People with non-small cell lung cancer (NSCLC) may wonder about participating in a clinical trial. A clinical trial is a study that tests a new treatment for effectiveness in a target population before it can be approved by the United States Food and Drug Administration (FDA) or equivalent entities in other countries. There are many important points to discuss with a health care provider when considering participation in a clinical trial.
In recent years, research studies and clinical trials have led to significant advances in effective treatment options for NSCLC. Many newly approved lung cancer treatments are targeted therapies, which are drugs that specifically target certain proteins or groups of proteins that play a role in cancer growth.
One such class of drugs is called epidermal growth factor receptor (EGFR) inhibitors. These drugs work by targeting cells with mutations in the EGFR gene that commonly occur in NSCLC. Two drugs for people with EGFR mutations — Rybrevant (amivantamab-vmjw) and Exkivity (mobocertinib) — became available in 2021.
Immunotherapy is a newer type of treatment that has also shown promise in treating NSCLC. Immunotherapy drugs help to use the body’s immune system to recognize and attack cancer cells. Keytruda (pembrolizumab) was approved by the FDA in 2016 to treat metastatic NSCLC that has the immune protein PD-L1.
New therapies have made it possible to selectively kill cancer cells without as many widespread side effects as standard radiation therapy and chemotherapy. Consequently, there are fewer deaths due to NSCLC with the death rate decreasing by 6.3 percent each year between 2013 and 2016. Participation in clinical trials has made it possible to explore effective new therapies, resulting in more people living longer with NSCLC.
Clinical trials usually take place at a large cancer center and involve at least three distinct phases to determine if a new therapy is safe and effective. Each phase of the trial has a different objective.
The goal of a phase 1 clinical study is to determine the best dose of the drug and if there are any major side effects (also called adverse events). Phase 1 is focused on the general safety of the treatment and may include participants with different types of cancer and different stages of disease.
Phase 2 studies are designed to determine if a treatment is effective in the target population. Here, only people with the type of cancer that the drug was made for are included. Based on the information learned in phase 1, participants receive the decided dose of the drug and are monitored to see if their condition improves.
A phase 3 study, which is the final phase before FDA approval, compares the effectiveness of the new drug to the standard of care, which is the drug or regimen currently used by doctors to treat the disease. A phase 3 study includes a larger number of people with the cancer of interest and includes two treatment groups: one group of people that gets the new drug and one group that gets the current treatment regimen.
Less commonly, some phase 3 studies may also include a placebo. A placebo, also called a sugar pill, is a “mock” medicine that is given to participants but has no effect. In clinical trials for cancer, placebos are used only in cases when there is no standard treatment, or in addition to standard treatment. Participants who do not receive the trial treatment receive treatment that’s considered standard of care — widely used and accepted as best.
After a drug is approved by the FDA, a phase 4 trial may take place. The purpose of the phase 4 study is to observe how a drug works in a large group of people over a much longer period of time and to find out whether there are side effects.
Before deciding whether to participate in a clinical trial, there are many key points to discuss with a doctor to determine if it is a good fit.
Initial discussions about participating in a clinical trial will focus on eligibility criteria. Eligibility for participation may depend on:
Due to the emerging importance of targeted therapies for the treatment of NSCLC, a person may also be required to have a certain genetic mutation targeted by the drug — often referred to as biomarkers. This may require a biopsy to obtain a sample of the tumor followed by molecular testing to determine the presence or absence of the mutation.
Each clinical study has a different set of eligibility criteria based on the overall goals of the study and the treatment being tested. A cancer care team can evaluate a person with NSCLC to see if they meet the requirements of a particular study.
Each phase of a clinical trial can range from several months to several years. The length of a clinical trial generally increases with each clinical trial phase. The exact length of time each phase lasts varies depending on the study’s design.
During initial discussions with a doctor, be sure to request information on what phase of the trial you might participate in and how long it is supposed to last.
Many people may be concerned about potential costs related to clinical trials. Questions to ask regarding trial-related costs include:
It is also helpful to understand the type of therapy being tested in the trial and the proposed treatment plan. Many emerging treatments like immunotherapy and targeted drugs are new and not as well understood. Potential participants should ask their doctor for information about how the treatment works and how it is administered.
It is important to understand the potential advantages of enrolling in a clinical trial. Some possible benefits include:
Of course, some risks may come along with participating in a clinical trial. Potential disadvantages include:
A doctor should go through potential benefits and risks to allow a person to make an informed decision about whether to participate in a trial. Questions you can consider asking are:
Clinical studies may require extensive follow-up with a health care team after completing the treatment regimen. Ask your doctor to explain the length of the follow-up period, who is on the health care team, and where to go for check-ups.
Historically, some participants in medical research were not told the truth about what was happening to them or risks to their health. Today, informed consent is a key component of all clinical studies. Before agreeing to participate in a trial, you must be provided with sufficient information to make an informed decision, sufficient time to read and review consent materials, and the opportunity to ask follow-up questions.
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