Cost-effectiveness analysis of trastuzumab emtansine (T-DM1) in human epidermal growth factor receptor 2 (HER2): positive advanced breast cancer.

PURPOSE: The EMILIA trial demonstrated that trastuzumab emtansine (T-DM1) significantly increased the median profession-free and overall survival relative to combination therapy with lapatinib plus capecitabine (LC) in patients with HER2-positive advanced breast cancer (ABC) previously treated with trastuzumab and a taxane. We performed an economic analysis of T-DM1 as a second-line therapy compared to LC and monotherapy with capecitabine (C) from both perspectives of the US payer and society. METHODS: We developed four possible Markov models for ABC to compare the projected life-time costs and outcomes of T-DM1, LC, and C. Model transition probabilities were estimated from the EMILIA and EGF100151 clinical trials. Direct costs of the therapies, major adverse events, laboratory tests, and disease progression, indirect costs (productivity losses due to morbidity and mortality), and health utilities were obtained from published sources. The models used 3 % discount rate and reported in 2015 US dollars. Probabilistic sensitivity analysis and model averaging were used to account for model parametric and structural uncertainty. RESULTS: When incorporating both model parametric and structural uncertainty, the resulting incremental cost-effectiveness ratios (ICER) comparing T-DM1 to LC and T-DM1 to C were $183,828 per quality-adjusted life year (QALY) and $126,001/QALY from the societal perspective, respectively. From the payer's perspective, the ICERs were $220,385/QALY (T-DM1 vs. LC) and $168,355/QALY (T-DM1 vs. C). CONCLUSIONS: From both perspectives of the US payer and society, T-DM1 is not cost-effective when comparing to the LC combination therapy at a willingness-to-pay threshold of $150,000/QALY. T-DM1 might have a better chance to be cost-effective compared to capecitabine monotherapy from the US societal perspective.

A Systematic Review of Health Economic Evaluation Studies Using the Patient's Perspective.

BACKGROUND: Patient-centered care has become increasingly important and relevant for informed health care decision making. OBJECTIVE: Our study aimed to perform a systematic review of health economic evaluation studies from the patient's perspective. METHODS: PubMed, EMBASE, and Cochrane Central databases were searched through May 2014 for cost-effectiveness, cost-utility, and cost-benefit studies using the patient's perspective in their analysis. The reporting quality of the studies was evaluated on the basis of Consolidated Health Economic Evaluation Reporting Standards. RESULTS: We identified 30 health economic evaluations using the patient's perspective, of which 7 were conducted in the United States, 9 in Europe, and 14 in Asian or other countries. Seventeen of 23 health conditions evaluated were chronic in nature. Among 12 studies that justified the use of the patient's perspective, patient's financial burden associated with medical treatment was the most commonly cited rationale. A total of 29, 17, and 15 studies examined direct medical, direct nonmedical, and indirect costs, respectively. Seventeen studies also included societal, governmental or payer's, and/or provider's perspective(s) in their analyses. Based on Consolidated Health Economic Evaluation Reporting Standards, more than 20% of the reporting items in these studies were either partially satisfied or not satisfied. CONCLUSIONS: There is a paucity of health economic evaluations conducted from the patient's perspective in the literature. For those studies using the patient's perspective, the true patient costs were not fully explored and study reporting quality was not optimal. With the increasing focus on patient-centered outcomes in health policy research, more frequent use of the patient's perspective in economic studies should be advocated.

Putting words into action: A simple focused education improves prescription label comprehension and functional health literacy.

OBJECTIVES: To assess the effectiveness of an educational intervention on prescription (Rx) label comprehension and functional health literacy (FHL) in older adults. Outcomes were compared between current and redesigned Rx labels. Additional objectives were to examine the correlation between 2 outcome measures and to determine the characterizing variables that are predictors for the outcome measures. SETTING: Southern California, January 2013 to March 2015. PRACTICE DESCRIPTION: Older adults (>55 y) taking 2 or more Rx medications daily were recruited at senior and community centers by a trained data collection team. PRACTICE INNOVATION: The validated Modified LaRue Tool (MLT) tested Rx label comprehension before and after a short, focused educational intervention and correlated it with FHL. INTERVENTIONS: A simple one-on-one education provided by student pharmacists that was focused on critical elements of an Rx label. EVALUATION: Short Test of Functional Health Literacy in Adults (STOFHLA) and MLT scores of all current and redesigned label participants at baseline and follow-up. RESULTS: Participants using redesigned Rx labels (n = 90) showed significantly higher MLT scores than with current Rx labels (n = 28) both before (23.0 ± 2.3 vs. 21.0 ± 2.4; P <0.001) and after educational intervention (23.8 ± 1.7 vs. 22.1 ± 3.1; P <0.001). With the use of analysis of covariance, intervention participants using redesigned label (n = 48) showed significant improvement in both MLT (23.1 ± 2.0 to 24.3 ± 1.0; P <0.001) and STOFHLA (29.8 ± 7.5 to 31.5 ± 5.7; P = 0.011) scores, whereas intervention participants using current Rx label (n = 16) did not show significant improvement in either MLT (P = 0.530) or STOFHLA (P = 0.215) scores. Effect size of intervention (redesigned label) was 0.77, indicating practical significance. MLT and STOFHLA scores were significantly correlated with each other, and age and education level were common significant predictors for both outcomes. CONCLUSION: Older adults using redesigned Rx labels showed improved Rx label comprehension and FHL after educational intervention, as well as higher comprehension compared with those using current Rx labels. Use of a redesigned Rx label and a simple educational intervention should be encouraged to improve Rx label comprehension and FHL.

Validating the Modified Drug Adherence Work-Up (M-DRAW) Tool to Identify and Address Barriers to Medication Adherence.

Barriers to medication adherence stem from multiple factors. An effective and convenient tool is needed to identify these barriers so that clinicians can provide a tailored, patient-centered consultation with patients. The Modified Drug Adherence Work-up Tool (M-DRAW) was developed as a 13-item checklist questionnaire to identify barriers to medication adherence. The response scale was a 4-point Likert scale of frequency of occurrence (1 = never to 4 = often). The checklist was accompanied by a GUIDE that provided corresponding motivational interview-based intervention strategies for each identified barrier. The current pilot study examined the psychometric properties of the M-DRAW checklist (reliability, responsiveness and discriminant validity) in patients taking one or more prescription medication(s) for chronic conditions. A cross-sectional sample of 26 patients was recruited between December 2015 and March 2016 at an academic medical center pharmacy in Southern California. A priming question that assessed self-reported adherence was used to separate participants into the control group of 17 "adherers" (65.4%), and into the intervention group of nine "unintentional and intentional non-adherers" (34.6%). Comparable baseline characteristics were observed between the two groups. The M-DRAW checklist showed acceptable reliability (13 item; alpha = 0.74) for identifying factors and barriers leading to medication non-adherence. Discriminant validity of the tool and the priming question was established by the four-fold number of barriers to adherence identified within the self-selected intervention group compared to the control group (4.4 versus 1.2 barriers, p < 0.05). The current study did not investigate construct validity due to small sample size and challenges on follow-up with patients. Future testing of the tool will include construct validation.