Trudy Lieberman has an important post on a story that has gone below the media radar, on how a bill based on the bogus premise that the FDA is impeding terminal patients’ ability to get access to experimental drugs, is moving forward in Congress and garnering unwarranted favorable commentary. Here is her overview:
Right before Congress adjourned for the August recess, the Senate rushed through “right to try” legislation that its sponsor Sen. Ron Johnson of Wisconsin argued gives terminally ill patients the “right to access treatments that have demonstrated a level of safety and could potentially save their lives.” Johnson was selling hope, a commodity never in short supply in America’s bag of marketing tricks. He also was selling something else–a way to circumvent the FDA’s role in a process that has been in place for several decades that lets very sick people try experimental approaches not yet on the market…
But the legislation, on its way to possibly an early vote in the House of Representatives in the next few weeks, eliminates the FDA in the process and with it any protections patients might have.
This might sound fine and logical if you don’t know the fact. The FDA already has a process for approving the experimental drugs for the terminally ill. The process is fast, with emergency sign-offs in 24 hours and the rest processed in 3-4 days. 99% of the applications are approved. So the idea that this is a problem is simply not true. Moreover, when there is a hangup, it’s typically that the drugmaker doesn’t want the use approved.
In addition, the FDA also provides useful input in these cases:
For example, the FDA may know about other drugs in the same class as the experimental approach and can warn of problems that have arisen with those drugs. It also can ask for safety adjustments in administering the experimental drug. For example, should the dosage be changed, or is a heart monitor needed?
On top of that, there are downsides to patients who go outside the FDA approved process:
…few patients in the 37 states that have adopted such laws are likely to know they may lose hospice coverage or they may be denied coverage for home health care if they are using an experimental drug. In Colorado, Connecticut, Oklahoma, and West Virginia, patients may lose their health insurance. Their coverage may be denied for six months after the experimental approach ends.
Lieberman describes how the legislation did get some good coverage when it was first proposed by the Washington Post, Vox, MedPage Today…in other words, stories oriented towards DC or medical industry insiders. And they were skeptical of the need for the bill. There was some cheerleading reporting in Johnson’s state of Wisconsin. Reporting was otherwise spotty to non-existant, save some op-eds also backing the proposal.
What interests me as least as much as Lieberman’s story is who might be behind this campaign to curb the FDA’s role. And it isn’t Big Pharma.
Admittedly, there is a clear moving force. An organization called Goldwater Institute, “a libertarian think tank,” is the leader of the campaign for this bill and similar legislation passed in 37 states. But even though individuals can have pet causes, it seems to be peculiar to be fixated on one where the driver has to be opposition to the FDA, as opposed to the issue at hand, since the notion that the FDA is a problem here does not stand up to scrutiny. In other words, this effort looks to be more about legitimating the use of experimental drugs and not about concern for the terminally ill.
And who might be in favor of that? Liberman again:
Who might benefit then, if not the drug companies? The organization Who.What.Why. reported that new drug startups might be interested in creating buzz for their products and finding a new revenue source–wealthy patients who can pay for their super-expensive experimental medicines. Dishonest doctors might be interested, too, in the fees they could collect for monitoring patients who use these drugs.
And who is the money behind “new drug startups”? Silicon Valley. Let us not forget that it was the FDA that effectively put an end to one-time tech darling Theranos. Peter Thiel, who is trying to live forever by getting blood transfusions from young people, has also been trying to pressure the FDA by launching an ethically questionable, non-FDA approved herpes vaccine trial in St. Kitts. But it’s hard to take that particular effort to challenge the regulator seriously, since his unapproved human “trial” had a grand total of 20 participants. Not only did the St. Kitts government launch an investigation, but the press uproar over the trials led to a climbdown within days. From CNBC:
The CEO of a herpes vaccine company at the center of a controversy over an offshore clinical trial on Friday said that future testing of the drug will follow U.S. Food and Drug Administration oversight rules, as demanded by a new group of financial backers that includes venture capitalist Peter Thiel.
In an impassioned interview, Rational Vaccines Chief Agustin Fernandez said he feared the great promise of the vaccine developed by the late university professor William Halford was being overshadowed by criticism over Halford’s decision to test the drug using American patients on the West Indian nation of St. Kitts and Nevis without monitoring by the FDA or an institutional review board (IRB), as is traditional.
Orc at Science Blogs connected the dots between the dodgy “right to try” legislation for the terminally ill and the broader libertarian war against the FDA. Forgive me for quoting at length:
I’ve also caught flak for characterizing right-to-try as being part of a broader war against the FDA (indeed, as part of a broader war against regulation in general) by libertarian-leaning free market fundamentalists, some of whom claim (literally) that the FDA is killing people. Lots of people. How can you say that? proponents of right-to-try would ask, drawing themselves up in their best self righteous fury. This is about helping terminally ill patients! Maybe. But, as I’ve said before, the FDA already has a Compassionate Use program that achieves the same purpose and rarely turns requests down, a program that’s been improved over the last couple of years to be speedier and more responsive, with much less application time. Consistent with its libertarian origins, right-to-try tries to cut the FDA out of decisions between a drug seller and patients, but at the cost of eliminating many protections for patients. For instance, unlike the case in “expanded access” or “compassionate use,” the institutional review board (IRB), an ethics panel that oversees clinical trials does not oversee patients in right-to-try. There is no financial help; so only patients whose families are rich or can raise a lot of money fast are likely to be able to benefit. Even worse, as I’ve described, most right-to-try bills not only do not require insurance companies cover experimental therapeutics (and why should they?), but allow insurance companies not to cover charges for complications from using experimental therapeutics. However, perhaps the most egregiously false argument made by right-to-try advocates is that the laws’ allow use of experimental therapeutics after they’ve been “proven safe” through phase I testing. Yes, you read that right. As I’ve discussed multiple times, there is the requirement that the drug or device has only passed phase 1 trials, which, given how few drugs that have passed phase 1 actually make it through to approval, is a really low bar, especially since most phase 1 trials involve fewer than around 25 patients.
Although not commonly seen as such, right-to-try laws are of a piece with libertarian and radical fundamentalist free market views, some of which claim that the FDA is basically unnecessary or that its role should be vastly constrained. You can see it all there: Minimal requirements for safety testing (phase I), no ethical oversight, the decision entirely up to the company and patient, and, of course, the patient is totally on his or her own. People with such views were even in consideration for the post of FDA Commissioner, for example, two cronies of the aforementioned Peter Thiel, both of whom believe that the FDA stifles their holy grail of “innovation.” The first was Jim O’Neill, who has advocated that the FDA test new drugs only for safety, not efficacy, thus returning the FDA to its pre-1962, pre-thalidomide role. The second was Balaji Srinivasan, a Silicon Valley entrepreneur who advocated basically doing away with the current system of FDA regulation and replacing it with online reviews, basically a Yelp or Über for drugs and medical devices. When asked “How do you prevent quacks?” Srinivasan replied, “Scaled Internet reputation systems. Works at massive scale in other areas.” So clueless were both of these men that it was a relief when a relatively normal Republican pharma shill named Scott Gottlieb was ultimately appointed. He was the “least bad,” most conventional choice, someone any conservative Republican administration might have appointed. Yes, he wants to “streamline” the process by which, for example, vaccines are approved, but he’s actually pretty conventional. He’s even decided to http://www.npr.org/sections/health-shots/2017/08/28/546719842/fda-cracks-down-on-stem-cell-clinics-selling-unapproved-treatments.
Stymied at the FDA, apparently Peter Thiel is now waging his war on the FDA by other means:
Defying U.S. safety protections for human trials, an American university and a group of wealthy libertarians, including a prominent Donald Trump supporter, are backing the offshore testing of an experimental herpes vaccine.
The American businessmen, including Trump adviser Peter Thiel, invested $7 million in the ongoing vaccine research, according to the U.S. company behind it. Southern Illinois University also trumpeted the research and the study’s lead researcher, even though he did not rely on traditional U.S. safety oversight in the first trial, held on the Caribbean island of St. Kitts.
Neither the Food and Drug Administration nor a safety panel known as an institutional review board, or an “IRB,” monitored the testing of a vaccine its creators say prevents herpes outbreaks. Most of the 20 participants were Americans with herpes who were flown to the island several times to be vaccinated, according to Rational Vaccines, the company that oversaw the trial.
“What they’re doing is patently unethical,” said Jonathan Zenilman, chief of Johns Hopkins Bayview Medical Center’s Infectious Diseases Division. “There’s a reason why researchers rely on these protections. People can die.”
Indeed it is. What Peter Thiel and Rational Vaccines, a company founded by Agustín Fernández III and William Halford (formerly of Southern Illinois University who died recently of cancer) did and are doing is patently unethical—shockingly, horrifyingly so. The very function of the IRB is to protect human subjects in clinical trials. Usually, when a clinical trial is proposed, it goes through two committees, first the scientific review board, or SRB, which determines whether the trial is scientifically justified and soundly designed. The second is the IRB, which is charged with protecting the human subjects. Its function is to examine the clinical trial design and to determine whether it is ethical and whether the risks to human subjects do not outweigh the potential benefits; e.g., that there is clinical equipoise. Once it approves the trial, the IRB continues to monitor the trial, examining reports of adverse events, both serious and minor, and sometimes demanding changes in the trial or even canceling it if it judges that there are too many adverse events or that one group is doing so much better than the other group that it would be unethical to continue the trial. These protections for human subjects flow from the Belmont Report and the Common Rule, both of which were designed to prevent the abuse of human research subjects like what the Nazis did and underlie all the federal regulation regarding human research protection. Unfortunately, these protections are not universal, as they only apply to organizations with federal funding (such as universities) or entities applying for FDA approval for one of its investigational drugs or devices.
Like right-to-try, Peter Thiel’s and Rational Vaccines’ offshore clinical trial of a new herpes vaccine is an attack on the FDA. Libertarians and free market fundamentalists even admit as much:
The push behind the vaccine is as much political as medical. President Trump has vowed to speed up the FDA’s approval of some medicines. FDA Commissioner Scott Gottlieb, who had deep financial ties to the pharmaceutical industry, slammed the FDA before his confirmation for over-prioritizing consumer protection to the detriment of medical innovations.
“This is a test case,” said Bartley Madden, a retired Credit Suisse banker and policy adviser to the conservative Heartland Institute, who is another investor in the vaccine. “The FDA is standing in the way, and Americans are going to hear about this and demand action.”
In other words, while I haven’t looked into whether there are direct ties between the Silicon Valley FDA opponents and the Goldwater Institute, they are at a minimum fellow travelers. And even though Thiel’s herpes vaccine gambit backfired, don’t expect him and his allies to give up easily.
And in case you need a reminder as to why rich men need to be protected from themselves, let me hoist some text from a 2008 post:
Consider the sad fate of Eben Byers, an athlete, industrialist, and man about town of the 1920s. After sustaining an arm injury that refused to heal, his doctor prescribed (and received a 17% patent rebate on) a patent medicine. Eben thought it did him a great deal of good and began taking the potion two to three times a day.
The drink, Radithor, was radium dissolved in water. Byers lost his teeth and most of the bone mass in his jaw, and before his death, developed abcesses on his brain and holes in his skull. But the maker of the toxic potion was never prosecuted, since selling radium drinks was not against the law.
By the time Byers had the vast misfortune to happen upon Radithor, the risks of radium were coming to light, as factory workers who painted radium onto clocks and would use their lips to establish a point on their brushes were developing lip and mouth cancers. But the potion-makers nevertheless continued to sell their tonics until the Byers death killed the industry.
Byer’s horrific and widely-publicized death was the impetus for greatly strengthening the powers of the FDA. But at least poor Byers experimented only upon himself. Thiel is perfectly comfortable with turning people into his guinea pigs.