Sen. Ron Johnson threatens to obstruct passage of the bill funding the FDA if “right-to-try” language isn’t added [Respectful Insolence]

Here we go again.

It’s been a while since I’ve written about the cruel sham that is known as “right to try.” Anyone who’s been reading this blog a while (or even read the first sentence of this paragraph) knows that I’m not a fan of right-to-try laws. Basically such laws, which have sprung up like kudzu since the movement to pass them gained momentum three years ago, claim to allow terminally ill patients the “right to try” experimental therapeutics. Thus far, they have been sold to the public as giving terminally ill patients “one last shot” and touting how such laws could save some of their lives. As a result, as I’ve grimly quipped on multiple occasions, to politicians opposing right-to-try laws has become very much akin to opposing motherhood, apple pie, and the American flag. So most of them, even ones with misgivings, find that it’s much easier to go along than to explain to the voters, the vast majority of whom do not understand the drug approval process and therefore do not understand why right-to-try laws are harmful to the very patients their proponents purport to be helping. So at the state level, right-to-try advocates have basically won. As of this writing, 37 states have passed such laws in a mere three years, and I wouldn’t be surprised if the remaining 13 states without a right-to-try law pass their own versions within the next year or two. I like to recount how when Governor Jerry Brown vetoed California’s first attempt at a right-to-try law, I was surprised that he had the guts to do it. Thus far, he’s the only governor to have vetoed a right=to-try bill, and even then the California legislature passed another one a year later that he signed. Basically, in any state where a right-to-try law is introduced, it passes.

Of course, part of the reason why I like ot refer to right-to-try as “placebo legislation” is because states don’t have the power to regulate drug approval; that is, unless a drug is entirely developed and marketed only within one state, which is basically never the case. The federal government does, and that process is controlled by the U.S. Food and Drug Administration. The libertarian “think tank” the Goldwater Institute, which developed the model legislation upon which virtually all state-level right-to-try laws are based, is quite blatant about understanding that, as a practical matter, state right-to-try laws do, in essence, nothing. Indeed, right-to-try laws, despite over three years on the books for the oldest ones, have thus far been a miserable failure in getting experimental drugs to patients, their claimed purpose, the claims of their proponents notwithstanding. Basically, no patient represented as having gotten drugs under right-to-try did not already have access to the FDA’s expanded access program.

Of course, the purpose of state laws is not really to make it easier for experimental therapeutics to reach terminally ill patients. Even the Goldwater Institute knows that states alone can do very little to do that; the real purpose behind these laws is to build pressure on Congress to pass a federal law and, at the same time, to gut the regulatory power of the FDA. As a byproduct, because such laws allow very early stage experimental therapeutics to be “tried,” such laws also have the potential to encourage quackery like Stanislaw Burzynski’s antineoplastons.

Thus far, right-to-try advocates have tried twice that I know of to pass a federal right-to-try law, first in the form of the Compassionate Freedom of Choice Act in 2014 and then last year in the form of the Trickett Wendler Right to Try Act of 2016. Neither went anywhere. But, like naturopaths seeking state licensure, right-to-try advocates are as relentless as the Terminator. So it’s not surprising that Senator Ron Johnson (R-WI), who introduced the last right-to-try bill, is at it again:

Sen. Ron Johnson is threatening to hold up a bill funding the Food and Drug Administration unless the House sends the Senate a version of the bill that expands terminally ill patients’ access to experimental drugs.

Johnson, R-Wis., is hoping the House includes the so-called “right to try” language in a funding bill it will take up on Wednesday under a suspension of House rules. But if not, he said he expects to prevent quick passage of the bill in the Senate once the upper chamber receives it.

“If the House bill comes to the Senate without ‘right to try’ language in it, I will have no choice but to object to any unanimous consent agreements related to it or any related bill unless right to try is added or the Senate is given an opportunity to vote on my right to try bill as an amendment,” Johnson said in a statement obtained first by the Washington Examiner. “It is time to stand up for terminally ill patients who just want reclaim their freedom by having the right to try and save their own lives – who want the right to hope.”

Sen. Johnson published a longer statement on his website.

The bill to which Sen. Johnson is recurring is the the Prescription Drug User Fee Act (PDUFA), which is is the law that allows the FDA to collect user fees from drug companies to fund the drug approval process. It turns out that the current legislative authority for the PDUFA expires at the end of September and needs to be renewed before then if the FDA is to continue to function. Right-to-try proponents thus see an opportunity to sneak a provision into the PDUFA renewal that would serve their ends as well as a federal right-to-try law—better, in fact, because getting a “right-to-try” provision into PDUFA would “bake” right-to-try into the FDA for at least the next seven years in its very funding. I first noted this effort three months ago.

Of course, Sen. Johnson isn’t just limiting his efforts to PDUFA. He’s resurrected his previous right-to-try bill, as well:

Johnson’s efforts on advancing right-to-try stalled in the Senate last year, something he blamed at the time on then-Minority Leader Harry Reid. Johnson’s 2017 version of the bill has 46 co-sponsors, including bipartisan support from Democratic Sens. Joe Donnelly and Joe Manchin of Indiana and West Virginia, respectively, and independent Sen. Angus King of Maine.

“I have spoken with House leadership and Chairman Walden over the last several weeks and encouraged them to support incorporating right-to-try legislation in the FDA reauthorization bill that is set to pass the House on Wednesday,” Johnson said. “It is my hope that they will support the millions of Americans and their families suffering from terminal illnesses by including right to try language in this bill.”

Notice how much Sen. Johnson oversells right-to-try in the form of his proposed amendment to PDUFA and, now, the S. 204, the Trickett Wendler Right to Try Act of 2017. He paints it as something that could potentially help millions of Americans suffering from terminal illnesses. Of course, the vast majority of Americans suffering from terminal illnesses suffer from diseases that derive from one thing: Old age. We’re talking heart failure, stroke, cancer, and in the coming years, dementia, such as Alzheimer’s disease. These are diseases for which granting access to experimental therapeutics before they’re approved is incredibly unlikely to save lives and very unlikely even to prolong very many lives. No one doubts that the legislators sponsoring such legislation feel real compassion for terminally ill patients and want to help. It’s just that they’re going about it in the wrong way. To see why, let’s step back and review the issues with state and federal right-to-try laws.

The basic premise behind right-to-try laws is that people are dying in droves because the FDA is too slow, bureaucratic, and hidebound to allow dying patients access to experimental drugs that are still undergoing clinical trials to be approved by the FDA. No, really, that’s the argument libertarians, at least, actually make, that the FDA is literally (yes, I mean literally—just ask Nick Gillespie and Ronald Bailey) “killing” people. Enter right-to-try, laws that purport to allow terminally ill patients (or, in some cases, patients with life-threatening but not necessarily terminal illnesses) to access experimental therapeutics in a desperate bid to save their lives.

It all sounds reasonable on the surface, right? What is very intentionally not emphasized are other aspects of these laws that are potentially profoundly harmful to patients. For instance, there is no mechanism in most right-to-try laws to help patients seeking to access experimental therapeutics financially. Indeed, as I’ve pointed out every time I’ve written about right-to-try, if you look at the language of the actual bills you’ll see that they go of their way to emphasize that health insurance companies do not have to pay for right-to-try treatments. Indeed, the language is often such that the laws could easily be interpreted to absolve health insurance companies from paying for the treatment of complications arising from the use of right-to-try drugs or devices! Given that such bills also allow pharmaceutical companies to charge for experimental therapeutics and such expenses can be very high, this effectively means that only the rich or those skilled (or whose families are skilled) at using social media to raise a lot of money fast could potentially access right-to-try.

All such bills also explicitly eliminate or very much limit the right of patients to sue for malpractice and the power of state medical boards to go after the license of physicians who use right-to-try. If something goes wrong that’s the physician’s, not the drug’s, fault, the patient using right-to-try basically has no recourse. Nor can drug manufacturers be sued; right-to-try gives them immunity. So, basically these laws tell terminally ill patients: Good luck. You’re on your own. Given that right-to-try laws also only require that experimental therapeutics have passed phase I trials and still be in clinical trials to be eligible, there’s a high probability of adverse events and harm. Indeed, I not uncommonly laugh derisively and contemptuously (and contemptuously and derisively) whenever I hear a Goldwater Institute flack claim with a straight face that right-to-try only allows drugs that have been shown to be safe to be used, because phase I trials generally only have a few dozen patients followed briefly. Let’s just put it this way: No one who knows what he’s talking about views drugs that have passed phase I trials as having been “shown to be safe.” At best, such drugs have been shown not to be obviously dangerous.

Of course, as I’ve discussed at least twice, a federal right-to-try law would be so much worse than state right-to-try laws because it would actually do something. It would not be placebo legislation; it would be harmful. Basically, it would federalize right-to-try, but that’s not all it would do. Let’s review the 2017 version, which is virtually identical to the 2016 version. As all previous versions have done, the 2017 version would make it impossible to sue any “producer, manufacturer, distributor, prescriber, dispenser, possessor, or user of an experimental drug, biological product, or device for the production, manufacture, distribution, prescribing, dispensing, possession, or use of an experimental drug, biological product, or device that is in compliance” with this law. This clause basically removes federal liability from physicians and drug companies that offer a drug under right-to-try. If a patient suffers because of the inappropriate use of such a drug, the patient (or, given that the patients under this bill have terminal illnesses, the family) has no recourse to sue the manufacturer under federal law, in addition to having no recourse under state law. That’s bad enough. Even worse is that the use of experimental drugs under right-to-try, unlike the use of experimental drugs under “compassionate use” or early access programs, is not overseen by an Institutional Review Board (IRB), the committee at each institution doing human subjects research whose function is to protect patient interests. If anything, the federal version even more anti-patient than state right-to-try laws.

Then there’s this:

(2)No use of outcomes
Notwithstanding any other provision of law, the outcome of any production, manufacture, distribution, prescribing, dispensing, possession, or use of an experimental drug, biological product, or device that was done in compliance with subsection (a) shall not be used by a Federal agency reviewing the experimental drug, biological product, or device to delay or otherwise adversely impact review or approval of such experimental drug, biological product, or device.

Obviously, this clause is intended to reassure drug and device manufacturers, whose main concern is that if a patient suffers a complication from an experimental medication or device approval of that drug or device could be in jeopardy. So what does Sen. Johnson do? He adds instructions in his bill to the FDA not to use outcomes data from patients using right-to-try drugs in its deliberations on approving drugs. Even if a patient death—heck, make it dozens or even hundreds of patient deaths—can be clearly attributed to the use of an experimental drug, if that drug was used under right-to-try in all the cases this law would tell the FDA that it has to turn a blind eye to all the carnage in considering whether the drug can be approved. I kid you not. Think I’m exaggerating? Look at the language of the bill!

Horrifyingly, given the change in administrations, this bill very well might pass.

Basically, right-to-try is based on two myths, as Alison Bateman-House, Kelly McBride Folkers, and Arthur Caplan describe:

Although promoted in many cases by individuals with benevolent intentions, the right-to-try approach is based on two myths. The first is that the FDA’s expanded access program, which allows patients to use an experimental drug outside of a clinical trial if the drug’s manufacturer agrees to provide it, is slow and ineffective. In fact, it is hard to envision how the agency could provide a faster turn-around time on expanded access requests while still conducting a thorough review of a patient’s medical history and proposed treatment plan. The form that physicians must submit to the FDA for review after a company agrees to supply a patient with an experimental drug requires less than one hour to complete. The FDA approves more than 99 percent of these requests, and it does so, on average, within four days. For emergency requests, the agency responds in one day or less. And this is no “rubber stamp” review: A recent study found that FDA reviewers weighed in with valuable suggestions about how to improve the proposal, making it more likely to help than hurt the patient.

The second myth—one that is especially pernicious—is that right-to-try legislation will allow patients faster and more guaranteed access to experimental drugs by cutting out the bureaucratic “middleman” (the FDA). Indeed, the central objective of right-to-try legislation is to end FDA oversight over the use of experimental drugs. For terminally ill patients, ending FDA oversight over experimental drugs would expose the patients to exploitation without guaranteeing access to the drugs they seek. And weakening the FDA puts everyone else who takes drugs or uses medical devices or vaccines at grave risk.

Exactly. Right-to-try is a Trojan horse designed to gut the FDA’s authority to regulate and approve experimental therapeutics. It’s also the “foot in the door” for more deregulation. Imnagine, for instance, that right-to-try is approved. You can bet that in a couple of years (or even sooner) the Goldwater Institute will start asking why right-to-try is restricted only to “terminally ill” patients. Why, it will ask, shouldn’t patients with serious medical conditions that are not immediately life-threatening “reap the benefits” of right-to-try?

The NYU School of Medicine Working Group on Compassionate Use and Pre-Approval Access has published a three-page fact sheet about right-to-try. Most important is to strengthen and streamline the FDA’s Expanded Access Program while at the same time making it appealing to drug companies to participate. Right-to-try might fails on all these counts. It is the very epitome of H.L. Mencken’s maxim, “For every complex problem there is an answer that is clear, simple, and wrong.” In the case of right-to-try, unfortunately, this answer is not only clear, simple, and wrong, but it’s incredibly appealing because is uses the compassion we all feel for the terminally ill to short circuit our critical thinking abilities for a purpose (gutting the FDA) that most of the bill’s sponsors and supporters don’t understand but that the Goldwater Institute does.