Marten J. Poley - Erasmus Universiteit Rotterdam

General Discussion: A Guided Tour Providing Four Different Views of the Results 1338.5 PERSPECTIVE 4: THE HEALTH ECONOMICS RESEARCHERWe have covered most of our tour. The final view offered is the perspective of thehealth economics researcher. Looking at the results of this thesis from a researchperspective, it attracts attention that evidence on the cost-effectiveness ofneonatal surgery was largely lacking in the past, and, to a lesser extent, still is.In Chapter 1, a review by Stolk and colleagues was cited to show that evidencewas still rudimentary in the year 2000. 21 The studies presented in this thesis andsome other studies brought expansion, but on the whole current evidenceremains limited (Chapter 7). Extending the evidence base on the conditionsstudied in this thesis (ARM, CDH, and, much less, MAS) would be welcome,because, as one reason, the studies in this thesis involved only one country andonly one, sometimes two, pediatric centers. Further evidence is all the moreneeded for the other neonatal surgical diseases, about which this thesis couldonly hypothesize. Apart from this, economic evaluation is not a one-timeexercise, but should be an ongoing effort. Changing patterns of disease andinnovations in treatments may render a once cost-effective technology less costeffective.As Chapter 7 discussed, it is not obvious that current favorable costeffectivenessratios in neonatal surgery will remain unchanged. To give anexample: when new alternative treatment modalities, such as exogenoussurfactant therapy, high-frequency oscillatory ventilation, and inhaled nitric oxide,evolve further, this will have an effect on the cost-effectiveness of ECMO (cf.Chapter 5). To conclude, we should try to get more, and better, data in thefuture. However, this confronts us with the problem that rigorously designedclinical research is difficult to realize in neonatal surgery, as explained inChapter 1. It will require the (inter-)national collaboration of pediatric surgicalcenters, because even the large centers alone do not have the number of patientsneeded to reach an optimal conclusion. Encouragingly, international collaborationhas already started in some areas, such as in CDH (the International CDH StudyGroup) 22 and in pediatric oncology (under the umbrella of the InternationalSociety of Pediatric Oncology (SIOP)).Then, several methodological issues will attract the attention of the researcher.Among the methodological challenges identified in this thesis on the specific areaof neonatal surgery were: the in- or exclusion of future health care costs in addedlife years (Chapters 2 and 3); measuring productivity losses regarding both paidand unpaid activities, in patients or in caregivers (ibidem); measuring utilities forhealth states in children (ibidem); more broadly measuring symptom status andHRQoL in infants and children, partly relying on proxies (in particular Chapter 4);costs and effects accruing to the parents (Chapters 2, 3, and 6); discounting(Chapters 2, 3, and 5); tracking patients who underwent treatment many yearsago, which bears the possibility of large and/or selective nonresponse (passim);and the consideration of equity when prioritizing health care programs forresource allocation (Chapter 7).