Marten J. Poley - Erasmus Universiteit Rotterdam

148SummaryThis thesis addresses the cost-effectiveness of neonatal surgery. Beginning afterthe Second World War, neonatal surgery has been making enormous progress.Mortality rates for the majority of anomalies belonging to the field fell fromalmost 100% to less than 10%. Contemporaneously with these medicaladvancements came new and pressing dilemmas. First, survival is of course animportant measure of success, but improved survival might come at the price ofpoor health-related quality of life (HRQoL) in later life. Second, the economicconsequences of health care have become larger, in an era where budgetaryrestrictions are becoming tighter. As in other branches of medicine, medicaladvancements contributed to increasing costs in neonatal surgery as well. It isagainst this background of concerns about the HRQoL of the surviving infant andthe increasing costs associated with neonatal surgery that the need forinformation about the cost and effects of neonatal surgery was recognized. Thisthesis directly emerges from these concerns, as explained in Chapter 1. Thischapter, which introduces and motivates this thesis, also explains why costsshould be considered in health care, and clarifies that we will not be able to offercertain medical practices that are known to have favorable effects, but whoseeffects are too small to justify the cost. It highlights the relevance of costeffectivenessanalyses of health care, which aim at establishing whether theeffects of a given treatment are worth the budget needed, compared to analternative treatment. For the case of neonatal surgery, results from costeffectivenessanalyses would make it possible to counteract critiques leveledagainst the discipline. Neonatal surgery has been criticized with arguments ofcost-effectiveness: the high costs of an operation have been feared to come withlow HRQoL after survival. Clearly, the current lack of evidence on the costeffectivenessof neonatal surgery is a main problem worth tackling. It threatensto give the discipline a weaker position in health care allocation decisions.This study's main objective is to collect evidence on the cost-effectiveness ofneonatal surgery and to place this evidence in its proper context. The explicitintention is to include a large group of newborns, to adopt a long-term timehorizon since treatment may have impacts on cost and outcomes basically over apatient's life time, and to study a wide range of relevant costs and effects. So, thestudy does not focus exclusively on the health care costs during the initialhospitalization, as earlier studies often did. Yet, a wide range of other costcategories is assessed, including direct non-healthcare costs (such as out-ofpocketexpenses), indirect health care costs (future costs in added life-years),and indirect non-healthcare costs (productivity costs). Furthermore, the timehorizon will be sufficiently long to capture all significant effects. After all, many ofthe most important outcomes of neonatal surgery, such as language or cognitiveabilities, educational attainment, and adult employment status, aredevelopmentally programmed to occur years if not decades after the intervention.Also, to gain a full understanding of the long-term outcomes of neonatal surgicaldiseases, we will include various outcome measures, such as measures ofsymptomatology and HRQoL, and not only traditional ones such as avoided