When last I discussed the cruel sham that is the tide of “right-to-try” laws that has been flowing through state legislatures to become law over the last year and a half. “Right-to-try” laws, as I pointed out when I first noted the earliest ones being promoted in Colorado, Louisiana, Arizona, and Missouri, referring to them as Dallas Buyers Club bills based on their seeming inspiration from that movie and pointing out how they are very, very bad policy that, contrary to the claims of its proponents, are far more likely to harm patients than help them. In every state in which such bills have been introduced they have passed with little opposition. I expected the same thing to happen with the right-to-try bill (AB 159) introduced in California, and it seemed to be happening, with the bill passing the legislature with relative ease.
Then Governor Jerry Brown did something unprecedented. He vetoed AB 159:
Gov. Jerry Brown vetoed a measure Sunday that would have allowed terminally ill patients easier access to experimental treatments that have not yet been approved by the federal government.
The measure by Assemblyman Ian Calderon (D-Whittier), dubbed the “right-to-try” act by supporters, would have authorized drug manufacturers to let patients facing imminent death obtain drugs that are still undergoing review by the Food and Drug Administration.
The FDA does allow for such patients to apply for “compassionate use” access to medications that are still in clinical trials, but proponents of the “right-to-try” legislation say the application can take too long for the gravely ill.
Brown, in his veto message of AB 159, noted the FDA recently streamlined the “compassionate use” program.
“Before authorizing an alternative state pathway, we should give this federal expedited process a chance to work,” Brown wrote.
I was amazed. I was awed. My respect for Gov. Brown, which had been rather low when he added an unnecessary signing statement to a law designed to make nonmedical exemptions to school vaccine mandates, thus neutering it, and recovered when he signed SB 277 into law, eliminating nonmedical exemptions altogether, took another tick upward. The reason is that his veto indicates an understanding of why right-to-try laws are a cruel sham, coupled with real political courage. The reason right-to-try laws have been passing so easily is that their proponents have successfully co-opted the terms of the debate, pitting suffering, terminally ill patients who believe that their only hope to live is an experimental drug against the cold, seemingly uncaring bureaucracy of the FDA. What politician wants to be portrayed as taking hope away from such patients? Not many. Indeed, California Bill AB 159 is even worse than most in at least one aspect: It doesn’t limit access to terminally ill patients but rather to patients with an “immediately life-threatening disease or condition,” defined as “a stage of disease in which there is a reasonable likelihood that death will occur within a matter of months.”
Basically, right-to-try laws all follow the same template provided by a libertarian think tank, the Goldwater Institute. The idea is to make it easier for terminally ill patients to access experimental drugs and devices. The requirements are risibly low and betray a total lack of understanding of how drug development works in that they only require that the experimental drug (1) have passed phase I trials and (2) still be in clinical trials. Of course, phase I trials are not designed to test efficacy. Their purpose is to work out optimal dosage, identify the maximal tolerated dose, and identify major side effects. Worse, they usually only consist of a few tens of patients, often less than 30. To propose letting seriously ill patients drugs that have been tested in so few people and not demonstrated to be efficacious and safe is to invite disaster. In addition, they provide no financial support for patients, who are basically on their own when it comes to paying what can be the substantial financial charges. In addition, right-to-try laws strip away patient protections, making it virtually impossible for a patient injured using such a drug to sue either the drug company or the physician administering the drug. That’s even leaving aside the fact that drug approval is controlled by the federal government, and drug companies will be highly reluctant to offer such drugs without the approval of the FDA, and the FDA already has programs for single patient INDs, also known as compassionate use, to allow patients access to experimental drugs.
Over the last year and a half, state legislature after state legislature, believing, based on the dishonest propaganda of advocates who claim that the FDA is killing people and the Goldwater Institute cynically featuring the sympathetic stories of dying patients (particularly those with Lou Gehrig’s disease) to advance its agenda, has fallen under the spell of right-to-try. It passed in Michigan last year, sneakily pushed through the legislature. Over the last year, state after state passed these ill-advised laws. Not surprisingly, several months ago and more than a year after the first of these laws passed, right-to-try advocates couldn’t point to a single patient helped by these laws, and even patients were starting to realize that they’ve been sold a bill of goods in the name of an antiregulatory fervor to weaken the FDA, which was the real reason all along for these laws. The Goldwater Institute just used terminally ill patients to lobby state legislatures, that and the fact that most people don’t understand drug development and think that the arguments for right-to-try sound reasonable because of it. It’s no wonder that patients feel betrayed and disappointed. Right-to-try laws, by their very design, create false expectation and false hope.
Because of the widespread misunderstanding of drug approval (specifically the primacy of the FDA over any state law) and the lack of attention paid to the patient-hostile provisions of the Goldwater Institute template for right-to-try laws that strip away legal protections and greatly weaken the right to legal recourse in the case of harm, these laws have passed 24 states. California would have been the 25th. No one seems to realize that the true purpose of right-to-try laws is to build a political consensus to weaken the FDA (as libertarian Nick Gillespie basically admits). Indeed, the Tenth Amendment Center virtually explicitly says as much:
Back in 1996, California voters recognized that keeping medicine away from sick people was a federal policy they could no longer ignore, and they passed Prop. 215 to legalize medical marijuana. The Right to Try Act is based on the same principle. And when enough people and enough states say no to federal bans, there’s not much that Washington D.C. can do about it.
“The Right to Try Act is a no-brainer,” Maharrey said. “When someone is on their deathbed, the fact that FDA regulations would let them die rather than try, has got to be one of the most inhumane policies of the federal government. Every state should nullify the FDA like this.”
Actually, as compelling as this sounds, this argument is far more about appealing to emotion rather than reason. In actuality, given the small proportion of drugs that make it successfully through the whole regulatory process after phase I, patients with terminal illnesses are far more likely to be harmed then helped by releasing experimental drugs that have only passed phase I trials. The frequent retort is that these patients are terminally ill and things can’t get worse, but there is something worse than being terminally ill. It’s being terminally ill and exhausting the last of your finances and even going into debt. It’s being terminally ill and harming yourself so that your are less able to do what you want to do in your short remaining time. It’s being terminally ill and dying sooner than you have to.
Moreover, as Gov. Brown noted, the FDA already has a compassionate use program that it is modernizing to make the process less onerous. It’s a process that is inherently superior than any state “right-to-try” process because patients who go through it do not give up any of their legal protections, must be monitored the same way clinical trial subjects receiving the experimental drug are, and remain under the regulation of an institutional review board (IRB), which protects human subjects and provides ethical oversight. Gov. Brown is correct; it is foolish for state legislatures to try to interfere with that process until it can be evaluated whether the changes the FDA has proposed in its draft guidance have had an effect. These guidelines even include a provision that, if the application process is still too slow for a particular patient, in an “emergency situation that requires the patient to be treated before a written submission can be made, the request to use the investigational drug for individual patient expanded access may be made by telephone (or other rapid means of communication) to the appropriate FDA review division, and authorization of the emergency use may be given by the FDA official over the telephone,” provided the physician explains how the use of the experimental drug meets the statutory requirements and a written submission is sent to FDA within 15 working days of the authorization to use the drug.
I mentioned earlier that, as of several months ago, no one has yet been able to identify a patient whom a right-to-try law had helped to obtain an experimental drug:
However, more recently, a number of questions have been raised as to whether these bills have actually brought experimental treatments to the patients in need, and whether they’re even necessary because of a more simplified FDA compassionate use program.
Dr. Arthur Caplan, director of the Division of Medical Ethics at New York University’s Langone Medical Center, who’s also part of the NYU Working Group on Compassionate Use and Pre-Approval Access, told reporters in late August: “It’s fair to say we haven’t yet found a case where right to try laws have facilitated access to anything.”
Caplan and several others also previously wrote in Health Affairs that although the “laws have created an expectation that terminally ill patients will be able to quickly access potentially life-saving treatments by being exempted” from FDA rules, “this expectation is, quite simply, false.
“Putting aside the invalidity of RTT laws [because of federal law preempting state law] and the false hope they create, these laws also fail to address more fundamental health and safety issues. RTT laws seek to grant vulnerable patients access to unproven and potentially harmful drugs or other medical products without any expert safeguards, such as scientific or ethical review by an Institutional Review Board or similar body,” Caplan and others wrote.
Right-to-try laws sound on the surface to be compassionate and reasonable. They are nothing of the sort. Unfortunately, it’s not at all a given that Gov. Brown’s veto will stand. The bill passed the California Senate by a vote of 40-0 (even the normally science-based Senator Arthur Pan, co-author of SB 277, voted for it) and the Assembly by a vote of 76-2. Not surprisingly, its supporters are already lobbying the legislature to override Gov. Brown’s veto. It wouldn’t surprise me if they succeed, but I hope that they don’t. Either way, Gov. Brown has shown us that it is possible to say no to this onrushing madness.