Nusinersen, one of several recently approved drugs intended for use in a rare disease, boasts an eye-popping price tag. Biogen Pharmaceuticals announced that nusinersen will cost $750 000 for the first year of treatment and $375 000 each year thereafter (prescribed indefinitely) for patients with spinal muscular atrophy (SMA).1 Other recently approved costly therapies for rare diseases include ivacaftor, costing $311 000 per year, for responsive patients with cystic fibrosis2; combination lumacaftor-ivacaftor, costing $272 000 per year, for patients with cystic fibrosis2; eteplirsen, costing $300 000 per year, for a 25-kg child with Duchenne muscular dystrophy3; and asfotase alfa, costing $285 000 per year, for patients with infantile and juvenile-onset hypophosphatasia.4 Many more such drugs are poised to enter the market, particularly in fields such as cancer medicine.5
Prasad V. Nusinersen for Spinal Muscular AtrophyAre We Paying Too Much for Too Little?. JAMA Pediatr. 2018;172(2):123–125. doi:10.1001/jamapediatrics.2017.4360
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