Published Cost-effectiveness Models Research Articles

MSR215 Are 237 Published Cost-Effectiveness Models Necessary for Non-Small-Cell Lung Cancer (NSCLC)? Can Open Source Model Platforms Improve Decision-Making and Save Resources?

MSR215 Are 237 Published Cost-Effectiveness Models Necessary for Non-Small-Cell Lung Cancer (NSCLC)? Can Open Source Model Platforms Improve Decision-Making and Save Resources?

Perks and Pitfalls of Performance-Linked Reimbursement for Novel Drugs: The Case of Sacubitril-Valsartan.

Rising drug costs have increased interest in performance-linked reimbursement (PLR) contracts that tie payment to patient outcomes. PLR is theoretically attractive to payers interested in reducing the risk of overpaying for expensive drugs, to manufacturers working to improve early drug adoption, and to patients seeking improved access. Multiple PLR contracts were developed for sacubitril-valsartan. We evaluated how the characteristics of a PLR contract influence its performance. We used a published cost-effectiveness model of sacubitril-valsartan. We evaluated hypothetical PLR contracts that adjusted drug payment based on observed therapy effectiveness. Ideally, these contracts reduce the uncertainty around the value obtained with purchasing sacubitril-valsartan. By reducing the financial risk in covering an ineffective therapy, PLR incentivizes insurers to increase patient access. We measured the uncertainty in value as the SD of the incremental net monetary benefit (INMB), an estimate of therapy value incorporating costs and clinical benefits. We evaluated the change in INMB SD under a variety of different assumptions regarding contract design, therapy effectiveness, and population characteristics. Over 2 years, sacubitril-valsartan led to 0.042 additional quality-adjusted life-years at an incremental cost of $4916. Using a willingness-to-pay of $150 000 per quality-adjusted life-year, this led to a mean INMB across simulations of $1416 (SD, $1720). A PLR contract that adjusted payment based on cardiovascular mortality reduced the INMB SD moderately by 20.7% while a contract based on all-cause mortality was more effective (INMB SD reduction of 27.3%). A contract based on heart failure hospitalization reduction was ineffective. PLR effectiveness increased with greater uncertainty regarding therapy effectiveness or in sicker cohorts (eg, New York Heart Association Class III/IV heart failure). Contracts required precise estimates of treatment effect in addition to trust or verifiability between manufacturers and payers concerning patient selection. The development of accurate prospective estimates of treatment effectiveness using actual enrollee characteristics will be critical for successful PLR. If able to meet these requirements, PLRs could incentivize insurers to expand access to expensive treatments by reducing financial risk.

POSB67 Cost-of-Illness for Invasive Meningococcal Disease Caused By Serogroup B Neisseria Meningitidis (MENB) in the Netherlands

Invasive meningococcal disease (IMD) is a rapidly progressive, unpredictable, rare disease that can lead to meningitis and sepsis. IMD is caused by Neisseria meningitidis, affecting mostly infant and adolescent cohorts, where high case fatalities and serious long-term sequelae are prevalent. The prevalent type in Europe and the Netherlands is serotype B. Previously published cost-effectiveness models of IMD prevention often do not include all costs related to the sequelae and therefore strongly underestimate the economic burden.

The Cost-Effectiveness of 13-Valent Pneumococcal Conjugate Vaccine in Seven Chinese Cities.

Objective: This study estimates the cost-effectiveness of vaccination with the 13-valent pneumococcal conjugate vaccine (PCV13) among infants in Beijing, Shanghai, Shenzhen, Chengdu, Karamay, Qingdao, and Suzhou. Methods: A previously published cost-effectiveness model comparing vaccination with PCV13 to no vaccination was localized to the included Chinese cities. A systematic literature review was undertaken to identify age-specific incidence rates for pneumococcal bacteremia, pneumococcal meningitis, pneumonia, and otitis media (AOM). Age-specific direct medical costs of treating the included pneumococcal diseases were taken from the Chinese Health Insurance Association database. The base case analysis evaluated vaccine efficacy using direct effect and indirect effects (DE+ IDE). A subsequent scenario analysis evaluated the model outcomes if only DE was considered. A vaccination rate of 70% was used. The model reported outcomes over a one-year period after it was assumed the vaccine effects had reached a steady state (5–7 years after vaccine introduction) to include the direct and indirect effects of vaccination. Health outcomes were discounted at 5% during the steady-state period. Results: Vaccination with PCV13 was cost-effective in the base case analysis for all included cities with the incremental cost-effectiveness ratio (ICER) ranging from 1145 CNY(Shenzhen) to 15,422 CNY (Qingdao) per quality-adjusted life-year (QALY) gained. PCV13 was the dominant strategy in Shanghai with lower incremental costs and higher incremental QALYs. PCV13 remained cost-effective in the DE-only analysis with all ICERs falling below a cost-effectiveness threshold of three times GDP per capita in each city. Conclusions: Vaccination with PCV13 was a cost-effective strategy in the analyzed cities for both the DE-only and DE + IDE analyses. PCV13 became very cost-effective when a vaccination rate was reached where IDE is observed.

Handling Uncertainty in Cost-Effectiveness Analysis: Budget Impact and Risk Aversion

Methods to handle uncertainty in economic evaluation have gained much attention in the literature, and the cost-effectiveness acceptability curve (CEAC) is the most widely used method to summarise and present uncertainty associated with program costs and effects in cost-effectiveness analysis. Some researchers have emphasised the limitations of the CEAC for informing decision and policy makers, as the CEAC is insensitive to radial shifts of the joint distribution of incremental costs and effects in the North-East and South-West quadrants of the cost-effective plane (CEP). Furthermore, it has been pointed out that the CEAC does not incorporate risk-aversion in valuing uncertain costs and effects. In the present article, we show that the cost-effectiveness affordability curve (CEAFC) captures both dimensions of the joint distribution of incremental costs and effects on the CEP and is, therefore, sensitive to radial shifts of the joint distribution on the CEP. Furthermore, the CEAFC also informs about the budget impact of a new intervention, as it can be used to estimate the joint probability that an intervention is both affordable and cost-effective. Moreover, we show that the cost-effectiveness risk-aversion curve (CERAC) allows the analyst to incorporate different levels of risk-aversion into the analysis and can, therefore, be used to inform decision-makers who are risk-averse. We use data from a published cost-effectiveness model of palbociclib in addition to letrozole versus letrozole alone for the treatment of oestrogen-receptor positive, HER-2 negative, advanced breast cancer to demonstrate the differences between CEAC, CEAFC and CERAC, and show how these can jointly be used to inform decision and policy makers.

The cost-effectiveness of therapeutic drug monitoring for the prescription drug-based treatment of chronic myeloid leukemia.

BACKGROUND: A recent study demonstrating the use of Therapeutic Drug Monitoring (TDM) in patients with chronic myeloid leukemia (CML) resulted in a higher response rate with imatinib (IM) than demonstrated in second-generation tyrosine kinase inhibitor studies. The cost-effectiveness of TDM combined with IM (IM TDM) in first-line CML treatment has not yet been studied. OBJECTIVES: To determine the cost-effectiveness of IM TDM for the first-line treatment of CML compared to tyrosine kinase inhibitor only treatment. METHODS: A recently published cost-effectiveness model of tyrosine kinase inhibitor-treatment in CML was modified to include IM TDM as a first-line tyrosine kinase inhibitor-based CML treatment option. Efficacy inputs for major molecular response (MMR) rates were taken from previously published studies: IM TDM 65%, dasatinib 52%, nilotinib 53%. Annual tyrosine kinase inhibitor drug prices were derived from the Federal Supply Schedule (FSS) and the average and lowest wholesale acquisition costs (WAC) reported in the Red Book; the annual cost of TDM was $228. Other input costs modeled in the original CML CEA model were updated to 2016 US dollars using the medical service component of the Consumer Price Index. A US payer perspective was used with a 5-year time horizon and a 3.0% discount rate. The model compared first-line IM TDM versus IM alone, nilotinib (NIL) or dasatinib (DAS) in terms of the following outcomes: costs, quality-adjusted life-years (QALYs), and cost-effectiveness (total cost/QALY). Deterministic and probabilistic sensitivity analyses were performed using all key clinical and economic parameters. RESULTS: This study found that IM TDM dominates IM alone with $15,452 to $36,940 in savings and 0.25 higher QALYs. Using FSS, per patient total costs for IM and IM TDM were $270,905 and $233,965, respectively.; Using average WAC, these costs were $461,657 and $446,205, and using lowest WAC, these costs were $366,966 and $350,090. The results comparing first line using of IM TDM to NIL/DAS found that TDM IM had higher QALYs and lower costs (0.08 QALYs lower, and $117,006 to $172,420 savings per patient [varying by price basis]). Thus, in terms of cost-effectiveness, IM TDM dominates NIL/DAS with both lower costs and higher QALYs. CONCLUSIONS: IM TDM is a clinically and economically viable first-line treatment option for CML. DISCLOSURES: This study was funded by Saladax Biomedical. Salamone is an employee of Saladax Biomedical. This study was presented at the IATDMCT Congress, September 2018, Brisbane, Australia.

996. The Potential for Reducing Opioid and Analgesic Prescriptions Via Herpes Zoster Vaccination

BackgroundHerpes zoster (HZ), or shingles, is a common neurocutaneous disease caused by the reactivation of latent varicella zoster virus that often includes rash and neuropathic pain that may last for months. Opioids and other analgesics may be prescribed. Recombinant zoster vaccine (RZV) is preferentially recommended for the prevention of HZ in adults aged 50 years and older. This study aimed to assess the impact of RZV vaccination on opioid and other analgesic prescription-related outcomes.MethodsEstimates of analgesic prescription rates (opioids, benzodiazepines, and other analgesics) among HZ cases were established using Truven claims data from 2012-2018 for adults aged 50 years and older. HZ case avoidance with RZV vaccination was calculated using a previously published cost-effectiveness model. This data was included in a calculator assessing the impact of RZV vaccination on analgesic prescription-related outcomes (compared to no vaccination).ResultsBetween 24.4% and 28.0% of HZ cases in the observed claims had at least one opioid prescription, dependent on age group (4.5%-6.5% and 8.6%-19.6% for benzodiazepines and other analgesics, respectively). The mean number of opioid prescriptions per person in each age group with at least one opioid prescription was between 1.7 and 1.9 (1.7-2.3 and 1.7-2.0 prescriptions for benzodiazepines and other analgesics, respectively). Assuming a 1-million-person population and 65% RZV coverage, the calculator predicts RZV vaccination will prevent 75,002 cases of HZ and will prevent 19,311 people from being prescribed at least 1 HZ-related opioid, 4,502 people from being prescribed benzodiazepines, and 12,201 people from being prescribed other analgesics. Additionally, 34,520 HZ-related opioid prescriptions will be avoided (9,413 benzodiazepine prescriptions; 22,406 other analgesic prescriptions).ConclusionHZ is associated with high levels of opioid, benzodiazepine, and other analgesic use. Primary prevention of HZ by vaccination could potentially reduce opioid and other medication exposure.DisclosuresJean-Etienne Poirrier, PhD, MBA, The GSK group of companies (Employee, Shareholder) Justin Carrico, BS, GlaxoSmithKline (Consultant) Jessica K. DeMartino, PhD, The GlaxoSmithKline group of companies (Employee, Shareholder) Katherine A. Hicks, MS, BSPH, GlaxoSmithKline (Scientific Research Study Investigator, GSK pays my company for my contractual services.) Saurabh P. Nagar, MS, RTI Health Solutions (Employee) Juliana Meyers, MA, GlaxoSmithKline (Other Financial or Material Support, This study was funded by GlaxoSmithKline.)

PIN12 THE COST-EFFECTIVENESS OF THE 13-VALENT PNEUMOCOCCAL CONJUGATE VACCINE IN CHINESE CITIES

The 13-valent pneumococcal conjugate vaccine (PCV13) offers protection against 13 pneumococcal serotypes, reducing the incidence of invasive pneumococcal disease (IPD), pneumonia, and otitis media (OM). This study estimates the cost-effectiveness of immunization of PCV13 in select Chinese cities. We localized a previously published cost-effectiveness model comparing PCV13 to no vaccination to Beijing, Shanghai, Suzhou, Chengdu, Karamay, Qingdao, and Shenzhen. A systematic literature review (SLR) through 10/11/2019 was undertaken to identify previously published PCV cost-effectiveness models in China. The SLR was utilized to identify sources for estimating the age-specific incidence of pneumococcal bacteremia, pneumococcal meningitis, pneumonia, and OM. Sources identified in the literature review were combined with data from the CHIRA database (2013-2015) to estimate incidence rates. Age-specific direct healthcare costs for each health state were sourced directly from the CHIRA database (2013-2015) and available for Tier 1 cities and nationally. The base case analysis considered a vaccination rate of 85%, herd effect, a 5% discount rate, and took a payer perspective. The number of IPD, pneumonia, and OM cases avoided due to PCV13 vaccination in each city ranged from 13 to 581, 1,039 to 43,769, and 174 to 7,112, respectively. Deaths avoided ranged 18 to 1,061 (1 to 29 for IPD and 17 to 1,032 for pneumonia), which resulted in 275 to 15,134 additional quality-adjusted life-years for PCV13 vaccination compared to no vaccination. The incremental total direct costs of the PCV13 vaccination program ranged between ¥5,462,884 and ¥156,224,157 in the included cities. The incremental cost-effectiveness ratio for PCV13 compared to no vaccination for each city was between ¥1,300 and ¥19,684 per QALY gained, below 1xGDP per capita of each city. PCV13 can be considered highly cost-effective when compared to no vaccination at a vaccination rate of 85% in Beijing, Shanghai, Suzhou, Chengdu, Karamay, Qingdao, and Shenzhen.

Comparative analyses of published cost effectiveness models highlight critical considerations which are useful to inform development of new models

Background: Comparative analyses of published cost effectiveness models provide useful insights into critical issues to inform the development of new cost effectiveness models in the same disease area.Objective: The purpose of this study was to describe a comparative analysis of cost-effectiveness models and highlight the importance of such work in informing development of new models. This research uses genotypic antiretroviral resistance testing after first line treatment failure for Human Immunodeficiency Virus (HIV) as an example.Method: A literature search was performed, and published cost effectiveness models were selected according to predetermined eligibility criteria. A comprehensive comparative analysis was undertaken for all aspects of the models.Results: Five published models were compared, and several critical issues were identified for consideration when developing a new model. These include the comparator, time horizon and scope of the model. In addition, the composite effect of drug resistance prevalence, antiretroviral therapy efficacy, test performance and the proportion of patients switching to second-line ART potentially have a measurable effect on model results. When considering CD4 count and viral load, dichotomizing patients according to higher cost and lower quality of life (AIDS) versus lower cost and higher quality of life (non-AIDS) status will potentially capture differences between resistance testing and other strategies, which could be confirmed by cross-validation/convergent validation. A quality adjusted life year is an essential outcome which should be explicitly explored in probabilistic sensitivity analysis, where possible.Conclusions: Using an example of GART for HIV, this study demonstrates comparative analysis of previously published cost effectiveness models yields critical information which can be used to inform the structure and specifications of new models.

PIN136 MODELLING CD4 TRANSITIONS IN HIV PATIENTS: VALIDATING A MARKOV MODEL BASED APPROACH

A key limitation of discrete-event simulation (DES) models in HIV is the requirement for repeated iterations to reach convergence. This is particularly problematic where clinical efficacy is informed by non-inferiority trials and small reported differences result in a prohibitively high number of iterations being required. Markov models offer an alternative to DES models that does not require multiple iterations to reach convergence. This study validates an alternative approach to estimating CD4 transitions within a Markov framework against data from a clinical study. A previously published cost-effectiveness model (CEM) was used to estimate the proportions of patients in each CD4 health state at the end of the study period. Transition probabilities between CD4 health states were estimated using data on baseline CD4 cell count and change in CD4 cell count over the study period. The proportions of patients in each CD4 health state estimated by the CEM were compared to those reported by a clinical study to validate the model data, with root mean squared error used to assess the level of agreement between the two. A high level of agreement was observed between proportions in each CD4 state as estimated by the CEM and as observed in a clinical study, successfully validating this method of modelling CD4 transitions within a Markov framework. The approach to modelling CD4 transitions presented here replicates CD4 profiles observed in clinical practice, without requiring large numbers of iterations to reach convergence. This makes it feasible to run the range of scenario and sensitivity analyses routinely required for health technology assessment in a reasonable timeframe, while maintaining robust and accurate estimates of health economic outcomes.

The value of persistence in treatment with subcutaneous TNF-alpha inhibitors for ankylosing spondylitis.

To estimate the impact of persistence on cost-effectiveness of subcutaneous tumor necrosis factor-α inhibitors (SC-TNFis) from healthcare and societal perspectives in a United Kingdom ankylosing spondylitis (AS) population using a recently published Markov cohort model. A recently published cost-effectiveness model developed for a National Institute for health and Care Excellence appraisal was extended to fit the current study; in brief, it is a Markov cohort model where treatment responders continue from the trial period with maintenance SC-TNFi treatment, while non-responders transition to conventional care. Costs and effects were modeled for a hypothetical SC-TNFi with average efficacy and price. Model outcomes included quality-adjusted life-years (QALYs), total direct and indirect lifetime costs, and incremental cost-effectiveness ratios (ICERs). The cost-effectiveness of SC-TNFi persistence was estimated by decreasing the annual discontinuation probability in five percentage point increments from 25 to 5% per annum. From a health care perspective, the ICERs for the modeled discontinuation rates compared to the baseline annual discontinuation rate (25%) ranged between GBP 17,277 and GBP 18,161. From a societal perspective, increased discontinuation rates resulted in decreased total costs and higher QALYs; hence, lower discontinuation rates dominated higher discontinuation rates from a societal perspective. In conclusion, this study shows that, all else equal, higher SC-TNFi treatment persistence in AS is cost effective from a health care perspective and dominant from a societal perspective. Hence, all else equal, prescribing the SC-TNFi with the highest persistence may be considered a cost-effective strategy.

PP41 Cost-Effectiveness Modeling Of Chimeric Antigen Receptor T-Cell Therapies

IntroductionThis study has two key aims. The first is to review cost-effectiveness (CE) models for chimeric antigen receptor T-cell (CAR-T) therapies that have been appraised by health technology assessment (HTA) authorities. The second is to identify the key challenges of CE modeling of CAR-T therapies based on the main points raised in the HTA appraisals.MethodsA targeted HTA review of published CE models for CAR-T therapies in the United Kingdom (UK) and United States (US) was undertaken.ResultsFour relevant CE models were identified – three from the UK and one in the US. Of the three UK models, two were single technology submissions to the National Institute for Health and Care Excellence (NICE) and one was a ‘mock’ appraisal undertaken by NICE with a hypothetical evidence dataset. The one US model was published by the Institute for Clinical and Economic Review (ICER) committee. Two key model structures were adopted across the appraisals: a three-health state partitioned survival analysis model and a short-term decision tree followed by a three-health state partitioned survival model. The key modeling challenges identified can by summarized into five main categories: comparator evidence generation, estimation of long-term survival, curative benefit, health-related quality of life, and infrastructure/training requirements.ConclusionsThere are many challenges associated with the CE modeling of CAR-T therapies, with the most critical issues related to how uncertainty for long-term efficacy and safety can be addressed and mitigated. With more mature evidence sets in the future, stakeholders will get a clearer picture for the long-term benefit and risk of CAR-T therapies, but until then it is likely that HTA authorities will take a conservative stand when appraising the comparative value of CAR-T therapies.

PCN104 - A Systematic Literature Review of the Metholodigcal Approaches and Conclusions from Published Cost-Effectiveness Models in the Treatment of Aggressive B-Cell Lymphomas

PCN104 - A Systematic Literature Review of the Metholodigcal Approaches and Conclusions from Published Cost-Effectiveness Models in the Treatment of Aggressive B-Cell Lymphomas

PUK11 - Systematic Assessment of Decision Analytic Models for the Cost Effectiveness of Immunosuppressive Agents Used In Kidney Transplant Patients

To systematically review the methodological approaches in published cost effectiveness models of Immunosuppressant approved as maintenance therapies used in kidney transplantation patients. A systematic literature search from different databases with an end date of December 18, 2017 was completed. The search was limited to English studies, cost effectiveness models comparing, immunosuppressant drugs approved as maintenance therapies used in kidney transplantation and those reporting quality adjusted life years. The quality of reporting the models was performed by using CHEERS statement. The initial search for title and abstract screening resulted in 903 articles. 7 economic evaluation studies were included in the final review. The reporting quality scores of most articles were rated as acceptable, between 61% and 100%. Of the included 7 studies, only 1 was done in the USA. All studies (100%) were modeled for adult age range between 43 and 50 years old. Only 2 studies (28%) evaluated standard of care versus Belatacept. All studies defined their health states by the existence or absence of functioning graft. No studies took the societal prospective. The time horizons varied, but 57% of studies used a lifetime horizon. Fifty seven percent of studies identified that maintaining graft function assumption and time horizon as the most sensitive and influential parameters. Forty two percent of studies did not conduct any model validation. Cost effectiveness models of Immunosuppressant had different modeling approaches and quality levels. Future cost effectiveness models should consider evaluating Belatacept in USA because no cost-effectiveness studies have addressed USA population in which the use of Belatacept is rising. Also, future models should include comorbidities in the model, since some studies have claimed that comorbidities may affect drug responses.

Abstract 143: Pearls and Pitfalls of Performance-linked Reimbursement Plans for Novel Drugs: The Case of Sacubitril-Valsartan

Introduction: Rising drug costs have sparked interest in tying payments to outcomes to reduce payer risk of adopting low-value interventions. However, performance-linked reimbursement (PLR) plans are poorly understood. We modeled the effects of PLR plans on the distribution of therapy value obtained using sacubitril-valsartan as an illustrative case. Methods: We modeled PLR plans that issued refunds/discounts based on reductions in cardiovascular mortality or heart failure hospitalization. We used probabilistic sensitivity analyses from a published cost-effectiveness model of sacubitril-valsartan to model real-world uncertainty. We compared the distribution of value obtained (net monetary benefit [NMB]) with PLR plans and conventional payment across 10,000 paired simulations. Price premiums were set so average reimbursement was equal with both schemes. We varied plan structure and treatment/population uncertainty. With successful plans, payment would decrease in simulations with less benefit and increase with more benefit - decreasing the NMB standard deviation. We also calculated incremental NMBs as valuations of the change in NMB variability using a risk-averse utility curve. Results: A plan that adjusted drug reimbursement based on the ratio of observed to expected reduction of cardiovascular mortality reduced the standard deviation of NMB by 16.6% (Table 1). The incremental NMB - an estimate of per-patient value of implementing the PLR plan - was only $35, 3.3% of the NMB with conventional payment. Cardiovascular mortality plans were more successful than heart failure hospitalization plans. With rare outcomes, refunds based on cohort event rates were larger than refunds for individual adverse events. Plans improved with greater treatment effect uncertainty, larger refunds, and more payer risk-aversion. Their value decreased with more natural history uncertainty. Plans unraveled if the payer asymmetrically selected higher risk patients. Conclusion: PLR plans require outcomes tightly associated with treatment value and minimally influenced by unrelated uncertainty. Both sides must agree on a predictable population and limit asymmetric selection. For rare outcomes, plans should evaluate population event rates or surrogate measures that consistently reflect the clinical benefit.

Systematic assessment of decision analytic models for the cost-effectiveness of bariatric surgery for morbid obesity

Bariatric surgery among patients with morbid obesity is very effective for providing long-term weight loss and remission of obesity-related co-morbidities. However, it is very expensive and its cost effectiveness is commonly argued. Long-term cost-effectiveness evaluations of bariatric surgery have often relied on decision models. A systematic review was performed on the methodologic approaches and their quality, evaluated the quality of reporting, and summarized findings and conclusions in published cost-effectiveness models of bariatric surgery for morbid obesity. A search from different databases with an end date of October 15, 2017 was completed. The initial search for title and abstract screening resulted in 741 articles. A total of 50 articles were included for full-text review and 23 economic evaluation studies were included in the systematic review. The reporting quality scores of most articles were rated as acceptable between 61% and 100%. Most studies (89%) were modeled for adult patients with age range between 25 and 75 years old. Sixty-one percent of studies defined their health states by the existence or absence of different obesity-related co-morbidities. Eleven percent of studies took the societal perspective. Most studies (61%) used a lifetime horizon. Thirty-nine percent of studies identified the extent of weight loss as the most sensitive and influential parameter. Seventeen (74%) did not report a formal model validation. Laparoscopic Roux-en-Y gastric bypass was reported as the most cost-effective strategy most often when it compared with no treatment or medical management. While most had acceptable quality of reporting levels, several gaps in the quality of reporting and quality of methods emerged, which led to recommendations for how to improve quality in future studies.

Exploring the potential value of improved care for secondary hyperparathyroidism with a novel calcimimetic therapy

Aims: This study explored the use of a value-based pricing approach for the new calcimimetic etelcalcetide indicated for the treatment of secondary hyperparathyroidism (SHPT) in patients receiving hemodialysis. It used the US payer perspective and applied the cost-effectiveness framework. Because etelcalcetide is an intravenous therapy that can be titrated for individual patients, and because its utilization is yet to be assessed in real world settings, a range of plausible doses were estimated for etelcalcetide to define a range of prices. These were either in relation to the existing oral calcimimetic cinacalcet or compared to no calcimimetic treatment.Materials and methods: The value-based price of etelcalcetide was determined via a Markov model. This model combined data from the etelcalcetide trials and previously published cost-effectiveness models in SHPT, and allowed extrapolation of treatment effects on mortality, cardiovascular events, fracture, and parathyroidectomy. Several dosing scenarios were explored covering the dose ranges of 30.0–64.18 mg per day for cinacalcet and 1.07–3.11 mg per day for etelcalcetide. These included the mean dose from the etelcalcetide trials, the preliminary defined daily dose, and the expected most common dose in real world. An acceptable price range for etelcalcetide was assessed by comparing the incremental cost-effectiveness ratios obtained with the willingness-to-pay threshold range of $100,000–$300,000/quality-adjusted life-years.Results: Cost-effectiveness analysis supported value-based prices for etelcalcetide ranging from $21.15–$49.97/mg vs cinacalcet, and $13.79–$119.45/mg vs no calcimimetics.Limitations: There is uncertainty around what the real-world dosing will be for etelcalcetide. Another important nuance is that no studies have examined etelcalcetide effects on hard outcomes and, therefore, this modeling exercise relied on an extrapolation approach.Conclusions: This cost-effectiveness analysis, including scenarios to address uncertainties, allowed estimation of a value-based price range to aid reimbursement decisions in the US.

VP100 Disease Modelling Approaches In Multiple Sclerosis

INTRODUCTION:In the past decades the cost-effectiveness of new effective disease-modifying therapies (DMTs) for Relapsing Remitting Multiple Sclerosis (RRMS) form was assessed through decision analytical models. Recently, new treatment option for the Primary Progressive (PPMS) form was developed. Aim of this work was assessing the similarities and differences of PPMS and RRMS and their impact in the development of decision analytical model for PPMS.METHODS:Literature review was performed to retrieve information on natural history of PPMS and RRMS and impact of DMTs agents on the progression of these conditions. Further, a review of the published cost-effectiveness models for RRMS was performed. Based on these data, an analysis on the difference and similarities between the two MS forms that could have an impact on the development of decision analytical model for PPMS was performed.RESULTS:Based on the analysis, similar structure model used for RRMS could be applied for PPMS. Health states of the model could be based on Expanded Disability Status Scale score as already done for RRMS. The relapse events considered for RRMS should not be included in PPMS model, and no possibility to develop another form, as the Secondary Progressive, should be included. While RRMS models should include at least a second line treatment option due to alternative DMTs available, only first treatment line should be considered for PPMS. Assessing data available to populate the model, poor data on the natural history, utility and cost associated to PPMS were available and assumption or expert opinions will be needed to overcome the lack of robust data.CONCLUSIONS:A decision analytical model for PPMS can use a similar structure used in the models for RRMS. However, more robust data on PPMS and some structural change are needed to provide a good tool to assess cost-effectiveness of DMTS in PPMS.

Systematic Review of Modelling Approaches for the Cost Effectiveness of HepatitisC Treatment with Direct-Acting Antivirals.

New direct-acting antivirals (DAAs) are highly effective for hepatitisC virus (HCV) treatment. However, their prices have been widely debated. Decision-analytic models can project the long-term value of HCV treatment. Therefore, understanding of the methods used in these models and how they could influence results is important. Our objective was to describe and systematically review the methodological approaches in published cost-effectiveness models of chronic HCV treatment with DAAs. We searched several electronic databases, including Medline, Embase and EconLit, from 2011 to 2015. Study selection was performed by two reviewers independently. We included any cost-effectiveness analysis comparing DAAs with the old standard of care for HCV treatment. We excluded non-English-language studies and studies not reporting quality-adjusted life-years. One reviewer collected data and assessed the quality of reporting, using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) statement. Another reviewer crosschecked the abstracted information. The development methods of the included studies were synthetized on the basis of good modelling practice recommendations. Review of 304 citations revealed 36 cost-effectiveness analyses. The reporting quality scores of most articles were rated as acceptable, between 67 and 100%. The majority of the studies were conducted in Europe (50%), followed by the USA (44%). Fifty-six percent of the 36 studies evaluated the cost effectiveness of HCV treatment in both treatment-naive and treatment-experienced patients, 97% included genotype1 patients and 53% evaluated the cost effectiveness of second-generation or oral DAAs in comparison with the previous standard of care or other DAAs. Twenty-one models defined health states in terms of METAVIR fibrosis scores. Only one study used a discrete-event simulation approach, and the remainder used state-transition models. The time horizons varied; however, 89% of studies used a lifetime horizon. One study was conducted from a societal perspective. Thirty-three percent of studies did not conduct any model validation. We also noted that none of the studies modelled HCV treatment as a prevention strategy, 86% of models did not consider the possibility of re-infection with HCV after successful treatment, 97% of studies did not consider indirect economic benefits resulting from HCV treatment and none of the studies evaluating oral DAAs used real-world data. The search was limited by date (from 1January 2011 to 8September 2015) and was also limited to English-language and published reports. Most modelling studies used a similar modelling structure and could have underestimated the value of HCV treatment. Future modelling efforts should consider the benefits of HCV treatment in preventing transmission, extra-hepatic and indirect economic benefits of HCV treatment, real-world cost-effectiveness analysis and cost effectiveness of HCV treatment in low- and middle-income countries.

Value of Information Analysis Applied to the Economic Evaluation of Interventions Aimed at Reducing Juvenile Delinquency: An Illustration.

ObjectivesTo investigate whether a value of information analysis, commonly applied in health care evaluations, is feasible and meaningful in the field of crime prevention.MethodsInterventions aimed at reducing juvenile delinquency are increasingly being evaluated according to their cost-effectiveness. Results of cost-effectiveness models are subject to uncertainty in their cost and effect estimates. Further research can reduce that parameter uncertainty. The value of such further research can be estimated using a value of information analysis, as illustrated in the current study. We built upon an earlier published cost-effectiveness model that demonstrated the comparison of two interventions aimed at reducing juvenile delinquency. Outcomes were presented as costs per criminal activity free year.ResultsAt a societal willingness-to-pay of €71,700 per criminal activity free year, further research to eliminate parameter uncertainty was valued at €176 million. Therefore, in this illustrative analysis, the value of information analysis determined that society should be willing to spend a maximum of €176 million in reducing decision uncertainty in the cost-effectiveness of the two interventions. Moreover, the results suggest that reducing uncertainty in some specific model parameters might be more valuable than in others.ConclusionsUsing a value of information framework to assess the value of conducting further research in the field of crime prevention proved to be feasible. The results were meaningful and can be interpreted according to health care evaluation studies. This analysis can be helpful in justifying additional research funds to further inform the reimbursement decision in regard to interventions for juvenile delinquents.