Clinical Trials: Risks, Safety, and Patient Protections

Participating in clinical research comes with risks and benefits. Jamie Holloway explores the many safeguards that protect clinical trial participants.
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Is it Safe for Me to Participate in a Clinical Trial? Is It Worth the Risk?
Since the goal of the clinical trial system is to determine the safety and efficacy of a new drug or device, there is always a possibility that the new drug or device isn’t safe or won’t work, and thus could cause unintended harm to trial participants. Clinical trials are how researchers find out what will happen when a new drug is used in humans. So yes, there are risks. The question is not whether risks exist, but rather, it is how we deal with those risks.

Behind the Scenes: Minimizing Risk Before the Drug Gets to Patients
Some risks are minimized before the clinical trial ever begins. Before any investigational new drug is tested in the human population, years of work have gone into its validation. Many researchers have tested the effects of the drug on cells in petri dishes in a lab, and after conditions for its use have been optimized, it is tested in animals. Safety, efficacy, and dosage are determined in animals, and many times, they are validated in more than one animal model. Investigators have an idea of what dose levels will be safe and effective in humans, and they have an idea of what side effects they might expect.

When the drug is ready to graduate to human clinical trials, the trial sponsor must submit an Investigational New Drug application to the FDA and the study protocol must be submitted to an Institutional Review Board whose primary role is to ensure participant safety.

Front and Center: Minimizing Risk During the Research Study
In addition to the behind-the-scenes steps to minimize risk, some of the things that protect participants in a research study are more obvious.

  • The participant needs to understand the risk.
    • The informed consent document that the participant is required to review and sign should list any specific risks that the investigator considers a possibility. It should also inform the participant that it is possible that there could be additional harms that are completely unanticipated. No participant should ever feel like they were taken advantage of and were subject to being harmed without understanding the risk.
    • The researchers should make sure that the participant has their contact information and feels welcome to reach out to the  study team with any questions or concerns, both about potential risks before agreeing to participate, and actual problems they may encounter while part of the study.
  • The risk should be minimized to the fullest extent possible.
    • Study design should reflect the breadth of knowledge about an investigational agent. When studying a drug in humans for the first time, in a phase 1 trial, investigators start with a dose that is much lower than what could safely be used in animals, and they increase the dose very gradually as they see that there are no unacceptable harms to patients. Once a drug is shown to be safe at a certain dose level, it can be given to a larger number of patients with the confidence that it poses them minimal risk, and so in phase 2, 3, and 4 trials, the dose given to patients reflects what investigators learned was safe in phase 1. Investigators also build on what was learned in earlier phases to find the dose or treatment schedule that is not only safe, but most effective in treating the disease. Learn more about the phases of clinical trials here.
    • Participants are closely monitored. Since there is uncertainty about the incidence and severity of specific side effects that participants might encounter, they are very closely monitored by the study team. This may mean more trips to the clinic, more blood draws, more check-ins with a nurse, or a clinic staff-member who is more responsive when a participant calls with concerns or questions.

Benefits of Participation
Understanding that there are risks when participating in a clinical trial is crucial. However, there are many safeguards built into the system to minimize these risks and the impact that any unintended harm can have on a participant. Having a full understanding not only of the risks, but also the safeguards and the potential benefits of participating in clinical research, allows patients to make a truly informed decision with the confidence that even if they do not directly benefit from the clinical trial, they are leaving a legacy of improved care for future patients.

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