The latest flashpoint in the ongoing debate over high drug prices is Emflaza, an $89,000-a-year drug that treats Duchenne muscular dystrophy.
People who have been watching the drug price issue closely, however, can reasonably ask why there is so much heat at that price tag? Late last year, two drugs went on the market for six-figure prices. Exondys 51 sells for $300,000 a year and Spinraza for a whopping $750,000.
While they did draw headlines, neither of those drugs sparked the bipartisan congressional firestorm and patient outcry that Emflaza ignited this week. Here are five reasons why:
1. It’s not a new drug.
Emflaza, the brand name for deflazacort, has never been approved for sale in the United States, but the steroid has been sold for decades in other countries — at much lower prices.
Some Duchenne patients in the U.S. have imported deflazacort from Europe and Canada for years for just $1,000 to $1,600 annually. Mohammed Haider, 27, of Mount Laurel, N.J., said he’s been buying it from European pharmacies since he was seven years old.
In medical circles, deflazacort is often compared with the widely used steroid, prednisone, which has been around since 1955.
2. It’s not a scientific breakthrough.
Exondys 51 and Spinraza are medical breakthroughs that target genes to treat underlying diseases.
Emflaza, on the other hand, addresses symptoms of muscle weakness and deterioration by decreasing inflammation and suppressing the immune system in Duchenne patients.
Dr. Aaron Kesselheim, an associate professor of medicine at Harvard Medical School, questions why Marathon won approval using the Orphan Drug Act, which was created by Congress to motivate companies to develop drugs to treat rare diseases.
Instead, Kesselheim said, this is a steroid that could be used more broadly.
“There’s no indication to me that this is a steroid specific to muscular dystrophy,” he said.
Marathon had considered researching whether Emflaza could treat patients with juvenile arthritis, but the company said last week it has no plans to pursue that use.
3. Emflaza doesn’t work alone.
The drug needs to be part of a cocktail of drugs Duchenne patients take, said Pat Furlong, the founder of the advocacy group Parent Project Muscular Dystrophy. She questions how many high-priced drugs insurers will pay for.
“Where is the breaking point where any given insurer says [that’s] too much,” and stops covering the drugs, Furlong said.
Marathon said the price was set based on a number of factors, including recouping its research costs. The FDA required the drug to be submitted as a new drug — regardless of its approval in other countries. A company spokeswoman said Marathon funded 17 studies for Emflaza’s approval.
4. Marathon has cultivated relationships in the Duchenne patient community.
At a heated meeting with patient advocates on Monday, Marathon CEO Jeff Aronin announced a delay in the rollout of the drug and later released an open letter saying the company would “not move forward with commercialization” until discussing options with Duchenne community leaders.
The company, which is a corporate sponsor for Parent Project Muscular Dystrophy, announced it will continue offering an expanded access program to patients. He also said patients currently receiving the drug from other countries can continue importing.
Joel Wood, whose Duchenne foundation has received money from Marathon, said he believed the company will uphold financial promises to patients.
“I will be the first one to pick up a pitch fork” if the company fails to do so, said Wood, who has a son with Duchenne.
5. Timing is everything.
There was muted criticism when Spinraza’s $750,000 price was announced over the Christmas holiday.
Perhaps emboldened by President Donald Trump who recently said drug makers are “getting away with murder,” lawmakers from both sides of the aisle are expressing anger about Emflaza’s price and demanding answers from the drugmaker.
Rep. Robert Aderholt, a Republican from Alabama who chairs the subcommittee that oversees FDA’s appropriations, said Monday that Emflaza is a “tipping point” on the issue.
Last week, Republican Sen. Chuck Grassley, chairman of the Senate Judiciary Committee, announced he has opened an inquiry into potential abuses of the Orphan Drug Act that may have contributed to high prices on commonly used drugs.
Sen. Bernie Sanders (I-Vt.) and Rep. Elijah Cummings (D-Md.) sent a letter to Marathon Monday calling Emflaza’s price “unconscionable.” They asked the company to explain itself and lower the price.
KHN’s coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation.
Source: Kaiser Health