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Comparing the cost-per-QALYs gained and cost-per-DALYs averted literatures.

ABSTRACT: Background: We examined the similarities and differences between studies using two common metrics used in cost-effectiveness analyses (CEAs): cost per quality-adjusted life year (QALY) gained and cost per disability-adjusted life year (DALY) averted. Methods: We used the Tufts Medical Center CEA Registry, which contains English-language cost-per-QALY gained studies, and the Global Cost-Effectiveness Analysis (GHCEA) Registry, which contains cost-per-DALY averted studies. We examined study characteristics, including intervention type, sponsor, country, and primary disease, and also compared the number of published CEAs to disease burden for major diseases and conditions across geographic regions. Results: We identified 6,438 cost-per-QALY and 543 cost-per-DALY studies published through 2016 and observed rapid growth for both literatures. Cost-per-QALY studies most often examined pharmaceuticals and interventions in high-income countries. Cost-per-DALY studies predominantly focused on infectious disease interventions and interventions in low and lower-middle income countries. We found that while diseases imposing a larger burden tend to receive more attention in the cost-effectiveness analysis literature, the number of publications for some diseases and conditions deviates from this pattern, suggesting "under-studied" conditions (e.g., neonatal disorders) and "over-studied" conditions (e.g., HIV and TB). Conclusions: The CEA literature has grown rapidly, with applications to diverse interventions and diseases. The publication of fewer studies than expected for some diseases given their imposed burden suggests funding opportunities for future cost-effectiveness research.

SUBMITTER: Neumann PJ

PROVIDER: S-EPMC5801595 | biostudies-literature |

REPOSITORIES: biostudies-literature

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Project description:IntroductionCalculating the cost per disability-adjusted life years (DALYs) averted associated with interventions is an increasing popular means of assessing the cost-effectiveness of strategies to improve population health. However, there has been no systematic attempt to characterize the literature and its evolution.MethodsWe conducted a systematic review of cost-effectiveness studies reporting cost-per-DALY averted from 2000 through 2015. We developed the Global Health Cost-Effectiveness Analysis (GHCEA) Registry, a repository of English-language cost-per-DALY averted studies indexed in PubMed. To identify candidate studies, we searched PubMed for articles with titles or abstracts containing the phrases "disability-adjusted" or "DALY". Two reviewers with training in health economics independently reviewed each article selected in our abstract review, gathering information using a standardized data collection form. We summarized descriptive characteristics on study methodology: e.g., intervention type, country of study, study funder, study perspective, along with methodological and reporting practices over two time periods: 2000-2009 and 2010-2015. We analyzed the types of costs included in analyses, the study quality on a scale from 1 (low) to 7 (high), and examined the correlation between diseases researched and the burden of disease in different world regions.ResultsWe identified 479 cost-per-DALY averted studies published from 2000 through 2015. Studies from Sub-Saharan Africa comprised the largest portion of published studies. The disease areas most commonly studied were communicable, maternal, neonatal, and nutritional disorders (67%), followed by non-communicable diseases (28%). A high proportion of studies evaluated primary prevention strategies (59%). Pharmaceutical interventions were commonly assessed (32%) followed by immunizations (28%). Adherence to good practices for conducting and reporting cost-effectiveness analysis varied considerably. Studies mainly included formal healthcare sector costs. A large number of the studies in Sub-Saharan Africa addressed high-burden conditions such as HIV/AIDS, tuberculosis, neglected tropical diseases and malaria, and diarrhea, lower respiratory infections, meningitis, and other common infectious diseases.ConclusionThe Global Health Cost-Effectiveness Analysis Registry reveals a growing and diverse field of cost-per-DALY averted studies. However, study methods and reporting practices have varied substantially.

Project description:For unresectable or metastatic melanoma, first-line ipilimumab has demonstrated long-term survival benefits over a 7-year period. First-line treatment with BRAF inhibitors has demonstrated efficacy in clinical trials with up to 3 years of follow-up. The long-term comparative efficacy and costs of ipilimumab and BRAF inhibitors are unknown.Patient-level data from 12 clinical studies for ipilimumab were used. Survival data were extracted from included clinical trials for BRAF inhibitors based on a systematic literature review. Different parametric survival models, including exponential, Gompertz, log-normal, and Weibull models, were used to fit reported overall survival (OS) data and to project long-term survival for BRAF inhibitors. Survival benefits were measured in terms of total life-months gained as calculated by the area under the curve of OS Kaplan-Meier curves for the observed ipilimumab data and projected BRAF inhibitor data. Total life-months gained and cumulative costs per life-month gained were compared between ipilimumab and BRAF inhibitors.The systematic literature review identified six randomized-controlled trials of BRAF inhibitors for subsequent analyses. With 7-year follow-up, ipilimumab was associated with a total of 28.5 life-months gained. Based on the Weibull model, the extrapolated total life-months gained for BRAF inhibitors were 26.5 months for dabrafenib, 21.3 months for trametinib, 14.3 months for vemurafenib, and 24.6 months for dabrafenib + trametinib. In sensitivity analyses, extrapolated total life-months gained varied across the three other models, ranging from 13.7 to 36.8 months across therapies. Cumulative costs per life-month gained with ipilimumab decreased steadily over time, while the costs remained constant for BRAF inhibitors due to continuous dosing. By year 3, cumulative costs per life-month gained were the lowest with ipilimumab; by year 7, the costs were $4281 for ipilimumab, compared with $8920 for dabrafenib, $10,211 for trametinib, $11,002 for vemurafenib, and $19,132 for the dabrafenib + trametinib combination therapy.Ipilimumab was associated with a better long-term cost-per-life month compared to BRAF agents. Long-term extrapolation of survival with BRAF agents was uncertain, and showed no evidence of prolonged survival compared to ipilimumab.

Project description:IntroductionPatients who require prolonged mechanical ventilation (PMV) are increasing and producing financial burdens worldwide. This study determines the cost per QALY (quality-adjusted life year), out-of-pocket expenses, and lifetime costs for PMV patients stratified by underlying diseases and cognition levels.MethodsA nationwide sample of 50,481 patients with continual mechanical ventilation for more than 21 days was collected during 1997-2007. After stratifying the patients according to specific diagnoses, a latent class analysis (LCA) was performed to categorise PMV patients with multiple co-morbidities into several homogeneous groups. The survival functions were estimated for individual groups using the Kaplan-Meier method and extrapolated to 300 months through a semi-parametric method. The survival functions were adjusted using an EQ-5D utility value derived from a convenience sample of 142 PMV patients to estimate quality-adjusted life expectancies (QALE). Another convenience sample of 165 patients was used to estimate the out-of-pocket expenses. The lifetime expenditures paid by the single-payer National Health Insurance (NHI) system and patients' families were estimated by multiplying average monthly expenditures by the survival probabilities and summing the values over lifetime.ResultsPMV therapy costs more than 100,000 U.S. dollars (USD) per QALY for all patients with poor cognition. For patients with partial cognition, PMV therapy costs less than 56,000 USD per QALY for those with liver cirrhosis, intracranial or spinal cord injuries, and 57,000-69,000 USD for patients with multiple co-morbidities under age of 65. The average lifetime cost of PMV was usually below 56,000 USD. The out-of-pocket expenses were often more than one-third of the total cost of treatment.ConclusionsPMV treatment for patients with poor cognition would cost more than 5 times Taiwan's GDP (gross domestic products), or less cost-effective. The out-of-pocket expenses for PMV provision should also be considered in policy decision.

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Comparing the cost-per-QALYs gained and cost-per-DALYs averted literatures.

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