You may have heard the buzz around clinical trials. Perhaps you’ve heard presentations on clinical trials. Or you may have heard talk of specific clinical trials. You may know of someone with a different type of cancer who tried a clinical trial as a “last resort.” Contrary to popular opinion, clinical trials may be considered at any time during your journey. Options range from first-line treatments for someone newly diagnosed to those who have already had multiple treatments. There are currently many clinical trials open for NET patients. This article explains why someone with NET may consider participating in a clinical trial.
What are Clinical Trials?
Clinical trials test the safety and efficacy of a treatment and strategies. They evaluate what works and doesn’t work. Clinical trials test to ensure a new treatment is safe and effective before it is brought to market and used in clinical practice. Clinical trials may also provide information about how drugs compare to each other.
Clinical trials are conducted in a series of steps called “phases.” Each phase has a different purpose and helps researchers answer different questions.
Phase 1: Researchers test a drug or treatment in a small group of people (20-80) for the first time. The purpose is to study the drug or treatment to learn about safety, side effects, and the maximum tolerated dose.
Phase 2: The new drug or treatment is given to a larger group of people (100-300) to determine its effectiveness and to further study its safety.
Phase 3: The new drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, and compare it with standard or similar treatments for the drug to be used safely.
Phase 4: After a drug is approved by the FDA and made available to the public, researchers track its safety in the general population, seeking more information about a drug or treatment’s benefits and optimal use.
5 Reasons to Participate in Clinical Trials:
Clinical trials help the NET community. You have the opportunity not only help to yourself, but also to help the entire community. If successful, clinical trials help advance treatment options for others. You can help bring new treatments to market so they are available to all.
Clinical trials can lead to important breakthroughs and discoveries. You can be part of shaping the future. You have the opportunity to contribute to moving the field of medicine into the future. (We all love moving the field of medicine into the future!)
Clinical trials are an opportunity to advocate. Your participation in clinical trials is a form of advocacy. Participating in clinical trials is especially important in rare diseases where there may be limited research and limited treatment options. Clinical trial enrollment shows that patients are interested and willing to participate.
Clinical trials must have inclusivity and diversity. It is important to include people from all backgrounds including race, gender, and age to give a better understanding of how treatments affect different people. This helps reduce biases, promote health equity, and produce more innovative science.
Clinical trials may help you.* Clinical trials may give you access to new and promising treatments or combinations to see if they will work better than the standard of care. Depending on the phase and type of trial, you may receive the study treatment or the current standard of care. When you enroll in a clinical trial, you may also have extra medical attention from the research team with frequent check-ups and monitoring as part of your treatment.
*The hope of a new or promising treatment often attracts people to clinical trials. While patients often seek clinical trials for their own personal benefit, it is important to clarify the difference between clinical care and clinical research:
The primary aim of clinical care is to benefit the patient.
The primary aim of clinical research is to gain new knowledge. The patient may or may not benefit. The benefit may be for future patients and for the NET community as a whole.
Clinical trials drive medical advances. Clinical trial participation is essential to help us find answers and new treatments for NETs.
In order for a randomized trial to go forward, each arm of the trial must be balanced, or have “equipoise,” meaning that there is a state of genuine uncertainty regarding the benefits or disadvantages of either arm of the trial. It would be unethical for a trial to not have equipoise. All clinical trials regardless of phase must be reviewed by an Institutional Review Board (IRB) to ensure that those that are entering clinical trials are protected. To read more about IRBs, please refer to the FDA description of IRBs and the Protection of Human Subjects in Clinical Trials.
For randomized phase 2 or 3 trials, patients sometimes wonder if they can drop out of the trial if they are randomized into the control (standard of care, non-experimental) arm. To do so would waste costly clinical trial resources and could delay or halt the trial from completion. Without randomized clinical trials, new treatments could not come to market. So, while there is hope that the clinical trial may benefit the patient, the bigger reason to participate in the trial is to offer hope to the whole NET community. This is because clinical trials offer opportunities to help researchers find better treatments for others in the future.
Clinical trials drive medical advances. Clinical trial participation is essential to help us find answers and new treatments for NETs. Consider how you might help the NET community by participating in a clinical trial.
NET patient Mary shares why she participated in a clinical trial
Watch “Clinical Trials” featuring medical oncologist Dr. Daneng Li of City of Hope
Watch “What’s New with NETs?" featuring medical oncologist Dr. Pamela Kunz of Yale Cancer Institute
*Special thanks to Dr. Jaydi Del Rivero, Dr. Andrew Hendifar, Dr. Dan Li, and Josh Mailman for their contributions to this blogpost.
*This blog post is for informational use only. LACNETS encourages you to discuss your individual needs and options with your medical team.
Reference: NIH Clinical Research Trials and You
Written by Lisa Yen, NP, NBC-HWC
Director of Programs & Outreach, LACNETS