Quantitative Evidence Synthesis Methods for the Assessment of the Effectiveness of Treatment Sequences for Clinical and Economic Decision Making: A Review and Taxonomy of Simplifying Assumptions
AbstractSequential use of alternative treatments for chronic conditions represents a complex intervention pathway; previous treatment and patient characteristics affect both the choice and effectiveness of subsequent treatments. This paper critically explores the methods for quantitative evidence synthesis of the effectiveness of sequential treatment options within a health technology assessment (HTA) or similar process. It covers methods for developing summary estimates of clinical effectiveness or the clinical inputs for the cost-effectiveness assessment and can encompass any disease condition. A comprehensive review of ...
Source: PharmacoEconomics - November 26, 2020 Category: Health Management Source Type: research

Budget Impact Analysis of Gemtuzumab Ozogamicin for the Treatment of CD33-Positive Acute Myeloid Leukemia
ConclusionsThe introduction of GO for newly diagnosed and R/R AML would have a minimal impact on the budget of a US health plan and could result in cost savings in the combination therapy setting for newly diagnosed AML. (Source: PharmacoEconomics)
Source: PharmacoEconomics - November 25, 2020 Category: Health Management Source Type: research

Comparing the Analysis and Results of a Modified Social Accounting Matrix Framework with Conventional Methods of Reporting Indirect Non-Medical Costs
ConclusionsIn contrast to conventional methods, the SAM approach provides detailed and comprehensive assessments of the impact of new interventions on the indirect non-medical costs and the financial interactions between agents, disclosing useful information for different stakeholders. (Source: PharmacoEconomics)
Source: PharmacoEconomics - November 25, 2020 Category: Health Management Source Type: research

Use of Productivity Loss/Gain in Cost-Effectiveness Analyses for Drugs: A Systematic Review
ConclusionsAlthough inclusion of productivity losses/gains was shown to have a favorable impact on evaluations for many studies, their impact and method of calculation was often not reported or was unclear. Further examination and discussion is needed to consider the optimal framework for considering productivity losses/gains in cost-effectiveness analyses, including the appropriate cost elements to include (e.g., patient absenteeism, caregiver absenteeism, presenteeism, unemployment, etc.) and how those costs should be estimated. (Source: PharmacoEconomics)
Source: PharmacoEconomics - November 24, 2020 Category: Health Management Source Type: research

Probabilistic One-Way Sensitivity Analysis with Multiple Comparators: The Conditional Net Benefit Frontier
AbstractAlthough there have been substantial developments in the analysis of uncertainty in economic evaluations of health care programmes, the development of methods for one-way sensitivity analysis has been notably slower. Conditional incremental net benefit was recently proposed as an approach for implementing probabilistic one-way sensitivity analysis for economic evaluations comparing two strategies. In this paper, we generalise this approach to economic evaluations that compare three or more strategies. We find that ‘conditional net benefit’ may be used to conduct probabilistic one-way sensitivity analysi...
Source: PharmacoEconomics - November 23, 2020 Category: Health Management Source Type: research

Comment on “Market Exclusivity for Drugs with Multiple Orphan Approvals (1983–2017) and Associated Budget Impact in the US”
(Source: PharmacoEconomics)
Source: PharmacoEconomics - November 9, 2020 Category: Health Management Source Type: research

Reply to Comment on “Market Exclusivity for Drugs with Multiple Orphan Approvals (1983–2017) and Associated Budget Impact in the US”
(Source: PharmacoEconomics)
Source: PharmacoEconomics - November 9, 2020 Category: Health Management Source Type: research

Evaluating New Zealanders ’ Values for Drug Coverage Decision Making: Trade-Offs between Treatments for Rare and Common Conditions
ConclusionsNew Zealanders ultimately value drug-related factors (e.g. quality of life and efficacy) and disease-related factors (e.g. severity of disease and equity) the most but did not value disease rarity. (Source: PharmacoEconomics)
Source: PharmacoEconomics - November 5, 2020 Category: Health Management Source Type: research

Budget Impact of Oral Semaglutide Intensification versus Sitagliptin among US Patients with Type 2 Diabetes Mellitus Uncontrolled with Metformin
ConclusionsUse of oral semaglutide 14  mg in patients currently receiving sitagliptin 100 mg substantially increases the budget impact for patients with T2DM whose blood glucose level is not controlled with metformin over a 5-year time horizon for US healthcare payers. (Source: PharmacoEconomics)
Source: PharmacoEconomics - November 5, 2020 Category: Health Management Source Type: research

The Budget Impact of Including Rucaparib on a US Payer Formulary for the Treatment of Patients with Metastatic Ovarian Cancer
ConclusionThe budget impact of adding rucaparib to the formulary for a health plan adds negligible PMPM costs of
Source: PharmacoEconomics - November 4, 2020 Category: Health Management Source Type: research

Lenalidomide with Rituximab for Previously Treated Follicular Lymphoma and Marginal Zone Lymphoma: An Evidence Review Group Perspective of a NICE Single Technology Appraisal
AbstractThe National Institute for Health and Care Excellence (NICE) invited the manufacturer (Celgene) of lenalidomide (Revlimid®), as part of the Single Technology Appraisal (STA) process, to submit evidence for the clinical effectiveness and cost-effectiveness of lenalidomide in combination with rituximab (MabThera®), together referred to asR2, for the treatment of adults with treated follicular lymphoma (FL) or marginal zone lymphoma (MZL). Kleijnen Systematic Reviews Ltd, in collaboration with Maastricht University Medical Centre+, was commissioned to act as the independent Evidence Review Group (ERG). This pa...
Source: PharmacoEconomics - November 4, 2020 Category: Health Management Source Type: research

Acknowledgement to Referees
(Source: PharmacoEconomics)
Source: PharmacoEconomics - November 4, 2020 Category: Health Management Source Type: research

Overlap of Depressive Symptoms with Health-Related Quality-of-Life Measures
AbstractBecause depressive symptoms are a part of health-related quality-of-life (HRQOL) measures, measures of depression will be empirically associated with HRQOL. We discuss examples of published research where authors ignored or did not fully account for overlap between depressive symptom and HRQOL measures. Future researchers need to recognize when their models include conceptually similar variables on the same side or both sides of the equation. This awareness will lead to more accurate conclusions about the prognostic value of depression and other HRQOL measures for health care utilization, mortality, and other outco...
Source: PharmacoEconomics - November 2, 2020 Category: Health Management Source Type: research

Dealing with Bad Risk in Cost-Effectiveness Analysis: The Cost-Effectiveness Risk-Aversion Curve
AbstractCost-effectiveness analysis has been advocated and is widely used to inform policy and decision makers in setting priorities for resource allocation. Since the costs and effects of health care interventions are uncertain, much research interest has focused on handling uncertainty in cost-effectiveness analysis. The most widely used method to summarize uncertainty in cost-effectiveness analysis is the cost-effectiveness acceptability curve, which estimates the probability that an intervention is cost effective for a wide range of threshold ratios. However, by estimating the uncertainty associated with incremental co...
Source: PharmacoEconomics - October 31, 2020 Category: Health Management Source Type: research

Correction to: Perspective and Costing in Cost ‑Effectiveness Analysis, 1974–2018
The article Perspective and  Costing in Cost-Effectiveness Analysis. (Source: PharmacoEconomics)
Source: PharmacoEconomics - October 21, 2020 Category: Health Management Source Type: research

An Updated Systematic Review of Cost-Effectiveness Analyses of Drugs for Osteoporosis
ConclusionsThis updated review provides an overview of recently published cost-effectiveness analyses. In comparison with a previous review, recent economic evaluations of anti-osteoporosis drugs were conducted in more countries and included more active drugs and sequential therapy as interventions/comparators. The updated economic evidence could help decision makers prioritize health interventions and the unmet/unreported quality issues indicated by the osteoporosis-specific guideline could be useful in improving the transparency, quality, and comparability of future economic evaluations in osteoporosis. (Source: PharmacoEconomics)
Source: PharmacoEconomics - October 6, 2020 Category: Health Management Source Type: research

The Relative Importance of Education and Criminal Justice Costs and Benefits in Economic Evaluations: A Best –Worst Scaling Experiment
ConclusionsThis study identified the most important education and criminal justice ICBs for economic evaluations of interventions targeting MBDs and suggests that it could be relevant to include these ICBs in economic evaluations. (Source: PharmacoEconomics)
Source: PharmacoEconomics - October 6, 2020 Category: Health Management Source Type: research

Health Economic Evaluation Alongside Stepped Wedge Trials: A Methodological Systematic Review
ConclusionsThe use of appropriate statistical methods that account for time, clustering and correlation between costs and outcomes is an important part of SWT health economics analysis, one that will benefit from an effort to communicate the methods available and their performance. (Source: PharmacoEconomics)
Source: PharmacoEconomics - October 4, 2020 Category: Health Management Source Type: research

Predictors of Health Utility in Relapsing –Remitting and Secondary-Progressive Multiple Sclerosis: Implications for Future Economic Models of Disease-Modifying Therapies
ConclusionsThis study suggests that in addition to EDSS and recent relapses, T25FW significantly predicts utility in RRMS and SPMS. These findings support the use of T25FW to supplement the EDSS and the occurrence of relapses to characterize the course of disease progression and to more accurately accrue quality-adjusted life-years in future economic evaluations of disease-modifying therapies for the treatment of RRMS. (Source: PharmacoEconomics)
Source: PharmacoEconomics - September 28, 2020 Category: Health Management Source Type: research

Recalibrating Health Technology Assessment Methods for Cell and Gene Therapies
AbstractRecently licensed cell and gene therapies have promising but highly uncertain clinical benefits. They are entering the market at very high prices, with the latest entrants costing hundreds of thousands of dollars. The significant long-term uncertainty posed by these therapies has already complicated the use of conventional economic evaluation approaches such as cost-effectiveness and cost-utility analyses, which are widely used for assessing the value of new health interventions. Cell and gene therapies also risk jeopardising healthcare systems ’ financial sustainability. As a result, there is a need to ...
Source: PharmacoEconomics - September 21, 2020 Category: Health Management Source Type: research

Cost Effectiveness of Teplizumab for Prevention of Type 1 Diabetes Among Different Target Patient Groups
ConclusionsCost-effective provision of teplizumab to target individuals depends on the price of teplizumab and genetic and antibody characteristics of treated individuals. As the drug makes its way to the  market, findings from this study will help inform policymakers and payers on cost-effective ways to provide this innovative but expensive drug to at-risk individuals. (Source: PharmacoEconomics)
Source: PharmacoEconomics - September 21, 2020 Category: Health Management Source Type: research

Cost-Effectiveness of Tofacitinib for Patients with Moderate-to-Severe Rheumatoid Arthritis in China
ConclusionsUsing the WHO-recommended ICER acceptability threshold of ≤ 1-time per capita Gross Domestic Product (GDP), our analysis suggests that the introduction of tofacitinib into the current treatment sequence for moderate-to-severe RA patients with csDMARDs-IR in China was a cost saving option as first- and second-line treatment, and cost-effective as a third- line treatment option. Of note, use of tofacitinib as first- and second-line treatment post-csDMARDs-IR appeared to be cost saving. (Source: PharmacoEconomics)
Source: PharmacoEconomics - September 14, 2020 Category: Health Management Source Type: research

Updating the Cost Effectiveness of Oral Anticoagulants for Patients with Atrial Fibrillation Based on Varying Stroke and Bleed Risk Profiles
ConclusionsFrom a US private payer ’s perspective, dabigatran appears cost effective compared with other anticoagulants. This study indicated risk stratification especially considering both stroke and bleed risk simultaneously is important not only in clinical practice but also in health technology assessment exercises among patien ts with atrial fibrillation. (Source: PharmacoEconomics)
Source: PharmacoEconomics - September 13, 2020 Category: Health Management Source Type: research

On Pandemic Preparedness: How Well is the Modeling Community Prepared for COVID-19?
(Source: PharmacoEconomics)
Source: PharmacoEconomics - September 13, 2020 Category: Health Management Source Type: research

The SF-6Dv2: How Does the New Classification System Impact the Distribution of Responses Compared with the Original SF-6D?
(Source: PharmacoEconomics)
Source: PharmacoEconomics - September 9, 2020 Category: Health Management Source Type: research

Sustainability of Publicly Funded Health Care Systems: What Does Behavioural Economics Offer?
AbstractThere has been a rapid increase in the use of behavioural economics (BE) as a tool for policy makers to deploy, including in health-related applications. While this development has occurred over the past decade, health care systems have continued to struggle with escalating costs. We consider the potential role of BE for making improvements to health care system performance and the sustainability of publicly funded health care systems, in particular. We argue that the vast majority of applications in this field have been largely focussed on BE and public health, or the prevailing level of risks to health in populat...
Source: PharmacoEconomics - September 7, 2020 Category: Health Management Source Type: research

Correction to: Estimating Transition Probabilities from Published Evidence: A Tutorial for Decision Modelers
In the original version of this article a number of equations were incorrectly represented. (Source: PharmacoEconomics)
Source: PharmacoEconomics - September 7, 2020 Category: Health Management Source Type: research

Voretigene Neparvovec for Treating Inherited Retinal Dystrophies Caused by RPE65 Gene Mutations: An Evidence Review Group Perspective of a NICE Highly Specialised Technology Appraisal
AbstractThe UK National Institute for Health and Care Excellence (NICE) considered evidence for voretigene neparvovec (VN; Luxturna®) for the treatment ofRPE65-mediated inherited retinal dystrophies (IRD) within its highly specialised technology programme. This paper summarises the evidence provided by the company; the appraisal of the evidence by the Peninsula Technology Appraisal Group, who were commissioned to act as the independent evidence review group (ERG); and the development of the NICE guidance by the appraisal committee. The evidence presented by the company highlighted the significant lifelong burden of IRD...
Source: PharmacoEconomics - September 1, 2020 Category: Health Management Source Type: research

Informing a Cost-Effectiveness Threshold for Health Technology Assessment in China: A Marginal Productivity Approach
ConclusionOur results suggest that the current cost-effectiveness threshold used in China is too high; continuing to use it risks decisions that reduce overall population health. (Source: PharmacoEconomics)
Source: PharmacoEconomics - August 27, 2020 Category: Health Management Source Type: research

A Systematic Review of Medical Costs Associated with Heart Failure in the USA (2014 –2020)
ConclusionsThe review underpins the conclusion drawn in earlier reviews, namely that hospitalization costs are the key driver of heart failure-related costs. Analyses of subgroups provide a clearer understanding of sources of heterogeneity in cost data. While current cost estimates provide useful indications of economic burden, understanding the nuances of the data is critical to support its application. (Source: PharmacoEconomics)
Source: PharmacoEconomics - August 18, 2020 Category: Health Management Source Type: research

Correction to: Calculating and Interpreting ICERs and Net Benefit
The original article can be found online. (Source: PharmacoEconomics)
Source: PharmacoEconomics - August 18, 2020 Category: Health Management Source Type: research

Simulating Progression-Free and Overall Survival for First-Line Doublet Chemotherapy With or Without Bevacizumab in Metastatic Colorectal Cancer Patients Based on Real-World Registry Data
ConclusionsExtensive internal validation showed that DES accurately estimated the outcomes of treatment combination strategies for specific subpopulations, with outcomes suggesting treatment could be optimized. Although results based on real-world data are informative, they cannot replace randomized trials. (Source: PharmacoEconomics)
Source: PharmacoEconomics - August 16, 2020 Category: Health Management Source Type: research

A Critical Appraisal and Recommendations for Cost-Effectiveness Studies of Poly(ADP-Ribose) Polymerase Inhibitors in Advanced Ovarian Cancer
ConclusionIn 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. (Source: PharmacoEconomics)
Source: PharmacoEconomics - August 13, 2020 Category: Health Management Source Type: research

Estimating Transition Probabilities from Published Evidence: A Tutorial for Decision Modelers
AbstractThis tutorial presents practical guidance on transforming various types of information published in journals, or available online from government and other sources, into transition probabilities for use in state-transition models, including cost-effectiveness models. Much, but not all, of the guidance has been previously published in peer-reviewed journals. Our purpose is to collect it in one location to serve as a stand-alone resource for decision modelers who draw most or all of their information from the published literature. Our focus is on the technical aspects of manipulating data to derive transition proba...
Source: PharmacoEconomics - August 13, 2020 Category: Health Management Source Type: research

Inclisiran as Adjunct Lipid-Lowering Therapy for Patients with Cardiovascular Disease: A Cost-Effectiveness Analysis
ConclusionsAs an adjunct therapy to statin treatment in those who have persistently elevated LDL-C despite optimal statin therapy, inclisiran is effective in reducing cardiovascular events in patients with atherosclerotic CVD. Inclisiran is not cost effective from the Australian healthcare perspective, assuming acquisition costs of current PCSK9 inhibitors. The cost of inclisiran would have to be 60% lower than that of evolocumab. (Source: PharmacoEconomics)
Source: PharmacoEconomics - August 12, 2020 Category: Health Management Source Type: research

Systematic Review and Meta-Analysis of Community- and Choice-Based Health State Utility Values for Lung Cancer
AbstractBackgroundUsing appropriate health state utility values (HSUVs) is critical for economic evaluation of new lung cancer interventions, such as low-dose computed tomography screening and immunotherapy. Therefore, we provide a systematic review and meta-analysis of community- and choice-based HSUVs for lung cancer.MethodsOn 6 March 2017, we conducted a systematic search of the following databases: Embase, Ovid MEDLINE, Web of Science, Cochrane CENTRAL, Google Scholar, and the School of Health and Related Research Health Utility Database. The search was updated on 17 April 2019. Studies reporting mean or median lung ca...
Source: PharmacoEconomics - August 4, 2020 Category: Health Management Source Type: research

Cost of Nine Pediatric Infectious Illnesses in Low- and Middle-Income Countries: A Systematic Review of Cost-of-Illness Studies
Conclusions and Implications of Key FindingsThe review yielded 37 articles and 267 sets of cost estimates. We found no cost-of-illness studies with cost estimates for hepatitis B, measles, rubella, or yellow fever from primary data. Most estimates were from countries in Gavi preparatory (28%) and accelerated (28%) transition, followed by those who are initiating self-financing (22%) and those not eligible for Gavi support (19%). Thirteen articles compared household expenses to manage illnesses with income and two articles with other household expenses, such as food, clothing, and rent. An episode of illness represented 1 &...
Source: PharmacoEconomics - August 3, 2020 Category: Health Management Source Type: research

Incorporating Pharmacometrics into Pharmacoeconomic Models: Applications from Drug Development
AbstractPharmacometrics is the science of quantifying the relationship between the pharmacokinetics and pharmacodynamics of drugs in combination with disease models and trial information to aid in drug development and dosing optimization for clinical practice. Considering the variability in the dose –concentration–effect relationship of drugs, an opportunity exists in linking pharmacokinetic and pharmacodynamic model-based estimates with pharmacoeconomic models. This link may provide early estimates of the cost effectiveness of drug therapies, thus informing late-stage drug development, pri cing, and reimbursem...
Source: PharmacoEconomics - July 30, 2020 Category: Health Management Source Type: research

Integrative Review of Managed Entry Agreements: Chances and Limitations
ConclusionsWe are likely to see a growth in MEAs with the continual launch of new high-priced and often complex treatments, coupled with increasing demands on resources. Whilst outcome-based MEAs could be an important tool to improve access to new innovative medicines, there are critical issues to address. Comparing knowledge, experiences, and practices across countries is crucial to guide high- and middle-income countries when designing their future MEAs. (Source: PharmacoEconomics)
Source: PharmacoEconomics - July 30, 2020 Category: Health Management Source Type: research

Taking the Analysis of Trial-Based Economic Evaluations to the Next Level: The Importance of Accounting for Clustering
ConclusionsAlthough OLS produced similar cost-effectiveness outcomes, it substantially underestimated the amount of variation in the data compared with MLM. To prevent suboptimal conclusions and a possible waste of scarce resources, it is important to use MLM in trial-based economic evaluations when data are clustered. (Source: PharmacoEconomics)
Source: PharmacoEconomics - July 29, 2020 Category: Health Management Source Type: research

Cost-Effectiveness of Cannabidiol Adjunct Therapy versus Usual Care for the Treatment of Seizures in Lennox-Gastaut Syndrome
ConclusionCBD does not appear to be a cost-effective therapeutic option in LGS patients at a willingness-to-pay threshold of $150,000/QALY. (Source: PharmacoEconomics)
Source: PharmacoEconomics - July 25, 2020 Category: Health Management Source Type: research

Perspective and Costing in Cost-Effectiveness Analysis, 1974 –2018
ConclusionResearchers should justify and be transparent about their choice of perspective and costing approaches. The use of the impact inventory and reporting of disaggregate outcomes can reduce inconsistencies and confusion. (Source: PharmacoEconomics)
Source: PharmacoEconomics - July 21, 2020 Category: Health Management Source Type: research

External Validation of the Core Obesity Model to Assess the Cost-Effectiveness of Weight Management Interventions
ConclusionThe predictions generated by the risk equations used in the COM showed good concordance both with the studies used to develop the model and with studies not included in the model. In particular, the concordance observed in the external dependent validation suggests that the COM accurately predicts obesity-related event rates observed in the studies used to develop the model. However, the impact of existing CV risk, as well as mortality, is a key area for future refinement of the COM. Our results should increase confidence in the estimates derived from the COM and reduce uncertainty associated with analyses using ...
Source: PharmacoEconomics - July 12, 2020 Category: Health Management Source Type: research

Producing Standardized Country-Level Immunization Delivery Unit Cost Estimates
ConclusionsAccurate immunization delivery costs are necessary for assessing the cost-effectiveness and strategic planning needs of immunization programs. The cost estimates from this analysis provide a broad indication of immunization delivery costs that may be useful when accurate local data are unavailable. (Source: PharmacoEconomics)
Source: PharmacoEconomics - June 27, 2020 Category: Health Management Source Type: research

Comment on “Replacing the NCCN’s Blocks with Wheels: How Should Consideration of Societal Spending be Incorporated into Oncology Practice?”
(Source: PharmacoEconomics)
Source: PharmacoEconomics - June 25, 2020 Category: Health Management Source Type: research

Response to Comment on “Replacing the NCCN’s Blocks with Wheels: How Should Consideration of Societal Spending be Incorporated into Oncology Practice?”
(Source: PharmacoEconomics)
Source: PharmacoEconomics - June 25, 2020 Category: Health Management Source Type: research

Novel Treatment Strategies for Secondary Prevention of Cardiovascular Disease: A Systematic Review of Cost-Effectiveness
ConclusionsBased on current pricing and willingness-to-pay thresholds, most CEA studies considered ezetimibe, n-3 PUFAs and the polypill to be cost effective compared with standard care but not PCSK9 inhibitors for secondary prevention of CVD. (Source: PharmacoEconomics)
Source: PharmacoEconomics - June 24, 2020 Category: Health Management Source Type: research

Surrogate Endpoints in Health Technology Assessment: An International Review of Methodological Guidelines
This study undertook an updated survey of methodological guidance for using surrogate endpoints across international HTA agencies. We reviewed HTA and economic evaluation methods guidance from European, Australian and Canadian HTA agencies. We considered how guidelines addressed the methods for handling surrogate endpoints, including (1) level of evidence, (2) methods of validation, and (3) thresholds of acceptability. Across the 73 HTA agencies surveyed, 29 (40%) had methodological guidelines that made specific reference to consideration of surrogate outcomes. Of the 45 methods documents analysed, the majority [27 (60%)] ...
Source: PharmacoEconomics - June 22, 2020 Category: Health Management Source Type: research

Does Cost-Effectiveness Analysis Really Need to Abandon the Incremental Cost-Effectiveness Ratio to Embrace Net Benefit?
(Source: PharmacoEconomics)
Source: PharmacoEconomics - June 14, 2020 Category: Health Management Source Type: research

Market Exclusivity for Drugs with Multiple Orphan Approvals (1983 –2017) and Associated Budget Impact in the US
AbstractObjectivesTheOrphan Drug Act extends exclusivity of branded drugs by 7  years for each rare disease approval. By extending market exclusivity, manufacturers can forestall generic competition. We determined the prevalence of drugs with multiple orphan approvals, the duration for which manufacturers are able to maintain exclusivity using this mechanism, and the budget i mpact of these additional exclusivity periods on US spending on orphan drugs.MethodsWe analyzed a retrospective cohort of US orphan drug approvals filed between 1983 and 2017. Drug costs throughout this time period were measured using IQVIA claim...
Source: PharmacoEconomics - June 11, 2020 Category: Health Management Source Type: research