Research: Is the cost of treating a hereditary neuromuscular disease affected by the timing of treatment?

In the analysis of data pertaining to 149 patients, (93 untreated, 42 treated after symptoms arose, and 14 treated after early diagnosis), the total societal cost was lower in untreated patients (due to high drug costs in treated patients), but costs were lower for treated patients.

Early detection and treatment of children with spinal muscular atrophy (SMA), a genetic ailment characterised by muscle weakness and wasting, can minimise the total financial expenditures associated with the condition, according to new research.

The research is published in the journal, "Developmental Medicine & Child Neurology."

In the analysis of data pertaining to 149 patients, (93 untreated, 42 treated after symptoms arose, and 14 treated after early diagnosis), the total societal cost was lower in untreated patients (due to high drug costs in treated patients), but costs were lower for treated patients who were identified by newborn screening than for treated patients identified due to the development of symptoms.

"These data are important as they are issued from a real-life perspective collection. They demonstrate clearly that as long as the decision to reimburse treatments for SMA has been made, newborn screening becomes a no-brainer--not only because it gives patients a much better future, but also because it saves a significant amount of money for the taxpayer," said senior author Laurent Servais, PhD, of the University of Liege, in Belgium and the University of Oxford, in the UK. "Using these data issued from the real world, we are working currently on a model that estimates the lifetime cost of the different strategies."

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