Clinical trials help researchers identify and refine better treatments
By Deborah Jeanne Sergeant
Every pill, vaccine and therapy prescribed to you by your medical provider once underwent a clinical trial to prove its safety and efficacy. While it is reassuring that the process exists, most people know little about how it works.
“Clinical trials are so important. This is how we advance healthcare. This is how we improve the quality of people’s lives. This is how we save lives: by these advancements,” said physician Tim Murphy, SUNY distinguished professor and director of UB’s Clinical and Translational Science Institute, and a professor in the Division of Infectious Diseases, Department of Medicine in the Jacobs School of Medicine and Biomedical Sciences.
Researchers must carefully select volunteers who fit a profile of the patients that the new drug or treatment would affect. Other factors of eligibility may include absence of certain conditions, age, gender and more.
Murphy said that people lacking good access to healthcare are often those hardest to recruit for trials, such as those underinsured, living in rural areas, older adults, minorities and people economically disadvantaged. Ironically, some of those people are often most benefited by participating in clinical trials.
“If you look at people who participate in clinical research, they have better outcomes and cost-effective care compared with those who don’t participate,” Murphy said. “Let’s say someone’s being studied for one disorder and at their regular visits, they have their blood pressure checked. The research coordinator might say to contact their primary care provider.”
For some medication not covered by insurance, participants in a trial for a new medication of that sort would receive free treatment—and even receive a stipend for their time.
“Trying to engage people who have traditionally not been engaged with clinical trials is a particular interest of mine,” Murphy said. “If we’re going to introduce new medications and treatments and if we test it only in Caucasians, we won’t know how it works in other ethnic groups. It’s important to know if it works in the national community.”
Trials are usually conducted in six steps: approval of the research protocol, screening, informed consent, data collection, study closure and reporting of findings.
Brent said that an institutional review board reviews every trial conducted in the US. The board is comprised of experts who look at the risks and benefits of the study to protect those participating.
Volunteers are screened for their eligibility. The informed consent ensures that the participants understand the study. As the study ensues, researchers carefully monitor patients. In some cases, they remain at a healthcare facility. In most cases, they participate in a number of visits to the facility so researchers can gather information. When the visits are done, the clinical trial is over.
Trials typically research in phases. The earliest phase is in a lab petri dish and then with animals. The first human trials use only a small, healthy group and focus on safety. As the phases continue, more individuals are added to the group and some receive the treatment and others (the control group) do not. This allows researchers to fairly compare the efficacy of what they are testing. As the phases continue, larger groups become involved, which can help prove efficacy among a variety of people.
Some groups are difficult to study for ethical reasons, such as pregnant women and babies.
“In almost every clinical trial, an exclusion for a new drug is almost always pregnancy,” Brent said. “Things are tested extensively on animals, but pregnant women are excluded on nearly every trial including drugs. Some drugs can have toxicities in pregnancy that we don’t know about and can be toxic for the fetus. It’s almost a staged process where we know drugs are effective and safe in a non-pregnant population and only after that is it tested in pregnant women.”
Older adults are also challenging to study because they may have a long list of medications and health issues that could skew the results if their group size is too small. But it is also important to accurately represent their demographic which statistically does have more health issues than younger people.
“The elderly are an important part of our community,” said Adam Larrabee, president of Rochester Clinical Research. “They need to be represented in clinical trials.”
He reassures anyone interested in volunteering for a study that “you’re not a guinea pig. Participating in a study is a positive. experience. We’re not a typical doctor’s office. You don’t come in and wait half an hour or an hour to see someone. We realize people are contributing their time to promote science. We realize their time is valuable and we see them right away.”
He views participating in studies as an opportunity to receive healthcare as well as experience the satisfaction of contributing to health improvements that will affect generations of people.
Medical studies are also highly regulated.
“It’s come a long way for safety protocols,” Larrabee said. “Patients are very closely monitored throughout their participation and their primary care providers are kept in formed. We’re not replacing anyone’s doctor but working with them. They’re human beings contributing to the advancement of new medicines. Medications are very, very expensive. And there are a lot of people who are underinsured. It’s expensive to pay for a medication. If you can get that through participating through a study in addition to receiving closer medical surveillance, why not?”
Look for clinical trials at www.clinicaltrials.gov.