Clinical Trials

The importance of clinical trials

All the advances in cancer treatment that are saving lives today are the result of clinical trials. Clinical trials are research studies that explore whether a medical strategy, treatment or device is safe and effective for people. Sponsored by government agencies (such as the National Cancer Institute), independent groups of doctors and health care institutions, or the pharmaceutical or biotechnology industries that have developed the new treatments, clinical trials also may show which strategies work best for certain illnesses or groups of people.

Understanding the clinical trial process can be overwhelming and confusing. This guide provides valuable information that will help you make an informed decision about participating in a clinical trial.

Types of clinical trials

Clinical trials are separated into three categories:

  • Treatment Trials evaluate whether a new type of treatment (drug, surgery, radiation therapy) or a combination of treatments is better than the treatment options that are currently available.
  • Quality-of-Life Trials study ways to improve the quality of life for people with cancer and survivors who experience cancer- and treatment-related symptoms. This type of trial may evaluate the effects of such things as nutrition, group therapy or counseling.
  • Prevention, Screening and Diagnostic Trials may be treatment or nontreatment trials that assess ways to reduce the chance of getting cancer in general. In these trials, many participants do not have cancer, but some have had cancer and are at risk of the cancer returning (recurring) or a second cancer type developing. Sometimes these trials consist of simply completing questionnaires and providing medical information.

Should you participate?

You may consider participating in a clinical trial for the following reasons:

  1. Your current treatment may not be working as well as expected, and a clinical trial may be a worthwhile alternative.
  2. A clinical trial may significantly improve your quality of life. Discuss your personal situation with your medical team, so they are aware of your expectations regarding side effects.
  3. You may have a rare type of cancer that hasn’t been studied as much as other types.
  4. By simply participating, you play an integral role in helping refine and improve the way millions of people with all types and stages of cancer are treated. You will not only help identify treatments that do work, you’ll help eliminate those that don’t.

Learning more about clinical trials by talking with other clinical trial participants may encourage you to volunteer. You won’t jeopardize your routine medical care if you participate. Institutional review boards or ethics committees carefully set up safeguards to make sure that all patients in the clinical trial remain safe throughout the process. Keep in mind that not everyone responds to treatments in the same way, so you cannot expect an identical experience in terms of response to treatment, side effects, etc. Regardless of the opinions and research you gather, participating in a clinical trial is ultimately your decision.

Before deciding whether to participate, do your research. Consult with your own doctor as well as the medical team who will be conducting the trial. Also, check with your insurance company to see what costs your policy covers and what you are required to pay if you participate.

What to Expect

Clinical trials are all highly strategic and performed in an extremely consistent manner so that all patients are treated exactly the same. Whether you’re at a small rural hospital or a large facility in a metropolitan area, your medical team is responsible for diligently following all of the same safety measures for your treatment plan across the board.

When you volunteer to participate in a clinical trial, you will receive specific instructions but you are encouraged to ask questions about anything you don’t fully understand. This is the ideal time to talk with your medical team about the many falsehoods that persist about clinical trials. For example, although there is fear to the contrary, participants are guaranteed to receive at minimum the current standard of care during the trial. Myth vs Fact addresses more of these common misconceptions.

You will be carefully monitored throughout the clinical trial. Even after the treatment ends, you will continue to be in close contact with the medical team.

Risks and benefits

Clinical trials present many potential benefits. At the same time, they also present many potential risks, such as side effects. If you consider volunteering for a clinical trial, talk with your medical team about the expected benefits and risks so you are not surprised by some of the effects.

Fear of the unknown is a common reason patients with cancer hesitate to volunteer in clinical trials. Rest assured, you can ask questions and share your concerns with your medical team before and throughout the clinical trial. Participation is always voluntary, even after the study begins. Even though you sign an agreement saying that you understand the potential risks involved, you can decide to leave the trial at any time. If your expectations aren’t met or if you experience too many side effects, you can withdraw and return to standard treatment at any time.

Weighing advantages and disadvantages of clinical trials

Potential advantages Potential disadvantages
Access to the best possible care through treatment with either the current standard of care or a treatment thought to offer more benefit than the standard of care Lack of clearly defined side effects or risks associated with experimental treatment
Potential for being treated with a new drug before it is widely available Chance that experimental treatment may not be better than the standard of care
Close monitoring during treatment and follow-up (often more so than in routine practice) Potential that not all participants will benefit from experimental treatment
Chance to play active role in health care Need for frequent tests and clinic visits
Opportunity to help future generations of individuals with cancer Possible need for travel to clinical trial site

 

The four phases of clinical trials

Clinical trials are conducted in four phases. Each phase is designed to address a separate research question about the treatment being evaluated. The information gathered in each phase helps build the next phase, providing researchers with the most reliable information possible. By completing the phases in order, researchers are able to systematically learn about treatments while keeping participants as safe as possible.

It can take as long as 14.5 to 15 years for a drug to complete all of the studies needed to become FDA-approved and available commercially.

Before a clinical trial moves to the first phase, it may go through about 4.5 years in animal and/or laboratory studies.

  • Phase I: This phase usually includes a small number of volunteers, and the research is primarily designed to determine the best dose, delivery method (for example, oral versus intravenous) and schedule (how often it’s given) for the drug. Drug safety and related side effects are closely monitored in phase I trials. This phase usually includes 15 to 30 participants.
  • Phase II: This phase helps determine how well a drug works and how safe it is by enrolling a greater number of patients with a specific type of cancer. Side effects and safety continue to be evaluated. This phase usually includes fewer than 100 participants.
  • Phase III: In this phase, the risks versus the benefits and overall efficacy of the new drug are compared with those associated with the current standard of care. This phase also evaluates safety. If the results of a phase III trial prove that the new drug leads to better outcomes for patients and is deemed safe (the benefits outweigh the risks), the FDA determines whether it will be approved and made available to the public. This phase includes anywhere between hundreds to thousands of participants.
  • Phase IV: Once a drug is approved, volunteers can be included in a phase IV clinical trial, in which a large number of people taking the drug continue to be monitored for long-term safety and side effects.

 

The more you know

If you find a clinical trial that interests you, start by asking your health care team these questions. The more you know, the better equipped you’ll be to decide if it is right for you.

  • Am I eligible for this clinical trial?
  • What is the treatment?
  • How long will the clinical trial last?
  • How do researchers think the treatment being studied differs from my current treatment plan?
  • How will I know if it is working?
  • Why is this trial being done?
  • Why is it believed that the treatment being studied may be better than the standard treatment?
  • What are my other options (standard treatments, other studies)?
  • What were the results of any previous studies of this new treatment?
  • What are the possible side effects or risks of the new treatment?
  • What are the possible benefits?
  • How will the doctor measure if the treatment is working?
  • Can I choose to continue to get this treatment after the trial ends?
  • What kinds of procedures or tests are involved?
  • What impact will the trial have on my daily life?
  • Will I have to travel to receive treatment? Will I be compensated for expenses?
  • How often will I need to travel to receive treatment?
  • Will I have to be hospitalized as part of the trial?
  • What type of long-term follow-up care will be done?
  • Will I still be under the care of my doctor, or will I see someone different?
  • What costs (if any) will be my responsibility to pay?
 

 

Dispelling the myths of cancer clinical trials

MYTH: Some participants in a clinical trial will get a placebo (sugar pill) instead of treatment.
FACT: Participants in cancer clinical trials will receive the standard of care as a foundation and then the experimental treatment or a placebo will be added to it. They will never receive a placebo instead of a cancer treatment.
 
MYTH: Clinical trials are not safe.
FACT: Clinical trials have many built-in safeguards to ensure that participants’ rights and safety are protected. These safeguards include an institutional review board, data and safety monitoring board, and an ongoing informed consent process.
 
MYTH: Clinical trials are only for people who have no other options for treatment (a “last resort”).
FACT: Clinical trials of cancer treatment may be available for individuals with cancer of all types and stages.
 
MYTH: The cost of care in a clinical trial is not covered by health insurance.
FACT: Under the new health care laws, patient-care costs (such as going to the doctor, hospital stays or certain testing procedures) may be covered by insurance. It’s common, but not guaranteed, for the trial sponsor to cover research costs directly related to the study, and insurance does not typically cover research costs. Any questions about coverage should be provided before entering into a clinical trial.
 
MYTH: Clinical trials only take place at large hospitals or cancer centers.
FACT: Many clinical trials take place at local hospitals, cancer centers and doctors’ offices. Clinical trials occur in all parts of the country, in both rural and urban areas.
 
MYTH: Signing a consent form to participate “locks” you into staying in a trial.
FACT: Once you have signed a consent form, you are free to change your mind and not participate. You can also decide to drop out of a trial at any time for any reason.
 
MYTH: A clinical trial must be recommended by a doctor in order for a person to participate.
FACT: If your doctor does not talk to you about clinical trials, raise the topic yourself. You can also search for clinical trials online; contact information for trials is given in the box below.
 
MYTH: Clinical trials involve treatments with unknown safety and efficacy.
FACT: Phase III clinical trials involve treatments that have been shown to be safe and effective in earlier phase trials. Phase III trials also include treatment with the standard of care, which has well-established evidence of safety and efficacy. Phase I and II trials are based on the results of preclinical trials showing the potential for efficacy and are carefully designed to ensure the safety of participants.
 
MYTH: Participants in clinical trials are treated like “guinea pigs.”
FACT: The overwhelming majority of clinical trial participants (97 percent) say they were treated with dignity and respect; 93 percent report having a positive experience in the trial.

 

 

Find a Clinical Trial

Your health care team can give you more information about clinical trials in your area, but they may not be aware of trials available in other locations. Using online tools, such as these, makes it easy to search for clinical trials that apply to you:

 
 

Words to know

As you investigate clinical trials, it will help if you’re familiar with the terminology used to describe trials and your medical coverage. Here are a few key terms to remember as you learn more about clinical trials and how they are affected by your medical plan and coverage:

Blinded: A study in which the participants do not know which treatment group they are in. In a single-blind trial, the participants don’t know what treatment they’re receiving. In a double-blind trial, neither the volunteers nor the researchers know who is receiving the experimental drug.

Claim: A request for payment based on the terms of your insurance policy.

Coinsurance: The percentage of medical care that you are financially responsible for paying after meeting your deductible.

Control group: The participants who are assigned to receive the standard treatment that is being compared with one or more experimental treatments.

Copay: The fixed amount you must pay for specific types of medical care.

Deductible: The amount of money you must pay before your insurance begins reimbursing fees.

Eligibility criteria: The guidelines defining who can participate (based on age, sex, health status, type and stage of cancer, for example).

Explanation of benefits (EOB): A statement provided by your health insurance company explaining what medical treatments and/or services were paid on your behalf.

Health insurance exchange/health insurance marketplace: A central hub for those seeking health insurance where the user can review plan options, see the premium rates for each and enroll in the plan they select.

Informed consent: A document that contains all the important information about the study, including the possible risks and benefits and the alternatives to the research being conducted. The document stresses that enrolling and remaining in the study are completely voluntary and that a volunteer may leave the study at any time. Generally, a person must sign an informed consent document to enroll in a clinical study.

Out-of-pocket costs: Expenses for medical care that aren't reimbursed by insurance and that you are responsible for paying. These costs may include deductibles, coinsurance and copayments for covered services plus all costs for services that aren't covered.

Placebo: An inactive drug given in a control group to simulate treatment with an active agent. Placebos are not used instead of treatment in cancer clinical trials. They are given along with the standard treatment.

Premium: The amount you pay each month to keep yourself insured.

Premium insurance tax credits: Tax credits from the federal government that reduce the cost of health insurance premiums given to individuals who qualify. Credits are available to use immediately to offset insurance costs, unlike other tax credits that are received when filing your taxes. Consumers whose income is between 100 to 400 percent of the federal poverty level may be eligible for these credits, which are also referred to as “insurance subsidies.”

Protocol: The set of rules that every participating clinic, hospital, university or cancer center must follow in a clinical trial. The protocol outlines the eligibility criteria, specifies the tests that will be used and procedures that will be performed, describes the medications and their doses and establishes the length of each study.

Reimbursement: Compensation or repayment from your insurance company for health care services that you paid for out-of-pocket.

Standard treatment (or therapy): The drug or treatment that experts agree is the most widely used and appropriate therapy for a particular type and stage of cancer. The standard treatment is used as a baseline against which an experimental drug or treatment is compared in a phase III trial.

 

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