How Drugs Get From the Test Tubes to Us, Part 4

What can happen, Finn said, is that a person has a serious illness, is very worried and hears that there is an experimental treatment under investigation. Handed an informed consent form, the patient may be quick to say, “OK, doc, where do I sign?” Finn said. Instead of rushing into it, he said to read the informed consent form very carefully, ask lots and lots of questions, think it over, talk to knowledgeable family members and friends, and consider getting a second medical opinion.

In the pull between the needs of research and those of the patient, you are still your own best advocate. Given the available options, being wary and well-informed can keep you on a decision-making course that is best for your own health.

“Do you want to participate in this trial, or another trial? Do you want to get the standard treatment? Or do you want to get no treatment at all? These are all valid, rational options,” Finn said.

For some patients, the problem is how to find out about clinical trials that might be beneficial for them. “Even if you have the best oncologist in the world, he or she may know about a couple of dozen clinical trials,” Finn said. The National Cancer Institute (NCI) runs about 1,700 trials at any given time and pharmaceutical companies probably have at least that number. Some doctors, especially those who are not affiliated with any major medical centers, may not be in the loop of the latest trial opportunities.

“Way too few people consider cancer clinical trials,” he said. For example, estimates are that between 2 and 5 percent of adults with cancer participate in clinical trials.

There are resources available for patients to find out information on their own. These include:

The National Cancer Institute — 800-4CANCER. Ongoing trials are listed
The CenterWatch Clinical Trials Listing service has more than 7,000 trials in all areas of medicine
The government has just opened a database of clinical trials for serious illnesses, with explanations, questions and contact information
You can also contact your local medical center directly to find out what trials are going on there. For example, the homepage of the University Medical Center at Stony Brook. Its cancer helpline is 800-UMC-2215.
The rate of participation in clinical trials is much higher for children with cancer, said Keene, who is a patient advocate with the Children’s Cancer Group, which is made up of nearly 3,000 doctors and researchers who design and implement cancer clinical trials.

“More than 75 percent of children with cancer are enrolled by their parents at some point in their treatment,” she said. “Even though there is a much smaller number of children than adults with cancer, there are so many of them in clinical trials that there’s a big enough sample size to make a lot of statistically significant findings.”

For example, Keene said, 30 years ago childhood leukemia was almost always fatal. Now, more than 80 percent of kids with acute leukemia survive.

Keene ascribes the large number of children in trials to motivation. “Parents will do anything to save their children,” she said.

If the child is old enough and willing to be involved, sometimes parents will include the children in the discussion about the trial and have them sign the consent form as well. If the trial involves a teen-ager, and there is disagreement between the teen and his parents, the resolution may be handled differently by each institution. Often, a medical center’s ethics committee gets involved, the teen or the parents may get representation, and, at some point, everyone hopefully sits down together and comes to a decision that’s mutually acceptable. “I don’t think the situation happens very often, but when it does, it is emotionally wrenching,” Keene said.

There are many considerations for adults who are thinking about joining a clinical trial. Some are uncomfortable about the lack of control over which arm of an experiment they end up in. For example, in a randomized study, you or your doctor can’t decide whether you’ll get the standard treatment or the experimental one.

A computer decides this.

Trials also can involve more treatments, hospital visits, checkups and discomfort. There is a question of traveling and, in some cases, who is going to pay for it.