Quality-adjusted Life Months Research Articles

Cost-effectiveness of sacituzumab govitecan versus single-agent chemotherapy for metastatic triple-negative breast cancer: a trial-based analysis

BackgroundSacituzumab govitecan (SG) has recently been approved in China for the post-line treatment of metastatic triple-negative breast cancer (mTNBC). SG substantially improves progression-free survival and overall survival compared with single-agent chemotherapy for pretreated mTNBC. However, in view of the high price of SG, it is necessary to consider its value in terms of costs and outcomes. This study aimed to estimate the cost-effectiveness of SG versus single-agent treatment of physician’s choice (TPC) in the post-line setting for patients with mTNBC from a Chinese healthcare system perspective.MethodsThe cohort characteristics were sourced from the ASCENT randomized clinical trial, which enrolled 468 heavily pretreated patients with mTNBC between November 2017 and September 2019. A partitioned survival model was constructed to assess the long-term costs and effectiveness of SG versus TPC in the post-line treatment of mTNBC. Quality-adjusted life-months (QALMs) and total costs in 2022 US dollars were used to derive incremental cost effectiveness ratio (ICER). QALMs and costs were discounted at 5% annually. The willingness-to-pay (WTP) threshold was defined as $3188 per QALM, three times China’s average monthly per capita gross domestic product in 2022. One-way sensitivity analysis, probabilistic sensitivity analysis, and scenario analyses were performed to estimate the robustness of the results.ResultsTreatment with SG yielded an incremental 5.17 QALMs at a cost of $44,792 per QALM, much above the WTP threshold of $3188/QALM in China. One-way sensitivity analysis showed that SG price was a crucial factor in the ICER. Probabilistic sensitivity analysis revealed that the cost-effective acceptability of SG was 0% in the current setting. Scenario analyses indicated that the result was robust in all subgroups in ASCENT or under different time horizons. Furthermore, SG must reduce the price to enter the Chinese mainland market. When the monthly cost of SG reduce to $2298, SG has about 50% probability to be a preferred choice than TPC.ConclusionsSG was estimated to be not cost-effective compared with TPC for post-line treatment for mTNBC in China by the current price in HK under a WTP threshold of $3188 per QALM. A drastic price reduction is necessary to improve its cost-effectiveness.

QOL-02. MONITORING GBM PATIENT HEALTH-RELATED QUALITY OF LIFE AND QUALITY ADJUSTED LIFE MONTHS DURING INDIVIDUALIZED MULTIMODAL IMMUNOTHERAPY USING EQ-5D-5L

Abstract DC vaccination emerges as effective treatment for patients with GBM. Besides overall survival, Health-Related Quality of Life (HRQoL) during and after treatment is an important qualitative assessment. We explored the usability of the self-reporting EQ-5D-5L instrument (), available in 14 languages, to determine the Health Utility Index (HUI) and calculate Quality Adjusted Life Months (QALM) in GBM patients during individualized multimodal immunotherapy (IMI). Selection criteria for this retrospective analysis were the start of IMI between 15 October 2021 and May 2023, and availability of at least 5 HRQoL measurements with EQ-5D-5L. Nineteen GBM patients (9 females, 10 males) met these criteria. Median age at diagnosis was 52 years (range 21-67). Median Karnofsky performance index was 80 (60-100). The HUI at time of first diagnosis was unknown. The median HUI at start of immunotherapy was 0.868 (1-0.346). Fifteen patients received IMI as part of first-line treatment. Four patients were treated at time of relapse. Patients received in median 2 IO-Vac® vaccines (0-3) and 6 immunogenic cell death immunotherapy courses (4-12). Sixteen patients were still alive in median 11 months (6-48) after first diagnosis. Three patients died respectively 11, 16, 34 months after diagnosis. The patient response rate to the EQ-5D-5L instrument was high. A median of 7 HRQoL measurements were registered per patient (5-15). One patient did not appropriately answer the EQ-5D-5L questionnaire due to active refusal. The median life time between start of IMI and last time point for measurement of HRQoL was 12.5 months (7.8-47.5). The Quality Adjusted life time between each time point was calculated based on the HUI for each patient. The life time reduction was -0.9 months (-4.2-0), resulting in a median QALM of 12 months (6.3-45.9). EQ-5D-5L can be used to determine the QALMs as qualitative basis for cost-effectiveness calculations for innovative treatments like IMI.

Short-term locally advanced gastric cancer management: A cost-effectiveness analysis.

e16065 Background: Across the nation, patients with locally advanced gastric cancer (LAGC) are managed with modalities including upfront surgery (US), neoadjuvant chemoradiation therapy (NCRT), and perioperative chemotherapy (PCT). Treatment modalities themselves are associated with adverse effects which can lead to difficulty with treatment adherence in the short-term. As preoperative therapies have demonstrated survival benefits over US, long-term outcomes are expected to vary, but it is not known if differences in adherence impacts short-term treatment outcomes. We sought to perform a decision analysis comparing costs and quality-of-life associated with short-term outcomes in the treatment of patients with LAGC to identify the most cost-effective option. Methods: We designed a decision tree model to investigate the survival and costs associated with the three utilized management modalities for T2 or higher gastric cancer in the United States: US, PCT, and NCRT. The tree described costs and treatment strategies over a six-month time horizon to incorporate the complete treatment regimen and various treatment failure points encountered during therapy. Costs were derived from 2022 Medicare reimbursement rates using the third-party payer perspective for physicians and hospitals. Effectiveness was represented using quality-adjusted life months (QALMs). One QALM equates to one month of perfect health, while zero QALMs is equivalent to death. One-way, two-way, and probabilistic sensitivity analyses were utilized to test the robustness of our findings. Results: PCT was the most cost-effective treatment modality for LAGC over US and NCRT with a cost of $40,792.16 with 4.14 QALMs. US had a cost of $55,575.57 with 4.20 QALMs. NCRT was dominated by the other options with a cost of $56,510.64 with 3.70 QALMs. Across 100,000 Monte-Carlo simulations, 55.2% of trials favored PCT and the remainder favored US (44.8%). NCRT was not favored in the probabilistic sensitivity analysis. Finally, one-way and two-way sensitivity analyses did not identify any situations where alternative treatment modalities were preferred over PCT. Conclusions: In our simulated patients with diagnosed gastric cancer, the most cost-effective treatment strategy was PCT. This further confirms the preference of this modality over others in LAGC. While US demonstrated improved QALMs over PCT, the associated cost was too great to justify the benefits. [Table: see text]

Cost-effectiveness of tapentadol immediate release versus oxycodone immediate release for acute post-operative pain after major hip surgeries

Objective To evaluate the cost-effectiveness of tapentadol immediate-release (IR) versus oxycodone IR for post-operative pain after a major hip surgery. Methods This study has been conducted using an Australian societal perspective, focusing on adult patients after a major hip surgery. A cost-effectiveness analysis was conducted using a decision-analytic model. The model incorporated drug and other resource costs, the probability of opioid-related adverse events, and quality-adjusted life months (QALM) in each treatment arm. A willingness to pay (WTP) threshold of AU$2500 was used per QALM gained. A probabilistic sensitivity analysis was conducted to examine the uncertainty of the assumptions. The primary outcome was the incremental cost-effectiveness ratio (ICER) of tapentadol IR versus oxycodone IR, expressed as Australian dollars (AU$) per QALM gained. Results Tapentadol IR dominated oxycodone IR, with a cost savings of AU$201 and an increase in QALM by 0.014. The ICER was −13,946 AU$/QALM (negative value attributed to numerator). In the probabilistic sensitivity analysis, 84.2% of the simulations were in favour of tapentadol IR at the WTP threshold. Conclusions Tapentadol IR may be more cost-effective than oxycodone IR for the treatment of acute postoperative pain after major hip surgeries.

Optimizing the management of patients with small renal masses in a Canadian context: A Markov decision-analysis model.

The management of patients with a small renal mass (SRM) varies significantly. The objective of this study was to determine which initial management strategy resulted in the greatest quality-adjusted life months (QALM) for an index patient with a SRM. A Markov decision analysis was used to determine the effect of 1) treating patients with a partial nephrectomy (PN); 2) active surveillance (AS); and 3) renal mass biopsy on QALM over a 10-year horizon. All relevant health states were modelled. Biopsy sensitivity and specificity were modelled assuming an 80% prevalence of cancer using procedural pathology as the gold standard. Health state utilities were obtained from the Tufts Medical Centre Cost-Effective Analysis Registry. Deterministic sensitivity analyses were used to test key assumptions. Over a 10-year time horizon for a 70-year-old male with a 2 cm SRM, the biopsy strategy resulted in 38.07 QALM, whereas treating all patients with PN resulted in 37.69 QALM and AS in 36.25 QALM. The model was most sensitive to the probability that a patient would remain alive at baseline. Biopsy was the preferred strategy when sensitivity was greater than 77%. As the underlying probability of cancer increased, the threshold of renal mass biopsy sensitivity to still favor biopsy increased. Renal mass biopsy is the preferred initial management strategy for an index patient with a SRM to optimize QALM. When the probability of cancer is high, centers should aim for a sensitivity of at least 77% in order to consider a biopsy as the first strategy.

Cost-Utility Analysis of Dolutegravir- Versus Efavirenz-Based Regimens as a First-Line Treatment in Adult HIV/AIDS Patients in Ethiopia.

BackgroundIn several countries, the dolutegravir (DTG)-based regimen is generally preferred as first-line antiretroviral therapy (ART) over the efavirenz (EFV)-based regimen, but the evidence in low-income countries is limited.ObjectiveOur study aimed to evaluate the cost effectiveness of DTG- versus EFV-based first-line human immunodeficiency virus (HIV) treatment in Ethiopia.MethodsWe developed a microsimulation model for the progression of HIV/acquired immune deficiency syndrome (AIDS) to examine the cost effectiveness of DTG-based first-line ART compared with an EFV-based regimen from a healthcare payer perspective. We used a lifetime horizon with a 1-month cycle length and a 3% annual discount rate. The primary outcomes were a lifetime cost in US dollars ($), quality-adjusted life-months (QALMs) that converted to QALYs using the formula QALY = QALM/12, and incremental cost-effectiveness ratio (ICER). Deterministic sensitivity analysis was conducted to account for parameter uncertainty.ResultsCompared with the EFV-based regimen, the DTG-based regimen was associated with an expected lifetime cost of $12,709 (vs. $12,701) and expected QALYs of 15.3 (vs. 14.7 QALYs) per patient, resulting in an ICER value of $13.33 per QALY. From an alternative analysis with a 5-year time horizon, DTG-based ART was found to be dominant, with expected gains of 0.17 QALYs at a lower cost of $1 per patient. The deterministic sensitivity analysis depicted that the maximum increase in ICER value was $72 per QALY, and all ICER values were below the estimated threshold value.ConclusionsThe DTG-based first-line regimen appears to be cost effective compared with the EFV-based regimen for the treatment of HIV/AIDS patients in an Ethiopian setting.

Pharmacoeconomic Evaluation of Erlotinib for the Treatment of Pancreatic Cancer

PurposeTo evaluate the cost-effectiveness of gemcitabine and gemcitabine plus erlotinib as first-line treatments for advanced pancreatic cancer. MethodsOn the basis of the Gemcitabine With/Out Erlotinib in Unresectable Locally Advanced/Metastatic Pancreatic Cancer (PA.3) trial, the Markov model was constructed to simulate the development of advanced pancreatic cancer. Cost-effectiveness analysis was used to determine the economic level of the treatments, according to the willingness-to-pay (WTP) threshold. The sensitivity analysis was conducted for cost-effectiveness and other indexes. FindingsThe results of the cost-effectiveness analysis revealed that the cost-effectiveness ratios for the first-line treatment of advanced pancreatic cancer were ¥60,492.78 (US$8892.44/€7568.88) per 6.34 quality-adjusted life-months (QALMs) for gemcitabine and ¥99,595.39 (US$14,640.52/€12,461.42) per 7.02 QALMs for gemcitabine plus erlotinib. The incremental cost-effectiveness of the 2 regimens was ¥57,503.84 ($8453.06/€7194.90) per QALM, which was higher than the WTP set in this study (¥16,161 [$2375.66/€2022.07] per QALM). The results of the sensitivity analysis indicate that the analysis results were stable. Gemcitabine was more cost-effective than gemcitabine plus erlotinib. ImplicationsCompared with gemcitabine, gemcitabine plus erlotinib was not cost-effective at the level of the WTP. Gemcitabine plus erlotinib therapy has no economic significance as a first-line medical treatment for pancreatic cancer.

Cost-effectiveness of concomitant and adjuvant temozolomide for glioblastoma patients with unmethylated O6-methylguanine-DNA methyltransferase promoter regions in the United States.

2058 Background: Promoter region methylation of the O6-methylguanine-DNA methyltransferase (MGMT) gene has emerged as a predictive factor for longer overall (OS) and progression-free (PFS) survival response to concomitant and adjuvant temozolomide (TMZ) chemotherapy in glioblastoma. Recently, several investigators have suggested the small survival benefit of TMZ for unmethylated patients is insufficient to warrant the increased risk of adverse events and that these patients should instead be enrolled in clinical trials. This study informs the debate by estimating the incremental costs and benefits associated with TMZ use in methylated and unmethylated patients based on available published evidence. Methods: Published OS and PFS Kaplan-Meier curves were digitized, parameterized, and extrapolated to ten years. Partitioned Survival Analysis trees were constructed in TreeAge Pro 2020. Costs of TMZ, RT, and inpatient hospitalization were included, among others. It was assumed that all patients receive surgery prior to beginning radiation, and that RT and TMZ (six adjuvant cycles) dosing was standard. Published utilities associated with the progression-free and post-progression health states, subject to each treatment, were used. A 3% annual discount rate and a willingness-to-pay of $150,000/quality-adjusted life year (QALY) were assumed. Probabilistic sensitivity analysis (PSA) was conducted. Results: In methylated patients, RT + TMZ versus RT is associated with $67,622 in additional costs and 10.91 additional quality-adjusted life months (QALMs). In unmethylated patients, RT + TMZ versus RT is associated with $9,203 in additional costs and 2.24 additional QALMs. The implied incremental cost effectiveness ratios (ICERs) support TMZ use in both populations and suggest it is more favorable for unmethylated patients ($74,378/QALY and $49,302/QALY for methylated and unmethylated patients, respectively). However, the incremental net monetary benefit (INMB) associated with TMZ use is substantially smaller for unmethylated patients ($18,797 versus $68,753) due to the smaller survival gains for this population. PSA results indicate that TMZ is more likely to be cost-effective for methylated (89.3%) than unmethylated (77.4%) patients. Conclusions: Post-radiation clinical trial enrollment should be considered instead of TMZ for unmethylated glioblastoma patients. While the estimated ICER justifies the use of TMZ for unmethylated patients – in fact, does so more strongly than for methylated patients – the relatively small incremental health benefit is plausibly outweighed by the opportunity cost of not enrolling these patients in clinical trials that offer the possibility of more substantive gains to current and future glioblastoma patients.

PCN213 Health IMPACT of Optimizing the Time to Patient Access

Prolonged time between marketing authorization and reimbursement leads to missed health gains, raising the question: what could be gained by ensuring earlier reimbursement? A health impact model was developed for two selected oncological drugs to calculate the impact of earlier reimbursement on the number of patients that could have been treated and accompanying health gains. Health impact outcome measures were obtained from national assessment dossiers and could differ per drug and country. Time to reimbursement was optimized in three hypothetical scenarios: 1) timing of reimbursement coinciding with EC marketing authorization (as is the case in Germany; ambitious scenario), 2) as fast as the fastest country (best practice), and 3) assessments within 180 days after the EC marketing authorization, unless faster in reality (as described in the EC Transparency Directive; base case). The number of new patients per month (uptake data) were retrieved from the respective companies marketing these drugs. Only countries with recorded uptake data for at least 1 year after reimbursement were used in the analysis. Midostaurin and pertuzumab and four European countries (England, Italy, the Netherlands, and Sweden) were included in the analysis. In the ambitious scenario, 1,689 more AML patients could have been treated with midostaurin in England, Italy, the Netherlands, and Sweden, accounting for 82,920 life months gained (LMG) and 8,665 months of event-free survival. With pertuzumab, 2,180 patients could have been treated in England, Netherlands, and Sweden, accounting for 12,718 LMG and 8,842 quality-adjusted life months gained. For the countries that lacked (sufficient) uptake data, even more health gains could be expected. Drugs gaining positive reimbursement decision are apparently worthwhile for patient- and population’s health, needing a quick access. Today, time to reimbursement varies greatly between countries, demonstrating the need for optimization. This analysis quantified the health gains demonstrating the urgency of rapid patient access.

Cost-effectiveness Analysis of Preoperative Screening Strategies for Obstructive Sleep Apnea among Patients Undergoing Elective Inpatient Surgery.

Obstructive sleep apnea is underdiagnosed in surgical patients. The cost-effectiveness of obstructive sleep apnea screening is unknown. This study's objective was to evaluate the cost-effectiveness of preoperative obstructive sleep apnea screening (1) perioperatively and (2) including patients' remaining lifespans. An individual-level Markov model was constructed to simulate the perioperative period and lifespan of patients undergoing inpatient elective surgery. Costs (2016 Canadian dollars) were calculated from the hospital perspective in a single-payer health system. Remaining model parameters were derived from a structured literature search. Candidate strategies included: (1) no screening; (2) STOP-Bang questionnaire alone; (3) STOP-Bang followed by polysomnography (STOP-Bang + polysomnography); and (4) STOP-Bang followed by portable monitor (STOP-Bang + portable monitor). Screen-positive patients (based on STOP-Bang cutoff of at least 3) received postoperative treatment modifications and expedited definitive testing. Effectiveness was expressed as quality-adjusted life month in the perioperative analyses and quality-adjusted life years in the lifetime analyses. The primary outcome was the incremental cost-effectiveness ratio. In perioperative and lifetime analyses, no screening was least costly and least effective. STOP-Bang + polysomnography was the most effective strategy and was more cost-effective than both STOP-Bang + portable monitor and STOP-Bang alone in both analyses. In perioperative analyses, STOP-Bang + polysomnography was not cost-effective compared to no screening at the $4,167/quality-adjusted life month threshold (incremental cost-effectiveness ratio $52,888/quality-adjusted life month). No screening was favored in more than 90% of iterations in probabilistic sensitivity analyses. In contrast, in lifetime analyses, STOP-Bang + polysomnography was favored compared to no screening at the $50,000/quality-adjusted life year threshold (incremental cost-effectiveness ratio $2,044/quality-adjusted life year). STOP-Bang + polysomnography was favored in most iterations at thresholds above $2,000/quality-adjusted life year in probabilistic sensitivity analyses. The cost-effectiveness of preoperative obstructive sleep apnea screening differs depending on time horizon. Preoperative screening with STOP-Bang followed by immediate confirmatory testing with polysomnography is cost-effective on the lifetime horizon but not the perioperative horizon. The integration of preoperative screening based on STOP-Bang and polysomnography is a cost-effective means of mitigating the long-term disease burden of obstructive sleep apnea.

COVID‐19 Screening with Chest CT in Acute Stroke Imaging: A Clinical Decision Model

ABSTRACTBACKGROUND AND PURPOSEAcute stroke patients may have undiagnosed coronavirus disease 2019 (COVID‐19) infection, transmissible to medical professionals involved in their care. Our aim was to determine the value of incorporating a chest computed tomography (CT) scan during acute stroke imaging, and the factors that influence this decision.METHODSWe constructed a probabilistic decision tree of the value of acquiring a chest CT scan or not, expressed in quality‐adjusted life months (QALM) of patients and medical professionals. The model was based on the chance of detecting infection by chest CT scan, the case fatality rates of COVID‐19 infection, the risk of COVID‐19 infection after exposure, the expected proportion of medical professionals exposed, and the exposure reduction derived from early disease detection.RESULTSThe decision to incorporate the chest CT scan was superior to not doing so (12.00 QALM vs 11.99 QALM, respectively), when the probability of patients having undetected COVID‐19 infection is 3.5%, potentially exposing 100% of medical professionals, and if early detection reduces exposure by 50%. The risk of developing symptomatic COVID‐19 infection following exposure casts uncertainty on the results, but this is offset by the potential for reducing exposure.CONCLUSIONSWe identified a measurable benefit of incorporating a chest CT into the urgent imaging protocol of acute stroke patients in reducing exposure of medical professionals without appropriate precautions. The clinical impact of this benefit, however, may not be materially significant.

Budget impact of sequential treatment with first-line afatinib versus first-line osimertinib in non-small-cell lung cancer patients with common EGFR mutations

BackgroundThe therapeutic landscape for non-small-cell lung cancer (NSCLC) patients that have common epidermal growth factor receptor (EGFR) mutations has changed radically in the last decade. The availability of these treatment options has an economic impact, therefore a budget impact analysis was performed.MethodsA budget impact analysis was conducted from a Dutch healthcare perspective over a 5-year time horizon in EGFR-mutant NSCLC patients receiving first-line afatinib (Gilotrif®) versus first-line osimertinib (Tagrisso®), followed by subsequent treatments. A decision analysis model was constructed in Excel. Scenario analyses and one-way sensitivity analysis were used to test the models’ robustness.ResultsSequential treatment with afatinib versus first-line treatment with osimertinib showed mean total time on treatment (ToT) of 29.1 months versus 24.7 months, quality-adjusted life months (QALMs) of 20.2 versus 17.4 with mean cost of €108,166 per patient versus €143,251 per patient, respectively. The 5-year total budget impact was €110.4 million for the afatinib sequence versus €158.6 million for the osimertinib sequence, leading to total incremental cost savings of €48.15 million.ConclusionsFirst-line afatinib treatment in patients with EGFR-mutant NSCLC had a lower financial impact on the Dutch healthcare budget with a higher mean ToT and QALM compared to osimertinib sequential treatment.

Cost-effectiveness of niraparib, rucaparib, and olaparib for treatment of platinum-resistant, recurrent ovarian carcinoma

BackgroundOlaparib was approved on December 19, 2014 by the US FDA as 4th-line therapy (and beyond) for patients with germline BRCA1/2 mutations; rucaparib was approved on December 19, 2016 as 3rd-line therapy (and beyond) for germline or somatic BRCA1/2-mutated recurrent disease. On October 23, 2019, niraparib was approved for treatment of women with damaging mutations in BRCA1/2 or other homologous recombination repair genes who had been treated with three or more prior regimens. We compared the cost-effectiveness of PARPi(s) with intravenous regimens for platinum-resistant disease. MethodsMedian progression-free survival (PFS) and toxicity data from regulatory trials were incorporated in a model which transitioned patients through response, hematologic complications, non-hematologic complications, progression, and death. Using TreeAge Pro 2017, each PARPi(s) was compared separately to non‑platinum-based and bevacizumab-containing regimens. Costs of IV drugs, managing toxicities, infusions, and supportive care were estimated using 2017 Medicare data. Incremental cost-effectiveness ratios (ICERs) were calculated and PFS was reported in quality adjusted life months for platinum-resistant populations. ResultsNon‑platinum-based intravenous chemotherapy was most cost effective ($6,412/PFS-month) compared with bevacizumab-containing regimens ($12,187/PFS-month), niraparib ($18,970/PFS-month), olaparib ($16,327/PFS-month), and rucaparib ($16,637/PFS-month). ICERs for PARPi(s) were 3–3.5× times greater than intravenous non‑platinum-based regimens. ConclusionHigh costs of orally administered PARPi(s) were not mitigated or balanced by costs of infusion and managing toxicities of intravenous regimens typically associated with lower response and shorter median PFS. Balancing modest clinical benefit with costs of novel therapies remains problematic and could widen disparities among those with limited access to care.

Pulse oximetry screening for critical congenital heart defects in Ontario, Canada: a cost-effectiveness analysis.

Previously conducted cost-effectiveness analyses of pulse oximetry screening (POS) for critical congenital heart defects (CCHDs) have shown it to be a cost-effective endeavour, but the geographical setting of Ontario in relation to its vast yet sparsely populated regions presents unique challenges. The objective of this study was to estimate the cost-effectiveness of POS for CCHD in Ontario, Canada. A cost-effectiveness analysis, comparing POS to no POS, was conducted from the Ontario healthcare payer perspective using a Markov model. The base case was defined as a well-appearing newborn at 24h of age. Outcome measures, including quality-adjusted life months (QALMs), lifetime costs, and incremental cost-effectiveness ratios (ICER) [ΔCost/ΔQALMs], were calculated over a lifetime horizon. All outcomes were discounted at 1.5% per year. Cost-effectiveness was assessed using an a priori ICER threshold of CAD$4166.67 per QALM (equivalent to CAD$50,000 per quality-adjusted life year). Deterministic and probabilistic sensitivity analyses were conducted to assess parameter uncertainty. Implementation of POS is expected to lead to timely diagnosis of 51 CCHD cases annually. The incremental cost of performing POS was estimated to be $27.27 per screened individual, with a gain of 0.02455 QALMs. This yielded an ICER of CAD$1110.79 per QALM, well below the pre-determined threshold. The probabilistic sensitivity analysis estimated a 92.3% chance of routine implementation of POS being cost-effective. Routine implementation of POS for CCHD in Ontario is expected to be cost-effective.

Cost-Effectiveness Analysis of the Surgical Management of Infants Less than One Year of Age with Feeding Difficulties

BackgroundWe compared the cost-effectiveness of the common surgical strategies for the management of infants with feeding difficulty. MethodsInfants with feeding difficulty undergoing gastrostomy alone (GT), GT and fundoplication, or gastrojejunostomy (GJ) tube were enrolled between 2/2017 and 2/2018. A validated GERD symptom severity questionnaire (GSQ) and visual analog scale (VAS) to assess quality of life (QOL) were administered at baseline, 1 month, and every 6 months. Data collected included demographics, resource utilization, diagnostic studies, and costs. VAS scores were converted to quality adjusted life months (QALMs), and costs per QALM were compared using a decision tree model. ResultsFifty patients initially had a GT alone (71% laparoscopically), and one had a primary GJ. Median age was 4 months (IQR 3–8 months). Median follow-up was 11 months (IQR 5–13 months). Forty-three did well with GT alone. Six (12%) required conversion from GT to GJ tube, and one required a fundoplication. Of those with GT alone, six (14%) improved significantly so that their GT was removed after a mean of 7 ± 3 months. Overall, the median GSQ score improved from 173 at baseline to 18 after 1 year (p < 0.001). VAS scores also improved from 70/100 at baseline to 85/100 at 1 year (p < 0.001). ED visits (59%), readmissions (47%), and clinic visits (88%) cost $58,091, $1,442,139, and $216,739, respectively. GJ tube had significantly higher costs for diagnostic testing compared to GT (median $8768 vs. $1007, p < 0.001). Conversion to GJ tube resulted in costs of $68,241 per QALM gained compared to GT only. ConclusionsMost patients improved with GT alone without needing GJ tube or fundoplication. GT and GJ tube were associated with improvement in symptoms and QOL. GJ tube patients reported greater gains in QALMS but incurred higher costs. Further analysis of willingness to pay for each additional QALM will help determine the value of care. Study and level of evidenceCost-effectiveness study, Level II.

Cost-effectiveness and budget impact of the management of uncomplicated urinary tract infection by community pharmacists

BackgroundUrinary tract infections (UTI) are one of the most common infections treated in primary care and the emergency department. The RxOUTMAP study demonstrated that management of uncomplicated UTI by community pharmacists resulted in high clinical cure rates similar to those reported in the literature and a high degree of patient satisfaction. The objective of this study was to assess the cost-effectiveness and budget impact of community pharmacist-initiated compared to family or emergency physician-initiated management of uncomplicated UTI.MethodsA decision analytic model was used to compare costs and outcomes of community pharmacist-initiated management of uncomplicated UTI to family or emergency physician-initiated management. Cure rates and utilities were derived from published studies. Costs of antibiotic treatment and health services use were calculated based on cost data from Canada. We used a probabilistic analysis to evaluate the impact of treatment strategies on costs and quality-adjusted-life-months (QALMs). In addition, a budget impact analysis was conducted to evaluate the financial impact of community pharmacist-initiated uncomplicated UTI management in this target population. This study was conducted from the perspective of the public health care system of Canada.ResultsPharmacist-initiated management was lower cost ($72.47) when compared to family and emergency physician-initiated management, $141.53 and $368.16, respectively. The QALMs gained were comparable across the management strategies. If even only 25% of Canadians with uncomplicated UTI were managed by community pharmacists over the next 5 years, the resulting net total savings was estimated at $51 million.ConclusionFrom a Canadian public health care system perspective, community pharmacist-initiated management would likely be a cost-effective strategy for uncomplicated UTI. In an era of limited health care resources, expanded roles of community pharmacists or other non-physician community based prescribers are important mechanisms through which accessible, high-quality and cost-effective care may be achieved. Further studies to evaluate other conditions which can be managed in the community and their cost effectiveness are essential.

Cost-effectiveness analysis of long-course oxaliplatin and bolus of fluorouracil based preoperative chemoradiotherapy vs. 5x5Gy radiation plus FOLFOX4 for locally advanced resectable rectal cancer

PurposeTo evaluate the cost-effectiveness of preoperative short-course radiotherapy (SCRT, 5 × 5 Gy) plus FOLFOX4 versus long-course oxaliplatin and bolus of fluorouracil based preoperative long-course chemoradiotherapy (LCCRT, 50.4 Gy in 28 fractions) in the management of cT4 or advanced cT3 rectal cancer (RC), both of which have been reported to achieve similar clinical effect in the NCT00833131 trial.Materials and methodsA Markov decision-analytic model compared SCRT plus chemotherapy and LCCRT, by simulating three health states (disease-free survival (DFS), progressive disease (PD) and death). The primary outcomes were quality-adjusted life months (QALMs), costs, and incremental cost-effectiveness ratios (ICERs). Transition probabilities were based on the NCT00833131 trial. The costs were calculated from a Chinese payers’ perspective. Strategies were evaluated with a willingness-to-pay (WTP) threshold of $2370.47 (3 × GDP) per QALM gained. Sensitivity analysis was performed to model uncertainty in these parameters.ResultsThe overall costs for SCRT plus chemotherapy and LCCRT were $78,937 and $38,140 with effectiveness of 29.92 QALMs and 22.99 QALMs, respectively. SCRT plus chemotherapy increased costs and QALM by $40,797.34 and 6.93 compared to LCCRT, resulting in an ICER of $5884.56/QALM gained. In the DFS state, the whole cost for SCRT plus chemotherapy and LCCRT were $11,490.03 and $10,794.06 with an effectiveness of 21.70 QALMs and 19.65 QALMs, respectively. SCRT plus chemotherapy increased cost and QALM by $695.97 and 2.05 compared to LCCRT, resulting in a ICER of $339.50/QALM gained, which below the WTP. The utility associated with the DFS state was the most influential factor on the cost-effectiveness of SCRT plus chemotherapy. When the cost of PD state below $1920, the ICER of SCRT compared with LCCRT below the WTP.ConclusionCompared with LCCRT, SCRT plus chemotherapy is a more cost-effective strategy for locally advanced resectable RC in the DFS state as well as in the all states when the cost of PD state below $1920.

Comparison of neoadjuvant and adjuvant therapy for resectable pancreatic cancer using Markov decision modeling.

448 Background: Meta-analysis of smaller studies have shown that neoadjuvant chemotherapy is more beneficial for patients with resectable pancreatic cancer than upfront surgery by comparing life expectancy (LE) and quality-adjusted life expectancy (QALE) computed from Markov models. The study results utilized literature data using several small clinical trials but no individual patient data was used and only gemzar based therapy was studied. Methods: Markov model was used to calculate the LE and QALE for adjuvant and neoadjuvant chemotherapy and individual patient parameters was used in the model to refine certain clinical outcome datapoints. We used 278 patients pancreatic cancer data from 2008 to 2017 from Stony Brook University and used the literature data from randomized clinical trials studying gemzar (GEM), gemzar and capecitabine (GEM+CAP) and modified FOLFIRINOX (mFOL). The median OS for each model was obtained by computer simulation. Results: Intensive adjuvant chemotherapy using mFOL had best simulation outcome with median OS (52.5 months), LE (81.5 months), and QALE (65.0 quality-adjusted life months) compared to using GEM (40.5, 66.5, and 52.9 months for median OS, LE, and QALE), GEM+CAP (16.5, 28.0, and 21.9 months for median OS, LE, and QALE), and 5-FU (16.5, 26.9, and 21.1 months for median OS, LE, and QALE). The neoadjuvant chemotherapy approach improved LE and QALE but not in median OS when compared to adjuvant therapy. Conclusions: Mathematical modeling confirms the improved clinical outcome for modified FOLFIRINOX in resectable pancreatic cancer. The benefit of neoadjuvant chemotherapy approach suggest further clinical trials are needed to determine the better treatment strategy for pancreatic cancer patients.

Optimal timing of hepatitis C treatment among HIV/HCV coinfected ESRD patients: Pre- vs posttransplant.

Patients with end-stage renal disease (ESRD) who are coinfected with hepatitis C virus (HCV) and human immunodeficiency virus (HIV) have access to effective treatment options for HCV infection. However, they also have access to HCV-infected kidneys, which historically afford shorter times to transplantation. Given the high waitlist mortality and rapid progression of liver fibrosis among coinfected kidney-only transplant candidates, identification of the optimal treatment strategy is paramount. Two strategies, treatment pre- and posttransplant, were compared using Monte Carlo microsimulation of 1000000 candidates. The microsimulation was stratified by liver fibrosis stage at waitlist addition and wait-time over a lifetime time horizon. Treatment posttransplant was consistently cost-saving as compared to treatment pretransplant due to the high cost of dialysis. Among patients with low fibrosis disease (F0-F1), treatment posttransplant also yielded higher life months (LM) and quality-adjusted life months (QALM), except among F1 candidates with wait times ≥ 18months. For candidates with advanced liver disease (F2-F4), treatment pretransplant afforded more LM and QALM unless wait time was <18months. Moreover, treatment pretransplant was cost-effective for F2 candidates with wait times >71months and F3 candidates with wait times >18months. Thus, optimal timing of HCV treatment differs based on liver disease severity and wait time, favoring pretransplant treatment when cirrhosis development prior to transplant seems likely.

Nivolumab in the Treatment of Metastatic Renal Cell Carcinoma: A Cost-Utility Analysis.

Nivolumab improves overall survival and health-related quality of life compared with everolimus in metastatic renal cell carcinoma (mRCC). This study assesses the cost-utility of nivolumab from the Canadian health care payer perspective. To evaluate the cost-utility of nivolumab, a Markov cohort model that incorporated data from the phase 3 CheckMate-025 trial and other sources was developed. The incremental cost per quality-adjusted life month (QALM) gained for nivolumab was calculated. A lifetime horizon was used in the base-case with costs and outcomes discounted 3% annually. The probabilities of progression and death from cancer and utility values were captured from the CheckMate-025 trial. Expected costs were based on Ontario fees and other sources. Scenario and sensitivity analyses were conducted to assess uncertainty. Compared with everolimus, nivolumab provided an additional 4.2 QALM at an incremental cost of $34,153. The resulting incremental cost-effectiveness ratio was $8138/QALM gained. Assuming a willingness to pay (WTP) threshold of $4167/QALM ($50,000/quality-adjusted life-year [QALY]), nivolumab was not cost-effective. In 1-way sensitivity analyses, nivolumab cost, median overall survival, and median treatment duration were sensitive to changes. Furthermore, the results were sensitive to the WTP threshold and nivolumab became a cost-effective strategy with a WTP of $8333/QALM ($100,000/QALY). Compared with everolimus, nivolumab is unlikely to be cost-effective for the treatment of mRCC from a Canadian health care perspective with its current price assuming a WTP of $50,000/QALY. Although mRCC patients derive a meaningful clinical benefit from nivolumab, considerations should be given to avoid drug wastage and increase the WTP threshold to render this strategy more affordable.