San Francisco Chronicle (SF); Thursday, July 11, 1991
Sabin Russell, Chronicle Staff Writer

The drive for so-called conditional approval of AIDS drugs has caused a fierce policy debate within the FDA, Congress, pharmaceutical companies and even among AIDS patients and those who treat them, some of whom fear the change would invite medical quackery.

Conditional approval represents the latest and most radical plan yet in the continuing battle to make experimental life-saving treatments available to patients. It would, for the first time, relax the rigorous FDA standards required to prove that a new drug is both safe and effective. It would also allow drug companies to profit from sales of medicines that today would be called experimental.

The concept, which will form the basis of new legislation, is a significant advance from a current FDA program, known as expanded access, that has been criticized for shortcomings in the delivery of experimental medicine to AIDS patients.

Because AIDS is a slow developing disease, it takes an agonizingly long time to prove that experimental AIDS drugs work. Thousands of patients have died waiting for new treatments to become available -- whatever the risk involved.

Jesse Dobson, who has been taking underground drugs in his battle against HIV infection for years, does not want to become another of those statistics.

"If an experimental drug kills some people, that's an acceptable risk," said Dobson, who supports anything that will speed the release of new drugs. "People are dying of AIDS anyway. I'm tired of being protected."

AIDS doctors, equally frustrated with the system, agree. "If we do all the studies that ultimately must be done, it will be three to five years before we know the results," said veteran San Francisco AIDS specialist Marcus Conant. "But I have patients who won't be here five years from now."

The current testing policy actually encourages AIDS patients to seek experimental drugs from underground labs, Dr. Conant said. Concealed from researchers, the illicit drug-taking taints the results of even the most carefully constructed scientific trials, he said.

Key elements of the concept are included in a bill to be introduced to Congress today by Representative Tom Campbell, R-Sunnyvale. Called the Access to LifeSavings Therapies Act, it empowers the FDA to change standards required for drugs to treat conditions such as AIDS, cancer, Alzheimer's and Parkinson's diseases.

Significantly, the legislation also would require insurers to reimburse patients who are prescribed drugs approved under the new standard.

The intent of Campbell's bill -- and others in preparation -- is to cut years off the time before a drug reaches the marketplace, unclogging the bureaucratic machinery. Ten years after the first AIDS cases were diagnosed, only one drug, AZT, is approved to treat the human immunodeficiency virus that causes the disease, while a dozen possibly useful treatments await results of lengthy clinical trials.

"If we find a drug that really works, a one-year reduction in approval time could translate into thousands, if not tens of thousands, of lives saved," said James Driscoll, who specializes in treatment issues for ACT UP Golden Gate, an organization that supports AIDS patients.

Earlier attempts to speed delivery of AIDS drugs have had mixed results, Driscoll said. The FDA's expanded access program -- which allows limited distribution of experimental treatments -- prompted Bristol-Meyers Squibb to give the experimental drug ddI free to more than 19,000 AIDS patients. But access tends to be limited to the well-educated, and smaller drug companies may be shying away from AIDS treatments for fear of bankrolling such a program, which by one estimate is costing $2 million a month.

Backers of conditional approval believe that they have an uphill battle to win support for the concept from Henry Waxman, D-Los Angeles, the influential congressman who heads the House Subcommittee on Health and Environment. Tests to prove that drugs are effective are critical, he warned. "Otherwise, we'll have a lot of snake oil being sold to vulnerable, desperate people." But Waxman said he is reserving judgment on the issue until he sees a forthcoming report from an FDA task force, which is expected to issue recommendations this month on ways to speed the drug approval process. Although most AIDS advocates solidly back conditional approval, some have reservations. "People are waiting for more information on this concept," said David Gold, a specialist in treatment issues for ACT UP in New York. "We do not want to see drugs that are unsafe or inefficacious on the market."

Drug industry support for the idea is lukewarm. Big pharmaceutical companies have little to gain if the rule change gives competitors a head start on drugs that could eat into existing sales. The concept is more popular among smaller firms.

A major concern of drug makers and AIDS patients alike is whether insurance companies and the federal Medicaid program will pay for conditionally approved drugs or will still treat them as experimental and thus exempt from coverage.

FDA spokesman Brad Stone said a task force of FDA and National Institutes of Health officials expects shortly to release a report focusing on ways to accelerate approval of AIDS drugs. Key to the FDA report will be recommendations on the use of so-called surrogate markers -- alternative measures of disease progression -- as standards of effectiveness.

Currently, the FDA has only two gruesome ways to judge how well an AIDS drug is working: monitoring the onset of deadly opportunistic infections and measuring the time it takes for AIDS patients to die.

For years, AIDS researchers have been debating the merits of surrogate markers such as T-cell blood counts. "There is a lot of speculation whether there really are good, reliable surrogate markers," said Stone.

Although current FDA practice requires that a drug meet the test of "substantial" effectiveness in clinical trials, the bill to be introduced by Campbell today would substitute a looser standard of "promising" evidence. By that new standard, a single favorable study rather than two such trials could be enough to clear a drug for sale. The bill encourages use of surrogate markers and requires drug companies to finance further testing.

Always watch for outdated information. This article first appeared in 1991. This material is designed to support, not replace, the relationship that exists between you and your doctor.

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