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 4, 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 31, 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 31, 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 30, 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 26, 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 22, 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 13, 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 28, 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 26, 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 26, 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 25, 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 23, 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 15, 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 12, 2020 Category: Health Management Source Type: research

Real World Cost-of-Illness Evidence in Hepatitis C Virus: A systematic review
ConclusionCost estimates for formal, informal, and non-health care services were identified in this review, but several challenges still exist in fully quantifying HCV burden. Future modeling studies including cost inputs should critically evaluate the risk of bias based on costing methods and data sources. (Source: PharmacoEconomics)
Source: PharmacoEconomics - June 12, 2020 Category: Health Management Source Type: research

Cost-Utility Analysis of Ravulizumab Compared with Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria
ConclusionsIn adults with paroxysmal nocturnal hemoglobinuria, ravulizumab is associated with improved health-related quality of life and provides a large cost saving from the perspective of a US payer, when compared with eculizumab. (Source: PharmacoEconomics)
Source: PharmacoEconomics - June 10, 2020 Category: Health Management Source Type: research

Cannabidiol for Adjuvant Treatment of Seizures Associated with Lennox –Gastaut Syndrome and Dravet Syndrome: An Evidence Review Group Perspective of a NICE Single Technology Appraisal
AbstractGW Research Ltd. provided two separate, but similar, submissions to the National Institute for Health and Care Excellence (NICE) on the clinical and cost-effectiveness of cannabidiol (CBD) 10  mg/kg/day, trade name Epidyolex®, for the adjunctive treatment of seizures associated with Lennox –Gastaut syndrome (LGS) and Dravet syndrome (DS). This paper highlights important methodological issues related to the company submissions, the Evidence Review Group (ERG) reports, and the subsequent development of the NICE guidance by the Appraisal Committee (AC) for the use of CBD. The company i dentified four ra...
Source: PharmacoEconomics - June 9, 2020 Category: Health Management Source Type: research

Future of Data Analytics in the Era of the General Data Protection Regulation in Europe
AbstractThe development of evidence to demonstrate ‘value for money’ is regarded as an important step in facilitating the search for the optimal allocation of limited resources and has become an essential component in healthcare decision making. Real-world evidence collected from de-identified individuals throughout the continuum of healthcare r epresents the most valuable source in technology evaluation. However, in the European Union, the value assessment based on real-world data has become challenging as individuals have recently been given the right to have their personal data erased in the case of consent ...
Source: PharmacoEconomics - June 8, 2020 Category: Health Management Source Type: research

Correction to: A Systematic Review of Methodological Variation in Healthcare Provider Perspective Tuberculosis Costing Papers Conducted in Low- and Middle-Income Settings, Using An Intervention-Standardised Unit Cost Typology
In the original version of this article, Fig.  3 was published in an incorrect format. The correct figure is published with this correction. (Source: PharmacoEconomics)
Source: PharmacoEconomics - May 27, 2020 Category: Health Management Source Type: research

Financing Drug Innovation in the US: Current Framework and Emerging Challenges
AbstractThe current US drug innovation financing framework rests on the notion that a defined period of marketing exclusivity combined with the expectation of reimbursement for clinically valuable, cost-effective therapies, followed by vigorous price competition from generic drugs and biosimilars ensures a sufficient return on investment (ROI) to incent private sector risk-based investment and research and development activities while providing access for new treatments to patients. While periodically, alternatives such as government prizes, direct purchases or development, and limits on certain incentives have been propos...
Source: PharmacoEconomics - May 26, 2020 Category: Health Management Source Type: research

What Value Does the NCCN Affordability Rating Really Provide to Patients, Providers, and Society?
(Source: PharmacoEconomics)
Source: PharmacoEconomics - May 15, 2020 Category: Health Management Source Type: research

Economic Evaluation of Cannabinoid Oil for Dravet Syndrome: A Cost-Utility Analysis
ConclusionCompared with clobazam/valproate, adjunctive cannabinoid oil may be a cost-effective treatment for Dravet syndrome, if a decision maker is willing to pay at least $32,399 for each QALY gained. The opportunity costs of continuing to fund stiripentol, but not cannabinoid oil, should be considered. (Source: PharmacoEconomics)
Source: PharmacoEconomics - May 14, 2020 Category: Health Management Source Type: research

Correction to: Real-World Evidence on the Patterns of Increased Societal Economic Relapse Costs in Patients with Multiple Sclerosis
In the original version of this article, the title of the article has been published incorrectly. (Source: PharmacoEconomics)
Source: PharmacoEconomics - May 13, 2020 Category: Health Management Source Type: research

Comparing the Cohort and Micro-Simulation Modeling Approaches in Cost-Effectiveness Modeling of Type 2 Diabetes Mellitus: A Case Study of the IHE Diabetes Cohort Model and the Economics and Health Outcomes Model of T2DM
ConclusionsThere was little evidence that estimated cost-effectiveness metrics, the outcomes that matter most to stakeholders, differed systematically. (Source: PharmacoEconomics)
Source: PharmacoEconomics - May 13, 2020 Category: Health Management Source Type: research

Health State Utility Values in Juvenile Idiopathic Arthritis: What is the Evidence?
ConclusionsWe have identified critical gaps in the literature reporting HSUV in patients with juvenile idiopathic arthritis including a lack of HSUV measures for distinct health states, particularly in adults with a history of juvenile idiopathic arthritis. The current reported HSUV data in juvenile idiopathic arthritis are insufficient for a full cost-utility analysis with a short or lifetime horizon. (Source: PharmacoEconomics)
Source: PharmacoEconomics - May 11, 2020 Category: Health Management Source Type: research

Calculating and Interpreting ICERs and Net Benefit
AbstractFor several decades, the incremental cost-effectiveness ratio has been routinely used by health technology assessment agencies around the world to summarise the results of economic evaluations of health interventions. Yet reporting and considering incremental cost-effectiveness ratios is unnecessary. Alternative summary measures exist, based on the concept of ‘net benefit’. The incremental cost-effectiveness ratio and measures of net benefit share several commonalities but some important distinctions. As a result, different methods are required to calculate and interpret incremental cost-effectiveness r...
Source: PharmacoEconomics - May 11, 2020 Category: Health Management Source Type: research

Why it ’s Time to Abandon the ICER
(Source: PharmacoEconomics)
Source: PharmacoEconomics - May 11, 2020 Category: Health Management Source Type: research

Budget Impact of 12-Month Fixed Treatment Duration Venetoclax in Combination with Obinutuzumab in Previously Untreated Chronic Lymphocytic Leukemia Patients in the United States
ConclusionsIntroducing VEN+G among first-line CLL treatments to a US health plan resulted in cost savings compared to a plan with chemoimmunotherapies and Ibr-based therapies only. Economic benefits of VEN+G, a novel agent with fixed treatment duration, coupled with proven clinical benefits should help inform formulary adoption decisions and treatment recommendations. (Source: PharmacoEconomics)
Source: PharmacoEconomics - May 8, 2020 Category: Health Management Source Type: research

Real-World Evidence on the Societal Economic Relapse Costs in Patients with Multiple Sclerosis
AbstractBackgroundRelapses are the hallmark of multiple sclerosis (MS). Analyses have shown that the cost of MS increases during periods of relapse. However, results are inconsistent between studies, possibly due to different study designs and the different implications of relapses with respect to patient characteristics.ObjectivesThe aims were to estimate and describe direct and indirect relapse costs and to determine differences in costs with respect to patient characteristics. Furthermore, we describe the pharmacoeconomic impact during the relapse follow-up.MethodsData were extracted from two German, multicenter, observ...
Source: PharmacoEconomics - May 4, 2020 Category: Health Management Source Type: research

A Systematic Review of Methodological Variation in Healthcare Provider Perspective Tuberculosis Costing Papers Conducted in Low- and Middle-Income Settings, Using An Intervention-Standardised Unit Cost Typology
ConclusionVariability in the methods and reporting used makes comparison difficult and makes it hard for decision makers to know which unit costs they can trust. The study quality rating system used in this review as well as the classification tree enable focus on specific reporting aspects that should improve variability and increase confidence in unit costs. Researchers should endeavour to be explicit and transparent in how they cost interventions following the principles as laid out in the Global Health Cost Consortium ’s Reference Case for Estimating the Costs of Global Health Services and Interventions, which in...
Source: PharmacoEconomics - May 4, 2020 Category: Health Management Source Type: research

Economic Costs of Myasthenia Gravis: A Systematic Review
ConclusionsWe show that the current body of literature of costs of MG is sparse, limited to a few geographical settings and resource categories, mostly dated, and subject to non-trivial variability, both within and between countries. Our synthesis will help researchers and decision-makers identify gaps in the local health economic context of MG and inform future cost studies and economic evaluations in this patient population. (Source: PharmacoEconomics)
Source: PharmacoEconomics - May 4, 2020 Category: Health Management Source Type: research

Real-World Evidence on the Patterns of Increased Societal Economic Relapse Costs in Patients with Multiple Sclerosis
AbstractBackgroundRelapses are the hallmark of multiple sclerosis (MS). Analyses have shown that the cost of MS increases during periods of relapse. However, results are inconsistent between studies, possibly due to different study designs and the different implications of relapses with respect to patient characteristics.ObjectivesThe aims were to estimate and describe direct and indirect relapse costs and to determine differences in costs with respect to patient characteristics. Furthermore, we describe the pharmacoeconomic impact during the relapse follow-up.MethodsData were extracted from two German, multicenter, observ...
Source: PharmacoEconomics - May 4, 2020 Category: Health Management Source Type: research

Adherence to Discounting Guidelines: Evidence from Over 2000 Published Cost-Effectiveness Analyses
AbstractPrevious studies have shown that not all cost-effectiveness analyses (CEAs) adhere to recommended guidelines on intertemporal discounting. This analysis investigates adherence in a sample of over 2000 CEAs from seven countries. Guideline discount rates were retrieved for Australia, Belgium, Canada, Ireland, The Netherlands, New Zealand and the UK. Data on the rates applied in published CEAs were retrieved from the Tufts CEA Registry from the sample countries within the periods covered by the discounting guidelines. The relationship between adherence and candidate explanatory factors were assessed using logistic reg...
Source: PharmacoEconomics - April 28, 2020 Category: Health Management Source Type: research

Replacing the NCCN ’s Blocks with Wheels: How Should Consideration of Societal Spending be Incorporated into Oncology Practice?
(Source: PharmacoEconomics)
Source: PharmacoEconomics - April 24, 2020 Category: Health Management Source Type: research

Keeping Pace with Pharmaceutical Innovation: The Importance of the NICE Methods Review
(Source: PharmacoEconomics)
Source: PharmacoEconomics - April 24, 2020 Category: Health Management Source Type: research

Health Economic Models for Metastatic Colorectal Cancer: A Methodological Review
ConclusionsModel-based cost-effectiveness analyses of systemic mCRC treatments have adopted varied modelling methods and structures, resulting in substantially different outcomes. As models generally focus on first-line treatment without consideration of downstream treatments, there is a profound source of structural uncertainty implying that the cost-effectiveness of treatments across the mCRC pathway remains uncertain. (Source: PharmacoEconomics)
Source: PharmacoEconomics - April 22, 2020 Category: Health Management Source Type: research

Valuing the AD-5D Dementia Utility Instrument: An Estimation of a General Population Tariff
ConclusionThe DCE-based utility value set is now available to use to generate QALYs for the economic evaluation of treatments and interventions targeting people with dementia and/or their family caregivers. (Source: PharmacoEconomics)
Source: PharmacoEconomics - April 21, 2020 Category: Health Management Source Type: research

Experience-Based Swedish TTO and VAS Value Sets for EQ-5D-5L Health States
ConclusionsTime trade-off and visual analogue scale value sets for the EQ-5D-5L are now available for Sweden. The time trade-off value set is the first such value set based on experience-based time  trade-off valuation. For decision makers with a preference for experience-based valuations of health states from a representative population-based sample, the reported value sets may be considered fit for purpose to support resource allocation decision as well as evaluating population health and h ealthcare performance. (Source: PharmacoEconomics)
Source: PharmacoEconomics - April 20, 2020 Category: Health Management Source Type: research

International Valuation Protocol for the EQ-5D-Y-3L
AbstractThe EQ-5D-Y-3L is a generic, health-related, quality-of-life instrument for use in younger populations. Some methodological studies have explored the valuation of children ’s EQ-5D-Y-3L health states. There are currently no published value sets available for the EQ-5D-Y-3L that are appropriate for use in a cost-utility analysis. The aim of this article was to describe the development of the valuation protocol for the EQ-5D-Y-3L instrument. There were several researc h questions that needed to be answered to develop a valuation protocol for EQ-5D-Y-3L health states. Most important of these were: (1) Do we need...
Source: PharmacoEconomics - April 16, 2020 Category: Health Management Source Type: research

Conducting Value for Money Analyses for Non-randomised Interventional Studies Including Service Evaluations: An Educational Review with Recommendations
This article provides an educational review covering the consideration of  conducting ‘value for money’ analyses as part of non-randomised study designs including service evaluations. These evaluations represent a vehicle for producing evidence such as value for money of a care intervention or service delivery model. Decision makers including charities and local and national governing bodies often rely on evidence from non-randomised data and service evaluations to inform their resource allocation decision-making. However, as randomised data obtained from randomised controlled trials are considered the &ls...
Source: PharmacoEconomics - April 15, 2020 Category: Health Management Source Type: research

Estimating the Value of New Antimicrobials in the Context of Antimicrobial Resistance: Development and Application of a Dynamic Disease Transmission Model
ConclusionsThe development of new antibiotics and the appropriate use of existing antibiotics are key to addressing the threat of AMR. This study presents a validated model that quantifies the value of new antibiotics through clinical and economic outcomes of relevance, and accounts for disease transmission of infection and development of AMR. In this context, the model may be a useful tool that could contribute to the decision-making process alongside other potential models and expert advice. (Source: PharmacoEconomics)
Source: PharmacoEconomics - April 6, 2020 Category: Health Management Source Type: research

Comment on ‘Fluocinolone Acetonide Intravitreal Implant for Treating Recurrent Non-infectious Uveitis: An Evidence Review Group Perspective of a NICE Single Technology Appraisal’
(Source: PharmacoEconomics)
Source: PharmacoEconomics - April 6, 2020 Category: Health Management Source Type: research

Response to Comment on “Fluocinolone Acetonide Intravitreal Implant for Treating Recurrent Non-Infectious Uveitis: An Evidence Review Group Perspective of a NICE Single Technology Appraisal”
(Source: PharmacoEconomics)
Source: PharmacoEconomics - April 6, 2020 Category: Health Management Source Type: research

Cost-Effectiveness Analysis of Patiromer in Combination with Renin –Angiotensin–Aldosterone System Inhibitors for Chronic Kidney Disease in Sweden
ConclusionsThe results indicate that patiromer may demonstrate value for money in Swedish patients with stage 3 –4 CKD, by enabling RAASi treatment. However, there is a considerable degree of uncertainty. (Source: PharmacoEconomics)
Source: PharmacoEconomics - April 2, 2020 Category: Health Management Source Type: research

Examining Approaches to Estimate the Prevalence of Catastrophic Costs Due to Tuberculosis from Small-Scale Studies in South Africa
ConclusionsThis paper presents a novel application of existing data to estimate the national prevalence of catastrophic costs due to TB. This type of model could be useful for researchers and policy makers looking to inform certain policy decisions; however, some uncertainties remain due to limitations in data availability. There is an urgent need for standardized reporting of cost data and improved guidance on methods to collect income data to improve these estimates going forward. (Source: PharmacoEconomics)
Source: PharmacoEconomics - April 1, 2020 Category: Health Management Source Type: research

R and Shiny for Cost-Effectiveness Analyses: Why and When? A Hypothetical Case Study
ConclusionsThe findings of this explicit comparison are used to produce recommendations for when R might be more suitable than Excel in contemporary cost-effectiveness analyses. We conclude that selection of appropriate modelling software needs to consider case-by-case modelling requirements, particularly (i) intended audience, (ii) complexity of analysis, (iii) nature and frequency of updates and (iv) anticipated model run time. (Source: PharmacoEconomics)
Source: PharmacoEconomics - March 31, 2020 Category: Health Management Source Type: research

The Accuracy and Usefulness of the National Comprehensive Cancer Network Evidence Blocks Affordability Rating
ConclusionsEvidence Blocks Affordability generally correlated with treatment costs but contained discrepancies, which may limit its usefulness to clinicians in comparing costs. This study suggests that the Affordability score may be improved by indexing more directly to specified dollar value thresholds. (Source: PharmacoEconomics)
Source: PharmacoEconomics - March 23, 2020 Category: Health Management Source Type: research

Development of Population Tariffs for the CarerQol Instrument for Hungary, Poland and Slovenia: A Discrete Choice Experiment Study to Measure the Burden of Informal Caregiving
ConclusionFor the first time in the Central and Eastern European region, country-specific tariffs are now available for the Hungarian, Polish and Slovenian versions of the CarerQol instrument. This facilitates inclusion of the impact of informal care in economic evaluations. Our results can be used to develop and evaluate country-specific health policy strategies to support informal caregivers. The differences found in informal care preferences highlight the limited transferability of CarerQol tariffs across European regions. (Source: PharmacoEconomics)
Source: PharmacoEconomics - March 23, 2020 Category: Health Management Source Type: research

A Comparison of the EQ-5D-3L and EQ-5D-5L
ConclusionThe 5L had superior measurement properties than the 3L and should be preferred in general population surveys and for use in individuals with multimorbidity. At increasing levels of morbidity, the 5L is currently associated with higher utility values than the 3L. (Source: PharmacoEconomics)
Source: PharmacoEconomics - March 17, 2020 Category: Health Management Source Type: research

A Budget Impact Model of the Addition of Telotristat Ethyl Treatment to the Standard of Care in Patients with Uncontrolled Carcinoid Syndrome
ConclusionsThe expected budget impact of adding telotristat ethyl to the standard of care in Sweden was relatively low, largely because of the rarity of carcinoid syndrome. (Source: PharmacoEconomics)
Source: PharmacoEconomics - March 11, 2020 Category: Health Management Source Type: research