"Some sort of model where there was a partial payment over time could work well," Brennan said in an interview.
The Price Of Progress
Gene therapy has been a target of Big Pharma for more than 20 years, but research has been dogged by a series of disappointments and safety concerns. More recent scientific advances have paved the way for the potentially life-changing treatments.
No gene therapies have been approved in the United States, but Europe approved its first gene therapy last year. Glybera treats a rare disorder that clogs the blood with fat and has been cleared for reimbursement in Germany at a price of 850,000 euros, or around $1 million. It will be sold for a one-time payment because it is too difficult to measure how well it works, said Joern Aldag, CEO at Dutch biotech firm UniQure NV , which developed Glybera.
Aldag said gene therapies for larger patient populations in development at UniQure, including hemophilia and congestive heart failure, would likely be priced on an amortized basis.
"Gene therapy may be an initial very high burden for the healthcare system," Aldag said. "The general consensus is that asking for a one-off payment of over a million dollars becomes unacceptable."
U.S. health insurers and other group payers routinely pay annual prices in the hundreds of thousands of dollars for enzyme replacement therapies for extremely rare, "orphan" diseases. BioMarin's Vimizin costs nearly $400,000 per year for a genetic disorder that causes skeletal malformation and other problems.
But insurers are much less willing to foot that kind of bill for drugs used in large numbers of patients. Last year, they pressured Gilead Sciences Inc to cut the $1,000 per-pill cost of its new hepatitis C drug, saying the cost of treating more than 3 million Americans infected with the virus was unacceptable. Gilead said recently that it is discounting its prices by nearly 50 percent.
About 20,000 Americans are estimated to have hemophilia, which is caused by a defect in a gene carried on the X chromosome.
"Distributing reimbursement over many years recognizes the fact that we don't know how long these (gene therapy treatments) are going to last," said Dr James Wilson, a gene therapy expert at the University of Pennsylvania.