Real-world antiseizure medication treatment outcomes in drug-resistant focal epilepsy patients.

OBJECTIVE: To gather real-world evidence on antiseizure medications (ASMs) treatment patterns and related outcomes in patients with drug-resistant focal epilepsy. METHODS: Medical insurance claims from the start of 2014 till the end of 2019 were used. Patient selection criteria included International Classification of Diseases (ICD) codes followed by documented ASM use. Baseline patient demographics along with ASM and rescue medication use patterns and related patient outcome were documented for first (index) ASM regimen. Patients who failed the first regimen and then failed the second regimen were considered drug resistant. Multivariate analyses were performed to identify risks and other characteristics for positive or negative treatment outcomes. RESULTS: Study cohort consisted of 46 474 patients with a mean age of 47.23 (SD: 16.94). Levetiracetam was the most first-encountered ASM (37.94%). At baseline, 87.14% were treated with ASMs prior to having study-confirmed diagnoses. Mental comorbidities were present in 37.86% of patients. After first-year ASM treatment, 34.61% of patients persisted on their index regimen and 5.91% were seizure-free. Patients failing first ASM regimen numbered 12 868 (27.69%). Drug-resistant patients who failed first and then second ASM regimens numbered 6335 (49.23%). Percentages of patients who had successful second treatment and seizure-free were 21.32 and 3.65, respectively. Initiating patients on lamotrigine or carbamazepine (relative to levetiracetam), baseline use of index ASM, rescue medications, and older age or male gender all lowered the risk for treatment failure. Having higher comorbidity, comorbid mental illness, headache, or neoplasty increased such a risk. Baseline use of index ASM, depressive episode, or anxiety disorder all entailed higher risk of failing second ASM treatment. SIGNIFICANCE: Overall, reported findings indicated that patient history at baseline and the early selection of an ASM all influenced treatment outcomes. Findings pointed to the complex nature of ASM treatment in drug-resistant focal epilepsy patients calling for additional research to identify the optimal treatment to achieve beneficial patient outcomes.

Treatment Patterns and Healthcare Resource Use in Medicare Beneficiaries with Parkinson's Disease.

Background: Studies on real-world treatment patterns and long-term economic burden of Parkinson's disease (PD) have been limited. Objective: To assess treatment patterns, healthcare resource utilization (HRU), and costs associated with PD symptoms and treatment-related adverse events (AEs) among Medicare beneficiaries in the United States. Methods: A 100% Medicare Fee-For-Service data (2006-2020) of patients with PD were analyzed. PD treatment patterns were described for the subset of patients who had no previously observed PD treatments or diagnoses (ie, the incident cohort). HRU and healthcare costs associated with PD symptoms were assessed for all patients with PD (ie, the overall cohort) and that associated with treatment-related AEs were assessed for the subset of patients who received PD treatments after PD diagnosis (ie, the active treatment cohort), using longitudinal models with repeated measures. Results: Overall, 318,582 patients were included (mean age at PD diagnosis: 77.4 years; 53.3% female). Among patients in the incident cohort (N=214,829), 51.1% initiated levodopa monotherapy and 5.9% initiated dopamine agonists (DAs) monotherapy as first-line treatment. The proportion of incident patients treated with DAs and other PD therapies generally increased from post-diagnosis years 1 to 10. The median time from diagnosis to PD treatment initiation was 2.0 months; the median time to treatment discontinuation was the longest with levodopa (18.7 months), followed by DAs (9.5 months). In the overall cohort, PD symptoms, especially motor symptoms and severe motor symptoms, were associated with significantly higher rates of HRU and costs. In the active treatment cohort (N=234,298), treatment-related AEs were associated with significantly higher rates of HRU and medical costs. Conclusion: While levodopa is still the mainstay of PD management, considerable heterogeneity exists in real-world treatment patterns. Overall, PD symptoms and AEs were associated with significantly higher HRU and healthcare costs, suggesting unmet medical needs for PD treatments with better tolerability profiles.

Work Loss and Direct and Indirect Costs Associated with Parkinson's Disease.

Purpose: To examine work loss and indirect costs during the three-year periods prior to and following initial diagnosis of Parkinson's disease (PD) in patients and in spouses of PD patients, as well as direct costs of healthcare. Patients and Methods: This is a retrospective, observational cohort study using the MarketScan Commercial and Health and Productivity Management databases. Results: A total of 286 employed PD patients and 153 employed spouses met all diagnostic and enrollment criteria for short-term disability (STD) analysis (PD Patient cohort and Caregiving Spouse cohort). The proportion of PD patients having a STD claim increased from roughly 5% and plateaued at around 12-14% starting in the year prior to first diagnosis of PD. The mean number of days lost from work due to STD per year increased from 1.4 days in the 3rd year prior to diagnosis to 8.6 days in the 3rd year after diagnosis (corresponding to an increase in indirect costs from $174 to $1104). STD use for spouses of patients with PD was lowest in the year after their spouses were diagnosed and then rose dramatically in the 2nd and 3rd years after the spouse's diagnosis. Total all-cause direct health-care costs increased during the years leading up to PD diagnosis and were highest in the years following diagnosis, with PD-related costs contributing ~20-30% of the total. Conclusion: PD has both a significant direct and indirect financial burden on patients and their spouses when analyzed for 3 years before and after diagnosis.

Dopamine agonist monotherapy utilization in patients with Parkinson's disease.

Objectives: To characterize patients with Parkinson's disease (PD) who initiated dopamine agonist (DA) monotherapy, describe medication utilization and provider types, and estimate medication adherence and discontinuation rates. Methods: Retrospective study identified patients with PD in the Optum Research Database and included those with ≥1 claim for DA or levodopa between 09/01/2012 and 12/31/2018, ≥2 PD diagnoses, commercial or Medicare Advantage Part D (MAPD) insurance, ≥40 years old, and continuous medical and pharmacy coverage ≥12 months before and after index date. A subset of patients receiving DA monotherapy was selected for this analysis. Variables were analyzed descriptively. Adherence was measured with medication possession ratio (MPR) and proportion of days covered (PDC); defined as ≥0.80. Results: Patients (N = 642) had mean (SD) age of 70.2 (9.9) years, 70.6 % had MAPD coverage, and 61.7 % were male. Neurologists prescribed 64.6 % of DA monotherapy, and 56.9 % of patients had ≥2 PD diagnoses before or on the index date. Index therapy was discontinued by 44.1 % of patients, and 55.9 % persisted for 12 months without change. Mean (SD) time to discontinuation was 102 (79) days. Mean (SD) MPR for patients (n = 562) with ≥2 fills was 0.84 (0.2); 70.3 % were MPR adherent. Mean (SD) PDC for all 642 patients was 0.66 (0.3); 50.5 % were PDC adherent. Conclusion: Adherence and continuation of therapy were suboptimal, which could translate into poor patient outcomes. Future studies could provide insights on the impact of low adherence and persistence with DA monotherapy.

Levodopa treatment patterns in Parkinson's disease: A retrospective chart review.

BACKGROUND: Medication regimens for Parkinson's disease (PD) may change as the disease progresses, symptoms fluctuate, or medication-related adverse events occur. This study evaluated treatment trends by observation year for patients initially receiving monotherapy with levodopa and a peripheral dopa decarboxylase inhibitor (PDDI). METHODS: In this retrospective chart review, therapy changes were evaluated for patients across the US diagnosed with PD on or before 6/30/2014 who initially received levodopa-PDDI monotherapy. Index date was the first clinic visit. Post-index was any time between the first 31 days after index and study end (6/30/2019). Index Hoehn-Yahr (H-Y) score and medication changes were also analyzed by index low (<400 mg/day) or high (≥400 mg/day) levodopa doses in the levodopa-PDDI combinations. RESULTS: In the levodopa-PDDI cohort (n = 95), there were 0.39 dose escalations, 0.16 dose reductions, 0.12 discontinuations, 0.19 therapy switches, and 0.24 add-ons per patient per year during the study. Most dose escalations or add-ons occurred within the first 6 months post-index. Of those who ever stopped levodopa-PDDI (n = 34), 31 (91%) restarted within the study period. Most (83%) patients who restarted levodopa-PDDI did so in the same year as stopping treatment. Index low dose users were associated with lower H-Y scores, were more inclined to escalate their dose, and were less inclined to reduce their dose in the first 2 years of treatment than index high dose users. CONCLUSIONS: Prescribers and patients tend to experiment with levodopa-PDDI treatment. Although many patients appeared to stop levodopa-PDDI after an initial course of treatment, most subsequently restarted treatment.

Natural history and patterns of treatment change in Parkinson's disease: A retrospective chart review.

BACKGROUND: Parkinson's disease (PD) management seeks to balance the benefits and harms of current medications and evolves as the disease progresses. The natural history of PD and associated patterns of treatment change were analyzed to identify unmet needs in treatment of PD symptoms. METHODS: Medical charts of patients from clinics across the US diagnosed on or before June 30th, 2014 were retrospectively reviewed. Index date was the first clinic visit, and the post-index period was through study end (June 30th, 2019). Outcomes included the frequency of therapy changes in the post-index period, reasons for therapy change, and adverse events (AE). RESULTS: Patients (n = 203) at index were receiving levodopa-peripheral dopa decarboxylase inhibitor (PDDI) monotherapy (47%), dopaminergic agonist (DA) monotherapy (15%), monoamine oxidase B inhibitor (MAOBI) monotherapy (14%), or combination therapies. The percentage of patients in Hoehn-Yahr disease Stage 1-2 was 52% at index and 20% by the end of the study. Frequencies of motor, non-motor, and neuropsychiatric symptoms increased during the enrollment. Levodopa-PDDI monotherapy and levodopa-PDDI + MAOBI had the lowest rates of therapy changes. Symptom relapse was the most common reason for dose escalation, add-on, and dose reduction, whereas AEs were the most common reason for discontinuation and switching. Dose escalation, add-on, and forward switch were most likely to occur in the first 6 months of treatment. CONCLUSIONS: Therapy changes during the study period reflected the challenging and evolving management of PD as the disease progresses. New or add-on symptomatic treatments are needed that are well-tolerated and able to control PD symptoms.

An assessment of Parkinson's disease medication treatment patterns in the Medicaid population.

INTRODUCTION: Most Parkinson's disease (PD) medication adherence studies have focused on patients with commercial or Medicare health insurance coverage. However, less is known regarding medication treatment patterns within the Medicaid population. METHODS: This retrospective cohort study utilized 2011-2019 administrative healthcare claims from 7 state Medicaid programs. We compared newly diagnosed patients with PD started on either levodopa or a dopamine agonist (DA). Baseline comorbidities were compared. Outcomes were assessed during a 12-month post-index observation period, and included total medication days, proportion of days covered (PDC), adherence status, persistence to initiating PD medication, and time to non-persistence of initiating PD medication. RESULTS: Our study sample of 805 Medicaid patients had an average age of 54.1 years, with 52.0% being female. Levodopa was the predominant PD medication at initiation (75.4%). Roughly half of patients had a baseline depressive disorder and nearly 40% had an anxiety disorder. Levodopa patients had a significantly higher PDC compared to DA patients (0.621 vs. 0.546, p = 0.007). An adjusted logistic regression model showed no significant difference in the number of adherent patients between the two groups (p = 0.058). An adjusted Cox proportional hazards model controlling for demographic and baseline variables showed a 26% lower risk of non-persistence for levodopa patients versus DA patients (HR 0.740, CI 0.597-0.917, p = 0.006). CONCLUSIONS: Adherence and persistence rates were suboptimal following initiation of either levodopa or DA medication for patients with PD in Medicaid programs, though rates were better for those initiated on levodopa.

Healthcare resource use and direct costs in severe aplastic anemia (SAA) patients before and after treatment with eltrombopag.

Purpose: This study evaluated healthcare resource utilization (HCRU), and direct costs among severe aplastic anemia (SAA) patients treated with eltrombopag (EPAG) using US claims data.Methods: This retrospective, real-world claims database study identified SAA patients aged ≥2 years treated with EPAG who initiated any SAA treatment between 1 July 2014 and 31 December 2017 (identification period) using the Truven MarketScan databases. A subset of 82 patients treated with EPAG during the identification period were evaluated for all-cause and SAA-related HCRU and direct costs as well as blood transfusion 1 month before EPAG initiation (baseline) and at Month 6 after EPAG initiation (follow-up period).Results: The average patient age was 50.8 (SD = 20.6) years old, predominantly female (n = 43, 52.4%), and had a mean CCI at baseline of 1.1 (SD = 1.7). Hospitalizations, and ER, office, and outpatient visits were significantly lower at Month 6 after EPAG initiation compared with 1 month before EPAG initiation (p < .05 for all four all-cause HCRU and SAA-related hospitalizations). An almost two-fold decrease in reliance on biweekly blood transfusions was observed: 1.0 at weeks 1-2 to 0.5 at Month 6 after EPAG initiation. Although prescription costs (mean [SD]) were significantly higher at Month 6 after EPAG initiation compared with 1 month before EPAG initiation (difference of $11,045 USD [SD = $18,801]), these increases were offset by savings in direct costs. Overall, a mean reduction in total all-cause costs of $29,391 USD [SD = $137,770] was reported at Month 6 after EPAG initiation due to substantial reductions in hospitalization ($40,060 USD [SD = $123,198]) and outpatient visits ($2,043 USD [SD = $25,264]).Conclusion: All-cause and SAA-related HCRU were reduced following EPAG treatment. Prescription costs were higher following treatment; however, these costs were generally offset by reductions in direct costs. These results provide real-world evidence around the role of EPAG in SAA treatment.

Treatment patterns and economic burden of sickle-cell disease patients prescribed hydroxyurea: a retrospective claims-based study.

BACKGROUND: This study aimed to evaluate sickle-cell disease (SCD) treatment patterns and economic burden among patients prescribed hydroxyurea (HU) in the US, through claims data. METHODS: SCD patients with pharmacy claims for HU were selected from the Medicaid Analytic Extracts (MAX) from January 1, 2009 - December 31, 2013. The first HU prescription during the identification period was defined as the index date and patients were required to have had continuous medical and pharmacy benefits for ≥6 months baseline and 12 months follow-up periods. Patient demographics, clinical characteristics, treatment patterns, health care utilization, and costs were examined, and variables were analyzed descriptively. RESULTS: A total of 3999 SCD patients prescribed HU were included; the mean age was 19.24 years, most patients were African American (73.3%), and the mean Charlson comorbidity index (CCI) score was 0.6. Asthma (20.3%), acute chest syndrome (15.6%), and infectious and parasitic diseases (20%) were the most prevalent comorbidities. During the 12-month follow-up period, 58.9% (N = 2357) of patients discontinued HU medication. The mean medication possession ratio (MPR) was 0.52, and 22.3% of patients had MPR ≥80%. The average length of stay (LOS) for SCD-related hospitalization was 13.35 days; 64% of patients had ≥1 SCD-related hospitalization. The mean annual total SCD-related costs per patient were $27,779, mostly inpatient costs ($20,128). CONCLUSIONS: Overall, the study showed the patients had significant unmet needs manifest as poor medication adherence, high treatment discontinuation rates, and high economic burden.

Healthcare costs and resource utilization in patients with severe aplastic anemia in the US.

Purpose: This study aimed to evaluate the healthcare resource utilization (HCRU) and costs for patients with severe aplastic anemia (SAA) using US claims data. Methods: This retrospective, observational database study analyzed claims data from the Truven MarketScan databases. SAA patients aged ≥2 years identified between 2014 and 2017 who were continuously enrolled for 6 months before their first SAA treatment or blood transfusion, with a ≥6-month follow-up, were included. Baseline demographics and comorbidities were evaluated. Monthly all-cause and SAA-related HCRU and direct costs in the follow-up period were analyzed and differences were presented for all patients and across age groups. Results: With an average follow-up period of 21.5 months, 939 patients were included in the study. Monthly all-cause and SAA-related HCRU [mean (SD)] were 1.65 days (2.61 days) and 0.18 days (0.70 days) for length of stay, 0.18 (0.23) and 0.01 (0.04) for hospital admissions, 0.25 (0.30) and 0.02 (0.07) for ER visits, 2.24 (1.40) and 0.46 (0.99) for office visits, and 2.90 (2.64) and 0.55 (1.31) for outpatient visits, respectively. On average, SAA patients received 0.15 (0.57) blood transfusions per month. Mean monthly all-cause direct costs were $28,280 USD ($36,127) [US dollars, mean (SD)]. Direct costs related to admissions were $11,433 USD (SD $25,040), followed by $624 USD ($1,703) for ER visits, $528 USD ($694) for office visits, $7,615 USD ($13,273) for outpatient visits, and $5,998 USD ($11,461) for pharmacy expenses. Monthly SAA-related direct costs averaged $7,884 USD (SD $16,254); of these costs, $1,608 USD ($7,774) were from admissions, $47 USD ($257) from ER visits, $127 USD ($374) from office visits, $1,462 USD ($4,994) from outpatient visits, and $4,451 USD ($10,552) from pharmacy expenses. Conclusion: SAA is associated with high economic burden, with costs comparable to blood malignancies, implying that US health plans should consider appropriately managing SAA while constraining the total healthcare costs when making formulary decisions.

Evaluation of Vaso-occlusive Crises in United States Sickle Cell Disease Patients: A Retrospective Claims-based Study.

Sickle cell disease (SCD) is a life-threatening vascular disease that burdens affected persons physically. SCD-related vaso-occlusive crises (VOCs) are one of the primary causes of morbidity and mortality. Our objective was to examine the epidemiology of pain crises and the relationship between pain crises and major acute complications among SCD patients. Using the Medicaid Analytic Extracts from 2009-2013, patients with SCD were selected and the first clinical claim indicating SCD during the identification period was defined as the index date. Patients were required to have continuous Medicaid enrollment for ≥6 months pre- and 12 months post-index period. Clinical outcomes included mortality, inpatient pain crises, and complications. Cox regressions were applied to examine the relationship between pain crises and deaths or acute complications, respectively. A total of 20 909 patients were included with a mean age of 17.9 years. The rate of VOC events in 100 person-years was 142.20 for adults and 53.91 for pediatric patients. Patients with VOCs were associated with a higher risk for death (hazard ratio=1.56; 95% confidence interval: [1.19-2.05]) or acute complications including acute chest syndrome, stroke, pulmonary embolism, splenic sequestration, and pulmonary hypertension. SCD patients have a substantial burden of disease-related complications. This study suggests that inpatient vaso-occlusive crisis is a key risk factor for acute complications.

Placement of selected new FDA-approved drugs in Medicare Part D formularies, 2009-2013.

OBJECTIVES: To assess formulary decisions by Part D plans for selected newly approved drugs. STUDY DESIGN: Retrospective cohort study. METHODS: Formulary placement and restrictions were identified for 33 drugs in 8 therapeutic classes (antihyperglycemics, anticoagulants, antiplatelets, disease-modifying agents for multiple sclerosis [MS] and rheumatoid arthritis [RA], chronic obstructive pulmonary disease [COPD] drugs, antiepileptics, and antipsychotics) in 863 Part D plans with continuous CMS contracts between 2009 and 2013. Multivariable models estimated the impact of drug characteristics and Part D plan characteristics on probability of drug adoption and, for adopters, evaluated factors associated with months to adoption and requirements for prior authorization (PA) or step therapy (ST). RESULTS: First Part D formulary placements varied from 2 to 14 months post FDA approval. On average, 56.7% of plans placed each drug within 6 months and 64.1% placed within 1 year of the National Drug Code assignment date. The most rapid adoption was for antipsychotics and antiepileptics. The slowest was for COPD drugs. More than 90% of disease-modifying agents for MS and RA were subject to PA. ST was uncommon except for antihyperglycemic agents. In adjusted analyses, enhanced benefit plans had a 4% higher probability of formulary placement (P <.01), and each additional star in the CMS star rating system increased the probability of adoption by 4% (P <.01). Overall, Medicare Advantage prescription drug plans had higher placement rates due to greater reliance on enhanced plan offerings and higher star ratings. CONCLUSIONS: We found significant heterogeneity in formulary placement and restrictions for 33 new drugs in the Part D marketplace between 2009 and 2013. Further research is necessary to determine whether this pattern applies to other drug classes.

Remote Patient Monitoring via Non-Invasive Digital Technologies: A Systematic Review.

BACKGROUND: We conducted a systematic literature review to identify key trends associated with remote patient monitoring (RPM) via noninvasive digital technologies over the last decade. MATERIALS AND METHODS: A search was conducted in EMBASE and Ovid MEDLINE. Citations were screened for relevance against predefined selection criteria based on the PICOTS (Population, Intervention, Comparator, Outcomes, Timeframe, and Study Design) format. We included studies published between January 1, 2005 and September 15, 2015 that used RPM via noninvasive digital technology (smartphones/personal digital assistants [PDAs], wearables, biosensors, computerized systems, or multiple components of the formerly mentioned) in evaluating health outcomes compared to standard of care or another technology. Studies were quality appraised according to Critical Appraisal Skills Programme. RESULTS: Of 347 articles identified, 62 met the selection criteria. Most studies were randomized control trials with older adult populations, small sample sizes, and limited follow-up. There was a trend toward multicomponent interventions (n = 26), followed by smartphones/PDAs (n = 12), wearables (n = 11), biosensor devices (n = 7), and computerized systems (n = 6). Another key trend was the monitoring of chronic conditions, including respiratory (23%), weight management (17%), metabolic (18%), and cardiovascular diseases (16%). Although substantial diversity in health-related outcomes was noted, studies predominantly reported positive findings. CONCLUSIONS: This review will help decision makers develop a better understanding of the current landscape of peer-reviewed literature, demonstrating the utility of noninvasive RPM in various patient populations. Future research is needed to determine the effectiveness of RPM via noninvasive digital technologies in delivering patient healthcare benefits and the feasibility of large-scale implementation.

Increasing Patient Involvement in Drug Development.

BACKGROUND: To ensure the creation of treatments that maximize value at the lowest cost, all aspects of the health care system need to align with patient needs and preferences. Despite growing efforts to engage patients in research and regulatory activities, the pharmaceutical industry has yet to maximize patient involvement in the drug development process. OBJECTIVE: To gain a better understanding of the present state of patient involvement in drug development. METHODS: Through a semistructured interview methodology, we sought to identify opportunities, barriers, and examples of patient involvement in the drug development process. Telephone interviews were conducted with six senior leaders of evidence generation within the pharmaceutical industry and four patients. These interviews were supplemented with interviews with a research funder, a regulator, a patient advocacy group, and a caregiver. RESULTS: Although our interviewees spoke of the potential benefits of aligning research around the needs of patients, there were few examples they could share to suggest this was occurring at scale. A number of barriers were identified including the added burden associated with adverse event reporting, concerns about patient representativeness or their ability to participate in drug development conversations, and the costs in time and resources involved relative to returns on investment. CONCLUSIONS: As health care systems continue to evolve and establish patients as the primary stakeholder in their health care decision making, the pharmaceutical industry will need to be innovative to demonstrate the value of their products relative to the outcomes experienced by patients. Pharmaceutical companies should recognize the value of involving patients across the entire product life cycle and work to transform present perceptions and practices throughout their organizations.

Difference-in-Differences Method in Comparative Effectiveness Research: Utility with Unbalanced Groups.

BACKGROUND: Comparative effectiveness research (CER) often includes observational studies utilizing administrative data. Multiple conditioning methods can be used for CER to adjust for group differences, including difference-in-differences (DiD) estimation. OBJECTIVE: This study presents DiD and demonstrates how to apply this conditioning method to estimate treatment outcomes in the CER setting by utilizing the MarketScan® Databases for multiple sclerosis (MS) patients receiving different therapies. METHODS: The sample included 6762 patients, with 363 in the Test Cohort [glatiramer acetate (GA) switched to fingolimod (FTY)] and 6399 in the Control Cohort (GA only, no switch) from a US administrative claims database. A trend analysis was conducted to rule out concerns regarding regression to the mean and to compare relapse rates among treatment cohorts. DiD analysis was used to enable comparisons among the Test and Control Cohorts. Logistic regression was used to estimate the probability of relapse after switching from GA to FTY, and to compare group differences in the pre- and post-index periods. RESULTS: Crude DiD analysis showed that in the pre-index period more patients in the Test Cohort experienced an MS relapse and had a higher mean number of relapses than in the Control Cohort. During the pre-index period, numeric and relative data for MS relapses in patients in the Test Cohort were significantly higher than in the Control Cohort, while no significant between-group differences emerged during the post-index period. Generalized linear modeling with DiD regression estimation showed that the mean number of MS relapses decreased significantly in the post-index period among patients in the Test Cohort compared with patients in the Control Cohort. CONCLUSION: In this study, an MS population was utilized to demonstrate how DiD can be applied to estimate treatment effects in a heterogeneous population, where the Test and Control Cohorts varied greatly. The results show that DiD offers a robust method for comparing diverse cohorts when other risk-adjustment methods may not be adequate.

Health economics and outcomes research fellowship practices reviewed.

The guidelines for health economics and outcomes research (HEOR) fellowship training programs devised by the American College of Clinical Pharmacy (ACCP) and the International Society of Pharmacoeconomics and Outcomes Research (ISPOR) suggest that continuous improvements are made to ensure that postgraduate training through didactic and professional experiences prepare fellows for HEOR research careers. The HEOR Fellowship Program at Novartis Pharmaceuticals Corporation was standardized to enhance the fellows' HEOR research understanding and align professional skill sets with the ACCP-ISPOR Fellowship Program Guidelines. Based on feedback from an internal task force comprised of HEOR employees and current and former fellows, the HEOR Fellowship Program was normatively and qualitatively assessed to evaluate the current curricular program. Fellowship program activities were instituted to ensure that the suggested minimum level requirements established by the guidelines were being met. Research opportunities enabling fellows to work hand-in-hand with other fellows and HEOR professionals were emphasized. Curricular enhancements in research methodology and professional training and development, and materials for a structured journal club focusing on specific methodological and HEOR research topics were developed. A seminar series (e.g., creating SMART Goals, StrengthsFinder 2.0) and professional courses (e.g., ISPOR short courses, statistics.com) were included to enhance the fellows' short- and long-term professional experience. Additional program attributes include an online reference library developed to enrich the current research facilities and a Statistical Analysis Software training program. Continuously assessing and updating HEOR fellowship programs keeps programs up-to-date in the latest HEOR concepts and approaches used to evaluate health care, both professionally and educationally.

Group-based trajectory modeling to assess adherence to biologics among patients with psoriasis.

BACKGROUND: Proportion of days covered (PDC), a commonly used adherence metric, does not provide information about the longitudinal course of adherence to treatment over time. Group-based trajectory model (GBTM) is an alternative method that overcomes this limitation. METHODS: The statistical principles of GBTM and PDC were applied to assess adherence during a 12-month follow-up in psoriasis patients starting treatment with a biologic. The optimal GBTM model was determined on the basis of the balance between each model's Bayesian information criterion and the percentage of patients in the smallest group in each model. Variables potentially predictive of adherence were evaluated. RESULTS: In all, 3,249 patients were included in the analysis. Four GBTM adherence groups were suggested by the optimal model, and patients were categorized as demonstrating continuously high adherence, high-then-low adherence, moderate-then-low adherence, or consistently moderate adherence during follow-up. For comparison, four PDC groups were constructed: PDC Group 4 (PDC ≥75%), PDC Group 3 (25%≤ PDC <50%), PDC Group 2 (PDC <25%), and PDC Group 1 (50%≤ PDC <75%). Our findings suggest that the majority of patients (97.9%) from PDC Group 2 demonstrated moderate-then-low adherence, whereas 96.4% of patients from PDC Group 4 showed continuously high adherence. The remaining PDC-based categorizations did not capture patients with uniform adherence behavior based on GBTM. In PDC Group 3, 25.3%, 17.2%, and 57.5% of patients exhibited GBTM-defined consistently moderate adherence, moderate-then-low adherence, or high-then-low adherence, respectively. In PDC Group 1, 70.8%, 23.6%, and 5.7% of patients had consistently moderate adherence, high-then-low adherence, and continuously high adherence, respectively. Additional analyses suggested GBTM-based categorization was best predicted by patient age, sex, certain comorbidities, and particular drug use. CONCLUSION: GBTM is a more appropriate way to model dynamic behaviors and offers researchers an alternative to more traditional drug adherence measurements.

Inhaled corticosteroid use in patients with chronic obstructive pulmonary disease and the risk of pneumonia: a retrospective claims data analysis.

BACKGROUND: The use of inhaled corticosteroids in patients with chronic obstructive pulmonary disease (COPD) has been associated with an increased risk of pneumonia in controlled clinical trials and case-control analyses. OBJECTIVE: Using claims databases as a research model of real-world diagnosis and treatment, to determine if the use and dose of inhaled corticosteroids (ICS) among patients with newly diagnosed COPD are associated with increased risk of pneumonia. PATIENTS AND METHODS: This was a retrospective cohort analysis of patients diagnosed with COPD between January 01, 2006 and September 30, 2010, drawn from databases (years 2006-2010). Patients (aged ≥45 years) were followed until first pneumonia diagnosis, end of benefit enrollment, or December 31, 2010, whichever was earliest. A Cox proportional hazard model was used to assess the association of ICS use and risk of pneumonia, controlling for baseline characteristics. Daily ICS use was classified into low, medium, and high doses (1 µg-499 µg, 500 µg-999 µg, and ≥1000 µg fluticasone equivalents daily) and was modeled as a time-dependent variable. RESULTS: Among 135,445 qualifying patients with a total of 243,097 person-years, there were 1020 pneumonia incidences out of 5677 person-years on ICS (crude incidence rate, 0.180 per person-year), and 27,730 pneumonia incidences out of 237,420 person-years not on ICS (crude incidence rate, 0.117 per person-year). ICS use was associated with a dose-related increase in risk of pneumonia, with adjusted hazard ratios (versus no use; (95% confidence interval) of 1.38 (1.27-1.49) for low-dose users, 1.69 (1.52-1.88) for medium-dose users, and 2.57 (1.98-3.33) for high-dose users (P < 0.01 versus no use and between doses). CONCLUSION: The use of ICS in newly diagnosed patients with COPD is potentially associated with a dose-related increase in the risk of pneumonia.

Identifying barriers to hypertension care: implications for quality improvement initiatives.

The role of clinical inertia in the treatment of patients with hypertension was assessed by evaluating health care providers' knowledge, attitudes, and clinical practices regarding hypertension management. A cross-sectional survey was conducted at the Forsyth Medical Group in North Carolina. Participants were physicians (N = 18, 10 sites) and support staff (N = 20, 12 sites), who were surveyed in 2006. Physician and support staff questionnaires consisted of 29 and 15 items, respectively, and were administered by trained interviewers. Though most physicians (94%) cited familiarity with the Seventh Report of the Joint National Committee on Prevention, Detection, Evaluation, and Treatment of High Blood Pressure (JNC-7) guidelines and affirmed that hypertension management guidelines are relevant to their patients, no physicians interviewed routinely document patient hypertension management plans. Although 1 in 3 physicians cited the inability to devote enough time to patients for the discussion of hypertension management, physicians predominantly cited patient- and support-staff- related factors as most important to patients not attaining blood pressure (BP) goal. Patient lifestyle modification (89%), education (67%), and medication compliance (56%) were cited as the most important reasons for uncontrolled BP. Only one-third of physicians believe that clinical staff always obtain accurate BP measurements, and 61% believe that resistant hypertension is a reflection of inaccurate BP measurement. Many support staff claimed to be rushed when measuring patient BP, and 65% recommended BP competency training. Contradictions were evident between provider knowledge of hypertension management standards and how this knowledge is applied in clinical practice. Standardized collection of BP is critical to measuring clinical improvement in hypertension. Results are being utilized to develop clinical improvement initiatives including staff education and competency training.

A cost comparison of alternative regimens for treatment-refractory partial seizure disorder: an econometric analysis.

BACKGROUND: Partial seizure disorder is typically treated by monotherapy with antiepileptic drugs (AEDs). However, when the condition is refractory to the initial treatment regimen, patients may be switched to monotherapy with another AED or to combination therapy with the initial AED plus a second AED. OBJECTIVES: The purpose of this study was to examine the economic costs associated with treatment-refractory partial seizure disorder and to compare the costs of 2 alternative approaches: a switch to oxcarbazepine (OXC) monotherapy or the addition to the regimen of another AED (AED add-on). METHODS: Adult patients with a diagnosis of partial seizure disorder who received initial AED monotherapy between January 1, 2000, and March 31, 2003, were identified from the PharMetrics Patient-Centric Database, a health plan administrative claims database. The medical and pharmacy history of these patients was analyzed from 6 months before a change to either OXC monotherapy or AED add-on therapy through 12 months after the change in treatment. Total health care resource utilization and the associated costs were compared within each cohort before and after the change, as well as between cohorts, with statistical differences tested using Wilcoxon rank sum tests. Multivariate econometric analyses were performed to examine the impact of age, sex, geographic location, Charlson Comorbidity Index, and the presence of specific comorbidities. RESULTS: Demographic and clinical characteristics 102 were similar between the OXC monotherapy cohort (n = 259) and the AED add-on cohort (n = 795). Annual direct treatment costs increased in both groups in the period after the failure of initial monotherapy, increasing from 10,462 US dollars to 11,360 US dollars in the OXC cohort and from 10,137 US dollars to 12,201 US dollars in the AED add on cohort (P < 0.01). Increased pharmacy costs were the primary driver behind cost increases in both cohorts. Patients in the AED add-on cohort were significantly more likely to have an emergency department visit during the period after the failure of initial monotherapy compared with the OXC monotherapy cohort (odds ratio = 1.52; P < 0.05). CONCLUSION: Despite limitations, the results of retrospective analysis of claims data suggest that the care of patients with treatment-refractory partial seizure disorder is costly and may vary significantly based on the pattern of care.