Quality-adjusted Life Year Gain Research Articles

PCV27 COST-EFFECTIVENESS OF INSERTABLE CARDIAC MONITORS VERSUS STANDARD OF CARE OR SHORT-TO-LONG-TERM ECG MONITORING TO IDENTIFY ATRIAL FIBRILLATION AFTER CRYPTOGENIC STROKE

Insertable cardiac monitors (ICMs) are more likely than standard of care (SoC) intermittent external ECG monitors to detect atrial fibrillation (AF) in patients with cryptogenic stroke (CS). Anticoagulation is proven to decrease recurrent stroke risk in patients with documented AF. We aimed to evaluate the cost-effectiveness of immediate ICM at hospital discharge versus a ‘short-to-long-term monitoring’ (STLM) approach (7-day ECG monitor followed by ICM in patients remaining undiagnosed) and versus SoC, in a CS population. A lifetime Markov model assessed the cost-effectiveness of immediate ICM vs. STLM vs. SoC, from a US payer perspective. Patient characteristics and AF detection rates were based on the CRYSTAL-AF trial: three-year diagnostic yield was 30% with ICM and 3% in SoC; 1% of STLM patients are diagnosed with initial 7-day monitor. AF detection resulted in a change from aspirin to NOAC, unless precluded by prior bleeds. Subsequent risks of ischemic strokes and bleeding events were modeled based on published literature. Costs and effects were discounted at 3% annually. Immediate ICM was associated with a 0.20 quality-adjusted life year (QALY) gain compared to SoC (6.99 vs. 6.79) and $6,588 higher per-patient costs, resulting in an incremental cost-effectiveness ratio of $33,169 per QALY gained. ICM’s expected value improved with increased recurrent stroke risk. Compared to STLM, immediate ICM was associated with lower costs ($54,480 vs. $54,665), and a modest benefit in outcomes (gain of 0.0011 QALYs) making it economically dominant for AF detection. In sensitivity analysis, the immediate ICM approach remained cost-saving when varying the duration of initial short-term monitor (1-, 2-, 21-, and 30-days). ICMs are a cost-effective diagnostic tool for the prevention of recurrent stroke in a US cryptogenic stroke population. Immediate ICM was cost-saving compared to STLM, due to the cost and low diagnostic yield of short-term monitoring and delay in AF detection.

Cost-Effectiveness Analysis of Xpert MTB/RIF for Multi-Outcomes of Patients With Presumptive Pulmonary Tuberculosis in Thailand

ObjectivesThe cost-effectiveness of screening adult patients for pulmonary tuberculosis is not clear. As such, this study aims to identify the cost-effectiveness between the Xpert MTB/RIF assay and the sputum acid-fast bacilli (AFB) smear. Multi-outcomes were correct diagnosis, time to achieve correct diagnosis, and gain in quality-adjusted life-years (QALYs). MethodsA decision tree model was constructed to reveal a possible clinical pathway of tuberculosis diagnosis. The researchers used a clinical study to establish the probability of all clinical pathways for input into this model. The sample size was calculated following the correct diagnosis. Participants were randomly divided into 2 groups. A structural questionnaire and the Thai version of quality of life (EQ-5D-5L) were used for interviewing. ResultsThe results showed that the time to achieve the correct diagnosis for the group using Xpert MTB/RIF was shorter than that for the group using the sputum AFB smear. Both the correct diagnosis and QALYs of the base case analysis presented the Xpert MTB/RIF method as dominant. A Monte Carlo model, which analyzed the Xpert MTB/RIF method, revealed that the average number of patients who were correctly diagnosed was 673, the QALYs were 945.85 years, and the total cost was $143 110.64. For the sputum AFB smear method, the average number who received a correct diagnosis was 592, the QALYs were 940.40 years, and the total cost was $196 666.84. Probabilistic and one-way sensitivity analysis confirmed that the Xpert MTB/RIF remained dominant. ConclusionsThese results provide useful information for the National Strategic Plan to screen all adult patients for pulmonary tuberculosis.

PDB19 COST-EFFECTIVENESS ANALYSIS OF INSULIN DEGLUDEC/INSULIN ASPART VERSUS INSULIN GLARGINE IN PATIENTS WITH TYPE 2 DIABETES IN CHINA

To evaluate the long-term cost effectiveness of insulin degludec/insulin aspart (IDegAsp) versus insulin glargine (IGlar) in the treatment of type 2 diabetes mellitus (T2DM) from a Chinese healthcare perspective. The validated and peer-reviewed IQVIA Core Diabetes Model was employed to simulate disease progression and determine the total direct medical cost, life years (LYs) and quality-adjusted life years (QALYs) over 30 years. Simulated cohorts and treatment effects were based on Onishi study (identifier in ClinicalTrials.gov: NCT01272193) which was a phase 3, open-label, randomized, parallel-group and multicenter trial among insulin-naïve patients with T2DM. The insulin prices were the averaged national drug procurement prices until December 2019 in China. Management and complication costs were obtained from Chinese published data and adjusted to the price level of 2018 with consumer price index. An annual discount rate of 5% was used for both costs and health outcomes. One-way and probabilistic sensitivity analyses were performed. Most of the complications’ cumulative incidence in IDegAsp group was lower than IGlar (Myocardial infarction 35.31% vs.35.37%, Stroke 13.15% vs.13.23%; Ulcer 42.05% vs. 43.22%, Neuropathy 74.40% vs. 75.86%; Microalbuminuria 35.06% vs. 35.22%; Background diabetic retinopathy 32.63% vs. 33.09%, Macular edema 26.39% vs. 26.69%, Severe visual lost 11.44% vs.12.27%). Treatment with IDegAsp was associated with LYs gain of 0.150 (11.959 vs. 11.809) and QALYs gain of 0.188 (8.355 vs. 8.167) compared with IGlar over 30 years. IDegAsp saved CNY 22,282 for total cost than IGlar (CNY 196,545 vs. 218,827), which was mainly induced by lower treatment cost (CNY-14,937, CNY 66,128 vs. 81,065) and complication cost (CNY -7,364, CNY 120,840 vs. 128,204). Sensitivity analysis further demonstrated the robustness of the results. Compared with IGlar, IDegAsp was a dominant treatment option for patients with T2DM in China, which obtained more LYs and QALYs, and reduced total direct medical cost.

PUK7 COST-EFFECTIVENESS OF MIRABEGRON VERSUS ANTIMUSCARINICS FOR THE TREATMENT OF OVERACTIVE BLADDER IN ARGENTINA

To assess the cost-effectiveness of mirabegron (50 mg) versus oxybutynin (10 mg) and tolterodine extended release (ER [4 mg]) in patients with overactive bladder (OAB) treated in the Argentinean healthcare system. Cost-effectiveness was assessed over a 5-year time horizon using a Markov model that characterizes the course of disease over time. Model health states were based on disease severity levels, and therapeutic management and complications in hypothetical cohorts of patients with OAB were tracked over one-month cycles. Key model outcomes included costs, quality-adjusted life-years (QALYs), micturition state improvement (MSI), and incontinence state improvement (ISI). Transition probabilities were estimated from a mirabegron trial and a mixed treatment comparison study. Other inputs such as resource use and costs, including costs of anticholinergic burden, comorbidity treatment, drug acquisition, and physician visits, were obtained from published literature and expert opinion. All costs are reported in 2019 US dollars (USD). Deterministic and probabilistic sensitivity analyses were performed. Mirabegron conferred greater improvement in terms of micturition, incontinence and QALYs than its comparators, oxybutynin and tolterodine ER, although it was associated with higher acquisition costs. The projected QALY gain for mirabegron was similar compared to both tolterodine ER and oxybutynin (0.022 and 0.020). Mirabegron had an incremental cost effectiveness ratio (ICER) of $3,953 per MSI, $4,789 per ISI and $23,522 per QALY versus tolterodine ER and an ICER of $2,699 per MSI, $3,188 per ISI and $17,313 per QALY versus oxybutynin. Probabilistic sensitivity analyses showed that mirabegron was cost effective in 100% of simulations considering a willingness-to-pay (WTP) of 3x per capita GDP of Argentina ($23,995 USD 2019). Compared to the most commonly used antimuscarinics in Argentina, mirabegron is cost-effective at a WTP of 3x per-capita GDP per outcome of improvement for the treatment of OAB in the public healthcare system of Argentina.

Computerised speech and language therapy or attention control added to usual care for people with long-term post-stroke aphasia: the Big CACTUS three-arm RCT.

People with aphasia may improve their communication with speech and language therapy many months/years after stroke. However, NHS speech and language therapy reduces in availability over time post stroke. This trial evaluated the clinical effectiveness and cost-effectiveness of self-managed computerised speech and language therapy to provide additional therapy. A pragmatic, superiority, single-blind, parallel-group, individually randomised (stratified block randomisation, stratified by word-finding severity and site) adjunct trial. Twenty-one UK NHS speech and language therapy departments. People with post-stroke aphasia (diagnosed by a speech and language therapist) with long-standing (> 4 months) word-finding difficulties. The groups were (1) usual care; (2) daily self-managed computerised word-finding therapy tailored by speech and language therapists and supported by volunteers/speech and language therapy assistants for 6 months plus usual care (computerised speech and language therapy); and (3) activity/attention control (completion of puzzles and receipt of telephone calls from a researcher for 6 months) plus usual care. Co-primary outcomes - change in ability to find treated words of personal relevance in a bespoke naming test (impairment) and change in functional communication in conversation rated on the activity scale of the Therapy Outcome Measures (activity) 6 months after randomisation. A key secondary outcome was participant-rated perception of communication and quality of life using the Communication Outcomes After Stroke questionnaire at 6 months. Outcomes were assessed by speech and language therapists using standardised procedures. Cost-effectiveness was estimated using treatment costs and an accessible EuroQol-5 Dimensions, five-level version, measuring quality-adjusted life-years. A total of 818 patients were assessed for eligibility and 278 participants were randomised between October 2014 and August 2016. A total of 240 participants (86 usual care, 83 computerised speech and language therapy, 71 attention control) contributed to modified intention-to-treat analysis at 6 months. The mean improvements in word-finding were 1.1% (standard deviation 11.2%) for usual care, 16.4% (standard deviation 15.3%) for computerised speech and language therapy and 2.4% (standard deviation 8.8%) for attention control. Computerised speech and language therapy improved word-finding 16.2% more than usual care did (95% confidence interval 12.7% to 19.6%; p < 0.0001) and 14.4% more than attention control did (95% confidence interval 10.8% to 18.1%). Most of this effect was maintained at 12 months (n = 219); the mean differences in change in word-finding score were 12.7% (95% confidence interval 8.7% to 16.7%) higher in the computerised speech and language therapy group (n = 74) than in the usual-care group (n = 84) and 9.3% (95% confidence interval 4.8% to 13.7%) higher in the computerised speech and language therapy group than in the attention control group (n = 61). Computerised speech and language therapy did not show significant improvements on the Therapy Outcome Measures or Communication Outcomes After Stroke scale compared with usual care or attention control. Primary cost-effectiveness analysis estimated an incremental cost per participant of £732.73 (95% credible interval £674.23 to £798.05). The incremental quality-adjusted life-year gain was 0.017 for computerised speech and language therapy compared with usual care, but its direction was uncertain (95% credible interval -0.05 to 0.10), resulting in an incremental cost-effectiveness ratio of £42,686 per quality-adjusted life-year gained. For mild and moderate word-finding difficulty subgroups, incremental cost-effectiveness ratios were £22,371 and £28,898 per quality-adjusted life-year gained, respectively, for computerised speech and language therapy compared with usual care. This trial excluded non-English-language speakers, the accessible EuroQol-5 Dimensions, five-level version, was not validated and the measurement of attention control fidelity was limited. Computerised speech and language therapy enabled additional self-managed speech and language therapy, contributing to significant improvement in finding personally relevant words (as specifically targeted by computerised speech and language therapy) long term post stroke. Gains did not lead to improvements in conversation or quality of life. Cost-effectiveness is uncertain owing to uncertainty around the quality-adjusted life-year gain, but computerised speech and language therapy may be more cost-effective for participants with mild and moderate word-finding difficulties. Exploring ways of helping people with aphasia to use new words in functional communication contexts is a priority. Current Controlled Trials ISRCTN68798818. This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 24, No. 19. See the NIHR Journals Library website for further project information. The Tavistock Trust for Aphasia provided additional support to enable people in the control groups to experience the intervention after the trial had ended.

The Utility of Cervical Spine Bracing As a Postoperative Adjunct to Multilevel Anterior Cervical Spine Surgery.

Use of cervical bracing/collar subsequent to anterior cervical spine discectomy and fusion (ACDF) is variable. Outcomes data regarding bracing after ACDF are limited. Here, we study the impact of bracing on short-term outcomes related to safety, quality of care, and direct costs in multilevel ACDF. Retrospective cohort analyses of all consecutive patients undergoing multilevel ACDF with or without bracing from 2013 to 2017 was undertaken (n = 616). Patient demographics and comorbidities were analyzed. Tests of independence and logistic regressions were used to assess differences in length of stay (LOS), discharge disposition (home, assisted rehabilitation facility [ARF], or skilled nursing facility [SNF]), quality-adjusted life year (QALY), direct cost, readmission within 30 days, and emergency room (ER) visits within 30 days. Amongst the study population, 553 were braced and 63 were not braced. There was no difference in comorbidities (P > .05) such as obesity, smoking, chronic obstructive pulmonary disease, hypertension, coronary artery disease, congestive heart failure, and problem list number. A significant difference in American Society of Anesthesiologists (ASA) score was found, with more ASA 2 patients in the braced cohort and more ASA 3 patients in the unbraced cohort (P = .007). LOS was extended for the unbraced group (median 156.9 ± 211.4 versus 86.67 ± 130.6 h, P = .003), and ER visits within 30 days were 0.21 times less likely in the braced group (P = .006). There was no difference in readmission (P = .181), QALY gain (P = .968), and direct costs (P = .689). Bracing following multilevel cervical fixation does not alter short-term postoperative course or reduce the risk for early adverse outcomes in a significant manner.

Cardiac Resynchronisation Therapy in Patients with Moderate to Severe Heart Failure in Germany: A Cost-Utility Analysis of the Additional Defibrillator

BackgroundCardiac resynchronisation therapy (CRT) is a well-established form of treatment for patients with heart failure and cardiac dyssynchrony. There are two different types of CRT devices: the biventricular pacemaker (CRT-P) and the biventricular defibrillator (CRT-D). The latter is more complex but also more expensive. For the majority of patients who are eligible for CRT, both devices are appropriate according to current guidelines. The purpose of this study was to conduct a cost-utility analysis for CRT-D compared to CRT-P from a German payer’s perspective.MethodsA cohort Markov-model was developed to assess average costs and quality-adjusted life-years (QALY) for CRT-D and CRT-P. The model consisted of six stages: one for the device implementation, one for the absorbing state death, and two stages (“Stable” and “Hospital”) for either a CRT device or medical therapy. The time horizon was 20 years. Deterministic and probabilistic sensitivity analyses and scenario analyses were conducted.ResultsThe incremental cost-effectiveness ratio (ICER) of CRT-D compared with CRT-P was €24,659 per additional QALY gained. In deterministic sensitivity analysis, the survival advantage of CRT-D to CRT-P was the most influential input parameter. In the probabilistic sensitivity analysis 96% of the simulated cases were more effective but also more costly.ConclusionsTherapy with CRT-D compared to CRT-P resulted in an additional gain of QALYs, but was more expensive. In addition, the ICER was subject to uncertainty, especially due to the uncertainty in the survival benefit. A randomised controlled trial and subgroup analyses would be desirable to further inform decision making.Electronic supplementary materialThe online version of this article (10.1007/s40258-020-00571-y) contains supplementary material, which is available to authorized users.

Cost-utility analysis comparing surgical and nonsurgical interventions in the treatment of erectile dysfunction

With recent innovative developments in microsurgery, novel, safe, and effective surgical options for erectile dysfunction (ED) are quickly emerging which may replace mainstay medical treatments. In an age where health care is increasingly focused on financial accountability and clinical outcomes, cost-utility analysis between nonsurgical treatment (pharmaceutical agents, mechanical devices, and shockwave therapy) and emerging surgical options will prove relevant and impactful to all physicians that treat patients with ED. The purpose of this study was to use the cost-utility analysis to compare the costs, outcomes, and overall quality of life conveyed to the patient by either nonsurgical or surgical treatments for ED. These results will help patients and physicians form more informed decisions when it comes to the treatment of men suffering from ED. The cost-utility analysis makes use of costs, probabilities, and utilities of health outcomes (referred to as health states) that are used to evaluate opposing interventions. Amazon Mechanical Turk© was used to derive utility scores for health states associated with surgical and nonsurgical interventions. In addition, a systematic review was performed to identify common complications of treatment and associated probabilities. All direct costs were derived from national Medicare facility reimbursement data and national hospital billing charges for fiscal year 2017, and indirect costs were accounted for by our utility estimates. Baseline analysis revealed an incremental cost-utility ratio of $(1428.00) in favor of surgical intervention. The cost of lifetime medical therapy was $15,488.28 greater than that of surgery even when adjusted for inflation. Furthermore, surgery provided patients with a quality-adjusted life-year gain of 10.77 years greater than medical treatment. Long-term costs and utility data should be taken into consideration when determining the best treatment options for a patient with ED. Surgical interventions for erectile dysfunctions represent a significant cost savings to society compared with nonsurgical treatment. Phosphodiesterase type-5 (PDE5) inhibitors offered the greatest cost with the least clinical benefit in the eyes of the patient. Level of evidence: Not ratable.

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.

Five-year cost-effectiveness analysis of the European Fans in Training (EuroFIT) physical activity intervention for men versus no intervention

ObjectivesIncreasing physical activity reduces the risk of chronic illness including Type 2 diabetes, cardiovascular disease and certain types of cancer. Lifestyle interventions can increase physical activity but few successfully engage men. This study aims to investigate the 5 year cost-effectiveness of EuroFIT, a program to improve physical activity tailored specifically for male football (soccer) fans compared to a no intervention comparison group.MethodsWe developed a Markov cohort model in which the impact of improving physical activity on five chronic health conditions (colorectal cancer, Type 2 diabetes, coronary heart disease, stroke and depression) and mortality was modelled. We estimated costs from a societal perspective and expressed benefits as quality adjusted life years (QALYs). We obtained data from a 4-country (England, Netherlands, Portugal and Norway) pragmatic randomised controlled trial evaluating EuroFIT, epidemiological and cohort studies, and meta-analyses. We performed deterministic and probabilistic sensitivity analyses to assess the impact of uncertainty in the model’s parameter values on the cost-effectiveness results. We used Monte Carlo simulations to estimate uncertainty and presented this using cost-effectiveness acceptability curves (CEACs). We tested the robustness of the base case analysis using five scenario analyses.ResultsAverage costs over 5 years per person receiving EuroFIT were €14,663 and per person receiving no intervention €14,598. Mean QALYs over 5 years were 4.05 per person for EuroFIT and 4.04 for no intervention. Thus, the average incremental cost per person receiving EuroFIT was €65 compared to no intervention, while the average QALY gain was 0.01. This resulted in an ICER of €5206 per QALY gained. CEACs show that the probability of EuroFIT being cost-effective compared to no intervention is 0.53, 0.56 and 0.58 at thresholds of €10,000, €22,000 and €34,000 per QALY gained, respectively. When using a time horizon of 10 years, the results suggest that EuroFIT is more effective and less expensive compared to (i.e. dominant over) no intervention with a probability of cost-effectiveness of 0.63 at a threshold of €22,000 per QALY gained.ConclusionsWe conclude the EuroFIT intervention is not cost-effective compared to no intervention over a period of 5 years from a societal perspective, but is more effective and less expensive (i.e. dominant) after 10 years. We thus suggest that EuroFIT can potentially improve public health in a cost-effective manner in the long term.

Cost-Effectiveness Analysis of Supervised Exercise Training in Men with Prostate Cancer Previously Treated with Radiation Therapy and Androgen-Deprivation Therapy.

Exercise for prostate cancer (PCa) survivors has been shown to be effective in addressing metabolic function and associated co-morbidities, as well as sarcopenia and significant functional impairment resulting from long-term androgen deprivation. Evidence on the cost-effectiveness of exercise interventions for PCa, however, is lacking, thus the aim of this study was to determine the cost-effectiveness of a supervised exercise intervention for long-term PCa survivors who previously received radiation therapy and androgen-deprivation therapy. Cost-effectiveness analysis from an Australian healthcare-payer perspective was conducted using patient-level data from a multicentre randomised controlled trial (RCT) of supervised exercise training (resistance and aerobic) compared to receiving printed exercise material and a recommendation to exercise in long-term PCa survivors (> 5years post-diagnosis). Analysis was undertaken for the 6-month supervised exercise portion of the intervention, which involved 100 men aged between 62 and 85years, 50 in each arm. The primary outcome was cost per quality-adjusted life-years (QALYs). A 6-month supervised exercise intervention for PCa survivors resulted in an incremental cost-effectiveness ratio of AU$64,235 (2018 AUD) at an incremental cost of AU$546 per person and a QALY gain of 0.0085. At a willingness-to-pay of AU$50,000, the probability that the intervention is cost-effective was 41%. Sensitivity analysis showed that maintenance of benefits via a 6-month home-based intervention, immediately following the supervised intervention, lowered the cost per QALY gained to AU$32,051. This is the first cost-effectiveness analysis of exercise for PCa survivors. The intervention was effective, but unlikely to be cost-effective at the generally accepted willingness-to-pay of AU$50,000 per QALY. It is likely that evidence to support cost savings from post-intervention outcomes would reveal greater benefits and contribute to a more comprehensive cost-effectiveness analysis. Future RCTs should incorporate longer follow-up durations and collection of data to support modelling to capture future health benefits. Measures of quality of life or utility more sensitive to the impact of physical activity would also improve future economic evaluations.

Cost-effectiveness of inactivated influenza vaccination in children with medical risk conditions in the Netherlands

BackgroundIn many countries, annual immunization with inactivated influenza vaccine (IIV) is recommended for children with medical risk conditions. Prior cost-effectiveness analyses found such immunization to be cost saving, but assumed effectiveness against non-severe influenza outcomes and a higher effectiveness against severe influenza outcomes than recent studies would suggest. However, recent vaccine studies do not indicate any reduction in community or outpatient disease episodes in IIV immunized individuals. We therefore evaluated cost-effectiveness of IIV immunization in children with medical risk conditions in the Netherlands, assuming that IIV reduces influenza-related hospitalization and death, but has no meaningful impact on non-severe health outcomes. MethodsA health economic decision tree model was developed to evaluate health effects and costs of annual IIV immunization versus no immunization. Model inputs were based on our study on influenza-related primary care visits and other literature. Immunization was considered cost effective if associated costs were less than €20,000 per quality-adjusted life year (QALY) gained. Probabilistic sensitivity analyses were performed to assess robustness of results, and one-way sensitivity analyses and scenario analyses were done to assess the influence of individual parameters. ResultsAnnual IIV prevents an average of 1.59 influenza-related hospitalizations and 0.02 deaths per 1,000 children with medical risk conditions. This results in an expected QALY gain of 0.43 at incremental costs of €21,564 per 1,000 children, corresponding to an incremental cost-effectiveness ratio (ICER) of €50,297/QALY compared to no immunization. Under base case assumptions, immunization had a 5% probability of being cost effective. Results were most influenced by vaccine efficacy against fatal influenza, QALY loss due to death, and mortality rate. ConclusionsIf IIV only reduces severe disease outcomes, as current evidence suggests, annual immunization of medical risk children is unlikely to be cost effective. Results should however be interpreted with caution as cost-effectiveness is largely dependent on incidence and QALY losses for fatal influenza, for which evidence is scarce.

Telepresence-teleguidance to facilitate training and quality assurance in ERCP: a health economic modeling approach.

Background and study aims The aims of this study was to document the clinical and training relevance of endoscopic retrograde cholangiopancreaticography (ERCP) teleguidance (as a clinical model for applied telemedicine) with health economic modeling methodologies. Methods Probabilities and consequences of complications after ERCP performed by either a novice-trainee or supported through teleguidance (TM) by an expert formed the basis of the health economic model. Results The main clinical and economic outcomes originated from the base case scenario representing a low-volume center. In the cohort the patient age was 62 years, 58 % were females, the expert was doing ≥ 250 ERCPs per year and 50 for the novice-trainee. The expert knowledge transferred was set to 50 % and the average complexity grade to 1.98. Given a willingness to pay threshold of 56,180 USD/ quality-adjusted life years (QALY), the probability of cost-effectiveness of TM assistance was 98.9 %. The probability of a QALY gain for patients having an ERCP, to which was added TM, was 91.6 %. Adding TM saved on an average 111.2 USD (95 % CI 959 to 1021 SEK) per patient, and remained cost-effective basically insensitive to the level of willingness to pay. Conclusion Teleguidance during an ERCP procedure has the potential to be the prefered option in many low- to medium-volume hospitals. The main mechanisms behind these effects are positive impact on several adverse patient outcomes, QALY increase, and decreased costs. TM should be considered for integration into future teaching curriculums in advanced upper gastrointestinal endoscopy.

Abstract PD10-05: Cost-effectiveness of different neoadjuvant followed by adjuvant treatment combination strategies for women with HER2-positive breast cancer

Abstract Background: Several neoadjuvant and adjuvant treatment options exist for patients with HER2-positive (HER2+) stage II-III breast cancer. Recent results of the KATHERINE trial (NCT01772472) showed that adjuvant T-DM1 can reduce distant recurrence risk in patients without pathologic complete response (pCR) after neoadjuvant chemotherapy. However, pCR rates can range between 20-80% depending on treatment regimen and subtype. Given the high cost of T-DM1 and other HER2-targeted agents, understanding the relationship between the costs and health consequences of various neoadjuvant-adjuvant treatment combinations is needed. Our goal is to identify the treatment strategy that leads to the highest health benefit (i.e., lowest distant recurrence risk) at the least costs. Methods: We developed a decision-analytic Markov model to simulate clinical practice in a cohort of women aged ≥49 with HER2+ breast cancer similar to the KATHERINE trial population. We considered 4 neoadjuvant regimens: HP: trastuzumab (H) and pertuzumab (P) doublet; THP: paclitaxel (T), H and P triplet; ddAC/THP: dose dense anthracycline/cyclophosphamide (ddAC) followed by T, H, P; TCHP: docetaxel (T), carboplatin (C), H, and P, and 4 adjuvant treatments for patients with residual disease including: H; T-DM1; ddAC/THP followed by T-DM1; and ddAC followed by T-DM1. We combined these neoadjuvant and adjuvant treatment options to mimic four clinically plausible treatment de-escalation strategies and examined their cost effectiveness. All patients with pCR after any neoadjuvant treatment received adjuvant H. Strategies for residual disease: 1: neoadjuvant-ddAC/THP+adjuvant-H; 2: neoadjuvant-ddAC/THP+adjuvant-T-DM1; 3: neoadjuvant-THP+adjuvant-ddAC followed by T-DM1; 4: neoadjuvant-HP+adjuvant-ddAC/THP followed by T-DM1; 5: neoadjuvant-TCHP+adjuvant-T-DM1. We informed the effectiveness, cost and utility parameters from the KATHERINE trial, McKesson data, and published literature. We estimated costs and quality-adjusted life years (QALYs) over a lifetime for each strategy, and an incremental cost-effectiveness ratio (ICER), from a healthcare payer perspective and addressed the uncertainty with a probabilistic sensitivity analysis (PSA). Results: The mean per-patient costs varied between $274,550 (Strategy 3) and $354,652 (Strategy 4) and the mean per-patient QALYs varied between 9.44 (Strategy 1) and 10.56 (Strategy 3). When considering the five treatment strategies, Strategy 3 yielded a substantial gain in QALYs at decreased costs, representing the dominating strategy. The decision-analytic model identified Strategies 1, 2, 4 and 5 to be cost-ineffective due to their lower health effects and higher costs (i.e., dominated strategies). PSA indicated Strategy 3 to have the highest probability of cost-effectiveness and expected net benefit across a wide range of willingness-to-pay thresholds ($0-250,000). These findings persisted when changing a number of assumptions, including increasing starting age and varying pCR rates and distant recurrence probability after an initial local recurrence. Conclusions: Our study indicates that neoadjuvant trastuzumab+paclitaxel+pertuzumab followed by adjuvant trastuzumab for patients with pCR (around 46% of patients) and by adjuvant ddAC and T-DM1 for those with residual disease is the most cost-effective treatment strategy for women with HER2+, stage II-III breast cancer. StrategyCostsQALYsICERStrategy 3 Neoadjuvant T, H, P Adjuvant ddAC+T-DM1 for RD; H for pCR$274,55010.56-Strategy 1 Neoadjuvant T, H, P, ddAC Adjuvant H for RD; H for pCR$278,8829.44DominatedStrategy 2 Neoadjuvant T, H, P, ddAC Adjuvant T-DM1 for RD; H for pCR$293,98210.04DominatedStrategy 5 Neoadjuvant T, C, H, P Adjuvant T-DM1 for RD; H for pCR$316,26610.46DominatedStrategy 4 Neoadjuvant H, P Adjuvant T, H, P, ddAC+T-DM1 for RD; H for pCR$354,65210.15Dominated* “Dominated” strategy leads to fewer QALYs at higher costs than the next least costly treatment alternative. Citation Format: Natalia R. Kunst, Shi-Yi Wang, Annette Hood, Sarah Mougalian, Michael P. DiGiovanna, Kerin Adelson, Lajos Pusztai. Cost-effectiveness of different neoadjuvant followed by adjuvant treatment combination strategies for women with HER2-positive breast cancer [abstract]. In: Proceedings of the 2019 San Antonio Breast Cancer Symposium; 2019 Dec 10-14; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2020;80(4 Suppl):Abstract nr PD10-05.

Economic value of transcatheter valve replacement for inoperable aortic stenosis.

To project the social value of transcatheter aortic valve replacement (TAVR) for inoperable patients with severe, symptomatic aortic stenosis (SSAS). This study used an economic model with parameters obtained from the literature and from US Census Bureau population projections. Our model estimated the economic value that will accrue to inoperable patients with SSAS and to device manufacturers as a result of TAVR utilization. We estimated individual patient value as the monetized gain in quality-adjusted life-years as estimated in the cost-effectiveness literature, net of device costs and cost offsets. We estimated manufacturer value by applying an assumed profit margin to revenue from device sales. We created population-level estimates by combining these individual-level estimates with age-stratified Census Bureau population projections and estimates of the incidence of AS. We assessed model uncertainty through the use of probabilistic sensitivity analyses. Between 2018 and 2028, approximately 465,000 inoperable Americans with SSAS will be treated with TAVR. These procedures will yield a cumulative social benefit of up to $48 billion, with roughly 80% of that benefit accruing to patients and 20% accruing to device manufacturers. Policy makers and payers should take this social value into account when considering decisions related to the care of inoperable patients with SSAS.

An Economic Evaluation of Voretigene Neparvovec for the Treatment of Biallelic RPE65-Mediated Inherited Retinal Dystrophies in the UK.

IntroductionVoretigene neparvovec (VN) is a gene therapy and the first approved pharmacological treatment for biallelic RPE65-mediated inherited retinal dystrophies (IRD), a rare condition that starts in early life and causes vision to progressively deteriorate towards complete blindness. In a phase III trial, treatment with VN significantly improved functional vision and visual function, and in October 2019 the National Institute for Health and Care Excellence (NICE) Highly Specialised Technologies (HST) process recommended VN for patients in England and Wales. We assessed the cost-effectiveness of VN compared with best supportive care (BSC) in individuals with biallelic RPE65-mediated IRD in the UK.MethodsA Markov model was developed to estimate the incremental cost per quality-adjusted life-year (QALY) gained for VN compared with BSC, from the perspective of the UK National Health Service and Personal Social Services. Phase III trial data were used to inform transition probabilities up to year 1, after which the treatment effect was assumed to be maintained for 40 years, followed by a decline in vision. A bespoke elicitation exercise involving clinical experts, patients and carers was conducted to estimate utility values for each model health state.ResultsAt list price, VN is associated with incremental costs of £612,404 and incremental QALYs of 6.4, resulting in an incremental cost-effectiveness ratio (ICER) of £95,072 per QALY gained. Voretigene neparvovec is associated with a significant undiscounted QALY gain (20.5) and is therefore eligible for additional QALY weighting under the NICE HST process; an ICER of up to £205,000 per QALY gained could be considered cost-effective under this framework.ConclusionThe results of the model show VN to be a cost-effective use of healthcare resources in the UK at list price. The availability of a commercial discount in the UK (as considered in the NICE appraisal) means that in reality the ICER will be even lower.Plain Language SummaryPlain language summary available for this article.Electronic supplementary materialThe online version of this article (10.1007/s12325-020-01243-y) contains supplementary material, which is available to authorized users.

Applying a Constrained Optimization Portfolio Model to Aid Prioritization of Public Health Interventions in Malaysia

ObjectivesCountries have constrained healthcare budgets and must prioritize new interventions depending on health goals and time frame. This situation is relevant in the sphere of national immunization programs, for which many different vaccines are proposed, budgets are limited, and efficient choices must be made in the order of vaccine introduction. MethodsA constrained optimization (CO) model for infectious diseases was developed in which different intervention types (prophylaxis and treatment) were combined for consideration in Malaysia. Local experts defined their priority public health issues: pneumococcal disease, dengue, hepatitis B and C, rotavirus, neonatal pertussis, and cholera. Epidemiological, cost, and effectiveness data were informed from local or regionally published literature. The model aimed to maximize quality-adjusted life-year (QALY) gain through the reduction of events in each of the different diseases, under budget and intervention coverage constraints. The QALY impact of the interventions was assessed over 2 periods: lifetime and 20 years. The period of investment was limited to 15 years. ResultsThe assessment time horizon influenced the prioritization of interventions maximizing QALY gain. The incremental health gains compared with a uninformed prioritization were large for the first 8 years and declined thereafter. Rotaviral and pneumococcal vaccines were identified as key priorities irrespective of time horizon, hepatitis B immune prophylaxis and hepatitis C treatment were priorities with the lifetime horizon, and dengue vaccination replaced these with the 20-year horizon. ConclusionsCO modeling is a useful tool for making economically efficient decisions within public health programs for the control of infectious diseases by helping prioritize the selection of interventions to maximize health gain under annual budget constraints.

Early cost-utility analysis of tissue-engineered heart valves compared to bioprostheses in the aortic position in elderly patients

ObjectivesAortic valve disease is the most frequent indication for heart valve replacement with the highest prevalence in elderly. Tissue-engineered heart valves (TEHV) are foreseen to have important advantages over currently used bioprosthetic heart valve substitutes, most importantly reducing valve degeneration with subsequent reduction of re-intervention. We performed early Health Technology Assessment of hypothetical TEHV in elderly patients (≥ 70 years) requiring surgical (SAVR) or transcatheter aortic valve implantation (TAVI) to assess the potential of TEHV and to inform future development decisions.MethodsUsing a patient-level simulation model, the potential cost-effectiveness of TEHV compared with bioprostheses was predicted from a societal perspective. Anticipated, but currently hypothetical improvements in performance of TEHV, divided in durability, thrombogenicity, and infection resistance, were explored in scenario analyses to estimate quality-adjusted life-year (QALY) gain, cost reduction, headroom, and budget impact.ResultsDurability of TEHV had the highest impact on QALY gain and costs, followed by infection resistance. Improved TEHV performance (− 50% prosthetic valve-related events) resulted in lifetime QALY gains of 0.131 and 0.043, lifetime cost reductions of €639 and €368, translating to headrooms of €3255 and €2498 per hypothetical TEHV compared to SAVR and TAVI, respectively. National savings in the first decade after implementation varied between €2.8 and €11.2 million (SAVR) and €3.2–€12.8 million (TAVI) for TEHV substitution rates of 25–100%.ConclusionsDespite the relatively short life expectancy of elderly patients undergoing SAVR/TAVI, hypothetical TEHV are predicted to be cost-effective compared to bioprostheses, commercially viable and result in national cost savings when biomedical engineers succeed in realising improved durability and/or infection resistance of TEHV.

Cost-effectiveness of Tisagenlecleucel vs Standard Care in High-risk Relapsed Pediatric Acute Lymphoblastic Leukemia in Canada

Tisagenlecleucel, a chimeric antigen receptor T-cell therapy for relapsed or refractory pediatric acute lymphoblastic leukemia, has been approved for use in multiple jurisdictions. The public list price is US $475 000, or more than CaD $600 000. Assessing the cost-effectiveness of tisagenlecleucel is necessary to inform policy makers on the economic value of this treatment. To assess the value for money of tisagenlecleucel compared with current standard care for tisagenlecleucel-eligible pediatric patients with acute lymphoblastic leukemia under unknown long-term effectiveness. A cost-utility analysis of tisagenlecleucel compared with current standard care using a Canadian population-based registry of pediatric patients with acute lymphoblastic leukemia was performed. Results from 3 pooled single-arm tisagenlecleucel clinical trials and a provincial pediatric cancer registry were combined to create treatment and control arms, respectively. The population-based control arm consisted of patients meeting clinical trial inclusion and exclusion criteria, starting at second relapse. Multistate and individual-level simulation modeling were combined to predict patient lifetime health trajectories by treatment strategy. Tisagenlecleucel efficacy was modeled across long-term cure rates, from 10% to 40%, to account for limited information on its long-term effectiveness. Uncertainty was tested with 1-way and probabilistic sensitivity analysis. Data were collected in September 2017, and analysis began in December 2017. Tisagenlecleucel compared with current standard care for tisagenlecleucel-eligible patients. Relative health care costs, survival gains, and quality-adjusted life-years (QALYs) between tisagenlecleucel and current standard care. The treatment and control arms were modeled on 192 and 118 patients, respectively. The mean (SD) age of control individuals was 10 (4.25) years, and the mean (SD) age of the pooled clinical trial sample was 11 (6) years. The control individuals had 78 boys (66%), and the pooled clinical trial sample had 102 boys (53%). Treatment with tisagenlecleucel was associated with an additional 2.14 to 9.85 life years or 1.68 to 6.61 QALYs, compared with current care. The average additional cost of tisagenlecleucel was CaD $470 013 (US $357 031). Accounting for the total discounted cost over the patient lifetime resulted in an incremental cost of CaD $71 000 (US $53 933) to CaD $281 000 (US $213 453) per QALY gain. To our knowledge, this study offers the first cost-effectiveness analysis of tisagenlecleucel compared with current standard care for pediatric patients with acute lymphoblastic leukemia using a constructed population-based control arm. At a willingness-to-pay threshold of $150 000/QALY, tisagenlecleucel had a 32% likelihood of being cost-effective. Tisagenlecleucel cost-effectiveness would fall below $50 000/QALY with a long-term cure rate of over 0.40 or a price discount of 49% at its currently known effectiveness.

Cost-effectiveness of offering an area-level financial incentive on breast feeding: a within-cluster randomised controlled trial analysis

ObjectiveTo provide the first estimate of the cost-effectiveness of financial incentive for breastfeeding intervention compared with usual care.DesignWithin-cluster (‘ward’-level) randomised controlled trial cost-effectiveness analysis (trial registration number ISRCTN44898617).SettingFive local authority...