US Approves the Most Expensive Drug in the World

Plus, what Congress has to get done

The Most Expensive Drug in the World — for Now

The Food and Drug Administration on Friday approved a gene therapy from Novartis that treats spinal muscular atrophy, clearing the way to market for what’s set to become the world’s most expensive medicine and the first U.S. drug to break the million-dollar mark.

The drug, to be sold as Zolgensma, will cost $2.1 million for a one-time treatment. Novartis said it would allow insurance companies to spread out payments over five years, for an annual cost of about $425,000.

The gene therapy targets a rare disease that strikes 1 in 11,000 births, or about 400 new patients per year. Without treatment, many of the stricken children die before their second birthday.

A fair price? The company defended its pricing plan, saying that it’s cheaper than the current biologic treatment option, Spinraza, which was introduced in 2016. Spinraza costs $750,000 for the first year but requires additional annual treatments for the life of the patient, at a cost of $350,000 per year. By contrast, Zolgensma is intended to be administered just once.

The Institute for Clinical and Economic Review, an independent watchdog group, said that the price of Zolgensma “falls within the upper bound” of its value-based benchmark range, which takes into account the value of increased lifespans.

But that approach has its critics. David Mitchell of the advocacy group Patients For Affordable Drugs, said the price “is emblematic of our broken system that effectively forces us to pay whatever price drug corporations demand for lifesaving new drugs. … We didn’t pay for the polio vaccine based on the future cost savings for kids who didn’t need to live in iron lungs.”

The promise of big profits: Novartis acquired the biotech firm AveXis, which developed Zolgensma, for $8.7 billion just last year. Industry analysts expect the drug to hit peak sales of $1.8 billion to $2.6 billion per year, according to PMLiVE, a pharmaceutical industry news site.

The price record may not stand for long: Bloomberg’s Max Nisen says that Zolgensma could open the door to skyrocketing prices for dozens of new gene therapies that are currently in the works. And at those prices, patients without the right kind of insurance could find themselves locked out. “Miracle cures don’t do much good if they aren’t accessible,” Nisen writes.

5 Keys Items on Congress’s To-Do List for the Rest of the Year

Congress’s to-do list is growing. “With the first half of 2019 off to a slow legislative start, both chambers are facing a potential logjam of crucial deadlines and competing priorities heading into the back half of the year,” The Hill’s Jordain Carney writes.

Here’s a rundown of some high-priority items lawmakers still need to address:

Disaster aid: Before the holiday weekend, lawmakers struck a deal on a long-delayed $19.1 billion relief package, with President Trump saying the agreement has his “total approval.” The Senate passed the legislation last Thursday, but Rep. Chip Roy (R-TX) on Friday blocked a vote by unanimous consent in the House and Rep. Thomas Massie (R-KY) blocked another vote during a pro forma session Tuesday. Those lawmakers faced blowback, including from members of their own party, but their objections mean that a recorded vote will likely have to wait until early next month.

2020 spending: Lawmakers are looking to reach a two-year deal to raise budget caps and avoid automatic spending cuts, but progress in negotiations early last week appeared to stall out, with the two sides reportedly far apart on top-line levels for non-defense outlays.

Debt ceiling: Lawmakers will need to extend the government’s borrowing authority by late summer or early fall to avoid breaching the debt limit and potentially setting off a financial crisis. “Leadership and White House officials are considering combining the debt ceiling issue with a potential budget caps deal so that Congress can approve both simultaneously,” Carney reports.

Funding the government: Once the top-line budget levels are set, appropriators need to pass 12 spending bills by the start of the new fiscal year on October 1 or risk another government shutdown. Congress could also pass a stopgap measure to extend funding at 2019 levels and shift the deadline until later in the year.

Infrastructure: Don’t count on anything getting done.

For more on Congress’s to-do list, read Carney’s piece at The Hill.

Judge Blocks Trump from Using Pentagon Funds for Border Wall

In case you missed it, a federal judge issued an order Friday that put a halt to the Trump administration’s transfer of $1 billion from counter-narcotics efforts at the Department of Defense to border wall construction projects in Arizona and Texas. The judge’s ruling will allow some fund transfers to continue, including a shift of $600 million from the Treasury Department’s asset forfeiture fund to barrier construction in the Rio Grande Valley.

U.S. District Judge Haywood Gilliam Jr. said that the administration appears to be ignoring the will of Congress in its efforts to boost border wall construction, which lawmakers have refused to fund at the level requested by the White House. “Congress’s ‘absolute’ control over federal expenditures — even when that control may frustrate the desires of the Executive Branch regarding initiatives it views as important — is not a bug in our constitutional system. It is a feature of that system, and an essential one,” Gilliam wrote.

President Trump criticized the ruling while still on his trip to Japan, tweeting: “Another activist Obama appointed judge has just ruled against us on a section of the Southern Wall that is already under construction. This is a ruling against Border Security and in favor of crime, drugs and human trafficking. We are asking for an expedited appeal!”

Column of the Day

In a column published Sunday, The Washington Post’s E.J. Dionne Jr. recalls that, after the late Republican Rep. Steve LaTourette of Ohio decided to not seek reelection in 2012, he said it was because Congress “couldn’t even pass a highway bill anymore.” Dionne brings that up in light of last week’s infrastructure meeting between President Trump and Democratic leaders — the meeting that quickly imploded, with the president insisting that he can’t work with Democrats who continue to investigate him.

“Trump’s theatrics were also very convenient because they disguised the fact that he cannot now, or ever, deliver on his signature promise to create a ‘great’ infrastructure program,” Dionne writes. “This is why Trump ‘infrastructure weeks’ have become a standing joke in Washington. LaTourette was right: The Republican Party is no longer interested in spending public money to solve big problems if doing so gets in the way of cutting taxes.”

Read the full column at The Washington Post.

Infographic of the Day

The Government Accountability Office says that about 40% of the government’s discretionary spending goes to contracts “covering everything from health care to hand grenades.” The U.S. spent more than $550 billion on such contracts in 2018, up $100 billion from 2015 thanks largely to increased defense spending. Dig into the details of that spending with the infographic below or at the GAO blog.

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