What are cancer clinical trials?
Learning about them can help you talk with your doctor and decide what is right for you.

“There is one and only one goal for all of us—to ensure that all our intellectual efforts are directed toward the relief of humanity’s suffering.”
― Azra Raza, The First Cell: And the Human Costs of Pursuing Cancer to the Last

As a cancer patient or caregiver, there are so many questions that come to mind when you are offered a clinical trial as part of your treatment options, and this blog is meant to address common misconceptions as well as fears associated with clinical trials. For some patients, a clinical trial is the first line of treatment, and for others, it’s something that is brought in later.

A cancer clinical trial is a research study comparing the best and most effective known treatment with a novel sort of treatment for a certain kind or stage of cancer. This could be a novel drug mixture or a new approach to utilizing existing treatments. Clinical trials can be thought of in two ways: the medicines we use now were identified, investigated, and first made accessible to patients in clinical trials, and the drugs that will be used to treat advanced or metastatic cancer in the future are currently being evaluated in clinical trials.

Therapy trials, preventative trials, diagnostic trials, supportive and palliative care trials, and natural history studies are all examples of cancer clinical trials. Clinical trials are the culmination of a years-long process that begins with basic laboratory research. Before using a new medication on patients in clinical trials, the research team carefully investigates the drug or treatment method in the laboratory on animals to see how it affects cancer cells. They also try to figure out what kind of adverse effects it might have as well as its safety profile. Every new drug or therapy is tested for patient safety and effectiveness in phases as part of a highly controlled and closely supervised procedure. These phases include:

• Phase 0 which is the preclinical phase. In this phase, testing is performed in a lab on mice or some kind of animal model to first see if there will be any potential benefit and whether it is reasonably safe for humans. Researchers then submit an application to the FDA, which oversees the entire process.
• Phase I is where researchers work to establish the safest, most appropriate dose of the drug. Usually no more than 50 patients participate during this phase, and patients are closely monitored for side effects as they start and continue the treatment during this phase.
• Phase II is where researchers primarily focus on the efficacy of the drug while continuing to monitor for patient safety. Since the dose and safety profile is already established in phase I, the researchers focus on how effective the drug is against cancer.
• Phase III is typically a randomized, controlled trial. This means that patients are randomly assigned to either test new treatments under study or the established standard of care. These trials are designed to provide definitive evidence to support FDA approval of the drug or agent for public use. You may sometimes hear the word "placebo" in a phase III trial. In cancer trials, "placebo" does NOT mean someone goes home with fake or bogus medication. People with cancer are treated with a therapy that's already been approved by the FDA for that type of cancer. During phase III, every patient typically receives the standard-of-care treatment, but some patients also receive the novel drug that’s already been tested in phases I and II. Neither the researcher nor the patient knows which they’re getting. Typically, over 300 patients participate in a phase III trial.
• Phase IV trials take place after a drug is approved and are often called post-marketing trials. The goal is to make sure that no safety or other concerns come up after a drug is approved that may not have been seen in the pre-approval trials. It is important to follow patients for a number of years to determine if there are any long-term side effects or other issues that affect the way the treatment is used.

Potential benefits to consider when enrolling in a clinical trial

• You will gain access to novel therapies that are not accessible to the general public.
• You will have an extended care team, including doctors and nurses, available to answer questions and address concerns. This is a great benefit of joining a trial, as more frequent check-ins can be reassuring and provide a greater understanding of how your treatment is working.
• You may have the chance to contribute to science and help others get better treatment for their cancer in the future.
• You may be able to access information about support groups and resources that are generally not available.

Potential risks to consider when enrolling in a clinical trial

• The new treatment may not be superior to, or even comparable to, the existing treatment.
• New treatments may have unanticipated or worse-than-anticipated negative effects that clinicians are unaware of.
• You may be obliged to visit the doctor more frequently than you would if you were undergoing regular treatment.
• You may incur additional costs as a result of more visits to your treatment center.
• You may require additional testing. Some of the examinations may be distressing or time-consuming.
• Even though a new treatment has been shown to be beneficial in some people, it may not be effective for you.
• In a clinical study, medical insurance may not reimburse all of the costs of patient treatment.

Eligibility

Every clinical trial contains protocols or criteria that explain what will be done during the trial, how it will be executed, and why each step is required. You must also meet the protocol's standards in order to participate in a clinical trial. Eligibility criteria help ensure that people participating in a clinical trial are similar in terms of factors like the type of cancer they have and their general health. When all participants meet the same eligibility criteria, it is more likely that the trial’s outcomes are the result of the treatment being tested than of other factors, such as health conditions or chance. Eligibility requirements are also important for patient safety. They decrease the chances that patients who might experience dangerous side effects from a study drug are enrolled in the trial.

Types Of Cancer Clinical Trials: 

There are two types of eligibility criteria in every cancer clinical trial:

1. Inclusion criteria. These are conditions a person with cancer must meet before joining the clinical trial. One example of an inclusion criteria can be that the person has to have a specific cancer type and stage, like “stage IV lung cancer”.

2. Exclusion criteria. These are conditions a person with cancer cannot have in order to join the clinical trial. For example, the exclusion criteria may say that someone who is diabetic may not participate in the study.

Clinical trials for cancer provide a way to explore the most effective means to treat or prevent cancer. Future generations benefit from the incredible progress in advanced cancer treatment options made by clinical trials.

Reason to hope: A study in JAMA showed the importance of conducting more clinical trials that include older populations with health conditions that might exclude them under more traditional trial criteria. The paper showed that immunotherapy among older Medicare-insured patients with advanced non–small cell lung cancer was associated with shorter overall survival than observed in key clinical trials, providing patients and physicians with estimates of outcomes for older patients who have lung cancer and are being treated with immunotherapy.
https://jamanetwork.com/journals/jamanetworkopen/fullarticle/2780273