Can States Overturn the FDA’s Limits on Compassionate Use?

At National Review Online, Jillian Kay Melchior, reports on a cancer patient who has run out of therapies. There are medicines under development that might help, but drug-makers won’t let her have them under the FDA’s “compassionate use” doctrine.

ClVariety of Medicine in Pill Bottlesinical trials accept only “typical” patients — Mikaela’s rare form of kidney cancer has ruled her out, but for others in her situation, complications as common as diabetes or high blood pressure could also be reason for ineligibility. And the Food and Drug Administration’s approval process for “compassionate use” of an experimental drug outside of clinical trials is extremely arduous.

First, a pharmaceutical company has to be willing to provide the experimental drug — a high-risk proposition, given that an atypical result in an atypical patient can prompt the FDA to delay approval or require significant and expensive additional testing…And approval from a pharmaceutical company is just the first step. Next, patients and doctors must fill out an exhaustive paperwork disclosure to submit to the FDA, which is estimated to take at least 100 hours to complete. The FDA then begins a review, which is supposed to take no longer than a month — but if the agency’s reviewers have any additional questions or need more information, that 30-day clock is reset. Finally, the hospital’s institutional review board has to give approval — and if a patient is being treated at a small hospital that lacks such an administrative panel, the case has to be reviewed by a bigger facility, often one unfamiliar with the patient.

The FDA bottleneck has been a frequent topic of this blog (e.g. here). Congress has never come close to fixing this problem. So, the Goldwater Institute has developed a new approach: State-based “right to try” laws:

In the past week, the state houses in Louisiana and Colorado both unanimously passed Right to Try bills. In Missouri, the bill has won unanimous support in both the House and a Senate subcommittee. Another bill won approval in the Arizona house recently, and the state senate is expected to vote on the legislation in the next few weeks. “You shouldn’t be putting a bureaucrat in the middle of medical decisions, especially when you’re talking about terminal patients,” says Christiana Corieri, a health-care policy analyst for the Goldwater Institute, which helped draft the legislation. “There’s no right that could be more fundamental than the right to try and save your own life.”

Will these laws hold up? There’s nothing the Goldwater Institute relishes more than a constitutional challenge.

Comments (15)

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  1. James L says:

    As in everything, there are pros and cons to the FDA. It is true the agency has made its bureaucracy to meddle with their mandate, thus they delay approval of drugs that would significantly improve the average health of Americans, something that was discussed in this blog recently (http://healthblog.ncpa.org/i-bet-you-didnt-know-the-fda-regulates-sunscreens/). The good thing about the FDA is that it lowers costs. It will be more expensive for every state to approve a drug, under different mechanisms, than it is for the FDA to do so. I don’t know if the pros outweigh the cons, but still, there are clear indications that this agency must be reformed.

    • John R. Graham says:

      I’m not sure anyone has suggested that states grant approval to drugs like the FDA does. It would be very challenging, as there is so much mail-order pharmacy, and pharmaceutical manufacturers are global concerns, that they would not receive such a proposal well.

      However, if such a reform did happen, I expect states would collaborate and outsource to commercial labs.

  2. Thomas says:

    I am curious as to what constitutes as a “typical” patient to be receiving this drug. If the experimental drug in question is used to treat forms of cancer, is there a typical form of cancer it is to be used for?

    • Matthew says:

      I would assume they are just trying to limit as much liability as possible.

    • Fernando G says:

      Maybe it constitutes healthy patients that are willing to risk their lives for a few extra dollars, not those individuals that actually needs the drug. We have to remember that most of these cancer related drugs don’t actually cure the cancer; they just delay its effects.

      • Thomas says:

        And they also kill cells as a way to kill cancer cells. I would hope that healthy patients do not take forms of cancer medication that can cure rare forms of kidney cancer for financial gain.

      • Jay says:

        We should just leave it to the rats as the test dummies.

  3. James M. says:

    “There’s no right that could be more fundamental than the right to try and save your own life”

    I agree with this sentiment. But I also see the side of the pharmaceutical company’s hesitance for allowing the drug to be used in therapies when it is still in the experimentation process.

  4. Buddy says:

    “You shouldn’t be putting a bureaucrat in the middle of medical decisions, especially when you’re talking about terminal patients,”

    Bureaucrats have no place to be in the medical decision making process. Leave it to the patient and medical professionals

  5. Esteban E says:

    I believe that allowing states to decide individually decide which drugs to use will create more problems than what they will solve. I would believe that if a pharmaceutical company located in X state came up with a new drug; there will be strong incentives for the state to approve the drug, even if the studies are not optimal.

    • Christina says:

      Under Right to Try, states are not approving any medications. The investigational medications become available only after they have successfully completed Phase I of an FDA approved trial. Moreover, the medications must continue to be part of an on-going trial.

  6. Sue says:

    I hope these laws hold up! It’s hard to understand how we have a terminally ill patient and a potential cure, and the FDA just stands in the way.