Using observational analysis of multiple sclerosis relapse to design outcomes-based contracts for disease-modifying drugs: a feasibility assessment

Abstract

Objective:

To assess predictors and costs of multiple sclerosis (MS) relapse, a potential outcome measure in payer-manufacturer risk-sharing agreements for disease-modifying drugs (DMDs).

Methods:

A retrospective cohort analysis of medical/pharmacy claims was used. Study patients had ≥1 DMD (interferon beta, glatiramer, natalizumab) claim, without DMD claims in a 6-month pre-period before DMD initiation; were aged 18–64 years and continuously enrolled from the pre-period through a 24-month post-period; and had ≥2 MS medical claims during the 30-month study period. Post-period relapse cohorts included: (1) severe (hospitalization with MS diagnosis); (2) moderate (outpatient services including intravenous methylprednisolone); and (3) none. Poisson regression modeled severe relapse frequency, logistic regression modeled ≥1 severe relapse, and generalized linear modeling predicted healthcare costs. Tested predictors included demographics, insurance type, index DMD, pre-period health status, and DMD medication possession ratio (MPR).

Results:

Severe relapse was experienced by 14.5% and moderate relapse by 13.8% of 2291 patients. In logistic regression, severe relapse was predicted by plan type; age (odds ratio [OR]?=?1.018, 95% confidence interval [CI]?=?1.005–1.031); pre-period Charlson Comorbidity Index (OR?=?1.307, 95% CI?=?1.166–1.464); pre-period proxy measure indicating impaired activities of daily living (OR?=?1.470, 95% CI?=?1.134–1.905); pre-period MS hospitalization (OR?=?2.174, 95% CI?=?1.537–3.074); and DMD non-adherence (MPR OR?=?0.101, 95% CI?=?0.068–0.151). Poisson regression results were similar. Predicted mean [standard deviation] all-cause healthcare expenditures were tripled for patients with severe compared with moderate relapse ($48,173 [$8665] and $13,334 [$1929], respectively).

Limitations:

Commercially insured patients from a single payer; use may have been inconsistent with approved indications; proxy relapse measure may have misclassified patients.

Conclusions:

Severe MS relapses requiring hospitalization, although affecting less than 15% of patients initiating DMD treatment, are associated with high medical costs. The only actionable predictor of severe relapse identified in observational analysis was MPR, raising questions about the feasibility of using observational data to guide outcomes-based contracting.     

Estimates of the direct and indirect cost savings associated with heart disease that could be avoided through dietary change in the United States

Aims: Diets high in saturated fat are associated with elevated risk of heart disease. This study estimates the savings in direct (medical care) costs and indirect (job absenteeism) costs in the US from reductions in heart disease associated with substituting monounsaturated fats (MUFA) for saturated fats.

Materials and methods: A four-part model of the medical care cost savings from avoided heart disease was estimated using data on 247,700 adults from the 2000–2010 Medical Expenditure Panel Survey (MEPS). The savings from reduced job absenteeism due to avoided heart disease was estimated using a zero-inflated negative binomial model of the number of annual work loss days applied to data on 164,577 adults from the MEPS.

Results: Estimated annual savings in medical care expenditures resulting from a switch from a diet high in saturated fat to a high-MUFA diet totaled ～ $25.7 billion (95% CI = $6.0–$45.4 billion) in 2010, with private insurance plans saving $7.9 billion (95% CI?=?$1.8–$14.0 billion), Medicare saving $9.4 billion (95% CI?=?$2.1–$16.7 billion), Medicaid saving $1.4 billion (95% CI?=?Aims: Diets high in saturated fat are associated with elevated risk of heart disease. This study estimates the savings in direct (medical care) costs and indirect (job absenteeism) costs in the US from reductions in heart disease associated with substituting monounsaturated fats (MUFA) for saturated fats.

Materials and methods: A four-part model of the medical care cost savings from avoided heart disease was estimated using data on 247,700 adults from the 2000–2010 Medical Expenditure Panel Survey (MEPS). The savings from reduced job absenteeism due to avoided heart disease was estimated using a zero-inflated negative binomial model of the number of annual work loss days applied to data on 164,577 adults from the MEPS.

Results: Estimated annual savings in medical care expenditures resulting from a switch from a diet high in saturated fat to a high-MUFA diet totaled ～ $25.7 billion (95% CI = $6.0–$45.4 billion) in 2010, with private insurance plans saving $7.9 billion (95% CI?=?$1.8–$14.0 billion), Medicare saving $9.4 billion (95% CI?=?$2.1–$16.7 billion), Medicaid saving $1.4 billion (95% CI?=?$0.2–$2.5 billion), and patients saving $2.2 billion (95% CI?=?$0.5–$3.8 billion). The annual savings in terms of reduced job absenteeism ranges from a lower bound of $600 million (95% CI?=?$100 million to $1.0 billion) to an upper bound of $1.2 billion (95% CI?=?$0.2–$2.1 billion) for 2010.

Limitations: The data cover only the non-institutionalized population. Decreased costs due to any decreases in the severity of heart disease are not included. Cost savings do not include any reduction in informal care at home.

Conclusions: Diets high in saturated fat impose substantial medical care costs and job absenteeism costs, and substantial savings could be achieved by substituting MUFA for saturated fat.     

Outcomes associated with concordance of oral antidiabetic drug treatments to prescribing information in patients with type 2 diabetes mellitus and chronic kidney disease

Abstract

Objectives:

This retrospective study aims to examine the association between prescribing information (PI)-concordant oral antidiabetic drug (OAD) treatment and clinical and economic outcomes in patients with type 2 diabetes mellitus and stages 3–5 chronic kidney disease (CKD).

Methods:

The study used a large, national administrative claims database with laboratory findings to identify patients with a diagnosis of diabetes and indication of stages 3–5 CKD (first observed indication as the index date) between 1/1/2005 and 30/06/2009. OADs prescribed during 6 months following the index date (baseline period) were evaluated and patients were considered non-PI-concordant if any did not meet the recommendations regarding patients with renal impairment. Glycemic control and measures of healthcare costs (standardized to 2010 US dollars using the Consumer Price Index) and resource utilization were assessed during the 12 months following the baseline period. Severe hypoglycemic events were assessed after the baseline period until lost to follow-up. Regression analyses were performed after adjusting for demographic and clinical characteristics.

Results:

Among the 3300 patients included in the study, 2454 (74.4%) were non-PI-concordant. The non-PI-concordant patients had higher risk of severe hypoglycemic events identified in all settings (HR?=?1.35, 95% CI: 1.10–1.67) and events identified in inpatient hospital setting (HR?=?2.51, 95% CI: 1.49–4.22), were more likely to have inpatient hospital admissions (OR?=?1.27, 95% CI: 1.02–1.57), and were less likely to have glycemic control (OR?=?0.56, 95% CI: 0.44–0.71). Annual diabetes-related cost was $1638 higher in the non-PI-concordant cohort (p?=?0.0048).

Limitations:

The retrospective cohort design does not allow for conclusions to be drawn on the causal effect of PI-concordant use based on the associations observed.

Conclusion:

Our findings suggest PI-concordant treatment to be associated with better clinical and diabetes-associated economic outcomes. Future research is warranted to confirm the associations found in this study.     

Characterizing the financial burden of pulmonary arterial hypertension within an integrated healthcare delivery system

Abstract

Purpose:

Financial burden associated with providing healthcare to patients with pulmonary arterial hypertension (PAH) is poorly characterized. This study sought to quantify 3-year healthcare expenditures and determine whether expenditures differed between incident and prevalent PAH cases.

Methods:

This was a retrospective cohort study of Kaiser Permanente Colorado (KPCO) patients with confirmed diagnosis of PAH. Included patients were followed from study entry until 3 years, death, or termination of KPCO membership, whichever came first. All expenditures were reported in 2011 US dollars from the KPCO perspective.

Results:

In total, 157 patients were included: 44 (28%) prevalent and 113 (72%) incident cases. Mean age (prevalent vs incident cases) was 61 years vs 67 years and 13.6% vs 27.4% were males. The majority of patients (55%) were classified as WHO Group 1 PAH. Prevalent cases had less follow-up (843 vs 975 days; p?=?0.033). Overall, median total per patient per day (PPPD) and 3-year total expenditures were $56 (interquartile range (IQR?=?$29–$166) and $50,599 (IQR?=?$25,958–$135,535), respectively. After adjustment for patient characteristics and chronic disease burden, median PPPD ($54 vs $56; p?=?0.950) and 3-year ($37,340 vs $55,073; p?=?0.111) total expenditures were equivalent between prevalent and incident cases; however, the risk of death during the 3-year follow-up was lower among incident cases (hazard ratio?=?0.41, 95% CI?=?0.18–0.91). No significant differences were detected in pharmacy, inpatient, medical office, emergency department, or other expenditures. Median PAH specialty medication PPPD expenditures were also equivalent, also ($226 vs $223 among specialty medication users; p?=?0.861).

Conclusion:

Healthcare expenditures related to PAH represent substantial financial burden. Significant differences according to prevalent or incident case status appeared to be driven by median ED and inpatient expenditures; however, PAH specialty medication expenditures represented a substantial cost-driver overall. Future efforts should focus on optimizing care for patients with PAH to avoid unnecessary harm or waste.     

Hospital costs associated with intraoperative hypotension among non-cardiac surgical patients in the US: a simulation model

Objective: Recent studies indicate intraoperative hypotension, common in non-cardiac surgical patients, is associated with myocardial injury, acute kidney injury, and mortality. This study extends on these findings by quantifying the association between intraoperative hypotension and hospital expenditures in the US.

Methods: Monte Carlo simulations (10,000 trial per simulation) based on current epidemiological and cost outcomes literature were developed for both acute kidney injury (AKI) and myocardial injury in non-cardiac surgery (MINS). For AKI, three models with different epidemiological assumptions (two models based on observational studies and one model based on a randomized control trial [RCT]) estimate the marginal probability of AKI conditional on intraoperative hypotension status. Similar models are also developed for MINS (except for the RCT case). Marginal probabilities of AKI and MINS sequelae (myocardial infarction, congestive heart failure, stroke, cardiac catheterization, and percutaneous coronary intervention) are multiplied by marginal cost estimates for each outcome to evaluate costs associated with intraoperative hypotension.

Results: The unadjusted (adjusted) model found hypotension control lowers the absolute probability of AKI by 2.2% (0.7%). Multiplying these probabilities by the marginal cost of AKI, the unadjusted (adjusted) AKI model estimated a cost reduction of $272 [95% CI?=?$223–$321] ($86 [95% CI?=?$47–$127]) per patient. The AKI model based on relative risks from the RCT had a mean cost reduction estimate of $281 (95% CI?=?–$346–$750). The unadjusted (adjusted) MINS model yielded a cost reduction of $186 [95% CI?=?$73–$393] ($33 [95% CI?=?$10–$77]) per patient.

Conclusions: The model results suggest improved intraoperative hypotension control in a hospital with an annual volume of 10,000 non-cardiac surgical patients is associated with mean cost reductions ranging from $1.2–$4.6 million per year. Since the magnitude of the RCT mean estimate is similar to the unadjusted observational model, the institutional costs are likely at the upper end of this range.     

The effect of pharmacogenetic profiling with a clinical decision support tool on healthcare resource utilization and estimated costs in the elderly exposed to polypharmacy

Objective:

To compare healthcare resource utilization (HRU) and clinical decision-making for elderly patients based on cytochrome P450 (CYP) pharmacogenetic testing and the use of a comprehensive medication management clinical decision support tool (CDST), to a cohort of similar non-tested patients.

Methods:

An observational study compared a prospective cohort of patients ≥65 years subjected to pharmacogenetic testing to a propensity score (PS) matched historical cohort of untested patients in a claims database. Patients had a prescribed medication or dose change of at least one of 61 oral drugs or combinations of ≥3 drugs at enrollment. Four-month HRU outcomes examined included hospitalizations, emergency department (ED) and outpatient visits and provider acceptance of test recommendations. Costs were estimated using national data sources.

Results:

There were 205 tested patients PS matched to 820 untested patients. Hospitalization rate was 9.8% in the tested group vs 16.1% in the untested group (RR?=?0.61, 95% CI?=?0.39–0.95, p?=?0.027), ED visit rate was 4.4% in the tested group vs 15.4% in the untested group (RR?=?0.29, 95% CI?=?0.15–0.55, p?=?0.0002) and outpatient visit rate was 71.7% in the tested group vs 36.5% in the untested group (RR?=?1.97, 95% CI?=?1.74–2.23, p?<?0.0001). The rate of overall HRU was 72.2% in the tested group vs 49.0% in the untested group (RR?=?1.47, 95% CI?=?1.32–1.64, p?<?0.0001). Potential cost savings were estimated at $218 (mean) in the tested group. The provider majority (95%) considered the test helpful and 46% followed CDST provided recommendations.

Conclusion:

Patients CYP DNA tested and treated according to the personalized prescribing system had a significant decrease in hospitalizations and emergency department visits, resulting in potential cost savings. Providers had a high satisfaction rate with the clinical utility of the system and followed recommendations when appropriate.     

Healthcare costs in patients with advanced non-small cell lung cancer and disease progression during targeted therapy: a real-world observational study

Aims: To assess healthcare costs during treatment with epidermal growth factor receptor (EGFR) tyrosine kinase inhibitors (TKIs) and following disease progression in patients with advanced non-small cell lung cancer (NSCLC).

Methods: A retrospective analysis of medical records of US community oncology practices was conducted. Eligible patients had advanced NSCLC (stage IIIB/IV) diagnosed between January 1, 2008 and January 1, 2015, initiated treatment with erlotinib or afatinib (first-line or second-line), and had disease progression. Monthly Medicare-paid costs were evaluated during the TKI therapy period and following progression.

Results: The study included 364 patients. The total mean monthly cost during TKI therapy was $20,106 (95% confidence interval [CI]?=?$16,836–$23,376), of which 47.0% and 42.4% represented hospitalization costs and anti-cancer therapy costs, respectively. Following progression on TKI therapy (data available for 316 patients), total mean monthly cost was $19,274 (95% CI?=?$15,329–$23,218), and was higher in the 76.3% of patients who received anti-cancer therapy following progression than in the 23.7% of those who did not ($20,490 vs $15,364; p?<?.001). Among patients who received it, anti-cancer therapy ($11,198; 95% CI?=?$7,102–$15,295) represented 54.7% of total mean monthly cost. Among patients who did not receive anti-cancer therapy, hospitalization ($13,829; 95% CI?=?$4,922–$22,736) represented 90.0% of total mean monthly cost. Impaired performance status and brain metastases were significant predictors of increased cost during TKI therapy.

Limitations: The study design may limit the generalizability of findings.

Conclusions: Healthcare costs during TKI treatment and following progression appeared to be similar and were largely attributed to hospitalization and anti-cancer therapy. Notably, almost one-quarter of patients did not receive anti-cancer therapy following progression, potentially indicating an unmet need; hospitalization was the largest cost contributor for these patients. Additional effective targeted therapies are needed that could prolong progression-free survival, leading to fewer hospitalizations for EGFR mutation-positive patients.     

Distribution and drivers of costs in type 2 diabetes mellitus treated with oral hypoglycemic agents: a retrospective claims data analysis

Objective:

To describe the distribution of costs and to identify the drivers of high costs among adult patients with type 2 diabetes mellitus (T2DM) receiving oral hypoglycemic agents.

Methods:

T2DM patients using oral hypoglycemic agents and having HbA1c test data were identified from the Truven MarketScan databases of Commercial and Medicare Supplemental insurance claims (2004–2010). All-cause and diabetes-related annual direct healthcare costs were measured and reported by cost components. The 25% most costly patients in the study sample were defined as high-cost patients. Drivers of high costs were identified in multivariate logistic regressions.

Results:

Total 1-year all-cause costs for the 4104 study patients were $55,599,311 (mean cost per patient?=?$13,548). Diabetes-related costs accounted for 33.8% of all-cause costs (mean cost per patient?=?$4583). Medical service costs accounted for the majority of all-cause and diabetes-related total costs (63.7% and 59.5%, respectively), with a minority of patients incurring >80% of these costs (23.5% and 14.7%, respectively). Within the medical claims, inpatient admission for diabetes-complications was the strongest cost driver for both all-cause (OR?=?13.5, 95% CI?=?8.1–23.6) and diabetes-related costs (OR?=?9.7, 95% CI?=?6.3–15.1), with macrovascular complications accounting for most inpatient admissions. Other cost drivers included heavier hypoglycemic agent use, diabetes complications, and chronic diseases.

Limitations:

The study reports a conservative estimate for the relative share of diabetes-related costs relative to total cost. The findings of this study apply mainly to T2DM patients under 65 years of age.

Conclusions:

Among the T2DM patients receiving oral hypoglycemic agents, 23.5% of patients incurred 80% of the all-cause healthcare costs, with these costs being driven by inpatient admissions, complications of diabetes, and chronic diseases. Interventions targeting inpatient admissions and/or complications of diabetes may contribute to the decrease of the diabetes economic burden.     

The cost-effectiveness of extended-release calcifediol versus paricalcitol for the treatment of secondary hyperparathyroidism in stage 3–4 CKD

Abstract

Aims: Patients with chronic kidney disease (CKD) not on dialysis frequently have vitamin D insufficiency (VDI) and secondary hyperparathyroidism (SHPT), which are associated with an increased risk of cardiovascular (CV) disease, fracture, CKD progression, and death. This study estimated the cost-effectiveness of extended-release calcifediol (ERC) vs paricalcitol for the treatment of patients with CKD stages 3–4 that have SHPT and VDI.

Materials and methods: An economic analysis of SHPT treatments among a hypothetical cohort of 1,000 patients with CKD Stage 3 and 4 with SHPT and VDI was developed to estimate differences in the rates and costs of CV events, fractures, CKD stage progression, and mortality in patients treated with ERC and paricalcitol. A Markov model was developed with 1-year cycles and a 5-year time horizon from a US Medicare payer perspective with costs valued in 2017?US dollars.

Results: The outcomes of the model were rates of clinical events, total costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER). Across a 1,000-person cohort, ERC was the dominant (less costly, more effective) treatment strategy when compared with paricalcitol. Treatment with ERC resulted in cost savings of $14.8?M (95% CI = –$10.0?M–$45.2?M) and an incremental gain of 340 QALYs (95% CI = 200–496) compared to treatment with paricalcitol.

Limitations: Bridging biochemical levels to clinical outcomes may not represent real-world risk of the clinical events modeled. Future real-world outcomes of patients treated with ERC and paricalcitol may be used to evaluate the model results.

Conclusions: This model demonstrated favorable short- and long-term clinical benefits associated with the use of ERC in patients with CKD Stage 3 and 4 with SHPT and VDI, suggesting ERC may be cost-effective from the Medicare perspective compared to paricalcitol.     

Relationship of hospital-associated bleeding with length of stay and total hospitalization costs in patients hospitalized for atrial fibrillation

Background:

While literature has focused on the impact of bleeding beginning outside the hospital setting among patients with atrial fibrillation (AF), there is little information regarding bleeding that first occurs within a hospital setting. This study was performed to determine the association between hospital-associated bleeding in patients admitted for AF on outcomes of length of stay (LOS) and total hospitalization cost.

Methods and results:

The Premier research database was queried to identify adult inpatients discharged between 2008–2011 having a primary diagnosis code for AF where a bleeding diagnosis code was not present on admission. Regression was used to adjust for baseline differences in patients to estimate outcomes comparing patients with and without a hospital-associated bleed. There were 143,287 patients that met the study criteria. There were 2991 (2.1%) patients identified with a hospital associated bleed. After adjustment for covariates, the mean estimated LOS was significantly greater in the bleed group, at 6.0 days (95% CI?=?5.8–6.1) vs the no bleed group at 3.3 days (95% CI?=?3.3–3.3) (p?<?0.0001). Similarly, the adjusted mean estimated total hospitalization cost was also significantly greater in the bleed group, $12,069 (95% CI?=?$11,779–$12,366) vs $6561 (95% CI?=?$6538–$6583) in the no bleed group (p?<?0.0001).

Conclusions:

After adjustments for baseline differences the data show that the 2.1% (n?=?2991) of patients with hospital associated bleeding accounted for an estimated additional 8106 hospitalization days and $16.4 million dollars in cost over the study period compared to non-bleeders.     

Major bleeding risk and healthcare economic outcomes of non-valvular atrial fibrillation patients newly-initiated with oral anticoagulant therapy in the real-world setting

Aims: This study compared the risk for major bleeding (MB) and healthcare economic outcomes of patients with non-valvular atrial fibrillation (NVAF) after initiating treatment with apixaban vs rivaroxaban, dabigatran, or warfarin.

Methods: NVAF patients who initiated apixaban, rivaroxaban, dabigatran, or warfarin were identified from the IMS Pharmetrics Plus database (January 1, 2013–September 30, 2015). Propensity score matching (PSM) was used to balance differences in patient characteristics between study cohorts: patients treated with apixaban vs rivaroxaban, apixaban vs dabigatran, and apixaban vs warfarin. Risk of hospitalization and healthcare costs (all-cause and MB-related) were compared between matched cohorts during the follow-up.

Results: During the follow-up, risks for all-cause (hazard ratio [HR]?=?1.44, 95% confidence interval [CI]?=?1.2–1.7) and MB-related (HR?=?1.57, 95% CI?=?1.0–2.4) hospitalizations were significantly greater for patients treated with rivaroxaban vs apixaban. Adjusted total all-cause healthcare costs were significantly lower for patients treated with apixaban vs rivaroxaban ($3,950 vs $4,333 per patient per month [PPPM], p?=?.002) and MB-related medical costs were not statistically significantly different ($100 vs $233 PPPM, p?=?.096). Risk for all-cause hospitalization (HR?=?1.98, 95% CI?=?1.6–2.4) was significantly greater for patients treated with dabigatran vs apixaban, although total all-cause healthcare costs were not statistically different. Risks for all-cause (HR?=?2.22, 95% CI?=?1.9–2.5) and MB-related (HR?=?2.05, 95% CI?=?1.4–3.0) hospitalizations were significantly greater for patients treated with warfarin vs apixaban. Total all-cause healthcare costs ($3,919 vs $4,177 PPPM, p?=?.025) and MB-related medical costs ($96 vs $212 PPPM, p?=?.026) were significantly lower for patients treated with apixaban vs warfarin.

Limitations: This retrospective database analysis does not establish causation.

Conclusions: In the real-world setting, compared with rivaroxaban and warfarin, apixaban is associated with reduced risk of hospitalization and lower healthcare costs. Compared with dabigatran, apixaban is associated with lower risk of hospitalizations.     

Time spent on erythropoietin stimulating agents administration in hemodialysis centers in Panama: a time and motion study

Objectives: There is a lack of data in Panama on the potential differences in total healthcare professional (HCP) time between routine administrations of short-acting erythropoietin simulating agents (ESAs) (i.e. epoetin alfa) and continuous erythropoietin receptor activator (CERA) (i.e. methoxy polyethylene glycol–epoetin beta). This study aimed to quantify the HCP time associated with a single administration of epoetin alfa and CERA for the treatment of anemic patients with chronic kidney disease (CKD) on hemodialysis.

Methods: This was a multi-center, cross-sectional study, using a time-and-motion methodology. Costs related to HCP time and consumables usage associated with administration of epoetin alfa and CERA were estimated.

Results: Based on 60 administrations of either CERA or epoetin alfa, the estimated savings in mean total active HCP time were 2.34 (95% confidence interval?=?1.87–2.81) min (–30%) per administration. When extrapolating to a full year’s treatment with intravenous ESA, it would require a total of 20.3 (95% CI?=?19.90–20.71) h of HCP time for epoetin alfa vs 1.1 (95% CI?=?1.01–1.19) h for CERA per patient per year. Estimated savings in active HCP time per patient per year were 19.20 (95% CI?=?19.20–19.21) h (–95%). This, in turn, translates into staff cost efficiency that favors Mircera with an estimated annual saving of $78.24 (95% CI?=?78.24–78.28) (–95%) per patient.

Conclusions: Data from a real-world setting showed that the adoption of CERA could potentially lead to a reduction in active HCP time.

• Highlights

• Few comparative data have explored the costs and potential savings of using long-acting erythropoietin–stimulating agents (ESA) instead of short-acting ESAs to treat anemia in CKD patients on hemodialysis.

• This time-and-motion study shows that use of CERA reduces total healthcare professional time and could represent a save for an institution in a real-world setting in Panama.

     

Healthcare costs for Crohn’s disease patients treated with infliximab: a propensity weighted comparison of the effects of treatment adherence

Objective:

The objective for the research was to evaluate the direct healthcare costs for Crohn’s disease (CD) patients categorized by adherence status.

Methods:

Adult patients with ≥1 claim for infliximab and ≥2 claims for CD who were continuously insured for 12 months before and after their first infliximab infusion (index date) were identified in a 2006–2009 US managed care database. Patients were excluded if they had rheumatoid arthritis claims, received infliximab billed as a pharmacy benefit, or received another biologic drug. Patients were categorized as being either adherent or intermittently adherent to infliximab using a pre-defined algorithm. Total and component direct costs, CD-related costs, rates of surgery, and days of hospitalization were estimated for the 360-day post-index period. Propensity weighted generalized linear models were used to adjust the cost estimates for potential confounding variables.

Results:

The total propensity weighted cost for infliximab adherent patients was $40,425 (95% CI?=?[$38,686, $42,242]), compared to $41,082 (95% CI?=?[$38,163, $44,223]) for the intermittently adherent (p?=?0.71). However, adherent patients had lower total direct medical costs, exclusive of infliximab, that were $13,097 (95% CI?=?[$12,141, $14,127]) compared with $20,068 (95% CI?=?[$17,676, $22,784]) for intermittently adherent patients as a result of substantially lower hospital and outpatient costs (p?Conclusions:

Greater drug-related costs for infliximab adherent patients were offset by lower costs from hospitalization and outpatient visits. These findings indicate that adherent patients have improved clinical outcomes, at a similar aggregate cost, than patients who are only intermittently adherent to therapy.     

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.     

The STARRT trial: a cost comparison of optimal vs sub-optimal initiation of dialysis in Canada

Abstract

Background:

Sub-optimal transitioning of patients from chronic kidney disease (CKD) to end stage renal disease (ESRD) may result in poor clinical outcomes and increased healthcare costs. The objectives of this study were to estimate the average total cost per patient who requires initiation of renal replacement therapy (RRT) stratified by status at initiation; optimal (RRT initiation as an outpatient with an arterio-venous [AV] Fistula, Graft or Peritoneal Dialysis [PD] catheter), and sub-optimal (RRT initiation as an inpatient and/or via central venous catheter [CVC]).

Methods:

Data from the Study To Assess Renal Replacement Therapy (STARRT), a Canadian, multi-centre, 6 month retrolective study (n?=?339), were used for this analysis. Unit costs for resources were obtained from participating hospitals, the literature, and/or standard costing sources. The analysis was performed from the perspective of healthcare payors and reported in 2011 Canadian Dollars (CAD). A propensity score technique was applied to control for potential confounders between the two groups.

Results:

Two hundred of the eligible patients for analysis (61.9%) were sub-optimally and 123 (38.1%) were optimally prepared. For this analysis, 106 “matched” pairs were used. The average total cost per patient was estimated to be $63,225 (with a 95% CI ranging from $58,663–$67,958) for the sub-optimally initiated patients, and $39,260 (with a 95% CI ranging from $35,683–$43,007) for the optimally initiated patients (p?<?0.001).

Limitations:

Costs were calculated utilizing a conservative approach, using the cheapest available prices for medications and other resources. Assumptions had to be made for the costing of dialyses.

Conclusion:

The results of this study indicate, after adjusting for potential confounders, that optimally initiated patients for RRT have significantly lower healthcare-associated costs compared to sub-optimally initiated patients.     

Outcomes of tumor necrosis factor inhibitor cycling versus switching to a disease-modifying anti-rheumatic drug with a new mechanism of action among patients with rheumatoid arthritis

Objectives: To examine treatment patterns, treatment effectiveness, and treatment costs for 1 year after patients with rheumatoid arthritis switched from a tumor necrosis factor inhibitor (TNFi) (adalimumab, certolizumab pegol, etanercept, golimumab, or infliximab), either cycling to another TNFi (“TNFi cyclers”) or switching to a new mechanism of action (abatacept, tocilizumab, or tofacitinib) (“new MOA switchers”).

Methods: This retrospective cohort study used administrative claims data for a national insurer. Treatment persistence (without switching again, restarting, or discontinuing), treatment effectiveness (defined below), and costs were assessed for the 12-month post-switch period. Patients were “effectively treated” if they satisfied all six criteria for a treatment effectiveness algorithm (high adherence, no dose increase, no new conventional synthetic disease-modifying anti-rheumatic drug, no subsequent switch in therapy, no new/increased oral glucocorticoids, and <2 glucocorticoid injections). Multivariable logistic models were used to adjust for baseline factors.

Results: The database included 581 new MOA switchers and 935 TNFi cyclers. New MOA switchers were 39% more likely than TNFi cyclers to persist after the switch (odds ratio [OR]?=?1.39; 95% confidence interval [CI]?=?1.12–1.74; p?=?.003) and 36% less likely to switch therapy again (OR?=?0.64; 95% CI?=?0.51–0.81; p?p?=?.006). New MOA switchers had 16% lower drug costs than TNFi cyclers (cost ratio?=?0.84; 95% CI?=?0.79–0.88; p?p?Limitations: Claims payments may not reflect rebates or other cost offsets. Medical and pharmacy claims do not include clinical end-points or reasons that lead to new MOA switching vs TNFi cycling.

Conclusions: These results support switching to a new MOA after a patient fails treatment with a TNFi, which is consistent with recent guidelines for the pharmacologic management of established rheumatoid arthritis.     

The association of pain interference and opioid use with healthcare utilization and costs,and wage loss among adults with osteoarthritis in the United States

Abstract

Aim: To examine associations of opioid use and pain interference with activities (PIA), healthcare resource utilization (HRU) and costs, and wage loss in noninstitutionalized adults with osteoarthritis in the United States (US).

Methods: Adults with osteoarthritis identified from the Medical Expenditure Panel Survey for 2011/2013/2015 were stratified by no-opioid use with no/mild PIA, no-opioid use with moderate/severe PIA, opioid use with no/mild PIA, and opioid use with moderate/severe PIA. Outcomes included annualized total HRU, direct healthcare costs, and wage loss. Multivariable regression analyses were used for comparisons versus no-opioid use with no/mild PIA (referent). The counterfactual recycled prediction method estimated incremental costs. Results reflect weighted nationally representative data.

Results: Of 4,921 participants (weighted n?=?20,785,007), 46.5% had no-opioid use with no/mild PIA; 23.2% had no-opioid use with moderate/severe PIA; 9.6% had opioid use with no/mild PIA; and 20.7% had opioid use with moderate/severe PIA. Moderate/severe PIA and/or opioid use were associated with significantly higher HRU and associated costs, and wage loss. Relative to adults with no/mild PIA, opioid users with moderate/severe PIA were more likely to have hospitalizations, specialist visits, and emergency room visits (all p?<?.001). Relative to the referent, opioid use with no/mild PIA had higher per-patient incremental annual total healthcare costs ($11,672, 95% confidence interval [CI]?=?$11,435–$11,909) and wage loss ($1,395, 95% CI?=?$1,376–$1,414) as did opioid use with moderate/severe PIA ($13,595, 95% CI?=?$13,319–$13,871; and $2,331, 95% CI?=?$2,298–$2,363) (all p?<?.001). Compared with the referent, estimated excess national total healthcare costs/lost wages were $23.3 billion/$1.3 billion for opioid use with no/mild PIA, and $58.5 billion/$2.2 billion for opioid use with moderate/severe PIA.

Limitations: Unobservable/unmeasured factors that could not be accounted for.

Conclusions: Opioid use with moderate/severe PIA had significantly higher HRU, costs, and wage loss; opioid use was more relevant than PIA to the economic burden. These results suggest unmet needs for alternative pain management strategies.     

Healthcare resource utilization and direct costs associated with frequent nausea in episodic migraine: results from the American Migraine Prevalence and Prevention (AMPP) Study

Abstract

Background:

Nausea is a common migraine symptom that is associated with impaired quality-of-life and functional disability. In this study, population-based data were used to elucidate the relationship between nausea frequency and headache-related healthcare utilization and costs in persons with migraine.

Research design and methods:

Participants with episodic migraine who completed the 2009 American Migraine Prevalence and Prevention (AMPP) Study survey rated their headache-related nausea as occurring never, rarely, <half the time, or ≥half the time with their headaches, and completed questions on symptom frequency and healthcare resource utilization.

Main outcomes measures:

Ordinal logistic regression models were used to assess the association between nausea frequency and headache-related healthcare utilization. Healthcare cost equivalents were calculated.

Results:

Among the 6488 respondents with episodic migraine, the number of respondents observed across headache-related nausea frequency strata were 6.9% for never, 14.5% for rarely, 29.1% for <half the time, and 49.5% for ≥half the time. In unadjusted models, the odds of having ≥1 healthcare encounter for headache in the preceding year increased with frequency of nausea for primary care/obstetrics-gynecology visits (OR?=?1.41; 95% CI?=?1.30–1.52, p?<?0.001), nurse practitioner/physician assistant visits (OR?=?1.52; 95% CI?=?1.25–1.85, p?<?0.001), neurology/headache clinic visits (OR?=?1.33, 95% CI?=?1.18–1.51, p?<?0.001), pain clinic visits (OR?=?1.31, 95% CI?=?1.01–1.71, p?<?0.05), emergency department visits (OR?=?1.85; 95% CI?=?1.56–2.19, p?<?0.01), and overnight hospital stays (OR?=?1.50, 92% CI?=?1.12–2.00, p?<?0.01). The odds of having ≥1 lifetime CT scan or MRI also increased with the frequency of nausea (p?<?0.001 for both). Results remained significant in these analyses when controlling for sociodemographics and overall symptom severity except in the case of pain clinic visits (p?<?0.107). Visits for Mental Health and visits for Chiropractic/Alternative care did not differ significantly by nausea group in unadjusted or adjusted models. Mean estimated direct headache-related healthcare cost equivalents per person per year generally increased with increasing headache-related nausea frequency across categories of healthcare utilization. Average per person healthcare cost for nausea ≥half the time vs nausea never was $179 and $49 yearly for outpatient services, $183 vs $20 yearly for overnight hospital stays, and $314 vs $257 for lifetime diagnostic services/imaging.

Conclusions:

Direct costs of migraine increase with increasing frequency of migraine-associated nausea. Both frequency and severity of headache-related nausea should be monitored as part of ongoing care of persons with migraine. Headache-related nausea, like headache pain, should be considered an area of central concern during clinical, diagnostic, and treatment optimization assessments.

Study limitations:

This study relied on self-reported headache frequency and healthcare costs which are subject to recall bias and under-reporting; however, reporting bias is unlikely to be different as a function of nausea frequency. In addition, medication use costs and indirect costs (which may be higher than direct costs for migraine) were not assessed.