What is a Clinical Trial?

It is important for you to know about clinical trials when considering your options for cancer treatment. Please read the following information to better understand clinical trials and how you can play a role in finding a cure!

What is a clinical trial?

A cancer clinical trial is a medical research study in which people participate as volunteers to test new methods of prevention, screening, diagnosis, or treatment of a disease. A cancer clinical trial can also be referred to as a cancer clinical study.

Some cancer patients may fear that enrolling in a cancer trial means they chance receiving no cancer treatment at all, but the reality is that patients in clinical trials receive either the best cancer treatment currently known for their cancer, or a new, and possibly more effective, therapy.

Why are trials important?

Clinical trials are important for many reasons

Cancer clinical trials are no longer seen as a last resort for cancer patients who had no other treatment choices. Today, patients often choose to receive their first cancer treatment in a clinical trial for many reasons.

First, cancer patients who take part in a trial may be helped by the treatment they receive. Trial participants receive either the standard of care—what scientists consider the most effective known treatment available—or the new medication or procedure under study. Cancer trial participants have access to the latest drugs, procedures, and other types of treatment. Although there is no guarantee that a new treatment will be effective, many trial participants conclude that the possible benefits outweigh the risks. But if a new treatment is effective, patients in clinical trials are among the first to receive it.

Second, clinical trials contribute to the overall knowledge and progress against cancer. Many of today’s standard treatments for cancer began in clinical trials. In fact, cancer clinical trials run by cooperative groups have helped establish the effectiveness of lumpectomy for breast cancer, new chemotherapy drugs for colon cancer, and the combination of chemotherapy and radiation for advanced cervical cancer. Many people treated for cancer are now living longer thanks to the knowledge gained through clinical trials.

Third, cancer can be a terrifying diagnosis. Taking an active part in your cancer treatment, and knowing that you might be helping to find a cure by enrolling in a trial, can be an empowering experience.

What are the Top 5 Clinical Trial Myths?

Myth #1: Clinical trial patients are treated like “guinea pigs.”

FACT: Safeguards have been put in place to protect research participants to ensure that legacy practices are not repeated. The informed consent process is an important step of a clinical trial where potential participants learn the purpose of the study, what will happen during the trial, potential risks and benefits, and individual rights in language that is understandable. The informed consent process continues throughout the trial and study participants must be informed of any new benefits, risks or side effects that may surface.

Myth #2: Clinical trials are used to test unproven treatments.

FACT: Clinical trial participants are monitored closely by a member of the medical team. Each trial has a detailed treatment plan (protocol), which must be followed. New treatments must go through a rigorous scientific evaluation and approval process to ensure patient rights and safety before they can begin. In addition, all studies must undergo laboratory preclinical testing, which helps identify treatments that may be ineffective or have side effects that are not tolerable.

Myth #3: Medical care associated with clinical trials costs more.

FACT: Studies have shown that routine patient care associated with clinical trial participation is about equal to costs for patients who don’t participate in trials. Most insurers cover normal costs associated with cancer clinical trial treatment. Many states mandate coverage of clinical trial treatment.

Myth #4: Doctors will always suggest cancer trials as a treatment option for an eligible patient.

FACT: The primary reason that patients participate in clinical trials is because their physicians suggested it; however, many
physicians don’t recommend clinical trials to their patients. Patients can take an active role in their healthcare by using a variety of resources to find a trial for which they may be eligible.

Myth #5: Only cancer patients can participate in clinical trials.

FACT: Cancer clinical trials exist for all types and stages of cancer from cancer prevention through long-term survivorship. There are trials available for healthy people that are at higher risk of cancer due to family history. There are also trials available for people who have had cancer and want to prevent recurrence or reduce the risk of developing a new type of cancer.

Where do clinical trials take place?

Cancer clinical trials are conducted in doctors’ offices, cancer centers, other medical centers, community hospitals and clinics, and veterans’ and military hospitals in cities and towns across the United States and around the world. Cancer clinical trials may include participants at one or two highly specialized centers or they may involve hundreds of locations at the same time.

Ask your doctor where clinical trials are being conducted near you that meet your needs, or go to CancerTrialsHelp.org.

What are the phases of a cancer clinical trial?

phases of cancer clinical trials

Cancer clinical trials have four steps, called “phases.” Each one answers a separate research question.

Cancer clinical trials are usually conducted in a series of steps, called “phases.” Each clinical trial phase is designed to answer a separate research question. Phase I trials evaluate safety, Phase II trials measure effectiveness, and Phase III trials test against best existing cancer treatment. Phase IV trials are conducted to further evaluate new uses or long-term effects of the treatment.

What is a randomized clinical trial?

A randomized cancer clinical trial is a study in which the participants are assigned by chance to separate groups to compare different cancer treatments; neither the researchers nor the participants choose which group the patient is entered into. People are assigned by chance to either a standard treatment group (control group) or investigational treatment arm. In cancer clinical trials the control group invariably receives the standard treatment currently available.

Why are randomized trials important in developing new treatments for cancer?

Randomization in a cancer clinical trial gives each cancer patient an equal chance of being assigned to any of the groups. It is one method used to prevent bias in cancer research

What is standard therapy?

In medicine, standard therapy is a cancer treatment that experts agree is appropriate, accepted, and widely used. Healthcare providers are obligated to provide patients with standard therapy. The terms standard of care or best practice are also used.

What is an investigational drug?

A drug is considered to be investigational if it is being tested in people, but has not yet been approved for marketing by the US Food and Drug Administration (FDA) by proving to be both safe and effective for the diagnosis, prevention, or treatment of a defined disease or condition. A drug may be approved by the FDA for use in one disease or condition, but be considered investigational in other uses.

What is the difference between standard therapy and experimental therapy in a clinical trial?

In medicine, standard therapy is the treatment that experts agree is appropriate, accepted, and widely used. Healthcare providers are obligated to provide patients with standard therapy. Standard therapy is also called standard of care or best practice. In cancer clinical trials, experimental therapy refers to a drug (including a new drug, new dose, combination with other drugs, or route of administration) or procedure that has undergone basic laboratory testing and received approval from the FDA to be tested in human subjects. A drug or procedure may be approved by the FDA for use in one disease or condition, but may be considered experimental in other diseases or conditions. Experimental therapy is also called investigational therapy.

What is a placebo?

A placebo is an inactive substance or treatment that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo. The use of placebos in cancer treatment studies is very uncommon and only with patient consent. Generally, in cancer trials “standard therapy” is given in place of a placebo.

If I enter a cancer clinical trial is there a chance I will receive a “placebo” (eg, sugar pill) instead of a therapy that could treat my cancer?

This misunderstanding is why some cancer patients are reluctant to enter cancer clinical trials. Placebos (also called sugar pills) are rarely used in cancer clinical trials and are never used in place of treatment. Patients who join cancer clinical trials are either given the standard of care, (best treatment available for their specific cancer) or receive a new treatment being investigated. Remember that although you are the only one who can decide whether to take part in a cancer clinical trial, your doctor and the rest of the healthcare team are important and valuable sources of balanced information about the risks and benefits of participation.

How would my cancer clinical trial “experience” compare with my “standard of care” treatment?

An important benefit to participating in a cancer clinical trial is the high level of patient care, as patients in cancer clinical trials are followed more closely by their doctors and nurses. This monitoring is an integral part of the cancer clinical trial process, and undoubtedly provides cancer patients with a high quality experience, whether they are in the treatment or control group.

Are cancer survivors satisfied with their cancer clinical trial experience?

Yes, very. Survivors who participated in a study conducted by the Coalition reported they were very satisfied with the cancer clinical trial experience, with 91% indicating they would recommend participating in a trial to a friend. Additional study results showed:

  1. 97% felt they were fully informed on risks and benefits
  2. 96% felt they were treated with dignity and respect
  3. 92% had a positive experience

The majority of cancer patients (more than 90%) rejected the idea that participants felt like a guinea pig or that they were subjected to more tests and procedures than were necessary.

Are cancer clinical trials just for advanced cancer?

No, although this is a common misconception. Cancer clinical trials exist for all types and stages of cancer, as well as for cancer prevention.

Are there cancer clinical trials for people who do not have cancer, but who are at a higher risk of the disease due to their family history?

Yes, there are cancer clinical trials for those at higher risk of the disease due to their family history. These are called cancer prevention trials. Prevention trials study ways to reduce the risk, or chance, of developing cancer. Most prevention trials are conducted with healthy people who have not had cancer. Some trials are conducted with people who have had cancer and want to prevent the return of cancer or reduce the chance of developing a new type of cancer.

What are the Barriers to Clinical Trial Recruitment & Retention?

Clinical Trial Barriers

Questions to ask your health care team about clinical trials

  1. Who can participate in this cancer clinical trial?
  2. What are the eligibility requirements?
  3. How do the potential benefits compare to other cancer treatment options?
  4. What are the trial’s tests and treatments?
  5. How long will the trial last?
  6. How will the trial and its possible side effects affect my daily life?
  7. Are there treatments to help alleviate side effects?
  8. Will my insurance company cover my costs?
  9. Who will monitor my care and safety?
  10. Who will review the information collected during the cancer clinical trial?
  11. Will I be hospitalized? If so, how often and for how long?
  12. Will I be able to see my own doctor? Who will be in charge of my care?
  13. How often will I need to visit a physician’s office?
  14. What support will be available for me and my caregivers during the trial? Can I talk to other people in the trial?
  15. What is the long-term follow-up care?

The choice of whether to enter a cancer clinical trial is up to you, and your medical team can help you weigh the options. Remember no question about your care is unimportant. These questions can help guide your discussion.