A clinical trial is a medical research study that tests new ways to prevent, screen, diagnose, or treat a disease. The word “clinical” means related to examining and treating patients.
Cancer clinical trials help to determine if a treatment, drug, or procedure shows a better way of treating a particular cancer, or provides a way to treat a condition for which there wasn’t a treatment before.
It takes years of thought and planning to have a clinical trial accepted by the U.S. Food and Drug Administration (FDA). The FDA must approve the trial before researchers can recruit patients. Clinical trials allow patients access to the latest research treatments before they are available to the public.
Who sponsors clinical trials?
Medical or research institutions, pharmaceutical and biotechnology companies, the government, and nonprofit groups who want to improve current cancer treatment can all sponsor clinical trials. Some trials are "investigator initiated," which means a researcher or doctor created the trial, but these trials face the same strict requirements.
What do the phases of clinical trials mean?
Phase I trials answer the questions: Is the drug or treatment safe? What is a safe dose? What are the side effects? Phase I trials are small, each only involving 20-80 participants. These trials are usually the first time a drug or treatment has been tested in humans.
Phase II trials tell researchers if the drug or treatment is effective in a larger group (100-300 participants) and help identify safety concerns.
Phase III trials determine within a large group (1,000 – 3,000 participants) if the experimental drug or treatment works better than what is currently used and if there are additional side effects. Most Phase III trials are randomized, meaning some participants will receive the experimental drug or treatment while others will receive the standard drug or treatment currently accepted by the medical community as best for their cancer type (standard of care).
Phase IV trials allow researchers to learn more about the drug or treatment and its risks, benefits, and best use.
Usually FDA approval comes after large phase III trials. However, sometimes special consideration is given to treatments that show great promise in early trials.
After enough data is gathered to prove that the new treatment is effective or more effective than current treatments, the FDA will investigate the findings and approve the treatment for use. After FDA approval, doctors can prescribe treatments to their patients without enrolling patients in clinical trials.
For a participant, what happens in a clinical trial?
Each clinical trial recruits and screens participants through participating treatment centers. In order to learn if a drug is effective, the participants must be similar in the type of diagnosis and other factors. Trials are designed to be very specific so researchers can know that the drug or treatment being studied is causing changes. For example, a clinical trial may be designed to answer a specific question, such as this: “Is this treatment effective for BRCA positive breast cancer in patients who have not received previous treatment?”
Participants must be eligible in order to qualify for the trial. Some possible requirements for eligibility include:
- Cancer type
- Specific tumor types or genetic markers
- Treatment history
- Other medical conditions
If you qualify for a trial, your healthcare team will provide you with all the information about the trial. They will tell you, in a process called informed consent, how the trial will be conducted and the possible risks and benefits of the trial. You will then sign a document stating that you understand the details of the trial and that you are willing to participate. Even after signing the document of informed consent, you may withdraw from the trial at any time for any reason.
The way you receive treatment depends on the specific clinical trial. Clinical trial treatments can come in many forms. These include but are not limited to the following:
- Intravenous (IV) medications given through a port or IV line
- Oral medications
As with any cancer treatment, clinical trial treatments may come with side effects. Your healthcare team will help you manage these side effects. It is very important to report to your healthcare team any side effects that you experience. Part of what researchers are studying is how the treatment affects the whole body, not just the cancer cells.
How is a clinical trial treatment different from other treatments?
Patients who decide to participate in clinical trials receive excellent care and attention. Throughout the trial, your healthcare team will closely monitor you for improvements and adverse side effects.
Researchers want to control as much about your care as they can to make sure it is the treatment that is helping your cancer improve, and not something else. Because of this, you may have restrictions on what you can eat and what other medicines you can take.
Although you should always follow your treatment plan and not miss appointments, doing so is especially important with a clinical trial. Clinical trials must have consistency among the participants to know if the treatment really is working.
Are clinical trials a “last resort” for cancer patients?
Patients who are no longer helped by existing standard of care treatments may find that a clinical trial provides hope, but many patients begin their treatment program with a clinical trial because the treatment best suited to their situation is still being tested. Some clinical trials even exclude patients who have already received another form of treatment.
When participating in a clinical trial that evaluates if a new treatment is better than the current standard treatment for your cancer type, you will always receive treatment – either the standard of care or the new treatment being studied. By being in a clinical trial, you may receive the added benefits of a new treatment before it is available to the public.
In many cases, cancer research moves at a faster pace than the FDA treatment approval process. Researchers have made important discoveries about biomarkers and mutations that affect how cancers respond to treatment. Because of the long time it takes for a treatment to be tested through the clinical trials process, many of these breakthrough treatments are still only available through participating in a clinical trial.
Here are the top reasons why you should consider a clinical trial:
The "standard of care" is what the medical community agrees is the treatment for a specific type of cancer given the patient's overall health. Clinical trials ALWAYS provide treatment that is the standard of care or better. For example, in a randomized Phase III trial for a promising new chemotherapy drug, some patients would get the treatment under study while others get the standard of care. Or some patients would get the current standard of care plus the treatment under study while others get just the current standard of care.
Many of the newest clinical trials focus on very specific tumor types such as tumors that carry specific genetic mutations, markers, or DNA sequencing. Basically, these treatments are tailored to individual cancer diagnoses. Many of these treatments are only available through clinical trials.
Because the promising new treatment is being carefully studied, patients are also carefully monitored by healthcare professionals to see how they are doing and to watch for side effects. So, patients enrolled in clinical trials tend to talk with their health care professionals more often and are watched more carefully than patients not involved in a trial.
Requirements for clinical trials are strictly managed by the Food and Drug Administration. Before a drug is approved for testing in humans, the FDA ensures that it has gone through strict testing protocols in laboratory animals.
Close to home
Most people are familiar with the tremendous research being conducted all over the country at research institutions, but in fact, many community cancer centers offer opportunities for their patients to participate in clinical trials. Depending on the type of clinical trial you seek, you may have many options that do not require you to travel.
Without clinical trials, there is no progress toward cures for cancer. By participating in scientific research through a clinical trial, you can help future cancer patients access new, better treatments. You can play a more active role in your own health care. People who are treated through clinical trials have access to the best new treatments before the general public.
What are the risks of clinical trials?
There are risks involved with the treatment for any life-threatening illness, including treatment offered through clinical trials:
- There may be difficult side effects from medications or treatments.
- The treatment may not be effective.
- The clinical trial may require extra time for trips to the study site, treatments, hospital stays, or complex dosage requirements.
However, through the process of "informed consent," your medical team will make sure you understand all the risks and benefits.
How can I find a clinical trial?
If you are interested in participating in clinical research, your first conversation should be with your doctor. She or he may be able to suggest trials or help you search for appropriate options.
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If you are interested in participating in a clinical trial, the first step is to talk to your healthcare team.
How To Start the Conversation
Here are some tips for talking to your doctor about clinical trials:
- Don’t be afraid to ask. Your doctor may not bring up clinical trials, but you are the person being treated so you should be involved in planning your treatment.
- Do not feel that you are second-guessing your doctor. Your doctor should be willing to discuss options with you and answer all your questions without feeling insulted.
- Do not be afraid to seek a second opinion if necessary.
- If possible, talk to your doctor about clinical trials before you begin treatment. Some trials require that participants have not received any other treatments.
- Write down your questions and what you want to talk about before your appointment. Organize your thoughts and your notes so you don’t forget anything.
- You may wish to have your caregiver go with you to your appointments. Your caregiver can take notes or ask any additional questions if you happen to forget.
- Keep in mind that while a clinical trial may be one survivor’s best option, there may not be a clinical trial option for your diagnosis. You should discuss ALL your treatment options with your healthcare team to create the best treatment plan for you.
What To Ask Before Starting a Trial
Once you find a trial and meet the eligibility requirements, you will go through a process called informed consent. During this process, your healthcare team and members of the research team conducting the trial will tell you all you need to know about the trial. Make sure you receive answers to the questions below:
- What is the purpose of the trial?
- How will the trial treatment be better for me than other treatment options?
- How will the healthcare team learn if I am eligible for the trial?
- Where will I receive treatment?
- What type of treatment will the trial involve?
- What will happen at my appointments? (For example, will I have scans, blood tests, and/or examinations?)
- During the treatment, who should I contact with questions and concerns?
- What are the risks and benefits of this trial?
- Will my insurance cover the cost of the trial?
- Will I receive any financial assistance for travel and lodging?
- What will my follow-up care be?