Quality-adjusted Life Years For Patients Research Articles

Pembrolizumab alone or with chemotherapy for squamous cell carcinoma of the head and neck: A cost-effectiveness analysis from Chinese perspective.

The KEYNOTE-048 trial indicated pembrolizumab plus chemotherapy is an appropriate first-line treatment for recurrent or metastatic head and neck squamous cell carcinoma (HNSCC), while pembrolizumab monotherapy is optimal for PD-L1 positive patient. This study was powered to determine the most cost-effective strategy for patient with different combined positive score (CPS). A Markov model was developed to predict progression-free survival, disease progression, or death in patients with recurrent or metastatic HNSCC based on data from the KEYNOTE-048 trial. Cost was obtained from West China Hospital, while utilities were referred to published studies. By using Monte Carlo simulations, acceptability curves were depicted to address the uncertainty of model inputs. Compared with cetuximab plus chemotherapy, pembrolizumab monotherapy resulted in an incremental cost-effectiveness ratio (ICER) of $14,995 per quality adjusted life year (QALY) in total population and $22,779 per QALY in patients with CPS≥1. Comparing pembrolizumab plus chemotherapy with standard therapy led to an ICER of $43,230 per QALY in total population and $26,157 per QALY in patients with CPS≥1. For patients with CPS≥20, ICERs yield by immunotherapy with or without chemotherapy exceeded the threshold of willingness to pay we set, when compared with standard therapy. Pembrolizumab plus chemotherapy was dominated by pembrolizumab alone in this patient population. For HNSCC patients with different CPS, pembrolizumab alone was the optimal choice for total population and patients with CPS≥1. Among patients with high CPS, immunotherapy with or without chemotherapy was not preferred over the standard therapy.

Number and Impact of Osteoporotic Forearm Fractures: An Analysis of German Hospital Data

Abstract Introduction Osteoporosis is a burden for Germany, however recent data on osteoporotic forearm fractures is missing. Methods Numbers of hospital diagnosed forearm fractures based on ICD-10 code S52.- for 2000 to 2017 were taken from GBE database. From this dataset, number of osteoporotic forearm fractures in patients aged 50+ years were calculated using age- and gender specific weighting factors. In addition, fracture rates per 100,000 people, total days in hospital and quality-adjusted life years (QALYs) lost due to osteoporotic forearm fractures were calculated. Results In 2017, a total of 69,046 osteoporotic forearm fractures were diagnosed in hospitals in patients aged 50+ years in Germany, which represents an increase of 48.8% since 2000. Age-adjusted fracture rates in women were 300/100,000 (standard error [SE] 1.25) in 2017 compared to 248/100,000 (SE 1.25) in 2000 (Odds ratio [OR]: 1.21 [95% confidence interval [CI]: 1.02; 1.43]; p = 0.026). In male patients fracture rates were 62/100,000 (SE 0.61) in 2017 and 53/100,000 (SE 0.65) in 2000 (OR: 1.17 [95% CI: 0.81; 1.69]; p = 0.401). However, highest numbers and rates of osteoporotic forearm fractures were seen in 2010.Osteoporotic forearm fractures resulted in 276,185 hospital days and 2,259 lost QALYs in patients aged 50+ years in 2017. Conclusion Number of osteoporotic forearm fractures increased from 2000 to 2017 in Germany and indicate a high burden of disease for patients and healthcare system.

Cost-Effectiveness of Alirocumab in Patients With Acute Coronary Syndromes: The ODYSSEY OUTCOMES Trial

BackgroundCholesterol reduction with proprotein convertase subtilisin-kexin type 9 inhibitors reduces ischemic events; however, the cost-effectiveness in statin-treated patients with recent acute coronary syndrome remains uncertain. ObjectivesThis study sought to determine whether further cholesterol reduction with alirocumab would be cost-effective in patients with a recent acute coronary syndrome on optimal statin therapy. MethodsA cost-effectiveness model leveraging patient-level data from ODYSSEY OUTCOMES (Evaluation of Cardiovascular Outcomes After an Acute Coronary Syndrome During Treatment With Alirocumab) was developed to estimate costs and outcomes over a lifetime horizon. Patients (n = 18,924) had a recent acute coronary syndrome and were on high-intensity or maximum-tolerated statin therapy, with a baseline low-density lipoprotein cholesterol (LDL-C) level ≥70 mg/dl, non–high-density lipoprotein cholesterol ≥100 mg/dl, or apolipoprotein B ≥80 mg/l. Alirocumab 75 mg or placebo was administered subcutaneously every 2 weeks. Alirocumab was blindly titrated to 150 mg if LDL-C remained ≥50 mg/dl or switched to placebo if 2 consecutive LDL-C levels were <15 mg/dl. Incremental cost per quality-adjusted life-year (QALY) was determined with the addition of alirocumab versus placebo and, based on clinical efficacy findings from the trial, was stratified by baseline LDL-C levels ≥100 mg/dl and <100 mg/dl. ResultsAcross the overall population recruited to the ODYSSEY OUTCOMES trial, using an annual treatment cost of US$5,850, the mean overall incremental cost-effectiveness ratio was US$92,200 per QALY (base case). The cost was US$41,800 per QALY in patients with baseline LDL-C ≥100 mg/dl, whereas in those with LDL-C <100 mg/dl the cost per QALY was US$299,400. Among patients with LDL-C ≥100 mg/dl, incremental cost-effectiveness ratios remained below US$100,000 per QALY across a wide variety of sensitivity analyses. ConclusionsIn patients with a recent acute coronary syndrome on optimal statin therapy, alirocumab improves cardiovascular outcomes at costs considered intermediate value, with good value in patients with baseline LDL-C ≥100 mg/dl but less economic value with LDL-C <100 mg/dl. (Evaluation of Cardiovascular Outcomes After an Acute Coronary Syndrome During Treatment With Alirocumab [ODYSSEY OUTCOMES]; NCT01663402)

PMS32 A COST EFFECTIVENESS ASSESSMENT OF DUAL MOBILITY BEARINGS IN REVISION HIP ARTHROPLASTY

Dislocation rates of traditional single bearing (SB) implants in revision total hip arthroplasty (THA) have been reported to be 8-10%. The use of Dual Mobility (DM) bearings can reduce this risk to 0.5-2%. However, DM bearings are more costly and is not clear if the additional clinical benefits constitute value for money for the payers. We aim to estimate the cost-effectiveness of DM bearing compared to SB for patients undergoing revision THA. We developed a Markov model over 5, 10 and 14 year time horizon, to estimate the expected cost and benefits of DM compared to SB in patients undergoing revision THA. The transition probabilities for revision, re-revisions, post-operative mortality was estimated from the published United Kingdom National Joint Registry and published literature and stratified by sex and age. Implant and healthcare costs were estimated from local procurement prices and national tariffs. Quality-adjusted life-years (QALY) were calculated using published utility estimates for patients undergoing THA. At a minimum five years follow up, DM was cost-effective with an estimated incremental cost-effectiveness ratio (ICER) of between £3,006 to £18,745/ QALY for patients <55 years and 64-75 years respectively. For those aged >75 years DM was only cost-effective if the time horizon was beyond 7 years or more. DM was cost-saving for ages less than 75 and cost-effective for over 75 years if the time horizon was 10 or more years. DM is cost-effective when compared with SB in patients undergoing revision THA. The younger the patient is, the more likely that a DM bearing is more cost-effective. The results are affected by the time horizon and cost of bearings for those aged over 75 years. For patients over 75 years, it must be left to the discretion of the clinician to consider if usage remains an economic and clinical option.

Cost-Utility Analysis of Pembrolizumab Versus Chemotherapy as First-Line Treatment for Metastatic Non-Small Cell Lung Cancer With Different PD-L1 Expression Levels.

To evaluate the cost–utility of pembrolizumab versus chemotherapy as the first-line setting for metastatic non-small cell lung cancer (NSCLC) from the US health care system perspective, a Markov model was developed to compare the lifetime cost and effectiveness of pembrolizumab versus chemotherapy for untreated metastatic NSCLC, based on the clinical data derived from phase III randomized controlled trial (KEYNOTE-042; ClinicalTrials.gov; NCT02220894). Weibull distribution was fitted to simulate the parametric survival functions. Drug costs were collected from official websites, and utility values were obtained from published literature. Total costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs) were computed as primary output indicators. The impact of different PD-L1 expression levels on ICER was also evaluated. One-way and probabilistic sensitivity analyses were performed to assess the model uncertainty. Compared with chemotherapy, patients treated with pembrolizumab provided an additional 1.13, 1.01, and 0.59 QALYs in patients with PD-L1 expression levels of ≥50%, ≥20%, and ≥1%, with corresponding incremental cost of $53,784, $47,479, and $39,827, respectively. The resultant ICERs of pembrolizumab versus chemotherapy were $47,596, $47,184, and $68,061/QALY, in three expression levels of PD-L1, respectively, all of which did not exceed the WTP threshold of 180,000/QALY. Probability sensitivity analysis outcome supported that pembrolizumab exhibited evident advantage over chemotherapy to be cost-effective. One-way sensitivity analysis found that ICERs were most sensitive to utility value of pembrolizumab in progression survival state. All the adjustment of parameters did not qualitatively change the result. For treatment-naive, metastatic NSCLC patients with PD-L1+, pembrolizumab was estimated to be cost-effective compared with chemotherapy for all PD-L1 expression levels at a WTP threshold of $180,000/QALY in the context of the US health care system.

Counting the cost of denying assisted dying

In this paper, we propose and defend three economic arguments for permitting assisted dying. These arguments are not intended to provide a rationale for legalising assisted suicide or euthanasia in and of themselves; rather, they are supplementary arguments that should not be neglected when considering the ethics of assisted dying. The first argument is that permitting assisted dying allows consenting patients to avoid negative quality-adjusted life years, enabling avoidance of suffering. The second argument is that the resources consumed by patients who are denied assisted dying could instead be used to provide additional (positive) quality-adjusted life years for patients elsewhere in the healthcare system who wish to continue living and to improve their quality of life. The third argument is that organ donation may be an additional potential source of quality-adjusted life years in this context. We also anticipate and provide counterarguments to several objections to our thesis. Taken together, the cumulative avoidance of negative quality-adjusted life years and gain in positive quality-adjusted life years suggest that permitting assisted dying would substantially benefit both the small population that seeks assisted suicide or euthanasia, and the larger general population. As such, denying assisted dying is a lose–lose situation for all patients.

The integrated care pathway for managing post stroke patients (iCaPPS\xa9) in public primary care Healthcentres in Malaysia: impact on quality adjusted life years (QALYs) and cost effectiveness analysis

BackgroundThe delivery of post stroke care is fragmented even in advanced public healthcare systems, globally. Primary care teams are entrusted to provide longer term care for stroke survivors in most developing countries. The integrated Care Pathway for Post Stroke patients (iCaPPS©) was designed to guide primary care teams to incorporate further rehabilitation and regular screening for post stroke complications among patients residing at home in communities, using the shared-care approach, especially in areas with limited access to specialist stroke care services. The iCaPPS© addressed coordination of rehabilitation and screening for post stroke complications which were absent in the current conventional care of patients managed at public primary care healthcentres. This study aimed to evaluate the cost effectiveness and impact of iCaPPS© on quality-adjusted- life-years (QALY) compared with current conventional monitoring at public primary care healthcentres.MethodsA pragmatic healthcentre-based cluster randomised controlled trial-within trial on 151 post stroke patients from 10 public primary care facilities in Peninsular Malaysia was conducted to evaluate QALY of patients managed with iCaPPS© (n = 86) vs conventional care (n = 65) for 6 months. Costs from societal perspective were calculated, using combination of top down and activity-based costing methods. The 5-level EQ5D (EQ-5D-5 L) was used to calculate health state utility scores. Cost per QALY and incremental cost effectiveness ratio (ICER) were determined. Differences within groups were determined using Mann-Whitney tests.ResultsTotal costs for 6 months treatment with iCaPPS© was MYR790.34, while conventional care cost MYR527.22. Median QALY for iCaPPS© was 0.55 (0,1.65) compared to conventional care 0.32 (0, 0.73) (z = − 0.21, p = 0.84). Cost per QALY for iCaPPS© was MYR1436.98, conventional care was MYR1647.56. The ICER was MYR1144.00, equivalent to 3.7% of per capita GDP (2012 prices).ConclusionsManagement of post stroke patients in the community using iCaPPS© costs less per QALY compared to current conventional care and is very cost effective.Trial registrationTrial Registration number ACTRN12616001322426. Registered 21 September 2016. (Retrospectively registered).

Predicting Cost-Effectiveness of Generic vs. Brand Dabigatran Using Pharmacometric Estimates Among Patients with Atrial Fibrillation in the United States.

Generic entry of newer anticoagulants is expected to decrease the costs of atrial fibrillation management. However, when making switches between brand and generic medications, bioequivalence concerns are possible. The objectives of this study were to predict and compare the lifetime cost‐effectiveness of brand dabigatran with hypothetical future generics. Markov microsimulations were modified to predict the lifetime costs and quality‐adjusted life years of patients on either brand or generic dabigatran from a US private payer perspective. Event rates for generics were predicted using previously developed pharmacokinetic‐pharmacodynamic models. The analyses showed that generic dabigatran with lower‐than‐brand systemic exposure were dominant. Meanwhile, generic dabigatran with extremely high systemic exposure was not cost‐effective compared with the brand reference. Cost‐effectiveness of generic medications cannot always be assumed as shown in this example. Combined use of pharmacometric and pharmacoeconomic models can assist in decision making between brand and generic pharmacotherapies.

Cost-Effectiveness of Natalizumab Compared With Fingolimod for Relapsing-Remitting Multiple Sclerosis Treatment in Colombia

ObjectivesMultiple sclerosis (MS) is a degenerative neurological disorder. Treatment aims to avoid relapses and disability progression. The purpose of this study was to evaluate the cost-effectiveness of natalizumab compared with fingolimod for treating highly active relapsing-remitting MS (RRMS) patients from the Colombian third-party payer perspective. MethodsWe used a Markov economic model from the perspective of the Colombian healthcare system to estimate the cost-effectiveness of natalizumab compared with fingolimod for RRMS with high disease activity or failure of interferons as first-line therapy. This model was centered on disability progression and relapses. We considered a 5-year time horizon with a 5% discount rate. We included only direct medical costs. Local experts were consulted to obtain resource utilization estimates, and local standardized costing methodologies and sources were used. Outcome was considered in terms of quality-adjusted life-years (QALYs). Utilities were extracted or calculated from the literature. Transition probabilities were calculated from available efficacy and safety information (1 USD = 3050.98 COP). ResultsNatalizumab showed lower total costs (USD 80 024 vs USD 98 137) and higher QALY yield (3.01 vs 2.94) than fingolimod, dominating it (incremental cost-effectiveness ratio = −$1861). Univariate sensitivity analysis showcased the relevance of the measures of effect on disability progression for natalizumab on model results. Probabilistic sensitivity analysis replicated base-case results in most simulations. ConclusionsThis study showed that natalizumab dominated fingolimod with lower costs and higher QALYs in patients with high-activity RRMS. These results are consistent with previous published international literature.

Projected Cost-Effectiveness for 2 Gene-Drug Pairs Using a Multigene Panel for Patients Undergoing Percutaneous Coronary Intervention

BackgroundFor patients undergoing percutaneous coronary intervention, gene-drug associations exist relevant to first-line treatment options–antiplatelet agent, clopidogrel, and pain medication, tramadol. Knowledge of genotype information may allow for avoidance of adverse drug events during critical clinical windows. ObjectiveThis evaluation estimated cost-effectiveness associated with a multi-gene panel pre-emptively testing two genes providing CYP2C19 genotype-guided strategy for antiplatelet therapy, with CYP2D6 genotype-guided pain management, compared to single gene test for CYP2C19 with random assignment for pain treatment, and to no testing (empiric clopidogrel with random assignment for pain treatment). MethodsDecision analysis modeling was used to project costs from a payer perspective and patient quality-adjusted life years (QALYs) from the three strategies. The model captured composite risks of major adverse cardiovascular events and pain therapy-related adverse drug events and associated utility estimates. We conducted sensitivity analyses to assess influential input parameters. ResultsOver 15 months, multi-gene testing was least costly and yielded more QALYs compared to both single gene and no testing; total incremental costs were $1646 lower with incremental gains of 0.04 QALYs for multi-gene compared with single gene and $11 368 lower with 0.17 QALY gains compared to no test. Base case analyses revealed multi gene was dominant compared to both single gene and no test, as it demonstrated cost savings with increased QALYs. ConclusionsFor these patients, a multi-gene-guided strategy yields a favorable incremental cost-effectiveness ratio compared to the other two treatment strategies. Pre-emptively ascertaining additional gene-drug pair information can inform clinical and economic decision-making at the point of care.

Cost-effectiveness analysis of pembrolizumab monotherapy and chemotherapy in the non-small-cell lung cancer with different PD-L1 tumor proportion scores

ObjectivesThis study aimed to assess the cost-effectiveness of pembrolizumab monotherapy compared with chemotherapy as first-line treatment in patients with locally advanced or metastatic non-small-cell lung cancer (NSCLC) with different tumor proportion scores (TPS), from perspectives of payers in China. Materials and methodsBasic information was derived from the KEYNOTE-042 trial. A Markov model was developed to simulate the process of NSCLC. Model inputs were based on published clinical trials and previous literatures. Costs were calculated from perspectives of payers in China. Sensitivity analyses were conducted to explore the impact of uncertainty. ResultsTreatment with pembrolizumab monotherapy for patients with high TPS (≥50%) was estimated to increase costs by $65,322 compared with chemotherapy, with a gain of 1.79 quality adjusted life years (QALYs) for an incremental cost-effectiveness ratio (ICER) of $36,493 per QALY. For patient population with TPS ≥ 20%, the ICER was $42,311 per QALY, while the corresponding ICER was $39,404 per QALY for patients with TPS ≥ 1%. Sensitive analyses for three different TPS populations were similar, which indicated the cost of PFS state in pembrolizumab arm and the price of pembrolizumab were the most influential factors in our study. ConclusionICERs yield by pembrolizumab monotherapy among different TPS populations were beyond the threshold we set, three times of the Gross Domestic Product per Capita of China in 2018 ($26,508/QALY). It is not a cost effective choice compared with standard chemotherapy for patients with locally advanced or metastatic NSCLC from the perspective of Chinese payer, regardless of TPS. Deeper discount of its current price would make pembrolizumab a preferable choice.

Factors associated with costs and health outcomes in patients with Back and leg pain in primary care: a prospective cohort analysis

BackgroundThere is limited research on the economic burden of low back-related leg pain, including sciatica. The aim of this study was to describe healthcare resource utilisation and factors associated with cost and health outcomes in primary care patients consulting with symptoms of low back-related leg pain including sciatica.MethodsThis study is a prospective cohort of 609 adults visiting their family doctor with low back-related leg pain, with or without sciatica in a United Kingdom (UK) Setting. Participants completed questionnaires, underwent clinical assessments, received an MRI scan, and were followed-up for 12-months. The economic analysis outcome was the quality-adjusted life year (QALY) calculated from the EQ-5D-3 L data obtained at baseline, 4 and 12-months. Costs were measured based on patient self-reported information on resource use due to back-related leg pain and results are presented from a UK National Health Service (NHS) and Societal perspective. Factors associated with costs and outcomes were obtained using a generalised linear model.ResultsBase-case results showed improved health outcomes over 12-months for the whole cohort and slightly higher QALYs for patients in the sciatica group. NHS resource use was highest for physiotherapy and GP visits, and work-related productivity loss highest from a societal perspective. The sciatica group was associated with significantly higher work-related productivity costs. Cost was significantly associated with factors such as self-rated general health and care received as part of the study, while quality of life was significantly predicted by self-rated general health, and pain intensity, depression, and disability scores.ConclusionsOur results contribute to understanding the economics of low back- related leg pain and sciatica and may provide guidance for future actions on cost reduction and health care improvement strategies.Trial registration13/09/2011 Retrospectively registered; ISRCTN62880786.

Home Biofeedback for the Treatment of Dyssynergic Defecation: Does It Improve Quality of Life and Is It Cost-Effective?

Biofeedback therapy, whether administered at home or in office settings, is effective for dyssynergic defecation (DD). Whether home biofeedback improves quality of life (QOL) and is cost-effective when compared with office biofeedback is unknown. QOL was assessed in 8 domains (SF-36) at baseline and after treatment (3 months), alongside economic evaluation during a randomized controlled trial (RCT) comparing home and office biofeedback in patients with DD (Rome III). Costs related to both biofeedback programs were estimated from the hospital financial records, study questionnaires, and electronic medical records. A conversion algorithm (Brazier) was used to calculate the patient's quality-adjusted life years (QALYs) from SF-36 responses. Cost-effectiveness was expressed as incremental costs per QALY between the treatment arms. One hundred patients (96 female patients, 50 in each treatment arm) with DD participated. Six of the 8 QOL domains improved (P < 0.05) in office biofeedback, whereas 4 of the 8 domains improved (P < 0.05) in home biofeedback; home biofeedback was noninferior to office biofeedback. The median cost per patient was significantly lower (P < 0.01) for home biofeedback ($1,112.39; interquartile range (IQR), $826-$1,430) than for office biofeedback ($1,943; IQR, $1,622-$2,369), resulting in a cost difference of $830.11 The median QALY gained during the trial was 0.03 for office biofeedback and 0.07 for home biofeedback (P = NS). The incremental cost-effectiveness ratio was $20,752.75 in favor of home biofeedback. Biofeedback therapy significantly improves QOL in patients with DD regardless of home or office setting. Home biofeedback is a cost-effective treatment option for DD compared with office biofeedback, and it offers the potential of treating many more patients in the community.

The Worth of a Quality-Adjusted Life-Year in Patients with Diabetes: An Investigation Study using a Willingness-to-Pay Method.

BackgroundA limited number of studies have specifically examined the value of quality-adjusted life-years (QALYs) from the patient’s perspective.ObjectiveThe goal of this study was to investigate the worth of QALYs from the perspectives of patients with diabetes using health and willingness-to-pay (WTP) measures.MethodsA hypothetical treatment characterized by a permanent cure was presented to 149 patients with diabetes in Tehran, Iran, to elicit the monetary value that they attach to QALYs. The QALY gains of the participants were determined using the EuroQol-5 Dimensions, 3 Levels instrument, the visual analogue scale, and the time trade-off method. A mixed closed-ended WTP model supported by an open-ended question was used to ascertain the monetary value of a QALY gained. Finally, we used each respondent’s ratio of WTP to QALY gained and the mean of the ratios to estimate the worth of a QALY to all respondents.ResultsIn total, 96% of respondents were willing to pay out of pocket for the restoration of full health, whereas 4% exhibited a zero WTP because of an inability to pay. The mean WTP per QALY varied depending on the health measure and discount rate used, ranging from $US1191 to $US5043 in sensitivity analysis, which is equal to 0.23–0.95 of Iran’s gross domestic product (GDP) per capita in 2015.ConclusionApplying the upper limit of the World Health Organization’s (WHO) cost-effectiveness threshold (i.e., three times the local GDP per capita) in resource allocation decisions requires caution and investigation, particularly in low- and middle-income countries with limited healthcare resources. To generalize our findings, especially for application to decision making, additional surveys involving more representative samples from different settings are recommended.Electronic supplementary materialThe online version of this article (10.1007/s41669-018-0111-2) contains supplementary material, which is available to authorized users.

Cost-effectiveness of adult spinal deformity surgery in a military healthcare system.

OBJECTIVEAdult spinal deformity surgery is an effective way of treating pain and disability, but little research has been done to evaluate the costs associated with changes in health outcome measures. This study determined the change in quality-adjusted life years (QALYs) and the cost per QALY in patients undergoing spinal deformity surgery in the unique environment of a military healthcare system (MHS).METHODSPatients were enrolled between 2011 and 2017. Patients were eligible to participate if they were undergoing a thoracolumbar spinal fusion spanning more than 6 levels to treat an underlying deformity. Patients completed the 36-Item Short Form Health Survey (SF-36) prior to surgery and 6 and 12 months after surgery. The authors used paired t-tests to compare SF-36 Physical Component Summary (PCS) scores between baseline and postsurgery. To estimate the cost per QALY of complex spine surgery in this population, the authors extended the change in health-related quality of life (HRQOL) between baseline and follow-up over 5 years. Data on the cost of surgery were obtained from the MHS and include all facility and physician costs.RESULTSHRQOL and surgical data were available for 49 of 91 eligible patients. Thirty-one patients met additional criteria allowing for cost-effectiveness analysis. Over 12 months, patients demonstrated significant improvement (p < 0.01) in SF-36 PCS scores. A majority of patients met the minimum clinically important difference (MCID; 83.7%) and substantive clinical benefit threshold (SCBT; 83.7%). The average change in QALY was an increase of 0.08. Extended across 5 years, including the 3.5% discounting per year, study participants increased their QALYs by 0.39, resulting in an average cost per QALY of $181,649.20. Nineteen percent of patients met the < $100,000/QALY threshold with half of the patients meeting the < $100,000/QALY mark by 10 years. A sensitivity analysis showed that patients who scored below 60 on their preoperative SF-36 PCS had an average increase in QALYs of 0.10 per year or 0.47 over 5 years.CONCLUSIONSWith a 5-year extended analysis, patients who receive spinal deformity surgery in the MHS increased their QALYs by 0.39, with 19% of patients meeting the $100,000/QALY threshold. The majority of patients met the threshold for MCID and SCBT at 1 year postoperatively. Consideration of preoperative functional status (SF-36 PCS score < 60) may be an important factor in determining which patients benefit the most from spinal deformity surgery.

Total Hip Arthroplasty Improves the Quality-Adjusted Life Years in Patients Who Exceeded the Estimated Life Expectancy

BackgroundHip osteoarthritis is a leading cause of functional decline and disability in the elderly. Although patients older than 80 years could significantly benefit from an elective total hip arthroplasty (THA), they pose a significant challenge to both anesthesiologist and arthroplasty surgeon. The purpose of this study was to report the clinical outcomes, complication rate, mortality, and quality-adjusted life year (QALY) of THA in patients who already exceeded the average life expectancy. MethodsPatients treated with elective THA for debilitating hip osteoarthritis and already exceeded the average life expectancy in Switzerland (n = 100) were included. The complication rate, QALY, and 30-day, 1-year, and midterm mortality were assessed retrospectively. ResultsThe overall complication rate was 12%. The 30-day and 1-year mortality was 3% and 6%, respectively. The Harris hip score increased significantly from an average of 50 preoperative to 93 points postoperative. Most of the patients (98%) had an improvement in the Harris hip score that was above the threshold for minimally significant change, whereas 75% reported an increase that exceeded the moderate improvement threshold. The average QALY was 4 years. ConclusionTHA might be a safe and cost-effective procedure for improving pain, function, and quality of life with low mortality in selected elderly patients who already exceeded the average life expectancy. Hence, the arthroplasty surgeons should not hesitate to operate relatively active, independent, and cognitively intact elderly patients having debilitating hip osteoarthritis based only on the patient’s age. Nevertheless, careful patient selection, surgical indications, and aggressive perioperative optimization might be necessary to minimize the risk of preoperative complications.

Modeling Long-Term Cost-Effectiveness of Sitagliptin and SGLT2i Combination Therapy for the Treatment of Type 2 Diabetes

Clinical benefits of dipeptidyl peptidase-4 inhibitors (DPP-4i) and sodium-glucose co-transporter 2 inhibitors (SGLT2i) combination therapy have been demonstrated through clinical trials; however, there is limited understanding of economic benefits of sequential use of these therapies. This study evaluated the long-term cost-effectiveness of a treatment strategy involving intensification with SGLT2is (pathway 1) compared to NPH insulin (pathway 2) in type 2 diabetes (T2D) patients not at goal on metformin and sitagliptin therapy in the UK. The QuintilesIMS CORE Diabetes Model was used to perform cost-effectiveness analysis over a patient’s lifetime. Treatment effect data were obtained from randomized clinical trials, and economic data were obtained from multiple published sources. Several scenario analyses were performed to assess the robustness of base case results. Pathway 1 increased total life years (13.49 vs. 13.37, respectively) and quality-adjusted life years (QALY) (9.40 and 9.22, respectively) compared to pathway 2. Although drug costs in pathway 1 were higher than pathway 2, they were offset by decreases in diabetes-related complications and body mass index, leading to lower total direct medical costs for pathway 1 (£25,747 vs. £26,095). Thus, pathway 1 dominates pathway 2, as it is both more effective and less costly. Results from scenario analyses assessing changes in treatment effect, hypoglycemia rate, body mass index, cardiovascular protective effect of SGLT2i consistently showed that pathway 1 was either dominant (incremental cost effectiveness ratio (ICER) &amp;lt;$0/QALY) or cost-effective [(ICER &amp;lt; £3,000/QALY for patients with lower baseline HbA1c (7%) or older age (65+years)]. Among patients not at goal on metformin and sitagliptin therapy, treatment intensification with SGLT2i is likely to be cost effective compared to intensification with NPH insulin as 3rd line therapy in T2D in the UK. Disclosure M. Pawaskar: Employee; Self; Merck &amp; Co., Inc. S. Bilir: Consultant; Self; Merck &amp; Co., Inc.. A. Graber-Naidich: None. C.D. Gonzalez: Employee; Self; Merck &amp; Co., Inc. S. Rajpathak: Employee; Self; Merck &amp; Co., Inc. G.M. Davies: Employee; Self; Merck &amp; Co., Inc..

Cost-effectiveness of carfilzomib plus dexamethasone compared with bortezomib plus dexamethasone for patients with relapsed or refractory multiple myeloma in the United States

ABSTRACTBackground: We assessed the economic value of carfilzomib 56 mg/m2 and dexamethasone (Kd56) vs. bortezomib and dexamethasone (Vd) for relapsed/refractory multiple myeloma (R/RMM) using ENDEAVOR trial results.Methods: Cost-effectiveness of Kd56 vs. Vd was assessed using a partitioned survival model by estimating progression-free survival, overall survival, and direct costs over a lifetime horizon. Surveillance Epidemiology and End Results (SEER) survival data were extrapolated after matching registry and ENDEAVOR patients. Utilities were sourced from the literature and mapped from patient-reported quality of life in ENDEAVOR to estimate quality-adjusted life-years (QALYs) from life-years (LYs).Results: The model predicted an average gain of 1.66 LYs and 1.50 QALYs with Kd56 vs. Vd, and lifetime additional costs of $182,699, resulting in an incremental cost-effectiveness ratio (ICER) of $121,828/QALY gained. The ICER was $114,793/QALY in patients with 1 prior treatment; $99,263/QALY in those not transplanted, and <$150,000/QALY up to an 85% discount in bortezomib price.Conclusions: Kd56 is cost-effective for patients with R/RMM at a willingness-to-pay threshold of $150,000/QALY. Trial data in the model may limit generalizability; however, SEER registry data mitigates this challenge. Kd56 provides additional value in key subgroups, and remains cost-effective after steep comparator discounts.

Cost-Effectiveness Analysis of Sensor-Augmented Insulin Pump Therapy with Automated Insulin Suspension Versus Standard Insulin Pump Therapy in Patients with Type 1 Diabetes in Sweden.

IntroductionIn Sweden an estimated 10,000 people with type 1 diabetes use continuous subcutaneous insulin infusion (CSII). Sensor-augmented pump therapy (SAP) is associated with higher acquisition costs but provides additional clinical benefits (e.g. reduced rate of hypoglycemic events) over and above that of CSII alone. The aim of the analysis was to assess the cost-effectiveness of SAP with automated insulin suspension relative to CSII alone in two different groups of patients with type 1 diabetes in Sweden.MethodsCost-effectiveness analyses were performed using the QuintilesIMS CORE Diabetes Model, with clinical and economic input data derived from published literature. Separate analyses were performed for patients at increased risk of hypoglycemia and for patients with uncontrolled glycated hemoglobin (HbA1c) at baseline. Analyses were performed from a societal perspective over a lifetime time horizon. Future costs and clinical outcomes were discounted at 3% per annum.ResultsSAP with automated insulin suspension was associated with an incremental gain in quality-adjusted life expectancy versus the CSII of 1.88 quality-adjusted life years (QALYs) in patients at high risk of hypoglycemia and of 1.07 QALYs in patients with uncontrolled HbA1c at baseline. Higher lifetime costs for SAP with automated insulin suspension resulted in projected incremental cost-effectiveness ratios for the SAP with automated insulin suspension versus CSII of Swedish Krona (SEK) 139,795 [euros (EUR) 14,648] per QALY gained for patients at increased risk for hypoglycemia and SEK 251,896 (EUR 26,395) per QALY gained for patients with uncontrolled HbA1c. In both groups, SAP with automated insulin suspension also reduced the incidence of diabetes-related complications relative to CSII.ConclusionsIn Sweden, SAP with automated insulin suspension likely represents a cost-effective treatment option relative to CSII for the management of patients with type 1 diabetes with a history of severe hypoglycemic events or patients who struggle to achieve good glycemic control despite the use of CSII.FundingMedtronic International Trading Sàrl.

Cost-Effectiveness Analysis of Afatinib versus Gefitinib for First-Line Treatment of Advanced EGFR-Mutated Advanced Non–Small Cell Lung Cancers

IntroductionThe irreversible ErbB family blocker afatinib and the reversible EGFR tyrosine kinase inhibitor gefitinib were compared in the multicenter, international, randomized, head-to-head phase 2b LUX-Lung 7 trial for first-line treatment of advanced EGFR mutation–positive NSCLCs. Afatinib and gefitinib costs and patients’ outcomes in France were assessed. MethodsA partitioned survival model was designed to assess the cost-effectiveness of afatinib versus gefitinib for EGFR mutation–positive NSCLCs. Outcomes and safety were taken primarily from the LUX-Lung 7 trial. Resource use and utilities were derived from that trial, an expert-panel questionnaire, and published literature, limiting expenditures to direct costs. Incremental cost-effectiveness ratios (ICERs) were calculated over a 10-year time horizon for the entire population, and EGFR exon 19 deletion or exon 21 L858R mutation (L858R) subgroups. Deterministic and probabilistic sensitivity analyses were conducted. ResultsFor all EGFR mutation–positive NSCLCs, the afatinib-versus-gefitinib ICER of was €45,211 per quality-adjusted life-year (QALY) (0.170 QALY gain for an incremental cost of €7697). ICERs for EGFR exon 19 deletion and L858R populations were €38,970 and €52,518, respectively. Afatinib had 100% probability to be cost-effective at a willingness-to-pay threshold of €70,000/QALY for patients with common EGFR mutations. ConclusionFirst-line afatinib appears cost-effective compared with gefitinib for patients with EGFR mutation–positive NSCLCs.