Clinical Trials- Navigating

Clinical Trials – An Overview

Hearing the words “you have cancer” may be some of the most frightening words to hear from a doctor. Even scarier are the words, “Your child has cancer.” All at once, your role as a parent expands beyond caregiver to nurse, advocate, medical decoder, and more. In a world of increasing complexities in the healthcare setting, this simple truth remains – always listen to your gut.

We live in a time where more treatment options are available than ever before. Most often, your doctor will prescribe a tried and true drug, walk you through the expected side effects, provide timelines for results, and share statistics on success rates. This is especially true for cancer drugs. But what happens when your doctor brings up a drug still under investigation? In this post, I hope to equip you with the knowledge that gives you confidence to make treatment decisions for your child and trust your gut.

Your initial reaction to the word “trial” might be “no way” or “guinea pig.” Even without bias, clinical trials carry a stigma. To help reduce that stigma, let’s look at a bit of history.

Clinical trials have existed for centuries. In the past, people didn’t call them clinical trials, and subject safety wasn’t a primary concern. However, since the mid-1900s, efforts have ensured that patient consent and safety take center stage in clinical trials.

In 1964, the World Medical Association adopted the Declaration of Helsinki (1), a significant step forward in establishing parameters for conducting clinical research. Since then, more specific guidelines have been put in place, such as those from the International Council for Harmonization (ICH) and the Good Clinical Practice (GCP) Guidelines (2). The ICH brings together regulatory bodies and pharmaceutical companies from different countries to establish safety guidelines for subjects (3). The GCP guidelines, based on ICH requirements, provide more specific protocols (4). In the U.S., clinical trials are also governed by the Code of Federal Regulations (5) and regulated by the Food and Drug Administration (6).

Locally, an Institutional Review Board (IRB), also called an Ethics Review Board, governs clinical trials. The IRB ensures that physicians conduct trials in the patient’s best interest. Additionally, pharmaceutical company representatives routinely monitor local trials to confirm patient safety and ensure that physicians follow study protocols properly.

Over the past 60 years, researchers have worked hard to oversee the ethical conduct of clinical trials. Despite this progress, patients and caregivers often wonder if a clinical trial is the right choice. Deciding to participate in a clinical trial is much like choosing a standard treatment for your child.

What You Can Expect from Clinical Trails

When a physician approaches you about a clinical trial, they should explain the drug being studied and the risks and benefits involved. You will receive a copy of the Informed Consent document, which outlines study details, including risks, benefits, study visits, and potential costs. Before making a decision, ask as many questions as needed to feel comfortable. You or your child must sign the Informed Consent form before undergoing any procedure required by the trial. Signing the form indicates that you understand the trial and agree to participate. You should never feel pressured to join a clinical trial. Trust your gut as you make this decision.

So, how do you decide?

Some benefits of participating in a clinical trial include access to treatments otherwise unavailable, treatments that may work better than standard care, and closer monitoring through frequent tests and doctor visits. Additionally, by participating, you contribute to advancing cancer care for future patients (8).

However, there are potential drawbacks. Clinical trials may require more time for appointments and monitoring. There could be unexpected side effects, and extra costs, such as travel expenses for gas or parking (8). Insurance might not cover certain tests required by the protocol, though pharmaceutical companies typically cover these costs.

Understanding the Risks

Like standard treatments, clinical trials come with risks and benefits. Let’s first address the risks.

To better understand the risks, it helps to know the different phases of clinical trials:

• Phase I – These studies involve human subjects for the first time, aiming to find the highest dose tolerated without severe side effects. The potential side effects are often unknown. Phase I studies help determine the best way to administer the treatment (9).
• Phase II – Once a treatment is deemed safe, Phase II studies explore which cancer types respond to it. Goals in Phase II vary widely, from completely eliminating cancer to improving quality of life (9).
• Phase III – These studies compare new treatments to existing ones. You may not receive the new treatment based on randomization. In double-blind trials, neither the patient nor the doctor knows which treatment is administered. Phase III trials aim to demonstrate the new treatment’s effectiveness to secure FDA approval (9).
• Phase IV – These observational studies involve approved drugs, monitoring patients long-term for rare side effects and tracking survival rates (9).

Each phase presents different risks. Phase I trials, often used when no other treatment options work, carry more risks, as the goal is often to prolong life. In contrast, Phase III studies involve better-known risks, with the primary goal of comparing treatments. Many Phase III trials include a crossover option, allowing patients to switch treatments if the assigned one doesn’t work.

Now that you know more about trial phases, you can better assess the risks and benefits.

The Benefits of Clinical Trials

The benefits of clinical trials vary and may be subtle. In Phase I studies, the benefit might simply be more time with your child. Relief from symptoms may also occur. Phase II trials offer these same benefits while identifying which cancer types respond to the treatment. It can be exciting to learn that your child’s cancer type may respond to the new drug.

Phase III trials offer a clearer understanding of what to expect from the treatment, similar to standard-of-care treatments. By the time a drug reaches Phase III, years of study have provided substantial information about its effects. Phase IV trials ensure continued close monitoring, even after FDA approval.

In Conclusion

Deciding to participate in a clinical trial is a deeply personal choice and may not be right for everyone. Educating yourself is essential to making the best treatment decision for your child. The medical professionals involved in your child’s care are there to provide support and information so that you can feel confident. Don’t hesitate to ask tough questions, and take the time you need to make the right choice. As a parent, you know your child best—trust that. And always, listen to your gut. It will never steer you wrong.

References

1. wma.net/what-we-do/medical-ethics/declaration-of-helsinki/
2. ich.org/page/efficacy-guidelines
3. ich.org/
4. fda.gov/science-research/clinical-trials-and-human-subject-protection/regulations-good-clinical-practice-and-clinical-trials
5. hhs.gov/ohrp/regulations-and-policy/regulations/45-cfr-46/index.html
6. fda.gov
7. fda.gov/about-fda/cder-offices-and-divisions/institutional-review-boards-irbs-and-protection-human-subjects-clinical-trials
8. cancerresearchuk.org/about-cancer/find-a-clinical-trial/what-you-should-be-told-about-a-clinical-trial/advantages-and-drawbacks
9. cancer.org/cancer/managing-cancer/making-treatment-decisions/clinical-trials/what-you-need-to-know/phases-of-clinical-trials.html