Michael S. Broder

Validity and Reliability of Value Assessment Frameworks for New Cancer Drugs

BACKGROUNDnSeveral organizations have developed frameworks to systematically assess the value of new drugs. These organizations include the American Society of Clinical Oncology (ASCO), the European Society for Medical Oncology (ESMO), the Institute for Clinical and Economic Review (ICER), and the National Comprehensive Cancer Network (NCCN).nnnOBJECTIVESnTo understand the extent to which these four tools can facilitate value-based treatment decisions in oncology.nnnMETHODSnIn this pilot study, eight panelists conducted value assessments of five advanced lung cancer drugs using the ASCO, ESMO, and ICER frameworks. The panelists received instructions and published clinical data required to complete the assessments. Published NCCN framework scores were abstracted. The Kendalls W coefficient was used to measure convergent validity among the four frameworks. Intraclass correlation coefficients were used to measure inter-rater reliability among the ASCO, ESMO, and ICER frameworks. Sensitivity analyses were conducted.nnnRESULTSnDrugs were ranked similarly by the four frameworks, with Kendalls W of 0.703 (P = 0.006) across all the four frameworks. Pairwise, Kendalls W was the highest for ESMO-ICER (W = 0.974; P = 0.007) and ASCO-NCCN (W = 0.944; P = 0.022) and the lowest for ICER-NCCN (W = 0.647; P = 0.315) and ESMO-NCCN (W = 0.611; P = 0.360). Intraclass correlation coefficients (confidence interval [CI]) for the ASCO, ESMO, and ICER frameworks were 0.786 (95% CI 0.517-0.970), 0.804 (95% CI 0.545-0.973), and 0.281 (95% CI 0.055-0.799), respectively. When scores were rescaled to 0 to 100, the ICER framework provided the narrowest band of scores.nnnCONCLUSIONSnThe ASCO, ESMO, ICER, and NCCN frameworks demonstrated convergent validity, despite differences in conceptual approaches used. The ASCO inter-rater reliability was high, although potentially at the cost of user burden. The ICER inter-rater reliability was poor, possibly because of its failure to distinguish differential value among the sample of drugs tested. Refinements of all frameworks should continue on the basis of further testing and stakeholder feedback.

Hospital cost and length of stay in idiopathic pulmonary fibrosis

Abstract Objective: To provide a detailed picture of the economic impact of hospitalization in idiopathic pulmonary fibrosis (IPF) and to identify factors associated with cost and length of stay (LOS). Methods: In this retrospective cross-sectional study using the Nationwide Inpatient Sample (NIS), this study included hospitalizations for IPF (ICD-9-CM 516.3) with a principal diagnosis of respiratory disease (ICD-9-CM 460-519) from 2009–2011; lung transplant admissions were excluded. Total inpatient cost, LOS, in-hospital death, and discharge disposition were reported. Linear regression models were used to determine variables predictive of LOS and cost. Results: From 2009–2011, 22,350 non-transplant IPF patients with a principal diagnosis of respiratory disease were admitted: mean (±SE) age was 70.0 (0.32), and 49.1% were female. While in hospital, 11.4% of patients received mechanical ventilation and 8.9% received non-invasive ventilation. Mean (±SE) LOS was 7.4 (0.15) days overall (pu2009<u2009.001). The mean (±SD) admission cost was

Measuring the Value of New Drugs: Validity and Reliability of 4 Value Assessment Frameworks in the Oncology Setting

16,042 (±631). Of hospitalized patients, 14.1% died, 20.6% transferred facilities, and 46.4% were routinely discharged. The adjusted LOS (95% CI) for patients with and without mechanical ventilation was 16.1 days (15–17.5) vs. 6.3 (6–6.5); adjusted costs were

The Incidence and Health Care Resource Burden of the Myelodysplastic Syndromes in Patients in Whom First‐Line Hypomethylating Agents Fail

48,772 (43,979–53,565) vs.

Mechanical ventilation in idiopathic pulmonary fibrosis: a nationwide analysis of ventilator use, outcomes, and resource burden

11,861 (11,292–12,431). Limitations: The positive predictive value of the algorithm used to identify IPF is not optimal. The NIS database does not follow patients longitudinally, and claims after admission are not available. Claims do not indicate whether listed diagnoses were present on admission or developed during hospitalization. The exclusion of transplant-related expenditures lead to under-estimation of cost. Conclusion: Using a nationally-representative database, we found IPF respiratory-related hospitalizations represent a significant economic burden with ∼7,000 non-transplant IPF admissions per year, at a mean cost of

0812 Disease Burden in Pediatric Narcolepsy: A Claims-based Analysis of Healthcare Utilization and Costs, and Medical Comorbidity

16,000 per admission. Mechanical ventilation is associated with statistically significant increases in LOS and cost. Therapeutic advances that reduce rates and costs of IPF hospitalizations are needed.

Mortality Risk of Patients With Idiopathic Pulmonary Fibrosis

BACKGROUNDnSeveral organizations have developed frameworks to systematically assess the value of new drugs.nnnOBJECTIVEnTo evaluate the convergent validity and interrater reliability of 4 value frameworks to understand the extent to which these tools can facilitate value-based treatment decisions in oncology.nnnMETHODSnEight panelists used the American Society of Clinical Oncology (ASCO), European Society for Medical Oncology (ESMO), Institute for Clinical and Economic Review (ICER), and National Comprehensive Cancer Network (NCCN) frameworks to conduct value assessments of 15 drugs for advanced lung and breast cancers and castration-refractory prostate cancer. Panelists received instructions and published clinical data required to complete the assessments, assigning each drug a numeric or letter score. Kendalls Coefficient of Concordance for Ranks (Kendalls W) was used to measure convergent validity by cancer type among the 4 frameworks. Intraclass correlation coefficients (ICCs) were used to measure interrater reliability for each framework across cancers. Panelists were surveyed on their experiences.nnnRESULTSnKendalls W across all 4 frameworks for breast, lung, and prostate cancer drugs was 0.560 (P= 0.010), 0.562 (P = 0.010), and 0.920 (P < 0.001), respectively. Pairwise, Kendalls W for breast cancer drugs was highest for ESMO-ICER and ICER-NCCN (W = 0.950, P = 0.019 for both pairs) and lowest for ASCO-NCCN (W = 0.300, P = 0.748). For lung cancer drugs, W was highest pairwise for ESMO-ICER (W = 0.974, P = 0.007) and lowest for ASCO-NCCN (W = 0.218, P = 0.839); for prostate cancer drugs, pairwise W was highest for ICER-NCCN (W = 1.000, P < 0.001) and lowest for ESMO-ICER and ESMO-NCCN (W = 0.900, P = 0.052 for both pairs). When ranking drugs on distinct framework subdomains, Kendalls W among breast cancer drugs was highest for certainty (ICER, NCCN: W = 0.908, P = 0.046) and lowest for clinical benefit (ASCO, ESMO, NCCN: W = 0.345, P = 0.436). Among lung cancer drugs, W was highest for toxicity (ASCO, ESMO, NCCN: W = 0. 944, P < 0.001) and lowest for certainty (ICER, NCCN: W = 0.230, P = 0.827); and among prostate cancer drugs, it was highest for quality of life (ASCO, ESMO: W = 0.986, P = 0.003) and lowest for toxicity (ASCO, ESMO, NCCN: W = 0.200, P = 0.711). ICC (95% CI) for ASCO, ESMO, ICER, and NCCN were 0.800 (0.660-0.913), 0.818 (0.686-0.921), 0.652 (0.466-0.834), and 0.153 (0.045-0.371), respectively. When scores were rescaled to 0-100, NCCN provided the narrowest band of scores. When asked about their experiences using the ASCO, ESMO, ICER, and NCCN frameworks, panelists generally agreed that the frameworks were logically organized and reasonably easy to use, with NCCN rated somewhat easier.nnnCONCLUSIONSnConvergent validity among the ASCO, ESMO, ICER, and NCCN frameworks was fair to excellent, increasing with clinical benefit subdomain concordance and simplicity of drug trial data. Interrater reliability, highest for ASCO and ESMO, improved with clarity of instructions and specificity of score definitions. Continued use, analyses, and refinements of these frameworks will bring us closer to the ultimate goal of using value-based treatment decisions to improve patient care and outcomes.nnnDISCLOSURESnThis work was funded by Eisai Inc. Copher and Knoth are employees of Eisai Inc. Bentley, Lee, Zambrano, and Broder are employees of Partnership for Health Analytic Research, a health services research company paid by Eisai Inc. to conduct this research. For this study, Cohen, Huynh, and Neville report fees from Partnership for Health Analytic Research. Outside of this study, Cohen receives grants and direct consulting fees from various companies that manufacture and market pharmaceuticals. Mei reports a grant from Eisai Inc. during this study. The other authors have no disclosures to report. Study concept and design were contributed by Bentley and Broder, with assistance from Elkin and Cohen. Bentley took the lead in data collection, along with Elkin, Huynh, Mukherjea, Neville, Mei, Popescu, Lee, and Zambrano. Data interpretation was performed by Bentley and Broder, along with Elkin, Cohen, Copher, and Knoth. The manuscript was written primarily by Bentley, along with Elkin and Broder, and revised by Bentley, Broder, Elkin, Cohen, Copher, and Knoth. Select components of this works methods were presented at ISPOR 19th Annual European Congress held in Vienna, Austria, October 29-November 2, 2016, and Society for Medical Decision Making 38th Annual North American Meeting held in Vancouver, Canada, October 23-26, 2016.

Longitudinal Changes in Comorbidity Rates in Patients With Idiopathic Pulmonary Fibrosis: Analysis of Medicare Data

BACKGROUNDnAlthough hypomethylating agents (HMAs) are effective and approved therapies for patients with myelodysplastic syndromes (MDS), many patients do not benefit from treatment, and nearly all ultimately stop responding to HMAs. The incidence and cost burden of HMA failure are unknown yet needed to appreciate the magnitude and significance of such failure.nnnMETHODSnWe analyzed a de-identified dataset of over 5 million individuals with private health insurance in the U.S. to estimate MDS incidence, prevalence, and treatments. Based on MDS provider interviews, a conceptual model of MDS patient management was constructed to create a new, claims-relevant and drug development-relevant definition of HMA treatment failure. This algorithm was used to define resource encumbrance of MDS patients in whom HMA treatment failed.nnnRESULTSnWe estimated an MDS incidence rate of ∼70 cases per 100,000 enrollees per year and a prevalence of 155 cases per 100,000 enrollees. The proportion of MDS patients receiving HMA treatment was low (∼3%), and treatment was typically initiated within 1 year of the first MDS claim. Notably, HMA-treated individuals were older and had more comorbidities than the overall MDS cohort. Total health care costs of managing MDS patients after HMA failure were high (∼

In-Hospital Length of Stay and Mortality for Patients With Idiopathic Pulmonary Fibrosis (IPF) in the US

77,000 during the first 6 months) and were driven primarily by non-pharmacy costs.nnnCONCLUSIONnThis study quantifies for the first time the burden of significant unmet need in caring for MDS patients following HMA treatment failure. The Oncologist 2017;22:379-385Implications for Practice: U.S.-based treatment patterns among MDS patients demonstrate the significant clinical, financial, and health care burden associated with HMA failure and call for active therapies for this patient population.

Annual Health Care Utilization And Costs In Cushing's Disease Patients In The United States

BackgroundIdiopathic pulmonary fibrosis (IPF) is associated with increased risk of respiratory-related hospitalizations. Studies suggest mechanical ventilation (MV) use in IPF does not improve outcomes and guidelines recommend against its general use. Our objective was to investigate MV use and association with cost and mortality in IPF.MethodsThis retrospective study, using a nationwide sample, included claims with IPF (ICD-9-CM: 516.3) in 2009–2011 and principal respiratory disease diagnosis (ICD-9-CM: 460–519); excluding lung transplant. Regression models were used to determine predictors of MV and association with cost, LOS, and mortality. Domain analysis was used to account for use of subpopulation. Costs were adjusted to 2011. Data on patient severity not available.ResultsTwenty two thousand three hundred fifty non-transplant IPF patients were admitted with principal respiratory disease diagnosis: Mean age 70.0 (SD 13.9), 49.1% female, mean LOS 7.4 (SD 8.2). MV was used in 11.4% of patients with a non-significant decline over time. In regression models, MV was associated with an increased stay of 9.78xa0days (95% CI 8.38–11.18) and increased cost of