Competencies for Professionals in Health Economics and Outcomes Research: The ISPOR Health Economics and Outcomes Research Competencies Framework.

The need for Health Economics and Outcomes Research (HEOR) has expanded globally, fueling demand for professionals trained in the discipline. By leveraging the expertise and perspectives of its members, the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) established a set of competencies for HEOR professionals. The resulting 41 competencies were organized into 13 topic domains that collectively comprise the ISPOR Health Economics and Outcomes Research Competencies Framework. In this article, we explain the collaborative process used by the ISPOR Institutional Council and Faculty Advisor Council to identify and validate the framework. This process entailed expertise from the council members combined with natural language processing to examine competencies included in ISPOR Career Center HEOR job postings, qualitative input from a focused Institutional Council-Faculty Advisor Council workgroup, and quantitative input from 3 surveys of mutually exclusive member groups: a general member survey to assess importance and relevance of each competency, a faculty member survey to assess the extent to which HEOR graduate degree programs cover each of the competencies, and a student member survey to assess exposure to each of the competencies. Organization of the competencies into topic domains was achieved by engaging the Education Council, which applied a taxonomy consistent with ISPOR's educational programming. The resulting ISPOR Health Economics and Outcomes Research Competencies Framework has the important potential of serving as a tool to guide academic curricula, fellowships, and continuing education programs, and assessment of job candidates. As the HEOR field advances, so do the job types and the breadth of topics in which professionals must demonstrate competence. Future work will entail revisiting the competencies to ensure their currency and comprehensiveness, and tailoring the framework according to major specialty areas.

Comparative Effectiveness of Proton Pump Inhibitors vs Histamine Type 2 Receptor Blockers for Preventing Clinically Important Gastrointestinal Bleeding During Intensive Care: A Population-Based Study.

BACKGROUND: Proton pump inhibitors (PPIs) and histamine type 2 receptor blockers (H2Bs) are used for stress ulcer prophylaxis. Although the PPIs have greater potency for acid suppression, their relative effectiveness for preventing clinically important GI bleeding (CIGIB) has not been established. The goal of this study was to determine whether prophylactic PPIs were associated with lower risk of CIGIB than H2Bs among critically ill adults. METHODS: This retrospective cohort study included adults with critical illness from January 1, 2008, to June 30, 2012, who had at least one stress ulcer risk factor and received a PPI or H2B for ≥ 3 days. Cox proportional hazards regression propensity score matching and instrumental variable analyses were used to control for selection bias and confounding by unmeasured factors. The Acute Physiology and Chronic Health Evaluation Score version IV score was used to adjust for differences of acuity. The main outcome and exposure was CIGIB. RESULTS: Among 70,093 patients at risk, 49,576 (70.7%) received prophylaxis for at least 3 days, and 424 patients (0.6%) met the definition for experiencing CIGIB. The hazard for CIGIB was two times greater for PPI users compared with H2B users (adjusted hazard ratio, 1.82 [95% CI, 1.19-2.78]; hazard ratio, 2.37 [95% CI, 1.61-3.5]). Sensitivity analyses failed to detect any plausible scenario in which PPIs were superior to H2Bs for the prevention of CIGIB. CONCLUSIONS: H2Bs were robustly and consistently associated with significantly lower CIGIB risk compared with PPIs in this population.

Tree-based Claims Algorithm for Measuring Pretreatment Quality of Care in Medicare Disabled Hepatitis C Patients.

BACKGROUND: To help broaden the use of machine-learning approaches in health services research, we provide an easy-to-follow framework on the implementation of random forests and apply it to identify quality of care (QC) patterns correlated with treatment receipt among Medicare disabled patients with hepatitis C virus (HCV). METHODS: Using Medicare claims 2006-2009, we identified 1936 patients with 6 months continuous enrollment before HCV diagnosis. We ran a random forest on 14 pretreatment QC indicators, extracted the forest's representative tree, and aggregated its terminal nodes into 4 QC groups predictive of treatment. To explore determinants of differential QC receipt, we compared patient-level and county-level (linked AHRF data) characteristics across QC groups. RESULTS: The strongest predictors of treatment included "liver biopsy," "HCV genotype testing," "specialist visit," "HCV viremia confirmation," and "iron overload testing." High QC [n=360, proportion treated (pt)=33.3%] was defined for patients with at least 2 from the above-mentioned metrics. Good QC patients (n=302, pt=12.3%) had either "HCV genotype testing" or "specialist visit," whereas fair QC (n=282, pt=7.1%) only had "HCV viremia confirmation." Low QC patients (n=992, pt=2.5%) had none of the selected metrics. The algorithm accuracy of predicting treatment was 70% sensitivity and 78% specificity. HIV coinfection, drug abuse, and residence in counties with higher supply of hospitals with immunization and AIDS services correlated with lower QC. CONCLUSIONS: Machine-learning techniques could be useful in exploring patterns of care. Among Medicare disabled HCV patients, the receipt of more QC indicators was associated with higher treatment rates. Future research is needed to assess determinants of differential QC receipt.

Determinants of quality of care and treatment initiation in Medicare disabled patients with chronic hepatitis C.

BACKGROUND: Aligning with a national priority to bridge health disparities in disadvantaged populations, we explored contextual determinants of pretreatment quality of care and treatment receipt of Medicare disabled patients with hepatitis C virus (HCV) infection. METHODS: We used Medicare claims (2006-2009) linked to the Area Health Resource Files. Ordinal partial proportional odds and weighted modified Poisson regressions were used to model the determinants of quality care receipt and interferon-based treatment, respectively. RESULTS: We identified 1936 Medicare disabled HCV patients, of whom 10.4% were treated with peg-interferon. Despite the high comorbidity burden among HCV disabled patients, greater engagement in care correlated with greater likelihood of quality care and treatment receipt. CONCLUSION: Our study highlights the need for process and linkage to care in Medicare disabled HCV patients, but future research relevant to novel interferon-free agents is needed to assess patterns of quality of care and treatment receipt in this vulnerable population.

Comparative effectiveness of different chemotherapeutic regimens on survival of people aged 66 and older with stage III colon cancer: a "real world" analysis using Surveillance, Epidemiology, and End Results-Medicare data.

OBJECTIVES: To compare the effectiveness and utilization trends of irinotecan (IRI)-based and oxaliplatin (OX)-based regimens with those of 5-fluorouracil and leucovorin (5FU/LV) alone in people aged 66 and older with Stage III colon cancer. DESIGN: Retrospective cohort study. SETTING: Data from the Surveillance, Epidemiology, and End Results (SEER)-Medicare data. PARTICIPANTS: People with Stage III surgically resected colon cancer who received adjuvant chemotherapy were categorized into 5FU/LV-alone (n = 3,581), OX-based regimen (n = 814), and IRI-based regimen (n = 219) subgroups. MEASUREMENTS: Multivariable Cox proportional hazards models examined the effect of chemotherapies on overall survival, colon cancer-specific survival, and non-colon cancer-specific survival. RESULTS: Use of the OX-based regimen increased, and use of the 5FU/LV-alone and IRI-based regimens decreased over time. OX was statistically significantly associated with longer overall survival (hazard ratio (HR) = 0.73, 95% confidence interval (CI) = 0.62-0.86, P < .001) and colorectal cancer-specific survival (HR = 0.39, 95% CI, 0.28-0.55, P < .001) than 5FU/LV alone. There was a greater risk of overall mortality (HR = 1.38, 95% CI = 1.14-1.67, P<.001) and cancer-specific mortality (HR = 1.92, 95% CI=1.49-2.47, P < .001) associated with IRI than with 5FU/LV. The superiority of OX on survival was found in participants aged 66 to 79 but not in those aged 80 and older. CONCLUSION: This "real world" comparative effectiveness research extends randomized controlled trial results by documenting the relative survival benefit of OX in older adults with Stage III colon cancer. The associated shift in treatment away from 5FU/LV alone or IRI toward OX is consistent with evidence-based medicine from real-world outcomes research.

Trends in disparities in receipt of adjuvant therapy for elderly stage III colon cancer patients: the role of the medical oncologist evaluation.

BACKGROUND: Race disparities in adjuvant chemotherapy for stage III colon cancer patients have been documented, and medical oncologist evaluation is a critical step in the treatment process. Recent healthcare system and environmental changes may have reduced treatment gaps. OBJECTIVES: To examine differential rates of oncologist evaluation and conditional treatment, by race, and to determine whether changing evaluation and treatment patterns reduced disparities. RESEARCH DESIGN: Retrospective analysis of Surveillance Epidemiology and End Results-Medicare registry, enrollment, and claims data. SUBJECTS: Patients age >65, white or African American race, diagnosed with American Joint Committee on Cancer stage III colon cancer between 1997 and 2002. N = 7176. KEY MEASURES: Oncology specialty evaluation and management visit or chemotherapy claim; receipt of 5-fluorouracil based chemotherapy. Time periods are grouped into early (1997-1998), middle (1999-2000), and late (2001-2002). RESULTS: Initial adjusted oncologist evaluation rates were higher for whites compared with African American patients (58.7% vs. 42.9%), but changes over time reduced the race gap substantially. We did not find significant race-time trends in treatment rates conditional on oncologist evaluation. CONCLUSIONS: Race disparities in medical oncologist evaluations diminished over time, possibly in response to increased provider supply or changing patient and provider attitudes, but there was no parallel reduction in disparities in conditional treatment rates. Projected decreases in oncologist supply suggest the need for further research on this relationship. Research on the role of supplemental medical insurance on disparities in treatment is needed, particularly as the cost of recommended adjuvant therapy increases.

Effect of age on survival benefit of adjuvant chemotherapy in elderly patients with Stage III colon cancer.

OBJECTIVES: To estimate the modifying effect of age on the survival benefit associated with adjuvant chemotherapy receipt in elderly patients with a diagnosis of Stage III colon cancer. DESIGN: Observational, retrospective cohort study using two samples: an overall sample of 7,182 patients to provide externally valid analyses and a propensity score-matched sample of 3,016 patients to provide more internally valid analyses by reducing the presence of treatment endogeneity. An interval-censored survival model with a complementary log-log link was used. Hazard ratios and 95% confidence intervals were obtained for all regressions. SETTINGS: Data from the National Cancer Institute's Surveillance, Epidemiology and End Results database and the linked Medicare enrollment and claims database were used. PARTICIPANTS: Selected patients were aged 66 and older and had a diagnosis of Stage III colon cancer. Patients were followed from surgery to time of death or censorship. MEASUREMENTS: The outcome was colon cancer-specific death during the follow-up period. Receipt of adjuvant chemotherapy was measured according to the presence of a claim for 5-fluorouracil or leucovorin within 6 months after surgery. RESULTS: All elderly patients had a significant survival benefit associated with adjuvant chemotherapy receipt, although the survival benefit of adjuvant chemotherapy was not uniform across all age groups. CONCLUSION: These findings have important clinical and policy implications for the risk-benefit calculation induced by treatment in older patients with Stage III colon cancer. The results suggest that there is a benefit from chemotherapy, but the benefit is lower with older age.

Ethnic disparities, hospital quality, and discharges against medical advice among patients with cardiovascular disease.

BACKGROUND: Nationally, cardiovascular disease is the third-ranked disease category in terms of discharges against medical advice (AMA). Disparities in discharges AMA have not been examined among patients with cardiovascular disease, nor has the moderating role of hospital quality been studied. METHODS: We examined the dual effect of race/ethnicity and hospital quality on discharges AMA by retrospectively analyzing hospital discharge data of patients who were admitted with a primary diagnosis of cardiovascular disease from 2000 through 2005. RESULTS: A total of 2619 of the 312,183 hospitalizations for cardiovascular disease (.8%) resulted in a discharge AMA. The sample was 50% male, 32% non-White, and an average age of 68 years of age. Non-White race was associated with a higher probability of a discharge AMA in a high-quality hospital (adjusted odds ratio [AOR] 1.2, P < .001). Non-White race/ethnicity was associated with a lower probability of a discharge AMA in a low-quality hospital (AOR .8, P = .01). A discharge AMA was less likely at a high-quality hospital (AOR .7, P < .001), regardless of race/ethnicity. The modifying effect of hospital quality is more apparent at the highest levels of hospital quality. CONCLUSIONS: Hospital quality is negatively correlated with discharges AMA and moderates the relationship between race/ethnicity and discharges AMA.

The total economic burden of overactive bladder in the United States: a disease-specific approach.

OBJECTIVE: To employ results from a recent US population-based survey to calculate disease-specific total costs of overactive bladder (OAB). STUDY DESIGN: Disease-specific total cost-of-illness method using population prevalence estimates. METHODS: Cases were identified as community-dwelling adults reporting the presence of urinary urgency or urgency urinary incontinence. Two OAB classifications were used based on Likert scale responses of OAB symptoms: "often" (base case) or "sometimes" (alternative). The study estimates disease-specific total costs of OAB from the societal perspective and using an average costing method. A population-based survey, a claims data analysis, and the published literature provided the prevalence and resource utilization data. RESULTS: The prevalence of OAB as defined in the base case (alternative) was 18.6% (28.7%) in the adult US population, accounting for 42.2 million (65.1 million) community-dwelling adults. The disease-specific total cost of OAB is estimated at $24.9 billion for the base case and $36.5 billion for the alternative case. Total direct costs were $22.3 billion in the base case and $33.5 billion in the alternative case. Costs were higher among adults younger than 65 years of age, compared with adults 65 years or older. This relative cost burden was lower for the base case compared with the alternative case in the full sample, with a larger gap among men. CONCLUSION: The total cost of OAB among community-dwelling adults is significant and varies with demographic groups. Future research is needed to determine whether the differential cost burden is robust to alternate cost-of-illness estimation methods.

Using cost-effectiveness analysis to sharpen formulary decision-making: the example of tiotropium at the Veterans Affairs health care system.

OBJECTIVE: To identify a cost-saving subset of criteria for the use of tiotropium at a Veterans Affairs Medical Center based on a cost-effectiveness analysis with ipratropium as the comparator. METHODS: Retrospective analysis of electronic medical records for the calendar year 2004 was conducted. The sample was drawn from a population at the Baltimore Veterans Affairs Medical Center that had a confirmed diagnosis of chronic obstructive pulmonary disease (COPD) and had filled prescriptions for ipratropium. The tiotropium sample was based on a modeled cohort of COPD patients who had received tiotropium. The analysis was conducted from the perspective of the Veterans Affairs Health Care System. The outcome was the incremental cost-effectiveness of tiotropium versus ipratropium. RESULTS: The incremental cost-effectiveness ratio (ICER) was $2360 per avoided exacerbation. Tiotropium cost-effectiveness increased with COPD severity and was cost-saving in patients with very severe disease (ICER = $-1818) and in patients with a previous COPD-related hospitalization (ICER = $-4472). The ICER was most sensitive to the relative effectiveness and price of tiotropium. Results identified the levels of treatment effectiveness and price beyond which tiotropium would become cost-saving relative to ipratropium. CONCLUSIONS: The results support the existing Veterans Affairs practice of offering tiotropium to patients with COPD-related hospitalizations. Periodic review of the effectiveness data to determine whether tiotropium would be cost-saving in patients with very severe COPD is suggested. Cost-effectiveness analyses that identify practical criteria-for-use should become an integral part of the formulary process.

Racial differences in hospital discharge disposition among stroke patients in Maryland.

OBJECTIVE: The objective of this retrospective study was to assess the evidence for racial differences in discharge disposition among patients hospitalized for stroke. DATA: Hospital discharge data from the Maryland Health Services Cost Review Commission were used in the analysis. The data covered the period from January 2000 to September 2003. STUDY DESIGN: Discharge-disposition categories were ordered such that higher numbers corresponded to less desirable outcomes: 1 = discharge to home; 2 = discharge to any medical care facility; 3 = death. We analyzed the influence of black race on the discharge disposition by estimating a partial proportional odds logit regression model that included demographic and clinical covariates. DATA EXTRACTION: The study inclusion criteria were 1) stroke (ICD9 431-434; 436-438) as a primary admission diagnosis and 2) patient race identified as black or white. Patients discharged against medical advice were excluded. The sample contained 51,564 stroke hospitalizations. PRINCIPAL FINDINGS: Based on the relative odds ratios (OR; 95% confidence interval [CI]), black males were more likely to be discharged to higher ranked (i.e., less desirable) discharge categories (OR = 1.66; CI 1.55-1.77) compared to white males. Black females were more likely to die (OR = 1.14; CI 1.02-1.28) and more likely either to die or to be discharged to medical care (OR = 1.38; CI 1.24-1.54) compared to white males. CONCLUSIONS: Blacks are at greater mortality risk following stroke hospitalizations and face less desirable discharge dispositions if they survive. These results are consistent with prior reports of lower survival rates among blacks and are robust to adjustments for various confounding factors.