Apr 16, 2002 By Lauran Neergaard Ap Medical Writer 4 min read

WASHINGTON (AP) – Standard treatment isn’t working so you’re desperately seeking an alternative. Is a clinical trial of an experimental drug a good idea, or too risky?

Can you trust the researchers?

Can you drop out if something happens?

Wading through all the scientific jargon to decide on a medical experiment is incredibly difficult for sick patients, a dilemma highlighted by several high-profile deaths in recent studies.

Now a new campaign aims to arm Americans with the best questions to ask before signing up, to ensure they’re fully informed. It’s education that research experts – and families who’ve struggled through the mysterious world of medical experiments – say is sorely needed.

“We didn’t know enough even to ask questions,” recalls Fernando Segura, a Pennsylvania engineer who helped his wife, dying of lung cancer, enter a study of a promising treatment. “It is frustrating. You feel like you are at everyone’s mercy.”

He’s not alone. A recent survey by CenterWatch, a company known for its Internet database of clinical trials, found 14 percent of study participants didn’t understand they could suffer side effects during an experiment even after signing required consent forms.

Maria Segura was too ill to be helped. But the couple’s experience advised health experts writing the free new patient guide, “Should I enter a clinical trial?”

“A lot of people don’t understand the whole context of research,” says co-author Haavi Morreim, a bioethicist at the University of Tennessee, Memphis, who also heads a unique patient advocacy council for a highly publicized study of an artificial heart.

So the guide starts with the basics: Clinical trials are not intended to treat an individual but to advance medical knowledge for future patients. Participants certainly may benefit, but there’s no guarantee. Indeed, many experimental therapies fail to work and occasionally even kill.

More than 5,000 clinical trials are conducted each year. Patients may learn about them from a doctor, a manufacturer’s ad, a patient support group or the Internet. The Food and Drug Administration just told manufacturers to list all trials testing the effectiveness of drugs for serious or life-threatening diseases on a federal Web site – www.clinicaltrials.gov – by early next month.

There are different kinds of studies, explains the patient guide, produced by the nonprofit health analysis agency ECRI. Funded by the American Association of Health Plans, it is being distributed by consumer and doctor groups, and the ECRI and AAHP Web sites.

A Phase 1 study marks the first time an experimental drug has ever been tried in humans – not to see if it heals but to see if it’s toxic. Only 3 percent to 5 percent of Phase 1 participants get any benefit, Morreim stresses, vital to know before signing up. “Maybe it wouldn’t change the person’s mind, but it’s an important understanding.”

If few people suffer serious reactions, then a slightly larger Phase 2 trial looks for signs that the drug works.

Phase 3 is the biggest test. Patients almost always are randomly divided so some get the experimental drug and others get a placebo – a dummy dose – or standard therapy.

To participate, the law requires that people get a full explanation of risks and potential benefits. Yet CenterWatch’s survey of 1,600 recent trial participants found many remained confused about risks after undergoing that “informed consent” – and, worse, 10 percent signed forms entering the experiment without even reading the explanations of risk.

Question not only the researchers and the study’s overseers or patient advocates, but a family doctor or other trusted adviser about the pros and cons, says CenterWatch president Kenneth Getz, who is selling his own patient guide.

Risk isn’t the only concern, the free ECRI guide stresses. Participants must understand:

– The biological rationale for the study, so they can compare all their options.

– That they can drop out for any reason.

– Who to go to if their illness worsens or they suffer a side effect.

– Who’s funding the research and whether the doctor owns stock in the therapy’s maker or has another conflict of interest that might affect patient welfare.

– While many trials are free, some incur costs that not all insurers cover.

– What tests and checkups are required, important in planning travel, lodging, child care and other possible commitments.