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 Atrophy: Are We Paying Too Much for Too Little? JAMA Pediatr. 2018;172(2):123–124. doi:10.1001/jamapediatrics.2017.4360
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