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Purpose: To address the need for more accurate risk stratification models for cancer immuno-oncology, this study aimed to develop a machine-learned Bayesian network model (BNM) for predicting outcomes in patients with metastatic renal cell carcinoma (mRCC) being treated with immunotherapy., Methods: Patient-level data from the randomized, phase III CheckMate 025 clinical trial comparing nivolumab with everolimus for second-line treatment in patients with mRCC were used to develop the BNM. Outcomes of interest were overall survival (OS), all-cause adverse events, and treatment-related adverse events (TRAE) over 36 months after treatment initiation. External validation of the model's predictions for OS was conducted using data from select centers from the International Metastatic Renal Cell Carcinoma Database Consortium (IMDC)., Results: Areas under the receiver operating characteristic curve (AUCs) for BNM-based classification of OS using baseline data were 0.74, 0.71, and 0.68 over months 12, 24, and 36, respectively. AUC for OS at 12 months increased to 0.86 when treatment response and progression status in year 1 were included as predictors; progression and response at 12 months were highly prognostic of all outcomes over the 36-month period. AUCs for adverse events and treatment-related adverse events were approximately 0.6 at 12 months but increased to approximately 0.7 by 36 months. Sensitivity analysis comparing the BNM with machine learning classifiers showed comparable performance. Test AUC on IMDC data for 12-month OS was 0.71 despite several variable imbalances. Notably, the BNM outperformed the IMDC risk score alone., Conclusion: The validated BNM performed well at prediction using baseline data, particularly with the inclusion of response and progression at 12 months. Additionally, the results suggest that 12 months of follow-up data alone may be sufficient to inform long-term survival projections in patients with mRCC.

Introduction: Biosimilars have the potential to enhance the sustainability of evolving health care systems. A sustainable biosimilars market requires all stakeholders to balance competition and supply chain security. However, there is significant variation in the policies for pricing, procurement, and use of biosimilars in the European Union. A modified Delphi process was conducted to achieve expert consensus on biosimilar market sustainability in Europe., Methods: The priorities of 11 stakeholders were explored in three stages: a brainstorming stage supported by a systematic literature review (SLR) and key materials identified by the participants; development and review of statements derived during brainstorming; and a facilitated roundtable discussion., Results: Participants argued that a sustainable biosimilar market must deliver tangible and transparent benefits to the health care system, while meeting the needs of all stakeholders. Key drivers of biosimilar market sustainability included: (i) competition is more effective than regulation; (ii) there should be incentives to ensure industry investment in biosimilar development and innovation; (iii) procurement processes must avoid monopolies and minimize market disruption; and (iv) principles for procurement should be defined by all stakeholders. However, findings from the SLR were limited, with significant gaps on the impact of different tender models on supply risks, savings, and sustainability., Conclusions: A sustainable biosimilar market means that all stakeholders benefit from appropriate and reliable access to biological therapies. Failure to care for biosimilar market sustainability may impoverish biosimilar development and offerings, eventually leading to increased cost for health care systems and patients, with fewer resources for innovation.

Objectives: The causes of oncology drug price growth remain unclear. Analyzing corresponding trends in revenue can help understand these causes. This study seeks to assess changes over time in prices, patient counts, and drug-level revenues in the US market for oncology therapies and to investigate whether price growth is driven by an increased ability by pharmaceutical firms to capture profits., Study Design: Nineteen-year retrospective study (1997-2015)., Methods: We used panel regression to investigate trends in prices, patient counts, and revenues within a US national data set consisting of targeted oncology therapies launched in different eras., Results: We find that prices have roughly tripled, whereas average patient counts per therapy have fallen by 85% to 90% over this period. However, the entire distribution of annual revenues has fallen: For instance, median revenues for drugs launched in the early 2010s are about half of what they were for drugs launched in the late 1990s., Conclusions: Future research on the causes of quantity decline can help inform pharmaceutical policy.

In the United States, osteoporosis affects over 10 million adults, has high societal costs ($22 billion in 2008), and is currently being underdiagnosed and undertreated. Given an aging population, this burden is expected to rise. We projected the fracture burden in US women by modeling the expected demographic shift as well as potential policy changes. With the anticipated population aging and growth, annual fractures are projected to increase from 1.9 million to 3.2 million (68%), from 2018 to 2040, with related costs rising from $57 billion to over $95 billion. Policy-driven expansion of case finding and treatment of at-risk women could lower this burden, preventing 6.1 million fractures over the next 22 years while reducing payer costs by $29 billion and societal costs by $55 billion. Increasing use of osteoporosis-related interventions can reduce fractures and result in substantial cost-savings, a rare and fortunate combination given the current landscape in healthcare policy. © 2019 The Authors. JBMR Plus published by Wiley Periodicals, Inc. on behalf of American Society for Bone and Mineral Research., (© 2019 The Authors. JBMR Plus published by Wiley Periodicals, Inc. on behalf of American Society for Bone and Mineral Research.)

Background: The objective of this study was to conduct an indirect treatment comparison between cabazitaxel, abiraterone and enzalutamide to determine the clinical efficacy and safety of cabazitaxel relative to comparators in the treatment of patients with metastatic castrate-resistant prostate cancer who progress on docetaxel-based therapies., Methods: A systematic literature review was conducted to inform the network meta-analysis of cabazitaxel, abiraterone and enzalutamide. Due to a lack of head-to-head trials, studies with a comparator arm of best supportive care were included in the analysis. Overall survival, progression-free survival, and adverse events were compared within both Bayesian and Frequentist frameworks. The ratios for survival outcomes were estimated using hazard ratios (HR), and the ratios for adverse events between groups were estimated using odds ratios (ORs); uncertainty was reported as 95% confidence (Frequentist) and credible (Baysesian) Intervals., Results: Three of thirteen trials identified for abstraction were relevant for analyses. Median overall survival was not statistically significantly different for abiraterone (HR = 1.04; 95% CI = 0.83-1.28) or enzalutamide (HR = 0.88; 95% CI = 0.69-1.11) when compared to cabazitaxel in the Bayesian analysis. Anaemia (OR = 3.71; 95% CI = 1.01-10.44), diarrhoea (OR = 16.60; 95% CI = 1.41-75.31) and haematuria (OR = 3.88; 95% CI = 1.03-10.09) were more likely to occur in the cabazitaxel group than the abiraterone group, while pyrexia risk was higher in cabazitaxel compared to enzalutamide (OR = 36.23; 95% CI = 1.14-206.40). Frequentist analyses produced similar results., Conclusions: The scarcity of clinical studies and lack of a common comparator limited analyses. The adverse event results must be interpreted with caution as many were based on small numbers. The results from this analysis indicate comparable survival outcomes and adverse event profiles. As these pivotal studies may not reflect the contemporary treatment landscape and patient profiles, additional research, including head-to-head clinical trials and real world observational studies, should be conducted to further elucidate the beneficial effects of these therapies.

Objective: Prostate cancer is a highly prevalent form of cancer in older men and is one of the leading causes of death from cancer in men across the globe. Many therapeutic agents have been approved for patients with metastatic castration-resistant prostate cancer (mCRPC), particularly as a post-docetaxel treatment strategy. The objective of this systematic literature review was to assess published efficacy and safety data for select mCRPC therapies - such as abiraterone, cabazitaxel, and enzalutamide - in the post-docetaxel setting., Methods: Database searches of MEDLINE, Embase, and Cochrane CENTRAL, in conjunction with hand searches of multiple congress abstracts, yielded 13 randomized studies and 107 non-randomized studies that met the inclusion criteria., Results: Randomized studies demonstrated significant improvements in median overall survival (OS) outcomes over placebo for abiraterone (15.8 vs. 11.2 months) and enzalutamide (18.4 vs. 13.6 months), and similar significant improvements were noted for cabazitaxel over mitoxantrone (15.1 vs. 12.7 months). Differences in progression-free survival (PFS) were similarly significant, although variance in the criteria for measuring PFS may limit the extent to which these outcomes can be compared between studies. Non-randomized evidence included multiple publications from several early access and compassionate use programs with a primary objective to report safety outcomes. Results from these studies largely reflected the findings in randomized trials., Conclusions: Overall, there is a growing body of evidence for post-docetaxel treatment options available in patients with mCRPC. Further head-to-head trials or indirect treatment comparisons may be a valuable method to assess the comparative efficacy of these therapies.

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

Objectives: To provide a comprehensive estimate of the total social value (TSV) delivered by granulocyte-colony stimulating factor (G-CSF) therapies in the United States in 2014., Study Design: Estimation of the TSV of G-CSF, based on a targeted literature review of pivotal studies., Methods: A literature review was conducted to obtain estimates of the adverse outcomes associated with myelosuppressive chemotherapy-induced febrile neutropenia (FN) and the positive impacts of G-CSFs. We monetized each outcome into a set of mutually exclusive value components that were aggregated to estimate the TSV. To estimate the share of TSV captured by manufacturers, we estimated 2014 profits from G-CSF using measures of industry revenues and operating costs., Results: In 2014, approximately 314,440 patients received G-CSFs. Compared with what they would have experienced without G-CSFs, these patients were less likely to be hospitalized or die from FN, incur reductions in chemotherapy relative dose intensity, receive antibiotics, miss work, or experience reduced health-related quality of life. We estimated the social value from fewer FN hospitalizations to be $770 million; from fewer FN-related deaths, $2.65 billion; from fewer deaths due to higher effective chemotherapy doses, $4.83 billion; from reductions in antibiotics, $2.3 million; from reductions in indirect costs, $230 million; and from improvements in health-related quality of life, $1.9 million. The estimated 2014 US TSV of G-CSFs was $8.5 billion. Industry profits associated with G-CSFs were estimated at $1.3 billion, accounting for approximately 15% of the TSV., Conclusions: Based on our calculations, the TSV generated by G-CSFs in the United States in 2014 was substantial, with the majority of this value accruing to patients.

Background: Psoriasis has significant economic impact on patients. However, its total economic burden has not been fully quantified., Objectives: To assess the annual economic burden of psoriasis in the United States., Methods: A systematic literature review was conducted to obtain estimates of the components of the economic burden of psoriasis. Prevalence estimates were used to estimate the 2013 psoriasis population. Incremental medical costs were calculated based on studies that compared psoriasis patients and controls. Productivity loss was estimated using measures of presenteeism, absenteeism, and unemployment. Reductions in health-related quality of life (HRQOL) were calculated from survey responses., Results: The prevalence of psoriasis in the US was estimated to be 7.4 million in 2013. Comparatively, psoriasis patients incurred incremental medical costs of $2284, experienced a $2203 reduction in HRQOL, and a $1935 reduction in productivity. The total burden of psoriasis was estimated as $35.2 billion, with $12.2 billion in incremental medical costs (35%), $11.8 billion from reduced HRQOL (34%), and $11.2 billion from productivity losses (32%)., Limitations: This study is constrained by the scope and populations of the existing literature., Conclusions: The economic burden of psoriasis in the US is significant, with a majority of it coming from indirect costs., (Copyright © 2015 American Academy of Dermatology, Inc. Published by Elsevier Inc. All rights reserved.)

The BRICS countries (Brazil, Russia, India, China, and South Africa) have experienced tremendous economic and health gains in recent decades. Two of the major health challenges faced by the BRICS and other low and middle income countries are decreasing inequity in health outcomes and increasing affordability of health insurance. One fiscally sustainable option for the BRICS governments is a public subsidy system for private health insurance plans. This essay lays out the potential applicability and impacts of public subsidies for private health insurance plans, as well as opportunities and challenges for implementation, in the BRICS countries. Overall, providing public subsidies rather than health insurance would enable the BRICS governments to avoid the open-ended financial liabilities that have plagued advanced economies, while still expanding access to health insurance and encouraging the develoment of a robust private health insurance market. We conclude by suggesting an array of pilot programs that could serve as the seeds for publicly subsidized health insurance schemes within the BRICS markets.

Background: Nutrition deficiency is common among hospitalized children. Although oral nutrition supplements (ONS) may improve malnutrition in this population, the benefits and healthcare costs associated with their use have not yet been fully explored. The objective of this study was to assess the effect of ONS use on inpatient length of stay (LOS) and episode cost in hospitalized children., Materials and Methods: Retrospective analysis of 557,348 hospitalizations of children aged 2-8 years in the Premier Research Database. The effect of ONS use on LOS and episode cost in a propensity score- matched sample was estimated in analyses with and without the use of instrumental variables (IVs) to reduce confounding from unobserved variables., Results: ONS were prescribed in 6066 of 557,348 inpatient episodes (1.09%). In IV analysis, using a matched sample of 11,031 episodes, hospitalizations with ONS use had 14.8% shorter LOS (6.4 vs 7.5 days; 1.1 days [95% CI, 0.2-2.4]). Hospitalizations with ONS use had 9.7% lower cost ($16,552 vs $18,320; $1768 [95% CI, $1924-$1612])., Conclusions: ONS use was associated with lower LOS and episode cost among pediatric inpatients. ONS use in hospitalized pediatric patients may provide a cost-effective, evidence-based approach to improving pediatric hospital care., (© 2014 Abbott Nutrition.)

With the implementation of the Affordable Care Act (ACA) under way, some policy makers have questioned the continued relevance of the Ryan White HIV/AIDS Program as a safety net for people living with HIV/AIDS. We surveyed HIV care providers to understand the role of the Ryan White Program and to identify concerns regarding the ACA implementation. We also addressed whether the program is still relevant after ACA implementation and, if so, what elements should be retained. We found that providers consider the Ryan White Program to be critical in facilitating high-quality care for people living with HIV/AIDS. Most of the providers highlighted the program's support for providing medical and nonmedical case management as especially valuable and important to the entire continuum of care and for all patient subpopulations. Whether care is supplied by the Ryan White Program, Medicaid, or other means, our findings suggest that case management services will remain critical in treating HIV/AIDS as the health care landscape continues to evolve.