In the world of science, clinical trials are important. They’re important because they’re required as part of ensuring patient safety as new treatment options make their way from the lab to pharmacies around the world. But at a broader level, clinical trials are important because they are the lifeblood of medical innovation in modern times. They’re the driving force behind the advances that allow people to manage their diabetes and be vaccinated for polio as well as countless other breakthroughs.
Okay, so they’re important…but are clinical trials safe? Maybe you’ve heard awesome stories that make you want to help. Maybe you’ve heard crazy stories that make you want to steer clear of them. If you or a loved one is thinking about participating in a trial, read on to learn about important safety considerations and answers to common concerns.
Testing on Humans vs. Animals
“Unfortunately, ‘guinea pig’ has entered our vernacular for those who don’t know much about clinical trials,” says Belinda Tan, MD, PhD, chief medical officer and co-founder of Science 37, a clinical trials company that allows volunteers to participate in research studies from home. She recalls a Time magazine cover that equated guinea pigs with human participation in clinical trials. But that connotation, Tan adds, is not a reflection of the reality of clinical trials. While there are inherent risks in human research, positive participant outcomes are the top priority for all involved, Tan says, with all aligned to achieve a common goal: ensuring trials are designed and executed as safely as possible.
If you’re concerned about being a guinea pig, just remember that a new treatment is tested on animals before it is used on humans. Let’s use a new acne drug for example. The drug developer, or sponsor, has to submit an Investigational New Drug application to FDA before any clinical trial begins. This application must include animal study data and toxicity data to show that the researchers have done their due diligence before using the investigational drug on humans. Then the real clinical research begins.
“All of the learning that happens in that span of time between animal testing and human testing gives us that confidence that we know all of the safety issues to look for and control for,” says Tan. “There’s so much rigor behind how the trials are phased [by FDA] — all of these phases are designed to protect human research participants from unnecessary harm.”
Understanding the Risks
Okay, so the trial phasing provides some reassurance, but as a potential participant, you might worry about experiencing side effects or other serious issues while participating in a trial.
That’s totally understandable. In many trials, participants are engaged in new, novel approaches to treatment that don’t always go as planned; as a result, there could be serious or life-threatening side effects. Unfortunately, the potential presence of these side effects has driven the perception that participating in a clinical trial is risky, as illustrated by a number of reports (despite some of the enterprising people who take part). Tan says this is why FDA and other regulatory agencies require that participants be informed — without bias, in a language they can understand — about all of the risks beforehand.
The process for reporting and documenting serious side effects will vary by trial, but all serious problems should immediately be reported to the study team.
So how do you decide whether contributing to science is worth it? As a potential trial volunteer or caregiver to one, seek out as much information as you can about a study (starting with the trial listing on ClinicalTrials.gov or the trial website), the level of risk that it presents, and assess your own level of comfort with the stated risks. All studies will give you the chance to discuss questions and concerns about the trial directly with the research staff.
The Benefits of Participation
Tan and her colleagues have discovered that clinical trials offer “tremendous value” in some cases simply because they provide volunteers with hope, even if the participants understand that the treatment might not do anything (especially if they receive a placebo). For others, it’s the altruistic aspect of contributing to science and helping others. Sometimes, it’s about the quality of care.
“Many volunteers feel like they get more attention and better care when they’re part of a clinical trial. The trial staff is very attentive and there for you, and that might be different than leaving a message for your primary care physician and waiting for a call back,” Tan explains. “Plus, and I know this tends to be the elephant in the room, people like getting compensated to do something.”
Other Safeguards for Participant Safety
If you choose to participate in a clinical trial, you won’t be on your own when it comes to safeguarding your health. U.S. clinical trials are approved and monitored by an institutional review board (IRB), which Tan says ensures “that all the decisions we make keep patient safety and choice in mind. The IRB also ensures ethical conduct in research.”
FDA adds another layer of protection for participants. It defines the rules so that governing bodies, such as an IRB, are involved, and it also makes sure that the rules are being followed, that the scientific questions being posed by the study make sense, and that the study design makes sense.
There’s also a growing movement to increase transparency around clinical trial data so that participants can have greater ownership of their part in the process. FDA has taken gradual steps to support this movement over the past several decades — first by requiring the registration of clinical trials on clinicaltrials.gov (1997) and then by requiring a reporting of summary results (the Final Rule for Clinical Trials Registration and Results Information Submission (42 CFR Part 11) was released in September 2016).
Although these measures don’t guarantee safety, they should provide reassurance that participants are not alone.
Moving Science Forward
These developments underscore that clinical trials, and the results that come from them, are valuable. You may not know what to expect in a clinical trial as a participant, but those working behind the scenes are working toward a common goal: to better understand your health and how a given treatment may help you, and work to improve outcomes for you and others with your condition.
“That sense of purpose is quite powerful,” says Tan. She’s touched by it, and hopes potential participants are as well.