Without clinical trials to test drugs and medical devices, we wouldn’t have the medications available to us today. While not without risk, certainly no one expects to die during a trial from an experimental drug.
Unfortunately, that’s exactly what happened in January 2016 in Rennes, France, when a botched clinical trial left one participant hospitalized and declared brain dead.The trial was conducted by Biotrial, a French research company contracted by the Portuguese drug manufacturer, Bial, to test a new drug for Parkinson’s and other disorders. The participant died a week later, and 5 other participants were also hospitalized.
What happened? A French government report uncovered 3 major errors that, according to Science magazine, created a perfect storm for medical disaster.
Hope for debilitating conditions, desperation over terminal illness, or simply earning a few extra dollars on the side are all factors that drive people to seek out clinical studies. The latest figures from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, shows the website “receives an average of more than 207 million page views per month and 65,000 unique visitors daily.”
Before you give yourself over to science, follow these general rules to know what you’re getting into:
1. Do Your Homework
There are currently more than 210,000 registered clinical studies on ClinicalTrials.gov, including both interventional and observational studies, and more than 37,000 studies recruiting participants being conducted in 50 states and 192 countries. (Up to date info, including studies currently accepting participants, is available on the site.) There is also a list of important questions would-be participants should ask before enrolling.
Researchers are required under a 2008 federal law (pdf) to publicly disclose study results on the site, but that doesn’t always happen. According to a 2015 investigation by STAT, a health and medicine news site, most research institutions, top universities and major hospitals — not to mention drug companies — routinely break the law. For that reason, it’s smart to reach out to the researchers directly, asking for the results of any previous such studies they’ve conducted on the drug. By law, that information should be made available to you.
Be sure the trial you’re interested in is registered. What that means: Under FDA regulations, responsible parties are required to register their clinical trial and submit their results with ClinicalTrials.gov.
If a trial isn’t registered, the government has no way of finding out about it, which is why companies get away without registering. If the results are not going well in an unregistered trial, the company can stop the trial and never report it, keeping negative information about a potential drug secret. Unfortunately, that’s important information, as much is learned from a failed trial as a successful one. And those patients subjected to the failed trial don’t even have the comfort that their sacrifice helped science.
2. Understand Trial Phases
Clinical trials are done in phases, including Phase 1, 2, 3, and 4. These test a drug’s efficacy, dosage, safety, and side effects. Phase 1 is the first time human volunteers are given the drug or biologic being tested — before that, testing is done on animals. (The Biotrial drug in France, for example, had been tested on mice, dogs, monkeys and rats without any toxicity.)
Phase 1, which tests a drug’s safety, has the potential to be the riskiest for participants, all of whom are healthy. A 2015 meta-analysis of the risks of Phase 1 studies, not related to cancer trials, found that the most adverse events, 27.4%, happened in neuropsychiatric studies, followed by studies of infectious diseases, at 20.9%. The majority of the adverse events were mild and the studies were allowed to continue without any additional treatment/ However, in a very small percentage the study had to be temporarily or permanently stopped — in some cases, requiring additional treatment for volunteers to address the adverse events.
3. Get Your Doctor’s Advice
Before you participate in a clinical trial, discuss it with your doctor. If you are suffering from an illness or disorder and think a clinical trial would be beneficial, your doctor can help you decide which clinical trial may offer the best hope. Your doctor can also help steer you clear of trials that may worsen your condition, especially if the trial requires you to forego your current course of treatment.
If you are a healthy individual interested in a clinical trial for the financial stipend it provides, your doctor can also help determine whether the trial would be a wise decision given your current state of health.
According to ClinicalTrials.gov, “While most clinical studies provide participants with medical products or interventions related to the illness or condition being studied, they do not provide extended or complete health care. By having his or her usual health care provider work with the research team, a participant can make sure that the study protocol will not conflict with other medications or treatments that he or she receives.”
4. Find Out About Your Care
Remember, while researchers running trials are aiming to produce a better, more effective treatment, that doesn’t mean that during the trial your personal health is always their primary concern. Ask these questions to determine the level of oversight of your health during the trial:
- What type of long-term follow-up care is part of this trial?
- If I benefit from the intervention, will I be allowed to continue receiving it after the trial ends?
- Who will oversee my medical care while I am participating in the trial?
- What are my options if I am injured during the study?
If the study doesn’t provide adequate oversight, and you still want to enroll, ask for guidelines to help your own healthcare provider follow your health.
5. Read the Fine Print
To enroll in a clinical trial, you will be asked to sign an informed consent document. It’s designed to protect the researchers from liability, so read the fine print — especially about who will pay for your long-term treatment and care in the unlikely event you are seriously injured during a trial. This document provides information on the study’s potential risks and benefits, and other alternatives available. You may also want to show this document to your doctor for his or her expert opinion on whether you should proceed. You may also be required to attend pre-trial question and answer sessions or seminars so that you fully understand what will occur.
That said, it’s not a binding contract; You can drop out at any time, even if the trial isn’t over. In fact, you should ask whether you’ll be routinely updated of adverse events which may change your mind.
Biotrial had such a statement in its informed consent documents. Yet, the Biotrial researchers failed to alert the other participants that someone had been hospitalized, curtailing their right to informed consent. On the morning the volunteer was hospitalized, the remaining 7 volunteers were still given their daily doses of either the experimental medicine or placebo, without researchers first finding out the status of the hospitalized patient.
6. Tell the Truth
Each clinical trial comes with its own set of prerequisites for participants to enroll in the study, also known as the “eligibility criteria.” The trial may require pre-trial testing, but may also rely on you to simply tell the truth about your health, medical history and other factors.
So, if you’re a former smoker applying for a trial that requires only previous non-smokers, fess up. Otherwise, you’ll not only waste your time and theirs, but, if you enrolled for monetary reason, at the very least, you may have to kiss that stipend goodbye. Withholding information about your health history, or that of immediate family members (which may signal you’re genetically at risk for certain conditions that the clinical trial may be trying to avoid), may lead to serious outcomes for your health.
7. Don’t Get Your Hopes Up
To be blunt: You might not get the therapeutic drug after all. Some participants in clinical trials are given a placebo, or sugar pill, to help the researchers determine whether the drug or biologic they are testing is actually doing what they think it’s doing. Placebo-takers are in what’s called the control group, but these participants often aren’t told that’s the group they’re in. So while you may be hopeful that you are getting a promising new drug to cure what ails you, you might not be after all.
8. Consider All the Requirements
Clinical trials can be taxing. Phase 1 trials may last several months. Phase 3 trials may last one to four years. Some trials require daily check-ins; others mean you’ll be going to a specified location for daily or weekly testing. You may even be required to be hospitalized during the trial. If needles or keeping a journal aren’t your thing, then trials that include those might not be for you. Figure out the total time commitment, and other requirements, before you say yes.
As the French study debacle tragically shows, there are many unknowns in any clinical trial. And yet participating in one can be a literal lifesaver or life-extender for, to use one example, cancer patients who sign up to receive experimental treatments. Just be sure, if you or a family member is looking at trials, to do your own investigation first.
- The Life of a Professional Guinea Pig (The Atlantic)
- The Deadly Corruption of Clinical Trials (Mother Jones)
- Do Clinical Trials Work? (New York Times)
- French Debacle Renews Concern About Clinical Trial Secrecy (STAT)
Rita Colorito specializes in covering health, safety, and caregiving for both people and pets. Her work has appeared in dozens of publications and media sites, including SELF, Costco Connection, Bankrate.com, Prevention.com, and Edmunds.com. A hockey/soccer mom, Rita lives outside Chicago with her husband, son, and three shelter cats.