Health economic modelling in Cystic Fibrosis:A systematic review

Introduction: Cystic Fibrosis (CF) is a heritable chronic condition. Due to the genetic and progressive nature of CF, a number of interventions are available for the condition. In the United Kingdom (U.K.) average annual cost of CF treatment is between €49,000 to €76,000 (2012) per patient [1]. A review of health economic modelling studies is warranted to provide decision makers and researchers with an in depth understanding of modelling practices in CF and guidance for future research. Methods: Online searches were performed in the 5 databases, studies were included if they were: a) Model based economic evaluation for management of Cystic Fibrosis. Articles were restricted to English language only, but no restriction was applied on publication year. Results: Nine studies were reviewed, most were Markov cohort models. Models evaluated pharmaceutical interventions and drug adherence. Modelling structure was consistent across most articles and a range of sources were used to populate the models. Cost and utility data were based on different sources and elicitation methods respectively. The majority of models failed to incorporate significant health events which impact both cost and disease progression. Conclusion: In our review we observed a lack of, application of European Medicines Agency (EMA) guidelines for clinical trial endpoints, model structure justifications and lastly, health-related quality of life derived utility information around important clinical events. Future work around conceptual modelling of CF progression, utility valuation of significant health events and meeting EMA guidelines for trial reporting is encouraged

Small intestinal bacterial overgrowth in patients with progressive familial intrahepatic cholestasis

Background & Aims: To date, no studies concerning the presence of small intestinal bacterial overgrowth in patients with progressive familial intrahepatic cholestasis were published. Based upon characteristic of progressive familial intrahepatic cholestasis one can expect the coexistence of small intestinal bacterial overgrowth. The aim of the study was to assess the incidence of small intestinal bacterial overgrowth in patients with progressive familial intrahepatic cholestasis. Methods: 26 patients aged 8 to 25 years with progressive familial intrahepatic cholestasis were included in the study. Molecular analysis of ABCB11 gene was performed in the vast majority of patients. In all patients Z-score for body weight and height, biochemical tests (bilirubin, bile acid concentration, fecal fat excretion) were assessed. In all patients hydrogen-methane breath test was performed. Results: On the basis of first hydrogen-methane breath test, diagnosis of small intestinal bacterial overgrowth was confirmed in 9 patients (35%), 5 patients (19%) had borderline results. The second breath test was performed in 10 patients: in 3 patients results were still positive and 2 patients had a borderline result. The third breath test was conducted in 2 patients and positive results were still observed. Statistical analysis did not reveal any significant correlations between clinical, biochemical and therapeutic parameters in patients with progressive familial intrahepatic cholestasis and coexistence of small intestinal bacterial overgrowth. Conclusions: Our results suggest that small intestinal bacterial overgrowth is frequent in patients with progressive familial intrahepatic cholestasis. Moreover, it seems that this condition has the tendency to persist or recur, despite the treatment

Single dose of green tea extract decreases lipid digestion and absorption from a test meal in humans

Background & Aims: Green tea is known worldwide for its high content of polyphenolic compounds and multifactorial beneficial effects on human health. The role of green tea as an inhibitor of lipid hydrolysis is widely discussed. The aim of the study was to assess the influence of green tea extract on lipid digestion and absorption. Methods: The study comprised 32 healthy volunteers aged 23 to 30 years with normal exocrine pancreatic function. In all subjects 13C-labelled mixed triglyceride breath test was performed twice with and without green tea extract ingestion. Cumulative percentage dose recovery was considered to reflect digestion and absorption of lipids. Values are expressed as medians and 1st-3rd quartile distribution. Results: In all subjects, cumulative percentage dose recovery values were normal in a placebo test (36.8% ). These results were significantly higher (p=0.021) than those obtained in green tea extract test (28.8% ). Results of six tests with GTE were abnormal. Conclusions: Single dose of green tea extract taken with a test meal decreases lipid digestion and absorption in humans

Relative cost-effectiveness of using an extensively hydrolyzed casein formula containing the probiotic Lactobacillus rhamnosus GG in managing infants with cow's milk allergy in Poland

Julian F Guest,1,2 Diana Weidlich,1 Maciej Kaczmarski,3 Elzbieta Jarocka-Cyrta,4 Natalia Kobelska-Dubiel,5 Agnieszka Krauze,6 Iwona Sakowska-Maliszewska,7 Anna Zawadzka-Krajewska8 1Catalyst Health Economics Consultants, Northwood, Middlesex, 2Faculty of Life Sciences and Medicine, King’s College, London, UK; 3Department of Pediatrics, Gastroenterology, and Allergology, Medical University of Bialystok, Białystok, 4Uniwersytet Warmińsko‑Mazurski, Wydział Nauk Medycznych, Katedra Pediatrii Klinicznej, Olsztyn, 5Department of Pediatric Gastroenterology and Metabolic Diseases, Poznań University of Medical Sciences, Poznań, 6Klinika Pneumonologii i Alergologii Wieku Dziecięcego, Warsaw, 7Poradnia Gastroenterologiczna Centrum Pediatrii, Sosnowiec, 8Department of Pediatric Pneumonology and Allergology, Medical University of Warsaw, Warsaw, Poland Objective: To estimate the cost-effectiveness of using an extensively hydrolyzed casein formula (eHCF) containing the probiotic Lactobacillus rhamnosus GG (eHCF + LGG; Nutramigen LGG) as an initial treatment for cow’s milk allergy compared with eHCF alone and amino acid formulas (AAF) in Poland from the perspective of the Polish National Health Fund (Narodowy Fundusz Zdrowia [NFZ]) and parents. Methods: Decision modeling was used to estimate the probability of cow’s milk allergic infants developing tolerance to cow’s milk by 18 months. The model also estimated the cost to the NFZ and parents (Polish Zloty [PLN] at 2013–2014 prices) for managing infants over 18 months after starting one of the formulas as well as the relative cost-effectiveness of each of the formulas. Results: The probability of developing tolerance to cow’s milk by 18 months was higher among infants who were fed eHCF + LGG (0.82) compared with those fed eHCF alone (0.53) or an AAF (0.22). An infant who is initially managed with eHCF + LGG is expected to consume fewer health care resources than infants managed with the other formulas. Hence, the estimated total health care cost incurred by the NFZ for initially feeding infants with eHCF + LGG (PLN 5,693) was less than that of feeding infants with eHCF alone (PLN 7,749) or an AAF (PLN 24,333). However, the total cost incurred by parents for initially feeding infants with an AAF (PLN 815) was marginally less than that of feeding with eHCF + LGG (PLN 993), which was less than that of feeding with eHCF alone (PLN 1,226). Conclusion: Using eHCF + LGG instead of eHCF alone or an AAF for first-line management of newly diagnosed infants with cow’s milk allergy affords a cost-effective use of NFZ-funded resources, since it improves outcome for less cost. Whether eHCF + LGG would be viewed as being cost-effective by parents is dependent on their willingness to pay an additional cost for additional tolerance acquisition to cow’s milk. Keywords: amino acid formula, cost-effectiveness, cow’s milk allergy, extensively hydrolyzed formula, Lactobacillus Rhamnosus GG, Polan