Noninvasive ventilation at home reduces mortality in COPD with CRF.

OBJECTIVES: Patients with chronic respiratory failure resulting from chronic obstructive pulmonary disease (COPD-CRF) have limited treatment options and poor health outcomes. We examined the effect of noninvasive ventilation at home (NIVH) treatment on all-cause mortality, hospitalizations, and emergency department (ED) visits. STUDY DESIGN: Retrospective cohort study. METHODS: Using Medicare claims data between 2012 and 2017, we divided patients with COPD-CRF into a treatment group, defined by NIVH receipt within 2 months of CRF diagnosis, and a control group without NIVH receipt in the entire follow-up period. We modeled time to death, first hospitalization, and first ED visit. Cox regressions were performed, mitigating selection bias using stabilized inverse probability of treatment weights with regression controls. Sensitivity analyses with time-varying exposure to NIVH were conducted on the full sample irrespective of treatment timing. RESULTS: We identified 410 patients treated with NIVH and 36,247 controls. We observed a reduced risk of hospitalizations (HR, 0.790; 95% CI, 0.592-0.988), ED visits (HR, 0.571; 95% CI, 0.457-0.686), and mortality (HR, 0.617; 95% CI, 0.462-0.772). The benefit of NIVH diminished over time for mortality and ED visits but remained constant for hospitalizations. However, no survival benefit was observed in the sensitivity analyses that accounted for immortal-time bias; further exploration suggests that earlier NIVH treatment following CRF diagnosis may be an important factor in improving survival outcomes. CONCLUSIONS: Patients with COPD-CRF who received NIVH had statistically significant reductions in hospitalizations and ED visits compared with patients not treated with NIVH. Further research is needed to examine the effect of NIVH on mortality.

Non-invasive ventilation at home improves survival and decreases healthcare utilization in medicare beneficiaries with Chronic Obstructive Pulmonary Disease with chronic respiratory failure.

BACKGROUND: Patients with Chronic Obstructive Pulmonary Disease with chronic respiratory failure (COPD-CRF) experience high mortality and healthcare utilization. Non-invasive home ventilation (NIVH) is increasingly used in such patients. We examined the associations between NIVH and survival, hospitalizations, and emergency room (ER) use in COPD-CRF Medicare beneficiaries. MATERIALS AND METHODS: Retrospective cohort study using the Medicare Limited Data Set (2012-2018). Patients receiving NIVH within two months of CRF diagnosis (treatment group) were matched on demographic and clinical characteristics to patients never receiving NIVH (control group). CRF diagnosis was identified using ICD-9-CM/ICD-10-CM codes. Time to death, first hospitalization, and first ER visit were estimated using Cox regressions. RESULTS: After matching, 517 patients receiving NIVH and 511 controls (mean age: 70.6 years, 44% male) were compared. NIVH significantly reduced risk of death (aHR: 0.50; 95%CI: 0.36-0.65), hospitalization (aHR: 0.72; 95%CI: 0.52-0.93), and ER visit (aHR: 0.48; 95%CI: 0.38-0.58) at diagnosis. The NIVH risk reduction became smaller over time for mortality and ER visits, but continued to accrue for hospitalizations. One-year post-diagnosis, 28% of treated patients died versus 46% controls. For hospitalizations and ER visits, 55% and 72% treated patients experienced an event, respectively, versus 67% and 92% controls. The relative risk reduction was 39% for mortality, 17% for hospitalizations, and 22% for ER visits. Number needed to treat were 5.5, 9, and 5 to prevent a death, hospitalization, or ER visit one-year post-diagnosis, respectively. CONCLUSION: NIVH treatment is associated with reduced risk of death, hospitalizations, and ER visits among COPD-CRF Medicare beneficiaries.

Economic value of transcatheter valve replacement for inoperable aortic stenosis.

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

Modeling the impact of patient treatment preference on health outcomes in relapsing-remitting multiple sclerosis.

Aims: Model how moving from current disease-modifying drug (DMD) prescribing patterns for relapsing-remitting multiple sclerosis (RRMS) observed in the United Kingdom (UK) to prescribing patterns based on patient preferences would impact health outcomes over time.Materials and methods: A cohort-based Markov model was used to measure the effect of DMDs on long-term health outcomes for individuals with RRMS. Data from a discrete choice experiment were used to estimate the market shares of DMDs based on patient preferences (i.e. preference shares). These preference shares and real-world UK market shares were used to calculate the effect of prescribing behavior on relapses, disability progression, and quality-adjusted life-years (QALYs). The incremental benefit of patient-centered prescribing over current practices for the UK RRMS population was then estimated; scenario and sensitivity analyses were also conducted.Results: Compared to current prescribing practices, when UK patients with RRMS were treated following patient preferences, health outcomes were improved. This population was expected to experience 501,690 relapses and gain 1,003,263 discounted QALYs over 50 years under patient-centered prescribing practices compared to 538,417 relapses and 958,792 discounted QALYs under current practices (-6.8% and +4.6%, respectively). Additionally, less disability progression was observed when prescribed treatment was based on patient preferences. In a scenario analysis where only oral treatments were considered, the results were similar, although the magnitude of benefit was smaller. Number of relapses was most sensitive to how the annualized relapse rate was modeled; disability progression was most sensitive to mortality rate assumptions.Limitations: Treatment efficacy estimates applied to various models in this study were based on data derived from clinical trials, rather than real-world data; the impact of patient-centered prescribing on treatment adherence and/or switching was not modeled.Conclusions: The population of UK RRMS patients may experience overall health gains if patient preferences are better incorporated into prescribing practices.

The Social Value of Improvement in Activities of Daily Living among the Advanced Parkinson's Disease Population.

OBJECTIVES: Quantify the value of functional status (FS) improvements consistent in magnitude with improvements due to levodopa-carbidopa intestinal gel (LCIG) treatment, among the advanced Parkinson's disease (APD) population. METHODS: The Health Economic Medical Innovation Simulation (THEMIS), a microsimulation that estimates future health conditions and medical spending, was used to quantify the health and cost burden of disability among the APD population, and the value of quality-adjusted life-years gained from FS improvement due to LCIG treatment compared to standard of care (SoC). A US-representative Parkinson's disease (PD)-comparable cohort was constructed in THEMIS based on observed PD patient characteristics in a nationally representative dataset. APD was defined from the literature and clinical expert input. The PD and APD cohorts were followed from 2010 over their remaining lifetimes. All individuals were ages 65 and over at the start of the simulation. To estimate the value of FS improvement due to LCIG treatment, decreases in activities of daily living (ADL) limitations caused by LCIG treatment were calculated using data from a randomized, controlled, double-blind, double-dummy clinical trial and applied to the APD population in THEMIS. RESULTS: Total burden of disability associated with APD was $17.7 billion (B). From clinical trial data, LCIG treatment versus SoC lowers the odds of difficulties in walking, dressing, and bathing by 76%, 42% and 39%, respectively. Among the APD population, these reductions generated $2.6B in value to patients and cost savings to payers. The added value was 15% of the burden of disability associated with APD and offsets 15% of the cost of LCIG treatment. CONCLUSIONS: FS improvements, consistent with improvements due to LCIG treatment, in the APD population created health benefits and reduced healthcare costs in the US.

The Societal Value of Broader Access to Antiobesity Medications.

OBJECTIVE: Obesity and its complications place an enormous burden on society. Yet antiobesity medications (AOM) are prescribed to only 2% of the eligible population, even though few individuals can sustain weight loss using other strategies alone. This study estimated the societal value of greater access to AOM. METHODS: By using a well-established simulation model (The Health Economics Medical Innovation Simulation), the societal value of AOM for the cohort of Americans aged ≥ 25 years in 2019 was quantified. Four scenarios with differential uptake among the eligible population (15% and 30%) were modeled, with efficacy from current and next-generation AOM. Societal value was measured as monetized quality of life, productivity gains, and savings in medical spending, subtracting the costs of AOM. RESULTS: For the 217 million Americans aged ≥ 25 years, AOM generated $1.2 trillion in lifetime societal value under a conservative scenario (15% annual uptake using currently available AOM). The introduction of next-generation AOM increased societal value to $1.9 to $2.5 trillion, depending on uptake. Finally, societal value was higher for younger individuals and Black and Hispanic individuals compared with White individuals. CONCLUSIONS: This study suggests that AOM provide substantial gains to patients and society. Policies promoting broader clinical access to and use of AOM warrant consideration to reach national goals to reduce obesity.

The effect of functional status impairment on nursing home admission risk among patients with advanced Parkinson's disease.

Aims: To estimate the relationship between functional status (FS) impairment and nursing home admission (NHA) risk in Parkinson's disease (PD) patients, and quantify the effect of advanced PD (APD) treatment on NHA risk relative to standard of care (SoC).Materials and methods: PD patients were identified in the Medicare Current Beneficiary Survey (MCBS) (1992-2010). A working definition based on the literature and clinical expert input determined APD status. A logit model estimated the relationship between FS impairment and NHA risk. The effect of levodopa-carbidopa intestinal gel (LCIG) on NHA risk relative to SoC was simulated using clinical trial data (control: optimized oral levodopa-carbidopa IR, ClinicalTrials.gov NCT00660387 and NCT0357994).Results: Non-advanced PD and APD significantly increased NHA risk when controlling for demographics (p < 0.01). APD status was no longer significant after controlling for FS limitations, implying that FS limitations explain the increased NHA risk in APD patients. Reduced impairment in FS in patients with APD treated with LCIG reduced risk of NHA by 13.5% relative to SoC.Limitations: This study applies clinical trial results to real-world data. LCIG treatment might have a different effect on NHA risk for the nationally representative population than the effect measured in the trial. Both data sources employ different instruments to measure FS, instrument wording and study follow-up differed, which might bias our estimates. Finally, there lacks consensus on a definition of APD. The prevalence of APD in this study is high, perhaps due to the specific definition used.Conclusions: Patients with APD experience a higher risk in NHA than those with non-advanced disease. This increased risk in NHA in patients with APD is explained by greater limitations in FS. The relative reduction in risk of NHA for the APD population treated with LCIG is quantitatively similar to doubling Medicaid home care services.

The long-term social value of granulocyte colony-stimulating factors.

OBJECTIVES: Febrile neutropenia (FN) is a life-threatening complication of chemotherapy that can lead to hospitalizations, chemotherapy dose reductions or delays, and mortality. Granulocyte colony-stimulating factor (G-CSF) prophylaxis reduces the incidence of FN, enabling patients to undergo and remain on myelosuppressive chemotherapy. We estimate the benefits of continuing current G-CSF use patterns and an alternative that aligns prophylactic G-CSF use with guideline recommendations. STUDY DESIGN: Using The Health Economics Medical Innovation Simulation microsimulation, we estimated lifetime social value (SV) of prophylactic G-CSF for a nationally representative US population with breast, lung, and gynecological cancers and non-Hodgkin lymphoma. METHODS: SV estimates included the cost of G-CSF, FN, chemotherapy relative dose intensity (RDI) less than 85% (RDI<85%), medical spending, and deaths for 3 scenarios: current use (current G-CSF use), targeted use (100% G-CSF use among patients with high FN risk), and reduced use (current G-CSF use reduced by 20% across all FN risk categories). RESULTS: Over 10 years, current use, compared with no G-CSF use, would decrease cases of FN by 3.3 million, prevent 354,000 cases of RDI<85%, and generate $96 billion in SV. Compared with current use, targeted use would decrease cases of FN by an additional 3.3 million, prevent 355,000 more cases of RDI<85%, and generate another $119 billion in SV. Reduced use would increase FN and RDI<85%, lowering SV by $18 billion compared with current use. CONCLUSIONS: Current use of G-CSF prophylaxis would provide $96 billion in SV over the next 10 years. Targeting G-CSF prophylaxis to align with guidelines would more than double SV, highlighting the substantial value of appropriate FN risk assessment and targeted G-CSF prophylaxis.

Lifetime benefits of early detection and treatment of diabetic kidney disease.

OBJECTIVES: Diabetic kidney disease (DKD) is a frequent complication of diabetes with potentially devastating consequences that may be prevented or delayed. This study aimed to estimate the health and economic benefit of earlier diagnosis and treatment of DKD. METHODS: Life expectancy and medical spending for people with diabetes were modeled using The Health Economics Medical Innovation Simulation (THEMIS). THEMIS uses data from the Health and Retirement Study to model cohorts of individuals over age 50 to project population-level lifetime health and economic outcomes. DKD status was imputed based on diagnoses and laboratory values in the National Health and Nutrition Examination Survey. We simulated the implementation of a new biomarker identifying people with diabetes at an elevated risk of DKD and DKD patients at risk of rapid progression. RESULTS: Compared to baseline, the prevalence of DKD declined 5.1% with a novel prognostic biomarker test, while the prevalence of diabetes with stage 5 chronic kidney disease declined 3.0%. Consequently, people with diabetes gained 0.2 years in life expectancy, while per-capita annual medical spending fell by 0.3%. The estimated cost was $12,796 per life-year gained and $25,842 per quality-adjusted life-year. CONCLUSIONS: A biomarker test that allows earlier treatment reduces DKD prevalence and slows DKD progression, thereby increasing life expectancy among people with diabetes while raising healthcare spending by less than one percent.

The well-being of long-term cancer survivors.

OBJECTIVES: To compare the well-being of long-term cancer survivors with that of US residents of similar age and demographic characteristics, patients recently diagnosed with cancer, and individuals with chronic illness. STUDY DESIGN: Retrospective observational study. METHODS: Using the Health and Retirement Study, a survey of US residents older than 50 years, we defined 4 cohorts: long-term cancer survivors (>4 years post diagnosis), individuals recently diagnosed with cancer (≤4 years post diagnosis), individuals with chronic illness, and US residents older than 50 years ("nationally representative cohort"). Well-being measures included self-reported health, utility, happiness, medical utilization and spending, employment, and earnings, and these measures were compared across cohorts, adjusting for survey year, demographic characteristics, smoking, and number of comorbidities. We imputed medical spending using the Medical Expenditure Panel Survey and the Medicare Current Beneficiary Survey. RESULTS: Long-term cancer survivors fared significantly better than those recently diagnosed with cancer, those with chronic illness, and individuals in the nationally representative cohort in the majority of well-being measures (P <.05), including fewer doctor visits, hospitalizations, and hospital nights; better utility and self-reported health; and greater likelihood of employment. Long-term cancer survivors had lower healthcare spending than those recently diagnosed with cancer (P <.01) and significantly greater happiness than the nationally representative cohort and those with chronic illness (P <.05). CONCLUSIONS: Although patients with cancer experience diminished well-being in the short term across a variety of measures, in the long term, cancer survivors do as well as or better than US residents of similar age and demographic characteristics. This finding is striking given that one might expect long-term cancer survivors to do worse than similar individuals without a history of cancer.

The impact of self- and physician-administered cancer treatment on work productivity and healthcare utilization.

BACKGROUND: Lost productivity in the workplace represents a significant portion of the economic burden of cancer in the United States. Cancer treatments have historically been physician-administered, while recent innovations have led to the development of self-administered, usually oral, agents. Self-administered treatments have the potential to reduce healthcare utilization and time away from work, but the magnitude of these effects is unknown. OBJECTIVE: To compare the effects of self- and physician-administered cancer treatment on work productivity and health care utilization. METHODS: Cancer subtypes with self- and physician-administered treatment options were selected. Patients with female breast, or lung or bronchus cancer diagnosed in 2004-2013 were identified in the Truven Health Analytics Commercial Claims and Encounters and Health and Productivity Management databases. Using multivariate regression models, work productivity and healthcare utilization were compared for patients receiving self- versus physician-administered treatment in the 12 months after initial diagnosis. Work productivity outcomes included the number of sick days and short-term disability claims. RESULTS: One month of self- versus physician-administered treatment significantly reduced cancer-related outpatient services, doctor visits, and infusions in the 12 months after initial diagnosis for both cancers of interest. In addition, breast and lung or bronchus cancer patients who received self-administered treatment were less likely to have short-term disability claims, and breast cancer patients with non-metastatic disease who received self-administered treatment had significantly fewer sick days. CONCLUSIONS: Self-administered cancer treatment was associated with fewer cancer-related outpatient services and reduced time away from work compared to physician-administered cancer treatment.

The Long-Term Impact of Price Controls in Medicare Part D.

Price controls for prescription drugs are once again at the forefront of policy discussions in the United States. Much of the focus has been on the potential short-term savings - in terms of lower spending - although evidence suggests price controls can dampen innovation and adversely affect long-term population health. This paper applies the Health Economics Medical Innovation Simulation, a microsimulation of older Americans, to estimate the long-term impacts of government price setting in Medicare Part D, using pricing in the Federal Veterans Health Administration program as a proxy. We find that VA-style pricing policies would save between $0.1 trillion and $0.3 trillion (US$2015) in lifetime drug spending for people born in 1949-2005. However, such savings come with social costs. After accounting for innovation spillovers, we find that price setting in Part D reduces the number of new drug introductions by as much as 25% relative to the status quo. As a result, life expectancy for the cohort born in 1991-1995 is reduced by almost 2 years relative to the status quo. Overall, we find that price controls would reduce lifetime welfare by $5.7 to $13.3 trillion (US$2015) for the US population born in 1949-2005.

Pharmaceutical Innovation in the Treatment of Schizophrenia and Mental Disorders Compared with Other Diseases.

OBJECTIVES: The objectives of this study were to assess the level of private and public investment in research and development of treatments for schizophrenia and other mental disorders compared to other diseases in order to present data on the economic burden and pharmaceutical innovation by disease area, and to compare the level of investment relative to burden across different diseases. DESIGN: The levels of investment and pharmaceutical innovation relative to burden across different diseases were assessed. Disease burden and prevalence for mental disorders (schizophrenia, bipolar disorder, and major depressive disorder); cancer; rheumatoid arthritis; chronic obstructive pulmonary disorder; diabetes; cardiovascular disease; and neurological disorders (dementia and epilepsy) were estimated from literature sources. SETTING: Pharmaceutical treatment innovation was measured by the total number of drug launches and the number of drugs launched categorized by innovativeness. Research and development expenditures were estimated using published information on annual public and domestic private research and development expenditures by disease area. Lastly, investment relative to disease burden was measured among the set of disease classes for which all three measures were available: schizophrenia, bipolar disorder, major depressive disorder, cancer, rheumatoid arthritis, chronic obstructive pulmonary disease, diabetes, cardiovascular disease, and neurology (dementia and epilepsy combined). RESULTS: The level of investment and pharmaceutical innovation in mental disorders was comparatively low, especially relative to the burden of disease. For mental disorders, investment was $3.1 per $1,000 burden invested in research and development for schizophrenia, $1.8 for major depressive disorder, and $0.4 for bipolar disorder relative to cancer ($75.5), chronic obstructive pulmonary disease ($9.4), diabetes ($7.6), cardiovascular disease ($6.3), or rheumatoid arthritis ($5.3). Pharmaceutical innovation was also low for mental disorders. CONCLUSION: Despite the significant burden mental disorders impose on society, investment and pharmaceutical innovation in this disease area remains comparatively low. Policymakers should consider new strategies to stimulate public and private investment in the research and development of novel and effective therapies to treat schizophrenia and other mental disorders.