FDA facing challenge on trial drugs

The Baltimore Sun

WASHINGTON -- At the same time that many Americans complain the government isn't doing enough to protect them from unsafe drugs, advocates for terminally ill cancer patients assert that health officials do too much.

Dying patients who have run out of other options have a constitutional right to take unproven medicines, say cancer patients who are challenging the government's authority to prevent them from taking potentially promising drugs that are under development.

"Their only alternative is death," said Frank Burroughs, who has fought for expanded access since his 21-year-old daughter, Abigail, died while trying to get experimental drugs during cancer treatment at Johns Hopkins Hospital in 2001.

Burroughs has mounted a legal challenge to the Food and Drug Administration's decades-old authority to regulate new drugs. He wants to greatly expand access to unproven medicines, which the agency restricts to the few patients participating in a scientific study or receiving special FDA permission.

His initiative has alarmed many in the medical and legal communities, especially after a federal appeals court in Washington ruled in his favor in May. At the request of the Bush administration, the court agreed to reconsider its ruling and will hear oral arguments in the case today.

Researchers worry that if Burrough's challenge holds up in court, it would seriously damage new drug development. They say it would encourage many patients who currently enroll in clinical trials to simply request the experimental treatments instead. In drug trials, a portion of those who participate receive a placebo, rather than the drug itself, and therefore cannot benefit immediately if the experimental drug works.

Health officials, meanwhile, worry that it would drastically weaken the FDA's authority by creating a constitutional gap in its gatekeeper role.

"It's a real bad policy idea," said Donald Kennedy, the FDA's commissioner from 1977 to 1979.

"It would cripple [the agency] to a considerable degree," Kennedy said, by undermining its power to monitor the safety of drugs and other products. For some patients, he said, its rules would no longer apply.

The legal attack on the FDA is, in effect, the flip side of a more common complaint: that the agency exercises too little caution when approving new treatments.

Some critics attack the agency out of a popular but mistaken belief - bolstered by frequent reports of new discoveries - that there is a cure for every illness, said Peter D. Jacobson, director of the University of Michigan Center for Law, Ethics and Health. To these critics, he said, the FDA, not the state of medicine, is blocking treatment.

The FDA generally bars patients from taking medicines until studies show they work safely. The agency makes exceptions for patients who participate in the studies. It also allows about 700 patients with life-threatening illnesses each year to try treatments under development; in December, the FDA proposed changes making it easier to obtain such drugs through its expanded access programs.

In challenging the FDA's authority, Burroughs points out that many patients don't qualify to participate in a study. He also says it's very difficult to obtain drugs through the FDA's expanded access programs.

After his daughter's death, he established the Abigail Alliance for Better Access to Developmental Drugs to advocate for greater and easier access.

"We are making huge strides in research, in treatment of cancer and other diseases," said Burroughs, a Fredericksburg, Va., engineer who is working full time on his cause. He said the FDA blocks dying patients and their doctors from trying promising treatments out of a wrongheaded focus on "statistics."

"They don't have patients' best interests at heart," he said.

The Abigail Alliance filed the lawsuit along with the Washington Legal Foundation. The nonprofit foundation's chief counsel, Richard A. Samp, says that obtaining experimental drugs for desperate patients is no different from the right of a homeowner to defend himself against a deadly intruder or of a woman to have a late abortion if her life is in danger.

The Abigail Alliance and the WLF are asking the court to endorse the idea that terminally ill patients who have run out of other options have a constitutional right to take promising but unapproved drugs.

Samp said his aim isn't to weaken the FDA - it's to help thousands of patients have access to new drugs that could mean the difference between life and death.

"Who should be the one making the decision?" Samp asked. "FDA or the person who's been told, "There's nothing we can do for you, so go off and die quietly'?"

Some opponents of the suit, such as former FDA chief Kennedy, said the WLF has a bigger goal: "This is an anti-regulatory move, and it's driven by people who don't think the government should be in this business" of guarding drug safety, Kennedy said.

Richard M. Cooper, a Washington lawyer who was the FDA's chief counsel from 1977 to 1979, said politicians and policymakers should decide, not judges. Otherwise, he said, patients who aren't terminally ill but have serious diseases could also demand experimental treatments before it's clear the drugs won't cause them harm.

Dr. Gary I. Cohen, director of the cancer center at the Greater Baltimore Medical Center, said cancer patients occasionally demand use of unproven drugs for misguided reasons. Often, he said, there is another approved option available. And whether approved or not, he said, treatments usually delay death, rather than cure the disease.

Many drug companies would object to supplying experimental medicines before approval for several reasons. They might not make enough to supply anyone outside of clinical trials. And they might leave themselves open to being sued if the experimental drugs proved harmful, Baltimore lawyer Mark D. Gately, who represents drug manufacturers, told a legal conference this week.

Many medical organizations and patient disease groups also oppose the establishment of a constitutional right to take experimental drugs for fear it will damage the clinical trial system.

Dr. Allen Lichter, executive vice president of the American Society of Clinical Oncology, said it could expose the sick to fraudulent cures.

"The legitimate players are not in favor of this," Lichter said, referring to companies and organizations opposing the lawsuit, including the Pharmaceutical Research and Manufacturers of America. "This is a potential tragedy."


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