FDA more cautious than compassionate

[Christine]

By Vindu Goel

Mercury News

Article Launched: 10/10/2007 01:36:35 AM PDT

If you were dying of cancer and there were an experimental drug that could give you an extra six months to live, you'd want access to it.

You'd still want it even if it had awful side effects, like turning your skin green and making your fingernails fall off - which can happen to people taking Xcytrin, a drug from a Sunnyvale company that shows promise for treating brain lesions caused by the spread of lung cancer.

But is it the government's responsibility to make it easier for you to get such life-prolonging therapies, even if they cost tens of thousands of dollars a year?

That's the question the Food and Drug Administration, Congress and the courts are wrestling with as some drugmakers and patient-rights groups crusade for more flexible rules in evaluating new drugs for terminal diseases.

We all want the best medicine available for ourselves and our loved ones. Yet we don't want to subsidize ineffectual treatments by paying higher hospital bills and insurance premiums. And we certainly don't want the government rushing to approve drugs that aren't safe.

The evidence suggests that when it comes to approving drugs for fatal diseases, especially cancer, regulators at the FDA are being too cautious.

New cancer drugs take an average of seven years to get FDA approval, and 92 percent of all applications are rejected, according to a study by Tufts University. That's a longer review time and a lower approval rate than most drugs face.

In the past year alone, the FDA has turned down or demanded more data on five experimental cancer drugs, including Provenge, a prostate cancer drug that got the thumbs-up from an FDA advisory panel.

The agency's actions have riled Dr. Richard A. Miller, a Stanford oncologist and chief executive of Pharmacyclics, a small publicly held biotech company which has about a year of cash left and badly needs the FDA to approve Xcytrin, its first drug. Miller has launched a Web site, www.yourcanceryourchoice.com, to push for better patient access to new cancer drugs.

In February, the FDA refused to accept his company's application to market Xcytrin. Its clinical trial data didn't pass a predefined test designed to determine whether the extra five months recipients lived was due to the drug or pure chance. Pharmacyclics is demanding a formal FDA review, saying some French doctors didn't follow the study protocols, which unfairly skewed the data.

Miller argues that the FDA is holding drugmakers to rigid, outdated scientific and statistical standards. "If a person is going to die in a few months, it's a different risk-benefit proposition than for someone with a hangnail," he said.

Steve Whitley, a sales manager at a Phoenix cement maker, thinks the trade-offs were worth it. When his wife, Cindy, was diagnosed with lung cancer that had spread to the brain, the couple quickly decided to sign her up for an Xcytrin drug trial.

Instead of living the expected three to six months, she got 25 months to spend with him and their three children. "It meant two more years of birthdays and Christmases," Steve Whitley said.

A 1992 law, passed in response to the U.S. AIDS epidemic, was supposed to speed approval of drugs for fatal diseases. But recently, the FDA has become more cautious just as drugmakers try to tackle more difficult diseases. One explanation is increased scrutiny of the FDA after Merck withdrew painkiller Vioxx in 2004 because of increased risk of heart attacks and strokes.

The agency declined to comment on specific drugs but said it works hard with companies to design measures of effectiveness for each drug and disease. Once those criteria are set, companies have to hit the targets.

"You need to win on what you think you're going to win on," said Dr. John Jenkins, director of the FDA's Office of New Drugs. Changing the rules in the middle of the process or selectively excluding some patients is "just not scientifically sound."

The Abigail Alliance for Better Access to Developmental Drugs disagrees, calling for a new law that would allow patients to buy experimental drugs and overhaul the FDA's scientific and statistical approach.

The advocacy group also has sued to force the FDA to give patients early access to drugs that have passed safety tests but are still being evaluated for effectiveness. Courts so far have backed the FDA.

If I were dying, I'd want the chance to weigh the risks and decide with my doctor whether to try something that might help me. The FDA, so dedicated to its traditional scientific standards, could use a dose of compassion.

As Whitley put it: "What's the risk? You're going to die."