'Right to try' is an ill-considered bill

'Right to try' is an ill-considered bill
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Food and Drug Administration Commissioner Scott Gottlieb is often given credit for straying from the Trump administration script by being public health and evidence-focused. But there’s one respect in which he, at times, takes his cues from the top — making policy by tweeting.

Take the controversy over “right to try” bills, which have sought to cut Food and Drug Administration (FDA) out of the process through which severely ill patients obtain access to drugs in development that have not been approved by FDA. That debate reached a climax on Tuesday when the House passed such a bill.

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In many ways the FDA’s expanded access process was a poor target for reform. For decades, the agency has had a carefully crafted program that balanced the desires of desperate patients for access to unproven drugs against the need to protect vulnerable patients from drug dangers and unscrupulous operators. It did all of this while ensuring the collection of data that can prove whether the drugs are safe and effective.

 

Under that program, the FDA quickly reviewed requests to use unapproved drugs, in order to not delay treatment, and has modified about 11 percent of the proposals it received to improve the likelihood of a good outcome. That safety protection would be lost with the right to try bill.

The legislation, the brainchild of a libertarian think tank with a much broader anti-regulatory agenda, targeted the FDA as part of a wider assault on drug-approval standards. However, the real obstacle to expanded access is not FDA, but rather the pharmaceutical industry. And right to try does nothing to compel companies to provide their investigational drugs.

One company acknowledged turning down 98 of 160 applications for just one drug in a six-month period, while another reportedly turned down “hundreds” of applications for its drug over a two-year period. In contrast, over six years, FDA turned down only 66 of the thousands of applications it received. That’s because the applications typically lacked critical data or because an approved product that the patient had not yet tried was available.

And the agency has embraced improvements, streamlining its application procedures (full disclosure: I led that effort), making sure ethical review is appropriate but not overly burdensome, and helping develop an online navigator to assist patients seeking expanded access.

So where was Dr. Gottlieb on all this? Well, in October 2017 when he testified before the House Energy and Commerce Committee’s Subcommittee on Health, he offered a fulsome defense of the FDA program and expressed a number of concerns about the right to try bill that had been passed by the Senate.

It included certain patients for whom effective treatments were available, he said, and appeared to limit FDA’s ability to protect patients from abuses, and excluded certain parties in the expanded access process. His concerns were shared by members of the House, who, after consultation with FDA, designed and passed their own right to try bill that addressed many of Dr. Gottlieb’s concerns.

In an interview with Stat, Dr. Gottlieb evinced a preference for the House-passed version, but then insisted in a Tweet that Stat had erred in its account. In a separate Tweet, he offered support for both bills, despite his previous criticisms of the Senate version and FDA’s longstanding concerns about right to try (see my 2016 testimony here). One of his last tweets before the vote, when he realized that the House would vote on the Senate bill, said it all, “FDA supports the [Senate] bill moving to the house floor and stands ready to implement it in a way consistent with the intent of Congress to promote access and protect patients.”

The fact is that both bills attempted to solve a problem that doesn’t exist: The FDA is not a roadblock for patients who want to try unapproved, last-ditch drugs — after all, the agency approves 99 percent of applications it receives.

However, the Senate’s version was the more dangerous of the bills. The House version contained more robust informed consent requirements and had a better procedure for reporting and assessing patient harm that may result from the investigational drug.

Four former FDA commissioners — two Republicans and two Democrats — opposed the right to try legislation, regardless of the version, as did approximately 100 patient groups.

Even Energy and Commerce Chairman Greg WaldenGregory (Greg) Paul WaldenHouse GOP blocks Trump-supported drug pricing provision from spending bill GOP turns its fire on Google Hillicon Valley: Twitter chief faces GOP anger over bias | DOJ convenes meeting on bias claims | Rubio clashes with Alex Jones | DHS chief urges lawmakers to pass cyber bill | Sanders bill takes aim at Amazon MORE, who had led the House effort to create better right to try legislation, abandoned his own bill under pressure from the president and vice president. The senate version passed the House 250-169 yesterday and now awaits the president’s signature.

This legislation is perfect for the Age of Trump: heedless of the facts, driven by emotion, mindlessly anti-regulatory and certain to be exploited for political purposes despite its failure to solve anything.

But the complexity of concerns involved was poorly suited to Twitter’s 280-character limit. No doubt when the president signs this ill-considered bill, we will hear about Dr. Gottlieb’s newfound enthusiasm for it in a Tweet #Sad.

Peter Lurie M.D. was associate commissioner at the FDA until 2017 and testified before the Senate on right to try legislation.