Impact of including productivity costs in economic analyses of vaccines for C. difficile infections and infant respiratory syncytial virus, in a UK setting.

OBJECTIVES: It has been estimated that vaccines can accrue a relatively large part of their value from patient and carer productivity. Yet, productivity value is not commonly or consistently considered in health economic evaluations of vaccines in several high-income countries. To contribute to a better understanding of the potential impact of including productivity value on the expected cost-effectiveness of vaccination, we illustrate the extent to which the incremental costs would change with and without productivity value incorporated. METHODS: For two vaccines currently under development, one against Cloistridioides difficile (C. difficile) infection and one against respiratory syncytial disease (RSV), we estimated their incremental costs with and without productivity value included and compared the results. RESULTS: In this analysis, reflecting a UK context, a C. difficile vaccination programme would prevent £12.3 in productivity costs for every person vaccinated. An RSV vaccination programme would prevent £49 in productivity costs for every vaccinated person. CONCLUSIONS: Considering productivity costs in future cost-effectiveness analyses of vaccines for C. difficile and RSV will contribute to better-informed reimbursement decisions from a societal perspective.

Shifting breast cancer surveillance from current hospital setting to a community based setting: a cost-effectiveness study.

BACKGROUND: This study explores the effectiveness and cost-effectiveness of surveillance after breast cancer treatment provided in a hospital-setting versus surveillance embedded in the community-based National Breast Cancer Screening Program (NBCSP). METHODS: Using a decision tree, strategies were compared on effectiveness and costs from a healthcare perspective over a 5-year time horizon. Women aged 50-75 without distant metastases that underwent breast conserving surgery in 2003-2006 were selected from the Netherlands Cancer Registry (n = 14,093). Key input parameters were mammography sensitivity and specificity, risk of loco regional recurrence (LRR), and direct healthcare costs. Primary outcome measure was the proportion true test results (TTR), expressed as the positive and negative predictive value (PPV, NPV). The incremental cost-effectiveness ratio (ICER) is defined as incremental costs per TTR forgone. RESULTS: For the NBCSP-strategy, 13,534 TTR (8 positive; 13,526 negative), and 12,923 TTR (387 positive; 12,536 negative) were found for low and high risks respectively. For the hospital-based strategy, 26,663 TTR (13 positive; 26,650 negative) and 24,883 TTR (440 positive; 24,443 negative) were found for low and high risks respectively. For low risks, the PPV and NPV for the NBCSP-based strategy were 3.31% and 99.88%, and 2.74% and 99.95% for the hospital strategy respectively. For high risks, the PPV and NPV for the NBCSP-based strategy were 64.10% and 98.87%, and 50.98% and 99.71% for the hospital-based strategy respectively. Total expected costs of the NBCSP-based strategy were lower than for the hospital-based strategy (low risk: €1,271,666 NBCSP vs €2,698,302 hospital; high risk: €6,939,813 NBCSP vs €7,450,150 hospital), rendering ICERs that indicate cost savings of €109 (95%CI €95-€127) (low risk) and €43 (95%CI €39-€56) (high risk) per TTR forgone. CONCLUSION: Despite expected cost-savings of over 50% in the NBCSP-based strategy, it is nearly 50% lower accurate than the hospital-based strategy, compromising the goal of early detection of LRR to an extent that is unlikely to be acceptable.

Early cost-effectiveness of tumor infiltrating lymphocytes (TIL) for second line treatment in advanced melanoma: a model-based economic evaluation.

BACKGROUND: An emerging immunotherapy is infusion of tumor infiltrating Lymphocytes (TIL), with objective response rates of around 50% versus 19% for ipilimumab. As an Advanced Therapeutic Medicinal Products (ATMP), TIL is highly personalized and complex therapy. It requests substantial upfront investments from the hospital in: expensive lab-equipment, staff expertise and training, as well as extremely tight hospital logistics. Therefore, an early health economic modelling study, as part of a Coverage with Evidence Development (CED) program, was performed. METHODS: We used a Markov decision model to estimate the expected costs and outcomes (quality-adjusted life years; QALYs) for TIL versus ipilimumab for second line treatment in metastatic melanoma patients from a Dutch health care perspective over a life long time horizon. Three mutually exclusive health states (stable disease (responders)), progressive disease and death) were modelled. To inform further research prioritization, Value of Information (VOI) analysis was performed. RESULTS: TIL is expected to generate more QALYs compared to ipilimumab (0.45 versus 0.38 respectively) at lower incremental cost (presently €81,140 versus €94,705 respectively) resulting in a dominant ICER (less costly and more effective). Based on current information TIL is dominating ipilimumab and has a probability of 86% for being cost effective at a cost/QALY threshold of €80,000. The Expected Value of Perfect Information (EVPI) amounted to €3 M. CONCLUSIONS: TIL is expected to have the highest probability of being cost-effective in second line treatment for advanced melanoma compared to ipilimumab. To reduce decision uncertainty, a clinical trial investigating e.g. costs and survival seems most valuable. This is currently being undertaken as part of a CED program in the Netherlands Cancer Institute, Amsterdam, the Netherlands, in collaboration with Denmark.

Effect of a Low vs Intermediate Tidal Volume Strategy on Ventilator-Free Days in Intensive Care Unit Patients Without ARDS: A Randomized Clinical Trial.

Importance: It remains uncertain whether invasive ventilation should use low tidal volumes in critically ill patients without acute respiratory distress syndrome (ARDS). Objective: To determine whether a low tidal volume ventilation strategy is more effective than an intermediate tidal volume strategy. Design, Setting, and Participants: A randomized clinical trial, conducted from September 1, 2014, through August 20, 2017, including patients without ARDS expected to not be extubated within 24 hours after start of ventilation from 6 intensive care units in the Netherlands. Interventions: Invasive ventilation using low tidal volumes (n = 477) or intermediate tidal volumes (n = 484). Main Outcomes and Measures: The primary outcome was the number of ventilator-free days and alive at day 28. Secondary outcomes included length of ICU and hospital stay; ICU, hospital, and 28- and 90-day mortality; and development of ARDS, pneumonia, severe atelectasis, or pneumothorax. Results: In total, 961 patients (65% male), with a median age of 68 years (interquartile range [IQR], 59-76), were enrolled. At day 28, 475 patients in the low tidal volume group had a median of 21 ventilator-free days (IQR, 0-26), and 480 patients in the intermediate tidal volume group had a median of 21 ventilator-free days (IQR, 0-26) (mean difference, -0.27 [95% CI, -1.74 to 1.19]; P = .71). There was no significant difference in ICU (median, 6 vs 6 days; 0.39 [-1.09 to 1.89]; P = .58) and hospital (median, 14 vs 15 days; -0.60 [-3.52 to 2.31]; P = .68) length of stay or 28-day (34.9% vs 32.1%; hazard ratio [HR], 1.12 [0.90 to 1.40]; P = .30) and 90-day (39.1% vs 37.8%; HR, 1.07 [0.87 to 1.31]; P = .54) mortality. There was no significant difference in the percentage of patients developing the following adverse events: ARDS (3.8% vs 5.0%; risk ratio [RR], 0.86 [0.59 to 1.24]; P = .38), pneumonia (4.2% vs 3.7%; RR, 1.07 [0.78 to 1.47]; P = .67), severe atelectasis (11.4% vs 11.2%; RR, 1.00 [0.81 to 1.23]; P = .94), and pneumothorax (1.8% vs 1.3%; RR, 1.16 [0.73 to 1.84]; P = .55). Conclusions and Relevance: In patients in the ICU without ARDS who were expected not to be extubated within 24 hours of randomization, a low tidal volume strategy did not result in a greater number of ventilator-free days than an intermediate tidal volume strategy. Trial Registration: ClinicalTrials.gov Identifier: NCT02153294.

Uncertainty of Physicians and Patients in Medical Decision Making.

Modern medicine has developed an apparently unlimited array of diagnostic and therapeutic tools; for example, treatment options for patients with hematologic malignancies are expanding rapidly. These developments, offering a spectrum of modalities, provide a new outlook on successful treatment for many patients. Nontheless, decisions regarding the optimum treatment strategy for any individual patient remain challenging, given that all prognostic projections are based on statistics. The challenge lies in identifying parameters that characterize individual patients as prospective responders or nonresponders and thus determine the prognosis. Both physicians and patients are confronted with the uncertainty of the success of treatment with any strategy in a particular disease condition and with the impact of treatment on overall prognosis. How certain can a physician be that an individual patient has the best prognosis with a particular intervention? How does a physician's own uncertainty affect decisions made by a less-informed patient? Here we examine various aspects of uncertainty and their relevance in the process of medical decision making as briefly illustrated by 2 clinical scenarios of hematologic malignancies.

Value of Implementation of Strategies to Increase the Adherence of Health Professionals and Cancer Survivors to Guideline-Based Physical Exercise.

BACKGROUND: To increase the adherence of health professionals and cancer survivors to evidence-based physical exercise, effective implementation strategies (ISTs) are required. OBJECTIVES: To examine to what extent these ISTs provide value for money and which IST has the highest expected value. METHODS: The net benefit framework of health economic evaluations is used to conduct a value-of-implementation analysis of nine ISTs. Seven are directed to health professionals and two to cancer survivors. The analysis consists of four steps: 1) analyzing the expected value of perfect implementation (EVPIM); 2) assessing the estimated costs of the various ISTs; 3) comparing the ISTs' costs with the EVPIM; and 4) assessing the total net benefit (TNB) of the ISTs. These steps are followed to identify which strategy has the greatest value. RESULTS: The EVPIM for physical exercise in the Netherlands is €293 million. The total costs for the ISTs range from €34,000 for printed educational materials for professionals to €120 million for financial incentives for patients, and thus all are cost-effective. The TNB of the ISTs that are directed to professionals ranges from €5.7 million for printed educational materials to €30.9 million for reminder systems. Of the strategies that are directed to patients, only the motivational program had a positive net benefit of €100.4 million. CONCLUSIONS: All the ISTs for cancer survivors, except for financial incentives, had a positive TNB. The largest improvements in adherence were created by a motivational program for patients, followed by a reminder system for professionals.

A SYSTEMATIC APPROACH FOR ASSESSING, IN THE ABSENCE OF FULL EVIDENCE, WHETHER MULTICOMPONENT INTERVENTIONS CAN BE MORE COST-EFFECTIVE THAN SINGLE COMPONENT INTERVENTIONS.

OBJECTIVES: Multicomponent interventions (MCIs), consisting of at least two interventions, are common in rehabilitation and other healthcare fields. When the effectiveness of the MCI versus that of its single interventions is comparable or unknown, evidence of their expected incremental cost-effectiveness can be helpful in deciding which intervention to recommend. As such evidence often is unavailable this study proposes an approach to estimate what is more cost-effective; the MCI or the single intervention(s). METHODS: We reviewed the literature for potential methods. Of those identified, headroom analysis was selected as the most suitable basis for developing the approach, based on the criteria of being able to estimate the cost-effectiveness of the single interventions versus that of the MCI (a) within a limited time frame, (b) in the absence of full data, and (c) taking into account carry-over and interaction effects. We illustrated the approach with an MCI for cancer survivors. RESULTS: The approach starts with analyzing the costs of the MCI. Given a specific willingness-to-pay-value, it is analyzed how much effectiveness the MCI would need to generate to be considered cost-effective, and if this is likely to be attained. Finally, the cost-effectiveness of the single interventions relative to the potential of the MCI for being cost-effective can be compared. CONCLUSIONS: A systematic approach using headroom analysis was developed for estimating whether an MCI is likely to be more cost effective than one (or more) of its single interventions.

Cost-effectiveness and resource use of implementing MRI-guided NACT in ER-positive/HER2-negative breast cancers in The Netherlands.

BACKGROUND: Response-guided neoadjuvant chemotherapy (RG-NACT) with magnetic resonance imaging (MRI) is effective in treating oestrogen receptor positive/human epidermal growth factor receptor-2 negative (ER-positive/HER2-negative) breast cancer. We estimated the expected cost-effectiveness and resources required for its implementation compared to conventional-NACT. METHODS: A Markov model compared costs, quality-adjusted-life-years (QALYs) and costs/QALY of RG-NACT vs. conventional-NACT, from a hospital perspective over a 5-year time horizon. Health services required for and health outcomes of implementation were estimated via resource modelling analysis, considering a current (4 %) and a full (100 %) implementation scenario. RESULTS: RG-NACT was expected to be more effective and less costly than conventional NACT in both implementation scenarios, with 94 % (current) and 95 % (full) certainty, at a willingness to pay threshold of €20.000/QALY. Fully implementing RG-NACT in the Dutch target population of 6306 patients requires additional 5335 MRI examinations and an (absolute) increase in the number of MRI technologists, by 3.6 fte (full-time equivalent), and of breast radiologists, by 0.4 fte. On the other hand, it prevents 9 additional relapses, 143 cancer deaths, 23 congestive heart failure events and 2 myelodysplastic syndrome/acute myeloid leukaemia events. CONCLUSION: Considering cost-effectiveness, RG-NACT is expected to dominate conventional-NACT. While personnel capacity is likely to be sufficient for a full implementation scenario, MRI utilization needs to be intensified.

Decisions on Further Research for Predictive Biomarkers of High-Dose Alkylating Chemotherapy in Triple-Negative Breast Cancer: A Value of Information Analysis.

OBJECTIVES: To inform decisions about the design and priority of further studies of emerging predictive biomarkers of high-dose alkylating chemotherapy (HDAC) in triple-negative breast cancer (TNBC) using value-of-information analysis. METHODS: A state transition model compared treating women with TNBC with current clinical practice and four biomarker strategies to personalize HDAC: 1) BRCA1-like profile by array comparative genomic hybridization (aCGH) testing; 2) BRCA1-like profile by multiplex ligation-dependent probe amplification (MLPA) testing; 3) strategy 1 followed by X-inactive specific transcript gene (XIST) and tumor suppressor p53 binding protein (53BP1) testing; and 4) strategy 2 followed by XIST and 53BP1 testing, from a Dutch societal perspective and a 20-year time horizon. Input data came from literature and expert opinions. We assessed the expected value of partial perfect information, the expected value of sample information, and the expected net benefit of sampling for potential ancillary studies of an ongoing randomized controlled trial (RCT; NCT01057069). RESULTS: The expected value of partial perfect information indicated that further research should be prioritized to the parameter group including "biomarkers' prevalence, positive predictive value (PPV), and treatment response rates (TRRs) in biomarker-negative patients and patients with TNBC" (€639 million), followed by utilities (€48 million), costs (€40 million), and transition probabilities (TPs) (€30 million). By setting up four ancillary studies to the ongoing RCT, data on 1) TP and MLPA prevalence, PPV, and TRR; 2) aCGH and aCGH/MLPA plus XIST and 53BP1 prevalence, PPV, and TRR; 3) utilities; and 4) costs could be simultaneously collected (optimal size = 3000). CONCLUSIONS: Further research on predictive biomarkers for HDAC should focus on gathering data on TPs, prevalence, PPV, TRRs, utilities, and costs from the four ancillary studies to the ongoing RCT.

A cost-effectiveness analysis of using TheraBite in a preventive exercise program for patients with advanced head and neck cancer treated with concomitant chemo-radiotherapy.

Previous studies have shown that a "Preventive Exercise Program" (PREP) is cost-effective compared to the standard exercise program provided in "Usual Care" (UC) in patients with advanced head and neck cancer. The current paper specifically estimates the cost-effectiveness of the TheraBite jaw rehabilitation device (TB) which is used as part of the PREP, compared to Speech Language Pathology (SLP) sessions as part of UC, and herewith intents to inform reimbursement discussions regarding the TheraBite device. Costs and outcomes [quality-adjusted life-years (QALYs)] of the TB compared to SLP were estimated using a Markov model of advanced head and neck cancer patients. Secondary outcome variables were trismus, feeding substitutes, facial pain, and pneumonia. The incremental cost-effectiveness ratio (ICER) was estimated from a health care perspective of the Netherlands, with a time horizon of 2 years. The total health care costs per patient were estimated to amount to €5,129 for the TB strategy and €6,915 for the SLP strategy. Based on the current data, the TB strategy yielded more quality-adjusted life-years (1.28) compared to the SLP strategy (1.24). Thus, the TB strategy seems more effective (+0.04) and less costly (-€1,786) than the SLP only strategy. At the prevailing threshold of €20,000/QALY the probability for the TB strategy being cost-effective compared to SLP was 70 %. To conclude, analysis of presently available data indicates that TB is expected to be cost-effective compared to SLP in a preventive exercise program for concomitant chemo-radiotherapy for advanced head and neck cancer patients.

Return-to-work intervention for cancer survivors: budget impact and allocation of costs and returns in the Netherlands and six major EU-countries.

BACKGROUND: Return-to-work (RTW)-interventions support cancer survivors in resuming work, but come at additional healthcare costs. The objective of this study was to assess the budget impact of a RTW-intervention, consisting of counselling sessions with an occupational physician and an exercise-programme. The secondary objective was to explore how the costs of RTW-interventions and its financial revenues are allocated among the involved stakeholders in several EU-countries. METHODS: The budget impact (BI) of a RTW-intervention versus usual care was analysed yearly for 2015-2020 from a Dutch societal- and from the perspective of a large cancer centre. The allocation of the expected costs and financial benefits for each of the stakeholders involved was compared between the Netherlands, Belgium, England, France, Germany, Italy, and Sweden. RESULTS: The average intervention costs in this case were €1,519/patient. The BI for the Netherlands was €-14.7 m in 2015, rising to €-71.1 m in 2020, thus the intervention is cost-saving as the productivity benefits outweigh the intervention costs. For cancer centres the BI amounts to €293 k in 2015, increasing to €1.1 m in 2020. Across European countries, we observed differences regarding the extent to which stakeholders either invest or receive a share of the benefits from offering a RTW-intervention. CONCLUSION: The RTW-intervention is cost-saving from a societal perspective. Yet, the total intervention costs are considerable and, in many European countries, mainly covered by care providers that are not sufficiently reimbursed.

Cost-effectiveness of heat and moisture exchangers compared to usual care for pulmonary rehabilitation after total laryngectomy in Poland.

The beneficial physical and psychosocial effects of heat and moisture exchangers (HMEs) for pulmonary rehabilitation of laryngectomy patients are well evidenced. However, cost-effectiveness in terms of costs per additional quality-adjusted life years (QALYs) has not yet been investigated. Therefore, a model-based cost-effectiveness analysis of using HMEs versus usual care (UC) (including stoma covers, suction system and/or external humidifier) for patients after laryngectomy was performed. Primary outcomes were costs, QALYs and incremental cost-effectiveness ratio (ICER). Secondary outcomes were pulmonary infections, and sleeping problems. The analysis was performed from a health care perspective of Poland, using a time horizon of 10 years and cycle length of 1 year. Transition probabilities were derived from various sources, amongst others a Polish randomized clinical trial. Quality of life data was derived from an Italian study on similar patients. Data on frequencies and mortality-related tracheobronchitis and/or pneumonia were derived from a Europe-wide survey amongst head and neck cancer experts. Substantial differences in quality-adjusted survival between the use of HMEs (3.63 QALYs) versus UC (2.95 QALYs) were observed. Total health care costs/patient were 39,553 PLN (9465 Euro) for the HME strategy and 4889 PLN (1168 Euro) for the UC strategy. HME use resulted in fewer pulmonary infections, and less sleeping problems. We could conclude that given the Polish threshold of 99,000 PLN/QALY, using HMEs is cost-effective compared to UC, resulting in 51,326 PLN/QALY (12,264 Euro/QALY) gained for patients after total laryngectomy. For the hospital period alone (2 weeks), HMEs were cost-saving: less costly and more effective.

Cost-Effectiveness of Reduced-Intensity Allogeneic Hematopoietic Cell Transplantation for Older Patients With High-Risk Myelodysplastic Syndrome: Analysis of BMT CTN 1102.

PURPOSE: BMT CTN 1102 was a phase III trial comparing reduced-intensity allogeneic hematopoietic cell transplantation (RIC alloHCT) to standard of care for persons with intermediate- or high-risk myelodysplastic syndrome (MDS). We report results of a cost-effectiveness analysis conducted alongside the clinical trial. METHODS: Three hundred eighty-four patients received HCT (n = 260) or standard of care (n = 124) according to availability of a human leukocyte antigen-matched donor. Cost-effectiveness was calculated from US commercial and Medicare perspectives over a 20-year time horizon. Health care utilization and costs were estimated using propensity score-matched cohorts of HCT recipients in the OptumLabs Data Warehouse (age 50-64 years) and Medicare (age 65 years and older). EuroQol 5 Dimension (EQ-5D) surveys of trial participants were used to derive health state utilities. RESULTS: Extrapolated 20-year overall survival for those age 50-64 years was 29% for HCT (n = 105) versus 13% for usual care (n = 44) and 31% for HCT (n = 155) versus 12% for non-HCT (n = 80) for those age 65 years and older. HCT was more effective (+2.36 quality-adjusted life-years [QALYs] for age 50-64 years and +2.92 QALYs for age 65 years and older) and more costly (+$452,242 in US dollars (USD) for age 50-64 years and +$233,214 USD for age 65 years and older) than usual care, with incremental cost-effectiveness ratios of $191,487 (USD)/QALY and $79,834 (USD)/QALY, respectively. For persons age 50-64 years, there was a 29% chance that HCT was cost-effective using a willingness-to-pay (WTP) threshold of $150K (USD)/QALY and 51% at a $200K (USD)/QALY. For persons age 65 years and older, the probability was 100% at a WTP >$150K (USD)/QALY. CONCLUSION: Among patients age 65 years and older with high-risk MDS, RIC HCT is a high-value strategy. For those age 50-64 years, HCT is a lower-value strategy but has similar cost-effectiveness to other therapies commonly used in oncology.

Cost-utility of metal-on-metal hip resurfacing compared to conventional total hip replacement in young active patients with osteoarthritis.

BACKGROUND: Metal-on-metal hip resurfacing arthroplasty (MoM HRA) has emerged as an alternative to total hip arthroplasty (THA) for younger active patients with osteoarthritis (OA). Birmingham hip resurfacing is the most common MoM HRA in Alberta, and is therefore compared with conventional THA. OBJECTIVE: The objective of this study was to estimate the expected cost-utility of MoM HRA versus THA, in younger patients with OA, using a decision analytic model with a 15-year time horizon. METHODS: A probabilistic Markov decision analytic model was constructed to estimate the expected cost per quality-adjusted life-year (QALY) of MoM HRA versus THA from a health care payer perspective. The base case considered patients with OA aged 50 years; men comprised 65.9% of the cohort. Sensitivity analyses evaluated cohort age, utility values, failure probabilities, and treatment costs. Data were derived from the Hip Improvement Project and the Hip and Knee Replacement Pilot databases in Alberta, the 2010 National Joint Replacement Registry of the Australian Orthopaedic Association, and the literature. RESULTS: In the base case, THA was dominated by MoM HRA (incremental mean costs of -$583 and incremental mean QALYs of 0.079). In subgroup analyses, THA remained dominated when cohort age was 40 years instead of 50 years or when only men were assessed. THA dominated when the cohort age was 60 years or when only women were assessed. Results were sensitive to utilities, surgery costs, and MoM HRA revision and conversion probabilities. At a willingness-to-pay of Can $50,000/QALY, there was a 58% probability that MoM HRA is cost-effective. CONCLUSIONS: The results show that, on average, MoM HRA was preferred to THA for younger and male patients, but THA is still a reasonable option if the patient or clinician prefers given the small absolute differences between the options and the confidence ellipses around the cost-effectiveness estimates.

Cost-Effectiveness of Unrelated Umbilical Cord Blood Transplantation versus HLA-Haploidentical Related Bone Marrow Transplantation: Evidence from BMT CTN 1101.

BMT CTN 1101 was a Phase III randomized controlled trial comparing reduced-intensity conditioning followed by double unrelated umbilical cord blood transplantation (UCBT) versus HLA-haploidentical related donor bone marrow transplantation (haplo-BMT) for patients with high-risk hematologic malignancies. Here we report the results of a parallel cost-effectiveness analysis of these 2 hematopoietic stem cell transplantation (HCT) techniques. In this study, 368 patients were randomized to unrelated UCBT (n = 186) or haplo-BMT (n = 182). We estimated healthcare utilization and costs using propensity score-matched haplo-BMT recipients from the OptumLabs Data Warehouse for trial participants age <65 years and Medicare claims for participants age ≥65 years. Weibull models were used to estimate 20-year survival. EQ-5D surveys by trial participants were used to estimate quality-adjusted life-years (QALYs). At a 5-year follow-up, survival was 42% for haplo-BMT recipients versus 36% for UCBT recipients (P = .06). Over a 20-year time horizon, haplo-BMT is expected to be more effective (+.63 QALY) and more costly (+$118,953) for persons age <65 years. For those age ≥65 years, haplo-BMT is expected to be more effective and less costly. In one-way uncertainty analyses, for persons age <65, the cost per QALY result was most sensitive to life-years and health state utilities, whereas for those age ≥65, life- years were more influential than costs and health state utilities. Compared to UCBT, haplo-BMT was moderately more cost-effective for patients age <65 years and less costly and more effective for persons age ≥65 years. Haplo-BMT is a fair value choice for commercially insured patients with high-risk leukemia and lymphoma who require HCT. For Medicare enrollees, haplo-BMT is a preferred choice when considering costs and outcomes.

Effectiveness of multidimensional cancer survivor rehabilitation and cost-effectiveness of cancer rehabilitation in general: a systematic review.

INTRODUCTION: Many cancer survivors suffer from a combination of disease- and treatment-related morbidities and complaints after primary treatment. There is a growing evidence base for the effectiveness of monodimensional rehabilitation interventions; in practice, however, patients often participate in multidimensional programs. This study systematically reviews evidence regarding effectiveness of multidimensional rehabilitation programs for cancer survivors and cost-effectiveness of cancer rehabilitation in general. METHODS: The published literature was systematically reviewed. Data were extracted using standardized forms and were summarized narratively. RESULTS: Sixteen effectiveness and six cost-effectiveness studies were included. Multidimensional rehabilitation programs were found to be effective, but not more effective than monodimensional interventions, and not on all outcome measures. Effect sizes for quality of life were in the range of -0.12 (95% confidence interval [CI], -0.45-0.20) to 0.98 (95% CI, 0.69-1.29). Incremental cost-effectiveness ratios ranged from -€16,976, indicating cost savings, to €11,057 per quality-adjusted life year. CONCLUSIONS: The evidence for multidimensional interventions and the economic impact of rehabilitation studies is scarce and dominated by breast cancer studies. Studies published so far report statistically significant benefits for multidimensional interventions over usual care, most notably for the outcomes fatigue and physical functioning. An additional benefit of multidimensional over monodimensional rehabilitation was not found, but this was also sparsely reported on. Available economic evaluations assessed very different rehabilitation interventions. Yet, despite low comparability, all showed favorable cost-effectiveness ratios. Future studies should focus their designs on the comparative effectiveness and cost-effectiveness of multidimensional programs.

A cost-effectiveness analysis of a preventive exercise program for patients with advanced head and neck cancer treated with concomitant chemo-radiotherapy.

BACKGROUND: Concomitant chemo-radiotherapy (CCRT) has become an indispensable organ, but not always function preserving treatment modality for advanced head and neck cancer. To prevent/limit the functional side effects of CCRT, special exercise programs are increasingly explored. This study presents cost-effectiveness analyses of a preventive (swallowing) exercise program (PREP) compared to usual care (UC) from a health care perspective. METHODS: A Markov decision model of PREP versus UC was developed for CCRT in advanced head and neck cancer. Main outcome variables were tube dependency at one-year and number of post-CCRT hospital admission days. Primary outcome was costs per quality adjusted life years (cost/QALY), with an incremental cost-effectiveness ratio (ICER) as outcome parameter. The Expected Value of Perfect Information (EVPI) was calculated to obtain the value of further research. RESULTS: PREP resulted in less tube dependency (3% and 25%, respectively), and in fewer hospital admission days than UC (3.2 and 4.5 days respectively). Total costs for UC amounted to €41,986 and for PREP to €42,271. Quality adjusted life years for UC amounted to 0.68 and for PREP to 0.77. Based on costs per QALY, PREP has a higher probability of being cost-effective as long as the willingness to pay threshold for 1 additional QALY is at least €3,200/QALY. At the prevailing threshold of €20,000/QALY the probability for PREP being cost-effective compared to UC was 83%. The EVPI demonstrated potential value in undertaking additional research to reduce the existing decision uncertainty. CONCLUSIONS: Based on current evidence, PREP for CCRT in advanced head and neck cancer has the higher probability of being cost-effective when compared to UC. Moreover, the majority of sensitivity analyses produced ICERs that are well below the prevailing willingness to pay threshold for an additional QALY (range from dominance till €45,906/QALY).

Is it economically viable to employ the nurse practitioner in general practice?

AIMS: This article provides insight into the potential economic viability of nurse practitioner employment in Dutch general practices. BACKGROUND: General practitioners face the challenging task of finding the most efficient and effective mix of professionals in general practice to accommodate future care demands within scarce health care budgets. To enable informed decision-making about skill mix issues, economic information is needed. DESIGN: Discursive paper. METHOD: A descriptive and explorative design was chosen to study the economic viability of nurse practitioner employment in general practice. The conditions under which the nurse practitioner is able to earn back his/her own cost of employment were identified. Preferences and expectations of general practitioners and health insurers about nurse practitioner reimbursement were made transparent. RESULTS: Although general practitioners and health insurers acknowledge the importance of the nurse practitioner in accommodating primary care demands, they have polarised views about reimbursement. The employment of nurse practitioners is seldom economically viable in current practices. It requires a reallocation of (80% of) the general practitioner's freed up time towards practice growth (12% number of patients). CONCLUSION: The economic viability of the nurse practitioner has proven difficult to achieve in every day health care practice. This study provided insight into the complex interaction of the (cost) parameters that result in economic viability and feeds a further discussion about the content of the nurse practitioner role in general practice based on optimal quality of care vs. efficiency. RELEVANCE TO CLINICAL PRACTICE: Effective and efficient health care can only be provided if the actual care needs of a population provide the basis for deciding which mix of professionals is best equipped to deal with the changing and increasing demand of care. A macro-level intervention is needed to help a broad-scale introduction of the nurse practitioner in general practice.

Quality of integrated chronic care measured by patient survey: identification, selection and application of most appropriate instruments.

OBJECTIVE: To identify the most appropriate generic instrument to measure experience and/or satisfaction of people receiving integrated chronic care. BACKGROUND: Health care is becoming more user-centred and, as a result, the experience of users of care and evaluation of their experience and/or satisfaction is taken more seriously. It is unclear to what extent existing instruments are appropriate in measuring the experience and/or satisfaction of people using integrated chronic care. METHODS: Instruments were identified by means of a systematic literature review. Appropriateness of instruments was analysed on seven criteria. The two most promising instruments were translated into Dutch, if necessary, and administered to a convenience sample of 109 people with a chronic illness. Data derived from respondents were analysed statistically. Focus-group interviews were conducted to assess the semantic and technical equivalence as well as opinions of people about the applicability and relevance of the translated instruments. RESULTS: From 37 instruments identified, the Patients' Assessment of Care for chronIc Conditions (PACIC) and the short form of the Patient Satisfaction Questionnaire III (PSQ-18) were selected as most promising instruments. Both instruments produced similar median scores across people with different chronic conditions. The overall PACIC and its subscales and the overall PSQ-18 were highly internally consistent, but not the PSQ-18 subscales. Overall, the PACIC demonstrated better psychometric characteristics. PACIC and PSQ-18 scores were found to be moderately correlated. Whereas more respondents preferred the PSQ-18, focus-group participants regarded the PACIC to be more applicable and relevant. The technical and semantic equivalence of both instruments were sufficient. CONCLUSIONS: Because of its psychometric characteristics, perceived applicability and relevance, the PACIC is the most appropriate instrument to measure the experience of people receiving integrated chronic care.

Cost-Effectiveness Analysis of a Procalcitonin-Guided Decision Algorithm for Antibiotic Stewardship Using Real-World U.S. Hospital Data.

Medical decision-making is revolutionizing with the introduction of artificial intelligence and machine learning. Yet, traditional algorithms using biomarkers to optimize drug treatment continue to be important and necessary. In this context, early diagnosis and rational antimicrobial therapy of sepsis and lower respiratory tract infections (LRTI) are vital to prevent morbidity and mortality. In this study we report an original cost-effectiveness analysis (CEA) of using a procalcitonin (PCT)-based decision algorithm to guide antibiotic prescription for hospitalized sepsis and LRTI patients versus standard care. We conducted a CEA using a decision-tree model before and after the implementation of PCT-guided antibiotic stewardship (ABS) using real-world U.S. hospital-specific data. The CEA included societal and hospital perspectives with the time horizon covering the length of hospital stay. The main outcomes were average total costs per patient, and numbers of patients with Clostridium difficile and antibiotic resistance (ABR) infections. We found that health care with the PCT decision algorithm for hospitalized sepsis and LRTI patients resulted in shorter length of stay, reduced antibiotic use, fewer mechanical ventilation days, and lower numbers of patients with C. difficile and ABR infections. The PCT-guided health care resulted in cost savings of $25,611 (49% reduction from standard care) for sepsis and $3630 (23% reduction) for LRTI, on average per patient. In conclusion, the PCT decision algorithm for ABS in sepsis and LRTI might offer cost savings in comparison with standard care in a U.S. hospital context. To the best of our knowledge, this is the first health economic analysis on PCT implementation using U.S. real-world data. We suggest that future CEA studies in other U.S. and worldwide settings are warranted in the current age when PCT and other decision algorithms are increasingly deployed in precision therapeutics and evidence-based medicine.