A victory and a more substantial defeat for the cruel sham known as “right to try”

I’ve referred to so-called “right to try” laws as a cruel sham.on more than one occasion. Since 2014, these laws, all based on a template provided by the libertarian Goldwater Institute, have been proliferating at the state level with the help of lobbying by the aforementioned Goldwater Institute and a concept that makes it pitifully easy to caricature opposition to these laws as wanting to heartlessly snatch away from terminally ill patients the last chance at life while laughing and twirling one’s mustache like Snidely Whiplash. Not surprisingly, state legislatures all over the country have found such laws irresistible, leading to their passage in over 30 states in just two and a half years. Over the last week, right-to-try has had a major victory in that, contrary to what he did last year and contrary to the hope of science advocates, California Governor Jerry Brown signed the right-to-try bill (AB-1668) that was passed earlier this month. However, it has also suffered a major defeat in that a couple of days ago the federal right-to-try bill was blocked in the Senate.

The basic premise behind right-to-try laws is that people are dying in droves because the FDA is too slow and too 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 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. Sounds reasonable on the surface, right? What is assiduously not mentioned are other libertarian-based aspects of these laws. For instance, there is no mechanism in most right-to-try laws to help patients seeking to access experimental therapeutics financially. Indeed, pointedly, such bills go out of their way to state that health insurance companies do not have to pay for suc treatments and can be interpreted to state that they don’t have to pay for treating 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.

These laws also explicitly remove patient protections in that most of them state that doctors recommending right-to-try can’t be sued for malpractice or disciplined by their state medical boards, seemingly no matter how inappropriate or incompetently executed such a request might be. Nor can drug manufacturers be sued. Basically, these laws tell terminally ill patients: Good luck. You’re on your own. And don’t sue if things go bad, no matter what. 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 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 have high levels of life-threatening toxicity.

Of course, the biggest flaw in these laws is that it is federal law, not state law, that controls drug approval. Right-to-try laws can say that terminally ill patients have the “right” to access experimental therapeutics, but it is the FDA that determines whether they, in fact, do. Companies are understandably reluctant to grant access to experimental therapeutics without the FDA’s prior approval because (1) the FDA will not look kindly upon it and they want FDA approval and (2) if there are any adverse events it could harm their chances of winning approval for their drugs. Also, the FDA does have what it calls its Expanded Access Program (sometimes referred to as Compassionate Use) already to allow terminally ill patients to access experimental therapeutics, and it does it without removing patient protections under Institutional Review Board (IRB) supervision. Moreover, the FDA already grants the overwhelming majority of Expanded Access requests. Indeed, as I pointed out, thus far, after two and a half years of existence, right-to-try has been a miserable failure. The Goldwater Institute can’t identify a single patient who has received an experimental drug under a state right-to-try law, although it claims to know of 40-60. Meanwhile a quack like Stanislaw Burzynski has abused right-to-try. Meanwhile, the only patient I’ve been able to find who actually used right-to-try died.

So it was that I was very disappointed to learn that Governor Jerry Brown had betrayed the citizens of the State of California by buckling under this time:

Terminally ill patients in California will be able to try potentially life-saving medication before it passes FDA final review thanks to a new piece of legislation inspired by the movie “Dallas Buyers Club.”

The bill, dubbed the “Right to Try” law, makes California the 32nd state to allow patients with terminal illnesses to try drugs that have passed the FDA’s Phase 1, but haven’t been fully approved.

Phase 1 is the first stage of drug testing in human patients. Drugmakers earn approval to conduct clinical trials on people after presenting the results of successful trials on animals, according to the FDA. After presenting the data — and a plan for human trials — the FDA determines whether drug companies can go forward with additional testing.

Patients can try experimental treatments only after exhausting all other options, according to the libertarian think tank Goldwater Institute, and the patients’ treatments with Phase 1 drugs cannot be included as data in ongoing clinical trials.

Of course, as I pointed out before when I discussed the California bill, this description is utter bollocks. In a way, the California bill (now law) is worse than the average state right-to-try law. It doesn’t actually require that the patient be terminally ill, only that he has an “immediately life-threatening disease or condition.” As I put it at the time, that’s incredibly broad. A severe case of pneumonia could be “immediately life-threatening.” A heart attack is definitely “immediately life-threatening.” A stroke is “immediately life-threatening.” “Immediately life-threatening” is not the same thing as “terminal.” Yet AB-1668 tries to have it both ways, as it defines “immediately life-threatening disease or condition” as “a stage of disease in which there is a reasonable likelihood that death will occur within a matter of months.” That implies something less acute, but “within a matter of months” encompasses more immediately life-threatening diseases as well.

You could ask, quite reasonably: Why does this matter? One reason is that it’s California, the most populous state in the country. Any law passed in California matters. California is always the biggest prize, and right-to-try advocates were bitterly disappointed when Gov. Brown vetoed a previous right-to-try bill last year.

Unfortunately, the Goldwater Institute has been very effective in co-opting terminally ill patients to use the considerable justified sympathy voters and legislators feel for them to lobby for right-to-try:

In a guest column for the Washington Post, 32-year-old Matthew Bellina says Right to Try laws are an improvement on the FDA’s Expanded Access program because they stipulate that the FDA won’t shut down or delay clinical trials if an experimental treatment goes wrong.

Because derailing clinical trials can deal a significant blow to drug companies that have poured millions of dollars into research and development, drug companies are less likely to sponsor terminally ill patients without guarantees that the FDA won’t retaliate for failed treatment.

Bellina, a military veteran and father who has terminal ALS — amyotrophic lateral sclerosis, also called Lou Gehrig’s disease — testified before the Senate on a federal version of Right to Try.

Turning down someone dying of Lou Gehrig’s disease is pretty much close to impossible for a politician, even if the legislation being proposed is profoundly anti-patient, as right-to-try is. Also, this is one of the most pernicious aspects of right-to-try, as you will see. So let’s segue to the federal right-to-try bill. Gov. Brown might have buckled and signed what I like to refer to as “placebo legislation,” which makes legislators feel good and believe that they’ve done something when in reality they’ve done nothing, but the federal bill is where the action is because that’s the real goal of the Goldwater Institute, to weaken and then ultimately neuter the FDA. The Goldwater Institute is politically savvy enough not to come right out and explicitly say this, but other libertarians are not.

A week ago, hearings were held on a federal right-to-try bill, S.2912, known as The Trickett Wendler Right to Try Act of 2016. This is a bill being pushed by Republican U.S. Sen. Ron Johnson. Interestingly, as the bill has been US Senate Committee on Health, Education, Labor, and Pensions, Johnson used his position as committee chair of the US Senate Committee on Homeland Security and Government Affairs to hold hearings on the bill, even though it has nothing to do with his committee’s purview. Sen. Johnson’s opening statement is basically a rehash of Goldwater Institute talking points, complete with the usual anecdotes about patients with terminal illnesses who might have been saved:

Despite the legal uncertainty there are doctors willing to jeopardize their practice to give patients needed, but unfortunately unapproved, treatments. One of them is Houston oncologist Dr. Ebrahim Delpassand. Even though the FDA has told him no, he bravely continues to treat patients under his state’s law. Now nearly 80 patients, whose chance of survival would be, as he puts it, “close to none,” are alive thanks to his treatment.

This caught my attention, as this is a potentially verifiable claim. There is a video of Dr. Delpassand giving a statement included in the testimony:

Whoa. He’s with Excel Diagnostics, the very same company that I discussed when I looked into the one patient in Texas whom I could find who had accessed right-to-try and who had not been saved. Contrary to Sen. Johnson’s claims, he is not an oncologist; he is a radiologist. Of course, nothing Dr. Delpassand claims in his video statement shows that 80 patients who would have died have been saved, thanks to his being a brave maverick doctor willing to buck the FDA. One notes a highly one-sided account designed to make Dr. Delpassand look as good as possible. In any case, the therapy discussed by Dr. Delpassand does have potential, as I mentioned before. However, one thing that stood out to me was how the Goldwater Institute reached out to Dr. Delpassand. So basically, right-to-try allowed Dr. Delpassand to charge for the use of his treatment, even though it is not FDA-approved. Not surprisingly, the Goldwater Institute is painting this example as the nefarious FDA preventing patients from saving their lives, even though this treatment is not curative, as I described. He’s also a flack for the Goldwater Institute, having participated in a promotional video touting right-to-try:

So his evidence is that “many of these patients were given three or six months to live” and are alive a year later? Seriously? Stanislaw Burzynski uses the same argument about the patients he treats.

In any case, as I pointed out three weeks ago, the federal right-to-try bill is even worse than state right-to-try bills because (1) it would actually do something and (2) what it would do would be very, very bad for patients indeed. For example, it would forbid the FDA from considering adverse events suffered by patients utilizing experimental drugs under right-to-try when considering a drug for approval. Seriously, it says that. A patient could die, clearly as a result of an experimental drug, and the FDA would be explicitly barred from considering that information when deciding whether to approve the drug or not.

Speaking of Stanislaw Burzynski, I couldn’t help but note the testimony of Peter Lurie, MD, MPH. He repeated the same points about how the FDA’s Expanded Access Program rarely rejects requests and then notes:

However, even patients with serious or life-threatening diseases and conditions require protection from unnecessary risks, particularly as, in general, the products they are seeking through expanded access are unapproved – and may never be approved. Moreover, FDA is concerned about the ability of unscrupulous individuals to exploit such desperate patients. Thus, with every request, FDA must determine that the potential patient benefit from the investigational drug justifies the potential risks and that the potential risks are not unreasonable in the context of the disease or condition to be treated.

“Unscrupulous individuals”? That would well describe Stanislaw Burzynski. It could also describe pharmaceutical companies willing to profit off of drugs that made it through phase I studies but are not approved yet.

Fortunately, for now at least, the federal right-to-try bill has been blocked:

Republican U.S. Sen. Ron Johnson’s push for a right-to-try bill ran up against the reality of hardball politics Wednesday.

Johnson’s measure to allow terminally ill patients to receive experimental drugs not approved by the Food and Drug Administration was blocked by Senate Minority Leader Harry Reid (D-Nev.).

Johnson sought to move the bill through unanimous consent, meaning one senator could halt its progress. And that’s what Reid did, blunting a Johnson initiative for the second time in recent months. In July, Reid blocked Johnson’s bill to protect federal whistleblowers from retaliation.

Johnson faces a tough re-election fight against Democrat Russ Feingold, so any move to get legislation through by a parliamentary maneuver was always going to be difficult.

And it’s even harder since Democrats are still upset that Republicans have blocked President Barack Obama’s nominee to the U.S. Supreme Court, Merrick Garland.

Reid said he understood the “seriousness” of the Johnson proposal and acknowledged “the urgency that patients and their families feel when they’re desperate for new treatments.”

In objecting to the measure, Reid said Johnson’s bill didn’t have bipartisan support — there were 40 Republican co-sponsors and two Democrats. He said the bill didn’t go through the hearing process where all the major players on the issue have voice. The Johnson-chaired Homeland Security & Governmental Affairs panel held two hearings on the subject.

“I think we should have had a hearing on Merrick Garland,” Reid said on the Senate floor.

This is what I would call doing the right thing for the wrong reason. Unfortunately, that’s what happens in politics a lot. I’ll take it, though. If state right-to-try bills are basically symbolic rants against the FDA, the passage of a federal right-to-try bill would be a disaster for patients and the clinical trial process.