House passes right-to-try on second try

The House of Representatives passed on party lines Wednesday evening a bill designed to let very sick patients request access to experimental medicines without government oversight.

The passage of the bill, known as right-to-try, is a big victory for the small libertarian think tank that crafted the proposal — and for a White House that has vigorously campaigned for the law. And it comes one week after Republicans failed to pass a similar measure through an expedited process that needed two-thirds support from the chamber.

The bill passed by a vote of 267-149 with 35 Democrats in favor and two Republicans against. It nowheads to the Senate, which passed a slightly different version of the legislation last summer. With a final law all but assured, health policy advocates who have vigorously opposed the idea are now looking toward the broader anti-regulatory health agenda being pushed by the Goldwater Institute.

Arizona-based Goldwater has a handful of other ideas that could dramatically weaken the authority of the FDA to oversee the safety and effectiveness of medicines and has already made progress getting one of those ideas — giving drug companies more freedom to market their products — on state lawmakers' agenda. Goldwater spearheaded passage of a law in Arizona's legislature last year that allows companies to promote non-FDA-approved uses of their products to doctors without fear of repercussions by state officials. It helped get similar bills introduced in Colorado and Missouri's legislative sessions this year and expects other states to follow suit soon.

Some are concerned that Goldwater could be emboldened by the right-to-try success.

"I think this is the first step, for sure. Tear down as many regulations as possible, take away all oversight, and let it be the Wild West of medicine," said Andrew McFadyen, executive director of the rare disease patient group Issac Foundation and a member of New York University's working group on compassionate use and pre-approval access.

"Republicans know this doesn't give access to patients. But they opened the door to Koch brothers and Goldwater to rip apart the FDA, and then other government bodies after that," McFadyen added.

The victory also highlights the success of Goldwater's strategy of pushing legislation state-by-state — 38 states have approved — and using that momentum to take it to the national level, said Christopher Robertson, an expert on health law and bioethics at the University of Arizona.

It's "easy for them to bend the ear of local politicians," Robertson said, and "even if the state legislation is completely ineffectual it does present one symbolic victory after the next." Moreover, the average state legislator doesn't need to understand the intricacies of FDA law, making it easier for Goldwater to sway state lawmakers. Besides drug marketing, Robertson said pushing to get more medicines available over the counter, without a doctor's prescription, is also on the libertarian group's agenda.

Senate Majority leader Mitch McConnell's office declined to provide any guidance on if and when the Senate might consider the House proposal, but the chamber is expected to eventually pass the House bill.

The Senate cleared its version of right-to-try over the summer on unanimous consent. But it stalled in the House due to concerns from patient advocacy groups, bioethicists, drug companies and President Donald Trump's own FDA commissioner, Scott Gottlieb.

After months of deliberation, the House released an updated version last week with tweaks designed to make it safer for patients and encourage more drug companies to participate — but Democrats continued to oppose the bill, arguing it wouldn't help more patients to get access to experimental medicines and could cause them harm.

With the GOP attempting to pass it under an expedited process requiring a two-thirds vote, Democrats sank the bill last week, knowing full well that it would pass easily when brought back under regular procedures requiring a simple majority for passage.

Trump, who called for swift passage of the bill during the State of the Union in January, called out Democrats for stalling the legislation at a speech Monday in New Hampshire.

"It is hope. It's incredible. They've been talking about this for years and years and years. We're going to get it approved. So important," Trump said.

But Democrats continued to push back against the law Wednesday. Rep. Frank Pallone (D-N.J.), ranking member of the House Energy and Commerce Committee, said right-to-try doesn't address the true barrier facing patients requesting experimental drugs — resistance from drug companies — and thus offers patients little more than "false hope."

"The fact is when a patient is denied access to an experiential treatment, it is because a company has said no, not the FDA. So let's be clear about what this legislation is. It's an attempt to undermine the authority of the expert public health agencies charged with reviewing drugs to ensure their safety and efficacy," Pallone said during House debate of the bill.

Pallone's argument got a boost from four former FDA commissioners who served under Republican and Democratic presidents. Margaret Hamburg, Robert Califf, Mark McClellan and Andrew von Eschenbach issued a statement earlier this week saying "there is no evidence" that either the House or Senate right-to-try bills "would meaningfully improve access for patients, but both would remove the FDA from the process and create a dangerous precedent that would erode protections for vulnerable patients,"

Ultimately, Republicans' messaging won the day.

"It's not false hope; It is hope. Support of this bill is compassionate. Support of this bill is fair," said Rep. Andy Biggs (R-Ariz.), one of the original sponsors of the House legislation.