Because you might help save lives
By Wendy Haaf
It’s a fact that most of us never think about: researchers can’t advance medical knowledge unless volunteers act as subjects in clinical trials, and only a very few people—an estimated five per cent of those who would qualify to participate—volunteer.
Perhaps you’ve never considered taking part in health research or you’ve dismissed the idea for some reason—because, for example, you wouldn’t want to take a chance on getting a placebo rather than a promising new treatment. In fact, the average person can help advance medical knowledge in a number of ways that involve no such placebo possibility, and in many cases, a person can contribute even if perfectly healthy. Joining a research trial can confer some surprising benefits, and while it can carry certain risks, every effort is made to minimize these.
Studies Aren’t All the Same
There are different types of studies for which someone might volunteer, including the classic drug study in which patients with a certain ailment are randomly assigned to take either a new drug or a placebo. (For brevity’s sake, our discussion of drug studies is limited here primarily to Phase III clinical trials—those carried out after a medication is tested for safety and effectiveness in relatively small numbers of patients.) However, even in some placebo-controlled studies, if the drug turns out to offer a benefit, “there’s the possibility of continuing with what’s called an open-label study,” notes Dr. Michael West, a nephrologist, professor, and assistant dean of research for clinical trials at the Faculty of Medicine at Dalhousie University in Halifax. “So you start with a placebo-controlled trial, where half the people get the drug and half get the placebo, and then, perhaps after six months, everybody gets the new drug,” he explains.
Moreover, many Phase III trials don’t use placebos at all; instead, they compare a promising new treatment with the best existing one. “No trial in cancer is going to give people anything less than the standard of care,” stresses Dr. Janet Dancey, director of the Canadian Cancer Trials Group and professor of oncology at Queen’s University in Kingston, ON.
Other trials test a treatment approach rather than a drug, in the hope of finding ways of more effectively using tools we already have. In a trial taking place in Edmonton, when people with a type of heart rhythm disturbance called atrial fibrillation (which is linked with an increased risk for stroke) are referred to the Mazankowski Alberta Heart Institute, they’re randomly assigned to receive care from either a cardiologist or a nurse practitioner.
“The cardiologist and the nurse practitioner might do exactly the same thing, but what we’re looking at is patient satisfaction,” explains Ross Tsuyuki, a doctor of pharmacy, professor of cardiology, and director of the EPICORE Centre at the University of Alberta in Edmonton. (EPICORE, or Epidemiology Coordinating and Research, is a clinical trials and health services research centre.) He points out that a similar trial in the Netherlands showed a reduction in deaths in the nursing care group. “We think that patient satisfaction and quality of life will be better because the nurse practitioners spend a lot more time with people,” he says.
In another recent trial, people with risk factors for heart disease (such as high cholesterol and high blood pressure) were randomly assigned either to receive care from their family physician or to get help from their pharmacist in managing these conditions. “We saw a 21 per cent reduction in the risk for bad heart disease-related events” in the latter group, Tsuyuki says.
Some studies don’t involve any intervention; they simply follow a group of people over a period of time (participants may undergo certain evaluations, have blood drawn at certain intervals, and/or give permission to allow researchers to access their health records). In the Canadian Longitudinal Study on Aging (CLSA), for instance, 50,000 Canadians aged 45 to 85 (on enrolment) will be tracked for at least 20 years in an attempt to understand how various biological, psychological, medical, social, economic, and lifestyle factors influence both the maintenance of good health and the development of disease and disability.
“I was interested because I have bad genes—I have two uncles who died of heart disease, one at age 42, and a father who died of cancer at 57—but I think that lifestyle mitigates on genetic predisposition, and I have strong beliefs in the value of nutrition and exercise in maintaining health,” says Roger McLeod, a CLSA participant and a professor of biochemistry and molecular biology and associate dean of research in the Faculty of Medicine at Dalhousie.
Similarly, the Atlantic PATH (Partnership for Tomorrow’s Health) study is investigating environmental, lifestyle, and genetic factors related to the development of cancer in residents of Eastern Canada.
“We learn a lot more from studies carried on with the same cohorts for a long period of time than from population snapshots,” McLeod says. For example, the risk factors we now know to contribute to heart disease were identified thanks to the Framingham Heart Study, which was launched in an effort to identify these variables. McLeod and a collaborator will be using some of the samples and data collected for the Atlantic PATH study to determine whether a protein released when fat tissue starts to function abnormally can reliably predict the development of Type 2 diabetes very early in its onset.
Patients, caregivers, and health care consumers can also contribute to health research, but in a completely different capacity: by working with researchers at various stages of the research process, from generating ideas about which questions to ask in a study, to helping to get the word out about research findings. This concept, called patient engagement in health research, is the wave of the future for all types of health research, including clinical trials. In fact, one of the major funders of health research in Canada, the Canadian Institutes of Health Research (CIHR), has made patient engagement one of the criteria investigators must meet to be awarded certain grants.
“Patient engagement goes well beyond the typical role of participant/subject to one that is very broad and respects and builds on the expertise of the patient or family member—expertise about their disease, condition, situation, or experience,” explains Virginia Vandall-Walker, associate professor with the Faculty of Health Disciplines at Athabasca University, adjunct associate professor with the Faculty of Nursing at the University of Alberta in Edmonton, and lead of the Alberta SPOR SUPPORT Unit’s Patient Engagement Platform (SPOR is the CIHR’s Strategy for Patient-Oriented Research).
For example, when a group of researchers designing a study on dialysis asked patient advisors what their biggest concerns were, the main answer was itching—a troublesome symptom that wasn’t even on the scientists’ radar as a possible outcome to measure. (Thus far, Alberta is the only province with an online Patient/Public Registry, bit.ly/peRegistry, where those interested can go to sign up and choose studies that appeal to them.)
What’s In It for You?
Now that we’ve outlined how you might get involved in health research, there’s the question of why you would consider it.
“The most common reason people cite for wanting to participate in research is wanting to give back” and to help advance medical knowledge, says Dr. Lawrence Richer, director of the Northern Alberta Clinical Trials and Research Centre (NACTRC).
“The notion of contributing to science has a lot of meaning for many people, especially people who’ve experienced complications of a disease like diabetes,” says Dr. Jan Hux, chief science officer at the Canadian Diabetes Association. “They want the next generation to have better outcomes.”
Arni Goodman, 74, of Edmonton, has seen first-hand how others have benefited from a research project in which he participated a number of years ago; he helped connect Vandall-Walker with men who’d been through prostate cancer treatment to find out what they wished they’d known beforehand, and then collaborated on a patient-education booklet based on the results.
“When I was going through prostate cancer surgery, I felt that some of the direction I was given, and the training given to the person directing me, was inadequate,” Goodman says. For example, he was simply given printed instructions on how to perform Kegel exercises (used to help regain urinary control after prostate surgery), which didn’t provide adequate detail. Since the patient booklet was released, however, Goodman says he’s watched other members of the support group he’s involved in benefit from it. “A fellow who went through prostate cancer shortly after the booklet was published commented that it gave him a wealth of information that wouldn’t have been available beforehand.”
For John Hawse, 60, a former Toronto fire chief, participating in a study on early insulin treatment for Type 2 diabetes helped provide a sense of purpose during a period when a neck injury left him unable to work or do many other activities. “I was feeling pretty vulnerable,” he says. “I think it helped a lot psychologically that I could contribute.”
For Laura Saunders of Edmonton, providing input on a survey that was going to be given to parents of kids visiting a specialist left her feeling empowered to ask questions of her own health care providers. “It was a wonderful experience,” she says. “We’ve all complained about the health care system at one point or another: patient-engaged research is a way to take these feelings and opinions and have them heard by people who have the means to create change. Having physicians open to engagement and working collectively could change the way health care is not only delivered but managed across our country.”
Of course, in some cases as a study participant, you may get access to a promising new treatment that might offer more of a benefit than currently available treatments. “The first people to have access to Herceptin to treat breast cancer were those who enrolled in the trials,” Queen’s University’s Dr. Dancey notes.
In addition, in research studies, drugs, including any given to people in the treatment groups, are often provided at no cost.
Enrolling in a clinical trial can also bring you into contact with some of the leading experts of a particular disease or condition, since they’re often involved in running such studies. Participants can receive quite a bit of extra education and support, as well, which may help them better understand and manage their condition.
“There were so many plusses that I didn’t know about when I signed up,” John Hawse says. Because he received one-on-one support and advice during the two weeks each month that he injected himself with insulin for the study, he became skilled at matching the dose to his food intake and at planning ahead for special events and dinners out—all of which are crucial in proper management of the condition. “If I ever had a question, I was only a phone call or an e-mail away from the nurse research coordinator, and she’d get right back to me,” he says. “I was thrilled with the amount of information I got out of the study, to help myself down the road, to be prepared. If I ever have to go on insulin permanently, I won’t be nervous at all.”
There’s also evidence that people who receive treatment at centres with a thriving clinical trial culture fare better than their peers, even when they’re not enrolled in a study. This may be due to the extra training people who conduct clinical trials have to take, as well as to the fact that investigators involved in a positive trial can quickly apply their findings to treating other patients, Dancey says.
All of these factors may partially account for another benefit of participating in trials. Research suggests that, “even if they’re in a placebo group, people tend to do better in a study than if they’re not in a study,” says Ross Tsuyuki, director of the Alberta clinical trials and health services research centre EPICORE. “Part of that,” he acknowledges, “is probably because people who go into studies are typically more interested in their health, so they do better to begin with,” but he says that the process of reviewing the risks and benefits of the study (as a prelude to providing written consent) may also help. “You’re going to learn more about your condition because we have to tell you about it, so you become even more engaged.”
To be fair, there can be downsides to participating in studies. For one thing, not everyone is willing to accept being randomly assigned to one treatment or another.
“Some people can’t live with that uncertainty, even if it’s a 50 per cent chance of getting something better than current treatment,” the Canadian Diabetes Association’s Dr. Hux says. And with new medications and devices that haven’t been broadly tested, “there’s the possibility treatment won’t be helpful or may actually be harmful,” she adds.
However, by the time a treatment reaches Phase III trials, quite a bit is known about its potential risks and benefits—and these tend to be laid out explicitly in the consent form. Trials also have to be approved by an ethics review board, and in the case of new drugs and devices, any significant events must be reported to Health Canada; the agency will also do inspections at trial sites to ensure regulations are being followed, Dancey says. “So it is an experiment, but it’s one that’s designed to try to ensure that patients are safe and have the best chance of a good outcome.”
Participants must also accept that—depending on the study and such factors as the distance between the study site and subjects’ homes, as well as the frequency of follow-up visits—being involved in a trial can be time-consuming.
“It can be an additional burden that people may or may not be willing to take on,” particularly during cancer treatment, says Sian Bevan, vice-president of research of the Canadian Cancer Society Research Institute (CCSRI) in Toronto.
And in some cases, trial participants incur financial costs that may not be reimbursed. “You can pay for parking and some travel expenses specific to the study, but you can’t remunerate people for things like lost wages because it would be considered an inducement, which might not be considered ethical,” Dalhousie’s Dr. West explains.
But let’s say the potential risks haven’t dissuaded you and you’re interested in the possibility of participating in a study. Before making a final decision, be sure to ask lots of questions so you understand as much as possible about the commitment you’re making—from nuts and bolts details such as how many visits you’ll be required to make and what kinds of tests will be involved to broader issues such as why researchers believe the new treatment will be effective and how it differs from current therapies or standard care.
There are several resources to help you come up with a list specific to your situation: itstartswithme.ca (see the “Getting Started” section) is a good place to begin. The site features a wealth of clearly written information on many different aspects of clinical trials, including a glossary of terms.
Richard Smith, who blogs for the BMJ (formerly British Medical Journal) website, also has some recommendations, including asking about who’s funding the study and whether researchers will receive a fee for enrolling participants or have other financial conflicts of interest, such as a patent. (Search for Smith’s name on blogs.bmj.com/bmj: the relevant entry is dated October 21, 2016.) It may also be worth getting input from your family physician, specialist, and family members before you decide. It’s also important to know that you retain the right to withdraw from a study at any time.
Jack Shore of Port Colborne, ON, is one person who’s grateful that people are willing to enrol in clinical trials.
Three years after surgery and radiation treatment for prostate cancer, his cancer recurred, so his oncologist recommended hormone therapy. For the type of cancer Shore has, these injections are typically administered every three months, indefinitely. Having researched the side effects, Shore was less than enthusiastic about the therapy, so he consulted his original surgeon, whom he’d been seeing every three months for follow-up testing. This doctor was closely tracking an ongoing clinical trial of intermittent hormone therapy and offered to try the approach on him.
After his first two shots, Shore was in the throes of miserable side effects, including fatigue, breast tenderness and enlargement, weight gain, muscle loss, and debilitating hot flashes—but his PSA level had dropped considerably (a high PSA level can indicate prostate cancer). Shore and his physician therefore decided to stop the injections until his PSA began to climb again. Consequently, rather than enduring continuous treatment, so far, Shore has had to undergo only one more two-shot round. That was three years ago.
Where Can I Find Out About Ongoing Studies?
- Ask your family physician or specialist.
- Call your local hospital or community health centre.
- Contact the nearest medical school or research institute.
- Check with charities that fund research on a particular health condition, such as heart disease or ovarian cancer.
- Check one of the following online registries: