Million-dollar drugs pose new challenge for Congress

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A new drug approved for the first time in May offers hope to children born with a rare genetic disease who previously often did not live past early childhood. 

The drug, Zolgensma, injected through the veins, provides a crucial functioning gene for people with spinal muscular atrophy who otherwise would have their muscles break down. 

With a one-time treatment, the drug has the potential to treat patients for a lifetime. But there’s one big problem: It costs $2.1 million. 


That eye-popping price tag gives Zolgensma the distinction of being the world’s most expensive drug — and there are others like it. 

A wave of innovation is bringing on new breakthrough treatments for terrible diseases, but also record highs in the prices of those medicines. The challenge will only mount as more drugs are developed. 

Zolgensma is part of a new type of treatment known as gene therapies that can replace a mutated gene that causes disease with a healthy one. 

In cancer treatment, the new field of CAR T-cell therapy trains a patient’s immune system to fight cancer. 

One such treatment, Kymriah, was approved in 2017 with a price tag of $475,000. 

“We are entering a new phase,” said Dr. Walid Gellad, director of the Center for Pharmaceutical Policy and Prescribing at the University of Pittsburgh, with a burst of “potentially curative” drugs that are “much more expensive.”

The question, of course, is whether these prices are justified. To patients with a fatal disease, almost any price could seem worth it for a cure. But the health care system as a whole would strain to afford massive sums for a whole range of drugs. 

“We’re going to have to do something,” said David Mitchell, president of Patients for Affordable Drugs. “We can’t possibly pay whatever these drug companies demand.”

Mitchell himself has multiple myeloma, an incurable blood cancer, and said that the development of new cures is “very, very important to me.”

“I personally want these drugs, but we have to figure out a way to have them but have them be affordable and accessible,” he said. 


Drug companies argue that the price needs to be viewed in comparison to what people would otherwise be paying for the cost of the old treatments, which would often be spaced out over years. 

AveXis, a unit of Novartis, which makes Zolgensma, argues that its $2.1 million cost is about 50 percent of the cost of the old treatments over 10 years. 

“The $2 million could be seen as outrageous, but the reality is we’ve been spending that kind of money to care for these kids and no one noticed,” Dave Lennon, president of AveXis, told The Hill. 

“We’re actually providing a highly cost-effective method for treating these kids,” he added. 

Mitchell counters that paying based on what the cost otherwise would be is the wrong way to look at the issue. “We don’t pay anybody else in our health care system that way,” he said. “Doctors and surgeons who repair genetic defects at birth don’t make $2 million for doing it.”

Lawmakers are confronting these questions as they craft legislation aimed at lowering drug prices, a rare area of possible bipartisan cooperation this year. 

Senate Finance Committee Chairman Chuck Grassley (R-Iowa) is floating the idea of allowing Medicaid to pay for expensive drugs over time, rather than all at once, comparing it to buying a house. 

House Democrats are working on legislation to allow Medicare to negotiate drug prices, a long-held goal for the party that they hope President Trump will also endorse given his attacks on drug companies. 

The cost problem could intensify once new areas like gene therapy have time to mature and more drugs come on the market. Many of the drugs with high prices have treated diseases that affect a relatively small number of people so far, but that might not always be the case, which opens the possibility of treating more people but also racking up even higher spending. 

Lennon, the AveXis president, said having a multitude of new treatments would be “a great problem to have” because of the innovation that it would entail. 

He said each new drug would have to be priced based on the value it brings. 

“I don’t think our product sets a precedent that everyone can price at that level,” he said. 

Gellad, the University of Pittsburgh professor, said that prices have to be high enough to incentivize innovation. 

But there is no consensus on how much profit there needs to be or how high the price needs to be to ensure innovation does not suffer. 

“We want the price to encourage enough new development of therapy, and that’s where no one can agree,” Gellad said. “The issue is no one knows the right answer.”

Tags Chuck Grassley Donald Trump Drug prices drug pricing Future of Healthcare Summit Gene therapy

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