5 reasons why an $89,000 drug has Congress fuming

This Oct. 14, 2015, file photo shows the Food and Drug Administration campus in Silver Spring, Md. On Sept. 19, 2016, the FDA granted tentative approval to the first drug for muscular dystrophy, following an intense public campaign from patients and doctors who pushed for the largely unproven medication. The FDA cleared Sarepta Therapeutics’ Exondys 51 for a rare form of Duchenne muscular dystrophy, a deadly inherited disease that affects boys.

The latest flashpoint in the 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.

Although they did draw headlines, neither of those drugs sparked the bipartisan congressional firestorm and patient outcry that Emflaza ignited last 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 United States have imported deflazacort from Europe and Canada for years for just $1,000 to $1,600 a year. Mohammed Haider, 27, of Mount Laurel,  said he’s been buying it from European pharmacies since he was 7 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.

Aaron Kesselheim, an associate professor of medicine at Harvard Medical School, questions why Marathon Pharmaceuticals won approval using the Orphan Drug Act, which was created by Congress to motivate companies to develop drugs to treat rare diseases.

Emflaza, Kesselheim said, 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 recently that 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 that 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 announced a delay in the rollout of the drug. Later in the day, Marathon CEO Jeff Aronin released an open letter saying the company would “not move forward with commercialization” until it discussed 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. Aronin said patients currently receiving the drug from other countries can continue importing.

Joel Wood, whose Duchenne foundation has received money from Marathon, said he believes the company will uphold financial promises to patients. “I will be the first one to pick up a pitchfork” 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 around Christmas.

Perhaps emboldened by President Trump, who recently said drugmakers 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 (R., Ala.), who chairs the subcommittee that oversees FDA’s appropriations, said Monday that Emflaza is a “tipping point” on the issue.

Sen. Chuck Grassley (R., Iowa), chairman of the Senate Judiciary Committee, announced this month that he has opened an inquiry into possible 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 on Monday calling Emflaza’s price “unconscionable.” They asked the company to explain itself and lower the price.

Kaiser Health News is a national health policy news service that is part of the nonpartisan Henry J. Kaiser Family Foundation.  Coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation.