Whose Life Is It Anyway?

The FDA versus dying cancer patients.

Washington, DC - University of Virginia student Abigail Burroughs died of head and neck cancer at age 21 on June 9, 2001. She died while fighting to gain access to promising experimental anti-cancer drugs recommended by her oncologist at Johns Hopkins University Hospital. Her father, Frank Burroughs, founded the Abigail Alliance for Better Access to Developmental Drugs and sued the Food and Drug Administration, arguing that terminal cancer patients have a constitutional right to try to gain access to developmental medicines that the agency has not yet approved.

In May 2006, the Alliance won its case before the U.S. Court of Appeals for the District of Columbia which ruled that "barring a terminally ill patient from the use of a potentially lifesaving treatment impinges on this right of self-preservation." The Appeals Court sent the case back to District Court to consider if the protected liberty interests of terminally patients outweigh the FDA's interest in insuring the provision of safe and effective drugs. Yesterday, March 1, the full Appeals Court reheard the case at the request of the FDA.

Earlier this week, the Food and Drug Law Institute held a colloquium, "Whose Life Is It Anyway?," on the issue. Scott Ballenger, the lawyer who is representing the Abigail Alliance before the Appeals Court, noted that the legal question before the court is what standard should apply to the case. Is trying to gain access to potentially life-saving medicines unapproved by the FDA a fundamental right or merely an economic right subject to regulations established by political authorities? Can the government properly assert that it has a compelling interest to deny dying patients access to potentially life-saving drugs?

Ballenger compared the situation to self-defense. "Self defense is the most obvious and self-evident rights of men," he asserted. "No state can deny someone self-defense in the face of an attack." Ballenger argued that if the law recognizes that people have the right to defend themselves from attack by a bear or infectious bacteria, then surely they have the right to defend themselves against a rogue cancer cell.

At the colloquium, food and drug lawyer Richard Cooper agreed that the issue is whether some rights are so fundamental that we do not entrust them to decisions made by elected officials. Until recently, establishing agencies to regulate the safety and efficacy of drugs was thought to be within the purview of Congress. "I doubt that most people thought that they had a constitutional right to buy investigational drugs," said Cooper. "It's a wholly new, unheard of right with no antecedents in Anglo-American law." If people want to change the way the FDA regulates patient access to investigational drugs, Cooper argued, they can petition the FDA and Congress and eventually vote out members of Congress who refuse to change the regulations.

Cooper noted that the Supreme Court ruled in 1979 that cancer patients did not have a constitutional right to access the cancer treatment laetrile. The court reasoned that access to the drug might be restricted on the grounds that laetrile had not passed the hurdle of FDA safety testing. However, the Alliance argues that this ruling does not apply because it is asking only for the same right of access enjoyed by those terminally ill patients who are lucky enough to be enrolled in an FDA-approved efficacy trial.

Ballenger continued that federal courts have recognized a number of fundamental rights and not all of them deal with life and death issues, including the right to teach your children German, educate them in private school, live with family members under one roof, view pornography at home, and engage in homosexual sodomy. Some other fundamental rights recognized by federal courts are the right to interracial marriage, to use contraception, to worship, and to obtain abortions.

University of Pennsylvania bioethicist Arthur Caplan pointed out that it was difficult to figure out who qualifies for the category "terminally ill." He also asked wyhy terminal illness in and of itself should give a patient a privilege or a right. After all, a patient who has exhausted standard treatments for some kind of severe disability, say, Parkinsonism, macular degeneration, or dementia, could argue that they have a right to access potentially better drugs that the FDA has not yet approved.

Scott Gottlieb, who has just stepped down as deputy commissioner at the FDA, thinks that "terminal patients should have access to experimental drugs after exhausting other treatments" He noted that FDA bureaucrats are concerned that opening access to investigational drugs to terminal patients might imperil the agency's ability to collect good data on drug safety and efficacy. They think that they are balancing individuals' interests in getting cutting edge treatments now against society's interest in obtaining more information in order to get more drugs to more people eventually.

Gottlieb noted that there is a marketplace issue. Companies have a disincentive to offer access outside of clinical trials. Why? Because side effects or other adverse events would be reported from patients who had not met the criteria for clinical trials. The companies fear that an already hyper-cautious agency would use such adverse event reports from outside of clinical trials as a justification for slowing the approval of a drug. Gottlieb told the colloquium that many inside the FDA believe that it is more important and better for society in the long run to gain good clinical data about a drug than it is to try to save the lives of individual patients. "I don't think the choice is that stark," said Gottlieb. "The FDA could pursue both goals."

Gottlieb asserted that the FDA is failing to use its authority to strike a balance on this issue. He suggested that the agency could start to develop alternatives to randomized placebo controlled clinical trials, perhaps incorporating Bayesian techniques. The FDA could also develop and validate surrogate clinical endpoints so that results can be known sooner than the results from trials that rely on long term survival rates. Gottlieb pointed out that new cancer drugs are rarely held up on safety grounds, so research should focus more quickly on efficacy. He does worry that the agency has begun to refuse to approve drugs that have fewer side effects but are less efficacious than earlier more brutal treatments. The agency fears that patients would trade off a higher risk of dying for fewer side effects. Why mentally competent people in consultation with their physicians should not be allowed to make such tradeoffs is not at all clear.

Mark Gately, a Baltimore attorney who defends pharmaceutical firms, pointed out another big issue in this debate -- the fact that federal law forbids a patient using experimental drugs from waiving negligence. Gately litigated the case involving patients enrolled in clinical trials for the drug GDNF, developed by Amgen to control Parkinsonism. The clinical trial did not find the drug to be efficacious, but many patients, who believed that they did benefit from it, sued the company demanding continued access to it. The company refused because it was worried by research that showed that the drug caused some brain damage in monkeys. According to Gately, a Kentucky judge asked the lawyer who represented two of a clinical trial participants, "What happens when one dies?" The lawyer answered, "They know what they are getting into. There will be no lawsuit." The judge replied, "This is America. If one dies, there will be a lawsuit." The bottom line for Gately: "Drug companies will be hard pressed to provide these drugs unless they are provided some protection from legal attacks."

For Frank Palumbo, who is the executive director of the University of Maryland's School of Pharmacy Center on Drugs and Public Policy, the central question is: "If patients are allowed access to these investigational drugs for the purpose of treating terminal illness, how can they afford them?" Newer cancer therapies are very expensive, some costing $10,000 per month.

After the somewhat dispassionate presentations of the panelists, representatives from the Abigail Alliance made themselves heard during the question and answer period. Steve Walker, the chief advisor to the Alliance explained what had happened to Abigail. She had exhausted all of the approved therapies. Her oncologist believed that the then-investigational anti-cancer therapy, Erbitux, being developed by ImClone might work for her because the drug was targeted against the EGFR receptor that encourages cancers to grow. Abigail's cancer had a very high EGFR expression. Despite preliminary evidence that it was an effective treatment for head and neck cancer, she did not qualify for clinical trials which were being run at the time only for colon cancer. The FDA approved the drug for treating head and neck cancer in March 2006. Erbitux boosts average head and neck cancer survival rates by about 2 years.

Walker noted that the current clinical trial system was devised 50 years ago in an era of medical ignorance. Today, researchers use genomic information to develop targeted drugs. Walker pointed to a current clinical trial involving a drug that early trials show works "unbelievably well" against metastatic kidney cancer. According to Walker, the FDA is insisting on randomized placebo controlled trial for approval. "Everyone knows the drug works," said Walker. So the end result is that the cancers in patients enrolled in the placebo end of the trial will have gotten worse by the time the FDA approves the drug in the next 4 or 5 months.

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