US Healthcare Payers Research Articles

Atezolizumab plus bevacizumab and chemotherapy as first-line therapy for cervical cancer: a cost-effectiveness analysis in the US

ObjectiveMedication is the predominant therapy for advanced cancers. However, the use of novel anticancer medications is a major contributor to disease-related financial hardships. Recently, numerous countries have mandated the pharmacoeconomic assessments of novel oncological agents to mitigate patient financial risks and optimize resource allocation. The present study evaluated the cost-effectiveness of adding atezolizumab to standard therapy (atezolizumab plus bevacizumab [BC]) for metastatic, persistent, and recurrent cervical cancer from the perspective of US healthcare payers, with the aim of supporting policymaking and promoting the rational use of healthcare resources.MethodsUsing clinical efficacy and safety data from the BEATcc clinical trial, in addition to cost and utility values from publicly available databases and published literature, a partitioned survival model over a 20-year lifetime horizon was developed to assess the cost-effectiveness of atezolizumab plus bevacizumab and chemotherapy (ABC) versus BC. The primary output of the model was the incremental cost-effectiveness ratio (ICER) and sensitivity analyses were performed to assess its robustness.ResultsAt both 20 and 4.5 y of time horizon, ABC therapy showed poor cost-effectiveness, with ICER of $193926.48/QALY and $168482.26/QALY, respectively, which were higher than the $150,000/QALY willingness-to-pay threshold. One-way sensitivity analysis showed that the price of atezolizumab had the most significant impact on the model results. When the price of atezolizumab was reduced by 10%, ABC changed from being not cost-effective to cost-effective (ICER = $121531.24/QALY). Probabilistic sensitivity analysis showed a 32.6% probability that ABC would be cost-effective, which increased to 58.6% when the price of atezolizumab was reduced by 10%.ConclusionsFor patients with metastatic, persistent, and recurrent cervical cancer in the US, ABC was not as cost-effective as BC. Appropriate price reduction (10%) is recommended for atezolizumab to improve cost-effectiveness of ABC therapy.

Payer perceptions and use of value assessment tools in the United States.

BACKGROUND: As the United States transitions toward value-based payment, value assessment tools to measure the value of health care interventions are emerging. As the field evolves, it is important to evaluate how these tools are influencing treatment and coverage decisions. OBJECTIVE: To examine payer perceptions and use of US value assessment tools and identify how these tools inform payer decision-making. METHODS: A double-blind, web-based survey was conducted from June to July 2022 to assess health care payers' perceptions and use of value assessment tools developed by the American Society of Clinical Oncology, Drug Pricing Lab, Institute for Clinical and Economic Review (ICER), Innovation and Value Initiative, and National Comprehensive Cancer Network. RESULTS: 51 respondents completed the survey. 86% of payers were familiar with at least 4 of 5 value assessment tools. Both ICER and National Comprehensive Cancer Network tools are perceived as very useful for informing formulary decisions (57% and 49%, respectively). When selecting a value assessment tool, payers identified the inclusion of appropriate metrics and outcomes (92%), comparative clinical effectiveness information (88%), and reliance on rigorous, unbiased methods (86%) to be very/extremely important. Payers reported the inclusion of the patient, provider, and societal perspectives as lower importance (32%, 31%, and 20% identify these elements as very/extremely important, respectively). Payers reported using ICER evidence reports to both expand and restrict coverage decisions. To advance more useful and relevant value assessment tools, payers identified the need for greater stakeholder awareness of existing tools, and some recommended that value assessors increase the volume of assessments conducted. CONCLUSIONS: US health care payers perceive select value assessment tools to be useful for informing health care decisions. As policy momentum behind value assessment builds, additional examination of value assessment tools is needed to inform appropriate application of value assessment in US health care decision-making. DISCLOSURES: This study was funded by Xcenda/AmerisourceBergen. Ms Buelt, Ms Loo, Ms Westrich, and Drs Hydery and Zheng report employment with Xcenda/AmerisourceBergen. Drs Dharbhamalla and Graff report employment with AMCP.

Cost-Effectiveness of Vonoprazan-Based and Rifabutin-Based vs Other Regimens as First-Line Treatment of Helicobacter pylori Infection in the United States.

The economic and clinical implications of eradicating Helicobacter pylori ( H. pylori ) with vonoprazan-based and rifabutin-based regimens vs other existing prepackaged first-line treatment options in the United States are unknown. Therefore, we evaluated the cost-effectiveness of vonoprazan-based and rifabutin-based and other prepackaged regimens for the first-line treatment of H. pylori from the perspective of US healthcare payers. We used the state-transition Markov model to conduct a cost-effectiveness analysis of H. pylori eradication with clarithromycin triple, bismuth quadruple, vonoprazan dual, vonoprazan triple, and rifabutin triple regimens. In a cycle length of 2 months, the model estimated the expected costs (expressed in 2022 US$), expected quality-adjusted life-years (QALY), incremental cost-effectiveness ratios, and expected net monetary benefit over 20 years. In addition, we accounted for the present value of future costs and QALY by applying a 3% discounting rate. In this study, rifabutin triple therapy had a lower expected cost but was more effective than clarithromycin triple, bismuth quadruple, and vonoprazan dual regimens; hence, it dominated them. Vonoprazan triple therapy had a higher expected cost (US$ 1,172 vs US$ 1,048) and expected QALY (14.262 vs 14.256) than rifabutin triple therapy, yielding an estimated incremental cost-effectiveness ratio of US$ 22,573/QALY. The study suggested that vonoprazan triple treatment had the highest expected net monetary benefit and was the most cost-effective at willingness-to-pay thresholds between US$50,000 and US$150,000 per QALY, followed by rifabutin triple therapy. H. pylori infection eradication with vonoprazan triple therapy would provide the greatest net health and monetary benefit from the perspective of US healthcare payers.

Cost-effectiveness of a 12-month fixed-duration venetoclax treatment in combination with obinutuzumab in first-line, unfit chronic lymphocytic leukemia in the United States.

BACKGROUND: Chronic lymphocytic leukemia (CLL) is a significant health and economic burden in the United States. Treatments include chemoimmunotherapy, such as obinutuzumab (G) plus chlorambucil (Clb) or bendamustine plus rituximab (BR), and chemotherapy-free regimens incorporating oral targeted therapies such as ibrutinib (Ibr), acalabrutinib (Acala), or venetoclax (Ven). Most chemotherapy-free regimens require continuous treatment to progression, while Ven plus G (VenG) is given for a fixed duration of 12 months, based on the CLL14 trial that led to its approval. Fixed-duration VenG has the potential for cost savings compared with treat-to-progression chemotherapy-free regimens. OBJECTIVE: To evaluate the cost-effectiveness of 12 months fixed-duration VenG in first-line treatment of unfit patients with CLL from a US health care payer perspective compared with GClb, BR, Ibr, Ibr + G, Ibr + R, Acala, and Acala + G. METHODS: A partitioned survival model was developed with 3 health states: progression-free survival (PFS), postprogression survival, and dead. The patient population, as based on the CLL14 trial, comprised previously untreated unfit patients with CLL (mean age 71.1 years, 33.1% female). The distribution of patients in each health state over time was estimated using extrapolated PFS and overall survival (OS) curves for VenG and GClb, based on CLL14 data 2 or more years after treatment cessation. PFS and OS for the other comparators were estimated using hazard ratios vs VenG, based on a network metaanalysis. Adverse events, utility values, and costs were obtained from published literature. The model estimated life-years gained, quality-adjusted life-years (QALYs) gained, and costs. The time horizon was 20 years, with a cycle time of 28 days. Outcomes and costs were discounted at 3.0% per year, and costs were estimated from a US health care payer perspective. One-way and probabilistic sensitivity analyses were conducted. RESULTS: In this cross-trial analysis of unfit CLL patients, in the base case, VenG had lower projected total costs than all comparators investigated. VenG also had larger projected health benefits (more QALYs gained) than GClb, BR, Ibr, and Ibr + R. VenG was therefore more effective and less costly than these comparators (dominant). Ibr + G, Acala, and Acala + G showed higher QALYs gained vs VenG (0.022, 0.672, and 0.961, respectively), and substantially higher projected costs vs VenG ($1,488,400, $1,579,737, and $1,656,154, respectively). Thus, Ibr + G, Acala, and Acala + G would cost more than $1,000,000 per QALY gained vs VenG. At the commonly used willingness-to-pay threshold of $150,000 per QALY gained, Ibr + G, Acala, and Acala + G were not cost-effective compared with VenG. CONCLUSIONS: Fixed-duration VenG for 12 months is a cost-effective first-line treatment option for unfit CLL patients compared with other available options and provides value for money to US health care payers at a threshold of $150,000 per QALY gained. Future studies with longer trial follow-up and more mature survival data may help to confirm longer-term cost benefits of VenG. DISCLOSURES: Genentech Inc. and AbbVie provided financial support for this study. Genentech Inc., AbbVie, and Pharmerit - An OPEN Health Company participated in the design, study conduct, analysis, and interpretation of data, as well as the writing, review, and approval of the manuscript. Venetoclax is being developed in a collaboration between Genentech Inc. and AbbVie. Ravelo and Shapouri are employed by Genentech Inc. and have ownership interests. Manzoor and Sail are employed by AbbVie and have ownership interests. Chatterjee, van de Wetering, and Qendri, employees of Pharmerit - An OPEN Health Company, received consultancy fees from AbbVie. Davids has received consultancy fees from AbbVie, AstraZeneca, Eli Lilly, Genentech Inc., Janssen, MEI Pharma, Novartis, Pharmacyclics, and Verastem; research funding from Ascentage Pharma, Genentech Inc., MEI Pharma, Pharmacyclics, Surface Oncology, TG Therapeutics, and Verastem; and has served on board of directors or advisory committees for AbbVie, Adaptive Biotechnologies, AstraZeneca, BeiGene, Eli Lilly, Genentech Inc., Janssen, Pharmacyclics, TG Therapeutics, and Verastem. This study was presented as a poster at ASH 61st Annual Meeting and Exposition; December 7-10, 2019; Orlando, FL.

Budget Impact of the Vest™ High Frequency Chest Wall Oscillation System for Managing Airway Clearance in Patients with Complex Neurological Disorders: A US Healthcare Payers' Perspective Analysis.

ObjectiveAs a recognized therapy to improve airway clearance, high-frequency chest wall oscillation (HFCWO) is used to manage reduced vital capacity in patients with complex neuromuscular disorders (cNMD). This study aimed to investigate the budget impact of HFCWO versus chest wall physical therapy (CWPT) from a US-commercial payer perspective.MethodsIn combination with a previously developed cost-effectiveness model, a budget impact model was developed to evaluate the incremental budgetary impact associated with introducing a HFCWO device over a 5-year time horizon. The model compared the cost implications associated with the commonly used CWPT procedure, as the current scenario, with a new scenario consisting of 80% of market share for HFCWO. The resource use and costs included in the analyses were costs associated with the HFCWO device (Vest™ System) and its consumables, patient training, and medical services such as hospitalization, medications, emergency room, and outpatient visits. The primary outcome measures included total and incremental budgetary impact per member per year (PMPY).ResultsIn a hypothetical plan of 1,000,000 members (men: 49.2%), 2099 patients with cNMD were estimated to be eligible to receive airway clearance services over 5 years. The new scenario (HFCWO and CWPT [US$24 PMPY]) was cost-saving compared with the current scenario (CWPT only [$34 PMPY]) with a cost reduction of US$9.46 PMPY. The model estimated a net cost-saving of US$1,594,131 and US$9,591,343 over 1 and 5 years, respectively.ConclusionThis study suggests the HFCWO technique to manage the reduction in vital capacity in patients with cNMD would lead to favorable budget impact results.

Cost-effectiveness and budget impact of dolutegravir/lamivudine for treatment of human immunodeficiency virus (HIV-1) infection in the United States.

BACKGROUND: Dolutegravir(DTG)/lamivudine(3TC) is the first 2-drug regimen recommended as an initial treatment for people living with HIV (PLHIV). OBJECTIVE: To assess the cost-effectiveness and potential budget impact of DTG/3TC in the US healthcare setting. METHODS: A previously published hybrid decision-tree and Markov cohort state transition model was adapted to estimate the incremental costs and health outcome benefits over a patients' lifetime. DTG/3TC was compared with current standard of care in treatment naive and treatment experienced virologically suppressed PLHIV. Health states included in the model were based upon virologic response and CD4 cell count, with death as an absorbing state. Clinical data was informed by the Phase III GEMINI 1 and 2 clinical trials, a published network meta-analysis (NMA) in treatment-naive patients and the Phase III TANGO clinical trial in treatment experienced patients. Costs and utilities were informed by published data and discounted annually at a rate of 3%. A separate 5-year budget impact analysis was conducted assuming 5%-15% uptake in eligible treatment naive and 10%-30% uptake in eligible treatment experienced patients. RESULTS: In the treatment naive analyses based on GEMINI 1 and 2, DTG/3TC dominated, i.e., was less costly and more effective, than all comparators. DTG/3TC resulted in 0.083 incremental quality-adjusted life-years (QALYs) at a cost saving of $199,166 compared with the DTG + tenofovir disoproxil(TDF)/emtricitabine(FTC) comparator arm. The incremental QALY and cost savings for DTG/3TC compared with DTG/abacavir(ABC)/3TC, cobicistat-boosted darunavir(DRV/c)/tenofovir alafenamide(TAF)/FTC, and bictegravir (BIC)/TAF/FTC, based on NMA results were 0.465, 0.142, and 0.698, and $42,948, $122,846, and $44,962, respectively. In the analyses of treatment-experienced virologically suppressed patients based on TANGO, DTG/3TC offered slightly lower QALYs (-0.037) with an estimated savings of $78,730 when compared with continuation of TAF-based regimen (TBR). Sensitivity analyses demonstrated that these conclusions were relatively insensitive to alternative parameter estimates. The budget impact analysis estimated that by 5th year a total of 70,240 treatment naive patients and 1,340,480 treatment experienced patients could be eligible to be prescribed DTG/3TC. The estimated budget savings over 5 years ranged from $1.12b to $3.35b (corresponding to 27,512 to 82,536 on DTG/3TC by year 5) in the lowest and highest uptake scenarios, respectively. CONCLUSION: In conclusion, DTG/3TC with its comparable efficacy and lower drug acquisition costs, has the potential to offer significant cost savings to US healthcare payers for the initial treatment of treatment naive patients and as a treatment switching option for virologically suppressed patients. DISCLOSURES: This study was funded in full by ViiV healthcare, Brentford, UK. Medical writing to support this study was also funded in full by ViiV Healthcare, Brentford, UK. Butler, Hayward, and Jacob are employees of HEOR Ltd, the company performing this study funded by ViiV Healthcare. Anderson is an employee of GlaxoSmithKline and owns shares in the company. Punekar, Evitt, and Oglesby are employees of ViiV Healthcare and own stocks in GlaxoSmithKline.

Nivolumab vs Pembrolizumab for Treatment of US Patients With Platinum-Refractory Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma

Nivolumab and pembrolizumab are approved for treating platinum-refractory recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC). Physicians and patients are uncertain which drug is preferable, rendering a cost-effectiveness comparison between them necessary. To evaluate the cost-effectiveness of nivolumab vs pembrolizumab in treating platinum-refractory R/M HNSCC. Both the network meta-analysis and cost-effectiveness analysis included patients from the CheckMate 141 and the KEYNOTE 040 phase 3 randomized clinical trials. The Checkmate 141 trial started on May 1, 2014, with the present analysis based on a September 2017 data cutoff. The KEYNOTE 040 trial started on November 17, 2014, with the present analysis based on a May 15, 2017, data cutoff. A bayesian network meta-analysis that included 856 patients was carried out, and a cost-effectiveness analysis that included 487 patients was conducted by developing a partitioned survival model, both between February and November 2020. The robustness of the model was assessed via 1-way, 2-way, and probabilistic sensitivity analyses; subgroup analyses were included; and scenario analyses were conducted to investigate the associations of dosage adjustment of nivolumab with cost-effectiveness. Life-years, quality-adjusted life-years (QALYs), overall costs, and incremental cost-effectiveness ratios (ICERs) were measured. In the cost-effectiveness analysis that included 487 patients, for US health care payers, when nivolumab was administered based on patient weight (3 mg/kg biweekly), at a willingness-to-pay (WTP) threshold of $100 000 per QALY, the probability of nivolumab being cost-effective compared with pembrolizumab was 56%; at a WTP threshold of $150 000 per QALY, the probability was 62%. When nivolumab was administered at a fixed dose of 240 mg biweekly or 480 mg monthly, at a WTP threshold of $100 000 per QALY, the probability of nivolumab being cost-effective was 42% to 45%; at a WTP threshold of $150 000 per QALY, the probability was 52% to 55%. Findings from this network meta-analysis and cost-effectiveness analysis suggest considering both WTP threshold and patient body weight when choosing between nivolumab and pembrolizumab for the treatment of patients with platinum-refractory R/M HNSCC. When the WTP threshold was $100 000 per QALY, for patients weighing less than 72 kg, nivolumab (3 mg/kg, biweekly) was considered cost-effective; otherwise, pembrolizumab was preferable. When the WTP threshold was $150 000 per QALY, nivolumab (3 mg/kg biweekly) was considered cost-effective for patients weighing less than 75 kg; otherwise, fixed-dose nivolumab (240 mg biweekly or 480 mg monthly) provided more cost savings.

Budget Impact of Oral Semaglutide Intensification versus Sitagliptin among US Patients with Type 2 Diabetes Mellitus Uncontrolled with Metformin.

BackgroundOral semaglutide was approved in 2019 for blood glucose control in patients with type 2 diabetes mellitus (T2DM) and was the first oral glucagon-like peptide 1 receptor agonist (GLP-1 RA). T2DM is associated with substantial healthcare expenditures in the US, so the cost of a new intervention should be weighed against clinical benefits.ObjectiveThis study evaluated the budget impact of a treatment pathway with oral semaglutide 14 mg daily versus oral sitagliptin 100 mg daily among patients not achieving target glycated hemoglobin (HbA1c) level despite treatment with metformin.MethodsThis study used the validated IQVIA™ CORE Diabetes Model to simulate the treatment impact of oral semaglutide 14 mg and sitagliptin 100 mg over a 5-year time horizon from a US healthcare sector (payer) perspective. Trial data (PIONEER 3) informed cohort characteristics and treatment effects, and literature sources informed event costs. Population and market share data were from the literature and data on file. The analysis evaluated the estimated budget impact of oral semaglutide 14 mg use for patients currently using sitagliptin 100 mg considering both direct medical and treatment costs to understand the impact on total cost of care, given underlying treatment performance and impact on avoidable events.ResultsIn a hypothetical plan of 1 million lives, an estimated 1993 patients were treated with sitagliptin 100 mg in the target population. Following these patients over 5 years, the incremental direct medical and treatment costs of a patient using oral semaglutide 14 mg versus sitagliptin 100 mg was $US16,562, a 70.7% increase (year 2019 values). A hypothetical payer would spend an additional $US3,300,143 (7.1%) over 5 years for every 10% of market share that oral semaglutide 14 mg takes away from sitagliptin 100 mg. Univariate and scenario analyses with alternate inputs and assumptions demonstrated consistent results.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.Electronic supplementary materialThe online version of this article (10.1007/s40273-020-00967-7) contains supplementary material, which is available to authorized users.

Treatment of venous leg ulcers using bilayered living cellular construct.

Background: Venous leg ulcers (VLUs) present a significant economic burden on the US healthcare system and payers (US$14.9 billion). Aim: To evaluate the quality of life (QoL) of patients with VLUs; to analyze the limitations of standard of care (SOC) for VLUs; and to explain how using bilayered living cellular construct (BLCC) with SOC for treatment of VLUs can help heal more VLUs faster (than using SOC alone) as well as help improve QoL and help reduce the burden on the US healthcare system and payers. Materials & methods: This is a review study. The search was conducted in February 2020 by way of electronic databases to find relevant articles that provided information related to QoL of patients with VLUs, limitations of SOC for VLUs and economic analyses of using BLCC for treatment of VLUs. Results: VLUs impact patients' physical, functional and psychological status and reduce QoL. A total75% of VLU patients who used SOC alone failed to achieve healing in a timely fashion, which led to increased healthcare costs and healthcare resource utilization. Although the upfront cost is high, the greater effectiveness of BLCC offsets the added cost of the product during the time period of the studies. Therefore, BLCC helps to improve the QoL of VLU patients. As an example, for every 100 VLU patients in a healthcare plan, the use of BLCC can create cost savings of US$1,349,829.51. Conclusion: Payers' coverage of BLCC results in reduction of the overall medical cost for treating VLU patients.

Associations of Renin-Angiotensin System Antagonist Medication Adherence and Economic Outcomes Among Commercially Insured US Adults: A Retrospective Cohort Study.

BackgroundMedication non‐adherence can result in considerable morbidity, mortality, and costs. The Pharmacy Quality Alliance hypertension medication adherence measure is used by US healthcare payers and providers to assess renin‐angiotensin system antagonist medication adherence. However, associations between renin‐angiotensin system antagonist adherence as calculated in quality measures, and healthcare service use and expenditure in commercial populations over a 1‐year timeframe has not been assessed.Methods and ResultsThis retrospective cohort study used eligible commercially insured individuals from the Truven Health MarketScan Commercial Claims and Encounters Research Databases (2009–2015). Generalized linear models with log link and gamma distribution (expenditure) or negative binomial distribution (usage) assessed relationships between hypertension adherence (≥80% proportion of days covered) and healthcare use and expenditures (in 2015 US dollars) while adjusting for covariates (age, sex, geographic region; health plan; Deyo‐Charlson Comorbidity Index, number of chronic medications, and treatment naivety). Beta coefficients were used to compute cost ratios and rate ratios. A total of 4 842 058 subjects were eligible; of those, 3 310 360 (68%) were adherent (adherent mean age 53.3±8.0 years, 55.9% men; non‐adherent mean age 50.3±9.1 years, 53.1% men). Adherence was associated with fewer inpatient (rate ratios, 0.612; 95% CI, 0.607–0.617) and outpatient visits (rate ratios, 0.995; 95% CI, 0.994–0.997); and lower total costs (cost ratios, 0.876; 95% CI, 0.874–0.878) compared with non‐adherence. Adherence was associated with lower average per member per month total costs ($97.98) compared with non‐adherence.ConclusionsAdherence to renin‐angiotensin system antagonists was associated with fewer outpatient and inpatient visits, and lower total costs compared with non‐adherence in a 1‐year time frame.

Using ICD-10 codes to identify elective epilepsy monitoring unit admissions from administrative billing data: A validation study

Video-electroencephalogram (EEG) monitoring in the epilepsy monitoring unit (EMU) is essential for managing epilepsy and seizure mimics. Evaluation of care in the EMU would benefit from a validated code set capable of identifying EMU admissions from administrative databases comprised of large, diverse cohorts. We assessed the ability of code-based queries to parse EMU admissions from administrative billing records in a large academic medical center over a four-year period, 2016–2019. We applied prespecified queries for admissions coded as follows: 1) elective, 2) receiving video-EEG monitoring, and 3) including diagnoses typically required by major US healthcare payers for EMU admission. Sensitivity (Sn), specificity (Sp), and predictive value positive/negative (PVP, PVN) were determined. Two approaches were highly effective. Incorporating epilepsy, seizure, or seizure mimic codes as the admitting diagnosis (assigned at admission; Sn 96.3%, Sp 100.0%, PVP 98.3%, and PVN 100.0%) or the principal diagnosis (assigned after discharge; Sn 94.9%, Sp 100.0%, PVP 98.8%, and PVN 100.0%) identified elective adult EMU admissions with comparable reliability (p = 0.096). The addition of surgical procedure codes further separated EMU admissions for intracranial EEG monitoring. When applied to larger, more comprehensive datasets, these code-based queries should enhance our understanding of EMU utilization and access to care on a scalable basis.

Biosimilars in the USA: Will New Efforts to Spur Approvals and Access Spur Uptake and Cost Savings?

Unlike in Europe, US healthcare systems and payers are still awaiting significant savings related to biosimilar utilization. Costs related to biologic use continue to rise at double-digit rates, and biosimilars are seen as a major tool to control costs and increase access to biologic drugs. However, one 2018 report indicated that US$3.2 billion (only 3%) of biologic spending is subject to competition from biosimilar products. Although the European Medicines Agency did a great deal of pioneering work in biosimilar regulation, the US Food and Drug Administration is moving at approximately the same pace as the European Medicines Agency, based on the number of approvals at the same time after implementation of its regulatory pathway. Several unique factors in the USA have conspired to limit biosimilar access (e.g. delayed regulatory policies, extended patent litigation activities, federal reimbursement policies, the widespread use of rebate contracting, and limited competition). The US Federal Government is taking the initiative in an attempt to address these factors, and speed both biosimilar development and patient access. To date, the most significant cost savings in the US system associated with the introduction of biosimilars may be their ability to halt price increases of the reference product. The complexity of the healthcare delivery system, and how it is financed, will remain challenging to payers, manufacturers, health providers and patients as they seek ways to manage health expenditure growth.

PHP171 - Drivers of Genomic Precision Medicine Adoption in the US: Payer Preferences

The current literature on the drivers of genomic precision medicine adoption is limited. The objective of this study was to estimate healthcare payers’ preferences for genomic precision medicine tests. Using qualitative research methods (n=6 payers), we identified six key attributes for payers’ uptake of genetic tests: type of information the test provides (screening vs. treatment prediction), probability that the member has an informative genetic marker, experts’ agreement on changing medical care based on the marker, members’ quality-of-life gains, life expectancy gains with statistical uncertainty, and cost to the plan. We designed a stated preference discrete choice experiment using these attributes and administered a web-survey on a representative sample of US healthcare payers. We used effects coding and analyzed the data using a nested logit modeling approach. The survey response rate was 58% (150 participants completed the survey). Approximately 53% respondents had prior experience evaluating genetic tests for reimbursements and 85% had greater than 5 years of healthcare decision-making experience. Based on the payers’ preferences, the relative importance of the attributes was in the following order of decreasing importance: quality-of-life, gains in life expectancy, expert agreement in recommendations, probability that the member has an informative genetic marker, cost to the plan, and screening vs treatment prediction. Payers’ marginal willingness to pay (mWTP) was highest for genetic tests that lead to improvement in quality-of-life ($544 to $5856) and lowest for tests that resolved uncertainty in life expectancy gains ($656 to $1679). The mWTP for an increase in the marker probability ranged from $434 to $2846. Payers exhibited a strong preference for genetic tests that improved quality of life and life expectancy. The results of this study can be used to predict genetic test adoption, guide future investment in and design of genetic tests, and inform pricing and reimbursement decisions.

PHP24 - Icer Reports – 4 Years of Cost/Qaly Drug Evaluations in the USA… what Does the Future Hold?

In 2014, the Institute for Clinical and Economic Review (ICER) became the focus of attention for publishing a report concluding that new drug treatments for Hepatitis C offered low value for US healthcare payers based on a clinical and economic assessment. This research systematically analyzes ICER reports of drugs and discusses their potential future role in drug pricing. All publically available final ICER reports of pharmaceuticals (01/01/2014-12/31/2017) were screened and key data extracted. 19 ICER reports of pharmaceuticals were identified, encompassing 65 drugs in 37 conditions/subgroups. 56 panel votes of their clinical benefits deemed 64% clinically effective by majority vote. 39% were clearly (≥80% votes) clinically effective, with 25% clearly not clinically effective. Clinical ratings were provided for 58 drugs : 52% achieved ≥B rating, 17% C–D and 31% I or P/I. A cost-utility assessment was undertaken for 49 drug:indication pairings. Even at the highest WTP threshold of 150,000/QALY, only 29% therapies were deemed cost-effective, with a mean 46% discount required for an ICER<$150,000. Of 30 votes on the “care value” of a product, based on their clinical and cost-effectiveness, 33% were rated as low value, 3% low/intermediate, 43% intermediate, 7% intermediate/high, and 13% high. Of 11 votes on the “health system value” of products, based upon their anticipated budget impact and affordability, 100% were deemed low value. ICER reports have generally concluded that assessed therapies offer important clinical benefits but generally do not justify their economic impact. These reports currently do not influence coverage/pricing decisions but there is anecdotal evidence of insurers using these in negotiations. For example they were formally cited as part of Medicare Part B payment model reform discussions, indicating ICER reports may play a greater role in future reforms to US drug reimbursement as payers struggle to manage increasing costs.

Role of Cochrane Reviews in informing US private payers' policies.

To assess the use of Cochrane Reviews to inform the medical policy documents of major US private payers. The publically available drug policy documents of the five major US private payers (covering about 50% market) namely: Anthem, Aetna, Cigna, Humana and United Healthcare (UHC) were assessed in February 2017 and reviewed to find whether they had used Cochrane Reviews. We extracted information such as use of Cochrane Reviews, number of Cochrane Reviews used, context of use and impact of Cochrane Reviews on policy, and Cochrane Review Group (CRG) and Cochrane Center linked to the Cochrane Review. Among the selected payers, less than half of the policy documents used Cochrane Reviews: maximum for Aetna (52%) and minimum for Humana (2%). Three hundred and sixty-one Cochrane Reviews from 42 CRGs have been used to inform 118 drug policy documents: Aetna (221) Anthem (64), Cigna (30), and UHC (25). The highest number of reviews used from any Cochrane Center was 79 (UK). The most reviews used from any CRG were 27 (Musculoskeletal Group). The most reviews used to inform any one policy was 18 (Drug: Botulinum Toxin, Payer: Aetna). Overall, 66% of the Cochrane Reviews were used to inform the background section of the policy document and 34% supported the clinical usage of the drug. Furthermore, 42% of the reviews had cited inline in the policies they were used to inform. Cochrane Reviews are used to inform the US healthcare payers' policies, but there is still scope to encourage the further usage of Cochrane Reviews in healthcare decision making.

Economic impact of using fesoterodine for the treatment of overactive bladder with urge urinary incontinence in a vulnerable elderly population in the United States

Objectives:To assess the costs of treating overactive bladder (OAB) with fesoterodine compared to no OAB pharmacotherapy among vulnerable elderly from the US payer perspective.Methods:A decision analytic cost model was developed to estimate the 52-week costs of a cohort of vulnerable elderly with OAB initiating treatment with fesoterodine or no OAB pharmacotherapy. Vulnerable elderly OAB patients were defined as those aged ≥65 years with self-reported urge urinary incontinence (UUI) symptoms for ≥3 months, 2–15 UUI episodes/day, and at risk of deteriorating health by a score of ≥3 on the Vulnerable Elders Survey (VES)-13. Patients were evaluated for fesoterodine treatment response (defined as no UUI episodes) and persistence at weeks 12, 26, and 52. The model included a hypothetical health plan with 100,000 elderly members. A total of 7096 vulnerable elderly subjects were identified as the model target population based on the percentage of vulnerable elderly and annual prevalence of OAB among vulnerable elderly. OAB-related costs included fesoterodine drug acquisition costs, healthcare resource use (inpatient hospitalization, outpatient visits, and physician office visits), and OAB-related co-morbidities (falls/fractures, urinary tract infections, depression, and nursing home admissions). All costs were inflated to 2013 US$ using the medical care component of the consumer price index (CPI).Results:When 7096 vulnerable elderly OAB patients were treated with fesoterodine, US healthcare payers could save $11,463,981 per year, or $1616 per patient vs no OAB pharmacotherapy. Univariate one-way sensitivity analyses supported the robustness of the findings and showed results were most sensitive to changes in fesoterodine efficacy followed by annual costs of inpatient hospitalization.Conclusions:From a US payer perspective, treating vulnerable elderly OAB patients with fesoterodine was cost-saving compared to no OAB pharmacotherapy.

Determining the quality and effectiveness of surgical spine care: patient satisfaction is not a valid proxy

Background contextGiven the unsustainable costs of the US health-care system, health-care purchasers, payers, and hospital systems are adopting the concept of value-based purchasing by shifting care away from low-quality providers or hospitals. Legislation now allows public reporting of these quality rankings. True measures of quality, such as surgical morbidity and validated questionnaires of effectiveness, are burdensome and costly to collect. Hence, patients' satisfaction with care has emerged as a commonly used metric as a proxy for quality because of its feasibility of collection. However, patient satisfaction metrics have yet to be validated as a measure of overall quality of surgical spine care. PurposeWe set out to determine whether patient satisfaction is a valid measure of safety and effectiveness of care in a prospective longitudinal spine registry. Study designProspective longitudinal cohort study. Patient populationAll patients undergoing elective spine surgery for degenerative conditions over a 6-month period at a single medical center. Outcome measuresPatient-reported outcome instruments (numeric rating scale [NRS], Oswestry disability index [ODI], neck disability index [NDI], short-form 12-item survey [SF-12], Euro-Qol-5D [EQ-5D], Zung depression scale, and Modified Somatic Perception Questionnaire [MSPQ] anxiety scale), return to work, patient satisfaction with outcome, and patient satisfaction with provider care. MethodsAll patients undergoing elective spine surgery for degenerative conditions over a 6-month period at a single medical center were enrolled into a prospective longitudinal registry. Data collected on all patients included demographics, disease characteristics, treatment variables, readmissions/reoperations, and all 90-day surgical morbidity. Patient-reported outcome instruments (NRS, ODI, NDI, SF-12, EQ-5D, Zung depression scale, and MSPQ anxiety scale), return to work, patient satisfaction with outcome, and patient satisfaction with provider care were recorded at baseline and 3 months after treatment. Receiver-operating characteristic (ROC) curve analysis was performed to determine whether extent of improvement in quality of life (SF-12 physical component summary [PCS]) and disability (ODI/NDI) accurately predicted patient satisfaction versus dissatisfaction. Standard interpretation of area under the curve (AUC) was used: less than 0.7, poor; 0.7 to 0.8, fair; and greater than 0.8, good accuracy. Multivariate logistic regression analysis was performed to determine if surgical morbidity (quality) or improvement in disability and quality of life (effectiveness of care) were independently associated with patient satisfaction. ResultsFour hundred twenty-two (84%) patients completed all questionnaires 3 months after surgery during the reviewed time period (mean age 55±14 years). Lumbar surgery was performed in 287 (68%) and cervical surgery in 135 (32%) patients. There were 51 (12.1%) 90-day complications, including 21 (5.0%) readmissions and 12 (2.8%) return to operating room. Three hundred fifty-eight (84.8%) patients were satisfied with provider care and 288 (68.2%) with their outcome. Satisfaction with provider care: In ROC analyses, extent of improvement in quality of life (SF-12) and disability (ODI/NDI) differentiated satisfaction versus dissatisfaction with care with very poor accuracy (AUC 0.49–0.69). In regression analysis, 3-month morbidity (odds ratio [95% confidence interval]: 1.45 [0.79–2.66]), readmission (0.66 [0.24–1.80]), improvement in quality of life (SF-12 PCS), or improvement in general health (health transition index) were not associated with satisfaction with care. Satisfaction with outcome: In ROC analyses, improvement in quality of life (SF-12) and disability (ODI/NDI) failed to differentiate satisfaction with good accuracy (AUC 0.76). Neither 90-day morbidity (1.05 [0.46–2.34]) nor 90-day readmission (0.27 [0.04–2.04]) was associated with satisfaction with outcome in regression analysis. ConclusionsPatient satisfaction is not a valid measure of overall quality or effectiveness of surgical spine care. Patient satisfaction metrics likely represent the patient's subjective contentment with health-care service, a distinct aspect of care. Satisfaction metrics are important patient-centered measures of health-care service but should not be used as a proxy for overall quality, safety, or effectiveness of surgical spine care.

Cost-effectiveness of dronedarone and standard of care compared with standard of care alone: US results of an ATHENA lifetime model.

BackgroundThe first antiarrhythmic drug to demonstrate a reduced rate of cardiovascular hospitalization in atrial fibrillation/flutter (AF/AFL) patients was dronedarone in a placebo-controlled, double-blind, parallel arm Trial to assess the efficacy of dronedarone 400 mg bid for the prevention of cardiovascular Hospitalization or death from any cause in patiENts with Atrial fibrillation/atrial flutter (ATHENA trial). The potential cost-effectiveness of dronedarone in this patient population has not been reported in a US context. This study assesses the cost-effectiveness of dronedarone from a US health care payers’ perspective.Methods and resultsATHENA patient data were applied to a patient-level health state transition model. Probabilities of health state transitions were derived from ATHENA and published data. Associated costs used in the model (2010 values) were obtained from published sources when trial data were not available. The base-case model assumed that patients were treated with dronedarone for the duration of ATHENA (mean 21 months) and were followed over a lifetime. Cost-effectiveness, from the payers’ perspective, was determined using a Monte Carlo microsimulation (1 million fictitious patients). Dronedarone plus standard care provided 0.13 life years gained (LYG), and 0.11 quality-adjusted life years (QALYs), over standard care alone; cost/QALY was $19,520 and cost/LYG was $16,930. Compared to lower risk patients, patients at higher risk of stroke (Congestive heart failure, history of Hypertension, Age ≥ 75 years, Diabetes mellitus, and past history of Stroke or transient ischemic attack (CHADS2) scores 3–6 versus 0) had a lower cost/QALY ($9580–$16,000 versus $26,450). Cost/QALY was highest in scenarios assuming lifetime dronedarone therapy, no cardiovascular mortality benefit, no cost associated with AF/AFL recurrence on standard care, and when discounting of 5% was compared with 0%.ConclusionsBy extrapolating the results of a large, multicenter, randomized clinical trial (ATHENA), this model suggests that dronedarone is a cost-effective treatment option for approved indications (paroxysmal/persistent AF/AFL) in the US.

Healthcare cost attributable to recently-diagnosed breast cancer in a privately-insured population in the United States

Objective:To evaluate breast cancer-associated healthcare cost from the payer perspective for the initial year after diagnoses of invasive breast cancer.Background:Breast cancer is the second most common malignancy in American women. While lifetime burden-of-care studies have reported spending between $20,000 and $100,000 per patient, previous studies have not outlined first year cost in managing this disease in recently diagnosed patients.Methods:This study was a retrospective, matched cohort study of privately-insured patients. Data were from a large US employers’ health claims database (January 2003–September 2008). Breast cancer cases were identified by ICD-9-CM diagnostic codes on index and confirmatory claims. A control group was identified with a ratio of 3:1, matched by demographic and health plan characteristics. Comorbidities were analyzed using the Charlson comorbidity index and AHRQ Comorbidity Software. A multivariate, log-linked, generalized linear model evaluated cost contributions of breast cancer in relation to demographic factors, comorbidities, and plan type.Results:The study included 35,057 cases and 105,171 matched controls (mean age 52 years). Common comorbidities included hypertension, diabetes, hypothyroidism, chronic pulmonary disease, and deficiency anemia. In the generalized linear model, the adjusted difference in total healthcare cost was $42,401 per patient within a year, with outpatient services responsible for most of this sum. Breast cancer-associated incremental annual costs per patient in inpatient, outpatient, and prescription categories were $5100, $37,231, and $1037, respectively.Limitations:These results may not be representative of the entire US, as data were derived from breast cancer patients with private, employer-based health insurance, and lacked covariates including race/ethnicity, education, income, and disease stage.Conclusions:Recently diagnosed breast cancer represents a substantial economic burden for US healthcare payers.

Incremental cost burden to US healthcare payers of atrial fibrillation/atrial flutter patients with additional risk factors

Atrial fibrillation (AF) and atrial flutter (AFL) patients often have cardiovascular (CV) comorbidities, and have an increased risk of hospitalization and death. Little is known about the real-world cost burden of AF/AFL patients with additional risk factors (ARF). We evaluated the medical resource use and cost burden of AF/AFL patients with ≥1 ARF (other than heart failure [HF]), in comparison with non-AF/AFL controls. This retrospective cohort study included patients from the MarketScan Medicare database who had ≥1 inpatient or ≥2 outpatient AF/AFL claims. Patients were (1) ≥75 years of age or (2) 70-74 years of age with ≥1 ARF (hypertension, diabetes, systemic embolism, or stroke/transient ischemic attack), but without HF. The AF/AFL patients were matched on age, gender, region, and enrollment status with non-AF/AFL patients. Hospital resource use and costs over the 12-month post-index period were compared across cohorts. The impacts of comorbidity were seen by subcategorizing hospitalization as all-cause, CV-related, and AF/AFL-related. AF/AFL patients with ≥1 ARF had a higher prevalence of comorbidity than non-AF/AFL patients (n=58,555/cohort). Hospitalizations (all-causality) were more than three times more frequent and of longer duration in AF/AFL patients with ≥1 ARF than in non-AF/AFL controls (mean [SD]: 0.72 [0.87] vs. 0.21 [0.51] hospitalizations per patient per year and 3.85 [9.30] and 1.03 [4.53] days, respectively; both P<0.0001). Overall mean (SD) costs over the 12-month post-index period were higher in AF/AFL patients with ≥1 ARF versus the non-AF/AFL control patients for inpatient ($9613 [25,407] vs. $2625 [11,597]; P<0.0001; incremental cost $6988), outpatient ($9447 [15,062] vs. $4906 [11,715]; P<0.0001; incremental cost $4541), and prescription drug costs ($3430 [3637] vs. $2618 [3374]; P<0.0001; incremental cost $812). AF/AFL patients with ≥1 ARF had significantly greater levels of comorbidity, hospitalizations, prescription, and outpatient claims than non-AF/AFL patients. The incremental costs of AF/AFL patients with ≥1 ARF are largely due to higher CV-related inpatient costs.