The Wall Street Journal - May 13, 1996
Marilyn Chase

Trials can offer advantages, if patients carefully weigh risks and benefits, but they aren't a ticket to Lourdes.

"People have to have their eyes wide open," says Cliff Lane, clinical director of the National Institute of Allergy and Infectious Diseases. Miracle-seekers may be disappointed, he says, because most experimental drugs don't pan out.

Clinical trials test a new treatment against standard care. If no standard drug exists, the control group may get a placebo, or dummy drug. Ethical qualms and patient protests have made placebos rarer these days. Phase 1 studies test safety; phases 2 and 3 weigh efficacy. The lower the number, the greater the uncertainty -- and risk. The government compiles databases for many major disease studies.

Volunteers don't always get their drug of choice. To prevent bias, major trials are randomized, and patients are assigned treatments by computerized coin toss. In double-blind studies, neither doctor nor patient knows who is getting what.

"You need to be comfortable with the notion of a coin toss," says Robert Wittes, director of the National Cancer Institute's division of cancer treatment. People sure of their best course, he adds, shouldn't participate in a study.

STUDIES HAVE layers of safeguards, if properly designed. A peer-review committee scrutinizes the quality of science. An institutional review board oversees ethics. A data-safety and monitoring board stops studies that go awry. Patients sign informed-consent forms spelling out risks and benefits.

Still, participants sometimes get hurt, and some patients have died from drug toxicity. During a study of interferon plus antivirals that cleared his hepatitis B, Martin Delaney of San Francisco suffered painful nerve damage. "I'd do it again," he says. "It's a personal choice."

Logistics can be tricky. "The disadvantage of going into a clinical trial is that they tend to be conducted at large centers, and it's not fun to travel if you're sick," says Greg Curt, clinical director of the National Cancer Institute. "The advantage is the care. The diagnostic studies, treatment regimens and dose modifications are spelled out very carefully, like a recipe. It's not seat-of-the-pants."

Amy Langer, executive director of the National Alliance of Breast Cancer Organizations, says, "The good news is patients receive at least the state-of-the-art in the standard treatment" regimen; new agents offer a possible response or cure, or a quality of life that's superior to standard care.

Additional benefits are a chance to advance research, and close health monitoring for the duration of the trial. A drawback: health plans and insurers often rule out reimbursement for medical care in studies.

Dr. Wittes urges consumers to ask their carriers about coverage.

Before you sign, ask yourself several basic questions, says Barbara Fabian Baird, research study coordinator at Niaid:

"Do I have the time and energy to participate?"

"Is this drug going to be safe for me?"

"Is the trial placebo-controlled, and what are my risks if I'm in the placebo group?"

"What are the side effects and am I willing to tolerate them?"

Ms. Baird faced such questions 15 years ago when she was diagnosed with breast cancer. A recent divorcee with a new job and young children, she didn't have time for the study, and opted for a then-standard mastectomy. Ironically, the study vindicated lumpectomy.

Diane Walstad, of Elburn, Ill., made the opposite choice. After surgery, she was offered standard chemotherapy, or a chance to test a new high-dose regimen.

SHE LOOKED at the trial "as something that might bring me benefit," she says.

"I wouldn't describe it as pleasant," she says of the 100-mile round trips to get nauseating therapy. "The side effects were expected but manageable." Among them: hair loss and early menopause. Six years later, she's cancer free and recalls the study as "a very positive thing."

For Mark Frey of San Francisco, clinical trials were a way to face life with HIV. "I got monthly monitoring for my blood, a miniphysical, [lab tests] every three months. It was a way of getting good health care while helping out."

Be clear and vocal about your practical needs, advises Ken Fornataro, executive director of New York-based AIDS Treatment Data Network, which counsels 25,000 people a year about clinical trials.

Write down the schedule of treatments, tests and clinic visits, he suggests. "Put it all into a day planner and see if it fits into your life."

If the trial is placebo-controlled, ask if the study has a "crossover" phase, when the placebo group gets the real drug, or a promise of treatment at the study's end.

A trial isn't for people in very fragile condition, he says. "We'd advocate for that person to get the drug under a compassionate-use program rather than a trial," he says.

To find clinical trials, you can call the U.S. Cancer Information Service or the AIDS Clinical Trials Information Service using 1-800 directory assistance.

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