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 U.S. 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 Wednesday it decided on the lower price after hearing concerns from health insurers about the affordability of the treatment.
Consternation over skyrocketing drug prices, especially in the U.S., has led to intense scrutiny from patients, politicians, insurers and hospitals.
“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 with The Associated Press.
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 U.S.
Pharmaceutical industry critics said the slightly lower cost is a distraction from the ongoing problem of unsustainable drug prices.
Approved last month, 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, which Spark estimates will cost between $4,000 and $5,000.
The treatment is part of an emerging field of medicine that could produce dozens of new gene-targeting medications in the next few years.
Like Luxturna, these therapies are generally intended to be taken once, a fact which drug developers argue sets them apart from traditional drugs taken for months or years. Even compared to other one-time gene therapies Luxturna is still an outlier. Two customized gene therapies for blood cancer approved last year are priced at $373,000 and $475,000.
Drug prices are not regulated in the U.S., as they are in many other countries, so drugmakers can price their goods like any other manufacturer. Drugmakers have historically offered little explanation for the prices they charge, other than to cite the high cost of developing a drug and the fact that so many drugs fail during trials and must be abandoned. However, some companies have begun to offer more detailed reasoning as the backlash against drug prices has grown more heated.
Spark Therapeutics, based in Philadelphia, has said that the cost for a lifetime of blindness — including lost earnings and caregiver wages — can easily exceed $1 million.
