Cost-effectiveness and willing-to-pay thresholds for vertebral augmentation of osteoporotic vertebral fractures, what are they based on: a systematic review.

OBJECTIVE: Although there is substantial clinical evidence on the safety and effectiveness of vertebral augmentation for osteoporotic vertebral fractures, cost-effectiveness is less well known. The objective of this study is to provide a systematic review of cost-effectiveness studies and policy-based willingness-to-pay (WTP) thresholds for different vertebral augmentation (VA) procedures, vertebroplasty (VP) and balloon kyphoplasty (BK), for osteoporotic vertebral fractures (OVFs). DESIGN: A systematic review targeting cost-effectiveness studies of VA procedures for OVFs. DATA SOURCES: Six bibliographic databases were searched from inception up to May 2021. ELIGIBILITY CRITERIA FOR STUDY SELECTION: Studies were eligible if meeting all predefined criteria: (1) VP or BK intervention, (2) OVFs and (3) cost-effectiveness study. Articles not written in English, abstracts, editorials, reviews and those reporting only cost data were excluded. DATA EXTRACTION AND SYNTHESIS: Information was extracted on study characteristics, cost-effective estimates, summary decisions and payer WTP thresholds. Incremental cost-effective ratio (ICER) was the main outcome measure. Studies were summarised by a structured narrative synthesis organised by comparisons with conservative management (CM). Two independent reviewers assessed the quality (risk of bias) of the systematic review and cost-effectiveness studies by peer-reviewed checklists. RESULTS: We identified 520 references through database searching and 501 were excluded as ineligible by titles and abstract. Ten reports were identified as eligible from 19 full-text reviews. ICER for VP versus CM evaluated as cost per quality-adjusted life-year (QALY) ranged from €22 685 (*US$33 395) in Netherlands to £-2240 (*US$-3273), a cost-saving in the UK. ICERs for BK versus CM ranged from £2706 (*US$3954) in UK to kr600 000 (*US$90 910) in Sweden. ICERs were within payer WTP thresholds for a QALY based on historical benchmarks. CONCLUSIONS: Both VP and BK were judged cost-effective alternatives to CM for OVFs in economic studies and were within WTP thresholds in multiple healthcare settings.

Cost-effectiveness studies of vertebral augmentation for osteoporotic vertebral fractures: a systematic review.

BACKGROUND CONTEXT: Osteoporotic vertebral fractures (OVF) dramatically increase with age and are serious life altering adverse events for seniors resulting in increased rates of institutionalization, morbidity and mortality. Given the expanding population of the elderly and increasing prevalence of OVFs, cost-effective treatment strategies need to be considered. Percutaneous vertebral augmentation (VA) procedures such as vertebroplasty (VP) or balloon kyphoplasty (BK) are increasingly employed to treat painful vertebral fractures not responding to conservative management (CM) of bedrest and analgesia. Both VA procedures have been shown to be effective treatments for OVFs in multiple systematic reviews of randomized controlled trials. In this systematic review, analytical strategies, designs and results were compared for health economic studies evaluating cost-effectiveness of VA procedures, VP or BK for OVFs. Furthermore, assessments of quality (risk of bias) were conducted for the systematic review and the individual studies with peer-reviewed checklists recommended for cost-effectiveness studies. PURPOSE: To provide an up-to-date systematic review of peer-reviewed studies evaluating cost-effectiveness of VA procedures, VP or KP for OVFs to support treatment and health care funding decisions. STUDY DESIGN: This study is a systematic literature review and structured narrative synthesis. STUDY SAMPLE: Peer reviewed health economic studies reporting cost-effectiveness for VA procedures, VP or BK for OVFs OUTCOME MEASURES: The following information extracted from the studies included: report country and year, study design, comparators, population, perspective, health valuations, costing sources and cost-effectiveness measures. For economic studies involving modeling, information was also extracted for model type, time horizon, key model drivers, and handling of uncertainty. Incremental cost-effectiveness ratio (ICER), the ratio of differences between comparator treatment groups in costs and health benefits, was considered the main cost-effectiveness measure. METHODS: A systematic review was conducted of MEDLINE, EMBASE, CINAHL, ECONLIT, Cochrane Library and DARE databases up to the review date May 2021. Studies were reviewed for those reporting cost-effectiveness analyses on VA procedures including VP or BK for OVFs. Studies including only costs, abstracts, editorials, methodologies and reviews were not included. The selection of articles was reported in line with PRISMA guidance. A descriptive framework was developed to classify types of cost-effectiveness studies based on methodological differences and a structured narrative synthesis was used to summarize studies. Quality assessments were made with British Medical Journal checklist for individual cost-effectiveness studies and the CiCERO checklist for systematic reviews of cost-effectiveness studies. RESULTS: In this systematic review, 520 references were identified through database searching and 501 were excluded as ineligible by titles and abstract based on prior eligibility criteria. From full-text reviews of 19 reports, ten were identified as eligible for the systematic review evaluating cost-effectiveness of VA procedures for OVFs. All references were published between 2008 and 2020. The ten cost-effectiveness studies, three for VP, three for BK and four for both VP and BK, all involved CM for OVFs as a treatment comparator. The studies involved different methods of economic analysis, modeling assumptions, cost and health valuations conducted in different health care setting over different time periods. A framework for the review outlines key features of cost-effectiveness study designs consisting of unmatched, matched, or randomized controls involving cost-effectiveness or cost-utility) analyses. Both VP and BK were cost-effective alternatives to CM for OVFs with earlier health gains and significantly shorter hospital stays. Cost-effectiveness estimates, ICERs, remained relatively stable and within willingness-to-pay thresholds under a range of sensitivity analyses. Comparisons between VP and BK were variable depending on modeling assumptions, but generally the procedures had similar health benefit gains with VP having lower acute procedural costs. CONCLUSIONS: Both VP and BK, have been shown to be cost-effective alternatives to CM for osteoporotic vertebral fractures in diverse cost-effectiveness studies conducted in multiple health care settings. Trial-based cost-utility contributed the strongest evidence supporting cost-effectiveness determination for VP and BK for OVFs.

PUBLIC AND PATIENT INVOLVEMENT IN HEALTH TECHNOLOGY ASSESSMENT: A FRAMEWORK FOR ACTION.

OBJECTIVE: As health technology assessment (HTA) organizations in Canada and around the world seek to involve the public and patients in their activities, frameworks to guide decisions about whom to involve, through which mechanisms, and at what stages of the HTA process have been lacking. The aim of this study was to describe the development and outputs of a comprehensive framework for involving the public and patients in a government agency's HTA process. METHODS: The framework was informed by a synthesis of international practice and published literature, a dialogue with local, national and international stakeholders, and the deliberations of a government agency's public engagement subcommittee in Ontario, Canada. RESULTS: The practice and literature synthesis failed to identify a single, optimal approach to involving the public and patients in HTA. Choice of methods should be considered in the context of each HTA stage, goals for incorporating societal and/or patient perspectives into the process, and relevant societal and/or patient values at stake. The resulting framework is structured around four actionable elements: (i) guiding principles and goals for public and patient involvement (PPI) in HTA, (ii) the establishment of a common language to support PPI efforts, (iii) a flexible array of PPI approaches, and (iv) on-going evaluation of PPI to inform adjustments over time. CONCLUSIONS: A public and patient involvement framework has been developed for implementation in a government agency's HTA process. Core elements of this framework may apply to other organizations responsible for HTA and health system quality improvement.