BENEFIT-RISK ANALYSIS IN ABSENCE OF CLINICAL EVIDENCE- DECIDING FOR TREATMENT OF SKULL DEFORMITY IN BABIES AGED 5 MONTHS

Author(s)

van Til JA1, van der Maas EW1, Boere-Boonekamp MM1, van Vlimmeren L2, IJzerman MJ11University of Twente, Enschede, Overijssel, Netherlands, 2Radboud University Nijmegen, Nijmegen, Gelderland, Netherlands

OBJECTIVES: Skull Deformaty (SD) is a flattening of the head as a result of pressure on the malleable skull in infants in the first months of life. Presently, a RCT is conducted to compare the effect of an orthotic helmet to the natural recovery of skull shape in the first three years of life. Burden of treatment is considerable; the helmet has to be worn 23 hours a day for at least 6 months. Possible harms include acceptation problems, pressure wounds and severe skin rash or eczema. The harms of treatment are perceived as an important reason for low adherence to and parental refusal of helmet treatment. The objective of this study is to estimate the risk-benefit trade-off in SD management in pediatric physiotherapists. METHODS: A discrete choice experiment was performed with the most important attributes of SD management. A total of 267 pediatric physiotherapists stated their preference for treatment of a 5 month old child with SD. A three scenario design was chosen Each scenario was characterized by its effect, its burden and the harms of treatment. Logistical regression analysis was performed to analyze the results of the discrete choice experiment. RESULTS: Not surprisingly, child physiotherapists’ ideal treatment has a high probability of timely success with low burden and minimal harms. At present, most attributes indicate a strong preference for awaiting natural recovery. Risk benefit assessment favoring the helmet will only be attained if the helmet can show highly significant clinical benefit. CONCLUSIONS: This study shows that risk benefit analysis can give early indications on the potential of a treatment, by estimating the effectiveness at which the treatment becomes more favorable than its comparator. Whether the risk-benefit analysis will be in favor of helmet treatment in the case of SD, is questionable, as earlier studies have not demonstrated superiority of the helmet

Conference/Value in Health Info

2011-11, ISPOR Europe 2011, Madrid, Spain

Value in Health, Vol. 14, No. 7 (November 2011)

Code

PMD78

Topic

Health Technology Assessment

Topic Subcategory

Decision & Deliberative Processes

Disease

Pediatrics

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