In a randomized controlled trial in patients with locally advanced squamous cell carcinoma of the head and neck (LA SCCHN), treatment with RT plus cetuximab resulted in improved survival compared to treatment with RT alone. Uncertainty exists about the generalizability of the trial results for the Dutch healthcare setting due to possible discrepancies in treatment allocation. Retrospective patient chart review was performed for 141 patients treated with first line RT plus cetuximab or RT alone, diagnosed in 2007-2010 in two head and neck treatment centers. Combined with aggregated population-based data from the Netherlands Cancer Registry and patient level clinical trial data, use of cetuximab in Dutch daily practice was assessed through comparison of patient characteristics, treatment characteristics and treatment outcomes between trial and daily practice. 61 daily practice patients fulfilled the selection criteria. In line with Dutch guidelines, RT plus cetuximab is prescribed in patients requiring combined therapy unfit to receive traditional platinum-based chemotherapeutics. These patients have unfavorable baseline characteristics, due to selection on - amongst others - high age of the patients. Beyond 1 year after treatment start, patients treated with RT plus cetuximab in daily practice died earlier than patients treated with RT plus cetuximab in the trial. Selective treatment allocation in daily practice limits generalizability of EMR 062202-006 trial results. Evidence is needed about the effectiveness of RT plus cetuximab compared to other treatments for patients with unfavorable clinical baseline characteristics.@en

For patients with recurrent and/or metastatic squamous cell carcinoma of the head and neck (R/M SCCHN), chemotherapy can prolong life and alleviate symptoms. However, expected gains may be small, not necessarily outweighing considerable toxicity and high costs. Treatment choice is to a large extent dependent on preferences of doctors and patients and data on these choices are scarce. The purpose of this study is to obtain real-world information on palliative systemic treatment and costs of R/M SCCHN in the Netherlands. In six Dutch head and neck treatment centers, data were collected on patient and tumor characteristics, treatment patterns, disease progression, survival, adverse events, and resource use for R/M SCCHN, between 2006 and 2013. 125 (14 %) out of 893 R/M SCCHN patients received palliative, non-trial first-line systemic treatment, mainly platinum + 5FU + cetuximab (32 %), other platinum-based combination therapy (13 %), methotrexate monotherapy (27 %) and capecitabine monotherapy (14 %). Median progression-free survival and overall survival were 3.4 and 6.0 months, respectively. 34 (27 %) patients experienced severe adverse events. Mean total hospital costs ranged from €10,075 (±€9,891) (methotrexate monotherapy) to €39,459 (±€21,149) (platinum + 5FU + cetuximab). Primary cost drivers were hospital stays and anticancer drug treatments. Major health care utilization and costs are involved in systemically treating R/M SCCHN patients with a limited survival.@en

Clinical trial EMR 62202-006 demonstrates prolonged median locoregional control (24.4 vs. 14.9 months), progression-free survival (17.1 vs. 12.4 months) and overall survival (49.0 vs. 29.3 months) for patients who receive cetuximab added to the comparator radiotherapy for locally advanced squamous cell carcinoma of the head and neck (LA SCCHN). In the Netherlands, hospitals receive reimbursement for cetuximab conditional on cost-effectiveness in daily practice. To estimate the real-world incremental cost per quality adjusted life-year (QALY) gained for radiotherapy + cetuximab over radiotherapy alone in first line treatment of LA SCCHN, a Markov model is constructed with health states “alive without progression”, “alive following progression” and “death”. Transition probabilities per month are estimated from clinical trial data and retrospectively collected real-world data from two Dutch head and neck cancer treatment centres (2007–2010, n = 141). 5-year, 10-year and lifetime horizons are used, without and with discounting (4 % costs, 1.5 % effects) to calculate incremental cost-effectiveness ratios. Two scenarios explore different assumptions on prognosis of real-world versus trial patients. Adding cetuximab to radiotherapy results in increased costs and health gains in both scenarios and across each of the time horizons. Incremental costs per QALY gained range between €14,624 and €38,543 in the base-case. For a willingness to pay of €80,000 per QALY, the acceptability curves for the different scenarios show probabilities between 0.76 and 0.87 of radiotherapy + cetuximab being cost-effective compared to radiotherapy alone. Current results show the combined treatment of radiotherapy + cetuximab to be a cost-effective treatment option for patients with LA SCCHN.@en