Introducing Clinical Trials: Safety Information to Consider

At myTomorrows, every day we speak to patients and caregivers who are on their unique treatment discovery journey. They are seeking to understand which options are relevant, and for many, this means researching clinical trials. That’s why we’ve pulled together information we think people will find helpful.

This blog focuses firstly on what a clinical trial is and, key safety information to consider. Part one of a three part series on Introducing Clinical Trials, you can read the other two here, ‘Phases explained‘, ‘How to find clinical trials for cancer‘.

A clinical trial is a type of research study where the effects of a medicinal treatment, medical device or other intervention are evaluated in people who volunteer to take part. The aim is to understand how safe and effective a treatment is for an intended health condition, before making it available to the wider public. In addition to treating a disease, clinical trials also study new methods for disease diagnosis and disease prevention.

Watch this short video which explains more about what a clinical trial is.

What safeguards are in place for clinical trial participants?

• Clinical trials are conducted in accordance with strict and internationally harmonized rules and law.
• Government organizations set rules and monitor clinical trials.
• Clinical trial protocols are reviewed by doctors, scientific experts and other people whose job it is to ensure participant safety.
• Participants are monitored closely for possible side effects.

Clinical trials are preceded by laboratory studies

Before a medicinal treatment even makes it to the clinical trial stage, a good understanding of how the investigational drug may work has already been acquired from laboratory experiments using animals or cultured cells. This preclinical research provides an indication of the potential benefits and risks of the investigational drug. Clinical trials occur in stages, starting will small numbers of participants at first and then later moving to larger studies once a basic sufficient indication of safety and efficacy safety is demonstrated in this early stage.

Informed consent

Before joining a clinical trial, there is a process of informed consent whereby potential participants must be given important information. Through informed consent, they receive information about how the clinical trial will proceed, including possible risks and benefits, so they can decide whether to take part. The process of informed consent includes a member of the research team explaining possible side effects. The person considering volunteering for the clinical trial will have a chance to have all of their questions answered.

If the person decides to participate in the clinical trial, they will be asked to sign a consent form that says they have been informed of the details about the study and choose to take part. Informed consent is not a contract. Clinical trial participants can change their minds and leave the study at any time for any reason.

Informed consent does not end when a person enrolls in a clinical trial. Throughout the clinical study, they must be informed of new information that may affect their decision to continue participating. If a person’s medical condition worsens or changes, they may wish to leave the study. There may be other reasons that a participant decides to leave, and they are not obligated to explain their decision.

Questions to ask before joining a clinical trial

People considering joining a clinical trial should talk to their doctor, clinical trial research staff or both about how their treatment options differ if they join or do not join the clinical trial. Here are some examples of questions potential clinical trial participants might ask.

1. What are my treatment options outside of clinical trials?
2. How is the clinical trial treatment different from the standard treatment?
   • Standard treatment is a widely used treatment that is accepted by medical experts as a proper treatment for a certain type of disease

1. What treatment will I receive if I am in the control group?
   • The control group in a clinical trial does not receive the treatment under investigation and is used for comparison. Depending on the clinical trial design, participants in the control group may receive a placebo or a standard treatment. A placebo is a substance or intervention that has no therapeutic effect but is administered in the same way as the treatment being investigated.
2. What are the risks and benefits of the standard treatment compared to the clinical trial treatment?
3. Why is this clinical trial treatment being studied or offered?
4. How will my privacy and personal information be protected?

Experts review the clinical trial protocol

A clinical trial protocol is a written plan or protocol, which explains in detail how medical tests, treatments, or procedures will be used in the clinical trial. The clinical trial protocol also explains what the study aims to accomplish and why. Details such as the number of people that will be enrolled, who is eligible, and how information will be collected are included in the clinical trial protocol.

Before a clinical trial can begin, the clinical trial protocol is reviewed by an independent expert committee. In the United States, this is done by an institutional review board (IRB). An IRB is comprised of scientific experts, doctors, clergy, and patient advocates. An IRB exists at every healthcare facility in the United States where clinical research takes place. The job of the IRB is to protect people who participate in clinical trials. The IRB checks that the study is well designed, legal, and ethical, that safety is taken into account, and that there are no unnecessary risks to participants.

Clinical trial safety monitoring

As the clinical trial proceeds, IRB members regularly review the results of the study to ensure that the risk of harm to participants is as low as possible. Clinical trial staff must monitor every participant by following strict rules. Rules for participant safety in clinical trials are enforced by the Federal Government.

Many clinical trials in the United States are supervised by a data and safety monitoring committee (DSMB), which includes members with expertise in biostatistics, clinical trials, and the disease and treatment being studied. This committee periodically checks in on the study results and if they find that the experimental treatment is not working or harming participants, they may suspend the study or recommend changes to the clinical trial. In addition to checking that the clinical trial is safe, the DSMB also makes sure the clinical trial provides doctors with useful information.

In the United States, clinical trials receive advice on ethical and regulatory issues and guidance on the clinical trial process from the Office of Human Research Protections (OHRP), which is part of the U.S. Department of Health and Human Services. OHRP protects the safety of clinical trial participants and ensures that rules are followed. Clinical trials are also monitored by the National Institutes of Health (NIH).

Clinical trials that involve experimental drugs or medical devices follow rules set up by the FDA. The FDA helps protect the rights, safety, and welfare of people volunteering in clinical trials and ensures clinical trials follow good clinical practice (GCP) regulations. GCP is the international standard for ethical and scientific quality in clinical trials.

Side effects in clinical trials

Clinical trial participants should talk to clinical trial research staff about what side effects they might expect. However, there may also be unknown side effects for new treatments. Besides minor discomfort, participants should be aware that complications or side effects can occur that require medical attention. Participants are closely monitored for side effects during clinical trials. If clinical trial participants are having unexpected or severe side effects, oversight committees such as the IRB and DSMB may decide to stop the trial. A clinical study may be stopped if the treatment is causing more harm than benefit to participants.

Conclusion

No clinical trial can be guaranteed to be completely safe for everyone. Safeguards that aim to help keep clinical trial participants safe include expert review of clinical trial protocols and continuous monitoring of clinical trials by government organizations. The rules and regulations that clinical studies must follow help to keep the risks to the participants to a minimum so that in most cases the benefits outweigh the risks.

We hope this blog was helpful when considering the safety information relating to clinical trials.

To learn more about possible clinical trials and get help finding clinical trials, schedule a call with a myTomorrows Patient Navigator. Find out more here.

The information in this blog is not intended as a substitute for a medical consultation. Always consult a doctor before receiving a diagnosis or treatment.

The myTomorrows team
Anthony Fokkerweg 61-2
1059CP Amsterdam
The Netherlands