When Barbara Holtz of Wayland, Mass was diagnosed with breast cancer in 2001, she remembers being told she had “a frightening tumor that would need rapid and aggressive treatment.” But she also learned she was eligible for a randomized clinical trial of a new drug called Hercpetin, a so-called targeted treatment, that showed promise against early stage breast cancer.
Holtz wanted in but there was one stumbling block. “What if I didn’t get the drug?” she recalls, knowing that a control group would be assigned to receiving the current standard treatment. But telling herself she wanted to make a contribution to science, she decided to go for it.
Holtz is among the estimated two million people who participate in clinical trials each year in the U.S. Some people volunteer because they want to help science; others have a more personal reason. “They want access to a novel treatment,” says Kenneth Getz, founder and chair of the board of The Center for Information & Study on Clinical Research Participation (CISCRP), a nonprofit organization designed to raise awareness of clinical research participation.
Soon after being accepted into the trial, Holtz was relieved to learn she would, in fact, get the Herceptin. “I was thrilled but mystified because I didn’t know if it would really work,” she says.
Almost six months into the year-long study, though, she had to stop taking the drug after testing revealed it was affecting her heart, putting her at risk for congestive heart failure. “I felt like I was being kicked out of paradise,” Holtz recalls.
As it turned out, nearly six months on the drug had been enough. “Here I am after 10 years and I am doing fine,” she says—heart and all. “I believe the drug saved my life.”
Whether you’re interested in entering a trial to further medical research, like Holtz, or to get cutting edge help for a condition, Getz recommends asking the following questions to learn as much as you can about the study. It’s all part of a process called informed consent. But keep in mind: Often you won’t be able to get all the answers until you’ve been formally accepted into the trial.
1. Exactly what will happen during the study? You need to know its purpose and who’s going to be involved as well as how the study will be conducted. Will you be trying a new medication or having an experimental surgical procedure, for instance? Ask whether or not the treatment has been studied before, what the results were and whether the researchers think the treatment will work.
2. How long have you been conducting this research? Other questions to broach: How many similar studies have you worked on? What are the credentials of the people who will be running the study? “You want to get a good intuitive sense of the kind of people with whom you’ll be interacting,” says Getz. “Do they seem forthright and eager to put you at ease?”
3. What are the risks? Ask about possible short- and long-term side effects and what will happen if you develop one. “If it’s a reputable center, the researchers will have a very open discussion about the critical importance of being able to drop out at any time and about any adverse event procedure,” says Getz, author of Informed Consent: A Guide to the Risks and Benefits of Volunteering for Clinical Trials
4. What kind of time commitment do I need to make? You may need to visit a center weekly or monthly for a few months or a year or more. “Being in a trial is a big commitment,” says Getz.
5. Will I be paid? Some studies cover travel and parking expenses. Others may pay volunteers a flat fee. One big perk has no monetary value: top-notch health care. “You will have people paying more attention to you, conducting more procedures and giving you insights into disease you would not otherwise have had access to,” says Getz.
6. Will I have access to the experimental treatment? You’ll need to know whether everyone in the study will receive the therapy or whether, as was the case in Holtz’s study, you’ll be randomized to treatment and non-treatment groups. Also, ask how you’ll be treated if you don’t end up in the investigational group. (Usually, you’ll receive the standard of care.) Once a study ends, people who didn’t receive the study treatment may be given access to it if it has proven effective. So ask about that.
7. What will happen if the treatment being studied comes into question? “If that happens, a study has to be terminated, “ explains Getz. Make sure that is in the policy guiding the researchers for your study.
8. Has the study been approved by The Institutional Review Board Services? Required by law to approve all trial protocols, the IRB determines if a study will be safe for volunteers and whether the benefits outweigh any risks, says Getz.
Whatever you decide, keep in mind: “Participating in a trial is a big leap of faith,” says Getz. You should also bring “a healthy dose of skepticism” to the table, he adds: “Just because one patient has had a positive experience doesn’t mean that will happen to everyone.”