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Background: Five diseases are currently screened on dried blood spots in France through the national newborn screening programme. Tandem mass spectrometry (MS/MS) is a technology that is increasingly used to screen newborns for an increasing number of hereditary metabolic diseases. Medium chain acyl-CoA dehydrogenase deficiency (MCADD) is among these diseases. We sought to evaluate the cost-effectiveness of introducing MCADD screening in France. Methods: We developed a decision model to evaluate, from a societal perspective and a lifetime horizon, the cost-effectiveness of expanding the French newborn screening programme to include MCADD. Published and, where available, routine data sources were used. Both costs and health consequences were discounted at an annual rate of 4%. The model was applied to a French birth cohort. One-way sensitivity analyses and worst-case scenario simulation were performed. Results: We estimate that MCADD newborn screening in France would prevent each year five deaths and the occurrence of neurological sequelae in two children under 5 years, resulting in a gain of 128 life years or 138 quality-adjusted life years (QALY). The incremental cost per year is estimated at €2.5 million, down to €1 million if this expansion is combined with a replacement of the technology currently used for phenylketonuria screening by MS/MS. The resulting incremental cost-effectiveness ratio (ICER) is estimated at €7 580/QALY. Sensitivity analyses indicate that while the results are robust to variations in the parameters, the model is most sensitive to the cost of neurological sequelae, MCADD prevalence, screening effectiveness and screening test cost. The worst-case scenario suggests an ICER of €72 000/QALY gained. Conclusions: Although France has not defined any threshold for judging whether the implementation of a health intervention is an efficient allocation of public resources, we conclude that the expansion of the French newborn screening programme to MCADD would appear to be cost-effective. The results of this analysis have been used to produce recommendations for the introduction of universal newborn screening for MCADD in France. [ABSTRACT FROM AUTHOR]

INTRODUCTION:Budget impact analysis (BIA) provides short and medium-term estimated effect of new health interventions on budgets and resources. Since January 2016, BIA is required as part of economic dossiers submitted to the French National Authority for Health (HAS) by manufacturers for innovative drugs with an expected 2-year sales revenue above EUR50 million. To this end and in order to promote good practices for conducting BIA, HAS developed a guide for BIA.Our objectives are: –to present the guide development method and the resulting recommendations;–to compare the HAS BIA guide with existing BIA guides.METHODS:The HAS guide development process rests on findings derived from a systematic literature review on BIA methodology, an HAS retrospective investigation of BIA, public consultation, international expert advice, and approval from the HAS Board and Committee of Health Economic and Public Evaluation (CEESP). Relevant publications were identified through Pubmed and EMBASE and the grey literature (search dates: January 2000 to June 2016).RESULTS:The search strategy captured 144 publications of which 31 were retained (14 methodological papers, 12 national guides and 5 learned society recommendations). On the basis of this result, an extraction template was designed to synthesize the methodological aspects of BIA. Based on its research findings, HAS developed its first BIA guide which includes recommendations on the following main topics: BIA definition, perspective, populations, time horizon, compared scenarios, BIA models, costing, discounting, choice of clinical data, reporting of BIA and uncertainty exploration.Compared to existing BIA guides from other Health Technology Assessment (HTA) agencies, the HAS guide specifically described issues relating to off-label use of drugs, disease-related costs and scenario analysis.CONCLUSIONS:It is expected that the HAS BIA guide will improve the quality, transparency and standardization of BIA in France. It should also enhance the usefulness of BIA as an essential part of a comprehensive economic assessment of health care interventions. [ABSTRACT FROM PUBLISHER]