Health care resource utilization, quality metrics, and costs of bladder cancer within the Oncology Care Model.

OBJECTIVES: New and emerging therapies have significantly changed the bladder cancer (BC) treatment landscape and can potentially affect spending and patient care in CMS' Oncology Care Model (OCM), a service delivery and payment model for voluntarily participating practices. The objectives of this analysis were to estimate health care resource utilization (HCRU) and benchmark spending per OCM episode of BC, and to model spending drivers and quality metrics. STUDY DESIGN: Retrospective cohort study. METHODS: A retrospective cohort study was conducted of OCM episodes triggered by receipt of anticancer therapy among Medicare beneficiaries from 2016 to 2018. Based on this, an average performance estimation was conducted to assess the impact of hypothetical changes in novel therapy use by OCM practices. RESULTS: BC accounted for approximately 3% (n = 60,099) of identified OCM episodes. Relative to low-risk episodes, high-risk episodes were associated with greater HCRU and worse OCM quality metrics. Mean spending per high-risk episode was $37,857 (low-risk episode: $9204), with $11,051 spent on systemic therapies and $7158 on inpatient services. In the estimation, high- and low-risk BC exceeded the spending target by 1.7% and 9.4%, respectively. This did not affect payments to practices and no retrospective payments were necessary. CONCLUSIONS: As 3% of OCM episodes were attributed to BC, with only one-third classified as high-risk, controlling expenditure on novel therapies for advanced BC is unlikely to affect overall practice performance. The average performance estimation further emphasized the minimal impact that novel therapy spending in high-risk BC has on OCM payments to practices.

Understanding Treatment Patterns and Outcomes among Patients with De Novo Unresectable Locally Advanced or Metastatic Urothelial Cancer: A Population-Level Retrospective Analysis from Alberta, Canada.

Despite a high disease burden, real-world data on treatment patterns in patients with unresectable locally advanced or metastatic urothelial carcinoma (la/mUC) in Canada are limited. This retrospective, longitudinal cohort study describes treatment patterns and survival in a population of patients with de novo unresectable la/mUC from Alberta, Canada, diagnosed between 1 January 2015 and 31 December 2019, followed until mid-2020. The outcomes of interest were systemic therapy treatment patterns and overall survival (OS). Of 206 patients, most (65.0%, n = 134) did not receive any systemic therapies. Of 72 patients (35.0%) who received first-line systemic therapy, the median duration of treatment was 2.8 months (IQR 3.3). Thirty-five patients (48.6% of those who received first-line therapy) received subsequent second-line therapy, for a median of 3.0 months (IQR 3.3). In all patients (n = 206), the median OS from diagnosis was 5.3 months (95% CI, 4.5-7.0). In patients who received treatment, the median OS from the initiation of first-line and second-line systemic therapy was 9.1 (6.4-11.6) and 4.6 months (3.9-19.2), respectively. The majority of patients did not receive first-line systemic therapy, and, in those who did, survival outcomes were poor. This study highlights the significant unmet need for safe and efficacious therapies for patients with la/mUC in Canada.

Clinical and Patient-Reported Outcomes of Advanced Urothelial Carcinoma Following Discontinuation of PD-1/L1 Inhibitor Therapy.

INTRODUCTION: The patterns of care and attrition of locally advanced or metastatic urothelial carcinoma (la/mUC) patients eligible for systemic therapy following PD-1/L1 inhibitors are unclear. The objective of this study was to evaluate the clinical characteristics and treatment patterns among patients with la/mUC following discontinuation of first-line (1L) or second-line (2L) PD-1/L1 inhibitor therapy. METHODS: An ambispective, multisite, chart review study was conducted in the United States, including patients with la/mUC. Eligible patients had initiated and subsequently discontinued PD-1/L1 therapy in the 1L or 2L setting for la/mUC between May 2016 and July 2018; with follow-up through October 2019. Patient characteristics, treatments, and overall survival (OS) were described. Patients had the option to complete a 1-time patient reported outcomes (PRO) survey. RESULTS: Among 300 patients included in the chart review, 198 (66%) received 1L PD-1/L1 inhibitor and 102 (34%) received 2L PD-1/L1 inhibitor. Following discontinuation of PD-1/L1 inhibitor therapy, 34% (n = 68) received subsequent therapy in 2L and 29% (n = 30) in third-line (3L). The median OS post-1L PD-1/L1 inhibitor was 9.4 (95% CI 8.6-NA) and 2.5 months (95% CI 2.24-3.50) for those who received and did not receive subsequent therapy, respectively. Following 2L PD-1/L1 inhibitor discontinuation, the median OS was 5.7 (95% CI 5.1-7.8) and 3.98 (95% CI 3.29-4.87) months for those who received and did not receive subsequent therapy, respectively. Among those with PRO data, 64% reported experiencing cancer-related pain and 29.6% received an opioid. Only 12.7% reported having a caregiver, requiring approximately 13 h/d of service. CONCLUSION: The symptom and caregiver burden are high among real-world patients with la/mUC who discontinued 1L or 2L PD-1/L1 inhibitors and outcomes are dismal, with a minority receiving subsequent therapy. Patterns of care in the setting of 1L maintenance avelumab and novel agents require further investigation.

Treatment with opioids in patients with locally advanced or metastatic urothelial carcinoma and matched non-cancer controls.

BACKGROUND: Locally advanced or metastatic urothelial carcinoma (la/mUC) is an aggressive disease with a poor long-term survival. While patients frequently report pain, there are limited data on the patient experience with pain and pain medication use. This study used real-world data to quantify treatment with opioids, as a proxy for pain, in patients with la/mUC compared with matched non-cancer controls. METHODS: This was a retrospective claims analysis, using the IBM® MarketScan® databases, of adults diagnosed with urothelial carcinoma and initiating ≥1 la/mUC therapy between May 2016 and June 2019. Index date was date of first systemic therapy claim for la/mUC; baseline was the 6 months pre-index; follow-up was from index until disenrollment or study end. Proportion with treatment with opioids, number of opioid prescriptions, and daily morphine-equivalent dose (MEQ; in morphine milligram equivalents/day) in patients with la/mUC and matched non-cancer controls from the same databases were assessed. RESULTS: We identified 1293 patients with la/mUC and matched 1:3 with 3862 non-cancer controls. Mean (SD) follow-up was 1.26 (0.74) years in patients with la/mUC and 1.29 (0.72) years in controls. A greater proportion of patients with la/mUC, compared with controls, used opioids during both baseline (63.6% vs. 19.4%) and follow-up (61.4% vs. 27.9%). Among those who used opioids, mean monthly prescriptions (number of medications claims/patient/month) were 0.55 both in patients with la/mUC and controls during baseline, and 0.49 and 0.39, respectively, at follow-up. Daily MEQ among those who used opioids was 53.6 and 45.7 during baseline, and 74.7 and 40.8 at follow-up, in patients with la/mUC and controls, respectively. In patients with la/mUC, mean opioid prescriptions and daily MEQ increased during later lines of therapy. CONCLUSION: In patients with la/mUC, pain requiring opioids is common at diagnosis, worsens as the patient progresses, and is consistently higher than in matched controls. Improvement in disease control with more effective therapies may reduce cancer pain in this population.

Cost-savings for biosimilars in the United States: a theoretical framework and budget impact case study application using filgrastim.

BACKGROUND: Biosimilars can directly reduce the cost of treating patients for whom a reference biologic is indicated by offering a highly similar, lower priced alternative. We examine factors related to biosimilar regulatory approval, uptake, pricing, and financing and the potential impact on drug expenditures in the U.S. METHODS: We developed a framework to illustrate how key factors including regulatory policies, provider and patient perception, pricing, and payer policies impact biosimilar cost-savings. Further, we developed a budget impact cost model to estimate savings from filgrastim biosimilars under various scenarios. The model uses publicly available data on disease incidence, treatment patterns, market share, and drug prices to estimate the cost-savings over a 5-year time horizon. RESULTS: We estimate five-year cost savings of $256 million, of which 18% ($47 million) are from reduced patient out-of-pocket costs, 34% ($86 million) are savings to commercial payers, and 48% ($123 million) are savings for Medicare. Additional scenarios demonstrate the impact of uncertain factors, including price, uptake, and financing policies. CONCLUSIONS: A variety or interrelated factors influence the development, uptake, and cost-savings for Biosimilars use in the U.S. The filgrastim case is a useful example that illustrates these factors and the potential magnitude of costs savings.

Estimated economic impact of vaccinations in 73 low- and middle-income countries, 2001-2020.

OBJECTIVE: To estimate the economic impact likely to be achieved by efforts to vaccinate against 10 vaccine-preventable diseases between 2001 and 2020 in 73 low- and middle-income countries largely supported by Gavi, the Vaccine Alliance. METHODS: We used health impact models to estimate the economic impact of achieving forecasted coverages for vaccination against Haemophilus influenzae type b, hepatitis B, human papillomavirus, Japanese encephalitis, measles, Neisseria meningitidis serogroup A, rotavirus, rubella, Streptococcus pneumoniae and yellow fever. In comparison with no vaccination, we modelled the costs - expressed in 2010 United States dollars (US$) - of averted treatment, transportation costs, productivity losses of caregivers and productivity losses due to disability and death. We used the value-of-a-life-year method to estimate the broader economic and social value of living longer, in better health, as a result of immunization. FINDINGS: We estimated that, in the 73 countries, vaccinations given between 2001 and 2020 will avert over 20 million deaths and save US$ 350 billion in cost of illness. The deaths and disability prevented by vaccinations given during the two decades will result in estimated lifelong productivity gains totalling US$ 330 billion and US$ 9 billion, respectively. Over the lifetimes of the vaccinated cohorts, the same vaccinations will save an estimated US$ 5 billion in treatment costs. The broader economic and social value of these vaccinations is estimated at US$ 820 billion. CONCLUSION: By preventing significant costs and potentially increasing economic productivity among some of the world's poorest countries, the impact of immunization goes well beyond health.

Funding gap for immunization across 94 low- and middle-income countries.

Novel vaccine development and production has given rise to a growing number of vaccines that can prevent disease and save lives. In order to realize these health benefits, it is essential to ensure adequate immunization financing to enable equitable access to vaccines for people in all communities. This analysis estimates the full immunization program costs, projected available financing, and resulting funding gap for 94 low- and middle-income countries over five years (2016-2020). Vaccine program financing by country governments, Gavi, and other development partners was forecasted for vaccine, supply chain, and service delivery, based on an analysis of comprehensive multi-year plans together with a series of scenario and sensitivity analyses. Findings indicate that delivery of full vaccination programs across 94 countries would result in a total funding gap of $7.6 billion (95% uncertainty range: $4.6-$11.8 billion) over 2016-2020, with the bulk (98%) of the resources required for routine immunization programs. More than half (65%) of the resources to meet this funding gap are required for service delivery at $5.0 billion ($2.7-$8.4 billion) with an additional $1.1 billion ($0.9-$2.7 billion) needed for vaccines and $1.5 billion ($1.1-$2.0 billion) for supply chain. When viewed as a percentage of total projected costs, the funding gap represents 66% of projected supply chain costs, 30% of service delivery costs, and 9% of vaccine costs. On average, this funding gap corresponds to 0.2% of general government expenditures and 2.3% of government health expenditures. These results suggest greater need for country and donor resource mobilization and funding allocation for immunizations. Both service delivery and supply chain are important areas for further resource mobilization. Further research on the impact of advances in service delivery technology and reductions in vaccine prices beyond this decade would be important for efficient investment decisions for immunization.

Return On Investment From Childhood Immunization In Low- And Middle-Income Countries, 2011-20.

An analysis of return on investment can help policy makers support, optimize, and advocate for the expansion of immunization programs in the world's poorest countries. We assessed the return on investment associated with achieving projected coverage levels for vaccinations to prevent diseases related to ten antigens in ninety-four low- and middle-income countries during 2011-20, the Decade of Vaccines. We derived these estimates by using costs of vaccines, supply chains, and service delivery and their associated economic benefits. Based on the costs of illnesses averted, we estimated that projected immunizations will yield a net return about 16 times greater than costs over the decade (uncertainty range: 10-25). Using a full-income approach, which quantifies the value that people place on living longer and healthier lives, we found that net returns amounted to 44 times the costs (uncertainty range: 27-67). Across all antigens, net returns were greater than costs. But to realize the substantial positive return on investment from immunization programs, it is essential that governments and donors provide the requisite investments.

Household Size and the Decision to Purchase Health Insurance in Cambodia: Results of a Discrete-Choice Experiment with Scale Adjustment.

BACKGROUND: Community-based health insurance (CBHI) schemes have been introduced in low- and middle-income countries to increase health service utilization and provide financial protection from high healthcare expenditures. OBJECTIVE: We assess the impact of household size on decisions to enroll in CBHI and demonstrate how to correct for group disparity in scale (i.e. variance differences). METHODS: A discrete choice experiment was conducted across five CBHI attributes. Preferences were elicited through forced-choice paired comparison choice tasks designed based on D-efficiency. Differences in preferences were examined between small (1-4 family members) and large (5-12 members) households using conditional logistic regression. Swait and Louviere test was used to identify and correct for differences in scale. RESULTS: One-hundred and sixty households were surveyed in Northwest Cambodia. Increased insurance premium was associated with disutility [odds ratio (OR) 0.61, p < 0.01], while significant increase in utility was noted for higher hospital fee coverage (OR 10.58, p < 0.01), greater coverage of travel and meal costs (OR 4.08, p < 0.01), and more frequent communication with the insurer (OR 1.33, p < 0.01). While the magnitude of preference for hospital fee coverage appeared larger for the large household group (OR 14.15) compared to the small household group (OR 8.58), differences in scale were observed (p < 0.05). After adjusting for scale (k, ratio of scale between large to small household groups = 1.227, 95 % confidence interval 1.002-1.515), preference differences by household size became negligible. CONCLUSION: Differences in stated preferences may be due to scale, or variance differences between groups, rather than true variations in preference. Coverage of hospital fees, travel and meal costs are given significant weight in CBHI enrollment decisions regardless of household size. Understanding how community members make decisions about health insurance can inform low- and middle-income countries' paths towards universal health coverage.

Costs of vaccine programs across 94 low- and middle-income countries.

While new mechanisms such as advance market commitments and co-financing policies of the GAVI Alliance are allowing low- and middle-income countries to gain access to vaccines faster than ever, understanding the full scope of vaccine program costs is essential to ensure adequate resource mobilization. This costing analysis examines the vaccine costs, supply chain costs, and service delivery costs of immunization programs for routine immunization and for supplemental immunization activities (SIAs) for vaccines related to 18 antigens in 94 countries across the decade, 2011-2020. Vaccine costs were calculated using GAVI price forecasts for GAVI-eligible countries, and assumptions from the PAHO Revolving Fund and UNICEF for middle-income countries not supported by the GAVI Alliance. Vaccine introductions and coverage levels were projected primarily based on GAVI's Adjusted Demand Forecast. Supply chain costs including costs of transportation, storage, and labor were estimated by developing a mechanistic model using data generated by the HERMES discrete event simulation models. Service delivery costs were abstracted from comprehensive multi-year plans for the majority of GAVI-eligible countries and regression analysis was conducted to extrapolate costs to additional countries. The analysis shows that the delivery of the full vaccination program across 94 countries would cost a total of $62 billion (95% uncertainty range: $43-$87 billion) over the decade, including $51 billion ($34-$73 billion) for routine immunization and $11 billion ($7-$17 billion) for SIAs. More than half of these costs stem from service delivery at $34 billion ($21-$51 billion)-with an additional $24 billion ($13-$41 billion) in vaccine costs and $4 billion ($3-$5 billion) in supply chain costs. The findings present the global costs to attain the goals envisioned during the Decade of Vaccines to prevent millions of deaths by 2020 through more equitable access to existing vaccines for people in all communities. By projecting the full costs of immunization programs, our findings may aid to garner greater country and donor commitments toward adequate resource mobilization and efficient allocation. As service delivery costs have increasingly become the main driver of vaccination program costs, it is essential to pay additional consideration to health systems strengthening.