“Right to try” goes federal, thus far unsuccessfully

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.”

Next up:

(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.

Finally:

(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.