Are you considering taking part in a clinical trial?
If so, you are probably already aware that clinical trials are a vital step for new drugs trying to make it through the FDA’s rigorous approval process1. Most likely, too, you know that for numerous patients, these novel drugs provide the opportunity for improved, possibly life-saving management of their illness.
Those are two pretty good reasons to say “Yes!” to being a part of a clinical trial. What holds most people back, however, is the question of safety.
In this article, we will assess how safe clinical trials are by considering both the measures in place to protect clinical trial volunteers and the possible risks volunteers face. By weighing both, you will see that clinical trials are actually quite safe for their participants.
The levels of safety and risk are somewhat related to the phase or stage of the clinical trial you participate in. For this reason, we will start off with an outline of each phase.
Phases of a Clinical Trial
A clinical trial involves the testing of a new drug, procedure or device on groups of human patients.2 The trial will only be conducted when there is sufficient reason to believe that the treatment may be an effective way to improve patient care. The clinical trial then proceeds in phases. Data gathered from one phase is analyzed before the trial progresses to the next phase.
Like many other sources, both the World Health Organization3 and Cancer.net4, the patient information website of the American Society of Clinical Oncology, describe four phases (I – IV) of a clinical trial.
Quite often, however, there is mention of an initial, optional stage referred to as Phase 0, as seen at ClinicalTrials.gov5, the clinical trials registry administered by the U.S. National Library of Medicine at the National Institutes of Health.
Here is an outline of each of the five phases.
A very small dose of the drug is given to a group of between 10 and 15 volunteers. The investigators (or researchers) gather information to determine how the body processes the drug and the effects the drug has on the body.
Patients in Phase I are started on very low doses of the drug and the amount increased slowly. The aim of the investigators is to pinpoint the best dose of the new drug – the amount of the drug that is both effective and carries the fewest side effects. Phase I usually has between 20 and 80 volunteers and lasts anywhere from a few months to a year.
Phase II can last up to 2 years and is carried out to further verify the safety of the drug. The investigators may try different dosages of the drug on different groups of volunteers. Intense monitoring is done to identify any adverse effects the drug has. This phase typically involves 100 to 300 participants.
This is usually the last phase before a new drug is approved. The number of patients now ranges from 1,000 to 3,000. They are drawn from a wide cross-section of the population, varying in age, gender, and ethnicity. The drug is compared to commonly used or standard treatments by dividing the participants into groups, usually without them knowing which group they belong to.
Did you know that the U.S. Food and Drug Administration (FDA) approved a record-setting 59 new drugs in 2018?6 A drug that receives FDA approval can be freely prescribed by doctors. For some drugs, however, the FDA may require investigators to monitor reported adverse events involving the drug and continue collecting information on its effectiveness. This is Phase IV. Some investigators may choose to conduct Phase IV trials even if the FDA does not require it.
7 Reasons Why It Is Safe to Participate in Clinical Trials
There are many safety measures built into clinical trials. These protect patients from harm and also protect the sponsors, investigators, and government from potential lawsuits. Here are seven ways in which clinical trials ensure the safety of participants.
1. You must provide informed consent.
Anyone participating in a clinical trial must be given sufficient information about it. The information usually includes the duration of the trial and the treatment being administered, as well as possible risks, side effects, and benefits of participating in the clinical trial.7 Prospective participants must also be given time to go over the information.
You are also quite free to ask questions about the trial to satisfy yourself that it is indeed something you want to be a part of. Furthermore, you can seek the advice of your primary health care provider and loved ones on whether you should participate. These persons may also suggest other questions you should ask first before signing up for the trial.
Furthermore, along with monitoring patient-response to the new treatment, investigators also let patients know of any new risks discovered as the trial progresses. So at all times, your decision to remain in the trial is an informed one.
Note: Children cannot provide informed consent. This is done on their behalf by a parent or guardian. What a child can give is informed assent to being a part of a clinical trial.
2. All patients are carefully monitored.
Investigators diligently look for signs of how the drug is working and for any side effects it may be causing. They also encourage participants to share any and all information they can on how they are affected by the drug.
Close monitoring helps to guarantee that the investigators can quickly identify if there is a need to adjust your treatment in any way. They may also decide that there are better ways to handle any side effects you are experiencing. At no point are they seeking to cause you undue discomfort or to jeopardize your safety.
3. There is a meticulous screening process.
Investigators must identify the eligibility criteria for participation in the trial before it begins. These criteria clearly outline who can and cannot take part in the clinical trial. The criteria help the investigators to identify persons from whom they can obtain the most relevant results.
The eligibility criteria are also a way to ensure your safety. That is because some of the criteria are based on your medical history, such as treatments you have received in the past and your current diagnosis. Past or current conditions or treatment may put you at risk depending on the treatment the clinical trial is testing.
You will not be accepted as a participant in the trial if the investigators feel that doing so exposes you to excessive or unwarranted risks.
4. You will be given standard of care.
The history of clinical trials is marred by incidents such as the disastrous 1932 – 1972 Tuskegee syphilis experiment.8 As an article from the Centers for Disease Control and Prevention points out, that trial was carried out without the informed consent of the participants and without them receiving the appropriate standard of care for their condition.
Thanks to modern legislation governing clinical trials, events such as the Tuskegee syphilis experiment will never happen again.9 Along with giving their informed consent, participants in modern clinical trials receive the standard treatment for their condition whether or not they are in the group being given the test treatment.
This is especially true when the participants are taken from high-risk groups, such as cancer patients. If there is the possibility that a participant will receive a placebo treatment, then they are duly informed.
5. Oversight is provided by independent bodies.
Sponsors (the source of funding for the trial) and investigators are legally bound to ensure your safety when you participate in a clinical trial. They are not the only ones in charge of ensuring your safety, however, should you decide to participate in a clinical trial. There are independent supervisory bodies, such as Institutional Review Boards (IRBs), within each state.
The role of an IRB (also called a research ethics committee) is to approve and monitor the clinical trial while ensuring that participants are exposed to minimal levels of risks.10 You have the right to ask the clinical trial sponsor or investigator if the trial was reviewed and approved by an IRB.
There are also regulatory agencies that routinely investigate clinical trials as a way to guarantee the safety of participants. Each country has its own regulatory body as shown by this informative page11 on the website of the National Institute of Allergy and Infectious Disease. Within the U.S, the regulatory body is the FDA.
6. You are free to withdraw at any time.
Participants in a clinical trial can decide to drop out if they feel their safety is threatened in any way. Furthermore, you do not need to give a reason for dropping out and you cannot be penalized in any way for your decision to stop.
Keep in mind that the informed consent form that participants sign is not a contract. That means legal action cannot be taken against you for opting out of a clinical trial. Sponsors and investigators take participant dropout rates into consideration when designing their trial.
Many persons who take part in clinical trials do report, however, that the experience was a positive one. In fact, according to a recent Healthline survey12, 86% of trial participants would be willing to participate in another clinical trial.
7. The trial is done in controlled stages.
As outlined in the previous section, clinical trials are conducted in phases with safety being a major concern at each and every stage. There is a thorough review of any instances where safety seems to be compromised and measures are put in place to decrease these risks both in the current and future phases.
4 Risks of Participating in A Clinical Trial
A clinical trial is a form of research into the action of a new drug. Because it is new, the drug is not yet fully understood by the investigators. This is the underlying reason for many of the risks which come with participation in a clinical trial. Here are four risks commonly encountered.
1. The new treatment may not work.
You may find that the new treatment has little to no effect on you. Although it may prove to be very beneficial to other participants, it may be deemed to be less effective in your case that standard treatment options.
2. The side effects may be overwhelming.
Despite careful patient screening and the use of small doses of the drug, you may be one of those persons who have serious adverse reactions to the treatment. These side effects can range from simply being very uncomfortable to negatively impacting your health.
Serious adverse events are most often reported in Phase I and Phase II clinical trials. In any event, the investigators will make the best decision as to whether to adjust your treatment or stop it completely. Both the sponsors and the investigators are obligated to report all serious adverse events.13
3. You may be part of the control group.
It is most likely that you will not have a say as to which group you assigned. This is especially true in randomized trials.14 Many persons take part in clinical trials so they can get early access and benefits from cutting edge treatment options. However, you may be placed in the group which is given only standard care.15
4. You may face time constraints.
The investigators need to closely monitor how you progress on the new treatment. This means you may be asked to come in for more tests and observations than you normally would do for your condition. It can lead to you having to take more time off from work and spending more time visiting the hospital or in your doctor’s office.
There are inherent risks involved in every clinical trial. Participants can rest assured, however, that there are safeguards in place to protect them. These include the precautions they take on their own, such as with informed consent and the ability to opt out of the trial at any time.
Sponsors and investigators are also accountable for the safety of participants. This is why they carefully screen applicants before acceptance. What’s more, the government plays its role by appointing regulatory bodies to oversee the operation and progress of clinical trials so as to ensure the participants’ right to safety is not violated.
All in all, since the safety measures outweigh the risk, participants can rest assured that clinical trial participation is a good and safe idea.
Clinical trial participants contribute to the advancement of medical science and the improved care of those in need, now and in the future. Clinical trial participation is, in this regard, a noble act. If you are interested in taking part in a clinical trial, you can reach out to Clinical Match for help finding appropriate trials you may be eligible to take part in.