Right to Try

Congress Could Let All Patients Have the 'Right to Try' Without Going Through the FDA's Complicated Application Process

The FDA is slashing paperwork requirements for expanded access trials in response to state-level "right to try" laws. That's good, but it's not enough.



It is a rare thing to see Congress pass a law that increases personal freedom.

Yet that is exactly what it could do later this fall if the House follows the Senate's lead on a so-called "Right to Try" bill. The law would allow individuals with terminal illnesses, without having to first get permission from the Food and Drug Administration, to try drugs that have not been approved. Since 2012, 37 states have adopted "right to try" laws, giving patients to access experimental treatments that have cleared the first phase of the FDA's trials, with the permission of a doctor.

Passing a law at the federal level would be important for residents of those remaining 13 states, but would also help steer FDA policy, advocates say.

"Right to try is about the terminal patients who don't fit into a control group, who can't afford to travel or move to another country, and who simply want permission to seek the same treatments that other patients—sometimes patients in the same medical facility—are already receiving," says Naomi Lopez Bauman, director of healthcare policy for the Goldwater Institute, an Arizona-based free market think tank that has largely spearheaded the state-level Right to Try movement.

The U.S. Senate in August passed—by unanimous consent—Senate Bill 204, a right to try bill sponsored by Sen. Ron Johnson, R-Wis. That bill, and a similar proposal sponsored by Rep. Morgan Griffith, R-Va., were the subject of a hearing hosted Tuesday by the House Committee on Energy and Commerce. There is no immediate timetable for either bill to receive a vote on the House floor, and Tuesday's hearing made clear that right to try legislation faces more opposition in the lower chamber than it did in the Senate.

"The legislation being proposed could expose critically ill patients to greater harm," worries Rep. Frank Pallone, D-N.J., minority chairman of the committee. Other Democrats expressed similar worries, even while expressing sympathy for patients who are asking little more than for government to get out of the way during the final days of their lives. There are "very legitimate frustrations with the current system," for allowing patients access ot non-FDA-approved drugs, admitted Rep. Gene Green, D-Texas. But those problems are not a good reason to remove the FDA from the process, Green said.

Currently, the FDA runs a so-called "expanded access" program for terminally ill patients who cannot get into drug trials for various reasons. According to a Government Accountability Office report published in July, FDA had approved 99 percent of the 5,800 requests made from 2012 through 2015 by patients seeking access to the program.

Lack of access, then, is not the problem, but time is. Patients with terminal illnesses can wait as little as a few hours to as long as 30 days for the FDA to respond to a request to try a new drug, according to the GAO, and that wait could ending any slim hope of finding a successful treatment. If you think dealing with bureaucrats is awful when you're standing in line at the DMV or applying for a passport, imagine having to go through that same process when your life is on the line.

Under Scott Gottlieb, the newly appointed commissioner of the FDA, the administration has moved to slash the amount of paperwork necessary to get patients enrolled in expanded access programs. Earlier Tuesday, Gottlieb announced further reforms to streamline the experimental treatment review process for patients and doctors. The FDA "believes difficult decisions about individual treatment are best made by patients with the support and guidance of their treating physicians," Gottlieb told the committee Tuesday.

Those welcome changes do not accomplish as much as a federal right to try law, in part because the FDA's statistics are something of an illusion. Gottlieb says the FDA accepts 99 percent of all applicants, ignoring how many patients don't bother going through the process in the first place.

About 500,000 Americans die of cancer each year, but the FDA receives only 1,200 applications from all terminally ill patients for its expanded access programs. The gap suggests a large number of patients who aren't asking for access to experimental drugs because they either don't know about the FDA's programs or don't care to navigate the system.

If nothing else, the recent groundswell of support for state-level right to try laws suggests the status quo isn't working for many patients.

Advocates for right to try also point out that the FDA has only gotten its act together because of pressure applied by the widespread adoption of right to try laws in the states. It wasn't until last year that the FDA announced it was creating a web portal to help guide patients through the expanded access application process. The website is only now being rolled out for public use.

"Shorter forms and hand-holding bureaucrats don't fix the system's fundamental flaw," Christina Sandefur, vice president of the Goldwater Institute, told Reason via email in response to Gottlieb's comments Tuesday. "It requires dying patients who have exhausted all government-approved options to beg the government for permission to obtain treatment to save their lives."

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  1. If polio and smallpox still existed, and a vaccine was invented today, tens of millions would die before it was approved.

    1. And some very common drugs would never be approved.

    2. Eh?

      Some of the early polio vaccine trials involved over 50k test subjects. Even with those massive sample sizes, some of the polio vaccines still took five years to achieve approval.

      BTW, polio still exists.

      And tens of millions still died of smallpox even after vaccines were approved.

  2. How much is a pharma company allowed to charge for these drugs?

    What happens when pharma companies inevitably exploit this “loophole” for unapproved oncology drugs?

    Will the patients who die while on these drugs be recorded as adverse events and reported to the FDA during the review process?

    How will that affect the approval of the drug?

    1. Good questions, I’m curious whether the “right to try” is for drugs that intended (but not approved) for treating a particular disease state or whether a doctor can prescribe drugs that not only aren’t approved but also aren’t being tested for treating a disease state. And what the obligations and liabilities of the manufacturer will be under those circumstances.

      1. My experience is that pharma companies don’t charge for experimental unapproved drugs and one waives any claims against the providing company in return. But that it within the FDA single experimental use system, which precludes companies from charging anything.

        1. This is not a sustainable idea.

          It is incredibly expensive to conduct clinical trials, and this only makes it significantly more expensive because now clinical trials will take substantially longer (since more patients will demand to be in the intervention group and just about no one will agree to be in a control group), more workers will be required to conduct the trial, and more of the product will have to be manufactured.

          Someone will have to pay for it.

      2. Usually if a product isn’t even being tested for medical use, the authorities wouldn’t be involved at all. For example, say you wanted to try sawdust or Ajax or Dutch Boy white latex paint for a disease; you’d simply obtain sawdust or Ajax or Dutch Boy white latex, not even saying it was for medical use. Of course someone would need to compund it into suitable form, but that could be done by a compounding pharmacy. All legal under both state pharmacy laws & FFDCA, because the purveyor did not intend the product for medical use.

  3. If toothbrushes were invented today, they would be safety-studied to death…

    … and 20 years later, they be finally approved, but would require a prescription from your dentist!!!!

    1. Are you sure they’d be approved? “We find that any purported benefits of the so-called ‘toothbrush’ are heavily outweighed by their mechanical and bacterial precipitations of periodontal disease.”

      1. Are you sure they’d be approved?

        It would depend on whether there’s any powerful interest groups lobbying to keep them banned. Like “Big Mouthwash” for example.

  4. The FDA gets its power from the commerce clause and economic terror. You can currently use any drug produced in your own state without FDA approval if you can find an in-state drug manufacturer, hospital and physician who isnt terririfed of the FDA. You won’t be able to find any of the three. Without federal legislation forcing the FDA to allow interstate transportation of medicines to patients who want to use unapproved drugs, state right-to-try laws will accomplish nothing. It is dubious if even federal legislation will do much since the FDA can still retaliate against pharma companies that acomodate patient requests outside of the single use experimental straight-jacket the FDA currently enforces.

    1. You have a common misconception there. Check your state’s pharmacy laws; they’re all boilerplate. The states require drugs to be licensed by their state if they’re not sold legally in interstate commerce. Usually you’d gain nothing by seeking state approval vs. FDA’s.

      1. Yeah, and the Supreme Court pretty much tossed that entire concept on the trash heap with Wickard and Raich. I think the decisions read “we’ll do what we damn well please, and you can suck it.”. I may be paraphrasing a bit though.

  5. Unfortunately, the right to try is also the right to get scammed. There are plenty of out-and-out scams operating under the label of clinical trials already. It is too bad, but there are a lot of people with no ethics who will Prey Upon desperate people.

    We really need a better method of communicating the nature of these scams. Currently, we rely on websites like sciencebasedmedicine.org. Not exactly an efficient method. Particularly when the scam artists operate networks of alternative medicine websites dedicated to following people into lucrative scams.

      1. Burzynski’s no scammer. There’s just a difference of opinion between him & others over his treatments.

        1. Not an honest difference of opinion. Click through and read the articles about his “trials”. It has been over 30 years. A real set of trials would have some evidence supporting them by now.

          So he may truly believe in his treatments. Or he may cynically be scamming folk. It really shouldn’t matter what he believes. That is why you need a disinterested 3rd party to help evaluate such claims.

          There are plenty of true believers in things that provably don’t work, like reiki, homeopathy, etc. Belief doesn’t make it less of a scam.

    1. “Freedom is the right to make choices, and you might make bad ones in the eyes of your owners, so no freedom for you.”

      Fuck off, slaver.

      1. Go read the article I linked, and reread my post.

        I never said nobody had the right to make their own choices in life.

        There are people who already use the “research trial” workaround for FDA regulation in order to commit fraud on desperate people. Massive fraud. The people in the linked article get together huge fundraisers to put together hundreds of thousands of dollars to pay for their bogus treatments.

        Because we have the FDA, no other entity exists that could give good, reliable information about the potential validity of a trial. Because the FDA is a political entity, it has requirements laid upon it that do not have anything to do with science, medicine or protecting the health and safety of the public. Like the requirement that “alternative medicine” like homeopathy and herbal supplements be given exceptions.

        If there was an Underwriters Laboratory for medicine, there is no way in hell that Airborn would get their seal of approval.

        But because of the incentives involved, institutional review boards will give scammy clinical trials a pass, because it brings in millions. And there is no entity other than the federal government that can really stand against it, because of the financial incentives involved.

        But if consumers insisted on a UL rating on medicine, maybe we’d get better information.

        1. And before you go around chanting some mantra like “Fuck off Slaver!” go take a look here and see “what’s the harm?”

          It is a list compiled by a small group of skeptics based on published reports. It details hundreds of thousands killed by medical scams (otherwise known as Alternative Medicine).

          Be sure, the true scope of the harm dwarfs that which can be documented from published reports.

          This isn’t some fantastical, hypothetical potential side effect of eliminating the FDA. This is what is already happening today, even with tight FDA control over medicine. People are making millions of dollars feeding false hope to sick people, resulting in many thousands of deaths that could be avoided by proper medical treatment.

          People in general are sorely lacking in the knowledge and training needed to evaluate medical claims. And the people who are in most desperate need are paradoxically the least able to make rational decisions.

          The feds are currently letting them down, because they are a political entity and have had their hands tied in certain areas, like Chiropractic.

          A private entity would have no such limitations. They would call it straight, or they would lose the trust of the public.

  6. So many things wrong with this idea.

    First, this will kick off an expanded industry of fraudsters, “researchers” who will peddle hope to desperate people, and these researchers will have no burden to show that there is any efficacy.

    Second, this will not only impair clinical research data collection, it will open the door to selective inclusion of data. If there happens to be some benefit to those people who receive the experimental therapy outside the clinical trial protocol, researchers will happily capitalize on the information. If it does not work, you’ll never hear a word about it.

    Third, it will dismantle a fundamental element of clinical research which is the double-blind model. What I have found working in clinical research is that no one wants to be in the control group. If we say everyone has the ‘right to try,’ then the double-blind approach will be thrown to the side and objective data collection becomes impossible. Every patient can demand to be in the intervention group and no one can be put in the control group because we can’t say that just some people have the ‘right,’ can we?

    This legislation will lead to one more step away from the scientific method and the invite fraud of massive proportions which will be impossible to address. I’m not saying that the current clinical research process is perfect, but this sets it back to the time of snake oil being sold out of the back of wagons.

    1. I’m not saying that the current clinical research process is perfect…

      The current process = terminal patients fucking die and medicine can’t help them. By definition of the category. Just so we’re clear what option you would presume to restrict them to.

      1. Just so you are clear, what you are advocating for is more of this: 100k people harmed by naturopathic vitamine megadose treatments..

        This isn’t a zero harm game. Medical scams are a real thing, and they kill people.

        There is a reason we use “snake oil salesman” as a pejorative. That was a real thing once.

        We have forgotten because we abdicated our responsibility to the government, instead of finding private alternatives for vetting medical treatments. As a result, medicine is less scientific than it would otherwise be, and the information we get it more limited and flawed.

        1. I don’t need to hear little lectures about the risks of freedom to clarify my opinion on anything. Nor have scams remotely disappeared. Google your favorite disease and you will see plenty of real snake-oil salesmen selling “cures”. That infomercial asshole who keeps getting busted for example. Those people disgust me as well. But again, these people are terminal. Who are you to tell them to just lay down and die quietly to protect our “system” which can’t hope to approve something to help them in time? (or is it just to protect yourself from the frustration of seeing others scammed?).

          Meanwhile, on the larger issue, including non-terminally-ill people, do you accept that private firms may not actually be scammers but may actually seek to make money by legitimately employing the best methods possible and the best expertise in the world to help people? And (I know this is crazy) may actually make progress at it?

          Also perhaps I should remind both of you that a majority of states already allow this as noted in the article. It isn’t the wild west. These are often famous doctors trying radical (and desperate) new treatments that make very good sense.

        2. Also, I don’t like these historic references to the “evils” of unregulated capitalist systems before we had modern regulations etc, which also happens to be a picture drawn from a time which lacked modern science and technology. When snake-oil “was a real thing”, the legit doctors were prescribing leeches or directly draining blood from people. And delivering babies after handling cadavers and not washing their hands. Also please remember there is a hidden cost to regulations. New advances and cures we don’t have today because of inefficiency in the market caused by govt. Your 100k number is just one point on a trade-off somewhere.

          1. “Snake oil” got that reputation because there were scammers peddling fake snake oil. Real snake oil may not have had benefits, but at least it was offered in good faith.

          2. But it isn’t about regulations – at least not as performed by government.

            The same role could be served much better by private organizations. It is much more difficult to get started, but if everyone had an Underwriter’s Laboratory they could trust for this sort of information, we’d be much better off. We’d have much better information about doctors, hospitals, pricing, and drug performance.

            Ever tried to figure out if one cold medicine was better than another? Is one antacid better than the next? That sort of data is very rare in the public discourse. But someone other than the FDA could fill that role.

            The problem is that it is much easier for the government to create an agency than it is for consumers to band together and demand a trusted seal of approval.

    2. There’s really only 1 problem w blind testing: It’s unethical. I’m just passing along what I’ve been told by those who’ve studied it, & I don’t have anything to contradict them.

      To test something ethically, you have to have some reason to think it more likely than not that it will be helpful rather than harmful. But then it would be unethical for you to withhold that which you believe more likely to be helpful. So either you’re doing something unethical to both groups of subjects, or you’re doing something unethical to the control group.

      1. Clinical trials are ethical only according to the fiction that you have 2 choices, & no advance opinion on whether either is better than the other. It’s rare that that’s true. Even then, by keeping yourself in the dark as to which choice seems better until the trial is over, even an initial ethical choice becomes unethical as results come in.

        1. There is an ethical dilemma, but failure to do proper blinding can invalidate the results of the test. Plenty of earnest and well-intentioned folks have fooled themselves under these conditions.

          For a classic example, see N-rays.

          Or take a look at the work that James Randi (the magician) did in helping researchers into psychic phenomena understand the flaws in their testing methodologies regarding blinding.

          So if you want a valid test, you have to keep everyone involved in the dark as to which test group they are in.

          That being said, there have been cases where there seemed to be dramatic results (both positive and negative) and tests were unblinded early.

  7. Fundamental Flaw – we are not free.

    Free to buy the medicines and medical services we want from whom we want to buy them from.

  8. Do you want superpowered mutates?

    Because that’s how you get superpowered mutates.

  9. Congress Could Let All Patients Have the ‘Right to Try’ Without Going Through the FDA’s Complicated Application Process – Hit & Run : Reason.comis the best post by imo for pc Please visit imo app imo app snaptube for pc snaptube app

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