Considering a Clinical Trial? Here’s What You Need to Know
When, how, and why to enroll
Alex Trebek, the beloved host of Jeopardy!, has been fighting a very public battle with Stage 4 pancreatic cancer since mid-2018. For a while, it appeared that his cancer was in “near remission.” But it returned late in 2019, and in an ABC News interview Trebek said, “We may try a new protocol, a different chemo, or something in the trial stage that is not chemotherapy… I don’t mind experimenting. I’ve got nothing to lose, so let’s go for it.”
The best time to consider a clinical trial — for cancer or any other medical condition — is immediately after your initial diagnosis, since many clinical trials require patients to be “treatment naive.”
While it’s highly likely that Trebek has been receiving the best of care and the latest, most effective proven treatments, his statement reveals a common misconception: that experimental treatments in the form of a clinical trial are only reserved for hopeless cases after all conventional treatments have failed.
Who should consider a clinical trial?
The best time to consider a clinical trial — for cancer or any other medical condition — is immediately after an initial diagnosis, since many clinical trials require patients to be “treatment naive.” This means they haven’t received any previous treatment for their condition.
But here’s the thing: Doctors are unlikely to mention the possibility of a clinical trial because oftentimes it’s simply not on their radar. And unless you’re being treated in a large academic medical center, even specialists are sometimes reluctant to mention clinical trials because they may worry that you’d take that as a signal that your case is hopeless. Additionally, your current team has a vested interest in discouraging you from participating in a clinical trial, since they’re likely to lose you as a patient.
Fortunately, you’re not solely dependent on your doctor’s ability or willingness to share info on clinical trials. It’s relatively easy for ordinary people to find and enroll in reputable clinical trials for a wide variety of medical conditions, from Stage 4 pancreatic cancer to (no kidding) athlete’s foot.
What do clinical trials do?
Clinical trials are intended to answer four questions:
- Is the new treatment safe?
- If so, is it effective against the condition it’s designed to treat?
- If so, is it better than existing treatments?
- And, if it’s safe, effective, and better than existing treatments, how does it perform in widespread use?
Those four questions correspond roughly to the four clinical trial phases called, reasonably enough, Phase 1, Phase 2, Phase 3, and Phase 4.
Phase 1 trials are intended to see how safe the potential treatment is and to determine the maximum tolerable dose of a new drug. The primary focus is not whether a new treatment works, and in fact many Phase 1 studies are performed on relatively small numbers of healthy volunteers. But some treatments are so toxic that it would be unethical to test them on healthy volunteers. In those cases, the investigators will look for patients who have exhausted all conventional treatments.
It’s relatively easy for ordinary people to find and enroll in reputable clinical trials for a wide variety of medical conditions, from Stage 4 pancreatic cancer to (no kidding) athlete’s foot.
Phase 2 trials are intended to test a treatment’s effectiveness on patients with the condition and to further refine what dosages are best. While Phase 2 trials can (and often do) benefit the participants, there’s no assurance that the investigational treatment is better than conventional treatments.
Phase 3 trials generally involve a large number of patients who are divided randomly into one or more treatment groups (taking different doses of the investigational drug, for example) and one or more control groups. You may be worried that you’d be randomized into a group receiving a placebo. But if your condition is serious, and there’s an already approved treatment (however imperfect), it would be unethical to give you a placebo. In that case, the investigational treatment would be compared to the best available existing treatment.
After a drug is deemed safe and effective in Phase 1, 2, and 3, and is approved for widespread use, pharmaceutical companies will often conduct Phase 4 trials, which are intended to determine how the treatment works — and whether rare side effects appear — in real-world conditions, when thousands of patients start taking it.
How do you find and enroll in a clinical trial?
If you’re interested in exploring clinical trials, your first stop should be Clinicaltrials.gov, which is maintained by the U.S. National Library of Medicine, a part of the National Institutes of Health (NIH). Clinicaltrials.gov contains detailed information on more than 327,000 clinical trials in 209 countries and all 50 U.S. states.
For all types of cancer, for example, more than 15,000 studies are actively seeking participants. And narrowing that down to clinical trials in North America actively recruiting patients with pancreatic cancer, on a recent day in January 2020, I found no fewer than 344 clinical trials to choose from. Those trials are recruiting patients in every state in the U.S. and many foreign countries.
In addition to listing the different current trials, clinicaltrials.gov provides pertinent details on which phase the trials are in and who they’re seeking. For example, take a look at the listing for the one U.S.-based athlete’s foot trial: You’ll see that it’s a Phase 3 trial of a new anti-fungal cream that’s being compared to an already-approved cream and to placebo. You’ll see what kinds of patients they’re looking for (the “inclusion criteria”) and what would disqualify a potential participant (the “exclusion criteria” which in this trial rules out pregnant women and people with a fairly long list of medical conditions). You’ll also find telephone numbers and email addresses of the investigators if you think you may be interested in enrolling.
Another good option for locating trials: You can call the NIH Clinical Center at 800-411-1222. According to Nikita Curry, MHA, supervisor of the office of patient recruitment, that number connects 40,000 patients a year to one of the Clinical Center’s information specialists.
“We look for your diagnosis under one of the settings that we have, and we use screening questions to determine if you’re a candidate,” Curry said in an interview with Elemental. “We use those screening questions to determine if you’re a candidate for the study… Then we send that information to the investigator. The investigator chooses if they’re going to reach out to that patient or not.”
The Clinical Center only conducts trials that are being sponsored by the NIH, which has a huge advantage: All treatment for those 1,500 NIH sponsored trials is conducted at the Clinical Center’s state-of-the-art hospital in Bethesda, Maryland, and any treatment you receive there is without charge. The center also has resources for family members of patients participating in their trials, including a limited number of lodging spaces for those who have traveled long distances.
How do you decide which trial, if any, is right for you?
There are several factors you should be aware of and consider:
First of all, you need to remember that if doctors were already certain the new treatment was better, they wouldn’t need to conduct a clinical trial. That means the experimental treatment could be worse than conventional treatment. And even if it’s more likely to cure your condition, the side effects could be worse.
If you enroll in a clinical trial, you’ll receive treatment from highly experienced specialists who will follow your condition much more closely than if you were getting the standard treatment. That’s great, but it does have a downside: You’ll probably have more frequent appointments and will be subject to more poking and prodding.
For most people, Phase 3 trials are likely to be best. Dangerous or ineffective treatments get weeded out in Phases 1 and 2.
Generally, the worse a person’s prognosis with standard treatment, the more that person may want to choose an aggressive and risky Phase 1 or Phase 2 trial aimed at finding a cure. If you decide to take an aggressive course, you may wish to look for a trial that’s a real departure from the ordinary, one using an entirely new approach to the disease rather than an old drug in a new dose or a new combination. Some people choose to participate in an early phase trial not out of hope of personal benefit, but to help others down the road.
You can bring up the idea to join a trial with your current medical team and they may help you in the search, or they may not. And while you certainly don’t need your current team’s blessing, you should discuss your decision to participate in a clinical trial with them; the trial team will need to see your medical records and they may wish to discuss your case with your current physicians. Even if your current team of doctors opposes your decision to participate in a trial, they can’t refuse to turn over records. Depending on the details of the trial, you may be able to continue receiving some treatment from your original team, or you may have to receive all your treatment from the clinical trial team.