The "Compassionate Freedom of Choice Act of 2014": Pernicious "health freedom" nonsense that degrades human research subject protections

I was torn about what to blog about today. There were lots of things floating around that caught my eye and appear worthy of of a little taste of Insolence, either Respectful or not-so-Respectful, so much so that I couldn’t decide. None of them really stuck out. Then I saw this news story pop up in my Google Alerts, Houston-area doctor grateful for time spent with Pope John Paul II:

My first thought was that Stanislaw Burzynski must have one hell of a publicist. Either that, or he has a good buddy at KHOU in Houston. My second thought was of a bill that was recently introduced into the House of Representatives by Rep. Morgan Griffith (R-VA), known as the Compassionate Freedom of Choice Act of 2014, because it’s a bill that’s intimately related to Burzynski because basically it would free him from all the hassle of the FDA regulating his administration of antineoplastons to terminally ill patients. Before I get to the bill (HR 4475), I note that, not being from Houston, I had never heard of Jacqueline Crea, the reporter who did the story. I mean, I know that local news often has to do local human interest stories that are tied into big news events like the canonization of Pope John Paul II, to give them the “local angle,” but holy hell, KHOU was scraping the bottom of the barrel on this one! I know that Burzynski met the Pope back in the 1990s, and there were rumors that he was being treated for colon cancer and wanted Burzynski’s input. Those rumors turned out not to be true. Other rumors were that Burzynski had been summoned to treat a prominent Cardinal, but that’s probably not true, either. More likely, the Pope just liked Burzynski because he was (and remains) a prominent Polish Catholic, just like John Paul II.

In any case, surely there must have been someone else in the Houston area who had an audience with Pope John Paul II. It’s not as though such audiences are “rare” (JP had lots of them). In any case, if Burzynski has a publicist who pitched this story, that’s one awesome publicist. If it’s a KHOU producer who happens to be a Burzynski supporter, for shame.

Then I saw this from our old friend, Stanislaw Burzynski’s propagandist Eric Merola, Laura Hymas loses her battle with cancer; An Antineoplaston Phase 2 study is now published in the peer-reviewed literature (April 23, 2014). You’ll remember that I noted Laura Hymas’s death last week. Apparently it’s finally hit the media in the UK in the form of a story in Kent Online, where we find out that Hymas’s cancer had actually recurred in November, a mere three weeks after her fairy tale wedding to Ben Hymas after they had thought that her cancer was gone for good. Hymas, unfortunately, is a woman who was extensively used by Burzynski and his allies as a “success” story “proving” that he could cure brainstem cancers. Sadly, she turned out not to be. Not surprisingly, Merola is going to blame the FDA for Laura’s death, because it placed a partial clinical hold on Burzynski’s clinical trials due to the death of a child (Josia Cotto) in 2012 and numerous violations of FDA regulations with respect to clinical trials found in an inspection in 2013 and detailed in an FDA warning letter in December.

From the newspaper article:

Ben, 34, said: “When we found out the cancer had come back we asked for the same treatment [Antineoplastons] but Laura was denied it [by the FDA]. We were in contact with her doctor in America [Burzynski] and he applied for the legal right to treat her again but this was declined [by the FDA]. We could have fought it legally but we just didn’t have time.”

And from Merola:

It’s impossible to know if Laura would have survived her cancer a second time, if she were allowed to have access to these medications again. It certainly doesn’t change the fact that denying cancer patients the right to choose their own therapy when no other therapy has been proven safe or effective—is an act of violence upon a cancer patient.

That’s going to be the spin placed on poor Laura’s death by Burzynski and his allies. Poor Laura could have been saved if the nasty FDA hadn’t prevented her from getting her antineoplastons. Look for it: The concept that there are all sorts of “cures” out there but that the FDA is keeping people from using them. It’s coming up next. (Oh, and Burzynski met the Pope who will become a saint; so he must be good.)

All of which brings me back to yet another manifestation of the pernicious influence that is the “health freedom” movement, or, as I like to call it, the freedom of quacks to prey on desperate individuals unhindered by pesky things like consumer protection laws. (I need to think of a catchier phrase to boil that concept down to a term as pithy as “health freedom.”) In any case, this brings us back to the Compassionate Freedom of Choice Act of 2014, a bill championed by the Alliance for Natural Health USA, a “health freedom” group that has yet to see a pro-quackery bill it doesn’t like and that has defended Burzynski vigorously before, as well as attacked any attempt to regulate “alternative” therapies.

In any case, as the time I originally wrote about this bill, HR 4475, its text hadn’t yet been published to the Congressional website. The bill has been floating around for a while in various forms (for instance, former Representative Ron Paul (R-TX) apparently introduced it.) Now it is, as Guy Chapman has noted. He calls it a “quack’s charter,” and he’s only off by a bit. The bill doesn’t go quite as far as he believes, but the bill is still plenty bad, man. If enacted, HR 4475 would amend the Food, Drug, and Cosmetic Act by inserting after section 561 (21 U.S.C. 360bbb) the text of HR 4475. This section of the Food, Drug, and Cosmetic Act is entitled Expanded Access to Unapproved Therapies and Diagnostics, and it’s the section of the act that regulates exactly that. The interesting thing is that this particular section of existing law is the framework under which the whole system of single patient INDs (also known as “compassionate use exemptions”) is already based. As I’ve described before, single patients can receive promising unapproved drugs under what’s known as a single patient IND, which has to be approved by the Institutional Review Board and the FDA and allows single patients to receive unapproved drugs. You can (and many have) argued that the single patient IND process is too cumbersome and restrictive, but HR 4475 seeks to (mostly) nuke this requirement. You’ll see what I mean in a minute.

First, the law states that:

Nothing in this Act or section 351 of the Public Health Service Act prevents or restricts, and the Food and Drug Administration shall not implement or enforce any provision of law preventing or restricting, the manufacture, importation, distribution, or sale of an investigational drug or device intended for use by a terminally ill patient in accordance with subsection (b).

Guy Chapman went a bit beyond what the law would actually permit it wrong in his analysis, I’m afraid. HR 4475 clearly does not authorize the use of quackery like Hoxsey therapy, Gerson therapy, or anything else like that in terminally ill patients, although I suppose it is theoretically possible that advocates could get an IND for one of these quackeries and then carry out a phase I clinical trial, after which the quackery would fall under the definition of an investigational drug or device according to HR 4475. (In this, perhaps the Gonzalez protocol would qualify, given that it’s undergone two clinical trials, the last of which was disastrously negative, but it was a clinical trial.) HR 4475 does, however, basically allow unfettered access by “terminally ill” patients to any “investigational” drug or device, which is defined as a drug or device that “(1) has not yet been approved, licensed, or cleared for commercial distribution under section 505, 510(k), or 515 of this Act or section 351 of the Public Health Service Act, and cannot otherwise be lawfully marketed in the United States; and (2) is or has been the subject of one or more clinical trials.” In other words, it’s very much like one of those “right to try” laws that are such a horrible idea but nonetheless popping up like kudzu in state legislatures.

The difference is that “right to try” laws in states would have little or no effect, because the FDA would still rule supreme over drug approval, and federal law trumps state law. HR 4475 would in essence codify “right to try” in the federal law under which the FDA works, which would do many orders of magnitude more harm to cancer patients and terminally ill patients. Basically, this law would allow any “terminally ill” patient access to any investigational drug with an IND that’s gone through at least phase I trials. I’ve explained before in great detail why that’s such a horrible idea, but it’s worth mentioning again briefly this. Most investigational drugs that make it past phase I trials still end up failing. The chances for doing harm far exceed any chance for benefit. Drugs with seemingly miraculous activity against cancer, like Gleevec, that show up in phase I clinical trials are exceedingly rare. Indeed, HR 4475 says nothing about whether the clinical trials the investigational drug or device being to be used in terminally ill patients has undergone even need to show promise or not. Under HR 4475, a drug that completely failed in phase I clinical trials by showing unacceptable toxicity (for example) could be distributed to a terminally ill patient as long as the patient is told that the drug failed phase I clinical trials.

Next up in HR 4475, there’s this:

(b) Patient Requirements.–In order for an investigational drug
or device to be intended for use in accordance with this subsection,
such drug or device must be intended for use by a patient who has–
“(1) been diagnosed with a terminal illness by a licensed
physician;
“(2) been informed by a licensed physician that no drug or
device that is lawfully marketed in the United States is likely
to cure the illness; and
“(3) executed a written informed consent document that
states–
“(A) the known and potential risks and benefits of
such drug or device; and
“(B) any indications of the illness for which a
drug or device is lawfully marketed, or for which
treatment is otherwise available, in the United States.
“(c) Prohibition on Requiring the Disclosure, Collection, and
Reporting of Certain Information by Food and Drug Administration.–
“(1) In general.–The Commissioner of Food and Drugs may
not require the disclosure, collection, or reporting of–
“(A) any information related to the delivery,
administration, or use of an investigational drug or
device pursuant to this section; or
“(B) any information related to the clinical
outcomes experienced by a terminally ill patient
supplied an investigational drug or device pursuant to
this section.
“(2) Exception.–Nothing in this subsection prevents the
sponsor of a clinical trial from voluntarily disclosing,
collecting, or reporting information to the Food and Drug
Administration.

One notes that “terminal illness” is not defined. I could go all Orac-style snarky on you and point out that life itself is a terminal illness (one that’s sexually transmitted, as well). We all die eventually. On the other hand, there are other diseases that are arguably terminal that can take a long time to kill. Even metastatic breast cancer is now potentially survivable for several years, even longer if it’s an indolent type with a low volume of metastatic tumor. Where do you draw the line in defining “terminal”? Normally, for purposes of laws, “terminal illness” is defined according to a certain estimated prognosis for life expectancy, be it six months, a year, or whatever. Even Abraham’s law did that. My guess is that this is to allow the widest number and variety of people with “terminal” illnesses access to unapproved treatments as possible. All you have to do is to find a doctor to say you’re terminal and there are no approved drugs or devices that can save you, and you’re good to go! Really? Any licensed physician, regardless of specialty? Moreover, under these circumstances, as described in this bill, “informed consent” is a joke, a cruel parody of real informed consent. In clinical trials, even in single patient INDs, the informed consent process is lengthy and detailed. In any event, the bill reads as though it were written specifically for Stanislaw Burzynski, as it would allow someone like him to abuse the IND process without even the threat of the FDA bothering him.

Now here’s the kicker. One of the criticisms of “right to try” laws is that drug and device companies won’t want to allow patients to have access to their investigational drugs or devices because adverse outcomes might affect their application for approval to the FDA. HR 4475 has that covered:

(c) Prohibition on Requiring the Disclosure, Collection, and
Reporting of Certain Information by Food and Drug Administration.–
(1) In general.–The Commissioner of Food and Drugs may
not require the disclosure, collection, or reporting of–
(A) any information related to the delivery,
administration, or use of an investigational drug or
device pursuant to this section; or
(B) any information related to the clinical
outcomes experienced by a terminally ill patient
supplied an investigational drug or device pursuant to
this section.
(2) Exception.–Nothing in this subsection prevents the
sponsor of a clinical trial from voluntarily disclosing,
collecting, or reporting information to the Food and Drug
Administration.

In other words, the drug and device companies who allow “terminally ill” patients to use unapproved drugs and devices don’t even have to collect data on the outcomes patients who take their unapproved drugs or use their unapproved devices under this law, and, if they choose to do so, don’t have to report those outcomes to the FDA. It’s all voluntary. It’s hard to imagine a provision more irresponsible than this. Add to the parody of “informed consent” in this law the fact that the law basically makes any reporting of patient response or adverse events completely voluntary. Basically, if you’re a terminally ill patient, this law strips away all the copious protections for human subjects in research that the law currently provides. You’re completely on your own, and the drug company is not responsible for anything, so much so that the law even ends with this provision:

Except in the case of gross negligence or willful misconduct, any
person who manufactures, imports, distributes, prescribes, dispenses,
or administers an investigational drug or device in accordance with
section 561A shall not be liable in any action under Federal or State
law for any loss, damage, or injury arising out of, relating to, or
resulting from–
(1) the design, development, clinical testing and
investigation, manufacturing, labeling, distribution, sale,
purchase, donation, dispensing, prescription, administration,
or use of the drug or device; or
(2) the safety or effectiveness of the drug or device.

In other words, if something goes wrong, the terminally ill patient or his surviving relatives can’t even sue, unless there’s obvious gross negligence or willful misconduct, whatever that is, even if there turns out to be an obvious defect in the design of the drug or device. In other words, terminally ill patients, you’re completely on your own. Masquerading as a “health freedom” law that will let terminally ill patients have access to experimental drugs under a process less difficult than currently existing IND procedures, HR 4475 is a stealth assault on the very heart of clinical trial ethics. Remember, one of the main principles of the Common Rule and other statements of principles regarding human research subject protection is that vulnerable groups need more, not less protection. Those particularly vulnerable groups are traditionally defined as children, prisoners, and students, the former of whom rely on parents to exercise judgment and the latter two of whom are subject to potential coercion. I would argue that terminally ill patients are a particularly vulnerable population in that they are desperate and prone to grasp at anything represented as having a chance of saving their lives. If anything, they need more, not less, protection.

Basically, HR 4475 is a solution in search of a problem. It’s based on the assumption that there are just oodles and oodles of new investigational drugs out there that could save the lives of lots and lots of terminally ill patients, if only the government would get out of the way and let the people have access to them. This is libertarian magical thinking at its most pernicious; there’s no compelling evidence for this assumption and plenty of evidence against it. (And, no, my characterization of this assumption underlying HR 4475 is not a straw man.) Even if you view the IND process as being in dire need of liberalization, it’s still a “solution” that won’t fix the process but will, in essence, destroy the process. It’s also clearly one of a number of attempts by the health freedom movement to get a “foot in the door.” If successful, then similar arguments will be made to expand the “right to try” principle and decrease regulation on investigational agents for larger and larger populations. Although HR 4475 is clearly not explicitly designed to aid quacks who peddle treatments like the Gerson therapy or the Gonzalez protocol, it would be a godsend to someone like Burzynski, and it could potentially be a boon to unethical drug and device companies, who would be free to peddle their investigational drugs and devices, both promising and even those that failed, to that most vulnerable of patient populations, patients with no hope of survival. Health freedom, indeed. Apparently “health freedom” means the freedom of pharmaceutical companies to provide treatments whose efficacy is unknown and that are unlikely to make a significant difference in outcome (or even might be toxic) without actually having to learn anything from the exercise.