Real-World Treatment Patterns and Outcomes in Patients with Head and Neck Cancer: Point-in-Time Survey of Oncologists in Italy and Spain.

INTRODUCTION: Squamous cell carcinomas of the head and neck (SCCHN) account for approximately 4% of all malignancies and are associated with high morbidity and poor prognosis. For patients who are not eligible for surgery or radiotherapy, treatment options are limited, especially for those with recurrent or metastatic (R/M) disease. Current European guidelines recommend first-line (1L) treatment with palliative chemotherapy, using biologic or platinum-based regimens. In the absence of new clinical trials in SCCHN, the use of real-world data has facilitated the assessment of treatment patterns and outcomes in different healthcare systems. This study reports on the 1L treatment of platinum-eligible patients with R/M SCCHN in Italy and Spain. METHODS: A point-in-time survey of the management of patients with R/M SCCHN was completed by clinical oncologists in Italy and Spain between October 2018 and February 2019. Patient demographics and clinical characteristics were obtained by retrospective chart review, whilst participating patients self-reported the impact of their disease on their quality of life (QoL) and well-being. RESULTS: A total of 436 patients were recruited from Italy (216) and Spain (220). Patient demographics for both countries comprised mostly male patients, aged 65 years in Italy and 63 in Spain on average. The primary site for the SCCHN was the pharynx and 36% of patients had metastatic disease overall. EXTREME or cetuximab-based regimens were the most common treatments administered at 1L (52% in Italy and 78% in Spain). Scores on the FACT-G in both countries were substantially lower than those of the general and other advanced cancer populations, while scores on the EQ-5D were clinically meaningfully lower than local population norms. CONCLUSION: Despite 1L treatment of platinum-eligible patients with R/M SCCHN in Italy and Spain following current European guidelines, patients' QoL remains poor, which highlights the need for alternative treatments that could improve clinical outcomes.

Preferences for Renal Cell Carcinoma Pharmacological Treatment: A Discrete Choice Experiment in Patients and Oncologists.

INTRODUCTION: The purpose of this investigation was to explore patients' and oncologists' preferences for the characteristics of a pharmacological regimen for patients with advanced renal cell carcinoma (aRCC). MATERIAL AND METHODS: Cross-sectional observational study based on a discrete choice experiment (DCE) conducted in Spain. A literature review, a focus group with oncologists and interviews with patients informed the DCE design. Five attributes were included: progression survival gain, risk of serious adverse events (SAEs), health-related quality of life (HRQoL), administration mode, and treatment cost. Preferences were analyzed using a mixed-logit model to estimate relative importance (RI) of attributes (importance of an attribute in relation to all others), which was compared between aRCC patients and oncologists treating aRCC. Willingness to pay (WTP, payer: health system) for a benefit in survival or in risk reduction and maximum acceptable risk (MAR) in SAEs for improving survival were estimated from the DCE. Subgroup analyses were performed to identify factors that influence preference. RESULTS: A total of 105 patients with aRCC (77.1% male, mean age 65.9 years [SD: 10.4], mean time since RCC diagnosis 6.3 years [SD: 6.1]) and 67 oncologists (52.2% male, mean age 41.9 years [SD: 8.4], mean duration of experience in RCC 10.2 years [SD: 7.5]) participated in the study. The most important attribute for patients and oncologists was survival gain (RI: 43.6% vs. 54.7% respectively, p<0.05), followed by HRQoL (RI: 35.5% vs. 18.0%, respectively, p<0.05). MAR for SAEs was higher among oncologists than patients, while WTP (for the health system) was higher for patients. Differences in preferences were found according to time since diagnosis and education level (patients) or length of professional experience (oncologists). CONCLUSION: Patients' and oncologists' preferences for aRCC treatment are determined mainly by the efficacy (survival gain) but also by the HRQoL provided. The results of the study can help to inform decision-making in the selection of appropriate aRCC treatment.

Patient characteristics and stroke and bleeding events in nonvalvular atrial fibrillation patients treated with apixaban and vitamin K antagonists: a Spanish real-world study.

Aim: To compare the risk of stroke, systemic thromboembolism and bleeding, in patients initiating apixaban or acenocoumarol for the treatment of nonvalvular atrial fibrillation. Methods: An observational, retrospective study was performed using medical records of patients who initiated apixaban or acenocoumarol between 2015 and 2017. Propensity score matching was used to match patients; stroke, systemic thromboembolism, major and minor bleeding events were compared between the matched patients. Results: Patients who were prescribed apixaban had a lower rate of systemic embolism/stroke (hazard ratio [HR] = 0.54; 95% CI: 0.38-0.78; p = 0.001), minor bleeding (HR = 0.64; 95% CI: 0.52-0.79; p < 0.001) and major bleeding (HR = 0.51; 95% CI: 0.37-0.72; p < 0.001). Conclusion: Patients prescribed apixaban for the treatment of nonvalvular atrial fibrillation had lower rates of thromboembolic events and minor/major bleeding than patients on acenocoumarol.

Cost per response for abatacept versus adalimumab in patients with seropositive, erosive early rheumatoid arthritis in the US, Germany, Spain, and Canada.

BACKGROUND: Effective treatment of rheumatoid arthritis (RA) with biologic DMARDs poses a significant economic burden. The AMPLE (Abatacept versus adaliMumab comParison in bioLogic-naïvE RA subjects with background methotrexate) trial was a head-to-head, randomized study comparing abatacept with adalimumab. A post hoc analysis showed improved efficacy for abatacept in patients with versus without seropositive, erosive early RA. OBJECTIVE: The aim of the current study was to evaluate the cost per response (ACR20/50/70/90 and HAQ-DI) and patient in remission (DAS28-CRP, CDAI, and SDAI) for abatacept relative to adalimumab, in patients with seropositive, erosive early RA in the US, Germany, Spain, and Canada. METHODS: A previously published model was used to compare abatacept and adalimumab in a cohort of 1000 patients over 2 years. Clinical inputs were updated based on two subpopulations from the AMPLE trial. Cohort 1 included patients with early RA (disease duration ≤ 6 months), RF and/or ACPA seropositivity, and > 1 radiographic erosion. Cohort 2 included patients with RA in whom at least one of these criteria was absent. RESULTS: For cohort 1, all incremental costs per additional health gain (patient response or patient in remission) favoured abatacept in all countries, except for DAS28-CRP remission in Canada. Cost savings versus adalimumab were greater when more stringent response criteria were applied and also in cohort 1 patient (versus cohort 2 patients). CONCLUSION: The cost per responder and patient in remission favoured abatacept in patients with seropositive, erosive early RA across all the countries. In this patient population, the use of abatacept instead of adalimumab can lead to lower costs in the US, Germany, Spain, and Canada.

Patient preferences for treatment of multiple sclerosis with disease-modifying therapies: a discrete choice experiment.

OBJECTIVES: To assess disease-modifying therapy (DMT) preferences in a population of patients with multiple sclerosis (MS) and to estimate the association between sociodemographic and clinical factors and these preferences. METHODS: Preferences for DMTs attributes were measured using a discrete choice experiment. Analysis of preferences was assessed using mixed-logit hierarchical Bayes regression. A multilinear regression was used to evaluate the association between the preferences for each attribute and patients' demographic and clinical characteristics. A Student's t-test or Welch's t-test was used for subgroup comparisons. RESULTS: A total of 125 patients were included in the final analysis (62.9% female, mean age 44.5 years, 71.5% with relapsing-remitting MS diagnosis). The most important factor for patients was the possibility of suffering from the side effects of the treatment (relative importance [RI] =50%), followed by a delay in disease progression (RI =19.4%), and route and frequency of administration (RI =14.3%). According to maximum acceptable risk, patients were willing to accept an increase of 3.8% in severity of side effects, for a delay of 1 year in disease progression. Treatment duration was the most prevalent factor affecting preferences, followed by the age of patients, type of MS, level of education, and the type of current treatment. Patients treated orally were significantly more concerned about the route and frequency of administration (P=0.026) than patients on injectable therapy. Naïve patients stated significantly less importance to prevention of relapses (P=0.021) and deterioration of the capacity for performing usual daily life activities (P=0.015). Finally, patients with >5 years since diagnosis were significantly less concerned about preventing disease progression (P=0.021), and more concerned about treatment side effects (P=0.052) than compared with patients with <5 years of MS history. CONCLUSION: The most important attribute for MS patients was side effects of DMTs, followed by delay in disability progression. Experience with DMTs and time since MS diagnosis changed patients' preferences. These results give information to adjust new DMT treatment in order to satisfy patients' preferences and therefore, improve adherence to treatment.

Savings associated with high-dose hypoallergenic house dust mite immunotherapy in rhinitis and/or asthma patients in Spain.

OBJECTIVES: To quantify the cost difference between conventional symptomatic treatment of mite allergy and specific subcutaneous immunotherapy (SCIT). METHODS: Observational, retrospective, and multicenter study was carried out in Spain in 2013. The medical records of 419 patients diagnosed with rhinitis and/or bronchial asthma for mite allergy were retrieved. Mean age was 24.9 years (standard deviation 14.4). The use of symptomatic medication (rescue and daily), diagnostic tests, unscheduled medical care, and sick leave days associated with SCIT treatment versus no-SCIT treatment was compared. Also measured was the SCIT treatment to no-SCIT treatment costs ratio: used resources (symptomatic medication, unscheduled medical care, diagnostic tests, and 3 years SCIT treatment and sick leave days) were quantified in euros. Efficacy (decreased resource usage) of first-year treatment was assumed during the remaining 2 years and also during the 3-year follow-up period. RESULTS: After a single year of SCIT, all quantified resources diminished significantly (P<0.05) from baseline. Estimated reduction in cost items included hospital resources (100% in hospitalizations, 82% in visits to the allergist, and 79% in emergency room visits), therapies (56% in rescue medication and 63% in daily medication), diagnostic tests (77%), and sick leave days (94%). Ratio of comparative calculation described as SCIT treatment versus non-SCIT treatment (or conventional symptomatic treatment) is 0.8. CONCLUSION: Direct costs are reduced by 64% and indirect costs by 94%. SCIT of hypoallergenic preparation of dust mite (Acaroid(®)) allows cost savings versus conventional treatment. Estimated savings for the public National Health System are 5.7 times the cost of immunotherapy.

[Cost-effectiveness analysis of universal screening for thyroid disease in pregnant women in Spain]. / Análisis coste-efectividad del cribado universal de la enfermedad tiroidea en mujeres embarazadas en España

OBJECTIVE: To assess the cost-effectiveness of universal screening for thyroid disease in pregnant women in Spain as compared to high risk screening and no screening. METHODOLOGY: A decision-analytic model comparing the incremental cost per quality-adjusted life year (QALY) of universal screening versus high risk screening and versus no screening. was used for the pregnancy and postpartum period. Probabilities from randomized controlled trials were considered for adverse obstetrical outcomes. A Markov model was used to assess the lifetime period after the first postpartum year and account for development of overt hypothyroidism. The main assumptions in the model and use of resources were assessed by local clinical experts. The analysis considered direct healthcare costs only. RESULTS: Universal screening gained .011 QALYs over high risk screening and .014 QALYS over no screening. Total direct costs per patient were €5,786 for universal screening, €5,791 for high risk screening, and €5,781 for no screening. Universal screening was dominant compared to risk-based screening and a very cost-effective alternative as compared to no screening. Use of universal screening instead of high risk screening would result in €2,653,854 annual savings for the Spanish National Health System. CONCLUSIONS: Universal screening for thyroid disease in pregnant women in the first trimester is dominant in Spain as compared to risk-based screening, and is cost-effective as compared to no screening (incremental cost-effectiveness ratio of €374 per QALY). Moreover, it allows diagnosing and treating cases of clinical and subclinical hypothyroidism that may not be detected when only high-risk women are screened.