Evaluating Value-Based Frameworks Used for Relapsed or Refractory Multiple Myeloma Regimens: ICER Report, ASCO Value Framework, and NCCN Evidence Blocks

BACKGROUND: With the continuous rise in costs for oncology drugs, the Institute for Clinical and Economic Review (ICER), the American Society of Clinical Oncology (ASCO) and the National Comprehensive Cancer Network (NCCN) have developed value-based frameworks (VBFs) to assist stakeholders in formulary and treatment decisionmaking. While emerging VBFs have the potential to significantly impact therapeutic options for patients, it is important to understand the differences associated with those VBFs within a therapeutic area. OBJECTIVE: To compare ICER, ASCO, and NCCN VBFs across three therapeutic options for relapsed or refractory multiple myeloma (RRMM). METHODS: The values of carfilzomib (CFZ), elotuzumab (ELO), and ixazomib (IX) were generated using ICER, ASCO, and NCCN VBFs. Those regimens, used for second or third line treatment of RRMM, were chosen because they share a common comparator in clinical trials, lenalidomide + dexamethasone (LEN + DEX). The ICER 2016 report of treatment options for RRMM was used to obtain results of the comparative clinical effectiveness and the cost effectiveness analysis for those regimens compared to LEN + DEX. ASCO’s 2016 VBF, which incorporates clinical benefit, toxicity and bonus points was used to generate a net health benefit (NHB) score without a scale along with the drug wholesale acquisition cost (WAC) for each regimen compared to LEN + DEX. The NCCN VBF uses a score ranging from 1 to 5, with 1 as the least favorable and 5 as the most favorable, for each of five evidence blocks: efficacy, safety, quality, consistency, and affordability. The 2016 Multiple Myeloma NCCN evidence blocks was used to obtain the value of CFZ, ELO, and IX. RESULTS: The ICER VBF suggested with moderate certainty that CFZ, ELO, and IX provide a better NHB in patients with RRMM compared to LEN + DEX. Second-line and third-line treatment costs per QALY for CFZ, ELO, and IX were $199,982,$427,607 and $433,794, and$238,560, $481,244, and$484,582, respectively. The ASCO VBF generated a total NHB of 28.8, 23.7 and 23.0 with a monthly WAC of $17,364,$16,032 and $20,607 for CFZ, ELO, and IX, respectively. The monthly cost of LEN + DEX was$11,616. The NCCN VBF had an efficacy score of 5, 3, and 4 for CFZ, ELO, IX, respectively. Safety, quality, consistency, and affordability scores of 3, 4, 4, and 1, respectively, were the same across regimens. CONCLUSIONS: ICER, ASCO and NCCN VBFs suggest CFZ may be the most valued treatment out of the three regimens. However, their applicability in stakeholder’s decision-making remains unclear due to uncertainty and challenges associated with them. SPONSORSHIP: None.https://jdc.jefferson.edu/jcphposters/1012/thumbnail.jp

Towards a transparent, credible, evidence-based decision-making process of new drug listing on the Hong Kong Hospital Authority Drug Formulary: challenges and suggestions

The aim of this article is to describe the process, evaluation criteria, and possible outcomes of decision-making for new drugs listed in the Hong Kong Hospital Authority Drug Formulary in comparison to the health technology assessment (HTA) policy overseas. Details of decision-making processes including the new drug listing submission, Drug Advisory Committee (DAC) meeting, and procedures prior to and following the meeting, were extracted from the official Hong Kong Hospital Authority drug formulary management website and manual. Publicly-available information related to the new drug decision-making process for five HTA agencies [the National Institute of Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC), the Australia Pharmaceutical Benefits Advisory Committee (PBAC), the Canadian Agency for Drugs and Technologies in Health (CADTH), and the New Zealand Pharmaceutical Management Agency (PHARMAC)] were reviewed and retrieved from official documents from public domains. The DAC is in charge of systemically and critically appraising new drugs before they are listed on the formulary, reviewing submitted applications, and making the decision to list the drug based on scientific evidence to which safety, efficacy, and cost-effectiveness are the primary considerations. When compared with other HTA agencies, transparency of the decision-making process of the DAC, the relevance of clinical and health economic evidence, and the lack of health economic and methodological input of submissions are the major challenges to the new-drug listing policy in Hong Kong. Despite these challenges, this review provides suggestions for the establishment of a more transparent, credible, and evidence-based decision-making process in the Hong Kong Hospital Authority Drug Formulary. Proposals for improvement in the listing of new drugs in the formulary should be a priority of healthcare reforms

Delivering diabetes education through nurseled telecoaching : cost-effectiveness analysis

Background : People with diabetes have a high risk of developing micro-and macrovascular complications associated with diminished life expectancy and elevated treatment costs. Patient education programs can improve diabetes control in the short term, but their cost-effectiveness is uncertain. Our study aimed to analyze the lifelong cost-effectiveness of a nurse-led tele-coaching program compared to usual care in people with type 2 diabetes from the perspective of the Belgian healthcare system. Methods : The UKPDS Outcomes Model was populated with patient-level data from an 18-month randomized clinical trial in the Belgian primary care sector involving 574 participants; trial data were extrapolated to 40 years; Quality Adjusted Life Years (QALYs), treatment costs and Incremental Cost-Effectiveness Ratio (ICER) were calculated for the entire cohort and the subgroup with poor glycemic control at baseline ("elevated HbA1c subgroup") and the associated uncertainty was explored. Results : The cumulative mean QALY (95% CI) gain was 0.21 (0.13; 0.28) overall and 0.56 (0.43; 0.68) in elevated HbA1c subgroup; the respective incremental costs were (sic)1,147 (188; 2,107) and (sic)2,565 (654; 4,474) and the respective ICERs (sic)5,569 ((sic)677; (sic)15,679) and (sic)4,615 (1,207; 9,969) per QALY. In the scenario analysis, repeating the intervention for lifetime had the greatest impact on the cost-effectiveness and resulted in the mean ICERs of (sic) 13,034 in the entire cohort and (sic)7,858 in the elevated HbA1c subgroup. Conclusion : Taking into account reimbursement thresholds applied in West-European countries, nurseled telecoaching of people with type 2 diabetes may be considered highly cost-effective within the Belgian healthcare system

Cost effectiveness of school-located influenza vaccination programs for elementary and secondary school children.

BackgroundStudies have noted variations in the cost-effectiveness of school-located influenza vaccination (SLIV), but little is known about how SLIV's cost-effectiveness may vary by targeted age group (e.g., elementary or secondary school students), or vaccine consent process (paper-based or web-based). Further, SLIV's cost-effectiveness may be impacted by its spillover effect on practice-based vaccination; prior studies have not addressed this issue.MethodsWe performed a cost-effectiveness analysis on two SLIV programs in upstate New York in 2015-2016: (a) elementary school SLIV using a stepped wedge design with schools as clusters (24 suburban and 18 urban schools) and (b) secondary school SLIV using a cluster randomized trial (16 suburban and 4 urban schools). The cost-per-additionally-vaccinated child (i.e., incremental cost-effectiveness ratio (ICER)) was estimated by dividing the incremental SLIV intervention cost by the incremental effectiveness (i.e., the additional number of vaccinated students in intervention schools compared to control schools). We performed deterministic analyses, one-way sensitivity analyses, and probabilistic analyses.ResultsThe overall effectiveness measure (proportion of children vaccinated) was 5.7 and 5.5 percentage points higher, respectively, in intervention elementary (52.8%) and secondary schools (48.2%) than grade-matched control schools. SLIV programs vaccinated a small proportion of children in intervention elementary (5.2%) and secondary schools (2.5%). In elementary and secondary schools, the ICER excluding vaccine purchase was $85.71 and$86.51 per-additionally-vaccinated-child, respectively. When additionally accounting for observed spillover impact on practice-based vaccination, the ICER decreased to $80.53 in elementary schools -- decreasing substantially in secondary schools. (to$53.40). These estimates were higher than the published practice-based vaccination cost (median = $25.50, mean =$45.48). Also, these estimates were higher than our 2009-2011 urban SLIV program mean costs ($65) due to additional costs for use of a new web-based consent system ($12.97 per-additionally-vaccinated-child) and higher project coordination costs in 2015-2016. One-way sensitivity analyses showed that ICER estimates were most sensitive to the SLIV effectiveness.ConclusionsSLIV raises vaccination rates and may increase practice-based vaccination in primary care practices. While these SLIV programs are effective, to be as cost-effective as practice-based vaccination our SLIV programs would need to vaccinate more students and/or lower the costs for consent systems and project coordination.Trial registrationClinicalTrials.gov NCT02227186 (August 25, 2014), updated NCT03137667 (May 2, 2017)

Cost-effectiveness of a potential Zika vaccine candidate: a case study for Colombia

Background: A number of Zika vaccine platforms are currently being investigated, some of which have entered clinical trials. We sought to evaluate the cost-effectiveness of a potential Zika vaccine candidate under the WHO Vaccine Target Product Profile for outbreak response, prioritizing women of reproductive age to prevent microcephaly and other neurological disorders. Methods: Using an agent-based simulation model of ZIKV transmission dynamics in a Colombian population setting, we conducted cost-effectiveness analysis with and without pre-existing herd immunity. The model was parameterized with estimates associated with ZIKV infection, risks of microcephaly in different trimesters, direct medical costs, and vaccination costs. We assumed that a single dose of vaccine provides a protection efficacy in the range 60% to 90% against infection. Cost-effectiveness analysis was conducted from a government perspective. Results: Under a favorable scenario when the reproduction number is relatively low (R0 = 2.2) and the relative transmissibility of asymptomatic infection is 10% compared with symptomatic infection, a vaccine is cost-saving (with negative incremental cost-effective ratio; ICER) for vaccination costs up to US$6 per individual without herd immunity, and up to US$4 per individual with 8% herd immunity. For positive ICER values, vaccination is highly cost-effective for vaccination costs up to US$10 (US$7) in the respective scenarios with the willingness-to-pay of US$6610 per disability-adjusted life-year, corresponding to the average per capita GDP of Colombia between 2013 and 2017. Our results indicate that the effect of other control measures targeted to reduce ZIKV transmission decreases the range of vaccination costs for cost-effectiveness due to reduced returns of vaccine-induced herd immunity. In all scenarios investigated, the median reduction of microcephaly exceeded 64% with vaccination. Conclusions: Our study suggests that a Zika vaccine with protection efficacy as low as 60% could significantly reduce the incidence of microcephaly. From a government perspective, Zika vaccination is highly cost-effective, and even cost-saving in Colombia if vaccination costs per individual is sufficiently low. Efficacy data from clinical trials and number of vaccine doses will be important requirements in future studies to refine our estimates, and conduct similar studies in other at-risk populations. Keywords: Zika, Microcephaly, Vaccination, Agent-based modeling, Cost-effectivenessYork University Librarie

Is increased hepatitis C virus case-finding combined with current or 8-week to 12-week direct-acting antiviral therapy cost-effective in UK prisons? A prevention benefit analysis

UNLABELLED: Prisoners have a high prevalence of hepatitis C virus (HCV), but case-finding may not have been cost-effective because treatment often exceeded average prison stay combined with a lack of continuity of care. We assessed the cost-effectiveness of increased HCV case-finding and treatment in UK prisons using short-course therapies. A dynamic HCV transmission model assesses the cost-effectiveness of doubling HCV case-finding (achieved through introducing opt-out HCV testing in UK pilot prisons) and increasing treatment in UK prisons compared to status quo voluntary risk-based testing (6% prison entrants/year), using currently recommended therapies (8-24 weeks) or interferon (IFN)-free direct-acting antivirals (DAAs; 8-12 weeks, 95% sustained virological response, £3300/week). Costs (British pounds, £) and health utilities (quality-adjusted life years) were used to calculate mean incremental cost-effectiveness ratios (ICERs). We assumed 56% referral and 2.5%/25% of referred people who inject drugs (PWID)/ex-PWID treated within 2 months of diagnosis in prison. PWID and ex-PWID or non-PWID are in prison an average 4 and 8 months, respectively. Doubling prison testing rates with existing treatments produces a mean ICER of £19,850/quality-adjusted life years gained compared to current testing/treatment and is 45% likely to be cost-effective under a £20,000 willingness-to-pay threshold. Switching to 8-week to 12-week IFN-free DAAs in prisons could increase cost-effectiveness (ICER £15,090/quality-adjusted life years gained). Excluding prevention benefit decreases cost-effectiveness. If >10% referred PWID are treated in prison (2.5% base case), either treatment could be highly cost-effective (ICER<£13,000). HCV case-finding and IFN-free DAAs could be highly cost-effective if DAA cost is 10% lower or with 8 weeks' duration. CONCLUSIONS: Increased HCV testing in UK prisons (such as through opt-out testing) is borderline cost-effective compared to status quo voluntary risk-based testing under a £20,000 willingness to pay with current treatments but likely to be cost-effective if short-course IFN-free DAAs are used and could be highly cost-effective if PWID treatment rates were increased. (Hepatology 2016;63:1796-1808)

Patient-controlled intravenous morphine analgesia combined with transcranial direct current stimulation for post-thoracotomy pain: A cost-effectiveness study and a feasibility for its future implementation

This prospective randomized study aims to evaluate the feasibility and cost-effectiveness of combining transcranial direct current stimulation (tDCS) with patient controlled intravenous morphine analgesia (PCA-IV) as part of multimodal analgesia after thoracotomy. Patients assigned to the active treatment group (a-tDCS

Cost effectiveness of cascade testing for familial hypercholesterolaemia, based on data from familial hypercholesterolaemia services in the UK

Peer reviewedPublisher PD

Economic evaluation of HBV vaccination: a systematic review of recent publications (2000-2013).

Aim: To conduct a systematic review of the economic evaluations (EE) of HBV vaccination, taking also into account the studies published in the new millennium. Methods: An extensive scientific literature review was conducted using two electronic medical journal databases: Scopus and PubMed engines for published studies on EE of HBV vaccination. Results: 22 articles were reviewed, 9, 5 and 8 cost-effectiveness, cost-benefit and cost-utility analysis, respectively. Studies were mainly concerning EE of universal vaccination (UV), mostly with regards to low or low-medium income countries. For high income countries, EE were focused on the possible implementation of HBV vaccination in particular settings, such as diabetic, renal and other chronic conditions care, as well as infectious diseasesUV has usually a very good cost-effectiveness ratio (80%), ranging from cost-saving (China) or few Euro per LY/QALY gained (in Thailand, and Vietnam) to 630.00$/ QALY in USA (Asian and Pacific Islands) Moreover, EE of HBV vaccination are favorable in the infectious diseases field as well as for chronic conditions. In relation to diabetes the studies gave controversial results. Conclusion: This systematic review highlighted the importance of introducing HBV vaccination not only for infant UV program but also for other settings in which patients are people affected by communicable and non-communicable diseases

Introductory programming: a systematic literature review

As computing becomes a mainstream discipline embedded in the school curriculum and acts as an enabler for an increasing range of academic disciplines in higher education, the literature on introductory programming is growing. Although there have been several reviews that focus on specific aspects of introductory programming, there has been no broad overview of the literature exploring recent trends across the breadth of introductory programming. This paper is the report of an ITiCSE working group that conducted a systematic review in order to gain an overview of the introductory programming literature. Partitioning the literature into papers addressing the student, teaching, the curriculum, and assessment, we explore trends, highlight advances in knowledge over the past 15 years, and indicate possible directions for future research