Quality-adjusted Life Year Gain Research Articles

Distinct models to assess the cost-effectiveness of EGFR-tyrosine kinase inhibitors for the treatment of metastatic non-small cell lung cancer in the context of the Brazilian Unified Health Care System.

ABSTRACTObjective:Lung cancer is an important health problem due to its high incidence and mortality. The treatment of metastatic disease improved after the molecular pathways of cancer came to be known. However, targeted therapy is unavailable to many patients treated within the Brazilian Sistema Único de Saúde (SUS, Unified Health Care System). Our objective was to assess the cost-effectiveness of erlotinib, gefitinib, and afatinib versus that of chemotherapy for the treatment of non-small cell lung cancer in the context of the SUS. Methods:Different analytical models were developed based on data in the literature. The outcomes were presented in quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs) per QALY gained. All costs related to treatment and supportive therapies were included in the models. Results:In one model, data from retrospective studies showed 2.01 life-years saved and a mean QALY gain of 1.169. The ICER per QALY gained ranged from R$48,451.29 (for gefitinib) to R$85,559.22 (for erlotinib). In another model, data from a meta-analysis showed −0.01 life-years saved and a mean QALY gain of 0.178. The ICER per QALY gained ranged from R$27,028.30 (for gefitinib) to R$75,203.26 (for erlotinib). Conclusions:There is no ideal analytical model for the SUS. However, targeted therapy with EGFR-tyrosine kinase inhibitors has been shown to be cost-effective in various scenarios. The adoption of drug price discounts will improve the cost-effectiveness of treatment.

Early Childhood Education and Life-Cycle Health

This paper forecasts the life-cycle treatment effects on health of a high-quality early childhood program. Our predictions combine microsimulation using non-experimental data with experimental data from a midlife long-term follow-up. The follow-up incorporated a full epidemiological exam. The program mainly benefits males and significantly reduces the prevalence of heart disease, stroke, cancer, and mortality across the life-cycle. For men, we estimate an average reduction of 3.8 disability-adjusted years (DALYs). The reduction in DALYs is relatively small for women. The gain in quality-adjusted life years (QALYs) is almost enough to offset all of the costs associated with program implementation for males and half of program costs for women.

Group cognitive rehabilitation to reduce the psychological impact of multiple sclerosis on quality of life: the CRAMMS RCT.

People with multiple sclerosis have problems with memory and attention. The effectiveness of cognitive rehabilitation has not been established. The objectives were to assess the clinical effectiveness and cost-effectiveness of a cognitive rehabilitation programme for people with multiple sclerosis. This was a multicentre, randomised controlled trial in which participants were randomised in a ratio of 6 : 5 to receive cognitive rehabilitation plus usual care or usual care alone. Participants were assessed at 6 and 12 months after randomisation. The trial was set in hospital neurology clinics and community services. Participants were people with multiple sclerosis who had cognitive problems, were aged 18-69 years, could travel to attend group sessions and gave informed consent. The intervention was a group cognitive rehabilitation programme delivered weekly by an assistant psychologist to between four and six participants for 10 weeks. The primary outcome was the Multiple Sclerosis Impact Scale - Psychological subscale at 12 months. Secondary outcomes included results from the Everyday Memory Questionnaire, the 30-Item General Health Questionnaire, the EuroQol-5 Dimensions, five-level version and a service use questionnaire from participants, and the Everyday Memory Questionnaire - relative version and the Modified Carer Strain Index from a relative or friend of the participant. Of the 449 participants randomised, 245 were allocated to cognitive rehabilitation (intervention group) and 204 were allocated to usual care (control group). Of these, 214 in the intervention group and 173 in the control group were included in the primary analysis. There was no clinically important difference in the Multiple Sclerosis Impact Scale - Psychological subscale score between the two groups at the 12-month follow-up (adjusted difference in means -0.6, 95% confidence interval -1.5 to 0.3; p = 0.20). There were no important differences between the groups in relation to cognitive abilities, fatigue, employment, or carer strain at follow-up. However, there were differences, although small, between the groups in the Multiple Sclerosis Impact Scale - Psychological subscale score at 6 months (adjusted difference in means -0.9, 95% confidence interval -1.7 to -0.1; p = 0.03) and in everyday memory on the Everyday Memory Questionnaire as reported by participants at 6 (adjusted difference in means -5.3, 95% confidence interval -8.7 to -1.9) and 12 months (adjusted difference in means -4.4, 95% confidence interval -7.8 to -0.9) and by relatives at 6 (adjusted difference in means -5.4, 95% confidence interval -9.1 to -1.7) and 12 months (adjusted difference in means -5.5, 95% confidence interval -9.6 to -1.5) in favour of the cognitive rehabilitation group. There were also differences in mood on the 30-Item General Health Questionnaire at 6 (adjusted difference in means -3.4, 95% confidence interval -5.9 to -0.8) and 12 months (adjusted difference in means -3.4, 95% confidence interval -6.2 to -0.6) in favour of the cognitive rehabilitation group. A qualitative analysis indicated perceived benefits of the intervention. There was no evidence of a difference in costs (adjusted difference in means -£574.93, 95% confidence interval -£1878.93 to £729.07) or quality-adjusted life-year gain (adjusted difference in means 0.00, 95% confidence interval -0.02 to 0.02). No safety concerns were raised and no deaths were reported. The trial included a sample of participants who had relatively severe cognitive problems in daily life. The trial was not powered to perform subgroup analyses. Participants could not be blinded to treatment allocation. This cognitive rehabilitation programme had no long-term benefits on quality of life for people with multiple sclerosis. Future research should evaluate the selection of those who may benefit from cognitive rehabilitation. Current Controlled Trials ISRCTN09697576. This project was funded by the National Institute for Health Research Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 24, No. 4. See the National Institute for Health Research Journals Library website for further project information.

Personalized breast cancer screening strategies: A systematic review and quality assessment.

BackgroundThe effectiveness of breast cancer screening is still under debate. Our objective was to systematically review studies assessing personalized breast cancer screening strategies based on women’s individual risk and to conduct a risk of bias assessment.MethodsWe followed the standard methods of The Cochrane Collaboration and PRISMA declaration and searched the MEDLINE, EMBASE and Clinical Trials databases for studies published in English. The quality of the studies was assessed using the ISPOR-AMCP-NPC Questionnaire and The Cochrane Risk of Bias Tool. Two independent reviewers screened full texts and evaluated the risk of bias.ResultsOut of the 1533 initially retrieved citations, we included 13 studies. Three studies were randomized controlled trials, while nine were mathematical modeling studies, and one was an observational pilot study. The trials are in the recruitment phase and have not yet reported their results. All three trials used breast density and age to define risk groups, and two of them included family history, previous biopsies, and genetic information. Among the mathematical modeling studies, the main risk factors used to define risk groups were breast density, age, family history, and previous biopsies. Six studies used genetic information to define risk groups. The most common outcome measures were the gain in quality-adjusted life years (QALY), absolute costs, and incremental cost-effectiveness ratio (ICER), while the main outcome in the observational study was the detection rate. In all models, personalized screening strategies were shown to be effective. The randomized trials were of good quality. The modeling studies showed moderate risk of bias but there was wide variability across studies. The observational study showed a low risk of bias but its utility was moderate due to its pilot design and its relatively small scale.ConclusionsThere is some evidence of the effectiveness of screening personalization in terms of QUALYs and ICER from the modeling studies and the observational study. However, evidence is lacking on feasibility and acceptance by the target population.Review registrationPROSPERO: CRD42018110483

Cost-Utility Analysis of Electroconvulsive Therapy and Repetitive Transcranial Magnetic Stimulation for Treatment-Resistant Depression in Ontario.

To evaluate the cost-effectiveness of repetitive transcranial magnetic stimulation (rTMS) and electroconvulsive therapy (ECT), and combining both treatments in a stepped care pathway for patients with treatment-resistant depression (TRD) in Ontario. A cost-utility analysis evaluated the lifetime costs and benefits to society of rTMS and ECT as first-line treatments for TRD using a Markov model, which simulates the costs and health benefits of patients over their lifetime. Health states included acute treatment, maintenance treatment, remission, and severe depression. Treatment efficacy and health utility data were extracted and synthesized from randomized controlled trials and meta-analyses evaluating these techniques. Direct costing data were obtained from national and provincial costing databases. Indirect costs were derived from government records. Scenario, threshold, and probabilistic sensitivity analyses were performed to test robustness of the results. rTMS dominated ECT, as it was less costly and produced better health outcomes, measured in quality-adjusted life years (QALYs), in the base case scenario. rTMS patients gained an average of 0.96 additional QALYs (equivalent to approximately 1 year in perfect health) over their lifetime with costs that were $46,094 less than ECT. rTMS remained dominant in the majority of scenario and threshold analyses. However, results from scenarios in which the model's maximum lifetime allowance of rTMS treatment courses was substantially limited, the dominance of rTMS over ECT was attenuated. The scenario that showed the highest QALY gain (1.19) and the greatest cost-savings ($46,614) was when rTMS nonresponders switched to ECT. From a societal perspective utilizing a lifetime horizon, rTMS is a cost-effective first-line treatment option for TRD relative to ECT, as it is less expensive and produces better health outcomes. The reduced side effect profile and greater patient acceptability of rTMS that allow it to be administered more times than ECT in a patient's lifetime may contribute to its cost-effectiveness.

Cost-Effectiveness of Revascularisation in Patients with Intermittent Claudication on Best Medical Treatment and Unsupervised Training in a Randomised Controlled Trial

Introduction - Invasive vascular procedures are an integral part of intermittent claudication (IC) treatment, despite the relative lack of evidence to support its long-term benefits and cost-effectiveness. We wanted to compare the cost-effectiveness of revascularisation together with best medical therapy (BMT) with that of BMT alone in patients with IC. Methods - In this prospective, randomised controlled trial, consecutive patients with mild-to-severe IC due to aortoiliac or femoropoliteal disease were allocated either to BMT alone (including a structured, non-supervised exercise programme) or to revascularisation together with BMT. In-patient and out-patient costs were obtained prospectively over 24 months of follow-up. Mean improvement in quality-adjusted life years (QALYs) was calculated using the EuroQol 5D-3L questionnaire, and the cost-effectiveness was assessed as the cost per QALY gained. Results - The mean cost per patient in the BMT group was €1 901 whereas it was €8 280 in the group treated with revascularisation in addition to BMT (difference in cost: p < 0.01). Revascularisation in addition to BMT resulted in a mean gain in QALYs of 0.16 per patient (p < 0.01), giving an incremental cost-effectiveness ratio of €42 881 per QALY. Conclusion - The costs associated with revascularisation together with BMT in patients with IC were about four times higher than for BMT alone. However, since health-related quality of life also improved, the incremental cost-effectiveness ratio of revascularisation was within the accepted threshold for public willingness to pay according to the Swedish national guidelines, but exceeded that in the British National Institute for Health and Care Excellence guidelines.

Different strategies for pharmacological thromboprophylaxis for lower-limb immobilisation after injury: systematic review and economic evaluation.

Thromboprophylaxis can reduce the risk of venous thromboembolism (VTE) during lower-limb immobilisation, but it is unclear whether or not this translates into meaningful health benefit, justifies the risk of bleeding or is cost-effective. Risk assessment models (RAMs) could select higher-risk individuals for thromboprophylaxis. To determine the clinical effectiveness and cost-effectiveness of different strategies for providing thromboprophylaxis to people with lower-limb immobilisation caused by injury and to identify priorities for future research. Ten electronic databases and research registers (MEDLINE, EMBASE, Cochrane Database of Systematic Reviews, Database of Abstracts of Review of Effects, the Cochrane Central Register of Controlled Trials, Health Technology Assessment database, NHS Economic Evaluation Database, Science Citation Index Expanded, ClinicalTrials.gov and the International Clinical Trials Registry Platform) were searched from inception to May 2017, and this was supplemented by hand-searching reference lists and contacting experts in the field. Systematic reviews were undertaken to determine the effectiveness of pharmacological thromboprophylaxis in lower-limb immobilisation and to identify any study of risk factors or RAMs for VTE in lower-limb immobilisation. Study quality was assessed using appropriate tools. A network meta-analysis was undertaken for each outcome in the effectiveness review and the results of risk-prediction studies were presented descriptively. A modified Delphi survey was undertaken to identify risk predictors supported by expert consensus. Decision-analytic modelling was used to estimate the incremental cost per quality-adjusted life-year (QALY) gained of different thromboprophylaxis strategies from the perspectives of the NHS and Personal Social Services. Data from 6857 participants across 13 trials were included in the meta-analysis. Thromboprophylaxis with low-molecular-weight heparin reduced the risk of any VTE [odds ratio (OR) 0.52, 95% credible interval (CrI) 0.37 to 0.71], clinically detected deep-vein thrombosis (DVT) (OR 0.40, 95% CrI 0.12 to 0.99) and pulmonary embolism (PE) (OR 0.17, 95% CrI 0.01 to 0.88). Thromboprophylaxis with fondaparinux (Arixtra®, Aspen Pharma Trading Ltd, Dublin, Ireland) reduced the risk of any VTE (OR 0.13, 95% CrI 0.05 to 0.30) and clinically detected DVT (OR 0.10, 95% CrI 0.01 to 0.94), but the effect on PE was inconclusive (OR 0.47, 95% CrI 0.01 to 9.54). Estimates of the risk of major bleeding with thromboprophylaxis were inconclusive owing to the small numbers of events. Fifteen studies of risk factors were identified, but only age (ORs 1.05 to 3.48), and injury type were consistently associated with VTE. Six studies of RAMs were identified, but only two reported prognostic accuracy data for VTE, based on small numbers of patients. Expert consensus was achieved for 13 risk predictors in lower-limb immobilisation due to injury. Modelling showed that thromboprophylaxis for all is effective (0.015 QALY gain, 95% CrI 0.004 to 0.029 QALYs) with a cost-effectiveness of £13,524 per QALY, compared with thromboprophylaxis for none. If risk-based strategies are included, it is potentially more cost-effective to limit thromboprophylaxis to patients with a Leiden thrombosis risk in plaster (cast) [L-TRiP(cast)] score of ≥ 9 (£20,000 per QALY threshold) or ≥ 8 (£30,000 per QALY threshold). An optimal threshold on the L-TRiP(cast) receiver operating characteristic curve would have sensitivity of 84-89% and specificity of 46-55%. Estimates of RAM prognostic accuracy are based on weak evidence. People at risk of bleeding were excluded from trials and, by implication, from modelling. Thromboprophylaxis for lower-limb immobilisation due to injury is clinically effective and cost-effective compared with no thromboprophylaxis. Risk-based thromboprophylaxis is potentially optimal but the prognostic accuracy of existing RAMs is uncertain. Research is required to determine whether or not an appropriate RAM can accurately select higher-risk patients for thromboprophylaxis. This study is registered as PROSPERO CRD42017058688. The National Institute for Health Research Health Technology Assessment programme.

A Cost-Effectiveness Analysis of Isolated Meniscal Repair Versus Partial Meniscectomy for Red-Red Zone, Vertical Meniscal Tears in the Young Adult

To evaluate the cost-effectiveness of treating isolated red-red zone, vertical meniscal tears with either isolated meniscal repair (IMR) or partial meniscectomy (PM) in the young adult using conservative modeling. Adecision-analytic Markov disease progression model with a 40-year horizon was created simulating outcomes after IMR or PM for an isolated meniscal tear. Event probabilities, costs, and utilities were used for the index procedures, and the development of osteoarthritis (OA) and subsequent need for knee arthroplasty were calculated or selected from the published literature. Differences in cost, difference in quality-adjusted life years (QALYs), and the incremental cost effect ratio were calculated to determine which index procedure is most cost effective. Total direct costs from PM were modeled at $38,648, and the total direct costs of IMR were $23,948, resulting in a projected cost savings of $14,700 with IMR. There was a modeled gain in QALYs of 17 for PM and 21 for IMR, resulting in an increase in 4 QALYs for the IMR treatment group. This results in an incremental cost effect ratio of $3,935 per QALY, favoring IMR as the dominant procedure. Meniscal repair for isolated red-red zone, vertical meniscal tears was predicted to have lower direct costs and improve QALYs compared with partial meniscectomy over 40-year modeling, indicating isolated meniscal repair to be the cost-effective procedure in the treatment of an isolated meniscal tear in the young adult population. Level 3: economic and decision analysis.

Real-World Effectiveness and Real-World Cost-Effectiveness of Intravitreal Aflibercept and Intravitreal Ranibizumab in Neovascular Age-Related Macular Degeneration: Systematic Review and Meta-Analysis of Real-World Studies.

IntroductionTreatment of neovascular age-related macular degeneration (nAMD) has evolved with the advent of anti-vascular endothelial growth factor agents such that intravitreally administered aflibercept and ranibizumab (RBZ) have become the standard of care. Randomized clinical trials (RCTs) have demonstrated the benefits of these agents in nAMD; however, results achieved under RCT protocols may not always be replicated in clinical practice. Assessing real-world outcomes is important to estimate the effectiveness and cost-effectiveness of these two agents. Our objective was to assess the real-world effectiveness of intravitreally administered aflibercept and RBZ in treatment-naive patients with nAMD and determine the cost-effectiveness of intravitreally administered aflibercept versus RBZ in a real-world setting.MethodsA multistage approach was undertaken. A systematic literature review (SLR) was completed to identify studies describing real-world outcomes in patients with nAMD treated intravitreally with aflibercept or RBZ. A meta-analysis of data identified in the SLR generated a pooled estimate of the effectiveness of intravitreally administered aflibercept and RBZ at 52 weeks and an estimate of treatment burden (injection frequency and monitoring). The impact of treatment effect modifiers, such as baseline visual acuity (VA) and age, were corrected through a multivariable meta-regression. A Markov state transition model was developed to estimate the real-world cost-effectiveness of intravitreally administered aflibercept using results from the pooled estimates for effectiveness and treatment burden as primary inputs. The analysis considered the perspective of the French National Healthcare System.ResultsPatients treated intravitreally with aflibercept had a mean age of 79.52 years and mean baseline VA of 55.80 Early Treatment Diabetic Retinopathy Study (ETDRS) letters. At week 52, mean VA gain was 5.30 ETDRS letters in patients reporting an average of 7.10 intravitreal injections of aflibercept and 8.65 visits (injection and/or monitoring). RBZ-treated patients were younger (77.28 years), with a lower mean baseline VA (52.81 ETDRS letters). At week 52, mean VA gain from baseline was 4.24 ETDRS letters, with an average of 5.88 injections and 10.10 visits (injection and/or monitoring). After correcting for differences in age (77.28 years) and baseline VA (52.81 ETDRS letters) and considering the current clinical practice with aflibercept and RBZ, the mean VA gain was 6.57 ETDRS letters for patients treated intravitreally with aflibercept and 4.42 ETDRS for patients treated intravitreally with RBZ. The cost-effectiveness analysis showed that intravitreally administered aflibercept is a more effective treatment option with an incremental gain in quality-adjusted life years (QALYs) (4.918 versus 4.880) and an incremental cost-effectiveness ratio (ICER) of €27,087 per QALY.ConclusionsThe analysis identified differences in the overall treatment approach and how ophthalmologists use intravitreally administered aflibercept and RBZ in clinical practice. These differences ultimately influence the mean real-world effectiveness of the two agents. Intravitreal treatment with aflibercept (injection frequency and patients follow-up) was consistent and in line with the European label recommendations. Patients treated intravitreally with aflibercept in clinical practice reported a mean gain in VA of similar magnitude to the mean VA gain reported in the pivotal RCT. Conversely, treatment with RBZ varied significantly across the different studies. On average, RBZ-treated patients reported a low injection frequency and a frequent follow-up, driven in part by the high number of patients treated with pro re nata (PRN) regimens. RBZ-treated patients reported gains in VA versus baseline; however, the magnitude of the gain in VA was not comparable to the VA gains reported in the RBZ pivotal RCT. Intravitreal treatment with aflibercept was associated with better mean VA outcomes and an incremental gain in QALYs compared with RBZ and can be considered cost-effective for the treatment of nAMD in patients in France despite a higher price for each individual intravitreal injection of aflibercept compared with RBZ.FundingBayer AG, Basel.Electronic supplementary materialThe online version of this article (10.1007/s12325-019-01147-6) contains supplementary material, which is available to authorized users.

Cost-Effectiveness Analysis of Gilteritinib Versus Best Supportive Care (BSC) for the Treatment of Relapsed or Refractory (R/R) FLT3 Mutation-Positive (FLT3mut+) Acute Myeloid Leukemia (AML)

Cost-Effectiveness Analysis of Gilteritinib Versus Best Supportive Care (BSC) for the Treatment of Relapsed or Refractory (R/R) FLT3 Mutation-Positive (FLT3mut+) Acute Myeloid Leukemia (AML)

Updated cost-effectiveness analysis of onabotulinumtoxinA for the prevention of headache in adults with chronic migraine who have previously received three or more preventive treatments in the UK

Aims: OnabotulinumtoxinA is recommended by NICE for the treatment of chronic migraine. This economic evaluation provides updated estimates of the cost-effectiveness of onabotulinumtoxinA for chronic migraine using new utility estimates in an existing model structure.Methods: A previously published model was revised to include EQ-5D utility estimates from a large observational study (REPOSE; n = 633). Efficacy data were taken from the pooled phase III PREEMPT clinical trial program, while resource utilization estimates were obtained from the International Burden of Migraine Study (IBMS). The model estimated costs and quality-adjusted life years (QALYs) gained over 2 years from the UK NHS perspective.Results: OnabotulinumtoxinA treatment resulted in total discounted incremental costs of £1,204 and an incremental discounted QALY gain of 0.07 compared with placebo in patients with chronic migraine who have previously failed three or more preventive treatments, corresponding to an incremental cost-effectiveness ratio (ICER) of £16,306 per QALY gained. Scenario analysis showed that the administration of onabotulinumtoxinA by a specialist nurse rather than a neurology consultant reduced the ICER from £16,306 to £13,832 per QALY gained. Removal of the positive stopping rule recommended in current NICE guidance increased the ICER to £20,768 per QALY for onabotulinumtoxinA vs. placebo. Combining these two scenarios produced an ICER of £17,686 per QALY gained.Conclusion: NICE recommended onabotulinumtoxinA for the prevention of chronic migraine in 2012 amid concerns about the uncertainty of ICER estimates, with a positive stopping rule used to manage some of these uncertainties. Since the publication of the NICE guidance, the REPOSE study provides a more recent source of utility data based on real-world evidence. The results of analyses including these utilities suggest that the application of the positive stopping rule may not be necessary to ensure cost-effectiveness and that this aspect of the current NICE guidance for onabotulinumtoxinA may merit reconsideration.

PSY18 COST-EFFECTIVENESS OF TOFACITINIB IN ACTIVE PSORIATIC ARTHRITIS AFTER ANTI-TUMOUR NECROSIS FACTOR (ANTI-TNF) FAILURE OR INTOLERANCE IN SPAIN

To evaluate the cost-effectiveness of initiating treatment for psoriatic arthritis (PsA) with tofacitinib in comparison to interleukin IL-23/IL-12 and IL-17A antagonists, after failure or intolerance to a first line of anti-TNF biologic disease-modifying antirheumatic drugs (bDMARD population), from the Spanish National Health System perspective. A lifetime Markov model was developed. A panel of experts defined two sets of therapeutic sequences consisting on six lines of treatment, where only the first line was modified in order to compare tofacitinib versus ustekinumab, secukinumab and ixekizumab in bDMARD population. After six months of no response, patients switched to next treatment line. Response was evaluated using both 20% and 50% improvement in American College of Rheumatology criteria (ACR20 and ACR50, respectively) as the efficacy measures. Comparative efficacy data were inferred from a network meta-analysis and utilities were obtained from a post-hoc analysis of tofacitinib OPAL-BROADEN clinical trial. Direct medical resources and local unitary costs (€, 2018) were considered. Costs and outcomes were discounted at 3% and a 25.000€/quality-adjusted life-years (QALY) threshold value was used. Probabilistic sensitivity analysis were conducted. Tofacitinib versus ustekinumab was associated with comparable QALYs gain (-0.04 and -0.006 for ACR20 and ACR50, respectively) with a cost reduction of 5,784.34€ and 3,140.65€. Similarly, secukinumab 300mg and ixekizumab generated a small QALYs gain while tofacitinib was associated in both cases with substantial incremental costs-savings (versus secukinumab: 18,104.33€ and 9,068.56€ for ACR20 & ACR50, respectively; versus ixekizumab: 6,622.14€ and 15,159.59€ for ACR20 & ACR50, respectively). Sensitivity analysis confirmed tofacitinib's probability of being cost-effective is over 90% for all comparisons. Yielding similar QALYs gain, tofacitinib is the most cost-saving therapeutic option for PsA treatment after failure or intolerance to anti-TNF biologics in comparison to IL-23/IL-12 antagonist ustekinumab, or IL-17A antagonist secukinumab and ixekizumab, with a probability of being cost-effective over 90%.

PUK12 ANALYSIS OF COST AND CONSEQUENCES RELATED TO THE PERSISTENCE OF MIRABEGRON AND ANTIMUSCARINIC TREATMENTS AND THEIR IMPACT ON QUALITY OF LIFE IN PATIENTS AGED ≥ 65 YEARS WITH OVERACTIVE BLADDER IN SPAIN

To evaluate whether a difference in treatment dropout rate with mirabegron in overactive bladder (OAB) patients aged ≥ 65 years could generate cost savings to the National Health System (NHS) and gain quality-adjusted life years (QALYs), compared with the most commonly prescribed antimuscarinics (AM) in Spain (tolterodine, fesoterodine, oxybutynin, solifenacin). A probabilistic model (second order Monte Carlo simulation) in a hypothetical cohort of 1,000 patients aged ≥ 65 years with OAB and a time horizon of 1 year was carried out. Persistence rates, defining treatment discontinuation after a period of at least 30 days without prescription renewal, for both mirabegron and AM were obtained from a Spanish observational study in 1,149 patients ≥ 65 years-old. Unit costs (€ 2019) and utility loss associated to treatment discontinuation were obtained from Spanish public prices and literature, respectively. Persistence rates were higher in patients aged ≥ 65 years treated with mirabegron (20.2%) versus AM (10.2%), leading to a QALY gain of 0.0192 ± 0.0027 QALYs per year. Treatment with mirabegron could generate savings of 68.32 ± 12.19 € per patient per year compared to AM, with a 100% probability of saving (in all patients in the cohort). For the discontinuation criteria at 45, 60 and 90 days, the gain of QALYs with mirabegron versus AM would be 0.0227 ± 0.0058, 0.0225 ± 0.0072 and 0.0230 ± 0.0099, respectively. Saving per patient/year would be 80.75 ± 40.14 €, 78.74 ± 56.23 € and 78.19 ± 83.35 €, respectively. The result was stable for all univariate deterministic sensitivity analyzes. The probabilistic model presented showed a greater persistence in patients aged ≥ 65 years treated with mirabegron compared to AM, leading to a positive impact in OAB patients' quality of life, as well cost savings to the NHS in Spain.

PGI32 EVALUATION OF THE COST-UTILITY OF THE YORK FAECAL CALPROTECTIN CARE PATHWAY

A previous health economics analysis evaluating the cost-effectiveness of the York Faecal Calprotectin Care Pathway (YFCCP) indicated that YFCCP is cost saving and of clinical benefit when compared with both published data for erythrocyte sedimentation rate (ESR) and c-reactive protein (CRP) testing, and with FC testing using a standard <50ug/g cut-off. However, the results indicated a trade-off when compared with published data from NICE guidance for FC testing (DG11), which correctly diagnosed more patients with irritable bowel syndrome (IBS). The current study has added health-related quality of life (HRQoL) data to the original model to measure the cost-utility of the YFCCP using costs and quality adjusted life years (QALYs), whereas previous analysis was used to demonstrate avoided costs from unnecessary resource use. A decision tree model which estimates accurate diagnosis of IBS and inflammatory bowel disease (IBD) for different diagnostic tests was updated to include HRQoL data. The analysis included five comparators. Costs were identified from publicly available sources and were reflective of the NHS. HRQoL for IBS and IBD was sourced from published NICE guidance documents. Diagnostic accuracy of the intervention and comparators were identified from published sources and clinical advice. The analysis had a time horizon of one year. Uncertainty was explored using deterministic sensitivity analysis. YFCCP is dominant (cost-saving with a QALY gain) when compared with four of the five comparators using published efficacy data of ESR + CRP and published data for the standard FC testing. When comparing with the fifth comparator of the NICE published efficacy of standard FC testing (Waugh et al) the YFCCP returned incremental costs of £14,000 and incremental QALYS of 1.62 QALYs per 1000 patients, giving an ICER of approximately £9,000/QALY. The updated model shows the YFCCP pathway is cost-effective against all comparators at a £20,000 threshold.

Cost-effectiveness of Operative versus Nonoperative Treatment of Adult Symptomatic Lumbar Scoliosis an Intent-to-treat Analysis at 5-year Follow-up.

Secondary analysis using data from the NIH-sponsored study on adult symptomatic lumbar scoliosis (ASLS) that included randomized and observational arms. The aim of this study was to perform an intent-to-treat cost-effectiveness study comparing operative (Op) versus nonoperative (NonOp) care for ASLS. The appropriate treatment approach for ASLS continues to be ill-defined. NonOp care has not been shown to improve outcomes. Surgical treatment has been shown to improve outcomes, but is costly with high revision rates. Patients with at least 5-year follow-up data were included. Data collected every 3 months included use of NonOp modalities, medications, and employment status. Costs for index and revision surgeries and NonOp modalities were determined using Medicare Allowable rates. Medication costs were determined using the RedBook and indirect costs were calculated based on reported employment status and income. Qualityadjusted life year (QALY) was determined using the SF6D. There were 81 of 95 cases in the Op and 81 of 95 in the NonOp group with complete 5-year follow-up data. Not all patients were eligible 5-year follow-up at the time of the analysis. All patients in the Op and 24 (30%) in the NonOp group had surgery by 5 years. At 5 years, the cumulative cost for Op was $96,000 with a QALY gain of 2.44 and for NonOp the cumulative cost was $49,546 with a QALY gain of 0.75 with an incremental cost-effectiveness ratio (ICER) of $27,480 per QALY gain. In an intent-to-treat analysis, neither treatment was dominant, as the greater gains in QALY in the surgery group come at a greater cost. The ICER for Op compared to NonOp treatment was above the threshold generally considered cost-effective in the first 3 years of the study but improved over time and was highly cost-effective at 4 and 5 years. 2.

PND31 COST-EFFECTIVENESS ANALYSIS OF 5% LIDOCAINE PLASTER COMPARED TO PREGABALIN IN PATIENTS WITH POSTHERPETIC NEURALGIA IN CHINA

Postherpetic neuralgia (PHN) is the most common complication of herpes zoster. Topical 5% lidocaine medicated plaster has been recommended as the first-line treatment for peripheral localized neuropathic pain, but cost-effectiveness (CE) assessment compared to systemic pregabalin in China was unclear. This study was to conduct a CE analysis for 5% lidocaine plaster versus pregabalin in relieving postherpetic neuralgia, to provide a reference for clinical practice and reimbursement policy making for treatment of PNH in China. Markov model was built according to PHN disease characteristics. The efficacy data were extracted from a RCT conducted in China, the transition probability, utility value and medical cost of each state in the model were collected through a systematic review of literature and public databases. The outcome measure was cost per quality-adjusted life-year (QALY) gained. Incremental cost-effectiveness ratios (ICER) were calculated. Sensitivity analysis was conducted to confirm the robustness of the model. In base case analysis, treatment of 6 months period by pregabalin and lidocaine plaster led to a mean QALY gain of 0.34012 and 0.41968, and mean incremental costs of RMB ¥5,720 and ¥14,314. The ICER of treatment by 5% lidocaine plaster was ¥108,014, which is less than 2 times GDP per capita in China (per-capita GDP in 2018 was ¥64,644). Monte Carlo simulation resulted in the estimate of 90% probability that the 5% lidocaine plaster treatment was cost-effective. In treatment of PHN, 5% lidocaine plaster has better remission effect, but more treatment costs compared to pregabalin. According to the CE threshold of three times the per-capita GDP per QALY gained, the 5% lidocaine plaster treatment was cost effective compared to pregabalin in China.

CC3 COST-EFFECTIVENESS ANALYSIS OF VORETIGENE NEPARVOVEC VERSUS BEST SUPPORTIVE CARE IN PATIENTS WITH RPE65-MEDIATED INHERITED RETINAL DYSTROPHY: A FRENCH HEALTHCARE SYSTEM PERSPECTIVE

An economic model was developed to evaluate the cost-effectiveness of voretigene neparvovec (VN) compared to best supportive care (BSC) in individuals with RPE65-mediated inherited retinal dystrophy (IRD), from a French healthcare system perspective. Methods were based on HAS guidelines and international good research practices for modelling. A Markov model was used and included six health states (HS) based on AMA visual deficiencies classification: Moderate Visual Impairment (VI), Severe VI, Profound VI, vision limited to “counting fingers”, vision limited to “hand motion” to “no light perception” and death. A lifetime horizon was used. Transition probabilities were calculated based on the results from the VN phase III trial and a natural history study. The economic endpoints used in the model were blindness-free years (BFY) and quality-adjusted life years (QALYs). Utility data from a vignette study were used. Resource utilization and costs included: disease-related costs (diagnosis, mutation testing, paramedical care and follow-up), drug costs (acquisition, administration and adverse events), costs of medical transport and accommodation and death. Patients treated with VN stayed longer in better HS while patients in the BSC arm progressed more quickly to worse HS. The treatment with VN results in a gain of 11.7 BFY and 4.5 QALY versus BSC. The ICER for VN versus BSC was €51, 552 per BFY and €132, 607 per QALY. Deterministic and probabilistic sensitivity analyses generally showed consistency with base case findings. When additional scenarios were explored, ICERs were most sensitive to variations in the multi-state model parameters and duration of treatment effect. VN is the first gene therapy approved in RPE65-mediated IRD and represents a clinically significant advancement in the management of this disease. VN was associated with an important gain of QALYs versus BSC and can be considered a cost-effective therapy in this orphan disease.

Abiraterone vs. docetaxel for metastatic hormone-sensitive prostate cancer: A microsimulation model.

Our aim was to determine whether androgen deprivation therapy (ADT) with abiraterone acetate (AA) or ADT with docetaxel chemotherapy (DC) resulted in improved quality-adjusted life years (QALYs) among men with de novo metastatic castration-sensitive prostate cancer (mCSPC) and the cost effectiveness of the preferred strategy using decision analytic techniques. A microsimulation model with a lifetime time horizon was constructed. Our primary outcome was QALYs. Secondary outcomes included cost, incremental cost effectiveness ratio (ICER), unadjusted overall survival (OS), rates of second- and third-line therapy, and adverse events. A systematic literature review was used to generate probabilities and utilities to populate the model. The base case was a 65-year-old patient with de novo mCSPC. A total of 100 000 microsimulations were generated. Initial AA resulted in a gain of 0.45 QALYs compared to DC (3.36 vs. 2.91 QALYs) with an ICER of $276 251.82 per QALY gained with initial AA therapy. Median crude OS was 51 months with AA and 48 months with DC. Overall, 46.6% and 42.6% of patients received second-line therapy and 8.7% and 7.9% patients received third-line therapy in the AA and DC groups, respectively. Grade 3/4 adverse events were experienced in 17.6% of patients receiving initial AA and 22.3% of patients receiving initial DC. Although ADT with AA results in a gain in QALYs and crude OS compared to DC, AA therapy is not a cost-effective treatment strategy to apply uniformly to all patients. The availability of AA as a generic medication may help to close this gap. The ultimate choice should be based on patient and tumor factors.

Cost-effectiveness of switching to insulin degludec from other basal insulins in real-world clinical practice in Italy

Aims: The costs associated with insulin therapy and diabetes-related complications represent a significant and growing economic burden for healthcare systems. The aim of this study was to evaluate the cost-effectiveness of switching to insulin degludec (degludec) vs continuing previous basal insulin, in Italian patients with type 1 (T1D) or type 2 (T2D) diabetes, using a long-term economic model.Materials and methods: Data were retrieved from a real-world population of patients from clinical practice in Italy. Clinical parameters included in the base-case model were change from baseline in HbA1c, rates of hypoglycemia, and basal and bolus insulin dose, at 6 months following switch to degludec. Costs of treatments were taken from official Italian pharmaceutical list prices and costs of hypoglycemia were based on the literature. The data were used to populate a long-term (lifetime) IQVIA CORE Diabetes Model to evaluate the incremental cost-effectiveness ratio (ICER) – cost per quality-adjusted life-year (QALY). The robustness of these results was tested with extensive sensitivity analyses by varying the time horizons and abolishing each of the treatment differences and previous basal insulins.Results: The total incremental cost for degludec vs previous basal insulin was €–6,310 and €–2,682 for patients with T1D and T2D, respectively; the switch to degludec resulted in a QALY gain of 0.781 and 0.628. The long-term ICER for degludec vs continuing the previous basal insulin regimen showed that degludec was dominant for both T1D and T2D, meaning that patient health was improved in terms of QALYs with lower healthcare costs. Sensitivity analyses showed that degludec remained dominant in most scenarios including after elimination of any benefit in non-severe hypoglycemia and insulin dose, in both T1D and T2D.Conclusions: Under routine care, switching to degludec is dominant, compared with continuing previous basal insulin, in Italian patients with T1D or T2D.

Operative treatment of displaced midshaft clavicular fractures is not cost-effective

Conventional treatment of displaced midshaft clavicular fractures is nonoperative. Recent studies have implied that operative treatment might result in a faster return to work, resulting in a decreased productivity loss for society. The cost utility of plate fixation vs. nonoperative treatment of displaced midshaft clavicular fractures has not previously been investigated using a societal perspective. Decision analytical modeling of incremental costs and quality-adjusted life-years (QALYs) was performed. Data on utility, hospitalization, and productivity costs were retrieved from a Danish randomized controlled trial. Supplementary data were taken from randomized controlled trials identified in the literature. A 1-year time horizon was applied, and all prices were reported with respect to a 2016 level. Operative treatment was associated with a larger QALY gain in patients and a higher cost compared with nonoperative treatment. The incremental cost-effectiveness ratio (ICER) was estimated in Danish currency (Danish krone [kr]) at kr1,360,000 (€182,306) per QALY from a health-sector perspective and kr1,388,738 (€186,158) per QALY from a societal perspective. Considering a subgroup analysis of patients with a high-load shoulder profession, operative treatment was dominated by nonoperative treatment from a health-sector perspective. Considering a societal perspective, the ICER was estimated at -kr889,091 (-€119,181) per reduction of 1 QALY. One-way and probabilistic sensitivity analyses showed that the results were subject to uncertainty. Operative treatment is not cost-effective when considering a threshold of €34,000/QALY. However, for a subgroup of patients with a high-load shoulder profession, operative treatment might be cost-effective compared with nonoperative treatment.