In the next few years the FDA is expected to approve dozens of new genetic therapies for rare diseases that affect just a small group of patients. Because these drugs cost nearly seven-figures insurance companies, hospitals and Congress are taking a closer look.
One gene therapy already approved, is Luxturna, costing $850,000. The surgical injection ($425,000 per eye) replaces a defective gene in the retina of a blind person. Within 24 hours of the treatment, patients suffering from this rare eye condition can see. The drug originally cost $1 million, but the pharmaceutical company that makes it, lowered the price after hearing concerns from health insurers.
Dr. Robert Sisk will do the surgery at Cincinnati Children's Hospital Medical Center this spring. He says Luxturna is a game changer. "I think this is incredibly exciting. This category of rare genetic diseases is one where we've not really been able to offer much hope for patients until the last five years."
Sisk says the therapy is designed for patients missing the gene RPE65. "So the virus, which is what the gene therapy is, is engineered in such a way that it can't replicate or destroy a cell but can deliver a working copy of this particular gene which is not present because of the genetic defect," he says.
Who is benefiting?
Two thousand to four thousand people in the U.S. have this eye defect. An equally small number of people suffer from a rare protein disorder. A genetic therapy to treat that cost $1.2 million and because there was a lack of demand for the drug, manufacturer uniQure stopped selling it last year. Yescarta, a cancer therapy could potentially treat a lot more patients, 7,500 people. It has a price tag of $373,000. Kymriah treats a form of childhood leukemia. It costs $475,000.
There are ethical questions. UC Professor of Molecular Genetics Anil Menon says the high priced drugs for small groups of people opens up a can of worms like social inequities and healthcare access. "It's possible that people who can afford these expensive drug methodologies will of course use them and others may not."
He says with people taking better care of themselves, "now the bottleneck is actually the genetic inheritance that we have." In the case of Luxturna, Dr. Sisk says it's worth the investment to the patient and society.
Drug companies say they are working with insurers. Spark Therapeutics, the manufacturer of Luxturna, may allow insurers to pay in installments. Also in some cases they repay part of the costs if the patient's vision doesn't improve.