A. Van Engen

Economic analysis of micafungin versus liposomal amphotericin B for treatment of candidaemia and invasive candidiasis in Germany

ABSTRACT Objective: To investigate the economic impact of micafungin (MICA) for treatment of invasive candidiasis and candidaemia (systemic Candida infections), a health economic analysis was conducted comparing MICA with liposomal amphotericin B (L-AMB). Research design and methods: The model was based on a phase III, randomised, double-blind, clinical trial which compared MICA with L-AMB. The model entailed a period of 14–20 weeks starting from initiation of treatment and was analysed from a German hospital perspective. Main outcome measures: The main outcome measures were defined as the percentage of patients achieving clinical and mycological response after initial treatment and who were alive at the end of the study (EOS), and the total treatment-associated costs over the study period. Results: The health economic analysis shows that with MICA, 52.9 % of patients are successfully treated and were alive at EOS compared to 49.1 % for L-AMB. In addition, MICA has, on average, lower treatment-associated costs than L-AMB with [euro]43 243 and [euro]49 216 per patient, respectively. Because the costs are lower and the effectiveness is higher for MICA in comparison with L-AMB, MICA is more cost-effective than L-AMB. However, the results of the probabilistic sensitivity analysis show that the differences cannot be considered significant due to a large variance, although MICA remained the most cost-effective option throughout the one-way sensitivity analyses. Conclusions: The lower costs and higher effectiveness reported for MICA versus L-AMB in this analysis indicate that MICA may be a more cost-effective therapy in the treatment of invasive candidiasis and candidaemia when compared with L-AMB.

Cost-effectiveness analysis of micafungin versus caspofungin for treatment of systemic Candida infections in the UK

ABSTRACT Objective: To evaluate the cost-effectiveness of micafungin compared to caspofungin in the treatment of systemic Candida infections (SCIs) in the UK, including invasive candidiasis and candidaemia. Research design and methods: Cost-effectiveness of both echinocandin antifungal drugs was estimated using decision analysis. Response to treatment, resource utilisation, and costs in the model were derived from a phase 3, head-to-head comparative trial. The model includes only data directly related to the treatment of the systemic Candida infection over the study duration (a maximum period of 14 weeks). Transition probabilities were calculated based on the efficacy results from the clinical trial. Main outcome measures: The models effectiveness outcome is surviving patients who are successfully treated, based on the absence of signs and symptoms, radiographic abnormalities, and culture/histologic evidence associated with the fungal infection. In addition, subgroup analyses were performed to identify cost-effectiveness in several specific patient groups. Results: The total medical treatment costs for the micafungin group were £29,095, which is similar to the total costs for the caspofungin group (£29,953). In the micafungin arm 60% of the patients and in the caspofungin arm 58% of the patients were successfully treated and alive. Cost-effectiveness ratio of micafungin was £48,771, and of caspofungin £52,066 per successfully treated patient. Because the costs are lower and the effectiveness is higher for micafungin in comparison with caspofungin, micafungin is more cost-effective than caspofungin. However, probabilistic sensitivity and subgroup analysis show that the differences cannot be considered significant due to a large variance although micafungin remained the most cost-effective option throughout all but one of the sensitivity analyses. Conclusions: Costs and effects of micafungin compare to those of caspofungin in the treatment of systemic Candida infections in the UK. The results indicate that micafungin is cost-effective compared to caspofungin, although the difference was not found to be significant.

Cost-effectiveness analysis of pancreatin minimicrospheres in patients with pancreatic exocrine insufficiency due to chronic pancreatitis

Abstract Objective: Chronic pancreatitis (CP) is the most common cause of pancreatic exocrine insufficiency (PEI). Management of PEI due to CP is achieved through lifelong treatment with pancreatic enzyme replacement therapy (PERT). To the authors’ knowledge, no cost-effectiveness analysis on the benefit of PERT in CP patients with PEI has been performed to date. The objective of this analysis was to examine the cost-effectiveness of Creon (pancreatin minimicrospheres [MMS]), one of the main PERTs available in Poland, in treating patients with CP-related PEI. Methods: The cost-effectiveness of pancreatin MMS in the treatment of patients with CP-related PEI vs no PERT treatment was estimated using a decision analysis based on clinical data from relevant studies. The model horizon was 20 years. Main outcomes included the percentage of patients with controlled PEI, survival, total medical costs, number of quality-adjusted life years (QALYs), and the incremental cost-effectiveness ratio (ICER). All costs were analysed from the Polish payer perspective. Results: The model included clinical data from 176 patients treated in five pancreatin MMS randomized trials. Treatment with pancreatin MMS resulted in a considerably higher proportion of patients with controlled PEI compared to those not treated with any PERT. Over a horizon of 20 years, the total treatment cost and the ICER for pancreatin MMS was €8223 and €6312 per QALY, respectively. Limitations: Important limitations include the lack of long-term and comparative clinical data available. The use of ‘no PERT treatment’ as a comparator against pancreatin MMS treatment may not accurately reflect current practice in Poland. Conclusions: Treatment of CP-related PEI with pancreatin MMS is cost-effective from a Polish payer perspective, with an ICER below the accepted ‘willingness to pay’ threshold of 3-times gross domestic product (GDP) per capita. These results are likely to apply to other European countries.

Payer/Hta Requirements In Metastatic Breast Cancer

INTRODUCTION • Triple-negative breast cancer (TNBC) represents 10%–20% of invasive breast cancers1 and has a very poor prognosis.2 There is a particular unmet need in TNBC, with a lack of clinically established targeted therapies3; chemotherapy is the only option for metastatic TNBC.4 • To facilitate access to new treatments, it is increasingly important to understand payer evidence needs in addition to regulatory evidence requirements.5 • Traditionally, payers focus on hard endpoints such as overall survival (OS), and recommendations for reimbursement would ideally be supported by statistically significant improvement in OS. However, this is sometimes difficult due to long follow-up durations and post-study treatment.

PMC11 IMPUTE INCOMPLETE PATIENT-LEVEL MEDICAL COSTS

PMC7 THE NATURE AND SCALE OF INADEQUATE REPORTING OF DICHOTOMOUS OUTCOMES FROM SIX SYSTEMATIC REVIEWS Bhanderi M, Dimri S, Mahajan A, Ubhadiya BS, Narvilkar P, Agrawal R, Singh N Heron Health Private Ltd, Chandigarh, India OBJECTIVES: Inadequate reporting of outcomes is a major problem while performing meta-analysis. The objective of this study is to estimate the nature and scale of inadequate reporting of dichotomous outcomes. METHODS: Reporting quality of dichotomous outcomes (efficacy and safety) was analysed across six systematic reviews conducted in four disease areas namely oncology (non-small cell lung cancer, colorectal cancer and ovarian cancer), overactive bladder, multiple sclerosis and rheumatoid arthritis. Reporting quality was considered inadequate when either number of patients analysed (N) was not reported or percentage was reported improperly. Reporting of percentage was considered as improper when the number of patients with outcome (n) could not be calculated from the given percentage. This may be the case when more than one value corresponds to same reported percentage (example 5% of 498 could have values between 23 and 27). Analyses were conducted using STATA 9.2. RESULTS: In total, 6408 reported outcomes were included in the analysis. Inadequate reporting of dichotomous outcomes was frequent and observed for 691 (10.78%) outcomes. The estimate of inadequate reporting varied across selected reviews and ranged from 7.36% to 17.41% in overactive bladder and multiple sclerosis, respectively. Estimate of inadequate reporting was similar for the safety and efficacy outcomes (10.53% and 11.19%, respectively). Improper reporting of percentage contributed for approximately two-third (65.56%) of the inadequately reported outcomes whereas for the remaining outcomes, N was missing. CONCLUSIONS: Inadequate reporting of dichotomous outcome was frequently observed among the selected reviews. It was observed that reporting of percentages in published reports are often imperfect (due to rounding of values). This leads to loss of data available for metaanalyses as number of patients with outcome could not be calculated accurately. Our results are indicative of outcome reporting bias which needs to be investigated further.