Last year, two medications went on the market for six-figure prices. Exondys 51 (eteplirsen, Sarepta Therapeutics) sells for $300,000 a year and Spinraza (nusinersen, Biogen) for a whopping $750,000. So people familiar with the current drug-price controversy might reasonably ask why there is so much heat over the $89,000-a-year cost of Emflaza (deflazacort, Marathon Pharmaceuticals), a treatment for Duchenne muscular dystrophy. An article posted on the Kaiser Health News (KHN) website lists five reasons for the uproar.
Deflazacort has never been approved for sale in the United States, but the steroid has been available for decades in other countries—at much lower prices, according to the KHN article. Some Duchenne patients in the U.S. have imported deflazacort from Europe and Canada for years for as little as $1,000 annually. In medical circles, deflazacort is often compared with the widely used steroid prednisone, which has been around since 1955.
Eteplirsen and nusinersen are scientific breakthroughs in that they target genes to treat underlying diseases. Deflazacort, on the other hand, addresses symptoms of muscle weakness and deterioration by reducing inflammation and suppressing the immune system in Duchenne patients.
Dr. Aaron Kesselheim, an associate professor of medicine at Harvard Medical School, questioned 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, deflazacort is a steroid that has no specific indication for Duchenne muscular dystrophy, Kesselheim told KHN.
Duchenne patients need to take deflazacort as part of a drug “cocktail,” according to Pat Furlong, founder of the advocacy group Parent Project Muscular Dystrophy. She questioned how many high-priced drugs insurers will accept for this indication.
Marathon has said that the price for deflazaort was set based on a number of factors, including recouping its research costs. The company said that the FDA required it to submit deflazacort as a new drug, regardless of its approval in other countries.
Marathon CEO Jeff Aronin recently released an open letter saying the company would “not move forward with commercialization” of deflazacort until discussing options with Duchenne community leaders. The company, which is a corporate sponsor for Parent Project Muscular Dystrophy, announced that it will continue to offer an expanded access program to patients. He also said patients currently importing the drug from other countries can continue to do so.
Joel Wood, whose Duchenne foundation has received money from Marathon, said he believed the company will uphold its financial promises to patients.
“I will be the first one to pick up a pitch fork” if the company fails to do so, he said.
The KHN article points out that there was muted criticism when the $750,000 price tag for nusinersen was quietly 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 now expressing anger about deflazacort’s price and demanding answers from Marathon.
Last week, Senator Chuck Grassley (R-Iowa), chairman of the Senate Judiciary Committee, announced that he had opened an inquiry into potential abuses of the Orphan Drug Act that may have contributed to high prices on commonly used drugs.
Senator Bernie Sanders (I-Vermont) and Representative Elijah Cummings (D-Maryland) sent a letter to Marathon calling deflazacort’s price “unconscionable.” They asked the company to explain itself and lower the price.
Source: Kaiser Health News; February 16, 2017.