Carolyn Y. Johnson
February 10, 2017
An old steroid treatment, long available outside the United States, received approval this week for a rare disease that afflicts about 15,000 Americans. Though not previously approved in the United States, the drug, deflazacort, has for years been available to patients suffering from the devastating and fatal disease Duchenne muscular dystrophy; families can import it from abroad for about $1,200 per year on average.
The new list price for the drug? $89,000 a year.
After rebates and discounts, the net price will be $54,000 a year, said Marathon Pharmaceuticals Chief Financial Officer Babar Ghias in an interview.
The company sought approval for deflazacort as an “orphan drug,” a special approval pathway intended to encourage the development of drugs for very rare diseases. With orphan designation, the company gets seven years of exclusive rights to sell the drug in the U.S., even though it has long been available as a generic in other countries.
The company also scored a valuable “priority review” voucher, essentially a ticket that it can use to get a future drug reviewed by regulators faster — or that it can sell to another company for hundreds of millions of dollars.
In theory, these vouchers exist for a good reason: Regulators want to encourage companies to invest in developing drugs for rare diseases that afflict children. But when old drugs already being used for a disease go through the approval process to earn a voucher — and a high price — it has raised questions about whether the incentives are being misapplied.
“It seems like it’s yet another example of gaming the system,” said Aaron Kesselheim, an associate professor of medicine at Harvard Medical School. “How many examples of this do we have to see before we can start to rethink the priority review voucher as a means of incentivizing innovation? This also seems to be another example of gaming the Orphan Drug Act, which was intended to try and encourage research into new therapeutic entities for people who have rare diseases — and it doesn’t seem like this is that.”
For patients, the cost of the drug — which goes by the brand name Emflaza, would be “zero to low out-of-pocket expense,” because of insurance and financial assistance programs that the company will offer, Ghias said. But to critics of old drugs that receive high prices, those measures are a public relations move that will simply insulate the company from its harshest potential critics — families with dying children who above all want access to the drug.
“Instead of making the price at a level that is reasonable for patients, they make it a very high price and offer this pathway that patients may not qualify for, they may not know about, there may be limitations on it. So it’s a marketing move and not really a public health solution,” Kesselheim said.
In recent years, companies that have gotten old or existing drugs approved to treat rare diseases have reaped big financial rewards. For example, tetrabenazine, a drug that was available from abroad and used for years to treat the uncontrollable tremors of Huntington’s disease, was approved as an orphan drug in 2008. In 1998, it cost $42.28 for a bottle of tetrabenazine pills from a European pharmacy, according to Joseph Jankovic, a neurologist at Baylor College of Medicine. After receiving approval as an orphan drug, that bottle of pills — now known by the brand name Xenazine — carried a list price of more than $6,000 in the U.S. in 2008. The price was repeatedly ratcheted up to more than $21,243 a bottle, according to Truven Health Analytics data. Xenazine accounted for $325 million in U.S. sales in 2015, the year it went generic, according to data from Evaluate, a market intelligence firm.