Patient follow up screening evaluations. Examples with regard to congenital hip dislocation and congenital heart disease

Objective - To discuss the merits of the patient follow up study design for the evaluation of some specific mass screening programmes. Design - Theoretical evaluation illustrated by two examples. Setting - Department of Public Health Erasmus University Rotterdam. Main results - The gold standard for evaluation of favourable effects of screening is the randomised controlled trial (RCT). Application of an RCT, however, is often not feasible, in which cases observational studies will have to be relied on. The case-control study design is generally considered to be second best. In some situations, however, a patient follow up study design may be applicable and may have some major advantages. The use of the patient follow up design for screening evaluation will often be very problematic or even unacceptable, particularly as far as screening for cancer is concerned. The most important objections are resulting from lead time bias, length bias, selection bias and over-treatment bias. For the evaluation of screening for congenital heart disease and congenital hip dislocation in Dutch child health care, however, these objections may relatively simply be overcome. Lead time bias will be of little importance, as the ages of onset of these disorders are fixed, namely at birth, and their ultimate outcomes may be expected within relatively short time. Length bias may largely be avoided by correction for severity of the disorder, which can be adequately assessed by modern diagnostic procedures. Selection bias is generally hard to rule out, but in these cases it probably plays a minor part. Over-treatment can be avoided by the policy of "watchful waiting", which in these disorders can be applied with little risk for fatal outcomes. In principle bias might be avoided more successfully in a case-control screening evaluation than in a patient follow up study. However, the patient follow up study is for both screening programmes discussed here the more feasible design and can provide more supplementary information. The results of two example studies suggest that both screenings probably yield considerable benefits Conclusion - Under a number of specific conditions a patient follow up study is an efficient alternatitve to more customary designs for screening evaluation.