Spinraza, which treats Spinal Muscular Atrophy (SMA), a rare genetic condition in children, costs $750,000 for the initial year of treatment. The Food and Drug Administration approved the first treatment for the condition two days before Christmas in 2016.
Across the country, similar stories are playing out as private insurers and already-squeezed state Medicaid programs wrestle with what, if any, limits to place on patients' access to break-the-bank drugs - weighing the needs of the ill against budget realities.
At the same time, policymakers and physicians increasingly demand to understand why drug manufacturers affix price tags that have risen to once unimaginable highs.
"It looks like a drug that works for a tragic condition that afflicts children and cripples and kills them. That's the good news, "
Dr. Jerry Avorn
, a professor of medicine at Harvard Medical School, says of Spinraza. But "how in the world did the price of $750,000 a year get chosen?"
Biogen, the maker of Spinraza, defends its price.
"We compared industry norms for other drugs in rare disease. We looked at the efficacy and safety profile of the drug itself," says Dr. Wildon Farwell, an epidemiologist and senior medical director of clinical development at