Cost-effectiveness of infant hypoallergenic formulas to manage cow's milk protein allergy in France.

Background: Clinician's choice of hypoallergenic formulas in the first-line management of cow's milk protein allergy (CMPA) should be informed by evidence on clinical efficacy and cost-effectiveness. Objective: We compare the cost-effectiveness of amino acid-based formula (AAF), extensively hydrolyzed casein formula with Lactobacillus rhamnosus Gorbach Goldin (EHCF+LGG), extensively hydrolyzed whey formula (EHWF), and rice hydrolyzed formula (RHF) in non-breastfed children in France. Methods: Immunotolerance and atopic manifestations' prevalence were based on a prospective non-randomized study with a 36-month follow-up. Resource utilization was sourced from a survey of French clinicians, and unit costs were based on national data. Costs and health consequences were discounted at 2.5% annually. Results were reported using the Collective and French National Health Insurance perspectives. Results: Children receiving EHCF+LGG were predicted to require less healthcare resources, given their reduced prevalence of CMPA symptoms at 3 years. In the base case, EHCF+LGG led to savings of at least €674 per child compared to AAF, EHWF, and RHF at 3 years, from both perspectives. Nutrition had the highest economic burden in CMPA, driven by hypoallergenic formulas and dietetic replacements costs. Results were robust to one-way and probabilistic sensitivity analyses. Conclusions: EHCF+LGG was associated with more symptom-free time, higher immune tolerance, and lower costs.

Managing Cow's Milk Protein Allergy in Indonesia: A Cost-effectiveness Analysis of Hypoallergenic Milk Formulas from the Private Payers' Perspective.

Background: Cow's milk protein allergy is very common in early childhood. Extensively hydrolyzed formulas are recommended in the first-line management of cow's milk protein allergy in non-breastfed children. Choice of formulas should be informed by efficacy and cost data. Objectives: This study aims to compare the cost-effectiveness of extensively hydrolyzed casein formula with Lacticaseibacillus rhamnosus Gorbach Goldin (EHCF+LGG), extensively hydrolyzed whey formula (EHWF), amino acid formula, and soy formula in the first-line management of cow's milk protein allergy in non-breastfed children in Indonesia. Methods: A trial-based decision analytic cohort model was adapted to simulate the occurrence of cow's milk protein allergy symptoms or being symptom free. The model was based on a prospective nonrandomized study that followed up children for 36 months. Costs and health consequences were discounted at 3% annually. Resources required to manage cow's milk protein allergy and unit costs for clinical appointments and exams were based on a panel of 15 clinicians, from a private payers' perspective. Other unit costs were based on publicly available national data. Results were reported as cost per additional child free from allergic manifestations or per additional immunotolerant child at 3 years, and per life-years under the same conditions. Uncertainty was assessed using deterministic and probabilistic sensitivity analysis. Results: Children receiving EHCF+LGG were associated with more symptom-free time, a higher probability of cow's milk tolerance at 3 years, and lower healthcare resources and transportation use when compared with children receiving other formulas (with 38%-49% lower costs). Formula costs were lower for soy, but EHCF+LGG was predicted to save 9% and 54% of overall costs compared with extensively hydrolyzed whey formula and amino acid formula, respectively. Results were robust to sensitivity analyses. Conclusion: Use of EHCF+LGG resulted in more symptom-free time and the highest 3-year probability of cow's milk tolerance. It also led to healthcare resource and transportation savings when compared with other hypoallergenic milk formulas. Soy formula remained an alternative if formula price represents a major constraint.

Cost-Effectiveness Analysis of Obinutuzumab for Previously Untreated Chronic Lymphocytic Leukaemia in Portuguese Patients who are Unsuitable for Full-Dose Fludarabine-Based Therapy.

BACKGROUND: Chronic lymphocytic leukaemia (CLL) mostly affects patients with comorbidities and limited therapeutic options. Obinutuzumab in combination with chlorambucil (GClb) is a new therapeutic option for previously untreated CLL patients who are unsuitable for full-dose fludarabine-based therapy. This combination delays disease progression but incurs additional costs; thus, an assessment of its value for money is relevant. OBJECTIVE: To estimate the incremental cost-utility ratio of GClb in comparison with (i) rituximab in combination with chlorambucil (RClb), and (ii) chlorambucil alone (Clb) from the perspective of the Portuguese National Health Service (NHS). METHODS: A Markov model was used to predict disease progression. Pre-progression clinical data were based on the latest CLL11 trial data, and post-progression clinical data were obtained from CLL5 trial data. Utility values are from Kosmas et al. (Leuk Lymphoma 56:1320-1326, 14). Only direct medical costs were included. The resource consumption was estimated by a panel of Portuguese experts, and the unit costs were obtained from official sources. A discount rate of 5% was applied to costs and consequences. RESULTS: GClb and RClb were associated with an increase of 1.06 and 0.39 quality-adjusted life-years (QALY) at an additional cost of €21,720 and €9836 when compared to Clb, respectively. The cost-utility ratio of GClb versus Clb was €20,397/QALY, while RClb was extendedly dominated. CONCLUSIONS: The use of GClb for previously untreated CLL patients who are unsuitable for full-dose fludarabine-based therapy incurs an incremental cost per QALY that is generally accepted in Portugal. Therefore, although there is some uncertainty, obinutuzumab is probably a cost-effective therapy in the Portuguese setting.

Colorectal cancer in Portugal.

Increasing diagnosis and deaths caused by colorectal cancer (CRC) warrant closer examination of affected patients and focus on management of CRC in Portugal. In order to assess the extent and quality of the information available in Portugal, we first analyse Portuguese cancer registries and then the management of CRC by discussing the diagnostic process and medical care provided, especially pharmaceuticals. Other cancer indications are mentioned in order to illustrate current approaches of cancer in Portugal. Current national data on cancer patients are scarce and there are divergencies in methods of data collection and treatment amongst regional cancer registries. However, the available data is sufficient enough to understand the dimension of CRC, with age-standardised incidence of 37 per 100,000 and mortality of 31 per 100,000 annually. An ongoing project is restructuring health services to improve efficiency and quality, however, some problems exist. The regional inequity of access to health care facilities and long waiting times for diagnostic examinations and surgery are major examples. Despite the non-availability of clinical guidelines, a pilot screening programme started at the beginning of 2009 in the Centre Region of the country. It is hoped that this overview will provide the basis for discussion on improvements in CRC management in Portugal and lead to better outcomes.