Drugs may win faster approval

FDA spearheading effort to speed up the process

December 01, 2006|By Jonathan D. Rockoff | Jonathan D. Rockoff,Sun reporter

WASHINGTON -- Federal health officials want to alter the foundations of the system entrusted with making sure new drugs are safe and effective.

By streamlining the way scientists determine a new drug's safety and effectiveness, advocates say, new cures can be delivered to dying patients much more quickly.

But many scientists outside Washington say this effort, spearheaded by the Bush administration and drug companies, could rush new treatments to market too quickly - and wind up hurting rather than helping patients.

Their concern is heightened by critics who already question the government's commitment to assuring the safety of new drugs.

Administration officials, led by Dr. Andrew C. von Eschenbach, acting director of the Food and Drug Administration, have dedicated an office in the agency to direct the overhaul effort. And they've given the initiative a name: Critical Path.

So far, the debate hasn't made waves outside medical circles, and prospects for change have never been better.

The plan has enjoyed bipartisan support on Capitol Hill - at least among the small group of key legislators who follow pharmaceutical issues. Thanks to its low profile, Critical Path never became an issue in last month's congressional elections.

How would it work? Since the 1960s, the FDA has required pharmaceutical companies to test new drugs on hundreds of subjects over many years. The agency reviews the final results to determine whether a drug is safe and effective before deciding whether to approve it for sale.

One proposed change would allow drugmakers to adjust the direction of an ongoing clinical trial to determine whether a new drug has a benefit that scientists weren't looking for originally. Currently, drug companies must finish testing for one benefit before working on another.

Another change would allow drugmakers to evaluate a new drug's impact by using shortcuts, known as markers, that predict its effect, instead of waiting for actual results. A drug might change a protein, molecule or some other marker, such as blood pressure or blood sugar, indicating that it will help a specific condition.

Scientists could use generally accepted markers to assert that a new drug will be effective, just as meteorologists seeing a radar blip can tell when a storm is starting without seeing a raindrop.

Neither the FDA nor the drug industry is ready for wholesale changes. Supporters and critics alike say additional research will have to support the use of markers.

That's why the future of drug development rests on scientists such as Dr. Jerald Radich, whose study of a cancer marker could help determine whether streamlining will work.

Radich believes that, somewhere in the blood of certain leukemia patients, there's a molecule that indicates whether a drug will successfully attack the disease. That molecule is a suspected marker, or chemical radar blip.

To see whether this marker performs the way Radich thinks it does, his lab in Seattle analyzes test tubes of blood from patients with a rare but insidious cancer.

Chronic myeloid leukemia can attack without symptoms, relentlessly taking over bone marrow. But a new drug called Gleevec targets the molecular underpinnings of the cancer, blocking diseased enzymes from creating cancerous blood cells in the first place.

Scientists want to see whether Gleevec works better at higher doses, or when it's combined with other drugs. That would normally take at least five years of testing, a period Radich wants to cut to a year with the chronic myeloid leukemia marker he's identified.

"It really could revolutionize the way we do therapy," he said.

Waiting for hope

Cancer activists are agitating for these shortcuts. They complain that too many patients die waiting for the FDA to approve promising treatments.

According to the industry-backed Tufts Center for the Study of Drug Development, it takes an average of seven years and $868 million to bring a drug to market. Just 20 new drugs won government approval last year, the lowest number in at least a decade, the FDA said.

"You don't want the regulatory process becoming an impediment to bringing new science to patients," said Dr. Scott Gottlieb, an FDA deputy commissioner.

In an unusual step for a regulator, the FDA - rather than academia or industry - is leading the charge. With the National Institutes of Health and the Centers for Medicare and Medicaid Services, it's paying researchers to find good markers. Meanwhile, FDA staffers have been writing new guidelines for the industry to follow when developing drugs.

The FDA's chief calls the changes a top priority. A cancer surgeon and cancer survivor, von Eschenbach served as a consultant to a prostate cancer foundation created by financier Michael R. Milken, who has promoted regulatory changes to accelerate approval of new drugs for desperate patients.

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