For almost 40 years, terminal patients have been trying to obtain access to unapproved new drugs. The latest effort has come from the Goldwater Institute (“GI”) which has made the strategic decision to bypass Congress and the federal courts and go directly to the state legislatures. GI’s legislative efforts have been wildly successful, having passed its “right to try” law in more than half of the states. That’s an almost unbelievable accomplishment and everyone should laud and support it, but it won’t do much, practically, until Congress is forced to act, and act in a big way. Before I go into why the state legislative angle won’t do the job, let’s review how we got to where we aren’t, namely open access to pipeline (and other) drugs for very sick people who can’t be helped by FDA approved drugs.
The history is instructive.
It started with laetrile in the 1970’s when terminal cancer patients sued the FDA for access to the apricot pit based cancer remedy. A compassionate and brave Oklahoma federal district court judge held in part that the FDA’s attempt to keep laetrile from terminal patients violated their constitutional privacy rights. https://www.courtlistener.com/opinion/1554980/rutherford-v-united-states/.
The court of appeals affirmed the result, but on a different ground, holding that as a matter of statutory interpretation, the FDA’s safety and efficacy requirements do not apply to terminally ill patients. The appellate court created this non application of safety and efficacy requirements because it wanted to give the patients laetrile but like most federal appellate courts, it wanted to reach the result without making a constitutional ruling. So the court decided that the statute didn’t apply to terminal patients.
In 1979, the liberal Supreme Court unanimously reversed the court of appeals. Ironically, Thurgood Marshall, – the man who argued Brown v Board of Education (“separate is not equal”) as a lawyer – wrote the opinion that there was no statutory exception to FDA law for terminal patients. Marshall ducked the district court’s right of privacy holding because the court of appeals hadn’t addressed it. The Supreme Court remanded the case to the court of appeals to decide the constitutional privacy issue. http://caselaw.findlaw.com/us-supreme-court/442/544.html. The appeals court decided that terminal patients do not have a right of privacy to take medicine of their choosing.
And so ended the right of privacy for terminal patients…
The right has stayed dead or nonexistent until this day, despite efforts by lawyers (including me) to revive it.
Interestingly, even though the federal government denied dying patients’ access to laetrile, many states stepped-up to the plate and passed laetrile access laws. Technically, these laws were in conflict with federal law, in the same way as the state laws legalizing marijuana are in violation of federal law. However, then as now, the feds basically let people have access in the states in which it is legal, or at least the feds didn’t go after the patients. The state laetrile laws in the 70’s may have been an inspiration to the right to try movement, but ultimately it is a false analogy, as I will explain later.
Access to unapproved drug was raised indirectly in Texas in the Burzynski cases in the 80’s and 90’s. In the early 80’s, a Texas judge held that although Burzynski couldn’t ship his unapproved drugs across state lines, he could provide them to patients in Texas since it appeared to be legal there. In response, the Texas legislature closed the loophole by passing a “mini-FDA act” which made the treatment illegal in Texas.
The issue came up again in Burzynski’s criminal case in the mid 1990’s when a Texas district court held that under Burzynski’s conditions of bail, he was not permitted to give his unapproved new drug to patients. We ended up going to Congress for help. Congressman Joe Barton and some of the Burzynski patients forced the FDA to back down, and the FDA provided a legal way for Burzynski’s patients to get the unapproved drug. (The FDA put all of his patients on a clinical trial protocol. See chapter 2 of my book, Galileo’s Lawyer for a more detailed discussion. http://www.amazon.com/Galileos-Lawyer-Alternative-Complementary-Experimental/dp/0980118301.
The book’s discussion about how that took place and my characterization of this one clinical trial protocol as a “joke” has become an oft repeated blog and media topic, much to my dismay. But more about another time.). The Burzynski access issue relating to his bail conditions also prompted congressional hearings on access in general, but nothing came of it.
The Thomas Navarro house bill
In the late 1990’s, and early 2000’s, there were other efforts to legislatively open up access to unapproved drugs. One was the Thomas Navarro house bill submitted by former Congressman Dan Burton. Thomas was a young terminal brain tumor patient who was initially refused access to Burzynski’s treatment. Thomas and his right to get treatment was discussed in a 2000 Republican presidential debate. However the bill went nowhere.
In the 2000’s there was another serious litigation effort in the form of the Abigail Alliance case based on a heart-wrenching story like Thomas Navarro’s. Like the laetrile case, a brave district court judge opened-up access to unapproved drugs on a constitutional basis. However, the DC circuit court overturned the lower court, holding that only the all-knowing FDA has the right to determine what drugs dying patient can take.
Patient access to investigational drugs hasn’t gone completely unnoticed by the FDA.
Because of the AIDS epidemic, since 1987, there has been some form of expanded access, i.e., access to investigational drugs beyond enrollment in clinical trials. Originally called “compassionate use” or “special exception” access. More recently expanded access has, well expanded somewhat, and is now called Single Patient Protocols (SPP). There are other larger programs as well (e.g. Treatment INDs for bigger groups). The FDA has even recently streamlined the SPP approval process with the introduction of a two page form instead of a formal IND. (more about that another time). However the FDA’s program is still woefully inadequate. Less than two thousand patients a year are treated under SPP’s, which is a miniscule amount compared to the amount of patients who could benefit from access to investigational drugs.
Which leads us back to the Goldwater Institute’s right to try state legislative efforts.
I think it’s now law in 28 states. Although there are some differences in the laws, the basic state law provides that medical doctors cannot be charged with a medical board violation for providing seriously ill patients with an investigational drug which has completed phase 1 (safety) clinical trials. Many of the laws purport to make it legal for drug manufacturers to provide the investigational drugs to patients. Some states disallow charging for the drugs, others allow it. Proper informed consent is required.
Of course, the physician and the patient have to obtain the investigational drug from the drug manufacturer, and therein lies the problem. There is and cannot be any way a state can compel a manufacturer of an investigational drug to give or sell it to a patient outside of clinical trials or expanded access. And from there, it just gets worse.
Suppose the drug manufacturer is in a different state from the doc? Can the manufacturer ship an investigations drug across state lines to a physician to administer to a patient in a right to try state? I don’t see how. The right to try law does not and cannot immunize a manufacturer from the federal law violation of shipping an unapproved new drug across state lines. The “law” just doesn’t work that way. While the federal government may not go after Colorado marijuana sellers who sell product to people in Colorado, if any of those sellers start shipping weed out of state, I guaranty the feds would shut them done very quickly.
Similarly, I don’t see how it would be legal for a drug manufacturer to ship an investigational drug across state lines without violating the FDA criminal statutory trifecta (introducing into interstate commerce an unapproved new drug, misbranding and adulteration). What interest would the FDA have in allowing that? None that I can think of. Depending on your perspective, for better or worse, the FDA is intent on controlling the use of investigational drugs and I don’t see it ceding any part of that power to the states.
Furthermore, I don’t see how any competent FDA attorney could advise a drug manufacturer to ship an investigational drug across state lines for use outside of a clinical trial or under formal FDA expanded access.
But even if a manufacturer is in the same state as the administering doctor, based on past precedent (the Regenerative Sciences case discussed in my first stem cell post), dollars-to-donuts the FDA would take the position that if the drug manufacturer buys a test tube or a pencil from an out-of-state source, that’s enough interstate commence for the FDA to assert jurisdiction over the intra state distribution of the investigational drug.
But even without the out-of-state purchase of a pencil, the FDA, backed up by federal jurisprudence, has a jurisdictional predicate of “affecting interstate commerce.” Basically, everything affects interstate commence in some way. So lawyers should be telling drug manufacturers that it is not FDA legal to ship unapproved drugs out of state or even within a state, for anything other than an FDA clinical trial or FDA expanded access. And on this I speak from professional experience.
But there is a bigger problem based on the naked realities of self-interest. There is only one reason a drug manufacturer tests an investigational drug; to get the FDA to approve it for marketing (so the executives can get their bonuses and/or cash-in their stock options after approval).
Why would pharma execs risk the FDA’s wraith to provide their investigational agents outside of FDA sanctioned expanded access programs? To save patients’ lives? Oh please, you’re listening to too many pharma commercials. Pharma hasn’t even provided wide access to investigational drugs under FDA expanded access, so it’s completely unrealistic to think it will do so in technical violation of federal law.
Going back to laetrile:
The state laws legalizing it were effective in allowing access to the drug, but only because the suppliers of laetrile were not pharma beholden to the FDA for drug approval. They were mom-and-pop, cottage industry folks from the herb and supplement field who didn’t give a whit about the FDA or drug approval. Pharma lives and dies in its relationship with the FDA, which is why there is the obscene revolving door between senior FDA regulators and pharma companies.
There is another hard reality.
The right to try target group consists of patients who are likely sicker or much sicker than patients who could get the drug in a clinical trial. Why? Because right to try covers terminal patients, and mostly very sick terminal patients who have tried and failed other treatments. These patients usually have many co-morbidities. Drug companies prefer to test their investigational agents on the healthiest people they can find for the drug’s indication. That’s what the entry criteria in the clinical trial protocol are all about. Why? Healthier people have fewer co-morbidities and will experience fewer side effects which could be attributed to the study drug. Side effects have to be reported or at least recorded.
Although the FDA says that side effects (adverse events “AE’s” or serious adverse events “SAE’s”) for SPP’s and compassionate use do not negatively impact a drug approval decision, drug companies don’t believe it. And they are worried that after approval, evil plaintiffs’ attorneys will use these SAE’s against the company in products liability cases. For these reasons, the drug companies are not and will not buy into the whole bypassing of federal law, state right to try thing.
So what’s the point of right to try?
For starters, it is a direct and impressive manifestation of the will of the people. It is very important for federal legislators to hear what people want, to give them the impetus to reign in the FDA. If all or most states pass right to try, I think Congress will have to act.
What kind of new federal law? Maybe there needs to be a new business model for drug approval. I would propose patient funded access to early stage investigational drugs, as a choice for patients. That might entice some smaller drug companies to open up access.
At its core, the issue is who has the right to decide on treatment for dying patients?
Is it government bureaucrats, talking heads in the cancer establishment, professional ethicists, mentally disturbed bloggers (more about that another time), or the dying patient and his or her family. Guess where I stand?
This is a bigger issue than right to try, or federal expanded access, both of which require that a drug be in a formal clinical trial before it is available to a patient. I go the next step, and re-ask the question I asked in the title. Why can’t dying patients have ANY drug they can get their hands on? I do not think the federal government should be in the business of overseeing or limiting the kinds of drugs or remedies which a terminal patient can take. I think a dying patient should be able to get any drug he/she can get their hands on, and let them weigh the risk and make the decision.
Let’s circle back to the laetrile case.
I think the court of appeals got it right when it said that the FDA’s safety and efficacy requirements should not apply to terminal cancer patients. These patients should be able to make their own decisions and take whatever treatment they want. Those terminal patients who want the full and glorious protection of the federal government and the mainstream cancer establishment can limit their treatments to FDA approved drugs and drugs in clinical trials or expanded access. But what right does the federal government or so-called cancer experts have to limit options for people who have no potentially life-saving options?
Each year almost 600,000 people a year die of cancer. I would venture to say that every one of them had some kind of treatment and died on it, as a result of it, or at the very least, the treatment they received obviously did not prevent their death. And the same is true with other diseases. This is a liberty issue. Either the patient or the government makes these decisions. Right now it’s the government. I think it should be the individual patient.
So the “go big or go home” solution is simple. Remove the FDA’s jurisdiction over terminal patients. That’s what the Tenth Circuit Court of Appeals did in the laetrile case, and 40 years later, it’s still the best solution I’ve ever heard. I think many people would agree.