Search

Objectives: To measure the value of survival gains attributable to the introduction of 3 novel therapies for myelodysplastic syndromes (MDS)., Study Design: Retrospective study of patients diagnosed with MDS in the Surveillance, Epidemiology and End Results Program (SEER) registry, clinical trial evidence for MDS therapies, and claims data., Methods: We used multivariate Cox proportional hazards models to estimate the increase in survival associated with the introduction of the 3 new therapies for patients diagnosed with MDS from 2001 to 2011 in the SEER cancer registry. Increases in survival associated with the 3 novel therapies were estimated using retrospective survival analyses and published clinical trial evidence. MDS treatment costs were estimated using Ingenix claims data and used to calculate the share of the value of survival gains retained by patients., Results: We estimated that the introduction of these 3 therapies is associated with a hazard ratio of 0.901 (P <.10), and a 73% increase in median survival from 33 to 57 months. We estimated that for current and future MDS patients, these 3 therapies will generate over $193 billion in cumulative value through extensions in patient survival., Conclusions: This study demonstrates that the value of recently approved innovative therapies in MDS is large and that the value of survival gains in MDS far outweighs their costs.

Effective pursuit of the science and management of heterogeneity of treatment effect (HTE) relies on the mutual understanding of the perspectives of, and collaboration among, the various stakeholders in health care. In this article, we compare, contrast, and endeavor to find areas of alignment across the perspectives of three such stakeholders -regulators, the biopharmaceutical and device industry, and U.S. payers. First, we discuss how evidence of HTE is generated and could be improved upon. For pharmaceuticals, much of the initial research is conducted by the pharmaceutical industry, guided by basic science but also delimited by potential markets, regulatory approval requirements, trial size considerations, and payer expectations for evidence of value. Once a drug is marketed, further evidence can be generated via combining trial data, conducting meta-analysis, and analyzing real-world results through observational research designs; we explore how these efforts can benefit from cooperation across these stakeholders. Second, we discuss the equally important utilization of HTE evidence so that physicians and patients have access to and can benefit from the learnings from this research. Research findings must be translated into actionable information and guidelines that can be incorporated into everyday practice. Doing so requires interaction and collaboration among all involved, based on facilitated communication as well as further evaluation research. We provide examples of several cross-sectorial initiatives that are under way in this area. Finally, we explore some economic aspects of HTE research as part of the drug development, marketing, and treatment process. Understanding the economic incentives present is fundamental to aligning those incentives to improve the availability and utilization of HTE evidence. Clear understandings among regulators, pharma, and payers about high-value targets, methods to efficiently generate and communicate information, and value propositions can lead to "win-win" scenarios for patients, individual payers, the health care system overall, and the future of drug development in producing new medicines., (Copyright © 2013, International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc.)

Objective: Azacitidine and decitabine are used to treat patients with myelodysplastic syndromes (MDS) in the United States (US). This study sought to assess their relative cost-effectiveness., Design and Methods: The authors developed a cost-effectiveness Markov model (1-month cycles) tracking hypothetical cohorts of MDS patients treated with azacitidine or decitabine over 2 years. The model used a US payer perspective and 2009 costs. Health states modeled included MDS with Transfusion Dependence, MDS with Transfusion Independence, Progression to Acute Myelogenous Leukemia (AML), and Death. Incremental cost-effectiveness outcomes included cost per quality-adjusted life year (QALY), cost per life year (LY), cost per patient-month of transfusion independence, and cost per case of AML progression avoided. One-way sensitivity analyses were performed on key model parameters., Results: Compared to decitabine, azacitidine was associated with better survival (1.512 LYs vs 1.292), more QALYs gained (1.041 vs 0.870), more patient-months with transfusion independence (8.328 vs 6.224), and a greater proportion of patients avoiding progression to AML (50.9% vs 28.5%). Total per-patient costs over 2 years for azacitidine were lower than for decitabine ($150,322 vs $166, 212)., Limitations: To inform and update the model over time, it will be important that randomized or observational clinical studies be conducted to directly compare azacitidine and decitabine, provide new information on how these medicines are used, and on their relative clinical effectiveness., Conclusion: Results demonstrate that azacitidine provides greater clinical benefit and costs less than decitabine across all key outcomes. These results accentuate the positive role of azacitidine in providing cost-effective care for MDS.

In the Valsartan Antihypertensive Long-Term Use Evaluation (VALUE) trial, the risk of new-onset diabetes was reported to be 23% lower among patients initiating therapy with valsartan versus amlodipine. The objective of our study was to examine whether this finding is generalizable to 'real-world' clinical practice. A retrospective cohort design and a large US health insurance database were employed for analyses. Study subjects included all hypertensive patients, aged >or=35 years, who were free from diabetes and who initiated treatment with valsartan (n=9999) or amlodipine (n=18 698) between January 1999 and March 2005. Unadjusted absolute risks of diabetes were 21.4 (95% confidence interval (CI) 18.9-24.3) and 26.3 (95% CI 24.3-28.3) per 1000 patient-years for valsartan and amlodipine, respectively; the corresponding relative risk (RR) for valsartan was 0.82 (95% CI 0.70-0.94). Multivariate analyses - controlling for age, sex, presence of hypercholesterolemia, cardiovascular disease and kidney disease, and pretreatment medical care expenditures - yielded similar results (RR=0.79, 95% CI 0.68-0.92). Our study thus corroborates the finding from VALUE that diabetes risk is lower for patients who receive valsartan versus amlodipine, and extends this finding to a 'real-world' setting.

Objectives: To: evaluate the impact of smoking status on objective productivity and absenteeism measures; evaluate the impact of smoking status on subjective measures of productivity; and assess the correlation between subjective and objective productivity measures., Design: Prospective cohort study in a workplace environment., Subjects: Approximately 300 employees (100 each of former, current, and never smokers) at a reservation office of a large US airline., Main Outcome Measures: Objective productivity and absenteeism data were supplied by the employer. Subjective assessments of productivity were collected using a self report instrument, the Health and Work Questionnaire (HWQ)., Results: Current smokers had significantly greater absenteeism than did never smokers, with former smokers having intermediate values; among former smokers, absenteeism showed a significant decline with years following cessation. Former smokers showed an increase in seven of 10 objective productivity measures as compared to current smokers, with a mean increase of 4.5%. While objective productivity measures for former smokers decreased compared to measures for current smokers during the first year following cessation, values for former smokers were greater than those for current smokers by 1-4 years following cessation. Subjective assessments of "productivity evaluation by others" and "personal life satisfaction" showed significant trends with highest values for never smokers, lowest for current smokers, and intermediate for former smokers., Conclusions: Workplace productivity is increased and absenteeism is decreased among former smokers as compared to current smokers. Productivity among former smokers increases over time toward values seen among never smokers. Subjective measures of productivity provide indications of novel ways of productivity assessment that are sensitive to smoking status.

Objective: The objective of this study was to evaluate the health care costs associated with the treatment of a new episode of depression with bupropion sustained release (SR) rather than with other antidepressants (selective serotonin reuptake inhibitors [SSRIs], tricyclic antidepressants [TCAs], and serotonin norepinephrine reuptake inhibitors [SNRIs])., Methods: This was a retrospective cohort study based on the private-pay, fee-for-service 1997 and 1998 MEDSTAT MarketScan databases. Individuals were included if they were 18 years of age or older, had an initial prescription for an antidepressant under study with an index prescription date between July 1997 and June 1998, and had a claim for a diagnosis of depression diagnosis within 30 days of the index date. All patients' claims from six months before and after receiving their index antidepressant prescription were examined. Total, outpatient, and pharmacy costs were compared among antidepressant groups using an intent-to-treat analysis with exponential regression models and bootstrapped 95% confidence intervals., Results: A total of 1771 patients were included in the study cohort. The mean age was 41.6 years, and 69.5% of subjects were female. Most patients (75%) continued with the index antidepressant during the 6-month follow-up period. Although the drug acquisition cost was lowest for TCAs, total costs were significantly higher for patients treated with TCAs than for those treated with bupropion SR (p < .05). In comparison with bupropion SR, patients initiating therapy with sertraline had significantly higher mental health payments (p < .05)., Conclusions: Initiating treatment of depression with bupropion SR was associated with lower total mental health care costs compared with TCAs and with sertraline. This study reaffirms that formulary and medical decision-makers should consider the overall impact of antidepressant treatment, including but not limited to drug acquisition costs, other health care costs, and drug efficacy and safety.

Objective: To conduct an economic analysis on the use of carboplatin versus cisplatin over multiple courses in patients with lung [nonsmall cell lung cancer (NSCLC) and small cell lung cancer (SCLC)] or ovarian cancer., Design: This 1-year study was a prospective, multicentre, cost-minimisation evaluation. Direct medical resource utilisation and costs associated with carboplatin and cisplatin administration over 3 to 6 courses of treatment were measured and compared. The perspective of this evaluation was that of the payer., Setting: A convenience sample of 16 sites representing a mix of cancer centres, outpatient clinics, medical centres and managed-care sites in a general practice oncology setting participated., Patients and Interventions: Patients were included in this study if they were newly diagnosed with NSCLC, SCLC or ovarian cancer, had not received prior chemotherapy, received either carboplatin or cisplatin as their treatment (additional chemotherapy agents were allowed), and received at least 3 courses of carboplatin or cisplatin therapy up to a maximum of 6 courses. Patients receiving more than 6 courses of therapy were included in this study, but data collection on those patients stopped after the sixth course. Individuals involved with data collection at all sites were trained via on-site and/or teleconference training. Site visits were made to assure reliability of at least 0.80. Data were collected and compiled via a fax transmission process that scans directly through optical mark and character recognition into a computer database. Outcome measures included costs of: medications, emergency room visits, physician/clinic/laboratory visits, home healthcare visits, transfusions, special procedures, consultations, hospitalisations and other/miscellaneous costs., Main Outcome Measures and Results: Of 220 patients, 164 met the study criteria (response rate = 74.2%) with 95 patients in the carboplatin group (NSCLC = 45, SCLC = 18, ovarian = 32) and 69 in the cisplatin group (NSCLC = 36, SCLC = 21, ovarian = 12). The average number of courses were: NSCLC = 4.3 and 4.2, SCLC = 4.3 and 4.8, and ovarian = 4.7 and 5.1, respectively, for carboplatin and cisplatin. The total costs (treatment and toxicity) associated with the use of carboplatin were higher in NSCLC, similar in SCLC but lower in ovarian cancer., Conclusions: These results indicate that overall treatment costs may vary depending on cancer type, even when the same drugs are used. The total costs (treatment plus toxicity costs) associated with the use of carboplatin were higher than those of cisplatin in patients with NSCLC, similar in SCLC, but lower in ovarian cancer.