Clinical, economic, and health-related quality of life outcomes in patients with overweight or obesity in the United States: 2016-2018.

Objectives: This study aimed to estimate clinical, economic (including productivity), and health-related quality of life (HRQoL) outcomes and associated individual characteristics among adults with overweight (OW) or obesity in the United States. Methods: This study included adult respondents with body mass index (BMI) ≥18.5 kg/m2 in the 2017-2018 National Health and Nutrition Examination Survey (NHANES) and 2016 Medical Expenditure Panel Survey. Respondents were classified according to BMI. Individual characteristics were described by BMI categories. Multivariable regression models estimated the association between BMI categories and outcomes, adjusting for individual characteristics. Results: Nearly three-quarters (73.7%) of NHANES participants were OW or obese. Relative to Normal weight (NW), respondents with Class 3 obesity had more obesity-related complications (2.07 vs. 4.62, p < 0.001). Higher BMI was associated with significantly lower HRQoL, lower productivity, and higher healthcare expenditures as well as more frequent weight loss attempts in the previous 12 months. Weight loss surgery and prescription anti-obesity medications (AOMs) were used only by a very small proportion of individuals. Despite frequent weight loss attempts, most respondents did not achieve clinically meaningful weight loss. Conclusions: Adults with OW or obesity experienced worse clinical, economic and HRQoL outcomes than those with NW. Better use of evidence-based obesity treatments, including prescription AOMs, should be considered to achieve more clinically meaningful weight reduction and improved outcomes in individuals with OW or obesity.

Diagnosis, testing, treatment, and outcomes among patients with advanced non-small cell lung cancer in the United States.

INTRODUCTION: Characteristics of patients in clinical trials may differ from those of real-world patients. Our objective was to describe biomarker testing and outcomes among patients with advanced non-small cell lung cancer (aNSCLC) in a real-world setting. METHODS: This retrospective cohort study included patients ≥18 years old, diagnosed with stage IIIB/C or IV NSCLC, and in the TEMPUS oncology dataset from January 1, 2012, to December 31, 2020. Patient characteristics associated with biomarker testing were evaluated in patients with positive biomarkers using univariate logistic regression models. Cox proportional hazard models were used to estimate median survival. RESULTS: Of 9540 patients included, 41.7% had biomarker testing, and 2158 had a positive biomarker result. Men (vs women; odds ratio [OR], 0.82; 95% CI: 0.74-0.91), Black patients (vs White; OR, 0.83; 95% CI: 0.72-0.97), patients with squamous (OR, 0.22; 95% CI: 0.19-0.25) or unknown histology (OR, 0.53; 95% CI: 0.45-0.61) (vs non-squamous histology), and patients with an Eastern Cooperative Oncology Group performance status (ECOG PS) of 2+ (OR, 0.69; 95% CI: 0.57-0.84) or missing (OR, 0.56; 95% CI: 0.48-0.66) (vs ECOG PS of 0) were less likely to undergo biomarker testing. Patients with positive biomarkers who received NCCN-recommended treatment options (55.7%) had significantly longer median overall survival (OS) (hazard ratio [HR], 0.84; 95% CI: 0.75-0.95) and real-world progression-free survival (rwPFS) (HR, 0.68; 95% CI: 0.62-0.75). CONCLUSION: More than 50% of patients were untested for biomarkers. Patients who were less likely to be tested included men, Black patients, current smokers, patients with squamous aNSCLC, and patients with an ECOG PS of 2+. Patients with positive biomarkers who received NCCN-recommended treatment options had significantly longer OS and PFS.

Outcomes of Warfarin Home INR Monitoring vs Office-Based Monitoring: a Retrospective Claims-Based Analysis.

BACKGROUND: Home INR testing (patient self-testing) is feasible and effective for warfarin patients but little is known about real-world differences in outcomes for patients using PST versus laboratory-based INR monitoring. OBJECTIVE: To compare the safety/efficacy of patient self-testing of real-world warfarin therapy versus office/lab-based monitoring of therapy. DESIGN/SETTING/PARTICIPANTS/EXPOSURE: A retrospective claims-based analysis of warfarin patients enrolled in the MarketScan® Commercial Claims and Encounters and Medicare databases between January 1, 2013, and March 30, 2020. Stratification was based on INR testing method: patient self-testing versus testing at physicians' offices/local laboratory. The probability of adverse events in each cohort was determined after adjusting for demographic and baseline clinical characteristics using a repeated measures analysis. MAIN MEASURES: Rates of all adverse events: deep venous thrombosis, pulmonary embolism, bleeding, and stroke. A secondary outcome of interest was emergency department visits. KEY RESULTS: A total of 37,837 patients were included in the analysis: 1592 patients in the patient self-testing group and 36,245 in the office-based therapy group. After adjusting for demographic and baseline clinical characteristics, patients in the office-based group had statistically significantly higher rates of all adverse events (incidence rate ratio [IRR]=2.07, 95% CI [1.82, 2.36]), and specific adverse events including thromboembolism (IRR=4.38, 95% CI [3.29, 5.84]), major bleed (IRR=1.45, 95% CI [1.28, 1.64]), and stroke (IRR=1.30, 95% CI [1.05, 1.61]) than patients in the patient self-testing group. Office-based patients also had a statistically significant higher rate of emergency department visits than patient self-testing patients (IRR = 1.65, 95% CI [1.47, 1.84]). CONCLUSIONS/RELEVANCE: This analysis of real-world claims data shows lower rates of stroke, thromboembolism, and major bleeding, as well as fewer emergency department visits, with patient self-testing compared to office-based/lab INR monitoring. Our finding that PST is safe and effective among current users suggests that more patients may benefit from its use.

Cognition and education benefits of increased hemoglobin and blood oxygenation in children with sickle cell disease.

BACKGROUND: Among individuals with sickle cell disease (SCD), decreased hemoglobin is associated with lower oxygen saturation (SpO2) and increased risk of stroke, both of which are associated with lower intelligence quotient (IQ) scores. Thus, increasing hemoglobin and SpO2 in individuals with SCD may increase IQ and educational attainment. METHODS: A cohort simulation model was built to determine academic performance and educational attainment based on cognitive function (measured by IQ) of a pediatric SCD cohort randomly assigned to treatment and control groups. The model contained two key stages: childhood (<10 years) and adolescence (≥10 years). In stage 1, increased hemoglobin and increased SpO2 (assigned to the treatment group) were determinants of higher IQ, prevention of IQ deterioration over time. Increased hemoglobin was also a determinant of decreased stroke risk. In stage 2, improvement in adolescent IQ as a result of treatment was a determinant of academic performance. RESULTS: In a simulated cohort of 2000 children and adolescents with SCD (52.5% female, 50% treated), stroke incidence was predicted to be 44.4% lower among the treated group than the untreated group (4.5% versus 8.1%, respectively). The average IQ among the treated group was estimated to be 91.1 compared with 82.9 in the untreated group (a 9.9% difference; P<0.001). Finally, high school (≥12 years of education) completion rates were estimated to be 64.7% higher among the treated group: 76.1% of the treated group was projected to complete high school compared with 46.2% of the untreated group. CONCLUSIONS: Our model predicts that an average improvement in hemoglobin of 1.1 g/dL (11 g/L) among individuals with SCD may be associated with improved neurocognition and educational outcomes. These improvements may also generate benefits not captured by our model, including improved quality of life, employment, and income.

Incremental Health Care Costs of Anxiety and Depression Among Medicare Beneficiaries With Cancer.

PURPOSE: Mental health comorbidities are commonplace among patients with cancer and have been associated with adverse health outcomes and elevated health care costs. Given the rapidly evolving cancer care landscape, an updated understanding of the prevalence and costs of mental health conditions among patients with cancer is needed. This study assessed the incremental costs of anxiety and depression among Medicare beneficiaries with cancer. METHODS: This retrospective cohort study used the SEER-Medicare database. Patients diagnosed with melanoma, breast, lung, prostate, or colorectal cancer between July 2013 and December 2017 were followed for at least 12 months and up to 36 months after cancer diagnosis. Patients were categorized on the basis of anxiety/depression (AD) diagnosis: (1) predating cancer, (2) onset after cancer, or (3) no AD. Multivariable regression was used to estimate differences in all-cause incremental costs (before v after cancer) between the three groups. RESULTS: Of 230,626 patients, 10% had AD before their cancer diagnosis and 22% were diagnosed after cancer. In the first year after cancer diagnosis, average monthly health care costs were $5,750 in US dollars (USD) for patients with newly onset, $5,208 (USD) for patients with preexisting, and $3,919 (USD) for patients without a diagnosis of AD. The incremental cost of cancer was the greatest among patients with newly onset AD-$1,458 (USD) per month greater than those with no AD. Similar patterns were observed across cancer types and stages. CONCLUSION: One in three Medicare beneficiaries with cancer in this study had a diagnosis of anxiety or depression. Newly onset AD is associated with an increase in health care costs of $17,496 (USD) per year. Screening and management of mental health conditions for patients with cancer should be part of coordinated oncology care.

Estimating Productivity Loss from Breast and Non-Small-Cell Lung Cancer among Working-Age Patients and Unpaid Caregivers: A Survey Study Using the Multiplier Method.

Background. Traditional approaches to capturing health-related productivity loss (e.g., the human capital method) focus only on the foregone wages of affected patients, overlooking the losses caregivers can incur. This study estimated the burden of productivity loss among breast cancer (BC) and non-small-cell lung cancer (NSCLC) patients and individuals caring for such patients using an augmented multiplier method. Design. A cross-sectional survey of BC and NSCLC patients and caregivers measured loss associated with time absent from work (absenteeism) and reduced effectiveness (presenteeism). Respondents reported pre- and postcancer diagnosis income, hours worked, and time to complete tasks. Exploratory multivariable analyses examined correlations between respondents' clinical/demographic characteristics-including industry of employment-and postdiagnosis productivity. Results. Of 204 patients (104 BC, 100 NSCLC) and 200 caregivers (100 BC, 100 NSCLC) who completed the survey, 319 participants (162 BC, 157 NSCLC) working ≥40 wk/y prediagnosis were included in the analysis. More than one-third of the NSCLC (33%) and BC (43%) patients left the workforce postdiagnosis, whereas only 15% of caregivers did. The traditional estimate for the burden of productivity loss was 66% lower on average than the augmented estimate (NSCLC patients: 60%, BC patients: 69%, NSCLC caregivers: 59%, and BC caregivers: 73%). Conclusions. Although patients typically experience greater absenteeism, productivity loss incurred by caregivers is also substantial. Failure to account for such impacts can result in substantial underestimation of productivity gains novel cancer treatments may confer by enabling patients and caregivers to remain in the workforce longer. Our results underscore the importance of holistic approaches to understanding this impact on both patients and their caregivers and accounting for such considerations when making decisions about treatment and treatment value. Highlights: Cancer can have a profound impact on productivity. This study demonstrates how the disease affects not only patients but also the informal or unpaid individuals who care for patients.An augmented approach to calculating health-related productivity loss suggests that productivity impacts are much larger than previously understood.A more comprehensive understanding of the economic burden of cancer for both patients and their caregivers suggests the need for more support in the workplace for these individuals and a holistic approach to accounting for these impacts in treatment decision making.

The value of survival gains from therapeutic innovations for US patients with relapsed/refractory multiple myeloma.

AIMS: This study quantifies the value of survival gains attributable to novel treatments approved since 2003 for United States (US) patients with relapsed/refractory multiple myeloma (RRMM). METHODS: We estimated the increase in survival attributable to lenalidomide and bortezomib for multiple myeloma (MM) patients in the 1983-2013 Surveillance, Epidemiology, and End Results (SEER) registry. To estimate the survival benefit of treatments approved since 2015 (carfilzomib, elotuzomab, daratumumab, used in combination with lenalidomide and dexamethasone) we used clinical trial data to calibrate survival estimated using the SEER data. We then conducted an economic valuation of the estimated shift in survival curves for all therapies. Finally, we estimated the share of the value accruing to patients and manufacturers using treatment costs estimated from MarketScan data. RESULTS: The introduction of bortezomib in combination with dexamethasone (Vd) and lenalidomide in combination with dexamethasone (Rd) resulted in substantial survival gains and societal value for multiple myeloma patients, generating 1.7 additional life-years per RRMM patient. More recently, approved novel treatments have improved survival over effective treatments (i.e. Rd/Vd) by an additional 2.5 life-years - the monetary value of this incremental survival benefit far exceeds the incremental cost of treatment. At the patient level, the incremental benefit of Rd/Vd is $335,500 and with novel treatments is $565,000. Applying this benefit to all future cohorts of US RRMM patients translates into a value of at least $75 billion and $130 billion with Rd/Vd and the novel treatments, respectively. CONCLUSIONS: SEER registry data were only available through 2013. Therefore, survival gains for recently approved treatments were estimated based on clinical trials, rather than observed survival. Our valuation analysis does not capture sources of value aside from survival gains, for example, better quality of life, increased productivity, or the value of surviving until subsequent novel therapies become available. Substantial extensions in life expectancy in RRMM since 2003 translate into real economic value gained by society.

Two steps forward, one step back: 50 years of societal value from LDL-C-lowering therapies.

OBJECTIVES: To assess the evolving landscape of low-density lipoprotein cholesterol-lowering therapies (LLTs) and quantify their effect on cardiovascular disease (CVD)-related mortality and morbidity. STUDY DESIGN: Secondary data came from LLT clinical trials and 1999-2014 National Health and Nutrition Examination Survey (NHANES) data. 1996-2016 Medical Expenditure Panel Survey (MEPS) data were used to estimate LLT spending. Nonfatal CVD events prevented by LLTs were calculated from clinical trials and NHANES. The value of nonfatal events prevented was calculated as the product of event treatment costs and the number of events prevented. The value of mortality reduction was calculated as the product of a value of a life-year and the life expectancy gain from LLTs. This was compared with LLT spending estimated using MEPS. METHODS: Total LLT expenditures were calculated based on MEPS LLT utilization and expenditure data. Values of prevented hospitalizations, prevented CVD events, and other LLT utilization-related outcomes were pulled from the published literature. RESULTS: Combined, statins and ezetimibe prevented 2.8 million nonfatal heart attacks and 1.7 million nonfatal strokes from 1999 to 2014. Statin use generated $2.6 trillion in societal value through CVD deaths avoided from 1987 to 2014, and 85% accrued to patients. CONCLUSIONS: LLTs have yielded significant societal value, and the majority of this value has accrued to patients.

Changes in mortality associated with cancer drug approvals in the United States from 2000 to 2016.

AIMS: To estimate the extent to which the approvals of new pharmacological therapies were associated with cancer mortality in the USA between 2000 and 2016. MATERIALS AND METHODS: The analysis quantified cancer drug approvals across the 15 tumor types with the highest incidence. Number of approvals in a given time period for each tumor was translated into a treatment stock measure, defined as a weighted sum of new indication approvals since 1976. The primary outcome was the annual tumor-specific cancer mortality, defined as the number of deaths per 100,000 U.S. population. The analysis used a multivariable ordinary least squares and a fixed effects model, controlling for incidence (new cases per 100,000 U.S. population) and the primary exposure, the treatment stock measure by year. RESULTS: Between 2000 and 2016, deaths per 100,000 population across the 15 most common tumor types declined by 24%. Additionally, 10.2 new indications were approved per year across the 15 most common tumor types. Cancer drug approvals were associated with statistically significant deaths averted in 2016 for colorectal cancer (4,991, p = 0.004), lung cancer (33,825, p < 0.001), breast cancer (11,502, p < 0.001), non-Hodgkin's lymphoma (6,636, p < 0.001), leukemia (4,011, p < 0.001), melanoma (1,714, p < 0.001), gastric cancer (758, p = 0.019), and renal cancer (739, p < 0.001). Between 2000 and 2016, new cancer treatments were correlated with 1,291,769 (p < 0.001) total deaths prevented across the 15 most common tumor types. LIMITATIONS AND CONCLUSIONS: Cancer drug approvals between 2000 and 2016 were associated with significant reduction in deaths from the most common cancers in the USA. Mortality changes were largest in prevalent tumor types with relatively more approvals, i.e. lung cancer, breast cancer, melanoma, lymphoma and leukemia. Future research evaluating the relationship between drug approvals and cancer mortality post 2016 is needed.

Non-Small Cell Lung Cancer Patient Preferences for First-Line Treatment: A Discrete Choice Experiment.

Background. There has been much innovation in the treatment of non-small cell lung cancer (NSCLC) in recent years. In particular, use of immuno-oncology (IO) therapies has been growing. Methods. Patients with NSCLC in the United States were surveyed online using a discrete choice experiment to elicit first-line (1L) treatment preferences across six treatment attributes: survival, adverse events (AEs), mechanism of action (MOA), subsequent treatment options (STOs), genetic testing treatment delay, and out-of-pocket cost (OOPC). Preferences were estimated using a latent-class model. Preference shares were estimated for IO-IO, IO-chemo, and chemo-like regimens. Results. Of the 199 patients who completed the survey, 55% were male, 76% were white, 19% had not begun or were on 1L treatment, and the median age was 43 years. Based on a latent-class model with 3 preference classes, 53.0% of patients considered survival and OOPC alone and were less likely to choose an option with a higher OOPC and lower survival, 12.7% of patients were likely to choose the more expensive option, and for 34.3% of patients, survival, AE risk, and treatment delays all significantly influenced choices. MOA and STOs did not significantly influence treatment choices in any preference class. Approximately 53%, 27%, and 20% of patients preferred IO-IO-like, IO-chemo-like, and chemo-like regimens in 1L, respectively. Respondents were younger, more likely to be Caucasian, and more likely to speak English than the general NSCLC patient population. Conclusions. OOPC, effectiveness, treatment delays, and safety influenced NSCLC patients' 1L treatment decisions, and most patients preferred an IO-IO followed by IO-chemo-like regimen in 1L. Cancer treatment decisions are complex and patient preferences are unique; therefore, patients' treatment objectives should be discussed in shared treatment decision making.

The Value of Progression-Free Survival in Metastatic Breast Cancer: Results From a Survey of Patients and Providers.

Background. Value assessments and treatment decision making typically focus on clinical endpoints, especially overall survival (OS). However, OS data are not always available, and surrogate markers may also have some value to patients. This study sought to estimate preferences for progression-free survival (PFS) relative to OS in metastatic breast cancer (mBC) among a diverse set of stakeholders-patients, oncologists, and oncology nurses-and estimate the value patients and providers place on other attributes of treatment. Methods. Utilizing a combined conjoint analysis and discrete choice experiment approach, we conducted an online prospective survey of mBC patients and oncology care providers who treat mBC patients across the United States. Results. A total of 299 mBC patients, 100 oncologists, and 99 oncology nurses completed the survey. Virtually all patients preferred health state sequences with contiguous periods of PFS, compared with approximately 85% and 75% of nurses and oncologists, respectively. On average, longer OS was significantly (P < 0.01) preferred by the majority (75%) patients, but only 15% of nurses preferred longer OS, and OS did not significantly affect oncologists' preferred health state. However, in the context of a treatment decision, whether a treatment offered continuous periods of stable disease holding OS constant significantly affected nurses' treatment choices. Patients and providers alike valued reductions in adverse event risk and evidence from high-quality randomized controlled clinical trials. Conclusions. The strong preference for observed PFS suggests more research is warranted to better understand the reasons for PFS having positive value to patients. The results also suggest a range of endpoints in clinical trials may have importance to patients.

Using Information on Patient Adherence to Antipsychotic Medication to Understand Their Adherence to Other Medications.

PURPOSE: To assess how patient adherence to atypical antipsychotic medications is associated with adherence to concurrently used medications that treat other serious mental illnesses (SMIs), type-2 diabetes, or hypertension. METHODS: Among patients who had been diagnosed with an SMI (i.e., bipolar disorder, major depressive disorder, or schizophrenia) in the previous year, we used health-insurance claims data to measure adherence based on medication fills. Patients diagnosed with an SMI were required to have 1) a prescription for an atypical oral antipsychotic, and 2) another SMI therapy or oral anti-diabetic or antihypertensive agent in the same year. The patient's concurrent adherence to an antipsychotic and one of 23 other medications was measured by the proportion of days covered (PDC) over a one-year period. Patients were considered adherent when the PDC was ≥ 80%. The strength of the association between their atypical antipsychotic adherence and their concurrent medication adherence was evaluated using the following metrics: accuracy, positive predictive value (PPV), and negative predictive value (NPV). RESULTS: The average (standard deviation) age of patients (N = 129,614) was 44.8 (14.8) years and 62.2% of patients were female. The median accuracy based on atypical antipsychotic adherence to the other 23 medications was 67% (range, 55-71%; statistically different from 50% accuracy in all cases, P < 0.001). Accuracy was higher than physician predictions of adherence from previous studies (53%). The negative predictive value of antipsychotic adherence (75%; range, 62-88%) was generally higher than the PPV (62%; range, 43-67%; all, P < 0.001). CONCLUSION: Information on patient adherence to antipsychotics provides significant insight into adherence to other medications often used by patients with SMI. Because NPV is higher than PPV, adherence to antipsychotics is likely to be a necessary but not sufficient condition for patients with SMI regarding adherence to non-SMI medications.

Medication Adherence Patterns Among Patients with Multiple Serious Mental and Physical Illnesses.

INTRODUCTION: Patients with mental and physical health conditions are complex to treat and often use multiple medications. It is unclear how adherence to one medication predicts adherence to others. A predictive relationship could permit less expensive adherence monitoring if overall adherence could be predicted through tracking a single medication. METHODS: To test this hypothesis, we examined whether patients with multiple mental and physical illnesses have similar adherence trajectories across medications. Specifically, we conducted a retrospective cohort analysis using health insurance claims data for enrollees who were diagnosed with a serious mental illness, initiated an atypical antipsychotic, as well as an SSRI (to treat serious mental illness), biguanides (to treat type 2 diabetes), or an ACE inhibitor (to treat hypertension). Using group-based trajectory modeling, we estimated adherence patterns based on monthly estimates of the proportion of days covered with each medication. We measured the predictive value of the atypical antipsychotic trajectories to adherence predictions based on patient characteristics and assessed their relative strength with the R-squared goodness of fit metric. RESULTS: Within our sample of 431,591 patients, four trajectory groups were observed: non-adherent, gradual discontinuation, stop-start, and adherent. The accuracy of atypical antipsychotic adherence for predicting adherence to ACE inhibitors, biguanides, and SSRIs was 44.5, 44.5, and 49.6%, respectively (all p < 0.001 vs. random). We also found that information on patient adherence patterns to atypical antipsychotics was a better predictor of patient adherence to these three medications than would be the case using patient demographic and clinical characteristics alone. CONCLUSION: Among patients with multiple chronic mental and physical illnesses, patterns of atypical antipsychotic adherence were useful predictors of adherence patterns to a patient's adherence to ACE inhibitors, biguanides, and SSRIs. FUNDING: Otsuka Pharmaceutical Development & Commercialization, Inc.

Economic burden of multiple myeloma among patients in successive lines of therapy in the United States.

This study characterized the costs of multiple myeloma (MM) during first-line (1L), second-line (2L) and third-line (3L) treatment from the US payer perspective. Patients with ≥2 outpatient or ≥1 inpatient claims with a primary MM diagnosis and 12 months continuous enrollment post index were identified in a retrospective claims database between 1 July 2006 and 30 June 2013. A cost per-patient per-month (PPPM) metric was used to calculate total all-cause and anti-MM pharmacy costs in 1L, 2L, and 3L treatment. Of 5704 patients included, 3626 initiated 1L treatment, 1797 initiated 2L and 817 initiated 3L. Average total all-cause PPPM costs were $22,527 in 1L, $35,266 in 2L and $47,417 in 3L. Anti-MM pharmacy costs represented 22%, 29% and 29% of total all-cause costs PPPM in 1L, 2L and 3L, respectively. Study results suggest that delaying 2L and/or 3L treatment initiation may result in lower treatment costs for patients with MM.

What do pharmaceuticals really cost in the long run?

OBJECTIVES: To estimate the long-run average cost (LAC) for a typical drug, accounting for the effects of generic competition and medical cost offsets. STUDY DESIGN: Descriptive analysis of retrospective cross-sectional survey data. METHODS: We estimated the LAC for a drug as the average price per unit paid over the lifecycle of the drug, discounted across all time periods using Medical Expenditure Panel Survey data, and accounted for the effects of generic competition and medical cost offsets attributable to the use of pharmaceuticals. RESULTS: The average market-weighted price fell rapidly after generic entry. As a result, the brand price in the year prior to generic market entry was 39% (95% confidence interval [CI], 37%-43%) higher than the LAC per 30-day supply or package. When accounting for medical cost offsets, the brand price in the year prior to generic market entry was 75% (95% CI, 69%-79%) greater than the LAC per 30-day supply or package. The brand price at launch was 11% more than the LAC, and 40% more than the LAC net after adjusting for medical cost offsets. CONCLUSIONS: Branded drug prices might overstate the true long-run cost of pharmaceuticals by 40% to 75%, accounting for generic price reductions and medical cost offsets. To ensure that all drugs providing long-run value end up entering the marketplace, market access and other policy decisions should consider the full range of long-term costs-and not just prices-at a particular point in time.

The relationship between adherence and total spending among Medicare beneficiaries with type 2 diabetes.

OBJECTIVES: This study examined the relationship between medication adherence, cost sharing measured as out-of-pocket spending, and total annual spending in Medicare beneficiaries with type 2 diabetes (T2D) to evaluate whether pharmacy cost-sharing programs have the potential to decrease adherence. These programs may unintentionally increase the risk of medical complications and may result in higher spending overall. STUDY DESIGN: This retrospective study used 2006 to 2009 Medicare claims data. The sample included patients 65 years or older with T2D (at least 1 claim with International Classification of Diseases, 9th Revision, Clinical Modification codes 250.x0 and 250.x2 and at least 1 antidiabetes drug claim). METHODS: Medication adherence was measured as proportion of days covered over the first 12 months of observation. Spending and adherence outcomes were defined in deciles. RESULTS: The sample included 12,305 patient-year observations. Pharmacy spending for patients in the most adherent (10th) decile was 59% higher than that for patients in the least adherent (1st) decile ($4839 vs $3046). Yet, patients in the 10th decile had 49% lower total ($12,531 vs $24,468) and 64% lower medical spending ($7692 vs $21,421) than patients in the 1st decile. Greater out-of-pocket spending was correlated with lower adherence and higher total and medical spending. CONCLUSIONS: This study describes a widespread variation in medication adherence, pharmacy cost sharing, and medical spending in a sample of Medicare beneficiaries with T2D. We found that lower adherence was correlated with higher cost sharing in the Medicare population, perhaps because of unobserved confounding factors. However, the existing literature on patients with employer-sponsored insurance suggests some of this correlation may be indicative of causal relationships.

Cost-effectiveness of sequenced treatment of rheumatoid arthritis with targeted immune modulators.

AIMS: To determine the cost-effectiveness of treatment sequences of biologic disease-modifying anti-rheumatic drugs or Janus kinase/STAT pathway inhibitors (collectively referred to as bDMARDs) vs conventional DMARDs (cDMARDs) from the US societal perspective for treatment of patients with moderately to severely active rheumatoid arthritis (RA) with inadequate responses to cDMARDs. MATERIALS AND METHODS: An individual patient simulation model was developed that assesses the impact of treatments on disease based on clinical trial data and real-world evidence. Treatment strategies included sequences starting with etanercept, adalimumab, certolizumab, or abatacept. Each of these treatment strategies was compared with cDMARDs. Incremental cost, incremental quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) were calculated for each treatment sequence relative to cDMARDs. The cost-effectiveness of each strategy was determined using a US willingness-to-pay (WTP) threshold of $150,000/QALY. RESULTS: For the base-case scenario, bDMARD treatment sequences were associated with greater treatment benefit (i.e. more QALYs), lower lost productivity costs, and greater treatment-related costs than cDMARDs. The expected ICERs for bDMARD sequences ranged from ∼$126,000 to $140,000 per QALY gained, which is below the US-specific WTP. Alternative scenarios examining the effects of homogeneous patients, dose increases, increased costs of hospitalization for severely physically impaired patients, and a lower baseline Health Assessment Questionnaire (HAQ) Disability Index score resulted in similar ICERs. CONCLUSIONS: bDMARD treatment sequences are cost-effective from a US societal perspective.

The value of survival gains in myelodysplastic syndromes.

OBJECTIVES: To measure the value of survival gains attributable to the introduction of 3 novel therapies for myelodysplastic syndromes (MDS). STUDY DESIGN: Retrospective study of patients diagnosed with MDS in the Surveillance, Epidemiology and End Results Program (SEER) registry, clinical trial evidence for MDS therapies, and claims data. METHODS: We used multivariate Cox proportional hazards models to estimate the increase in survival associated with the introduction of the 3 new therapies for patients diagnosed with MDS from 2001 to 2011 in the SEER cancer registry. Increases in survival associated with the 3 novel therapies were estimated using retrospective survival analyses and published clinical trial evidence. MDS treatment costs were estimated using Ingenix claims data and used to calculate the share of the value of survival gains retained by patients. RESULTS: We estimated that the introduction of these 3 therapies is associated with a hazard ratio of 0.901 (P <.10), and a 73% increase in median survival from 33 to 57 months. We estimated that for current and future MDS patients, these 3 therapies will generate over $193 billion in cumulative value through extensions in patient survival. CONCLUSIONS: This study demonstrates that the value of recently approved innovative therapies in MDS is large and that the value of survival gains in MDS far outweighs their costs.