An easy pass for Cures; so too for new meds?

Allaying concerns from some quarters that it wouldn’t be passed this year, and coming just one month after the presidential election, the 21st Century Cures Act has passed the Senate as it looks to shake up the way in which the FDA works.

It was an emphatic victory for those who backed the bill: in the end, 94-5 voted for its passing last night that will see $4.8 billion earmarked over the next 10 years to the NIH, and in turn will help fund the BRAIN Initiative, the Precision Medicine Initiative, and the Cancer Moonshot headed by outgoing Vice President Joe Biden.

There is also a $1 billion investment to fight the opioid epidemic, as well as $500 million for the FDA. The bill is in addition reauthorizing the Creating Hope Act pediatric priority review voucher program.

It also aims to speed up the time it takes new medicines to get to patients, while favoring the “voice” of the patient more in its reviews and decisions, and allowing clinical trials to be designed with fewer patients while being less expensive, and generally easier-to-achieve goals.

So-called real world evidence, outside of the traditional RCT, will also be given greater prominence, something favored by the industry given these data are easier for companies to collate.

My inbox was full of statements on its passing yesterday, with those from patient and pharma groups—who paid millions in lobbying costs for Cures to pass—overwhelmingly positive in their response, all congratulating those who worked on getting the bill passed.

But not everyone is happy. Senator Bernie Sanders, a fierce critic of pharma and high drug prices, was one of a handful of voices voting against the bill yesterday, with Public Citizen, long a critic of the shake-up, saying Congress had “stuffed pharma’s stocking” with its passing.

“It is sorely disappointing that Congress gave Big Pharma and the medical device industry an early Christmas present by passing the 21st Century Cures Act. This gift—which 1,300 lobbyists, mostly from pharmaceutical companies, helped sell—comes at the expense of patient safety by undermining requirements for ensuring safe and effective medications and medical devices.”

Speaking to the WSJ, Jerry Avorn, a medical professor at Harvard Medical School, said: “The worry is that this could be a potentially toxic combination, a kind of one-two punch. The bill instructs the FDA to use nonconventional methods to approve drugs, in the hands of a new FDA commissioner with a deregulatory bent.”

This also comes at a time when the FDA is at a crossroad of its own. Much has been made of its controversial decision to pass a new drug for DMD in September based on relatively weak data and a small patient group.

Many internal agency members and advisers argued against the approval of Sarepta’s drug, but these concerns were in essence overruled by Janet Woodcock and FDA Commissioner Robert Califf.

It led several FDA staffers to come out in the intervening months to argue that this was not a good model for the agency to take. One of those, the head of the FDA’s new drugs office, John Jenkins, is now set to retire early next year. Woodcock, who during the Sarepta debates was strongly in favor of an approval, will take over in the interim.

Ron Farkas, a vocal FDA critic of Sarepta’s Duchenne med, also left the agency in early September to join CRO Parexel, with no fanfare, official PR or formal explanation. There is no evidence that both left because of the Sarepta decision, but the timing of these departures has raised eyebrows.

This also all comes as Califf will tender his resignation to the new president (a formal part of the process), which Donald Trump can either accept or reject.

Yesterday, Bloomberg reported that Trump may in fact seek out his own man for the top FDA job, with tech capitalist Jim O’Neill said to be the top contender.

No formal decision either way has been made, but O’Neill would be something of an odd choice: he is an associate of Trump supporter Peter Thiel, the billionaire entrepreneur and venture capitalist who co-founded PayPal.

O'Neill also has no scientific background and little healthcare-related experience, working as an MD at Thiel’s tech investment firm Mithril.

This also all dovetails with Trump’s healthcare policy, still in its infancy, that promises to “reform the FDA,” although further details have not been forthcoming.

You might expect this to send another Trump bump into biotech stocks, but most were hit yesterday when Trump, named Time Magazine’s Person of the Year, told the publication in a brief statement that he would “bring down drugs prices. I don’t like what’s happened with drug prices.”

Cue the Nasdaq biotech index down by nearly 3% yesterday and the S&A biotech down 4%.

The legislation will now be passed along to the outgoing president Barack Obama, who has already said he will sign it “as soon as it reaches my desk.”