Families, lawmakers demand transparency for $89,000 drug

Life-saving drugs are getting more expensive
Life-saving drugs are getting more expensive

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Story highlights

  • A drug for muscular dystrophy has faced criticism for its $89,000-a-year price tag
  • The pharmaceutical company halted its rollout and released a statement justifying the cost

(CNN)A newly US-approved drug with an $89,000-a-year price tag has been put on hold, owing to the latest uproar from advocates and lawmakers over high drug prices.

The drug, a steroid called deflazacort, was approved by the Food and Drug Administration last week. It has been available in Europe for decades to treat Duchenne muscular dystrophy, a rare, fatal disease that slowly attacks the muscles.
The rollout of the drug was temporarily suspended by maker Marathon Pharmaceuticals on Monday. Shortly beforehand, Sen. Bernie Sanders and Rep. Elijah Cummings sent a letter to Marathon accusing the company of abusing a government drug incentive program and contributing to rising costs "for the entire health care sector."
    The company's yearly estimate of $89,000 is a stark contrast to the $1,200 that some families pay to import the drug from overseas, raising questions about what the ultimate cost to consumers will be -- particularly for children with rare diseases who have few treatment options.
    "We're really upset. I feel like there was no transparency about how they got to that price," said Joanne Wechsler, a parent advocate with Parent Project Muscular Dystrophy. Wechsler's oldest son, Adam, 20, has Duchenne muscular dystrophy.
    "They should be able to make a profit, but that's really crazy."
    But the company insists that people who need the drug will have access to it through insurance and the company's own patient assistance programs.
    "We expect patients will pay a standard co-pay of typically $20 or less per prescription," reads a statement by Marathon's CEO, Jeffrey Aronin.
    Now industry group PhRMA may want to kick out the company from its organization because it acted in a way that doesn't align with PhRMA's mission, according to a statement by PhRMA President Stephen J. Ubl. Marathon's Aronin sits on PhRMA's board of directors.
    A request to Marathon for Aronin's response was not immediately returned.

    Taking on Duchenne

    Duchenne muscular dystrophy is a rare genetic disease that causes muscles to slowly waste away. Patients, nearly all of whom are boys, will often have trouble walking or climbing steps before they are 5. They might need to use wheelchairs before they are teenagers. About half die by their mid- to late 20s, usually due to heart muscle failure.
    Deflazacort is a steroid that delays muscle damage in Duchenne patients, according to Dr. Stanley Nelson, who co-directs the Center for Duchenne Muscular Dystrophy at UCLA with his wife, fellow professor Carrie Miceli. They have one son with Duchenne who also takes deflazacort.
    With the drug, boys can walk for an average of three years longer than with no steroids at all, Nelson said. They can breathe on their own for longer, and they develop scoliosis less frequently thanks to stronger back muscles. Deflazacort is more effective and has milder side effects than the next best steroid, prednisone, which is widely available in the United States.
    Wechsler, the parent advocate, has seen the impact of the drug herself. Her son, now a junior at the University of Vermont, is living independently with the help of nighttime aides. He can still put a fork to his mouth.
    "He can do things we weren't expecting he'd still be able to do," she said. "We were told he was going to die before high school."
    Wechsler buys the drug from a pharmacy in the UK. Because Adam uses a smaller dose, it costs their family only $500 per year, which is covered by Vermont's Children with Special Health Needs program. But he will age out of the program in May, when he turns 21.
    "The company is telling us that we're not going to have to pay more than $20 out of pocket," Wechsler said of Marathon. "My big concern is ... I have to fight with my insurance company over everything."
    Although FDA approval opens the door to insurance coverage for the drug, Wechsler doesn't know for sure how her insurer will act once it hits the market. The company recently refused to cover a wheelchair Adam needed, she said; only after a "huge battle" did it concede.
    "We're in a position of always having to advocate for our son's needs," Wechsler said.
    "There's a lot of anxiety about what health care is going to look like in the US," UCLA's Dr. Stanley Nelson said, adding that the insurance conversations surrounding lifetime caps and preexisting conditions could hit Duchenne families directly.
    "We need a health care system that recognizes that there are rare diseases."

    Hitting the pause button

    In response to the backlash, Marathon's Aronin released a statement justifying the costs and announcing the halt of the drug's commercial launch.
    Aronin said Marathon was "pausing our commercialization efforts." In the meantime, company executives plan to speak with Duchenne leaders and devise plans to move forward. Marathon did not give a timeline.
    Debra Miller is one of these leaders. Like Wechsler, Miller -- the co-founder and CEO of the advocacy group CureDuchenne -- has a 20-year-old son with the disease.
    "We had never heard of the disease," said Miller, who has no family history of the genetic mutations that cause Duchenne. "It just popped up out of the blue and was completely shocking and devastating."
    CureDuchenne works with and invests in biotech companies to spur research for the disease. Marathon is one of these companies, though Miller said they had not discussed pricing until the past week.
    "We reached out to them immediately and expressed concern," she said.
    Miller's son, Hawken, has been on deflazacort since his 6th birthday. She gets the drug from the same UK pharmacy as Wechsler, adding that the $1,200 a year she pays can be prohibitive for many families. Getting the drug abroad, she said, has other challenges: Packages occasionally get held up in customs, and others don't show up at all.
    While Marathon re-evaluates, families like the Wechslers and the Millers will still be able to buy the drug online from foreign pharmacies. However, once the drug is available in the US, that door will be closed, according to the FDA. The only way to obtain the drug legally then will be from Marathon.
    Because of government incentives for drugs that treat rare diseases, Marathon will be the sole American distributor of deflazacort -- under the brand name Emflaza -- for seven years.

    Reasons and reassurances

    Marathon's CEO Jeffrey Aronin claimed that price would not be a barrier for families who need the drug. In his open letter, he said that early meetings with insurers "have gone well" and that Marathon's patient assistance program would continue to lower the financial burden for some families.
    "Anyone who needs this medicine will get this medicine," he wrote.
    In justifying Marathon's pricing, Aronin said that the company needed to recoup its investment in the clinical studies it designed to bring the drug to market. These studies explored the appropriate dosing, side effects and drug interactions. However, the letter by lawmakers Sanders and Cummings points out that Marathon also built upon data from the 1990s, which had already proved that it worked.
    "There are some people who think you can just bring a generic from Europe and sell it in the United States, and that's simply not the case," said Wanda Moebius, vice president of communications for Marathon.
    Aronin's letter also said that the price tag would increase Marathon's "ability to fund future research and development," a common refrain among drug companies responding to backlash over pricing -- from Mylan's price hike on its EpiPen to infamous pharma executive Martin Shkreli.
    In August, Harvard researchers found that the 10 largest pharmaceutical companies spent between 7% and 21% of total sales on research and development. Marathon was not one of the companies surveyed.
    The researchers concluded that there was "no evidence" of a relationship between how much companies charged for drugs and how much they spent on drug research and development; drug prices are based on "what the market will bear," the authors wrote. Lack of competition and the inability of Medicare to negotiate with pharmaceutical companies may also contribute to high prices, they said.
    This isn't the first time one of Aronin's companies has faced backlash for increasing the price of drugs. In 2008, the company he headed at the time, Ovation Pharmaceuticals, was sued by the Federal Trade Commission under accusations that it unlawfully acquired a drug for premature babies with a fatal heart condition, established a monopoly on the drug and raised the price nearly 1,300%.
    But, Ovation Pharmaceuticals won the suit.

    A call for transparency

    Despite the outcry, advocates like Miller are not so quick to blast incentives that have led drug companies to focus on rare diseases that affect only a handful of people. She is worried that the backlash will cause companies to abandon these so-called orphan drugs in search of more profitable ones.
    Muscular dystrophy advocates Joanne Wechsler and Bradley Stephenson, who has Becker muscular dystrophy, and his daughter Georgia met with Sen. Bernie Sanders this week.
    "I want to make sure that we don't win a battle and lose a war," she said. "We have to encourage companies ... to come in and be interested in rare diseases."
    New research is underway, UCLA's Nelson and Miceli said. Whereas deflazacort targets the inflammation of muscular dystrophy, other drugs may actually stabilize muscles and address genetic mutations that occur on the DNA level.
    In September, the FDA approved a drug called eteplirsen, which addresses just one mutation associated with Duchenne. The controversial move was based largely on a small study of 12 boys.
    Another drug approved in Europe, ataluren, was recently denied by the FDA but is still being pushed by advocacy groups for review and approval. Gene editing tool CRISPR/Cas9 is also being explored at the preclinical level as a potential treatment.
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    Nelson, however, acknowledges the pattern these drugs might continue.
    "Each of these rare-disease drugs comes out with a very shocking price," he said.
    Nelson thinks transparency in how Marathon prices its drugs "would go a long way." This would entail the company being open about its plans to recoup its research investment and turn an acceptable profit. This is also the information Sanders and Cummings requested in their letter to Marathon.
    On Wednesday, Wechsler returned home from Capitol Hill, where she met with Sanders as part of her advocacy work. She agrees that transparency is key and said that many were initially excited by deflazacort's approval -- until they saw the price.
    "We've been wanting it FDA approved for as long as I can remember," she said. "It is a roller coaster."