Cost-effectiveness of sotagliflozin for the treatment of patients with diabetes and recent worsening heart failure.

Aim: To assesses the cost-effectiveness of sotagliflozin for the treatment of patients hospitalized with heart failure and comorbid diabetes. Materials & methods: A de novo cost-effectiveness model with a Markov structure was created for patients hospitalized for heart failure with comorbid diabetes. Outcomes of interest included hospital readmissions, emergency department visits and all-cause mortality measured over a 30-year time horizon. Baseline event frequencies were derived from published real-world data studies; sotagliflozin's efficacy was estimated from SOLOIST-WHF. Health benefits were calculated quality-adjusted life years (QALYs). Costs included pharmaceutical costs, rehospitalization, emergency room visits and adverse events. Economic value was measured using the incremental cost-effectiveness ratio (ICER). Results: Sotagliflozin use decreased annualized rehospitalization rates by 34.5% (0.228 vs 0.348, difference: -0.120), annualized emergency department visits by 40.0% (0.091 vs 0.153, difference: -0.061) and annualized mortality by 18.0% (0.298 vs 0.363, difference: -0.065) relative to standard of care, resulting in a net gain in QAYs of 0.425 for sotagliflozin versus standard of care. Incremental costs using sotagliflozin increased by $19,374 over a 30-year time horizon of the patient, driven largely by increased pharmaceutical cost. Estimated ICER for sotagliflozin relative to standard of care was $45,596 per QALY. Conclusion: Sotagliflozin is a cost-effective addition to standard of care for patients hospitalized with heart failure and comorbid diabetes.

Quantifying the Value of Reduced Health Disparities: Low-Dose Computed Tomography Lung Cancer Screening of High-Risk Individuals Within the United States.

OBJECTIVE: This study aimed to measure the value of increasing lung cancer screening rates for high-risk individuals and its impact on health disparities. METHODS: The model estimated changes in health economic outcomes if low-dose computed tomography screening increased from current to 100% compliance, following clinical guidelines. Current low-dose computed tomography screening rates were estimated by income, education, and race, using 2017-2019 Behavioral Risk Factor Surveillance System data. The model contained a decision tree module to segment the population by screening outcomes and a Markov chain module to estimate cancer progression over time. Model parameters included information on survival, quality of life, and costs related to cancer diagnosis, treatment, and adverse events. Distributional cost-effectiveness analysis estimated the net monetary value from reduced health disparities-measured using quality-adjusted life expectancy-across income, education, and race groups. Outcomes were assessed over 30 years. RESULTS: Lung cancer screening eligibility using US Preventive Services Task Force guidelines was higher for individuals with income <$15 000 (47.2%) and without a high-school education (46.1%) than individuals with income >$50 000 (16.6%) and with a college degree (13.5%), respectively. Increasing lung cancer screening to 100% compliance was cost-effective ($64 654 per quality-adjusted life-year) and produced economic value by up to $560 per person ($182.1 billion for United States overall). Up to 32.2% of the value was due to reductions in health disparities. CONCLUSIONS: Significant value in increasing lung cancer screening rates derived from reducing health disparities. Policy makers and clinicians may not be appropriately prioritizing cancer screening if value from reducing health disparities is unconsidered.

Use of Conditionally Essential Amino Acids and the Economic Burden of Postoperative Complications After Fracture Fixation: Results from a Cost Utility Analysis.

Objective: To measure the economic impact of conditionally essential amino acids (CEAA) among patients with operative treatment for fractures. Methods: A decision tree model was created to estimate changes in annual health care costs and quality of life impact due to complications after patients underwent operative treatment to address a traumatic fracture. The intervention of interest was the use of CEAA alongside standard of care as compared to standard of care alone. Patients were required to be aged ≥18 and receive the surgery in a US Level 1 trauma center. The primary outcomes were rates of post-surgical complications, changes in patient quality adjusted life years (QALYs), and changes in cost. Cost savings were modeled as the incremental costs (in 2022 USD) of treating complications due to changes in complication rates. Results: The per-patient cost of complications under CEAA use was $12,215 compared to $17,118 under standard of care without CEAA. The net incremental cost savings per patient with CEAA use was $4902, accounting for a two-week supply cost of CEAA. The differences in quality-adjusted life years (QALYs) under CEAA use and no CEAA use was 0.013 per person (0.739 vs 0.726). Modeled to the US population of patients requiring fracture fixations in trauma centers, the total value of CEAA use compared to no CEAA use was $316 million with an increase of 813 QALYs per year. With a gain of 0.013 QALYs per person, valued at $150,000, and the incremental cost savings of $4902 resulted in net monetary benefit of $6852 per patient. The incremental cost-effectiveness ratio showed that the use of CEAA dominated standard of care. Conclusion: CEAA use after fracture fixation surgery is cost saving. Level of Evidence: Level 1 Economic Study.

A Decade of Health Innovation: The Impact of New Medicines on Patient Health and the Implications for NICE's Size of Benefit Multiplier.

OBJECTIVES: This study aimed to estimate the incremental health benefits of pharmaceutical innovations approved between 2011 and 2021 and the share that would surpass the National Institute for Health and Care Excellence (NICE) "size of benefit" decision weight thresholds. METHODS: We identified all US-approved drugs between 2011 and 2021. Health benefits, in terms of quality-adjusted life-years (QALYs) for each treatment, were extracted from published cost-effectiveness analyses. Summary statistics by therapeutic area and cell/gene therapy status identified the treatments with the largest QALY gains. RESULTS: The Food and Drug Administration approved 483 new therapies between 2011 and 2021 and of these 252 had a published cost-effectiveness analysis meeting our inclusion criteria. The average incremental health benefits produced by these treatments were 1.04 QALYs (SD = 2.00) relative to standard of care, with wide variation by therapeutic area. Pulmonary and ophthalmologic therapies produced the highest health benefits with 1.47 (SD = 2.17, n = 13) and 1.41 QALYs gained (SD = 3.53, n = 7), respectively; anesthesiology and urology had the lowest gains (< 0.1 QALYs). Cell and gene therapies produced an average health benefit that was 4 times greater than noncell and gene therapies (4.13 vs 0.96). Among the top treatments in terms of incremental QALYs gained, half (10 of 20) were oncology therapies. Three of 252 treatments (1.2%) met NICE's threshold for a "size of benefit" multiplier. CONCLUSIONS: Treatments for rare disease, oncology, and cell and gene therapies produced some of the highest level of health innovation relative to previous standard of care, but few therapies would have qualified for NICE's "size of benefit" multiplier as currently constructed.

Impact of suboptimal or inappropriate treatment on healthcare resource use and cost among patients with uncomplicated urinary tract infection: an analysis of integrated delivery network electronic health records.

BACKGROUND: Although uncomplicated urinary tract infections (uUTIs; occurring in female patients without urological abnormalities or history of urological procedures or complicating comorbidities) are one of the most common community infections in the United States (US), limited data are available concerning associations between antibiotic resistance, suboptimal prescribing, and the economic burden of uUTI. We examined the prevalence of suboptimal antibiotic prescribing and antibiotic resistance and its effects on healthcare resource use and costs. METHODS: This retrospective cohort study utilized electronic health record data from a large Mid-Atlantic US integrated delivery network database, collected July 2016-March 2020. Female patients aged ≥ 12 years with a uUTI, who received ≥ 1 oral antibiotic treatment within ± 5 days of index uUTI diagnosis, and had ≥ 1 urine culture with antimicrobial susceptibility test, were eligible for inclusion in the study. The study examined the proportion of antibiotics that were inappropriately or suboptimally prescribed among patients with confirmed uUTI, and total healthcare costs (all-cause and UTI-related) within 6 months after a uUTI, stratified by antibiotic susceptibility and/or inappropriate or suboptimal treatment. Patient outcomes were assessed after 1:1 propensity score matching of patients with antibiotic-susceptible versus not-susceptible isolates and then by other covariates (e.g., demographics and recent healthcare use). A similar propensity score calculation was used to analyze the effect of inappropriate/suboptimal treatment on health outcomes. Costs were adjusted to 2020 US dollars ($). RESULTS: Among 2565 patients with a uUTI included in the analysis, the most commonly prescribed antibiotics were nitrofurantoin (61%), trimethoprim-sulfamethoxazole (19%), and ciprofloxacin (15%). More than one-third of the sample (40.2%) had isolates that were not-susceptible to ≥ 1 antibiotic indicated for treating patients with uUTI. Two-thirds (66.6%) of study-eligible patients were prescribed appropriate treatment; 29.9% and 11.9% were prescribed suboptimal and/or inappropriate treatment, respectively. Inappropriate or suboptimally prescribed patients had greater all-cause and UTI-related costs compared with appropriately prescribed patients. Differences were most striking among patients with antibiotic not-susceptible isolates. CONCLUSIONS: These findings highlight how the increasing prevalence of antibiotic resistance combined with suboptimal treatment of patients with uUTI increases the burden on healthcare systems. The finding underlines the need for improved prescribing accuracy by better understanding regional resistance rates and developing improved diagnostic tests.

Estimating the impact of biosimilar entry on prices and expenditures in rheumatoid arthritis: a case study of targeted immune modulators.

OBJECTIVE: To model changes in prices, utilization, and expenditures of targeted immune modulators (TIMs) for rheumatoid arthritis, accounting for biosimilar entry. METHODS: Using IQVIA National Sales Perspective data between 2013 and 2019, we examined sales and expenditures of biologics and non-biological complex molecules, 20 quarters before and after patent exclusivity milestones. We estimated the impact of a molecule's exclusivity milestones and biosimilar entry on prices, using a regression discontinuity design (RDD). We then combined the RDD estimate with historical trends to assess the impact of adalimumab's exclusivity milestones on future TIM expenditures. RESULTS: Changes in average molecule prices were associated largely with biosimilar uptake. For molecules with relatively high biosimilar uptake (>60%), prices fell considerably (-21.2% to -59.3%) one year after exclusivity milestones, whereas molecules with lower biosimilar uptake (<10%) experienced smaller price decreases (-2.4% to -8.4%). Average price reduction at the molecule level after biosimilar entry was not significant (-18.6%; p = .657). When applying the RDD results after adalimumab's exclusivity milestones, its projected share of total TIM market expenditures decreased from 48.0% in 2019 to 26.0% in 2025, whereas expenditures on Janus kinase inhibitors increased from 4.0% to 34.0%. CONCLUSIONS: Biologics facing biosimilar competition may experience price decreases, potentially offering substantial savings to payers, patients, and society, although the magnitude of these estimates depends on biosimilar uptake. Formulary placement, along with manufacturer-payer dynamics, may also play a role in determining the impact on price and market uptake of biosimilars.

Health care utilization and costs associated with functional status in patients with psoriatic arthritis.

BACKGROUND: The Health Assessment Questionnaire Disability Index (HAQ-DI) has been validated and widely used in psoriatic arthritis (PsA) clinical trials for the assessment of patient functional status. Significant improvements in the HAQ-DI have been reported in response to therapeutic interventions; however, few US studies have evaluated the economic impact of functional disability in patients with PsA. OBJECTIVE: To evaluate the association of functional status with health care resource utilization (HCRU) and total health care costs in US patients diagnosed with PsA. METHODS: This retrospective study included adult patients with PsA enrolled in FORWARD between July 2009 and June 2019 who completed 1 or more HAQ-DI questionnaires between January 2010 and December 2019. Patient demographics, clinical characteristics, and patient-reported outcomes were collected from the most recent questionnaire. HCRU and total health care costs (2019 US dollars) for all hospitalizations, emergency department (ED) visits, outpatient visits, diagnostic tests, and procedures were assessed for the 6 months prior to survey completion. Negative binomial regression models (HCRU outcomes) and generalized linear models with γ distribution and log-link function (cost outcomes) were used to assess the relationship between HAQ-DI and HCRU and cost outcomes, respectively. RESULTS: A total of 828 patients with PsA who completed HAQ-DI questionnaires were included. The mean (SD) age was 58.5 (13.5) years, 72.3% were female, and 92.3% were White. The mean (SD) disease duration was 17.5 (12.4) years, and the mean (SD) HAQ-DI score at the time of the patients' most recent questionnaire was 0.9 (0.7). More severe functional disability, measured by higher HAQ-DI score, was significantly associated with increased risk (incident rate ratio [95% CI]) of hospitalizations (1.68 [1.11-2.55]), ED visits (2.09 [1.47-2.96]), outpatient visits (1.14 [1.05-1.24]), and diagnostic tests (1.42 [1.16-1.74]). There was also a significant positive association between greater HAQ-DI score and increased total annualized health care costs (incremental amount [95% CI], 1.13 [1.03-1.23]) and medical costs (1.38 [1.13-1.69]), but there was no significant association found with pharmacy costs. Total adjusted average patient medical costs increased with increasing HAQ-DI score. CONCLUSIONS: Among patients with PsA enrolled in FORWARD, more functional disability-as measured by higher HAQ-DI scores-was associated with greater HCRU and increased total health care costs. These results suggest that improving functional status in patients with PsA may reduce economic burden for health care payers and systems. DISCLOSURES: Dr Ogdie has received consulting fees from Amgen, AbbVie, Bristol Myers Squibb, Celgene, CorEvitas (formerly Corrona), Gilead, Janssen, Lilly, Novartis, Pfizer, and UCB and has received grant support from the National Institutes of Health/National Institute of Arthritis and Musculoskeletal and Skin Diseases, Rheumatology Research Foundation, National Psoriasis Foundation, Pfizer (University of Pennsylvania), Amgen (FORWARD), and Novartis (FORWARD). Dr Hwang has received consulting fees from Novartis and UCB and has received grant support (5KL2TR003168-03) from the University of Texas Health Science Center at Houston Center of Clinical and Translational Sciences KL2 program. Drs Veeranki and Shafrin were employees of PRECISIONheor at the time of this analysis. Ms Portelli and Mr Sison are employees of PRECISIONheor. Ms Pedro has nothing to disclose. Dr Hass is an employee of H. E. Outcomes, providing consulting services to Novartis. Dr Hur was an employee of Novartis at the time of this analysis. Dr Kim was a postdoctoral fellow at the University of Texas at Austin and Baylor Scott and White Health, providing services to Novartis at the time of this analysis. Dr Yi is an employee of Novartis. Dr Michaud received grant funding from the Rheumatology Research Foundation at the time of this analysis. This study was funded by Novartis Pharmaceuticals Corporation, East Hanover, NJ.

Association of health care utilization and costs with patient-reported outcomes in patients with ankylosing spondylitis.

BACKGROUND: Interventions for ankylosing spondylitis (AS) have improved patient-reported outcomes (PROs) in clinical studies. However, limited data exist associating these improvements with health care resource utilization (HCRU) or cost savings. Few studies have evaluated the economic impact of patient-reported physical status and related disease burden in patients with AS in the United States. OBJECTIVE: To assess the association of PRO measures with HCRU and health care costs in patients with AS from a national US registry. METHODS: This cohort study included adults with a diagnosis of AS enrolled in the FORWARD registry from July 2009 to June 2019 who completed at least 1 questionnaire from January 2010 to December 2019 and completed the Health Assessment Questionnaire Disability Index (HAQ-DI) (0-3) and/or Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) (0-10). Patient-reported data for demographics, clinical characteristics, and PROs were collected through questionnaires administered biannually and reported from the most recent questionnaire. Patient-reported HCRU and total health care costs (2019 US dollars) for hospitalizations, emergency department (ED) visits, outpatient visits, diagnostic tests, and procedures were captured during the 6 months prior to the most recent survey completion. The relationship between HAQ-DI or BASDAI and HCRU outcomes was assessed using negative binomial regression models, and the relationship between HAQ-DI or BASDAI and the cost outcomes was evaluated using generalized linear models with γ distribution and log-link function. RESULTS: Overall, 334 patients with AS who completed the HAQ-DI (n = 253) or BASDAI (n = 81) were included. The mean (SD) HAQ-DI and BASDAI scores at the time of patients' most recent surveys were 0.9 (0.7) and 3.7 (2.3), respectively. HAQ-DI score was positively associated with number of hospitalizations, ED visits, outpatient visits, and diagnostic tests, whereas BASDAI was not associated with HCRU outcomes. Overall annualized mean (SD) total health care, medical, and pharmacy costs for patients with AS were $44,783 ($40,595); $6,521 ($12,733); and $38,263 ($40,595), respectively. Annualized total health care, medical, and pharmacy costs adjusted for confounders increased by 35%, 76%, and 26%, respectively, for each 1.0-unit increase in HAQ-DI score (coefficient [95% CI]: 1.35 [1.15-1.58], 1.76 [1.22-2.55]; both P < 0.01 and 1.26 [1.04-1.52]; P < 0.05, respectively); BASDAI score was not significantly associated with cost outcomes. CONCLUSIONS: Higher HAQ-DI scores were associated with higher HCRU and total health care costs among patients with AS in FORWARD, but BASDAI scores were not. These findings indicate that greater functional impairment may impose an increased economic burden compared with other patient-reported measures of AS. DISCLOSURES: A. Ogdie has received consulting fees from Amgen, AbbVie, Bristol Myers Squibb, Celgene, CorEvitas (formerly Corrona), Gilead, Janssen, Lilly, Novartis, Pfizer, and UCB and has received grant support from the National Institutes of Health/National Institute of Arthritis and Musculoskeletal and Skin Diseases, Rheumatology Research Foundation, National Psoriasis Foundation, Pfizer (University of Pennsylvania), Amgen (FORWARD), and Novartis (FORWARD). M. Hwang has received consulting fees from Novartis and UCB and has received grant support (5KL2TR003168-03) from the University of Texas Health Science Center at Houston Center of Clinical and Translational Sciences KL2 program. P. Veeranki and J. Shafrin were employees of PRECISION-heor at the time of this analysis. A. Portelli and S. Sison are employees of PRECISION-heor. S. Pedro does not have anything to disclose. N. Kim was a postdoctoral fellow at the University of Texas at Austin and Baylor Scott and White Health, providing services to Novartis at the time of this study. E. Yi is an employee of Novartis. K. Michaud received grant funding from the Rheumatology Research Foundation at the time of this analysis. This study was funded by Novartis Pharmaceuticals Corporation, East Hanover, NJ.

Economic burden of antibiotic-not-susceptible isolates in uncomplicated urinary tract infection: Analysis of a US integrated delivery network database.

BACKGROUND: Uncomplicated urinary tract infections (uUTIs) are one of the most common bacterial infections in the United States (US). Contemporary data are important for understanding the health economic impact of antimicrobial-resistant uUTIs. We compared the economic burden among patients with uUTI isolates susceptible or not-susceptible to the initial antibiotic prescription. METHODS: This retrospective cohort study utilized electronic health record data (1 July 2016-31 March 2020) from a large Mid-Atlantic US integrated delivery network database. Patients were females aged ≥ 12 years with a uUTI, who received oral antibiotic treatment and had ≥ 1 urine culture within ± 5 days of diagnosis. The primary outcome was the difference in healthcare resource use and costs (all-cause, urinary tract infection [UTI]-related) among patients with susceptible versus not-susceptible isolates during the 6 months after the index uUTI diagnosis. Secondary outcomes included: pharmacy costs, hospital admissions and emergency department visits, as well as the probability of uUTI progressing to complicated UTI (cUTI) between patients with susceptible and not-susceptible isolates. Patient outcomes were compared using 1:1 propensity score matching. Winsorized costs were adjusted to 2020 quarter 1 US dollars ($). RESULTS: A total of 2565 patients were eligible for analysis. The propensity score-matched sample comprised 2018 patients, with an average age of 44.0 and 41.0 years for the susceptible and not-susceptible populations, respectively. In the 6 months post-index uUTI event, patients with not-susceptible isolates had significantly more all-cause prescriptions orders (+ 1.41 [P = 0.001]), UTI-related prescriptions orders (+ 0.26 [P < 0.001]) and a higher probability of all-cause inpatient (+ 1.4% [P = 0.009]), outpatient (+ 6.1% [P = 0.006]), or UTI-related outpatient (+ 3.7% [P = 0.039]) encounters. Patients with a uUTI and an antibiotic-not-susceptible isolate were significantly more likely to progress to cUTI than those with susceptible isolates (odds ratio: 2.35 [confidence interval: 1.66-3.33; P < 0.001]). Over 6 months, patients with not-susceptible versus susceptible isolates had significantly higher all-cause costs (+ $426 [P = 0.031]) and UTI-related costs (+ $157 [P = 0.034]). CONCLUSIONS: Patients with a uUTI caused by antibiotic-not-susceptible isolates had higher healthcare resource usage, costs, and increased likelihood of progressing to cUTI than those with antibiotic-susceptible isolates.

White Paper on the Value of Time Savings for Patients and Healthcare Providers of Breast Cancer Therapy: The Fixed-Dose Combination of Pertuzumab and Trastuzumab for Subcutaneous Injection as an Example.

Health technology assessments and value frameworks are becoming increasingly important for clinical decision-making. Most of these frameworks, however, focus on value to payers rather than patients and healthcare providers and may ignore other sources of economic value such as patient and physician time cost, impact on productivity, and direct health system costs. This article focusses on fixed-dose combination of pertuzumab and trastuzumab for subcutaneous injection (PH FDC SC) in the treatment of HER2-positive breast cancer. We review relevant clinical evidence, examine data on time and resource use of the subcutaneous administration of trastuzumab compared with intravenous treatment and how it can be extrapolated to PH FDC SC, and discuss the value PH FDC SC can bring to patients and healthcare providers. We will also provide our own experiences of PH FDC SC from the healthcare (oncologist, healthcare economist, pharmacist) and patient point of view. The data, combined with our personal experiences, suggest that switching from intravenous pertuzumab and trastuzumab to PH FDC SC could reduce non-drug costs for healthcare providers treating patients with HER2-positive breast cancer through time savings and other economic benefits. Furthermore, PH FDC SC could also save patient time given its shorter administration and post-injection observation time versus intravenous infusions, potentially resulting in reduced productivity loss. These benefits could be applied to other subcutaneous formulations, either currently available or in development.

New therapies are increasingly assessed by looking at their value to those who pay for them rather than their value to patients and healthcare providers. Value assessments conducted from the payers' perspective often ignore such things as patient and healthcare system time and costs. The fixed-dose combination of pertuzumab and trastuzumab for subcutaneous injection (also known as pertuzumab, trastuzumab, and hyaluronidase-zzxf, abbreviated to PH FDC SC), is injected under the skin to treat a subtype of breast cancer called HER2-positive breast cancer. PH FDC SC is as effective as pertuzumab and trastuzumab, which are infused separately into a vein, but takes a lot less time to administer to patients. This transition is similar to what was seen when a subcutaneous version of trastuzumab was developed and compared to the intravenous original. Also, subcutaneous trastuzumab reduced costs associated with treating patients compared with intravenous infusions. The same benefits of PH FDC SC to patients and healthcare providers can be expected, and our personal experiences as an oncologist, healthcare economist, patient, and pharmacist agree. PH FDC SC could save patient and healthcare provider time given its shorter injection and observation times versus intravenous infusions, potentially resulting in better productivity for these people and a smaller cost to healthcare providers. These benefits could be applied to other subcutaneous formulations, either currently available or in development.

Quantifying spillover benefits in value assessment: a case study of increased graft survival on the US kidney transplant waitlist.

AIM: To quantify the wider impacts of increased graft survival on the size of the kidney transplant waitlist and health and economic outcomes. MATERIALS AND METHODS: The analysis employed known steady-state solutions to a double-queueing system as well as simulations of this system. Baseline input parameters were sourced from the Organ Procurement and Transplant Network and the United States Renal Data System. Three increased graft survival scenarios were modeled: decreases in repeat transplant candidates joining the waitlist of 25%, 50%, and 100%. RESULTS: Under the three scenarios, we estimated that the US waitlist size would decrease from 91,822 to 85,461 (6.9% decrease), 80,073 (12.8% decrease), and 69,340 (24.4% decrease), respectively. Patient outcomes improved, with lifetime quality-adjusted life years (QALYs) for a 1-year cohort of transplant recipients increasing by 10,010, 16,888, and 43,345 over the three scenarios. Discounted lifetime costs for the cohort in the new steady state were lower by $1.6 billion, $2.3 billion, and $9.0 billion for each scenario, respectively. Spillover impacts (i.e. benefits that accrued beyond the patients who directly experienced increased graft survival) accounted for 41-48% of the QALY gains and ranged from cost increases of 3.3% to decreases of 5.5%. LIMITATIONS: The model is a simplification of reality and does not account for the full degree of patient heterogeneity occurring in the real world. Health economic outcomes are extrapolated based on the assumption that the median patient is representative of the overall population. CONCLUSIONS: Increasing graft survival reduces demand from repeat transplants candidates, allowing additional candidates to receive transplants. These spillover impacts decrease waitlist size and shorten wait times, leading to improvements in graft and patient survival as well as quality-of-life. Cost-effectiveness analyses of treatments that increase kidney graft survival should incorporate spillover benefits that accrue beyond the direct recipient of an intervention.

Measuring the Value Healthy Individuals Place on Generous Insurance Coverage of Severe Diseases: A Stated Preference Survey of Adults Diagnosed With and Without Lung Cancer.

OBJECTIVES: To compare the ex ante willingness to pay (WTP) of healthy individuals for generous insurance coverage of novel lung cancer treatments to the WTP for coverage of such treatment among individuals with lung cancer. METHODS: A survey was administered to 2 cohorts of US adults: (1) healthy individuals without cancer and (2) individuals diagnosed with lung cancer. A multiple random staircase survey design was used to elicit respondent WTP for coverage of novel lung cancer therapy associated with survival gains. RESULTS: Of the 84 937 healthy individuals invited, 300 completed the survey. Of the 36 249 in the lung cancer cohort invited, 250 completed the survey. Mean age by cohort was 50.0 (SD 14.6) and 48.4 (SD 16.8) years, and 55.2% and 47.2% were female, respectively. Respondents in the healthy and lung cancer cohorts were willing to pay $97.52 (95% confidence interval (CI) $89.89-$105.15) and $22 304 (95% CI $20 194-$24 414) per month, respectively, for coverage of a novel therapy providing 5-year survival of 15% versus standard-of-care therapy with a 5-year survival of 4%. After accounting for the likelihood that healthy individuals are diagnosed with lung cancer in the future, we estimated that 89.8% of the total value of new lung cancer treatments comes from the WTP healthy individuals place on generous insurance coverage. CONCLUSIONS: Total societal willingness to pay for lung cancer is much higher than conventionally thought, as most healthy individuals are risk-averse and highly value having lung cancer treatments available to them in the future.

For which diseases do broader value elements matter most? An evaluation across 20 ICER evidence reports.

BACKGROUND: U.S. value framework developers such as the Institute for Clinical and Economic Review (ICER) use cost-effectiveness analysis to value new health care technologies. Often, these value assessment frameworks use a health system perspective without fully accounting for societal and broader benefits and costs of an intervention. Although there is ongoing debate about the most appropriate methods for including broader value elements in value assessment, it remains unclear whether the inclusion of these value elements is likely to affect the quantitative estimates of treatment value. OBJECTIVE: To assess variations in the relevance of broader value elements to cost-effectiveness analysis across diseases. METHODS: Thirty-two broader value elements (e.g., caregiver burden, health equity, real option value, productivity) not traditionally included in health technology assessments were identified through a targeted literature review. Evidence reports published by ICER between July 2017 and January 2020 were evaluated to identify which broader value elements were discussed as relevant to each disease in the report text. The study examined whether there were associations among ICER's discussion of broader value elements, rare disease status, treatment cost, estimated treatment cost-effectiveness, and ICER committee voting results for contextual considerations and additional benefits/disadvantages. RESULTS: The most commonly cited broader value element category in the ICER evidence reports was household and leisure (e.g., absenteeism from normal activities and caregiver burden). More value elements were cited for inherited retinal disease (19 elements) and sickle cell disease (18 elements) than for other diseases. Cardiovascular disease and diabetes had the fewest number of value elements cited (7 elements). Rare diseases were more likely to have broader value elements cited compared with nonrare diseases (15.9 vs. 11.5, P < 0.001). Treatments with higher (i.e., less favorable) incremental cost-effectiveness ratios were more likely to have a greater number of broader value elements cited (ρ = 0.625, P < 0.001). CONCLUSIONS: The presence of broader value elements varied across diseases, with less cost-effective treatments more likely to have a higher number of relevant broader value elements. Inclusion of all relevant value elements in value assessments will more appropriately incentivize innovation and improve allocation of research funding. DISCLOSURES: This study was sponsored by Novartis Pharmaceutical Corporation. At the time of this study, Shafrin was employed by PRECISIONheor, a consultancy to the life sciences industry that received financial support from Novartis to conduct this study. Dennen, Pednekar, and Birch are employed by PRECISIONheor. Bhor was an employee of Novartis Pharmaceutical Corporation at the time this research was conducted and manuscript was developed and reports grants from Novartis, unrelated to this work. Kanter has served on scientific advisory boards and steering committees for and reports receiving consulting fees from Novartis Pharmaceutical Corporation and is a site principal investigator on studies funded by Novartis Pharmaceutical Corporation. Kantar also reports support from Sickle Cell Disease Association of America Inc. and National Heart, Lung, and Blood Institute, unrelated to this work. Neumann reports advisory boards or consulting fees from Novartis Pharmaceutical Corporation and PRECISIONheor, as well as advisory boards or consulting fees unrelated to this study from AbbVie, Amgen, Avexis, Bayer, Congressional Budget Office, Janssen, Merck, Novartis, Novo Nordisk, Precision Health Economics, Veritech, Vertex; funding from The CEA Registry Sponsors by various pharmaceutical and medical device companies; and grants from Amgen, Lundbeck, Bill and Melinda Gates Foundation, National Pharmaceutical Council, Alzheimer's Association, and the National Institutes for Health.

Adoption of Digital Health Technologies in the Practice of Behavioral Health: Qualitative Case Study of Glucose Monitoring Technology.

BACKGROUND: Evaluation of patients with serious mental illness (SMI) relies largely on patient or caregiver self-reported symptoms. New digital technologies are being developed to better quantify the longitudinal symptomology of patients with SMI and facilitate disease management. However, as these new technologies become more widely available, psychiatrists may be uncertain about how to integrate them into daily practice. To better understand how digital tools might be integrated into the treatment of patients with SMI, this study examines a case study of a successful technology adoption by physicians: endocrinologists' adoption of digital glucometers. OBJECTIVE: This study aims to understand the key facilitators of and barriers to clinician and patient adoption of digital glucose monitoring technologies to identify lessons that may be applicable across other chronic diseases, including SMIs. METHODS: We conducted focus groups with practicing endocrinologists from 2 large metropolitan areas using a semistructured discussion guide designed to elicit perspectives of and experiences with technology adoption. The thematic analysis identified barriers to and facilitators of integrating digital glucometers into clinical practice. Participants also provided recommendations for integrating digital health technologies into clinical practice more broadly. RESULTS: A total of 10 endocrinologists were enrolled: 60% (6/10) male; a mean of 18.4 years in practice (SD 5.6); and 80% (8/10) working in a group practice setting. Participants stated that digital glucometers represented a significant change in the treatment paradigm for diabetes care and facilitated more effective care delivery and patient engagement. Barriers to the adoption of digital glucometers included lack of coverage, provider reimbursement, and data management support, as well as patient heterogeneity. Participant recommendations to increase the use of digital health technologies included expanding reimbursement for clinician time, streamlining data management processes, and customizing the technologies to patient needs. CONCLUSIONS: Digital glucose monitoring technologies have facilitated more effective, individualized care delivery and have improved patient engagement and health outcomes. However, key challenges faced by the endocrinologists included lack of reimbursement for clinician time and nonstandardized data management across devices. Key recommendations that may be relevant for other diseases include improved data analytics to quickly and accurately synthesize data for patient care management, streamlined software, and standardized metrics.

Alternative payment models and innovation: a case study of US health system adoption of a sacubitril/valsartan to treat acute decompensated heart failure.

AIM: To understand the financial impact of health system adoption of novel heart failure medications under US alternative payment models (APMs). MATERIALS AND METHODS: This study used a decision tree model to assess the financial impact of health system adoption of sacubitril/valsartan to treat acute decompensated heart failure (ADHF). A comparator scenario modeled current health care utilization and cost for treating hospitalized ADHF patients with angiotensin-converting-enzyme inhibitors (ACEi) or angiotensin-receptor blockers (ARB). The study then measured the impact of adopting sacubitril/valsartan to treat ADHF on health system economic outcomes. Differences in treatment efficacy were based on the PIONEER-HF clinical trial. The financial impact of changes in patient outcomes under the sacubitril/valsartan and ACEi/ARB arms was assessed across three APMs: the Medicare Shared Savings Program, Bundled Payments for Care Improvement, and fee-for-service payments adjusted according to the Hospital Readmission Reduction Program. RESULTS: Sacubitril/valsartan reduced re-hospitalizations after an initial ADHF admission by 46.3% for individuals aged 18-64 years and 23.4% for individuals aged ≥65 years. Health systems' financial benefit of adopting sacubitril/valsartan was $740 per ADHF case per year (PCPY). Savings were larger for patients aged ≥65 years ($803 PCPY) compared to those <65 years ($653 PCPY). The majority of the health system financial benefit came from changes in APM bonus and penalty reimbursements. Value-based payments from the Hospital Readmission Reduction Program ($1,190 financial gain PCPY) and the Bundled Care Payment Improvement Initiative ($645 financial gain PCPY) produced larger financial benefits than participation in the Medicare Shared Savings Program ($253 financial gain PCPY). LIMITATIONS: The model uses clinical trial data, which may not reflect real-world outcomes. Further, the financial implications were modeled based only on three widely used APMs. CONCLUSION: Sacubitril/valsartan adoption decreased hospitalizations and led to a positive net financial impact on health systems after accounting for APM bonus payments.

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.

A Flexible Open-Source Decision Model for Value Assessment of Biologic Treatment for Rheumatoid Arthritis.

OBJECTIVE: The nature of model-based cost-effectiveness analysis can lead to disputes in the scientific community. We propose an iterative and collaborative approach to model development by presenting a flexible open-source simulation model for rheumatoid arthritis (RA), accessible to both technical and non-technical end-users. METHODS: The RA model is a discrete-time individual patient simulation with 6-month cycles. Model input parameters were estimated based on currently available evidence and treatment effects were obtained with Bayesian network meta-analysis techniques. The model contains 384 possible model structures informed by previously published models. The model consists of the following components: (i) modifiable R and C++ source code available in a GitHub repository; (ii) an R package to run the model for custom analyses; (iii) detailed model documentation; (iv) a web-based user interface for full control over the model without the need to be well-versed in the programming languages; and (v) a general audience web-application allowing those who are not experts in modeling or health economics to interact with the model and contribute to value assessment discussions. RESULTS: A primary function of the initial version of RA model is to help understand and quantify the impact of parameter uncertainty (with probabilistic sensitivity analysis), structural uncertainty (with multiple competing model structures), the decision framework (cost-effectiveness analysis or multi-criteria decision analysis), and perspective (healthcare or limited societal) on estimates of value. CONCLUSION: In order for a decision model to remain relevant over time it needs to evolve along with its supporting body of clinical evidence and scientific insight. Multiple clinical and methodological experts can modify or contribute to the RA model at any time due to its open-source nature.

The economic burden of switching targeted disease-modifying anti-rheumatic drugs among rheumatoid arthritis patients.

AIMS: To estimate real world healthcare costs and resource utilization of rheumatoid arthritis (RA) patients associated with targeted disease modifying anti-rheumatic drugs (tDMARD) switching in general and switching to abatacept specifically. MATERIALS AND METHODS: RA patients initiating a tDMARD were identified in IMS PharMetrics Plus health insurance claims data (2010-2016), and outcomes measured included monthly healthcare costs per patient (all-cause, RA-related) and resource utilization (inpatient stays, outpatient visits, emergency department [ED] visits). Generalized linear models were used to assess (i) average monthly costs per patient associated with tDMARD switching, and (ii) among switchers only, costs of switching to abatacept vs tumor necrosis factor inhibitors (TNFi) or other non-TNFi. Negative binomial regressions were used to determine incident rate ratios of resource utilization associated with switching to abatacept. RESULTS: Among 11,856 RA patients who initiated a tDMARD, 2,708 switched tDMARDs once and 814 switched twice (to a third tDMARD). Adjusted average monthly costs were higher among patients who switched to a second tDMARD vs non-switchers (all-cause: $4,785 vs $3,491, p < .001; RA-related: $3,364 vs $2,297, p < .001). Monthly RA-related costs were higher for patients switching to a third tDMARD compared to non-switchers remaining on their second tDMARD ($3,835 vs $3,383, p < .001). Switchers to abatacept had significantly lower RA-related monthly costs vs switchers to TNFi ($3,129 vs $3,436, p = .021), and numerically lower all-cause costs ($4,444 vs $4,741, p = 0.188). Switchers to TNFi relative to abatacept had more frequent inpatient stays after switch (incidence rate ratio (IRR) = 1.85, p = .031), and numerically higher ED visits (IRR = 1.32, p = .093). Outpatient visits were less frequent for TNFi switchers (IRR = 0.83, p < .001) compared to switchers to abatacept. LIMITATIONS AND CONCLUSIONS: Switching to another tDMARD was associated with higher healthcare costs. Switching to abatacept, however, was associated with lower RA-related costs, fewer inpatient stays, but more frequent outpatient visits compared to switching to a TNFi.

Long-Term Health and Economic Value of Improved Mobility among Older Adults in the United States.

BACKGROUND: Mobility impairments have substantial physical and mental health consequences, resulting in diminished quality of life. Most studies on the health economic consequences of mobility limitations focus on short-term implications. OBJECTIVES: To examine the long-term value of improving mobility in older adults. METHODS: Our six-step approach used clinical trial data to calibrate mobility improvements and estimate health economic outcomes using a microsimulation model. First, we measured improvement in steps per day calibrated with clinical trial data examining hylan G-F 20 viscosupplementation treatment. Second, we created a cohort of patients 51 years and older with osteoarthritis. In the third step, we estimated their baseline quality of life. Fourth, we translated steps-per-day improvements to changes in quality of life using estimates from the literature. Fifth, we calibrated quality of life in this cohort to match those in the trial. Last, we incorporated these data and parameters into The Health Economic Medical Innovation Simulation model to estimate how mobility improvements affect functional status limitations, medical expenditures, nursing home utilization, employment, and earnings between 2012 and 2030. RESULTS: In our sample of 12.6 million patients, 66.7% were female and 70% had a body mass index of more than 25 kg/m2. Our model predicted that a 554-step-per-day increase in mobility would reduce functional status limitations by 5.9%, total medical expenditures by 0.9%, and nursing home utilization by 2.8%, and increase employment by 2.9%, earnings by 10.3%, and monetized quality of life by 3.2% over this 18-year period. CONCLUSIONS: Interventions that improve mobility are likely to reduce long-run medical expenditures and nursing home utilization and increase employment.

The potential value of rapid, cloud-enabled onsite testing for the diagnosis of rheumatoid arthritis in the United States.

AIMS: Improvements in information technology have granted the recent development of rapid, cloud-enabled, onsite laboratory testing for rheumatoid arthritis (RA). This study aims to quantify the value to payers of such technologies. MATERIALS AND METHODS: To calculate the value of rapid, cloud-enabled, onsite laboratory testing to diagnose RA relative to traditional, centralized laboratory testing, an Excel-based decision tree model was created that simulated potential cost-savings to payers who cover routine evaluations of RA patients in the US. First, a conceptual framework was created to identify the value components of rapid, cloud-enabled onsite testing. Second, value associated with patient time savings, savings on visit fees, change in treatment costs, and QALY improvements was measured, leveraging existing literature and information from an observational study. Lastly, these value components were combined to estimate the total incremental value accruing to payers per patient-year relative to centralized laboratory testing. RESULTS: Rapid, cloud-enabled, onsite testing is estimated to save one office and 1.81 laboratory visits during the evaluation period for the average patient. Results from an observational study found that rapid, cloud-enabled testing increased the likelihood of completing diagnostic orders from 84.5% to 97%, resulting in an increased probability of early treatment (3.5 percentage points) with disease-modifying anti-rheumatic drugs among patients eligible for treatment. The combined total value was $5,648 per evaluated patient-year. This value is primarily attributed to health benefits of early treatment ($5,048), fewer visit payments ($459), and patient time savings due to fewer office ($216) and laboratory visits ($255). LIMITATIONS AND CONCLUSIONS: Data on the impact of rapid, cloud-enabled, onsite testing on patient health, care delivery, and clinical decision-making is scarce. More robust real-world data would confirm the validity of our model. Rapid, cloud-enabled, onsite testing has the potential to generate significant value to payers.