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Nearly all patients with type 1 diabetes and 20-30 percent of patients with type 2 diabetes use insulin to manage glycemic control. Approximately one-quarter of patients who use insulin report underuse because of cost. In response, more than twenty states have implemented monthly caps on insulin out-of-pocket spending, ranging from $25 to $100. Using a difference-in-differences approach, this study evaluated whether state-level caps on insulin out-of-pocket spending change insulin usage among commercially insured enrollees. The study included 33,134 people ages 18-64 who had type 1 diabetes or who used insulin to manage type 2 diabetes with commercial insurance coverage that was subject to state-level oversight and was included in the 25 percent sample of the IQVIA PharMetrics database during 2018-21. Insulin out-of-pocket caps did not significantly increase quarterly insulin claims for enrollees who had type 1 diabetes or who used insulin to manage type 2 diabetes. State-level caps on insulin out-of-pocket spending for commercial enrollees did not significantly increase insulin use; that may be in part because of out-of-pocket expenses being lower than cap amounts.
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Diabetes Mellitus Tipo 1 , Diabetes Mellitus Tipo 2 , Gastos em Saúde , Hipoglicemiantes , Insulina , Humanos , Insulina/uso terapêutico , Insulina/economia , Feminino , Pessoa de Meia-Idade , Adulto , Diabetes Mellitus Tipo 2/tratamento farmacológico , Diabetes Mellitus Tipo 2/economia , Diabetes Mellitus Tipo 1/tratamento farmacológico , Diabetes Mellitus Tipo 1/economia , Masculino , Estados Unidos , Gastos em Saúde/estatística & dados numéricos , Hipoglicemiantes/uso terapêutico , Hipoglicemiantes/economia , Adolescente , Revisão da Utilização de Seguros , Cobertura do Seguro/estatística & dados numéricos , Adulto Jovem , Seguro Saúde/economia , Seguro Saúde/estatística & dados numéricos , Custos de Medicamentos/estatística & dados numéricosRESUMO
In early 2024, the Centers for Medicare & Medicaid Services (CMS) will send initial price offers to the manufacturers of the first 10 drugs selected for the Medicare Drug Price Negotiation Program, established under the Inflation Reduction Act. However, CMS has not specified exactly how it will adjust the starting point for an initial price offer based on assessment of a drug's clinical benefit. This article addresses unanswered questions relating to CMS' methods for assessing clinical benefit. Specifically, we address how CMS can weigh various measures of evidence, ensure transparency and consistency, meaningfully incorporate patient and other stakeholder perspectives, and support addressing evidence gaps. We propose a 2-step approach for assessing the overall clinical benefit of a selected drug compared with its therapeutic alternatives that builds on the framework outlined by CMS. In step 1, CMS would evaluate conventional clinical benefit, defined in terms of outcomes commonly used in clinical studies for the selected drug and indications. In step 2, CMS would evaluate other outcomes broadly related to patient experience that are not adequately represented in the clinical literature. Overall, our approach incorporates the advantages of both qualitative and quantitative approaches to value assessment and decision-making. We describe a set of loose decision rules to improve transparency and consistency, recommend incorporating ranks and weights to signal to researchers and manufacturers which elements of clinical benefit and sources of data are the most important, and center meaningful deliberation with clinical experts, patients, and caregivers.
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Medicare , Negociação , Idoso , Estados Unidos , Humanos , Participação do Paciente , Projetos de PesquisaRESUMO
OBJECTIVES: Limitations in conventional cost-effectiveness methods have led to calls for incorporation of additional value elements in assessments of health technologies. However, gaps remain in how additional value elements may inform decision making. This study aimed to prioritize additional value elements from the perspective of healthy individuals without a specific condition or indicated for a specific treatment in the United States among a multistakeholder panel and compare the importance of perspective-specific value elements. METHODS: Additional value elements were prioritized in 2 phases: (1) we identified and categorized additional value elements in a targeted literature review, and (2) we convened a multistakeholder group-based preference elicitation study (N = 28) to evaluate the description of each value element and rank and generate normalized weights of each value element for its significance in value assessment. The importance of additional value elements was also weighted relative to patient-centric value elements. RESULTS: The rank and weight of contextual value elements among 28 stakeholders were "severity of the disease" (26.2%), "disadvantaged and vulnerable target populations highly represented" (21.8%), "broader economic impact" (17.3%), "risk protection" (13.8%), "rarity of the disease" (11.3%), and "novel mechanism of action" (9.7%). Relative weight of the additional value elements versus patient-centric value elements was 52% and 48%, respectively. CONCLUSIONS: Study findings may inform priority setting for value frameworks and emerging US government assessments. The group-based elicitation method is repeatable and useful for structured deliberative processes in value assessment and may help improve the consistency and predictability of what is important to stakeholders.
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Avaliação de Processos e Resultados em Cuidados de Saúde , Assistência Centrada no Paciente , Humanos , Estados Unidos , Participação dos InteressadosRESUMO
Importance: Parkinson disease and related disorders (PDRD) are the fastest growing neurodegenerative illness in terms of prevalence and mortality. As evidence builds to support palliative care (PC) for PDRD, studies are needed to guide implementation. Objective: To determine whether PC training for neurologists and remote access to a PC team improves outcomes in patients with PDRD in community settings. Design, Setting, and Participants: This pragmatic, stepped-wedge comparative effectiveness trial enrolled and observed participants from 19 community neurology practices supported by PC teams at 2 academic centers from March 8, 2017, to December 31, 2020. Participants were eligible if they had PDRD and moderate to high PC needs. A total of 612 persons with PDRD were referred; 253 were excluded. Patients were excluded if they had another diagnosis meriting PC, were receiving PC, or were unable or unwilling to follow study procedures. Patients received usual care or the intervention based on when their community neurologist was randomized to start the intervention. Data were analyzed from January 2021 to September 2023. Intervention: The intervention included (1) PC education for community neurologists and (2) team-based PC support via telehealth. Main Outcomes and Measures: The primary outcomes were differences at 6 months in patient quality of life (QOL; measured by the Quality of Life in Alzheimer Disease Scale [QOL-AD]) and caregiver burden (Zarit Burden Interview) between the intervention and usual care. Results: A total of 359 patients with PDRD (233 men [64.9%]; mean [SD] age, 74.0 [8.8] years) and 300 caregivers were enrolled. At 6 months, compared with usual care, participants receiving the intervention had better QOL (QOL-AD score, 0.09 [95% CI, -0.63 to 0.82] vs -0.88 [95% CI, -1.62 to -0.13]; treatment effect estimate, 0.97; 95% CI, 0.07-1.86; P = .03). No significant difference was observed in caregiver burden (Zarit Burden Interview score, 1.19 [95% CI, 0.16 to 2.23] vs 0.55 [95%, -0.44 to 1.54]; treatment effect estimate, 0.64; 95% CI, -0.62 to 1.90; P = .32). Advance directive completion was higher under the intervention (19 of 38 [50%] vs 6 of 31 [19%] among those without directives at the beginning of the study; P = .008). There were no differences in other outcomes. Conclusions and Relevance: PC education for community neurologists and provision of team-based PC via telehealth is feasible and may improve QOL and advance care planning. Overall treatment effects were small and suggest opportunities to improve both the intervention and implementation. Trial Registration: ClinicalTrials.gov Identifier: NCT03076671.
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Doença de Parkinson , Telemedicina , Masculino , Humanos , Idoso , Qualidade de Vida , Doença de Parkinson/terapia , Neurologistas , Cuidados Paliativos/métodos , Telemedicina/métodosRESUMO
OBJECTIVE: High upfront costs and long-term benefit uncertainties of gene therapies challenge Medicaid budgets, making value-based contracts a potential solution. However, value-based contract design is hindered by cost-offset uncertainty. The aim of this study is to determine actual cost-offsets for valoctocogene roxaparvovec (hemophilia A) and etranacogene dezaparvovec (hemophilia B) from Colorado Medicaid's perspective, defining payback periods and its uncertainty from the perspective of Colorado Medicaid. METHODS: This cost analysis used 2018-2022 data from the Colorado Department of Health Care Policy & Financing to determine standard-of-care costs and employed cost simulation models to estimate the cost of Medicaid if patients switched to gene therapy versus if they did not. Data encompassed medical and pharmacy expenses of Colorado Medicaid enrollees. Identified cohorts were patients aged 18+ with ICD-10-CM codes D66 (hemophilia A) and D67 (hemophilia B). Severe hemophilia A required ≥ 6 claims per year for factor therapies or emicizumab, while moderate/severe hemophilia B necessitated ≥ 4 claims per year for factor therapies. Patients were included in the cohort in the year they first met the criteria and were subsequently retained in the cohort for the duration of the observation period. Standard-of-care included factor VIII replacement therapy/emicizumab for hemophilia A and factor IX replacement therapies for hemophilia B. Simulated patients received valoctocogene roxaparvovec or etranacogene dezaparvovec. Main measures were annual standard-of-care costs, cost offset, and breakeven time when using gene therapies. RESULTS: Colorado Medicaid's standard-of-care costs for hemophilia A and B were $426,000 [standard deviation (SD) $353,000] and $546,000 (SD $542,000) annually, respectively. Substituting standard-of-care with gene therapy for eligible patients yielded 8-year and 6-year average breakeven times, using real-world costs, compared with 5 years with published economic evaluation costs. Substantial variability in real-world standard-of-care costs resulted in a 48% and 59% probability of breakeven within 10 years for hemophilia A and B, respectively. Altering eligibility criteria significantly influenced breakeven time. CONCLUSIONS: Real-world data indicates substantial uncertainty and extended payback periods for gene therapy costs. Utilizing real-world data, Medicaid can negotiate value-based contracts to manage budget fluctuations, share risk with manufacturers, and enhance patient access to innovative treatments.
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Hemofilia A , Hemofilia B , Estados Unidos , Humanos , Medicaid , Análise Custo-Benefício , Terapia GenéticaRESUMO
BACKGROUND: Current practice in health technology assessment (HTA) of pharmaceuticals conducts cost-effectiveness analyses (CEAs) based on a static price or the estimated price at market launch. Recent publications suggest incorporating dynamic pricing. To test the feasibility and importance of including dynamic pricing, we compared the standard static approach to four dynamic scenarios by replicating US-based HTA evaluations with dynamic pricing inputs. METHODS: The four case examples included omalizumab (Xolair®) for the treatment of allergic asthma, elagolix (Orilissa®) for the treatment of endometriosis, ocrelizumab (Ocrevus®) for the treatment of primary progressive multiple sclerosis (PPMS), and dupilumab (Dupixent®) for the treatment of atopic dermatitis (AD). The primary outcome was the relative percentage change in incremental cost-effectiveness ratios (ICERs) per quality-adjusted life-year (QALY) for two dynamic pricing scenarios versus static pricing. Secondary outcomes included the absolute difference in ICERs versus base-case and an assessment of decision uncertainty. RESULTS: Base-case ICERs were $327,000, $102,000, $700,000, and $102,000 for allergic asthma, endometriosis, PPMS, and AD, respectively. Across scenarios and case examples, the range of ICERs versus base-case varied from decreases of 56% to increases of 232%. The absolute difference in ICERs versus base-case ranged from decreases of $120,000 to increases of $758,000. Conclusions on cost effectiveness were altered in 2/16 scenarios across the four case examples. CONCLUSIONS: Given the decision context that US payers face, with prices varying over time, findings suggest further research to reduce uncertainty around price trajectories, as well as conducting or updating multiple assessments over the lifecycle of pharmaceutical products.
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Asma , Endometriose , Feminino , Humanos , Análise Custo-Benefício , Omalizumab , Asma/tratamento farmacológico , Análise de Custo-Efetividade , Anos de Vida Ajustados por Qualidade de VidaRESUMO
BACKGROUND AND OBJECTIVE: Several novel methods have been suggested to extend a conventional value assessment to capture a more comprehensive perspective of value from a patient perspective. The objective of this research was to demonstrate a framework for implementing a combined qualitative and quantitative method to elicit and prioritize patient experience value elements in rare diseases. Neuromyelitis optica spectrum disorder was used as a case study. METHODS: The method for eliciting and prioritizing patient experience value elements involved a three-step process: (1) collecting potential patient experience value elements from existing literature sources followed by deliberation by a multi-stakeholder research team; (2) a pre-workshop webinar and survey to identify additional patient-reported value elements; and (3) a workshop to discuss, prioritize the value elements using a swing weighting method. Outcomes were prioritized value elements with normalized weights for patients considering a treatment for neuromyelitis optica spectrum disorder. RESULTS: A literature review and deliberation resulted in the following initial value elements: ability to reach important personal milestones, patient's financial burden, value of hope/balance or timing of risks and benefits, Uncertainty about long-term benefits and safety of the treatment, Patient empowerment through therapeutic advancement and technology, Caregiver/family's financial burden, patient experience related to treatment regimen, Therapeutic options, and Caregiver/family's quality of life. Eight patients with neuromyelitis optica spectrum disorder participated in the case study. In the online survey, participants found the nine proposed patient experience value elements both understandable and important with no additions. During the workshop, 'Uncertainty about long-term benefits and safety,' 'Patient experience related to treatment regimen,' and 'Patient's financial burden' were found to be the most important patient experience value elements, with a respective weight of 25%, 19.2%, and 14.4% (out of total 100%). CONCLUSIONS: This case study provides a framework for eliciting and prioritizing patient experience value elements using direct patient input. Although elements/weights may differ by disease, and even in neuromyelitis optica spectrum disorder, additional research is needed, value frameworks, researchers, and manufacturers can use this practical method to generate patient experience value elements and evaluate their impact on treatment selection.
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BACKGROUND: Patients with atherosclerotic cardiovascular disease who require additional low-density lipoprotein cholesterol (LDL-C) lowering despite maximally tolerated statins have a significant unmet medical need and are at increased risk of future cardiovascular events and a reduced quality of life. OBJECTIVE: We aimed to estimate the percentage of cardiovascular events avoided following treatment with a fixed-dose combination of bempedoic acid plus ezetimibe (BA+EZE FDC) versus ezetimibe (EZE) in patients with atherosclerotic cardiovascular disease receiving maximally tolerated statins across a range of baseline LDL-C levels. METHODS: A Markov cohort simulation model estimated major adverse cardiovascular events avoided over a lifetime horizon among patients with atherosclerotic cardiovascular disease and baseline LDL-C levels from 80 to >200 mg/dL. BA+EZE FDC was compared with EZE based on mean percent LDL-C reductions versus placebo reported in a phase III trial. Health outcomes for the average patient were extrapolated to a US population of 100,000 persons using evidence on contemporary LDL-C levels from the National Health and Nutrition Examination Survey. RESULTS: Among patients with atherosclerotic cardiovascular disease not at the LDL-C goal with maximally tolerated statins, the addition of BA+EZE FDC compared with the addition of EZE was predicted to provide incremental absolute reductions in major adverse cardiovascular events dependent on baseline LDL-C levels at the population level. For those with baseline LDL-C of 101-110 mg/dL (n = 15,237), there were 4.9% (744) fewer events predicted, while for patients with baseline LDL-C of > 200 mg/dL (n = 1689), 10.9% (184) fewer events were predicted through the addition of BA+EZE FDC versus EZE. CONCLUSIONS: Further LDL-C reductions through the addition of BA+EZE FDC to maximally tolerated statins are predicted to reduce major adverse cardiovascular events compared with the addition of EZE. Benefits are potentially greater among those with higher starting LDL-C.
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Anticolesterolemiantes , Aterosclerose , Azetidinas , Doenças Cardiovasculares , Inibidores de Hidroximetilglutaril-CoA Redutases , Hipercolesterolemia , Humanos , Anticolesterolemiantes/efeitos adversos , Aterosclerose/tratamento farmacológico , Azetidinas/efeitos adversos , Doenças Cardiovasculares/epidemiologia , Doenças Cardiovasculares/prevenção & controle , Doenças Cardiovasculares/induzido quimicamente , LDL-Colesterol , Quimioterapia Combinada , Ezetimiba/efeitos adversos , Inibidores de Hidroximetilglutaril-CoA Redutases/efeitos adversos , Hipercolesterolemia/tratamento farmacológico , Qualidade de Vida , Resultado do TratamentoRESUMO
DISCLOSURES: Drs Rind, Campbell, Pearson, Ms Herce-Hagiwara, Ms Fluetsch, and Ms Herron-Smith report grants from Arnold Ventures; Kaiser Foundation Health Plan, Inc; The Patrick and Catherine Donaghue Medical Research Foundation; Blue Cross Blue Shield of Massachusetts; and California Healthcare Foundation during the course of this study.
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Anticorpos Monoclonais Humanizados , Asma , Anticorpos Monoclonais Humanizados/uso terapêutico , Asma/tratamento farmacológico , Análise Custo-Benefício , Humanos , MassachusettsRESUMO
BACKGROUND: Sepsis causes a major health burden in the United States. To better understand the role of sepsis as a driver of the burden and cost of foodborne illness in the United States, we estimated the frequency and treatment cost of sepsis among US patients hospitalized with 31 pathogens commonly transmitted through food or with unspecified acute gastrointestinal illness (AGI). METHODS: Using data from the National Inpatient Sample from 2012 to 2015, we identified sepsis hospitalizations using 2 approaches-explicit ICD-9-CM codes for sepsis and a coding scheme developed by Angus that identifies sepsis using specific ICD-9-CM diagnosis codes indicating an infection plus organ failure. We examined differences in the frequency and the per-case cost of sepsis across pathogens and AGI and estimated total hospitalization costs using prior estimates of foodborne hospitalizations. RESULTS: Using Explicit Sepsis Codes, sepsis hospitalizations accounted for 4.6% of hospitalizations with a pathogen commonly transmitted through food or unspecified AGI listed as a diagnosis; this was 33.2% using Angus Sepsis Codes. The average per-case cost was $35â 891 and $20â 018, respectively. Applying the proportions of hospitalizations with sepsis from this study to prior estimates of the number foodborne hospitalizations, the total annual cost was $248 million annually using Explicit Sepsis Codes and $889 million using Angus Sepsis Codes. CONCLUSIONS: Sepsis is a serious complication among patients hospitalized with a foodborne pathogen infection or AGI resulting in a large burden of illness. Hospitalizations that are diagnosed using explicit sepsis codes are more severe and costly, but likely underestimate the burden of foodborne sepsis.
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Doenças Transmitidas por Alimentos , Sepse , Efeitos Psicossociais da Doença , Doenças Transmitidas por Alimentos/epidemiologia , Hospitalização , Humanos , Incidência , Classificação Internacional de Doenças , Sepse/diagnóstico , Sepse/epidemiologia , Estados Unidos/epidemiologiaRESUMO
The pipeline for new treatments for Alzheimer's disease (AD) in the USA contains over 100 different agents, 80% of which can be categorized as disease-modifying therapies. The regulatory approval of the disease-modifying agent aducanumab has brought more attention to the complexity of the diagnosis, evaluation, and treatment of AD and the difficult decisions payers and policy makers will face over the next few years as innovation continues in this space. The value of AD treatment can vary widely according to the perspective of the analysis, sources of data, and methodological approach for the value assessment. This article focuses on AD-specific data gaps and measurement challenges and provides guidance for evidence generation to facilitate better value assessments for future AD treatments.
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Doença de Alzheimer , Doença de Alzheimer/tratamento farmacológico , HumanosRESUMO
Background: Defining the value of healthcare is an elusive target, and depends heavily on the decision context and stakeholders involved. Cost-utility analysis and the quality-adjusted life year (QALY) have become the method and value definition of choice for traditional value judgements in coverage and pricing decisions. Other criteria that may influence value are often not measured and therefore omitted from value assessments, or are only used to qualitatively contextualize assessments. The objective of this study was to engage two key stakeholders; patients and payers to elicit and rank the importance of additional value criteria, potentially assessed in Multiple Criteria Decision Analysis (MCDA). Methods: This study consisted of a focus group with cancer patients (n = 7), including follow-up questions through an electronic survey, and in-depth phone interviews with payers (n = 5). Results: For payers, value equated either with criteria that provided tangible benefits (from their perspective) such as new treatment options that respond to serious unmet need. For patients, population-level value equated to options that would potentially benefit them in the future and the value of hope. However, these criteria were seen by payers as difficult to measure and incorporate into objective decision making. Limitations: The findings from this study are primarily limited due to generalizability. Due to the small sample size, it was outside the scope of this study to calculate a weight for each criterion that could be used as part of a quantitative MCDA. Conclusion: MCDA, with particular attention to qualitative aspects, is an avenue to incorporate these additional criteria into value assessments, as well as provide an opportunity for reflecting the patient's preferences in assessing the value of a treatment.
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BACKGROUND: Harvard Pilgrim Health Care expanded coverage for non-invasive prenatal testing (NIPT) to include all pregnant, single-gestation women aged < 35 years, through a performance-based risk-sharing (PBRS) agreement with Illumina to offset costs from coverage expansion. NIPT analyzes cell-free DNA fragments from a maternal blood sample to screen for fetal aneuploidies and is considered a more accurate screening method than conventional serum biochemical screening and nuchal translucency ultrasound-based approaches. OBJECTIVE: This study assessed the impact of NIPT coverage expansion on prenatal screening strategies and payer expenditures. METHODS: This was a real-world comparison of utilization and expenditures of prenatal screening and diagnostic testing in pregnant women aged < 35 years pre- (1 March 2016-28 February 2018) and post- (1 March 2018-30 September 2019) coverage expansion. Incidence rate ratios (IRRs) with 95% confidence intervals (CIs) were estimated to compare changes in utilization of conventional and NIPT-based prenatal screening methods. Change in per member per month (PMPM) expenditures in $US year 2020 were assessed post-coverage expansion using a budget impact model. RESULTS: A total of 5041 and 4109 distinct pregnancies were identified in pre- and post-coverage expansion periods, respectively. Mean ± standard deviation maternal age was consistent between pre- and post-coverage expansion periods (30.35 ± 3.35 and 30.33 ± 3.28, respectively). Screening orders for conventional methods decreased, with an adjusted IRR in the post-expansion period of 0.87 (95% CI 0.85-0.90) times the rate in the pre-expansion period; orders for NIPT increased, with an adjusted IRR in the post-expansion period of 1.41 (95% CI 1.32-1.51) times the rate in the pre-expansion period. Invasive diagnostic testing was low at baseline (1.0%) and did not change post-coverage expansion. The change in PMPM is estimated at $US0.026 post-coverage expansion. CONCLUSION: The PBRS agreement to expand NIPT coverage for women aged < 35 years was associated with an increase in NIPT utilization, decreases in conventional screening methods, and a modest increase in PMPM expenditures.
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OBJECTIVES: The quality-adjusted life-year (QALY) has been long debated, but alternative estimation approaches have not been comprehensively evaluated. Our objective was to identify alternatives, characterize them by implementation feasibility, and evaluate the impact of implementing feasible options in cost-effectiveness models developed for the Institute for Clinical and Economic Review reports. METHODS: We conducted a literature review combining keywords relating to QALYs, methodology alternatives, and cost-effectiveness in PubMed, EconLit, Web of Science, and MEDLINE. Articles that discussed alternatives to the conventional QALY were included. Alternatives were characterized by type, data availability, calculation burden, and overall implementation feasibility. The subset of feasible alternatives, that is, sufficient data and methodology compatible with incorporation into common modeling approaches, were evaluated according to impact on incremental QALYs, incremental net monetary benefit (iNMB), intervention rankings, and proportion of interventions with a positive iNMB. RESULTS: We identified 28 articles discussing 9 alternatives. Feasible alternatives were using patient preference (PP) data; equity weighting according to baseline utility, fair innings, or proportional QALY shortfall; and the equal value of life-years-gained approach. All alternatives affected the incremental QALY and iNMB outcomes, rankings, and proportion of interventions with a positive iNMB. The PP alternative had the largest and most consistent impact. The PP impact on the proportion of interventions with a positive iNMB, was in the negative direction. CONCLUSIONS: Our work is the first comprehensive evaluation of proposed alternatives to the conventional QALY. We found robust literature but few options that were feasible to be implemented in current healthcare decision-making processes.
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Análise Custo-Benefício/métodos , Anos de Vida Ajustados por Qualidade de Vida , Estudos de Viabilidade , Humanos , Modelos EstatísticosRESUMO
BACKGROUND: Ovarian cancer is the fifth leading cause of cancer death in women in the US. With poly(ADP-ribose) polymerase (PARP) inhibitors having shown promising results in ongoing trials, there is interest in better understanding their economic value. OBJECTIVE: This study aimed to review and evaluate the quality of published cost-effectiveness analyses (CEAs), and provide recommendations for CEAs in this setting. METHODS: A systematic literature review of the MEDLINE and EMBASE databases was conducted in June 2019 to identify CEAs of PARP inhibitors in treating advanced ovarian cancer from peer-reviewed journals and conferences. Key information from the identified publications were extracted and reviewed. The quality of full-text studies was assessed using the Quality of Health Economic Studies instrument. Recommendations for future CEAs were developed based on the findings from the literature review. RESULTS: Eighteen CEAs (five in full texts) met the inclusion criteria. Most adopted a US healthcare or societal perspective. The majority of the studies did not clearly display the economic model structure. No studies reported the validation of model projections based on internal or external data. Surrogate outcomes such as incremental costs per progression-free life-year gained were the most common outcomes reported. The majority of studies drew their conclusions based on surrogate outcomes, even with no theoretical or empirical threshold for cost effectiveness. All five full-text studies included some type of sensitivity or scenario analyses. The key drivers of the incremental cost-effectiveness ratio were treatment duration, effects, and costs, health utility, and prevalence of BRCA mutations. CONCLUSION: In the existing CEAs for PARP inhibitors, there were uncertainties and challenges leading to variation in quality. We provided recommendations to improve consistency and quality of CEAs in this setting, which will help to better understand the value of PARP inhibitors, improve decision making, and reduce potential misallocation of resources.
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Neoplasias Ovarianas , Inibidores de Poli(ADP-Ribose) Polimerases/uso terapêutico , Análise Custo-Benefício , Feminino , Humanos , Modelos Econômicos , Neoplasias Ovarianas/tratamento farmacológicoRESUMO
DISCLOSURES: Funding for this summary was contributed by Arnold Ventures, Commonwealth Fund, California Health Care Foundation, National Institute for Health Care Management (NIHCM), New England States Consortium Systems Organization, Blue Cross Blue Shield of Massachusetts, Harvard Pilgrim Health Care, Kaiser Foundation Health Plan, and Partners HealthCare to the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the evidence on the value of health care interventions. ICER's annual policy summit is supported by dues from Aetna, America's Health Insurance Plans, Anthem, Allergan, Alnylam, AstraZeneca, Biogen, Blue Shield of CA, Cambia Health Services, CVS, Editas, Express Scripts, Genentech/Roche, GlaxoSmithKline, Harvard Pilgrim, Health Care Service Corporation, Health Partners, Johnson & Johnson (Janssen), Kaiser Permanente, LEO Pharma, Mallinckrodt, Merck, Novartis, National Pharmaceutical Council, Premera, Prime Therapeutics, Regeneron, Sanofi, Spark Therapeutics, and United Healthcare. Pearson is employed by ICER; Synnott was employed by ICER at the time of this report. Ollendorf, Campbell, and McQueen received grants from ICER for work on this review. Ollendorf also reports advisory board, consulting, and other fees from Sarepta Therapeutics, DBV Technologies, EMD Serono, Gerson Lehman Group, The CEA Registry Sponsors, Autolus, Analysis Group, Amgen, AbbVie, Cytokinetics, Aspen Institute/University of Southern California, and University of Colorado, unrelated to this review.