Cost-effectiveness of precision medicine: a scoping review.
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ABSTRACT:
SUBMITTER: Kasztura M
PROVIDER: S-EPMC6867980 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:IntroductionPrecision medicine (PM) involves gene testing to identify disease risk, enable early diagnosis or guide therapeutic choice, and targeted gene therapy. We aim to perform a systematic review and meta-analysis to quantify the cost-effectiveness profile of PM stratified by intervention type, identify sources of heterogeneity in the value-for-money of PM.Methods and analysisWe will perform a systematic search in Embase, MEDLINE, EconLit and CRD databases for studies published in English language or with translation in English between 1 January 2011 and 8 July 2021 on the topic of cost-effectiveness analysis of PM interventions. The focus will be on studies that reported health and economic outcomes. Study quality will be assessed using the Biases in Economic Studies checklist. The incremental net benefit of PM screening, diagnostic, treatment-targeting and therapeutic interventions over conventional strategies will be respectively pooled across studies using a random-effect model if heterogeneity is present, otherwise a fixed-effect model. Subgroup analyses will be performed based on disease area, WHO region and World Bank country-income level. Additionally, we will identify the potential sources of heterogeneity with random-effect meta-regressions. Finally, biases will be detected using jackknife sensitivity analysis, funnel plot assessment and Egger's tests.Ethics and disseminationFor this type of study ethics approval or formal consent is not required. The results will be disseminated at various presentations and feedback sessions, in conference abstracts and manuscripts that will be submitted to peer-reviewed journals.Prospero registration numberCRD42021272956.
| S-EPMC8984003 | biostudies-literature
Project description:Precision diagnostic testing (PDT) employs appropriate biomarkers to identify cancer patients that may optimally respond to precision medicine (PM) approaches, such as treatments with targeted agents and immuno-oncology drugs. To date, there are no published systematic appraisals evaluating the cost-effectiveness of PDT in non-small-cell lung cancer (NSCLC). To address this gap, we conducted Preferred Reporting Items for Systematic Reviews and Meta-Analyses searches for the years 2009-2019. Consolidated Health Economic Evaluation Reporting Standards were employed to screen, assess and extract data. Employing base costs, life years gained or quality-adjusted life years, as well as willingness-to-pay (WTP) threshold for each country, net monetary benefit was calculated to determine cost-effectiveness of each intervention. Thirty-seven studies (50%) were included for analysis; a further 37 (50%) were excluded, having failed population-, intervention-, comparator-, outcomes- and study-design criteria. Within the 37 studies included, we defined 64 scenarios. Eleven scenarios compared PDT-guided PM with non-guided therapy [epidermal growth factor receptor (EGFR), n = 5; programmed death-ligand 1 (PD-L1), n = 6]. Twenty-eight scenarios compared PDT-guided PM with chemotherapy alone (anaplastic lymphoma kinase, n = 3; EGFR, n = 17; PD-L1, n = 8). Twenty-five scenarios compared PDT-guided PM with chemotherapy alone, while varying the PDT approach. Thirty-four scenarios (53%) were cost-effective, 28 (44%) were not cost-effective, and two were marginal, dependent on their country's WTP threshold. When PDT-guided therapy was compared with a therapy-for-all patients approach, all scenarios (100%) proved cost-effective. Seven of 37 studies had been structured appropriately to assess PDT-PM cost-effectiveness. Within these seven studies, all evaluated scenarios were cost-effective. However, 81% of studies had been poorly designed. Our systematic analysis implies that more robust health economic evaluation could help identify additional approaches towards PDT cost-effectiveness, underpinning value-based care and enhanced outcomes for patients with NSCLC.
| S-EPMC8486593 | biostudies-literature
Project description:Over the past 20 years, with the incorporation of genetic sequencing and improved understanding regarding the mechanisms of cancer growth/metastasis, novel targets and their associated treatments have emerged in oncology and are now regularly incorporated into the clinical care of patients in the US. Novel, more tumor-specific, non-chemotherapy agents, including agents that are commonly used in the treatment of patients with gastric adenocarcinoma (GA) and gastrointestinal stromal tumor (GIST), fall under a broader treatment strategy, termed "precision medicine". While diagnostic testing and associated treatments in metastatic GA (mGA) are costly and may produce marginal benefit, those associated with GIST, despite being costly, produce significant improvements in patient outcomes. Despite the significant difference in impact, the agents associated with these cancers have similar acquisition costs. In this paper, we will review the current literature regarding cost and cost-effectiveness associated with precision medicine diagnosis and treatment strategies for GA and GIST.
| S-EPMC5506269 | biostudies-other
Project description:Precision medicine (PM), specifically genetic-based testing, is currently used in over 140,000 individual tests to inform the clinical management of disease. Though several databases (e.g., the NIH Genetic Testing Registry) demonstrate the availability of these sequencing-based tests, we do not currently understand the extent to which these tests are used. There exists a need to synthesize the body of real-world data (RWD) describing the use of sequencing-based tests to inform their appropriate use. To accomplish this, we performed a scoping review to examine what RWD sources have been used in studies of PM utilization between January 2015 and August 2021 to characterize the use of genome sequencing (GS), exome sequencing (ES), tumor sequencing (TS), next-generation sequencing-based panels (NGS), gene expression profiling (GEP), and pharmacogenomics (PGx) panels. We abstracted variables describing the use of these types of tests and performed a descriptive statistical analysis. We identified 440 articles in our search and included 72 articles in our study. Publications based on registry databases were the most common, followed by studies based on private insurer administrative claims. Slightly more than one-third (38%) used integrated datasets. Two thirds (67%) of the studies focused on the use of tests for oncological clinical applications. We summarize the RWD sources used in peer-reviewed literature on the use of PM. Our findings will help improve future study design by encouraging the use of centralized databases and registries to track the implementation and use of PM.
| S-EPMC9025578 | biostudies-literature
Project description:The biological fingerprint of environmental adversity may be key to understanding health and disease, as it encompasses the damage induced as well as the compensatory reactions of the organism. Metabolic and hormonal changes may be an informative but incomplete window into the underlying biology. We endeavored to identify objective blood gene expression biomarkers for psychological stress, a subjective sensation with biological roots. To quantify the stress perception at a particular moment in time, we used a simple visual analogue scale for life stress in psychiatric patients, a high risk group. Then, using a stepwise discovery, prioritization, validation, and testing in independent cohorts design, we were successful in identifying gene expression biomarkers that were predictive of high stress states, and of future psychiatric hospitalizations related to stress, more so when personalized by gender and diagnosis.
2019-03-15 | GSE125216 | GEO
Project description:Effective use of genetic and genomic data in cancer prevention and treatment depends on adequate communication with patients and the public. Although relevant empirical work has emerged, the scope and outcomes of this communication research have not been characterized. We conducted a comprehensive scoping review of recent published research (2010-2017) on communication of cancer-related genetic and genomic testing (CGT) information. Searches in six databases revealed 9243 unique records; 513 papers were included. Most papers utilized an observational quantitative design; fewer utilized an experimental design. More attention has been paid to outcomes of CGT results disclosure than to decision making regarding CGT uptake or the process of results disclosure. Psychosocial outcomes were most common across studies. This literature has a strong focus on BRCA1/2, with few papers focused on Lynch syndrome or next-generation technologies. Women, Caucasians, older adults, and those of higher socioeconomic status were overrepresented. Research gaps identified include the need for studies on the process of CGT communication; examining behavioral, decision making, and communication outcomes; and inclusion of diverse populations. Addressing these gaps can help improve the use of genomics in cancer control and reduce disparities in access to and use of CGT.
| S-EPMC7725534 | biostudies-literature
Project description:Activated B-cell-like (ABC) diffuse large B-cell lymphoma (DLBCL) is associated with worse survival after standard rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (RCHOP) chemoimmunotherapy compared to germinal center B-cell-like (GCB) subtype. Preliminary evidence suggests that benefits from novel agents may vary by subtype. Hypothesizing that treatment stratified by DLBCL subtype could be potentially cost-effective, we developed micro-simulation models to compare three first-line treatment strategies: (1) standard RCHOP for all patients (2) subtype testing followed by RCHOP for GCB and novel treatment for ABC DLBCL, and (3) novel treatment for all patients. Based on phase 2 evidence, we used lenalidomide?+?RCHOP as a surrogate novel treatment. The subtype-based approach showed a favorable incremental cost-effectiveness ratio of $15,015/quality-adjusted life year compared with RCHOP. Although our exploratory analyses demonstrated a wide range of conditions where subtype-based treatment remained cost-effective, data from phase 3 trials are needed to validate our models' findings and draw definitive conclusions.
| S-EPMC5918224 | biostudies-literature
Project description:BackgroundCost-effectiveness analysis of artificial intelligence (AI) in medicine demands consideration of clinical, technical, and economic aspects to generate impactful research of a novel and highly versatile technology.ObjectiveWe aimed to systematically scope existing literature on the cost-effectiveness of AI and to extract and summarize clinical, technical, and economic dimensions required for a comprehensive assessment.MethodsA scoping literature review was conducted to map medical, technical, and economic aspects considered in studies on the cost-effectiveness of medical AI. Based on these, a framework for health policy analysis was developed.ResultsAmong 4820 eligible studies, 13 met the inclusion criteria for our review. Internal medicine and emergency medicine were the clinical disciplines most frequently analyzed. Most of the studies included were from the United States (5/13, 39%), assessed solutions requiring market access (9/13, 69%), and proposed optimization of direct resources as the most frequent value proposition (7/13, 53%). On the other hand, technical aspects were not uniformly disclosed in the studies we analyzed. A minority of articles explicitly stated the payment mechanism assumed (5/13, 38%), while it remained unspecified in the majority (8/13, 62%) of studies.ConclusionsCurrent studies on the cost-effectiveness of AI do not allow to determine if the investigated AI solutions are clinically, technically, and economically viable. Further research and improved reporting on these dimensions seem relevant to recommend and assess potential use cases for this technology.
| S-EPMC9419048 | biostudies-literature
Project description:BackgroundCost-effectiveness analyses (CEAs) on prevention of non-communicable diseases (NCDs) are necessary to guide decision makers to allocate scarce healthcare resource, especially in Southeast Asia (SEA), where many low- and middle-income countries (LMICs) are in the process of scaling-up preventive interventions. This scoping review aims to summarize the cost-effectiveness evidence of primary, secondary, or tertiary prevention of type 2 diabetes mellitus (T2DM) and cardiovascular diseases (CVDs) as well as of major NCDs risk factors in SEA.MethodsA scoping review was done following the PRISMA checklist for Scoping Reviews. Systematic searches were performed on Cochrane Library, EconLit, PubMed, and Web of Science to identify CEAs which focused on primary, secondary, or tertiary prevention of T2DM, CVDs and major NCDs risk factors with the focus on primary health-care facilities and clinics and conducted in SEA LMICs. Risks of bias of included studies was assessed using the Consensus of Health Economic Criteria list.ResultsThis study included 42 CEAs. The interventions ranged from screening and targeting specific groups for T2DM and CVDs to smoking cessation programs, discouragement of smoking or unhealthy diet through taxation, or health education. Most CEAs were model-based and compared to a do-nothing scenario. In CEAs related to tobacco use prevention, the cost-effectiveness of tax increase was confirmed in all related CEAs. Unhealthy diet prevention, mass media campaigns, salt-reduction strategies, and tax increases on sugar-sweetened beverages were shown to be cost-effective in several settings. CVD prevention and treatment of hypertension were found to be the most cost-effective interventions. Regarding T2DM prevention, all assessed screening strategies were cost-effective or even cost-saving, and a few strategies to prevent T2DM complications were found to be cost-effective in certain settings.ConclusionThis review shows that the cost-effectiveness of preventive strategies in SEA against T2DM, CVDs, and their major NCDs risk factors are heterogenous in both methodology as well as outcome. This review combined with the WHO "best buys" could guide LMICs in SEA in possible interventions to be considered for implementation and upscaling. However, updated and country-specific information is needed to further assess the prioritization of the different healthcare interventions.Systematic review registrationhttps://osf.io, identifier: 10.17605/OSF.IO/NPEHT.
| S-EPMC10666286 | biostudies-literature
Project description:BackgroundEvidence supporting physical activity for older adults is strongly positive. Implementation and scale-up of these interventions need to consider the value for money. This scoping review aimed to assess the volume of (i) systematic review evidence regarding economic evaluations of physical activity interventions, and (ii) of cost utility analysis (CUA) studies (trial- or model-based) of physical activity interventions for older people.MethodsWe searched five databases (January 2010 to February 2022) for systematic reviews of economic evaluations, and two databases (1976 to February 2022) for CUA studies of physical activity interventions for any population of people aged 60+ years.ResultsWe found 12 potential reviews, two of which were eligible for inclusion. The remaining 10 reviews included eligible individual studies that were included in this review. All individual studies from the 12 reviews (n = 37) investigated the cost-effectiveness of structured exercise and most showed the intervention was more costly but more effective than no intervention. We identified 27 CUA studies: two investigated a physical activity promotion program and the remainder investigated structured exercise. Most interventions (86%) were more costly but more effective, and the remaining were cost-saving compared to no intervention.ConclusionsThere is a scarcity of reviews investigating the value for money of physical activity interventions for older adults. Most studies investigated structured exercise. Physical activity interventions were generally more effective than no intervention but more costly. As such an intervention could be cost-effective and therefore worthy of wider implementation, but there is a need for more frequent economic evaluation in this field.
| S-EPMC10024893 | biostudies-literature