 will be the threshold of patients' willingness to pay, it is more economical to use Xiangju Capsules + conventional western medicine. The drug belongs to grade A of the national medical insurance, with an average daily cost of 3.06 yuan, and the economy is grade B. This formula is modified from classic formulas and characteristic empirical formulas, be capable of improving immunity and preventing repeated attacks. It can be used for acute and chronic rhinitis-rhinosinusitis. It had a wide range of applicability, especially for the patients with head and face tenderness. Service innovation was reflected in the measures to guarantee supply, capacity, scalability, and coverage of grass-roots sales channels. The industrial innovation was improved through the management of medicinal resources, pharmaceutical industry, production technology, quality control, scientific research and development, and this formula won three national invention patents. Comprehensively, the innovation of Xiangju Capsules is grade B. According to the survey of 188 medical practitioners and 196 patients in 20 provinces, municipalities, and autonomous regions of China, the drug was characterized by easy preparation and administration, individualized medication, simple technology and management, convenient use, storage, and transport, and controllable adverse reactions, with the suitability is grade B. Xiangju Capsules showed the cost of 45.9 and 275.4 yuan for treatment of acute and chronic rhinitis-rhinosinusitis, respectively, being well affordable. It was sold in 35 000 medical institutions in China. The dosage form was suitable for transportation, storage, and grass-root application. With rich, sustainable, and available medicinal resources, the accessibility of Xiangju Capsules is grade A. This drug can be used for both acute and chronic rhinitis-rhinosinusitis, clearing heat and expelling pus, and strengthening the exterior to prevent relapse. After this drug was available on the market, over 4 000 cases were studied, with rich experience in human use accumulated, and characteristics of traditional Chinese medicine is grade B. Overall, the clinical value of Xiangju Capsules is class B. It is suggested that Xiangju Capsules should be used in accordance with the relevant policies of basic clinical drug administration to play its role.