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Sitting at your favorite waterfront restaurant, your meal is disturbed by the sounds of frantic splashing and cries for help. Someone is drowning. Running down the pier, you grab a large metal canister—the first thing at hand—but just as you're about to throw it to the drowning person, you stop. Will the canister float or sink? Will it save the person or drag him to the bottom? Should you wait for someone else to show up with a proven flotation device?
This is essentially the dilemma that faced the U.S. Court of Appeals for the District of Columbia Circuit recently as it considered the question of whether terminally ill patients had the right to access potentially lifesaving but still experimental drugs. Their decision, overturning an earlier ruling by the same court, was no.
The decision arose from a lawsuit launched in 2003 by the Abigail Alliance for Better Access to Developmental Drugs and the Washington Legal Foundation, which sued the FDA. The alliance was founded in 2001 by Frank Burroughs, who had fought to gain access to one of two investigational new cancer drugs for his daughter. Abigail died later that year after making it into a trial of a third drug. The two cancer drugs in question—Erbitux and Iressa—were eventually approved.
"Although terminally ill patients desperately need curative treatments…their deaths can certainly be hastened by the use of a potentially toxic drug with no proven therapeutic benefit," the judges wrote. "Thus, we must conclude that, prior to distribution of a drug outside of controlled studies, the Government has a rational basis for ensuring that there is a scientifically and medically acceptable level of knowledge about the risks and benefits of such a drug."
The court acknowledged that there are mechanisms in place for seriously and terminally ill patients to gain access to experimental drugs, including the FDA's treatment IND program. Despite the Abigail Alliance's contention that such access should be a right under considerations of "right to life" and "right of self-defense", however, the court decided that the FDA must be the final arbiter of such decisions.
"We therefore hold that the FDA's policy of limiting access to investigational drugs is rationally related to the legitimate state interest of protecting patients, including the terminally ill, from potentially unsafe drugs with unknown therapeutic effects," the judges continued.
The decision was not unanimous, however, and in a dissenting opinion, two judges wrote: "It is startling that the oft-limited rights to marry, to fornicate, to have children…and to control one's own body even if it results in one's own death or the death of a fetus have all been deemed fundamental rights…but the right to try to save one's life is left out in the cold despite its textual anchor in the right to life. This alone is reason the court should pause about refusing to put the FDA to its proof when it denies terminal patients with no alternative therapy the only option they have left, regardless of whether that option may be a long-shot with high risks."
It's a tough question.
I think anybody can understand the decision of Burroughs and the Alliance to fight this fight. Given similar circumstances, I like to think that I too would fight tooth and nail to gain access to potentially life-saving treatment for a family member or friend. But at the same time, I also appreciate the position in which the FDA finds itself. In periods of duress, we cannot always be expected to make the best decisions, particularly in questions about the health of a loved one, so it is beholden on organizations like the FDA to protect us from ourselves.
It is a gray area that will only get murkier as more companies tackle more human diseases and as more parents and friends become stronger advocates for access to experimental drugs, networking ever more extensively through television, radio, telephone, print, and the Web. Sadly, regardless of which way this battle swings, there will always be those who don't make it, whether through action or inaction.
OUR READERS RESPOND
Proponents of evidence-based medicine have to support the FDA's policies. The FD&C act charged them to protect patients by ensuring that drugs were SAFE and EFFECTIVE. The law doesn't say "somewhat" or "maybe" effective.
There have been few really new drugs over the last two decades, most are just cheap knock-offs of what is already available, even in the oncology area. Look how many EDGF receptor antagonists we already have. Any so-called gains in survival are minimal. Then there is the bigger issue of do these unproven remedies even work.
Remember the demand for peripheral blood stem cell transplants for breast cancer patients? Every women with breast cancer wanted that treatment because it was the best. Turns out, contrary to the internet and proponents, it didn't work. At least that is what the prospective, randomized, controlled trials showed. In addition, it hastened the death of many, and it cost a lot of money. This is just another example of modern day snake oil.
Dr. Alan Hopefl, Senior Clinical Manager
Amerinet, St Louis, MO
It is my perception that the advocates for the patients and their rights to experimental pharma often are not the medical professionals who treat the terminally ill, but the relatives, friends, and loved ones who helplessly watch conventional treatments fail. If true, this is one possible reason for FDA being more cautious than it could be. After all, what could the uneducated relatives of a patient know?
Facetious, but an understandable attitude. Most professionals in a given field are suspicious of the skills of laypeople. I know I am, and it is unlikely that anyone will die if I err. How much more difficult to trust someone who is not a peer, when potentially risking lives?
The argument of the majority of the Court seems to me to be along the lines of the bizarre and incomprehensible rulings that doctors who administer adequate palliative medication to their terminally ill patients are criminals because the patients could become addicted to drugs. Where is our compassion? Where are our brains? Does anyone (excluding from the question you and perhaps 2 Supreme Court judges) use logic anymore?
Last October a friend died of complications from relapsing polychondritis. Do you know of this rare and terrible disorder?
The drug pirfenidone seemed to me to be a potential treatment for the condition. Pirfenidone is very safe and (last I looked) in clinical trials was shown to have positive effects not only on its intended target of lung fibrocytis but also, as serendipitously discovered, on some forms of MS. Given the similarities in symptoms and progression, if not biochemical origin, of MS and relapsing polychondritis, it seemed pirfenidone was worth a try.
Despite many years of effort, the only response I got was from a specialist at Beth Israel Deaconess hospital in Boston: "If you can get someone to try it (in a clinical trial), I'd be interested." Who knows if pirfenidone would work, or not. It's certainly too late for
my friend, whose suffering might have been eased even if her disorder was not cured.
Patients may advocate, friends may advocate, parents may—and do—use their last stores of all resources to try to heal their children, but if the physicians and other medical professionals aren't willing to consider—and demand—access to treatments that might work, those treatments will not be available to those who cannot wait for proof of a drug's safety.
Life is fatal, but people ought to be able to get to the end of it with as much hope—and help—as possible.
Dr. Denise Lynn Merkle, President
SciConsult, Inc., Fort Worth, TX