Methodological guidelines and publications of benefit-risk assessment for health technology assessment: a scoping review.

OBJECTIVES: To map the available methodological guidelines and documents for conducting and reporting benefit-risk assessment (BRA) during health technologies' life cycle; and to identify methodological guidelines for BRA that could serve as the basis for the development of a BRA guideline for the context of health technology assessment (HTA) in Brazil. DESIGN: Scoping review. METHODS: Searches were conducted in three main sources up to March 2023: (1) electronic databases; (2) grey literature (48 HTA and regulatory organisations) and (3) manual search and contacting experts. We included methodological guidelines or publications presenting methods for conducting or reporting BRA of any type of health technologies in any context of the technology's life cycle. Selection process and data charting were conducted by independent reviewers. We provided a structured narrative synthesis of the findings. RESULTS: From the 83 eligible documents, six were produced in the HTA context, 30 in the regulatory and 35 involved guidance for BRA throughout the technology's life cycle. We identified 129 methodological approaches for BRA in the documents. The most commonly referred to descriptive frameworks were the Problem, Objectives, Alternatives, Consequences, Trade-offs, Uncertainty, Risk and Linked decisions and the Benefit-Risk Action Team. Multicriteria decision analysis was the most commonly cited quantitative framework. We also identified the most cited metric indices, estimation and utility survey techniques that could be used for BRA. CONCLUSIONS: Methods for BRA in HTA are less established. The findings of this review, however, will support and inform the elaboration of the Brazilian methodological guideline on BRA for HTA. TRIAL REGISTRATION NUMBER: https://doi.org/10.17605/OSF.IO/69T3V.

Methodological guidelines and publications of benefit-risk assessment for health technology assessment: a scoping review protocol.

BACKGROUND: Benefit-risk assessment (BRA) is used in multiple phases along the health technology's life-cycle to evaluate the balance between the benefits and risks, as it is fundamental to all stakeholders. BRA and its methodological approaches have been applied primarily in the context of regulatory agencies. However, BRA's application and extent in the context of health technology assessment (HTA) bodies remain less clear. Our goal is to perform a scoping review to identify and map methodological guidelines and publications on methods of BRA. This will be done considering the different phases of the life-cycle of health technologies to underline both the depth and extent of research concerning BRA, especially in the context of HTA. METHODS AND ANALYSIS: This scoping review protocol was developed following the framework proposed by Arksey and O'Malley, and the updated guidelines by the Joanna Briggs Institute. We will include methodological publications that provide recommendations or guidelines on methods for BRA. We will conduct electronic searches on Medline (PubMed) and EMBASE (Ovid) databases; manual searches on the main websites of HTA bodies and drug regulatory organisations; and contact experts in the field. Systematic extraction forms will be used to screen and assess the identified publications by independent assessors. We will provide a qualitative synthesis using descriptive statistics and visual tools. Results will be summarised in systematic evidence tables and comparative evidence scoping charts. ETHICS AND DISSEMINATION: This review will use data publicly available and does not require ethics approval. The results of this scoping review will contribute to scientific knowledge and act as a basis for methodologists, guideline developers and researchers for the development of BRA to inform regulatory decisions, reimbursement and coverage decision making. The results will be disseminated through peer-reviewed articles, conferences, policy briefs and workshops. TRIAL REGISTRATION NUMBER: Open Science Framework (https://doi.org/10.17605/OSF.IO/69T3V).

Economic evaluation of wheelchairs interventions: a systematic review.

OBJECTIVE: The overall aim of this systematic review was to identify and synthesise the best available evidence on effectiveness, resource use and costs involved in wheelchair interventions of adults with mobility limitations. METHODOLOGY: This systematic review was undertaken in accordance with the Centre for Reviews and Dissemination Guidelines. The protocol for this systematic review was registered with PROSPERO International Prospective Register of Systematic reviews. The following PICOS eligibility criteria were considered: (P) Population was individuals with mobility limitations that live in their community (e.g., non-institutionalized), with aged 18 or older; (I) Intervention was mobility assistive technologies (MAT), such as manual and powered wheelchairs; (C) Comparators (Not Applied); (O) Outcome, the primary outcome of interest, was established as the cost-effectiveness of wheelchair interventions. Direct and indirect costs per unit of effect were expressed in terms of clinical outcome units, quality-adjusted life years gained, utility scores, quality of life measures and incremental cost-effectiveness ratios to inform the economic outcomes. (S) Study design was considered as a health economic evaluation (i.e., including cost-effectiveness analysis, cost-utility analysis and cost benefit analysis as well as partial economic evaluations). The Consolidated Health Economic Evaluation Reporting Standards - CHEERS, checklist was used for summarising and interpreting the results of economic evaluations. RESULTS: Sixteen studies were included, two were identified as full health economic evaluations and 14 were considered partial health economic evaluations. CONCLUSION: Only two full health economic analyses of wheelchair interventions have been conducted and both focussed on powered wheelchair provision. There are important gaps in current knowledge regarding wheelchair health economic methods and available outcome measures, which there is a great need for further research.Implication for RehabilitationSystematic reviews of health economic evaluation studies are useful for synthesising economic evidence about health interventions and provide insight in new research development.Organisations involved in the provision of wheelchairs should apply cost-effectiveness outcome measures to help raise the standard of provision, to support evidence-based practice, and to improve resource utilisation.

Multicriteria decision analysis in health care decision in oncology: a systematic review.

INTRODUCTION: Multicriteria decision analysis (MCDA) has been used to inform health decisions in health technology assessments (HTA) processes. This is particularly important to complex treatment decisions in oncology. AREAS COVERED: Five databases (PubMed, EMBASE, LILACS, Web of Science and CRD's NHS Economic Evaluation Database) were searched for studies comparing health technologies in oncology, involving the concept MCDA. The ISPOR MCDA Good Practices Guidelines were used to assess the reporting quality. Study selection, appraisal, and data extraction were performed by two reviewers. Fifteen studies were included. The main decision problem was related to health technology assessment of cancer treatments. Clinicians and public health experts were the most frequent stakeholders. The most frequently included criteria comprised therapeutic benefit, and socio-economic impact. Value measurement approach, direct rating techniques, and additive model for aggregation were used in most studies. Uncertainty analysis revealed the impact of posology and costs on the studies' results. All studies showed some level of overlapping decision criteria. EXPERT OPINION: There is considerable diversity of methods in MCDA for healthcare decision-making in oncology. The evidence presented can serve as a resource when considering which stakeholders, criteria, and techniques to include in future MCDA studies in oncology.

Challenges for the development of alternative low-cost ventilators during COVID-19 pandemic in Brazil. / Desafios para o desenvolvimento de ventiladores alternativos de baixo custo durante a pandemia de COVID-19 no Brasil.

The COVID-19 pandemic has brought concerns to managers, healthcare professionals, and the general population related to the potential mechanical ventilators' shortage for severely ill patients. In Brazil, there are several initiatives aimed at producing alternative ventilators to cover this gap. To assist the teams that work in these initiatives, we provide a discussion of some basic concepts on physiology and respiratory mechanics, commonly used mechanical ventilation terms, the differences between triggering and cycling, the basic ventilation modes and other relevant aspects, such as mechanisms of ventilator-induced lung injury, respiratory drive, airway heating and humidification, cross-contamination risks, and aerosol dissemination. After the prototype development phase, preclinical bench-tests and animal model trials are needed to determine the safety and performance of the ventilator, following the minimum technical requirements. Next, it is mandatory going through the regulatory procedures as required by the Brazilian Health Regulatory Agency (Agência Nacional de Vigilância Sanitária - ANVISA). The manufacturing company should be appropriately registered by ANVISA, which also must be notified about the conduction of clinical trials, following the research protocol approval by the Research Ethics Committee. The registration requisition of the ventilator with ANVISA should include a dossier containing the information described in this paper, which is not intended to cover all related matters but to provide guidance on the required procedures.

A pandemia por COVID-19 tem deixado os gestores, os profissionais de saúde e a população preocupados com a potencial escassez de ventiladores pulmonares para suporte de pacientes graves. No Brasil, há diversas iniciativas com o intuito de produzir ventiladores alternativos para ajudar a suprir essa demanda. Para auxiliar as equipes que atuam nessas iniciativas, são expostos alguns conceitos básicos sobre fisiologia e mecânica respiratória, os termos comumente utilizados no contexto da ventilação mecânica, as fases do ciclo ventilatório, as diferenças entre disparo e ciclagem, os modos ventilatórios básicos e outros aspectos relevantes, como mecanismos de lesão pulmonar induzida pela ventilação mecânica, pacientes com drive respiratório, necessidade de umidificação de vias aéreas, risco de contaminação cruzada e disseminação de aerossóis. Após a fase de desenvolvimento de protótipo, são necessários testes pré-clínicos de bancada e em modelos animais, a fim de determinar a segurança e o desempenho dos equipamentos, seguindo requisitos técnicos mínimos exigidos. Então, é imprescindível passar pelo processo regulatório exigido pela Agência Nacional de Vigilância Sanitária (ANVISA). A empresa responsável pela fabricação do equipamento deve estar regularizada junto à ANVISA, que também deve ser notificada da condução dos testes clínicos em humanos, seguindo protocolo de pesquisa aprovado pelo Comitê de Ética em Pesquisa. O registro do ventilador junto à ANVISA deve ser acompanhado de um dossiê, composto por documentos e informações detalhadas neste artigo, que não tem o propósito de esgotar o assunto, mas de nortear os procedimentos necessários.