Dangerous and expensive screening and treatment for rare childhood diseases: the case of Krabbe disease.

[krabbe disease]

Public policy surrounding newborn screening is in flux . New technology allows more screening for more diseases at lower cost . Traditional criteria for target diseases have been criticized by leading health policymakers . The example of newborn screening for Krabbe disease highlights many of the dilemmas associated with population-based screening programs . Krabbe is difficult to diagnose , variable in its natural history , and does not always respond to treatment . The only available treatment is hematopoietic stem cell transplantation , which is expensive , risky , and of uncertain efficacy . This article analyzes the debate about Krabbe as an example of the sorts of debates that will likely arise for many more diseases over the next decade . I conclude that pilot programs in pioneer states should be carefully evaluated before testing for Krabbe is universalized .