Medically Reviewed by Jenny Blair, MD
Clinical trials represent a crucial series of steps for effective therapies to get to patients. These studies vet how well a therapy works and how much harm it might cause. They also help researchers establish the right dose and determine if certain patients are at greater risk for adverse events, especially people with vulnerabilities like kidney and liver disease.
Even though every prescription drug and many other interventions must go through this standardized series of trials before it hits a pharmacy shelf or rehabilitation room, myths about clinical trials abound.
Here, with a little help from Peter Higgins, director of the IBD Program at the University of Michigan, and Adam Cifu, professor of medicine at the University of Chicago, are six myths about clinical trials and the realities that patients, especially patients with IBD, need to understand.
Myth 1: All trials pair one patient receiving the tested intervention with one patient receiving a placebo (a mimic of the intervention lacking the active agent).
Reality: Many trials will involve 2 to 4 people receiving the intervention for every person getting a placebo, says Higgins. Trials in the later stages, nearer federal approval, especially may go this route. The cited reason is usually that it’s easier to attract patient participants if the odds that they’ll get the intervention instead of the placebo are higher.
Myth 2: Results from any kind of clinical trial are meaningful for patients.
Reality: Clinical trials come in different flavors, from “testing the waters” to involving huge populations of patients taking clearly defined doses of a drug. But not everyone can tell which kind of trial is which, especially in news stories about the findings.
“There is so much early research that gets out there these days,” Cifu says. He cites “hypothesis-generating” studies that may involve only a few people and no controls. Also not immediately useful for patients are the results of Phase 1 trials. These studies usually involve a very small number of people and are conducted largely to make sure an intervention doesn’t have any dramatic or disastrous safety consequences.
“Phase 1 trials really have no bearing on therapeutic decisions for patients,” says Cifu. Results that are most relevant for patients will come from Phase 3 or Phase 4 trials, which usually involve large numbers of patients and careful assessment of both the effect of the intervention and any safety concerns.
Myth 3: If the tested intervention helps patients in the trial with my condition, it will help me.
Reality: Higgins says that often, patients don’t even consider participating in trials until they’re quite ill. Thus, the intervention might show as much effect for people with less severe disease. The flip of that is also possible, though, says Higgins. Because sicker patients may be harder to treat, what works for them might work even better for the average patient.
IBD Reality: Cifu notes that generalizing to the IBD patient population is “incredibly complicated.” Their disease subtype, the kinds of treatments they’ve already had, and their stage of disease all can limit how much results of a specific trial apply to the broader population.
Myth 4: Clinical trials last for years, so if I am in the group receiving the therapy, I’ll get that treatment for a long time.
Reality: The early phase trials usually last only a few months. Later-phase, larger trials can last for up to 4 years, though. Some of these trials may go into an extension segment, says Higgins, where participants in the intervention group can continue on the treatment for a longer time.
Myth 5: When trials results are “positive,” that means the participants receiving the intervention all experienced benefit.
Reality: “I think it really is important for patients to recognize the number of participants who are not helped,” Cifu says. Even if a trial compares two interventions and finds that one is better, that doesn’t necessarily mean every patient should switch, he says. The results might show that 10% more people respond on drug 2 than on drug 1. That means only 1 in 10 people who switch from drug 1 to drug 2 will gain greater benefit, and the other 9 who switched will have nothing to show for it.
IBD Reality: In trials for IBD interventions, says Cifu, often the drug shows a benefit for only a fraction of patients. “We don’t have any drugs that go beyond maybe 60 percent,” he says. That means every patient should discuss such findings with their clinician to decide what might work best for them.
Myth 6: The outcome that the treatment affects is important for the patient.
Reality: Sometimes, a reported trial outcome involves a target that isn’t terribly relevant for a patient.
IBD Reality: Cifu gives an example of finding reduced C-reactive protein levels on treatment. C-reactive protein is a marker of inflammation, so sure, a drug that affects those levels might affect inflammation. But a patient can’t feel C-reactive protein levels rising or falling. On the other hand, a patient likely would be more interested in an outcome showing reduced hospitalization frequency or fewer episodes of bloody diarrhea.
Although how clinical trials are conducted is pretty standardized, each trial will have its quirks that distinguish it in some way. Important information to look for is how big the patient population is, which outcomes the researchers address, what the patient profile is, and how many people seem to have benefited compared to how many were treated.
And as always, ask your clinician if you have any questions about whether or not the results of a clinical trial are relevant for you. If you see an opportunity to participate in a trial yourself, you can keep this same information in mind as you consider joining up.
Jenny Blair is a writer and journalist covering science, medicine, and the humanities. She earned her MD at Yale University, then completed a residency in emergency medicine at the University of Chicago. After several years in practice, she transitioned to working with words and ideas full-time. Jenny has contributed to Discover, New Scientist, Washington Spectator, and Medtech Insight, among other publications. She lives in New York City.
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