Healthcare resource utilization and costs in patients with newly diagnosed acute myeloid leukemia

Abstract

Aim: Acute myeloid leukemia (AML) is associated with high disease burden. This analysis estimated HRU and costs among newly diagnosed AML patients in a US commercially insured population.

Materials and methods: This was a retrospective observational study using the IMS Health PharMetrics Plus and Hospital Charge Detail Master databases. Patients included adults who were newly diagnosed with AML between January 2007 and June 2016 (“study period”). Patients with <12 months of continuous enrollment prior to the index date were excluded, as were those whose first diagnosis was AML in remission/relapse, those diagnosed with acute promyelocytic leukemia, those on Medicare supplemental insurance, or those with a diagnosis of AML in remission/relapse without evidence of treatment during the study period. Patients were stratified by receipt of AML treatment (chemotherapy/hematopoietic cell transplantation [HCT]), and their follow-up was partitioned into initial, remission, and relapsed health states. Mean HRU and costs were tallied by treatment and, for treated patients, by health state and time since entry into health state (≤6 vs >6 months).

Results: A total of 9,455 patients met study criteria, including 6,415 (68%) treated and 3,040 (32%) untreated patients, with mean follow-up of 18.3 and 16.4 months, respectively. Mean age was 55 years in treated patients and 60 years in untreated patients. Mean total costs per patient were $386,077 in treated patients and $79,382 in untreated patients. For treated patients, 60% of total costs ($231,867 per patient) were incurred during the initial health state, representing time without remission/relapse. Mean monthly total healthcare costs were $21,055 and $4,854 among treated and untreated patients, respectively.

Limitations and conclusions: HRU and costs of managing AML patients are substantial. In treated patients, the majority of costs were incurred during the initial treatment period, without claims indicating remission/relapse.     

Cost analysis of plasma-derived factor VIII/von Willebrand factor versus recombinant factor VIII for treatment of previously untreated patients with severe hemophilia A in the United States

Background: Inhibitor development to factor VIII (FVIII) hemophilia therapy results in increased complications and substantial economic costs. The SIPPET study, the first randomized controlled trial to compare the immunogenicity of plasma-derived FVIII (pdFVIII)/von Willebrand factor (VWF) and recombinant-DNA-derived FVIII (rFVIII), demonstrated higher inhibitor rates in previously untreated patients (PUPs) treated with rFVIII than in PUPs treated with pdFVIII/VWF.

Objective: To quantify the economic impact of treating PUPs with pdFVIII/VWF vs rFVIII.

Methods: An Excel-based clinical and economic model was developed from a US healthcare payer perspective and run over a 5-year period. The analysis utilized a cohort approach to model patient treatment and outcomes over a monthly cycle to quantify differences in costs of FVIII, bypassing agents, and hospitalizations for serious bleeds. Rates of high-titer inhibitor development were obtained from the SIPPET study. Patients developing high-titer inhibitors were treated with immune tolerance induction (ITI). Patients who developed low-titer inhibitors and those who did not develop inhibitors continued their usual FVIII treatment. Patients who were successfully treated with ITI returned to FVIII treatment, while unsuccessfully treated patients received bypassing agents. Total costs per treated patient were estimated and a one-way sensitivity analysis was conducted to quantify the impact of parameter uncertainty on the model outcomes.

Results: Total cumulative costs per patient over 5 years were $834,621 for pdFVIII/VWF patients and $1,237,163 for rFVIII patients, representing a total saving of $402,542 per patient over the 5-year period, for an average annual saving of $80,508 per patient.

Conclusions: Based on data from the SIPPET study, this analysis found that initiating FVIII treatment in severe hemophilia A PUPs with pdFVIII/VWF has the potential to offer substantial cost savings to healthcare payers, amounting to a one-third reduction in costs.     

Semiparametric Difference-in-Differences Estimators

The difference-in-differences (DID) estimator is one of the most popular tools for applied research in economics to evaluate the effects of public interventions and other treatments of interest on some relevant outcome variables. However, it is well known that the DID estimator is based on strong identifying assumptions. In particular, the conventional DID estimator requires that, in the absence of the treatment, the average outcomes for the treated and control groups would have followed parallel paths over time. This assumption may be implausible if pre-treatment characteristics that are thought to be associated with the dynamics of the outcome variable are unbalanced between the treated and the untreated. That would be the case, for example, if selection for treatment is influenced by individual-transitory shocks on past outcomes (Ashenfelter's dip). This article considers the case in which differences in observed characteristics create non-parallel outcome dynamics between treated and controls. It is shown that, in such a case, a simple two-step strategy can be used to estimate the average effect of the treatment for the treated. In addition, the estimation framework proposed in this article allows the use of covariates to describe how the average effect of the treatment varies with changes in observed characteristics.     

Evaluation of the Reggio approach to early education

We evaluate the Reggio Approach using non-experimental data on individuals from the cities of Reggio Emilia, Parma and Padova belonging to one of five age cohorts: ages 50, 40, 30, 18, and 6 as of 2012. The treated were exposed to municipally offered infant-toddler (ages 0–3) and preschool (ages 3–6) programs in Reggio Emilia. The control group either did not receive formal childcare or were exposed to programs offered by municipal systems (outside of Reggio Emilia), or by state or religious systems (in all three cities). We exploit the city-cohort structure of the data to estimate treatment effects using three strategies: difference-in-differences, matching, and matched-difference-in-differences. Most positive and significant effects are generated from comparisons of the treated with individuals who did not receive formal childcare. Relative to not receiving formal care, the Reggio Approach significantly boosts outcomes related to employment, socio-emotional skills, high school graduation, participation in elections, and obesity. Comparisons with individuals exposed to alternative forms of childcare do not yield strong patterns of positive and significant effects. This suggests that differences between the Reggio Approach and other alternatives are not sufficiently large to result in significant differences in outcomes. This interpretation is supported by a survey we conduct, which documents increasing similarities in the administrative and pedagogical practices of childcare systems in the three cities over time.     

States as Ecologies of Political Enterprises

This paper seeks to overcome an antinomy within the theory of political economy: while market outcomes are treated as resulting from polycentric competition, political outcomes are treated as resulting from hierarchic planning. We seek to overcome this antinomy by treating political outcomes as likewise resulting from polycentric competition, taking due account of relevant institutional differences. For example, a parliamentary assembly is treated as an extra-ordinary form of investment bank that intermediates between the sponsors of enterprises and those within the citizenry who have means to support those enterprises. What results is a theory in which political programs emerge in largely bottom-up fashion through complex networks of transactions. Much of the inspiration for this paper arises from the Italian School of Public Finance, particularly Mazzola, Montemartini, Pantaleoni and de Viti de Marco.     

Implications for Australia of Cross-Country Comparisons in Economic Performance*

What can be learnt about the economic experience of Australia in the 1970s by comparing the performances of other countries with that of Australia? In particular, what do those comparisons suggest about the nature of and reasons for, the Australian outcomes for prices, production and employment? These are the main questions addressed in this paper. In Section II, comparisons are made for several indicators between the experiences of Australia and nine other countries. In Section III the outcomes are analyzed and in Section IV some implications for Australia are drawn.     

Guidelines-based treatment associated with improved economic outcomes in nontuberculous mycobacterial lung disease

Abstract

Background: The prevalence of nontuberculous mycobacterial lung disease (NTMLD) in the US has increased; however, data characterizing the associated healthcare utilization and expenditure at the national level are limited.

Objective: To examine associations between economic outcomes and the use of anti-Mycobacterium avium complex (MAC) guidelines-based treatment (GBT) for newly-diagnosed NTMLD in a US national managed care claims database (Optum^® Clinformatics^® Data Mart).

Methods: NTMLD was defined as having ≥2 claims for NTMLD (ICD-9 031.0; ICD-10 A31.0) on separate occasions ≥30?days apart (between 2007 and 2016). The cohort included patients insured continuously over a period of at least 36?months (12?months before initial NTMLD diagnostic claim and for the subsequent 24?months). Treatment was classified as GBT (consistent with American Thoracic Society/Infectious Diseases Society of America guidelines), non-GBT, or untreated. All-cause hospitalization rates and total healthcare expenditures at Year 2 were assessed as outcomes of the treatment prescribed in Year 1 after NTMLD diagnosis.

Results: A total of 1,039 patients met study criteria for NTMLD (GBT, n?=?294; non-GBT, n?=?298; untreated, n?=?447). After adjustment for baseline characteristics, GBT was associated with a significantly lower all-cause hospitalization risk vs non-GBT (odds ratio [OR]?=?0.53; 95% CI = 0.33–0.85, p?=?0.008), and vs being untreated (OR = 0.57; 95% CI = 0.35–0.91, p?=?0.020). Adjusted total healthcare expenditure in Year 2 with GBT ($69,691) was lower than that with non-GBT ($77,624) with a difference of ?$7,933 (95% CI = ?$14,968 to ?$899; p?=?0.03).

Conclusions: Patients with NTMLD in a US managed care claims database who were prescribed GBT had lower hospitalization risk than those who were prescribed non-GBT or were untreated. GBT was associated with lower total healthcare expenditure compared with non-GBT.     

A note on the triple difference in economic models

This article shows when a triple difference strategy using an imperfect control category improves on the double difference strategy for estimating an average treatment effect. For example, a product is treated in one place and not another leading to a double difference strategy. When does comparison with an untreated product in triple difference strategy improve accuracy?     

Medical resource utilization and costs among Australian patients with genotype 1 chronic hepatitis C: results of a retrospective observational study

Objective: To evaluate medical resource utilization (MRU) and associated costs among Australian patients with genotype 1 chronic hepatitis C (GT1 CHC), including both untreated patients and those receiving treatment with first-generation protease inhibitor-based regimens (telaprevir, boceprevir with pegylated interferon and ribavirin).

Methods: Medical records were reviewed for a stratified random sample of GT1 CHC patients first attending two liver clinics between 2011–2013 (principal population; PP), supplemented by all GT1 CHC patients attending one transplant clinic in the same period (transplant population; TP). CHC-related MRU and associated costs are reported for the PP by treatment status (treated/not treated) stratified by baseline fibrosis grade; and for the TP for the pre-transplant, year of transplant and post-transplant periods.

Results: A total 1636 patients were screened and 590 patients (36.1%) were included. Comprehensive MRU data were collected for 276 PP patients (F0–1 n?=?59, F2 n?=?58, F3 n?=?53, F4 n?=?106; mean follow-up?=?17.3 months). Thirty-eight (13.8%) were treatment-experienced prior to enrolment; 55 (19.9%) received triple therapy during the study. Data were collected for 112 TP patients (mean follow-up?=?29.9 months), 33 (29.5%) received a transplant during the study, and 51 (45.5%) beforehand. The annual direct medical costs, excluding drug costs, were higher among treated PP vs untreated PP (AU$: $1,954 vs $1,202); and year of transplant TP vs pre-/post-transplant TP (AU$: pre-transplant $32,407, transplant $155,138, post-transplant $7,358).

Limitations: To aid interpretation of results, note that only patients with GT1 CHC who are actively managed are included, and MRU data were collected specifically from liver outpatient clinics. That said, movement of patients between hospitals is rare, and any uncaptured MRU is expected to be minimal.

Conclusions: CHC-related MRU increases substantially with disease severity. These real-world MRU data for GT1 CHC will be valuable in assessing the impact of new hepatitis C treatments.     

The Effect of the Interaction between Price and Time Preference on Cigarette Consumption among Pregnant Women

In this study, I explore smoking behavior among pregnant U.S. women using the 1979 cohort of the National Longitudinal Survey of Youth. The key aspect of this study is the availability of smoking participation data before and during pregnancy. I consider the probabilities of smoking cessation while pregnant as the outcome. I find that pregnant women who smoke are less responsive to price changes when they are more future-oriented. Women who are more present-oriented are more likely to smoke and consume more cigarettes given that they smoke more than those who are future-oriented. Moreover, those who discount the future more heavily are more sensitive to the money price of cigarettes than those who are more future-oriented. I focus on the role of time preference and the interaction between time preference and price in determining these outcomes.     

Economic evaluation of the use of Innohep® in the treatment of acute pulmonary embolism

Summary

This article reports the cost implications of using Innohep® (tinzaparin), a low-molecular-weight heparin (LMWH), instead of intravenous (iv) unfractionated heparin (UFH) in the treatment of acute pulmonary embolism (PE). The expected cost per patient, including initial treatment costs and the cost of managing adverse outcomes (but excluding costs common to both regimes) was found to be £191.81 for patients treated with UFH and £186.64 for patients treated with Innohep® (costs estimated as of end 1997). Therefore, the use of Innohep® reduces the expected cost per patient by £5.17 or 3%. When the costs of managing adverse outcomes occurring after cessation of UFH/Innohep® therapy are excluded, expected costs per patient for UFH and Innohep® treatment are £136.70 and £120.22, respectively. Therefore, when outcomes not directly attributable to the choice between UFH and Innohep® treatment are reduced, the use of Innohep® reduces the expected cost per patient by £16.48 or 12%.

Innohep® reduces costs through greater ease of administration, by removing the need for extensive laboratory monitoring and by saving staff time. These results are generally robust to variations in key assumptions. Sensitivity analysis demonstrates that if patients treated with Innohep® can be discharged from hospital earlier, with their treatment continuing at home, substantial cost savings may result.     

Differences in the response heterogeneity of educational attainment and achievement to the same instrumental variable

The typical econometric application of instrumental variable (IV) estimates imposes a constant-treatment-effect model for all individuals. Using regression discontinuity design, we find while older school starters tend to have lower educational attainment, they are more likely to achieve qualifications at different educational levels for the UK. This finding joins the discussion on instruments for educational outcomes by offering some evidence that school starting age can affect treated individuals diversely. We highlight that identifying the treatment effect with methods similar to IV estimates should be concerned in the context of heterogeneous treatments; that is, the instrument in question has an effect on treatment for particular subpopulations only, and those subpopulations may not be the same for different treatments.     

Econometrics for Evaluations: An Introduction to Recent Developments

There has recently been a rapid expansion of interest in the econometrics of program evaluation, both within Australia and around the world. We provide a review of the key issues and recent developments in this field. A central feature of recent developments is the attempt to allow for program impacts that vary across individuals. This contrasts with earlier econometric approaches that implicitly assumed a homogeneous treatment effect. We survey alternative nonexperimental estimation strategies and note that they can be characterised by (1) an assumption about how untreated outcomes vary across individuals: this assumption in turn suggests how the counter-factual untreated outcomes of program participants should be estimated, and (2) the way in which the estimator aggregates or weights the program impacts of different individuals in the treatment group. We also emphasise the importance of good data.     

Clinical and economic benefits of professional CGM among people with type 2 diabetes in the United States: analysis of claims and lab data

Background: It is estimated that one in 10 people in the US have a diagnosis of diabetes. Type 2 diabetes accounts for 95% of all cases in the US, with annual costs estimated to be $246 billion per year. This study investigated the impact of a glucose-measuring intervention to the burden of type 2 diabetes.

Objective: This analysis seeks to understand how professional continuous glucose monitoring (professional CGM) impacts clinical and economic outcomes when compared to patients who are not prescribed professional CGM.

Methods: This study utilized a large healthcare claims and lab dataset from the US, and identified a cohort of patients who were prescribed professional CGM as identified by CPT codes 95250 and 95251. It calculated economic and clinical outcomes 1 year before and 1 year after the use of professional CGM, using a generalized linear model.

Results: Patients who utilized professional CGM saw an improvement in hemoglobin A1C. The “difference-in-difference” calculation for A1C was shown to be –0.44%. There was no statistically significant difference in growth of total annual costs for people who used professional CGM compared to those who did not ($1,270, p?=?.08). Patients using professional CGM more than once per year had a –$3,376 difference in the growth of total costs (p?=?.05). Patients who used professional CGM while changing their diabetes treatment regimen also had a difference of –$3,327 in growth of total costs (p?=?.0023).

Conclusion: Significant clinical benefits were observed for patients who used professional CGM. Economic benefits were observed for patients who utilized professional CGM more than once within a 1-year period or who used it during a change of diabetes therapy. This suggests that professional CGM may help decrease rising trends in healthcare costs for people with type 2 diabetes, while also improving clinical outcomes.     

Comparing olanzapine and ziprasidone in the treatment of schizophrenia: a case study in modeling

SUMMARY

When a new drug is introduced to the market, the availability of head-to-head data for comparisons of annual cost and health outcomes with other drugs already on the market is usually insufficient. When only limited head-to-head data are available, one alternative is to perform preliminary modeling using data from the best available studies. In this paper, we synthesise data from the most comparable available studies to create a model to compare the annual costs and health outcomes when initiating treatment for schizophrenia with one of two antipsychotic drugs: olanzapine, which was launched in the United States (US) market in September 1996, and ziprasidone, which was approved by the US Food and Drug Administration (FDA) in February 2001. Annual treatment costs were determined by response and relapse rates as well as acquisition costs. The results indicate similar annual treatment costs (US$48,676 for olanzapine; US$48,873 for ziprasidone), despite the lower drug acquisition cost for ziprasidone. Annual health outcomes differed between the olanzapine and ziprasidone groups with 23.5% and 25.2% relapsed, 36.7 and 37.4 hospital days, and 60.0 and 60.1 EPS days in the olanzapine and ziprasidone groups respectively. A head-to-head naturalistic trial is needed to validate the results of this modeling exercise.     

Institutional economic theory: the old versus the new

The object of this paper is to make some limited comparisons between the ‘new’ institutionalism of Williamson, Schotter, Hayek and others and the ‘old’ institutionalism, particularly of Veblen. It is argued that a fundamental feature of the ‘new’ institutionalism in which it contrasts with the ‘old’ is its close attachment to the idea of the abstract individual of classic liberal ideology. This attachment is found even in those minority subsets of ‘new’ institutionalists who are hostile to promarket policies, or non-neoclassical in matters of theory. Consequently, ‘old’ institutionalist warnings against proceeding on such individualistic assumptions should not be ignored, despite the value of much ‘new’ institutionalist analysis.     

A cost evaluation of treatment alternatives in mild-to-moderate bleeding episodes in haemophilia patients with inhibitors in Turkey

Summary

A decision-analysis model was constructed to assess total direct health care costs of four current first-line treatment options for mild-to-moderate bleeding episodes in haemophilia patients with inhibitors in Turkey: recombinant activated Factor VII (rFVIIa); high-dose Factor VIII; prothrombin complex concentrate (PCC); and activated PCC (aPCC). Resource utilisation was based on a retrospective analysis of 105 bleeding episodes treated during the period January 1996 to December 2002. Clinical outcomes were derived from a combination of the retrospective patient data and literature review, both validated by an expert panel of Turkish haematologists. rFVIIa was more effective and resolved bleeds more quickly than any of the alternatives. rFVIIa and PCC were associated with similar direct treatment costs that were relatively lower than those compared with the other options. Given the better efficacy, rFVIIa should be considered the preferred treatment option in the management of haemophilia patients with inhibitors in Turkey.     

Testing the Participation Constraint in the Executive Labour Market

This paper tests the participation constraint by examining the workings of the executive labour market in a panel of UK listed companies over a period of 14 years. Directors are found to move jobs regularly – both within companies and between companies. Consistent with agency theory, directors who are underpaid relative to their comparable peers are particularly likely to leave for higher paying jobs in other companies. Those who move between companies secure more favourable terms than those who move within their firm – even when the move does not involve promotion, calling into question the managerial power perspective of this area of employment.     

The Labour Market Effects of Vocational Education and Training in Australia

We provide estimates of the effects of completing a Vocational Education and Training (VET) qualification on individual labour market outcomes, particularly on the probability of employment and on earnings. Estimates are provided for 1997, 2001 and 2005. The estimation methodology is based on matched comparisons of persons at each level of VET qualification among Year 12 completers and non‐completers. We find that among Year 12 completers, there is little benefit from obtaining certificate level qualifications, but there are positive employment and earnings outcomes associated with obtaining diploma level qualifications. Among persons who did not complete Year 12, however, there are benefits from obtaining any kind of VET qualification, including the lower level Certificate I and II qualifications.