Utility Decrements Associated with Adult Overweight and Obesity in Australia: A Systematic Review and Meta-Analysis
ConclusionsOur results demonstrate that elevated weight status is associated with small but statistically significant reductions in utility compared with healthy weight, which will result in reduced quality-adjusted life years when extrapolated across time and used in economic evaluations. (Source: PharmacoEconomics)
Source: PharmacoEconomics - February 22, 2021 Category: Health Management Source Type: research

Methodological Challenges in the Economic Evaluation of a Gene Therapy for RPE65-Mediated Inherited Retinal Disease: The Value of Vision
This study underlines the findings from Drummond et al. that standard methods can be used to evaluate gene therapies. However, given uncertainty about (particularly long-term) out comes of gene therapies, guidance is required on the acceptable extrapolation of treatment effect of gene therapies and on how to handle the uncertainty around this extrapolation in scenario and sensitivity analyses to aid health technology assessment research and align submissions of future gene th erapies. (Source: PharmacoEconomics)
Source: PharmacoEconomics - February 19, 2021 Category: Health Management Source Type: research

Valuation of SF-6Dv2 Health States in China Using Time Trade-off and Discrete-Choice Experiment with a Duration Dimension
ConclusionsThe Chinese value set for the SF-6Dv2 was established based on the TTO approach, but the DCETTO also performed well. Minor issues of non ­monotonicity did present for DCETTO. (Source: PharmacoEconomics)
Source: PharmacoEconomics - February 18, 2021 Category: Health Management Source Type: research

Macrovascular Risk Equations Based on the CANVAS Program
ConclusionThese risk equations address some of the limitations of widely used risk equations, such as the UKPDS-OM2, for modeling cardioprotective treatments for individuals with T2DM and high cardiovascular risk, including derivation from overly healthy patients treated with agents that lack cardioprotection and have been described as reflecting a different therapeutic era. Future work is needed to examine external validity. (Source: PharmacoEconomics)
Source: PharmacoEconomics - February 13, 2021 Category: Health Management Source Type: research

EQ-5D-Y Value Set for Slovenia
ConclusionsThe study addresses an important research gap and presents the EQ-5D-Y value set for Slovenia. At the time of writing, no published value sets are available for the EQ-5D-Y-3L appropriate for use in QALY calculations, making this value set the first EQ-5D-Y value set in the world. (Source: PharmacoEconomics)
Source: PharmacoEconomics - February 10, 2021 Category: Health Management Source Type: research

Global Economic Burden of Attention-Deficit/Hyperactivity Disorder: A Systematic Review
ConclusionsDespite the wide variation in methodologies in studies reviewed, the literature suggests that ADHD imposes a substantial economic burden on society. There is a dire need for cost-of-illness research in low- and middle-income countries to better inform the treatment and management of ADHD in these countries. In addition, guidelines on the conduct and reporting of economic burden studies are needed as they may improve standardisation of cost-of-illness studies. (Source: PharmacoEconomics)
Source: PharmacoEconomics - February 8, 2021 Category: Health Management Source Type: research

The Cost-Effectiveness and Budget Impact of Ibalizumab-uiyk for Adults with Multidrug-Resistant HIV-1 Infection in the United States
ConclusionsIbalizumab may represent a cost-effective and affordable option to improve health outcomes for individuals with MDR HIV-1 infection. (Source: PharmacoEconomics)
Source: PharmacoEconomics - February 3, 2021 Category: Health Management Source Type: research

The Excess Costs of Depression and the Influence of Sociodemographic and Socioeconomic Factors: Results from the German Health Interview and Examination Survey for Adults (DEGS)
ConclusionsDepression was associated with high excess costs that varied by social determinants. Considerable differences between the socioeconomic and sociodemographic subgroups need further clarification as they point to specific treatment barriers as well as varying treatment needs. (Source: PharmacoEconomics)
Source: PharmacoEconomics - February 1, 2021 Category: Health Management Source Type: research

Multiple Myeloma in Portugal: Burden of Disease and Cost of Illness
ConclusionsThe mortality rate in MM means that most DALYs are due to years of life lost rather than years lost due to disability. This study generates comprehensive data on the burden and cost of MM in Portugal and provides updated insights into the costs associated with the management of MM. (Source: PharmacoEconomics)
Source: PharmacoEconomics - January 31, 2021 Category: Health Management Source Type: research

Modifying NICE ’s Approach to Equity Weighting
AbstractThe UK ’s National Institute for Health and Care Excellence (NICE) recently launched a consultation on the methods it uses to evaluate new health technologies, and has highlighted the issue of how ‘modifiers’, including equity weights, should be incorporated into its processes. The practice of applyi ng equity weights to specific population subgroups, as a means for increasing the effective cost-effectiveness threshold for some new health technologies, is well established in health technology assessment. It is also the subject of extensive discussion in the academic literature. In this paper, we d...
Source: PharmacoEconomics - January 31, 2021 Category: Health Management Source Type: research

Cost-Effectiveness of Erenumab for the Preventive Treatment of Migraine in Patients with Prior Treatment Failures in Sweden
ConclusionsOur analysis suggests that erenumab is a cost-effective treatment for migraine with a willingness-to-pay threshold of SEK300,000 per QALY. (Source: PharmacoEconomics)
Source: PharmacoEconomics - January 25, 2021 Category: Health Management Source Type: research

Assessment of the Effects of Active Immunisation against Respiratory Syncytial Virus (RSV) using Decision-Analytic Models: A Systematic Review with a Focus on Vaccination Strategies, Modelling Methods and Input Data
AbstractBackgroundSeveral vaccine and antibody candidates are currently in development for the prevention of lower respiratory tract infections caused by the respiratory syncytial virus (RSV).MethodsWe searched MEDLINE, Embase, and SCOPUS and included model-based evaluations of RSV vaccinations. Two reviewers performed the selection, data extraction, and quality evaluation with EVIDEM. Cost-effectiveness (CE) estimates were converted to $US purchasing power parity (PPP), year 2018 values. Potential economic and epidemiological outcomes were summarised for maternal, infant, children, and elderly vaccinations. The PROSPERO i...
Source: PharmacoEconomics - January 19, 2021 Category: Health Management Source Type: research

NICE ’s Discounting Review: Clear Thinking on Rational Revision Meets Obstacle of Industrial Interests
AbstractThe National Institute of Health and Care Excellence (NICE) recently published a review of discounting practice and theory as part of a consultation on its current methods guidelines. The review examines the case for revision or retention of current methods. The changes considered include eliminating favourable rates in certain special cases and the reduction of the base-case rate for costs and health effects from 3.5 to 1.5%. The review also notes the potential need to reduce the cost-effectiveness threshold to accommodate a discount rate reduction, explaining that an agreement between the UK government and the ph...
Source: PharmacoEconomics - January 19, 2021 Category: Health Management Source Type: research

Early-Phase Clinical Trials and Reimbursement Submissions to the Pan-Canadian Oncology Drug Review
(Source: PharmacoEconomics)
Source: PharmacoEconomics - January 19, 2021 Category: Health Management Source Type: research

What Did Time Tell Us? A Comparison and Retrospective Validation of Different Survival Extrapolation Methods for Immuno-Oncologic Therapy in Advanced or Metastatic Renal Cell Carcinoma
ConclusionsThis aRCC case study identified marked differences in the predictive accuracy of survival extrapolation methods for nivolumab but less so for everolimus. The dependent log-logistic model did not suffer from overfitting to early DBLs to the same extent as more complex methods. Methods that provide more degrees of freedom may accurately represent survival for IO therapy, particularly if data are more mature or external data are available to inform the long-term extrapolations. (Source: PharmacoEconomics)
Source: PharmacoEconomics - January 11, 2021 Category: Health Management Source Type: research

Productivity-Adjusted Life-Years: A New Metric for Quantifying Disease Burden
(Source: PharmacoEconomics)
Source: PharmacoEconomics - January 11, 2021 Category: Health Management Source Type: research

Cost-Utility Analysis of a Dolutegravir-Based Versus Low-Dose Efavirenz-Based Regimen for the Initial Treatment of HIV-Infected Patients in Cameroon (NAMSAL ANRS 12313 Trial)
ConclusionsAt 2020 antiretroviral drug prices, a DTG-based first-line regimen should be preferred over an EFV400-based regimen in sub-Saharan Africa.Trial RegistrationClinicalTrials.gov Identifier: NCT02777229. (Source: PharmacoEconomics)
Source: PharmacoEconomics - December 23, 2020 Category: Health Management Source Type: research

Common Problems, Common Data Model Solutions: Evidence Generation for Health Technology Assessment
AbstractThere is growing interest in using observational data to assess the safety, effectiveness, and cost effectiveness of medical technologies, but operational, technical, and methodological challenges limit its more widespread use. Common data models and federated data networks offer a potential solution to many of these problems. The open-source Observational and Medical Outcomes Partnerships (OMOP) common data model standardises the structure, format, and terminologies of otherwise disparate datasets, enabling the execution of common analytical code across a federated data network in which only code and aggregate res...
Source: PharmacoEconomics - December 18, 2020 Category: Health Management Source Type: research

The Application and Implications of Novel Deterministic Sensitivity Analysis Methods
AbstractDeterministic sensitivity analyses (DSA) remain important to interpret the effect of uncertainties in individual parameters on results of cost-effectiveness analyses. Classic DSA methodologies may lead to wrong conclusions due to a lack of or misleading information regarding marginal effects, non-linearity, likelihood and correlations. In addition, tornado diagrams are misleading in some situations. Recent advances in DSA methods have the potential to provide decision makers with more reliable information regarding the effects of uncertainties in individual parameters. This practical application discusses advances ...
Source: PharmacoEconomics - December 14, 2020 Category: Health Management Source Type: research

Health State Utilities of Patients with Heart Failure: A Systematic Literature Review
ConclusionsThere is a wealth of published utility values for heart failure to support economic evaluations. Data are heterogenous owing to specificities of the study population and methodology of utility value elicitation and analysis. Choice of value(s) to support economic models must be carefully justified to ensure a robust economic analysis. (Source: PharmacoEconomics)
Source: PharmacoEconomics - November 30, 2020 Category: Health Management Source Type: research

Comment on “External Validation of the Core Obesity Model to Assess the Cost-Effectiveness of Weight Management Interventions”
(Source: PharmacoEconomics)
Source: PharmacoEconomics - November 30, 2020 Category: Health Management Source Type: research

Authors ’ reply to Comment on “External Validation of the Core Obesity Model to Assess the Cost-Effectiveness of Weight Management Interventions”
(Source: PharmacoEconomics)
Source: PharmacoEconomics - November 30, 2020 Category: Health Management Source Type: research

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