Methodological Quality Assessment of Budget Impact Analyses for Orphan Drugs: A Systematic Review.
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ABSTRACT:
SUBMITTER: Abdallah K
PROVIDER: S-EPMC8098807 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:BackgroundBudget impact analyses (BIAs) are used for reimbursement decisions and drug access medical insurance, as a supplement to cost-effectiveness analyses (CEAs).ObjectivesWe systematically reviewed BIAs for antitumor drugs of lung cancer to provide reference for high-value drug budget impact analyses and decision making.MethodsWe conducted a literature search on PubMed, EMbase, The Cochrane Library, China National Knowledge Infrastructure and Wanfang Data Knowledge Service Platform from 2010 to 2019. The methodological indicators and result information of the budget impact analyses were extracted and evaluated for quality.ResultsA total of 14 studies on the budget impact for antitumor drugs of lung cancer were included, and the overall quality was good. Half of studies were from developed countries. Nine of the studies were designed using the BIA cost calculation model, and two were simulated using the Markov model Monte Carlo model. From all studies, only 14.3% reported model validation. The budget impact results of the same drug in different countries were inconsistent.ConclusionsIncluded studies evaluating budget impact analyses for anti-tumor drugs of lung cancer showed variability in the methodological framework for BIAs. The budget impact analyses of high-value drugs need to be more stringent to ensure the accuracy of the parameters, and should provide reliable results based on real data to decision-making departments, which should carefully consider access to lung cancer drugs.
| S-EPMC7706257 | biostudies-literature
Project description:Background: Budget impact analysis (BIA) is an economic assessment that estimates the financial consequences of adopting a new intervention. BIA is used to make informed reimbursement decisions, as a supplement to cost-effectiveness analyses (CEAs). Objectives: We systematically reviewed BIA studies associated with anti-diabetic drugs and assessed the extent to which international BIA guidelines were followed in these studies. Methods: We conducted a literature search on PubMed, Web of Science, Econlit, Medline, China National Knowledge Infrastructure (CNKI), Wanfang Data knowledge Service platform from database inception to June 30, 2021. ISPOR good practice guidelines were used as a methodological standard for assessing BIAs. We extracted and compared the study characteristics outlined by the ISPOR BIA Task Force to evaluate the guideline compliance of the included BIA. Results: A total of eighteen studies on the BIA for anti-diabetic drugs were identified. More than half studies were from developed countries. Seventeen studies were based on model and one study was based on real-world data. Overall, analysis considered a payer perspective, reported potential budget impacts over 1-5 years. Assumptions were mainly made about target population size, market share uptake of new interventions, and scope of cost. The data used for analysis varied among studies and was rarely justified. Model validation and sensitivity analysis were lacking in the current BIA studies. Rebate analysis was conducted in a few studies to explore the price discount that was required for new interventions to demonstrate cost equivalence to comparators. Conclusion: Existing studies evaluating budget impact for anti-diabetic drugs vary greatly in methodology, some of which showed low compliance to good practice guidelines. In order for the BIA to be useful for assisting in health plan decision-making, it is important for future studies to optimize compliance to national or ISPOR good practice guidelines on BIA. Model validation and sensitivity analysis should also be improved in future BIA studies. Continued improvement of BIA using real-world data is necessary to ensure high-quality analyses and to provide reliable results.
| S-EPMC8639520 | biostudies-literature
Project description:Background: With the increasing disease burden of cancer worldwide, more and more anticancer drugs have been approved in many countries, and the results of budget impact analyses (BIAs) have become important evidence for related reimbursement decisions. Objectives: We systematically reviewed whether BIAs for anticancer drugs consider the scope of costs rationally and compared the results of different cost scopes to provide suggestions for future analyses and decision-making. Methods: Eligible BIAs published in PubMed, Embase, Web of Science, and the Cochrane Library from 2016 to 2021 were identified based on Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. We extracted 15 terms from the included studies and analyzed how they considered the scope of costs. In addition, a budget impact model was developed for the introduction of geptanolimab to China's National Reimbursement Drug List to enable a comparison of two cost-scope scenarios. Results: A total of 29 studies were included in the systematic review. All 29 studies considered the costs of anticancer drugs, and 25 (86%) also considered condition-related costs, but only 11 (38%) considered subsequent treatment costs. In the comparative study, the predicted budget impacts from 2022 to 2024 were significantly impacted by subsequent treatment costs, with annual differences between the two cost-scope scenarios of $39,546,664, $65,866,161, and $86,577,386, respectively. Conclusions: The scope of costs considered in some existing BIAs for anticancer drugs are not rational. The variations between different cost scopes in terms of budget impact were significant. Thus, BIAs for anticancer drugs should consider a rational scope of costs that adheres to BIA guidelines. Researchers and decision-makers should pay more attention to the scope of costs to achieve better-quality BIAs for anticancer drugs and enhance reimbursement decision-making.
| S-EPMC8602071 | biostudies-literature
Project description:BackgroundGeneric drugs are used by millions of patients for economic reasons, so their evaluation must be highly transparent.ObjectiveTo assess the quality of reporting of bioequivalence trials comparing generic to brand-name drugs.Methodology/principal findingsPubMed was searched for reports of bioequivalence trials comparing generic to brand-name drugs between January 2005 and December 2008. Articles were included if the aim of the study was to assess the bioequivalency of generic and brand-name drugs. We excluded case studies, pharmaco-economic evaluations, and validation dosage assays of drugs. We evaluated whether important information about funding, methodology, location of trials, and participants were reported. We also assessed whether the criteria required by the Food and Drug Administration (FDA) and the European Medicine Agency (EMA) to conclude bioequivalence were reported and that the conclusions were in agreement with the results. We identified 134 potentially relevant articles but eliminated 55 because the brand-name or generic drug status of the reference drug was unknown. Thus, we evaluated 79 articles. The funding source and location of the trial were reported in 41% and 56% of articles, respectively. The type of statistical analysis was reported in 94% of articles, but the methods to generate the randomization sequence and to conceal allocation were reported in only 15% and 5%, respectively. In total, 65 articles of single-dose trials (89%) concluded bioequivalence. Of these, 20 (31%) did not report the 3 criteria within the limits required by the FDA and 11 (17%) did not report the 2 criteria within the limits required by the EMA.Conclusions/significanceImportant information to judge the validity and relevance of results are frequently missing in published reports of trials assessing generic drugs. The quality of reporting of such trials is in need of improvement.
| S-EPMC3157430 | biostudies-literature
Project description:BackgroundProbiotics are widely used for the induction and maintenance of remission in inflammatory bowel disease (IBD) and pouchitis. There are a large number of meta-analyses (MAs)/ systematic reviews (SRs) on this subject, the methodological quality of which has not been evaluated.ObjectivesThis study aimed to evaluate the methodological quality of and summarize the evidence obtained from MAs/SRs of probiotic treatments for IBD and pouchitis patients.MethodsThe PubMed, EMBASE, Cochrane Library and China National Knowledge Infrastructure (CNKI) databases were searched to identify Chinese and English language MAs/SRs of the use of probiotics for IBD and pouchitis. The Assessment of Multiple Systematic Reviews (AMSTAR) scale was used to assess the methodological quality of the studies.ResultsA total of 36 MAs/SRs were evaluated. The AMSTAR scores of the included studies ranged from 1 to 10, and the average score was 5.81. According to the Canadian Agency for Drugs and Technologies in Health, 4 articles were classified as high quality, 24 articles were classified as moderate quality, and 8 articles were classified as low quality. Most of the MAs/SRs suggested that probiotics had potential benefits for patients with ulcerative colitis (UC), but failed to show effectiveness in the induction and maintenance of remission in Crohn's disease (CD). The probiotic preparation VSL#3 may play a beneficial role in pouchitis.ConclusionThe overall methodological quality of the current MAs/SRs in the field of probiotics for IBD and pouchitis was found to be low to moderate. More MAs/SRs of high quality are required to support using probiotics to treat IBD and pouchitis.
| S-EPMC5179087 | biostudies-literature
Project description:BackgroundSystematic reviews (SRs) are considered the highest level of evidence to answer research questions; however, they are time and resource intensive.ObjectiveWhen comparing SR tasks done manually, using standard methods, versus those same SR tasks done using automated tools, (1) what is the difference in time to complete the SR task and (2) what is the impact on the error rate of the SR task?MethodsA case study compared specific tasks done during the conduct of an SR on prebiotic, probiotic, and synbiotic supplementation in chronic kidney disease. Two participants (manual team) conducted the SR using current methods, comprising a total of 16 tasks. Another two participants (automation team) conducted the tasks where a systematic review automation (SRA) tool was available, comprising of a total of six tasks. The time taken and error rate of the six tasks that were completed by both teams were compared.ResultsThe approximate time for the manual team to produce a draft of the background, methods, and results sections of the SR was 126 hours. For the six tasks in which times were compared, the manual team spent 2493 minutes (42 hours) on the tasks, compared to 708 minutes (12 hours) spent by the automation team. The manual team had a higher error rate in two of the six tasks-regarding Task 5: Run the systematic search, the manual team made eight errors versus three errors made by the automation team; regarding Task 12: Assess the risk of bias, 25 assessments differed from a reference standard for the manual team compared to 20 differences for the automation team. The manual team had a lower error rate in one of the six tasks-regarding Task 6: Deduplicate search results, the manual team removed one unique study and missed zero duplicates versus the automation team who removed two unique studies and missed seven duplicates. Error rates were similar for the two remaining compared tasks-regarding Task 7: Screen the titles and abstracts and Task 9: Screen the full text, zero relevant studies were excluded by both teams. One task could not be compared between groups-Task 8: Find the full text.ConclusionsFor the majority of SR tasks where an SRA tool was used, the time required to complete that task was reduced for novice researchers while methodological quality was maintained.
| S-EPMC8204237 | biostudies-literature
Project description:ObjectiveTo examine how the results of network meta-analyses are reported.DesignMethodological systematic review of published reports of network meta-analyses.Data sourcesCochrane Database of Systematic Reviews, Database of Abstracts of Reviews of Effects, Medline, and Embase, searched from inception to 12 July 2012.Study selectionAll network meta-analyses comparing the clinical efficacy of three or more interventions in randomised controlled trials were included, excluding meta-analyses with an open loop network of three interventions.Data extraction and synthesisThe reporting of the network and results was assessed. A composite outcome included the description of the network (number of interventions, direct comparisons, and randomised controlled trials and patients for each comparison) and the reporting of effect sizes derived from direct evidence, indirect evidence, and the network meta-analysis.Results121 network meta-analyses (55 published in general journals; 48 funded by at least one private source) were included. The network and its geometry (network graph) were not reported in 100 (83%) articles. The effect sizes derived from direct evidence, indirect evidence, and the network meta-analysis were not reported in 48 (40%), 108 (89%), and 43 (36%) articles, respectively. In 52 reports that ranked interventions, 43 did not report the uncertainty in ranking. Overall, 119 (98%) reports of network meta-analyses did not give a description of the network or effect sizes from direct evidence, indirect evidence, and the network meta-analysis. This finding did not differ by journal type or funding source.ConclusionsThe results of network meta-analyses are heterogeneously reported. Development of reporting guidelines to assist authors in writing and readers in critically appraising reports of network meta-analyses is timely.
| S-EPMC3949412 | biostudies-literature
Project description:Objective To evaluate the impact of searching clinical trial registries in systematic reviews.Design Methodological systematic review and reanalyses of meta-analyses.Data sources Medline was searched to identify systematic reviews of randomised controlled trials (RCTs) assessing pharmaceutical treatments published between June 2014 and January 2015. For all systematic reviews that did not report a trial registry search but reported the information to perform it, the World Health Organization International Trials Registry Platform (WHO ICTRP search portal) was searched for completed or terminated RCTs not originally included in the systematic review.Data extraction For each systematic review, two researchers independently extracted the outcomes analysed, the number of patients included, and the treatment effect estimated. For each RCT identified, two researchers independently determined whether the results were available (ie, posted, published, or available on the sponsor website) and extracted the data. When additional data were retrieved, we reanalysed meta-analyses and calculated the weight of the additional RCTs and the change in summary statistics by comparison with the original meta-analysis.Results Among 223 selected systematic reviews, 116 (52%) did not report a search of trial registries; 21 of these did not report the information to perform the search (key words, search date). A search was performed for 95 systematic reviews; for 54 (57%), no additional RCTs were found and for 41 (43%) 122 additional RCTs were identified. The search allowed for increasing the number of patients by more than 10% in 19 systematic reviews, 20% in 10, 30% in seven, and 50% in four. Moreover, 63 RCTs had results available; the results for 45 could be included in a meta-analysis. 14 systematic reviews including 45 RCTs were reanalysed. The weight of the additional RCTs in the recalculated meta-analyses ranged from 0% to 58% and was greater than 10% in five of 14 systematic reviews, 20% in three, and 50% in one. The change in summary statistics ranged from 0% to 29% and was greater than 10% for five of 14 systematic reviews and greater than 20% for two. However, none of the changes to summary effect estimates led to a qualitative change in the interpretation of the results once the new trials were added.Conclusions Trial registries are an important source for identifying additional RCTs. The additional number of RCTs and patients included if a search were performed varied across systematic reviews.
| S-EPMC5421496 | biostudies-literature
Project description:IntroductionRare diseases (RDs) are often chronic and progressive life-threatening medical conditions that affect a low percentage of the population compared with other diseases. These conditions can be treated with medications known as orphan drugs (ODs). Unfortunately, there is no universal definition of RDs or ODs. This systematic review (SR) will identify the quantitative and qualitative criteria and the underlying rationale used internationally to define RDs and ODs.Methods and analysisThis protocol follows the conventions for the Preferred Reporting Items for Systematic Review and Meta-Analysis Protocols (2015 guidelines). A SR will be conducted, including a search of the following databases: PubMed, MEDLINE, EMBASE, Scopus, Web of Science, GreyLit and OpenGrey. Eligible publications will be selected based on predetermined inclusion criteria. Extracted data will be analysed using thematic and content analyses of qualitative descriptors, whereas quantitative data will be analysed descriptively and reported in the form of frequencies and percentages.Ethics and disseminationNo ethical approval is required since this SR focuses on the secondary analysis of data retrieved from the scientific literature. The outcomes of this SR will be published as part of a PhD thesis, presented at conferences, and published in peer-reviewed journals.Prospero registration numberCRD42021252701.
| S-EPMC9345065 | biostudies-literature
Project description:This systematic review assessed the methodological quality of behavioural weight loss intervention studies conducted among adults and associations between quality and statistically significant weight loss outcome, strength of intervention effectiveness and sample size. Searches for trials published between January, 2009 and December, 2014 were conducted using PUBMED, MEDLINE and PSYCINFO and identified ninety studies. Methodological quality indicators included study design, anthropometric measurement approach, sample size calculations, intent-to-treat (ITT) analysis, loss to follow-up rate, missing data strategy, sampling strategy, report of treatment receipt and report of intervention fidelity (mean = 6.3). Indicators most commonly utilized included randomized design (100%), objectively measured anthropometrics (96.7%), ITT analysis (86.7%) and reporting treatment adherence (76.7%). Most studies (62.2%) had a follow-up rate > 75% and reported a loss to follow-up analytic strategy or minimal missing data (69.9%). Describing intervention fidelity (34.4%) and sampling from a known population (41.1%) were least common. Methodological quality was not associated with reporting a statistically significant result, effect size or sample size. This review found the published literature of behavioural weight loss trials to be of high quality for specific indicators, including study design and measurement. Identified for improvement include utilization of more rigorous statistical approaches to loss to follow up and better fidelity reporting.
| S-EPMC5996374 | biostudies-literature