Gene therapy for blindness to cost $850K
washington — A first-of-its kind genetic treatment for blindness will cost $850,000 per patient, making it one of the most expensive medicines in the world and raising questions about the affordability of a coming wave of similar gene-targeting therapies.
The injectable treatment from Spark Therapeutics can improve the eyesight of patients with a rare genetic mutation that affects just a few thousand people in the US. Previously there has been no treatment for the condition, which eventually causes complete blindness by adulthood.
Pricing questions have swirled around the treatment due to a number of unusual factors — it is intended to be a one-time treatment, it treats a very small number of patients and represents a medical breakthrough.
Previously, Spark suggested its therapy, Luxturna, could be worth more than $1 million. But the company said on Wednesday it decided on the lower price after hearing concerns from health insurers about the affordability of the treatment.
“We wanted to balance the value and the affordability concerns with a responsible price that would ensure access to patients,” said CEO Jeffrey Marrazzo, in an interview.
Luxturna is still significantly more expensive than nearly every other medicine on the global market, including two other gene therapies approved earlier last year in the US.
Pharmaceutical industry critics said the slightly lower cost is a distraction from the ongoing problem of unsustainable drug prices.
“The company very cleverly convinced everyone that they were going to charge a million dollars, so now they are being credited for being reasonable,” said Dr Peter Bach, director of a policy centre at Memorial Sloan Kettering Cancer Centre in New York.
Luxturna, is the nation’s first gene therapy for an inherited disease. It requires a 45-minute operation in which a tiny needle delivers a replacement gene to the retina, tissue at the back of the eye that converts light into electric signals that produce vision. The therapy will cost $425,000 per injection. The price does not include the cost of the operation. —