It's been nearly two weeks since a new "right to try" bill (AB 1668) passed the California legislature with overwhelming support and was sent to Governor Jerry Brown's desk to be signed. Thus far, he has not signed it, which is good, but neither have I seen a story that he has vetoed it either. In the meantime I learned some more about a federal version of the bill, which I will discuss after a brief recap of why right-to-try is such bad policy, which will lead into a discussion of the federal bill.
For those unfamiliar with right-to-try, such bills claim to allow terminally ill (or, in some states, "seriously ill") patients to bypass the FDA and receive potentially promising new experimental drugs that have passed phase I clinical trials and are still being tested in phase II or III clinical trials. I've discussed in detail why such "Dallas Buyers Club" laws are a cruel sham that is unlikely to help terminally ill patients and in the process strip patients of critical protections, such as the right to sue the recommending physician for malpractice or the company for negligence, and in some cases insurance coverage and access to hospice care. Worse, in some cases such laws leave the field open to quacks like Stanislaw Burzynski to use right-to-try to bypass the FDA. Basically, right-to-try is really right-to-buy. If you have a lot of money or can raise a lot of money, you can potentially access it. If you don't and can't, tough luck. Basically, as I've discussed so many times before, you have to remember that the function of right-to-try was never to actually help patients. Right-to-try was the brainchild of the libertarian Goldwater Institute, whose interest is far more in weakening and ultimately neutering the FDA, so that patients can be left to the tender mercies of the free market, which libertarians are deluded enough to believe can do a much better job of ensuring patient safety and drug efficacy than any government regulations. (Seriously, libertarians not infrequently falsely argue that the FDA is killing people by being so slow at approving drugs. I guess they forgot the time before the Food, Drug, and Cosmetics Act. Certainly they forgot the potential harms to patients whose desperation they enlisted to turn right-to-try into a cause few politicians can afford to be seen opposing.
Be that as it may, as you recall, at the time I pointed out that, although Gov. Brown had vetoed a prior version of this bill last fall, this time he might sign it. The reason is that his rationale for vetoing the bill last year was that he wanted to wait and see how well reforms of the Expanded Access/Compassionate Use program worked out. Although I argued that in fact the reforms have been quite successful thus far, for right-to-try advocates, anything short of allowing terminally ill patients unfettered access to experimental drugs. Again, remember, the real driving force behind right-to-try is to neuter the FDA; so expanding access to experimental therapeutics through the FDA just won't do, because the FDA would then still remain the primary determiner of what drugs are approved and who can get experimental drugs. If you don't believe me, ask the Goldwater Institute if any patients have managed to receive experimental therapeutics through right-to-try over the last two and a half years since the first such laws were passed. They'll tell you they're aware of 40 patients but will assiduously decline to provide any more information, as I found out when I took to Twitter to ask for more. In comparison, since 2009, the FDA has granted between 936 and 1,873 Expanded Access requests a year and approves the overwhelming majority of requests, including a 300% increase in emergency INDs, which are requests for single patients who don't have time to complete the regulatory paperwork. Right-to-try, even if it works as the Goldwater Institute claims it wants it to, is having a negligible effect compared to the FDA's already existing policies—just as I've told you it would many times.
Besides undermining the authority of the FDA, another purpose of state level right-to-try laws is to build pressure for a federal law weakening the FDA, and to provide a pretext for lawsuits designed to challenge the FDA's authority to regulate drugs. As Alice Bateman-House puts it:
@gorskon Yes but I'm afraid much more support now. Goldwater's playbook was get laws in at least 25 states & then claim mandate for fed law
— Alison Bateman-House (@ABatemanHouse) August 24, 2016
Unlike the case at the state level, the Goldwater Institute and right-to-try advocates have had a harder time achieving their aims at the federal level. For example, when right-to-try laws were first starting to be passed at the state level, the Compassionate Freedom of Choice Act of 2014 was introduced. It was basically a quack manifesto, and fortunately it didn't go anywhere. Of course, the Goldwater Institute's intent had been the long game all along. So it's not surprising that another federal bill has been introduced:
A bill introduced in the US Senate on Tuesday becomes the latest legislative effort to expand the ability of terminally ill patients to gain access to experimental medicines. The legislation, which joins a companion bill that was introduced in the House last summer, would prohibit the federal government, including the US Food and Drug Administration, from taking any action to prevent patient access.
The bill, which was introduced by Senator Ron Johnson (R-Wis.), comes after 28 states have passed so-called "Right to Try" laws. These allow patients to leapfrog a drug-development process that takes years before new treatments become available. And the laws reflect rising frustration with an FDA program called expanded access, in which people who are seriously ill can obtain a drug under development, even though they aren't enrolled in a clinical trial.
This story is from May; since then three more states have passed right-to-try, to bring the total number of states with such laws to 31. The federal bill, submitted to the Senate, is S.2912, The Trickett Wendler Right to Try Act of 2016. Fortunately, thus far the bill has been languishing in the US Senate Committee on Health, Education, Labor, and Pensions, as its companion bill, H.R.3012, The Right To Try Act of 2015 has been languishing in the relevant House committee since last summer. Given that this is an election year and not much of anything is happening legislatively (heck, Congress can't even seem to pass a budget), this bill is unlikely to pass this Congress, but it's not hard to imagine its being introduced next year, with much more support. Let's take a look at what it does.
I'm not a lawyer, as we say, but this law is clear enough that I understand what it could do. First, it explicitly states that state right-to-try laws trump the Federal Food, Drug, and Cosmetic Act and the Controlled Substances Act:
(a) IN GENERAL.—Notwithstanding the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 301 et seq.), the Controlled Substances Act (21 U.S.C. 801 et seq.), and any other provision of Federal law, the Federal Government shall not take any action to prohibit or restrict—
(1) the production, manufacture, distribution, prescribing, or dispensing of an experimental drug, biological product, or device that—
(A) is intended to treat a patient who has been diagnosed with a terminal illness; and
(B) is authorized by, and in accordance with, State law; and
(2) the possession or use of an experimental drug, biological product, or device—
(A) that is described in subparagraphs (A) and (B) of paragraph (1); and
(B) for which the patient has received a certification from a physician, who is in good standing with the physician's certifying organization or board, that the patient has exhausted, or otherwise does not meet qualifying criteria to receive, any other available treatment options.
In other words, this bill, if passed, would explicitly federalize each state's right-to-try law, in the process eliminating the FDA's ability to protect terminally ill patients from what could be dangerous or inappropriate drugs. Worse, it trusts the states not to produce right-to-try laws that are too dangerous, the sole exception is that it requires that the illness being treated be "terminal." The problem, of course, is that this bill does not define "terminal."
(1) NO LIABILITY.—Notwithstanding any other provision of law, no liability shall lie against a 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 subsection (a).
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. Add to that the lack of oversight by an IRB, and this law is profoundly anti-patient—just like state right-to-try laws.
(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.
This clause is clearly intended to address the concern of drug and device manufacturers that if they provide an experimental therapeutic to a patient under right-to-try and the patient suffers complications due to the treatment, the approval of its product might well be jeopardized. This, of course, is not an unreasonable concern on the part of manufacturers, given that it can easily cost $1 billion to bring a drug or device to market, and by the time the product has passed phase I clinical trials a great deal of that cost has already been invested in development. However, this clause goes way too far. Basically, it says that even if a patient death is clearly due to use of an experimental drug under right-to-try, that death should not be considered by the FDA in deciding whether to approve the drug. Think of it this way. Let's say dozens of patients die from using an experimental drug under right-to-try. This bill, if passed, would bar the FDA from even considering those deaths during its deliberations regarding whether to approve the drug for marketing or not.
Basically, the Trickett Wendler Right to Try Act of 2016 would not help terminally patients. It would endanger them. Proponents often ask, "What does a terminally ill patient have to lose?" The answers are simple. They can lose money, perhaps their life savings, given that accessing right-to-try could easily cost tens or hundreds of thousands of dollars. That's because, when you come right down to it, "right to try" is a misnomer. It's really "right to buy." If you're terminally ill and don't have the money or the means to raise it, right-to-try will not help you. Worse, thanks to right-to-try, terminally ill patients can lose some of their precious quality time remaining with their loved ones if they suffer complications that place them in a hospital or prematurely kill them.
The California right-to-try bill being considered by Gov. Jerry Brown and the Trickett Wendler Right to Try Act of 2016, like all of the other right-to-try laws based on the Goldwater Institute template and passed by other states, are profoundly anti-patient. Like the equally ill-advised 21st Century Cures Act, right-to-try bills are based on the delusion that there are oodles and oodles of cures for deadly diseases out there that could save thousands of lives if only the evil government and FDA would step aside and get out of the way of the free market. I say "delusion" because the FDA, despite being underfunded, is actually pretty efficient at new drug approvals, evaluating nearly all new drug applications within 6 to 10 months, an impressive turnaround for such complex assessments. Indeed, the FDA actually acts more rapidly than its European counterparts approving new drugs. Basically, there is no evidence that the FDA hampers overall medical innovation, nor is there evidence that the FDA's current requirements lead to higher drug prices or cost lives.
None of this is to say that the FDA is perfect. Far from it. Certainly there is a discussion to be had about how, in the case of desperate patients with terminal illnesses, we as a society should balance individual rights versus risk/benefit considerations in making decisions about how freely we allow experimental therapeutics to be used to try to save these patients. It's not as though we haven't had this discussion before, either. We had it in the 1980s during the AIDS epidemic, and we've periodically revisited it since then. This is another such time, and the FDA has actually responded by vastly simplifying its procedure for granting Expanded Access, and the vast majority of such requests are granted.
It must be reiterated that, as much as right-to-try is enormously popular because no one wants to deny a terminally ill patients his or her "last chance," the purpose of right-to-try was never to help terminally ill patients. There is no evidence, at least none that the Goldwater Institute has yet produced or that I've been able to find anywhere, that right-to-try has not, as far as I've yet been able to ascertain, allowed a single terminally ill patient to access an experimental drug or helped him or her to live longer, much less that it has saved a single life. (True, I have found one possible patient who might be receiving treatment under right-to-try, but I need to look into the case more before I feel comfortable blogging about it.) Unfortunately, right-to-try has been a cynically successful strategy to weaponize sympathetic patients and basic human empathy for patients facing imminent death to attack and ultimately greatly weaken the FDA.
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The wording of the bill is so transparent that it is hard to see how anybody could fail to see its intent.
Remind me again how its skeptics who are pursuing an industry-driven agenda?
If anything should be able to kill these bills, it should be the "No liability" and "No outcomes" clauses. And yet, we see incredible myopia on the part of legislatures.
@ 2 Todd W.
I'm relatively okay with the no liability clause. It's a bit like the MS study at the Ottawa Hospital where the consent form was described along the lines of "every second paragraph said that this treatment may kill you ".
The "No outcomes" clause is clearly insane. Whom the gods ...
And the rest of the world is going to have to figure out a way to monitor these instances as presumably CDC or FDA or whoever will not be doing so.
My problem with the no liability clause is that even if the manufacturer knows that the risks of the product outweigh any potential benefits, and fails to disclose that to the patient, the patient or their family has no legal recourse available to them. They cannot sue the manufacturer for damages. So even the libertarian's answer to everything, lawsuits, can't be used to correct the situation.
@Todd: Not only are there risks, but the law is designed to stop the manufacturer from ever having to declare what those risks are. They can continue to lie about the risks and the one agency tasked with assessing how accurate such claims are, will be prevented, by law, from doing so.
I want to know who drafted this bullshit. The most evil pharma corporate exec in the world would sure not be this brazen. Only quacks would do this. I suspect chronic lyme charlatans, personally.
It's one thing to waive liability in the context of informed consent. The patient is told that the treatment is experimental and has certain risks. Likewise, if I go cross-country skiing on trails for which I am expected to pay a trail fee, I have to sign a piece of paper saying that I know that skiing is inherently dangerous and if I get hurt, it's not the ski area's fault. But that usually only applies to risks that a patient or a skier can reasonably foresee. If there are dangers that the company running the drug trial knows about but didn't disclose, or the ski area puts a novice rating on a trail that should have been marked as a double-diamond run, the patient or skier may have recourse. RTT laws eliminate that possibility.
God knows what happens to clinical trials in the midst of that kind of free for all. They'd be be pretty much screwed, I guess.
Certainly we know that, in placebo controlled trials, participants often get tested to see if they're on placebo, and many lie about what they're taking on the side.
And the way recruitment of treatment-naive participants would dry up means it would be hard to mount them in any kind of timely manner.
Hell, I guess you'd just have to let people take whatever they want. As was said back in the days of high dose AZT in the 1980s: "they're gonna die anyway."
Funny how libertarians always say that product liability suits will keep the free market "honest" but then always seem to oppose laws that make suing easier and support policies that make it harder for consumers to sue manufacturers. Yes, funny that.
What if the last clause (no use of outcomes) got reversed, and there was instead an obligation to disclose and publish every treatment with experimental drugs, with the name of the person/clinic that prescribing treatment, the name of the substance, the manufacturer, the expected outcome and the actual outcome?
@VDG: That's only a slight improvement. Yes, we'd know about adverse outcomes. But anybody who was taking an experimental medication under a RTT regime is unlikely to be following a rigorously controlled regime, so all you would get out of this is anecdata. That may be of some use to fans of so-called pragmatic trials, but our host is on record as opposing pragmatic trials, for reasons I generally agree with (to the extent a non-expert like me is entitled to an opinion).
So, to someone like me, the answer to fixing the FDA is somewhat obvious. Instead of hamstringing them and then complaining about the poor job they do ( which is the libertarian MO but leaving that aside) they should put more money in to hire more people and increase efficiency. Only makes sense to me.
Besides, that way the FDA becomes a job creator.
Here's an open letter I wrote a couple years ago, when there were only 5 states with RTT laws on the books.
Brian Deer reminds me of another possible negative outcome of this sort of Federal legislation: creating a disincentive for completing clinical trials to get FDA approval. After all, clinical trials are freakin' expensive. If you can sell your product without having to go through the rigors of phase 2 and 3 trials, why bother? Especially since under RTT you could actually charge patients for the product, rather than the more ethical and usual practice of providing the experimental treatment for free. While the bill would require the product to be in trials, there is no requirement that those trials actually need to progress. So you could have people like Burzynski who register their trials, then let them just sit open for decades with no end in sight.
"After all, clinical trials are freakin’ expensive. If you can sell your product without having to go through the rigors of phase 2 and 3 trials, why bother? "
Bingo. Especially with the undefined "terminal" qualifier, which I could easily see being the target of later right-to-try legislation, softening it under the guise of "what's the harm?" combined with freedom of choice.
Well it seems clear that this draft is bad. But the idea isn't to me, we should be at least coming up with an alternative that could work and was fair.
What about incorporating it in with the clinical trials, once people get diagnosed as terminal, offer them 3-5 different experimental treatments. The companies get cost of materials only, capped at $500, it's a non profit obligation, call it Phase 1b or 2b. Limit it to a one year window per treatment to limit abuse from both sides. EVERYTHING gets reported, though allow patients to mix and match, that might still be interesting data. For actual clinical trials people get treated for free, though the government should part subsidise this part of the trial.
Actually I forgot, I wrote the above based on the UK's National Health Service, with all this being free to the end user.
But maybe if it was capped at something reasonable, it could help drive some of the outrageous prices down.
I read a great Science Fiction series once that opined that the government should maintain a nationalised industry in every endeavour to prevent private business from mischief, like monopolies, overcharging and bad worker treatment.
Nationalize "Right to Try"!
We already have an alternative to the RTT laws that are popping up: the FDA expanded use and compassionate use regulations.
The FDA does everything these laws propose to do with the following benefits: data must be gathered from these one-off uses to factor into the risk-benefit analysis; charging patients for the "privilege" of being guinea pigs must be reasonable and justifiable (and more of an exception than the rule); manufacturers, etc. can still be sued if they engage in malfeasance.
That one needs abolishing. It certainly is a boon to somebody with all the 'no studies to show'.
First cannabis and now kratom. The ban on kratom, which alleviates pain and staves off opiod withdrawl comes concurrent with a cracking down on pain meds. So now there will be buprenorphine and, ohh, maybe
Mmm, mmm. I'll bet it will be real expensive to.
Oh my gilbert wants to abolish the controlled substances act.
Thanks Todd, please pardon my British ignorance. So this Bill is double useless for actual patience then,
, so glad we have the NHS.
So, time for another letter to my senator (who happens to be the ranking member on this committee)?
Something along the lines of "This bill will kill patients, and destroy the biotech industry. Please vote no, and let all the researchers, clinicians and biotech/pharma workers keep working to ethically provide treatments that work."
No worries. There's a lot of similarity between this bill and the Saatchi bill, since you mention Britain.
@VDG #9: An outcomes clause would be useful for determining the who is using total quack therapies under "right to try" like those of Burzynski ( that is assuming Burzynski actually reported it , given how little of anything he reports.)
Luckily for Britain, by the time the "Saatchi" bill passed, it had been so modified that it no longer had any practical provisions. See here:
Thanks for sharing that. I hadn't really followed it too much after its first death. While I'm glad to see that Saatchi's original provisions completely and utterly failed, as they should have, what a colossal waste of time!
I won't comment too much on this specific legislation, INAL, I certainly support concepts for individuals (with friends and family) to try to save themselves, without necessarily giving Merck, Pfizer, Roche, et al a free pass for a new blockbuster like Vioxx, with their New Drug Entities.
...for which the patient has received a certification from a physician, ..., that the patient has exhausted, or otherwise does not meet qualifying criteria to receive, any other available treatment options... is actually over restrictive to my eyes.
The time to start if you have advanced therapies is much sooner, not later. Clinical medicine lags many years behind the literature for various low risk opportunities that seem to work fine in aggregate, just not as a single magic bullet.
The FDA should not be allowed to criminalize seriously ill individuals or their families for getting important medicines overseas. Right now, bringing in approved cancer medicines from overseas is a crime, subject to immediate seizure at anytime while in the US, even if fatal. FDA and ICE merely have a policy of "forebearance" for your "crimes".
My "science project" has more than triple the maximal OS estimate from an MD Anderson oncologist and over 4x the normal OS average, using drug and therapies not commercially available in the US. Also, I will note the maximal OS examples that I have tracked down, either had "whimpy biomarkers" or massive immunological events e.g. Coley's examples. Neither apply.
So I think many views here are ill informed and dangerous to me and mine.
Fifteen deaths world wide vs:
Why are they doing this?
Chuck Rosenberg is a fuckbag^2
Why are they doing this?
Chuck Rosenberg is a really swell guy.
Like the poorly-made Walmart key breaking off in the lock, This is what happens when one fakes nature for a patent instead of allowing one to use something perfectly safe they can grow in the back yard:
The same kind of thing happens with 'K2' or 'spice', synthetic thc. There is a natural alternative which already works. Sick. Greedy sick peddlers.
It is disgusting. Wikipedia locked the page after the DEA deflowering/raping.
compare and contrast
Same ol', same ol'.
As of May 10, 2016 SB226 makes kratom a schedule 1 controlled substance in Alabama.
There has been an explosive increase of the rate of overdoses from heroin, fentanyl, and prescription opiates since the ban went into effect -- Extrapolating from just this one county in Alabama, I predict an increase from 129 deaths per day nationwide to 300-500 deaths per day (It is hard to know how many have been using kratom to supplant heroin and pharma) starting in a month, or so -- Heckuvajob Rosenburg.
Though the DEA ban is not in effect yet, the threat of it has haulted commerce in kratom. While many have 'stocked up' for a month to a year, It's still a good bet to set the watch for today being the start of the pharma-induced (Rosenburg was a lobbyist for drug companies) apocolypse.
While the DEA is autonomous with there asshattery, Congress can defund them: