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July 25, 2007

Dying so that others may live

A major reason that the FDA exists is that people are concerned that if medicine is unregulated then people will take medicine that is dangerous or unproven. unfortunately, there is a class of people for whom contemporary licensed medicine is insufficient, it is a death sentance. So they are desperate to get access to the clinical trials which for now are pretty much the only mechanism of access to new drugs. But a bunch of people, mostly the dying and their families, who are fighting to try to get access to these drugs after they've been rejected from these trials. They call themselves the Abigail Alliance for Better Access to Developmental Drugs, and they are hard at work suing the government to change the policy. My understanding is that they seek "terminally ill, mentally competent adult patients have a due process right to informed access to potential lifesaving investigational new drugs determined by the Food and Drug Administration to be sufficiently safe for expanding human trials, where there are no alternative government-approved treatment options."

In the great reason article I read about this subject, Dying for Lifesaving Drugs, I learned that the situation is a bit more complicated. Currently, despite improvements in statistical techniques and a streamlined regulatory process, the best and clearest way to demonstrate the efficacy of a new drug is a double blind trial in which half of a specially selected group of participants are given a placebo and the other half the new drug. But in the sorts of diseases that kill, which victimize the very sorts of folks for whom the Abigail Alliance is fighting it is hard to attract people into these studies. People know that they may receive no help if they participate. So if there is a clear mechanism for getting the drug outside the trial it may be impossible to run these studies. That would have serious reputations in drug development.
Reason says the following about this:

There is a serious argument to be made that the entire clinical trial system is antiquated, that it is time to stop counting piles of bodies and to start using more sophisticated measures of drug efficacy. Biomarkers, substances whose presence in the body indicates a particular disease state, could provide objective evidence on how a drug is working on a particular patient. As such measures improve, the need for placebos may lessen. "Clinical trials are very cumbersome," says Thomas Garvey, a gastroenterologist and former FDA supervisor who has designed thousands of clinical trials. "Although they are not the be all and end all. The science is evolving and getting better."

A March 2005 editorial in The Wall Street Journal advocated scrapping placebo trials for cancer patients altogether, giving everyone access to the drugs, and using advanced statistical methods to measure patient progress versus the typical survival rate for a particular cancer. This is the kind of change the Abigail Alliance hopes for, but more incremental changes are already in play.

In an attempt to increase flexibility and reduce approval times, some pharmaceutical companies have begun to conduct what are known as "adaptive" trials. Standard trials are blinded: The findings remain secret from researchers until each phase of the trial is over, and they are conducted on general populations of patients with similar conditions. Even after a standard trial has ended, it can be difficult to know which patients within a population will respond most positively or suffer the most severe side effects from a particular drug.

Alternatively, adaptive trials allow researchers to analyze and respond to data as it comes in, personalizing treatments and assessing how patients with particular characteristics respond to particular dosages. Researchers can tweak the trial design as they move forward, perhaps dropping a method of treatment that proves unpromising or adding more of one type of patient that seems to be responding well. Trial flexibility may prompt shorter approval times and allow companies to sort good drugs from bad more efficiently. "It's a slightly less restrictive straitjacket," says Walker.

But the drug companies have every incentive to try to use these new statistical techniques alongside old ones to justify the safety and efficacy of their new drugs. So I must conclude that while they may be better than nothing, these alternative techniques are worse that the status-quo.

A solution which doesn't come up in the article is to give the right to the patients to take the drug but not a unlimited obligation of the pharmaceutical companies to provide it. That is, if no other treatment works and you are dying, then the drug company can sell you the drug if they want to under a regulatory environment that prevents the treatment from use as evidence for or against the drug and covers the drug companies from liability.

Sure I'd like to do away with the FDA, but maybe the above incremental reform would be a big step in the direction of freedom.

Reason points out that if the courts recognize this right of medical access, there will be serious implications:

If terminal cancer patients do have a constitutional right to lifesaving drugs, the limits of that right are hard to discern. What does it mean to be "terminally ill," after all, and where is the line between a lifesaving drug and a life-prolonging one? Could a suicidal cancer patient claim she has a constitutional right to marijuana in order to ease her pain? Does it make any sense to say a fundamental right hinges on the FDA's determination that a drug has passed Phase I testing? And if not, what does that say about the Controlled Substances Act, the Supreme Court's recent rulings on medical marijuana, and the FDA's role as medical gatekeeper?

Although the Abigail Alliance has chosen not to assert a broader right of individual autonomy, the narrow right it claims seems to imply profound change to U.S. law. "Wouldn't the right you're asserting here also apply, then, to the right to therapeutic cloning, or to organ purchase?" Judge Brett Kavanaugh asked a few minutes into Ballenger's oral argument. "Don't we have to take into account the repercussions of what you're asking for here?"

This is a slippery slope, and it alarms supporters of the status quo. Once you argue that the government has no authority to deny Jennifer McNeillie access to cancer drugs, that she has a constitutional right to accept a certain level of risk, it becomes difficult to know where the agency's authority stops and her autonomy begins.

Posted by OneEyedMan at July 25, 2007 10:14 AM

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