Breakthrough cancer pain (BTP) has a significant impact on patients' activities of daily living, family, and the society; however, the economic ramifications of BTP are largely unknown. This review aims to summarize the available pharmacoeconomics studies of BTP in the context of the availability of several formulations of rapid-onset opioids administered by various routes, which are significantly more expensive than oral opioids. A systematic literature search of PubMed and Tufts registry through August 2012 was conducted using key words including "breakthrough cancer pain" and "cost effectiveness." After exclusion of irrelevant articles, a total of six articles were included. Studies reviewed include two economic survey studies, two quality improvement projects, and two decision-analytic models. These studies demonstrate BTP causes significant financial burden to patients and society through increased hospitalization and health care utilization. Only one study comparing placebo with intranasal fentanyl spray, oral transmucosal fentanyl citrate, and oral transmucosal fentanyl buccal tablet has demonstrated the cost-effectiveness of these rapid-onset opioids for the treatment of BTP. Overall, there is a lack of pharmacoeconomic studies for BTP management with rapid-onset opioids. Further study is warranted assessing the net benefit of rapid-onset opioids to oral opioids to assist decision-making by patients, clinicians, and payers.

Outcomes research studies use clinical and administrative data generated in the course of patient care or from patient surveys to examine the effectiveness of treatments. Health care providers need to understand the limitations and strengths of the real-world data sources used in outcomes studies to meaningfully use the results. This paper describes five types of databases commonly used in the United States for outcomes research studies, discusses their strengths and limitations, and provides examples of each within the context of pain treatment. The databases specifically discussed are generated from (1) electronic medical records, which are created from patient-provider interactions; (2) administrative claims, which are generated from providers' and patients' transactions with payers; (3) integrated health systems, which are generated by systems that provide both clinical care and insurance benefits and typically represent a combination of electronic medical record and claims data; (4) national surveys, which provide patient-reported responses about their health and behaviors; and (5) patient registries, which are developed to track patients with a given disease or exposure over time for specified purposes, such as population management, safety monitoring, or research.

Retrospective review of imatinib monitoring through electronic health records (EHR) can provide valuable insight into the current management of chronic myelogenous leukemia (CML). This study retrospectively reviewed EHRs from 2001 to 2010 of patients with chronic phase CML (CP-CML) treated with first-line imatinib. Chart evaluations included a review of cytogenetic and molecular testing, overall survival, adverse drug events (ADEs), and therapy modifications. A total of 54 patients with CP-CML were treated with first-line imatinib and had either cytogenetic or molecular testing within 18 months of imatinib initiation. Within the first 18 months of treatment, 33 of 45 patients (73%) undergoing cytogenetic testing experienced a complete cytogenetic response (median, 241 days; range, 110-542 days) and 24 of 48 patients (50%) receiving molecular testing achieved at least a major molecular response (median, 253 days; range, 99-546 days). The average number of cytogenetic and molecular tests conducted within the first 18 months was 2.5 and 3.8, respectively. Nineteen of 54 (35%) had a dose increase of imatinib (>400 mg; median, 329 days; range, 21-1968 days). The 5-year estimated overall survival rate was 88.5%. Between 2006 and 2010 (n=30; 56%), 7 patients (23%) transitioned to dasatinib or nilotinib (median, 399 days from diagnosis; range, 180-1046 days) because of suboptimal response or treatment failure (n=5) and imatinib ADEs (n=2). Forty-six imatinib-associated ADEs occurred in 31 patients (57%), of which 10 (32%) received dose reductions (median, 52 days) and 6 (19%) had discontinuations (median, 139 days). Closely monitored patients with CML treated with imatinib at an NCCN Member Institution experienced outcomes comparable to those reported in key clinical trials.

Treatment patterns in the NCCN groupings correlated with recommended treatment strategies. Overall, the NCCN groupings have a linear relationship in overall survival.

A639 ment regimens for chronic myeloid leukemia. If further research were funded, studies should examine a combination of natural history, treatment, and quality of life parameters, especially the effectiveness of first-line TKI treatment.Objectives: The first goal was to adapt an existing Austrian decision-analytic model for chronic myeloid leukemia (CML) treatment to the US-American health care context. Secondly, we updated the model with new data and further treatment strategies to identify the most effective and most cost-effective strategy for the treatment of CML patients with different sequential tyrosine kinase inhibitors (TKIs). MethOds: We evaluated 18 different treatment strategies within the US-American setting in terms of survival, quality-adjusted survival and costs. For model parameters, data from literature, a US-American expert survey, the Utah Cancer Registry, and economic data from a US-American database were used. Evaluated treatment strategies included imatinib, dasatinib, nilotinib, bosutinib, ponatinib, stem-cell transplantation and chemotherapy. The Markov state-transition model was analyzed as a cohort simulation over a lifelong time horizon, a third-party payer perspective was adopted and a discount rate of 3% was used. Additionally, several deterministic and probabilistic sensitivity analyses were conducted. Results: Imatinib without second-line TKI resulted in an incremental cost-utility ratio (ICUR) of $148,700/QALY gained (incremental cost-effectiveness ratio (ICER) of $128,800/Lys) compared to baseline strategy 'chemotherapy'. Imatinib with second-line nilotinib yielded an ICUR of $217,100/QALY gained (ICER $242,200/ LY) compared to imatinib without second-line TKI. Imatinib followed by secondline bosutinib had an ICUR of $331,300/QALY gained (ICER $265,100/LY) compared to imatinib followed by second-line nilotinib. Imatinib with second-line dasatinib produced an ICUR of $343,200/QALY gained (ICER $279,600/LY) compared to imatinib with second-line bosutinib. All remaining strategies were excluded due to dominance. ICURs and ICERs obtained from the probabilistic sensitivity analysis deviated up to 6.5% (2.5%) compared to base-case ICURs (ICERs). cOnclusiOns: Based on our analysis and current treatment guidelines, we recommend imatinib followed by second-line nilotinib as the most cost-effective treatment strategy. Our model results may support clinicians and patients in CML treatment decision making.