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).

Impact of switching between reference biologics and biosimilars of tumour necrosis factor inhibitors for rheumatoid arthritis: a systematic review and network meta-analysis.

What is the impact of switching between biologics and biosimilars of adalimumab, etanercept, and infliximab on efficacy and safety for rheumatoid arthritis? A systematic review and network meta-analysis were performed to compare switching and non-switching groups of treatments. Pooled Risk Relative (RR) or standardised mean differences (SMD) with 95% credible intervals (95% CrIs) were obtained. Seventeen randomized trials with a switching phase involving 6,562 patients were included. Results showed that a single switch from biologics to biosimilars compared to continuing biologics had comparable effects for primary and co-primary outcomes, the American College of Rheumatology criteria with 20% response (ACR20) (7 trials, 1,926 patients, RR 0.98, 95% CrIs 0.93 to 1.03) and the Health Assessment Questionnaire-Disability Index (HAQ-DI) (5 trials, 1,609 patients, SMD - 0.07, 95% CrIs - 0.23 to 0.1), and within the equivalence margins: ACR20 [RR 0.94, 1.06] and HAQ-DI [SMD - 0.22, 0.22]. The risk of treatment-emergent adverse events, discontinuation, and positive anti-drug antibodies were comparable after switching. Safety results were imprecise, and the follow-up period might not be sufficient to evaluate long-term effects, especially malignancies. Overall, the practice of single switching between approved biologics and biosimilars of Tumour Necrosis Factor inhibitors is efficacious and safe for rheumatoid arthritis.