The question is no longer academic: On Thursday, Spark Therapeutics won unanimous support from a Food and Drug Administration advisory panel for its gene therapy drug, Luxturna. It seems likely to win FDA approval in the coming months. But the cost will be hefty: Analysts estimate that Luxturna, which has been shown to restore vision in children with an inherited form of blindness, could cost $1 million per patient.
Will private insurers be willing to pay? What about taxpayers, via Medicaid and Medicare? And, importantly: What happens if patients — or insurers — do foot a hefty bill, only to find out the drug simply did not work for them?