Sen. Ron Johnson threatens to obstruct passage of the bill funding the FDA if "right-to-try" language isn't added

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.

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.

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The FDA manages to adhere to the concept of do-no-harm under the most difficult circumstances (e.g., lethal injections).

http://www.nytimes.com/2011/01/28/opinion/28fri3.html

The right-to-try concept must have checks and balances, thereafter, making the FDA indispensable.

By Michael J. Dochniak (not verified) on 17 Jul 2017 #permalink

I appreciate this and join you in opposing "right to try" as extremely patient-hostile legislation that will not let patients gain access to investigational products.

Just wanted to point out that Hawaii also vetoed a right to try bill, SB 2181, in April 2016:
http://www.capitol.hawaii.gov/Archives/measure_indiv_Archives.aspx?bill…

A new bill, SB 1110, was introduced in Jan 2017 but hasn't moved, and the session is in recess till Jan 2018.

Regards,
Lisa Kearns

Lisa Kearns, MS, MA
Senior Research Associate
Member: Working Group on Compassionate Use and Pre-Approval Access
Division of Medical Ethics
NYU School of Medicine

By Lisa Kearns (not verified) on 17 Jul 2017 #permalink

OT: Over the day I got error messages along the line "too many requests" when I tried to open a post. Was there an attempted DoS attack today, did the Slashdot effect hit or was the problem likely on my side?

By aairfccha (not verified) on 17 Jul 2017 #permalink

@ aairfccha
I got exactly the same messages for a large part of the weekend and today, so I suppose the problem wasn't on your side.

And the problem still hasn't disapeared.

@MJD: As the bill is written, the FDA would have no say whatsoever in right-to-try cases. And the patient explicitly has no recourse should anything go wrong. The only people who could prevent a drug being used in a right-to-try case are the manufacturers, who could choose not to make it available (as it is, they typically make enough for the current trial phase, with some allowance for spoilage, but not more). And then to tell the FDA that they cannot consider outcomes of right-to-try cases is a mockery of evidence-based judgment: they are being explicitly ordered to ignore evidence.

@aairfccha: You are not alone; I have been getting that same error message since Saturday at home, and today here at work. This post is the first SB content I have seen since Friday afternoon. Orac, you may want to have the corporate (non-pharma) overlords at SB look into this, if they aren't already.

By Eric Lund (not verified) on 17 Jul 2017 #permalink

@MJD: As the bill is written, the FDA would have no say whatsoever in right-to-try cases. And the patient explicitly has no recourse should anything go wrong. The only people who could prevent a drug being used in a right-to-try case are the manufacturers, who could choose not to make it available (as it is, they typically make enough for the current trial phase, with some allowance for spoilage, but not more). And then to tell the FDA that they cannot consider outcomes of right-to-try cases is a mockery of evidence-based judgment: they are being explicitly ordered to ignore evidence.

@aairfccha: You are not alone; I have been getting that same error message since Saturday at home, and today here at work. This post is the first SB content I have seen since Friday afternoon. Orac, you may want to have the corporate (non-pharma) overlords at SB look into this, if they aren't already. Another possibility is that there is malicious code in one of the ads being served, because I shouldn't be getting such a result by simply surfing over to this site, and I have.

This will be a second attempt at posting this comment, because I got that error message again on the first attempt.

By Eric Lund (not verified) on 17 Jul 2017 #permalink

@MJD: As the bill is written, the FDA would have no say whatsoever in right-to-try cases. And the patient explicitly has no recourse should anything go wrong. The only people who could prevent a drug being used in a right-to-try case are the manufacturers, who could choose not to make it available (as it is, they typically make enough for the current trial phase, with some allowance for spoilage, but not more). And then to tell the FDA that they cannot consider outcomes of right-to-try cases is a mockery of evidence-based judgment: they are being explicitly ordered to ignore evidence.

@aairfccha: You are not alone; I have been getting that same error message since Saturday at home, and today here at work. This post is the first SB content I have seen since Friday afternoon. Orac, you may want to have the corporate (non-pharma) overlords at SB look into this, if they aren't already. Another possibility is that there is malicious code in one of the ads being served, because I shouldn't be getting such a result by simply surfing over to this site, and I have.

This will be a secondthird attempt at posting this comment, because I got that error message again on the first two attempts.

By Eric Lund (not verified) on 17 Jul 2017 #permalink

Sorry for the duplicate posts. As I said, I continued to get the error message on my first two attempts, so I had no way of knowing that those attempts had posted.

By Eric Lund (not verified) on 17 Jul 2017 #permalink

Another possibility is that there is malicious code in one of the ads being served.

Naa, the problem is with one superflous module of the security software Wordfence. So, I'll post the solution again:

We have since back in December seen some issues on Bluehost because they install something called Endurance cache and some version of this cache is too aggressive and will actually cache the Wordfence blocked page and then serve it to every visitor to the site. This is why it doesn’t change when you deactivate Wordfence. It’s not actually Wordfence that is serving the page, it’s the cache. We believe that it is one of the early versions of Endurance cache that is faulty, but it seems like on many sites it has not been upgraded to newer versions.

The easiest way to solve this may be to contact Bluehost and ask them to remove the faulting Endurance Cache from your website. If you want to try disabling it yourself you can:

1. Look in wp-content/mu-plugins and delete the endurance cache plugins from there
2. Check your .htaccess and manually remove any trace of the Endurance cache…

“Just ran into this issue with a client using Bluehost and just wanted to add that deleting/renaming the endurance-page-cache folder did nothing for me. It just regenerated. The only way to disable was to go to Plugins > Must Use and disable there.

https://wordpress.org/support/topic/your-access-to-this-site-has-been-l…

I seem to have access to RI again, but what I did earlier today was to use my TOR browser, and reset my circuit for the site when I got the error message (happened a few times even with TOR). Clunky but it worked well enough.

An issue with right to try that bothers me greatly is the reliance of libertarian and tea party types to think that free markets are the solution to everything. "If only" we'd let the free markets "work their magic" we'd have miracle cures for everyone.

And a line of bodies miles long.

People seem to confuse a chance with any chance, not realizing the latter is so unlikely as to come to fruition, but it makes for an emotional argument under the umbrella of American determination and fighting spirit. "If you had any chance, wouldn't you take it?"

And the answer to that question, in many instances, should be "No, no I wouldn't, not if I could live longer, in less pain and more dignity by taking hospice services."

What Senator Johnson knows about modern medicine, clinical trials, ethics or biology would not fill a teacup. Out of a dollhouse.

"Passed Phase I" does not mean "safe". It means something closer to "doesn't obviously kill patients super fast".
At least one Phase II immunotherapy trial had to be halted recently because patients were dying. How on earth is dead now better than dead in 3 months?

Or what about Novartis' new immunotherapy? That treatment requires a huge amount of supportive care so the patient doesn't die of the treatment. Under "right to try" if the prescribing physician didn't arrange that care and the patient died for lack of it, the family would be SOL, even though it was clear malpractice.

Startup pharma doesn't want this; they don't have the material to spare. Ethical pharma doesn't want this, it's nuts. I'm not even sure amoral pharma would want this; there's too much risk to reputation.
And evil pharma seems to do just fine themselves.

This serves nothing but ideology. And I , for one, have no truck with that.

By JustaTech (not verified) on 17 Jul 2017 #permalink

Sen. Johnson has been rather persistent with this issue. One wonders who is pushing this. Where is the funding? The fact that it has been introduced via many states over a short period of time implies a centralized well financed effort. Someone with big money to be made I'd wager.

By captian_a (not verified) on 17 Jul 2017 #permalink

Well, these laws are a product of the Goldwater Institute, which has been the single biggest promoter of right-to-try laws. Every state RTT law is based on a legislative template written by the Goldwater Institute three or four years ago. A couple of years ago when the Michigan legislature was considering RTT, the Goldwater Institute flew in its flacks and a bunch of patients with terminal illnesses and family members. Those standing up for science were hopelessly outgunned.

Unfortunately, right-to-try sounds so good; how could anyone object to giving patients more rights? For people thinking with their gut and not with their head, the answer is obvious. Since anti-intellectualism is rampant in our society right now, we run the risk of many laws being passed which don't work out very well because they try to mold reality to our beliefs.

I was having trouble connecting to Respectful Insolence via iPad. Clearing browsing history allowed access again.

Regarding funding: Jan Mayer's book, "Dark Money" details how wealthy people set up non-profit foundations so they can fund right-wing think-tanks with tax-free dollars. Some of that money can also be routed to political causes as long as it isn't their primary purpose. It's all a part of distorting our political landscape courtesy of all three branches of government.

By cloudskimmer (not verified) on 18 Jul 2017 #permalink

As a widow of someone who died from Stage 4 lung cancer, I have a different perspective. I was heartbroken when my husband was diagnosed. We both understood that a Stage IV diagnosis would mean that he would be given palliative instead of curative treatment. At that time, Nivolumab was showing incredible results in phase 3 clinical trials. During his illness, the drug was approved for squamous cell NSCLC but not adeno (he had adeno). We tried several times to get into trials but there were problems (distance, PDL-1 levels, drew the taxotere straw, etc..) Because he was treated at Kaiser, he couldn't get it approved off-label even after Opdivo was approved for squamous.
We switched insurance and discovered he had brain mets. He did wbr and gk for the brain mets and then his new dr. approved Opdivo off-label. His first scan showed improvement in his lung and liver tumours, but unfortunately, the cancer had settled in his meninges. He died 15 days later.
I can never know, but I always wonder if successful, systemic treatment of his primary tumor in the beginning could have prevented his cancer from spreading to his brain. I will never know. But I do know that the current system of clinical trials and off-label use is advantageous for the wealthy who can afford better insurance and travel. (of course wealth is an advantage in everything).
I don't think 'right to try' is the answer, but damn it, can't we figure something out for incredibly promising phase 3 drugs?

By PollylovesJoe (not verified) on 19 Jul 2017 #permalink

Polly,

I'm so sorry for your loss.

The short answer to your question is no. Otherwise there would be no point in holding a phase 3 clinical trial. Sometimes a drug does so well, they end the trial early but that's not the norm. Opdivo was not one of those drugs.

I'm not sure what "figuring out something for incredibly promising" really even means. The problem would be, everyone would clamor for the drug and say, "why not me?" That muddles the data and maybe we end up allowing people access to a drug that isn't all that hot after all.

That happens a lot with drugs. At first it sounds great, but as more data comes in we find either the drug doesn't work well enough to justify its high costs, or the side effects aren't worth the benefits.

I'm sure there are things we can do to improve the system but the bottom line is there will always be someone who will want access to a drug undergoing clinical trials and can't have it for whatever reason. This is one of those greater good issues, which isn't easy to hear when your loved one is the one being told no for the greater good.

Thanks for your kind response. Intellectually, I understand that allowing anyone and everyone to take a medication would muddle the data. But I can't tell you how sad/frustrating/heartbreaking it is to know there's something out there working and not being able to do anything to access it. Within a few months of diagnosis, I had heard about Nivolumab. The data coming out wasn't miraculous but it was much better than chemo and with much less severe side-effects. I wish my husband could have gotten his shot at taking Nivolumab sooner. Nothing anyone's ever going to say is going to talk me out of that position.
I am so grateful for the treatment my husband did receive.His doctors, nurses, technicians, etc.., all had so much compassion and did the best they could for him. He had 20 months of high quality life after his diagnosis. He didn't spend one night in the hospital (almost unheard of for lc), and he died peacefully at home in his own bed.
I am a solid proponent of science and so was he but ...I will leave it at that.

By PollylovesJoe (not verified) on 20 Jul 2017 #permalink

Polly, I would never disrespect you by saying you shouldn't feel what you feel.

I'm glad he was able to pass at home. That is a dignity that was denied my father because I couldn't get the logistics to work in time :(

I’m glad he was able to pass at home.

Same here, but for my mom (Herceptin worked well for a good four years, but the final evasion was to the lungs).