Frequently Asked Questions
Choosing whether or not to participate in a clinical trial is a decision that millions of individuals make each year. Before making that decision, it is important to learn about clinical trials and what it means to be a study participant. The frequently asked questions below help you learn about and what to consider before taking part in a trial.
Additional resources for information and frequently asked questions about clinical trials for patients and their families can be found further below.
Participants in clinical trials can play a more active role in their own health care, gain access to new research treatments before they are widely available, and help others by contributing to medical research.
Clinical trials that are well-designed and well-executed are the best approach for participants to:
- Play an active role in their own health care.
- Gain access to new research treatments before they are widely available.
- Help others by contributing to medical research.
There are also risks to clinical trials:
- There may be unpleasant, serious or even life-threatening side effects to treatment.
- The treatment may not be effective for the participant.
- The protocol may require more of their time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements
Side effects are any undesired actions or effects of drug or treatment. Negative or adverse effects may include headache, nausea, hair loss, skin irritation, or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects.
People should know as much as possible about the clinical trial and feel comfortable asking the members of the health care team questions about it, the care expected while in a trial, and the cost of the trial. The following questions might be helpful for the participant to discuss with the health care team. Some of the answers to these questions are found in the informed consent document.
- What is the purpose of the study?
- Who is going to be in the study?
- Why do researchers believe the new treatment being tested may be effective?
- Has it been tested before?
- What kinds of tests and treatments are involved?
- How do the possible risks, side effects, and benefits in the study compare with my current treatment?
- How might this trial affect my daily life?
- How long will the trial last?
- Will hospitalization be required?
- Who will pay for the treatment?
- Will I be reimbursed for other expenses?
- What type of long-term follow up care is part of this study?
- How will I know that the treatment is working?
- Will results of the trials be provided to me?
- Who will be in charge of my care?
- Plan ahead and write down possible questions to ask.
- Ask a friend or relative to come along for support and to hear the responses to the questions.
- Bring a tape recorder to record the discussion to replay later.
Every clinical trial in the U.S. must be approved and monitored by an Institutional Review Board (IRB) to make sure the risks are as low as possible and are worth any potential benefits. An IRB is an independent committee of physicians, statisticians, community advocates, and others that ensures that a clinical trial is ethical and the rights of study participants are protected. All institutions that conduct or support biomedical research involving people must, by federal regulation, have an IRB that initially approves and periodically reviews the research.
A control is the standard by which experimental observations are evaluated. In many clinical trials, one group of patients will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.
- Treatment trials test new treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.
- Prevention trials look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes.
- Diagnostic trials are conducted to find better tests or procedures for diagnosing a particular disease or condition.
- Screening trials test the best way to detect certain diseases or health conditions.
- Quality of Life trials (or Supportive Care trials) explore ways to improve comfort and the quality of life for individuals with a chronic illness.
Clinical trials are conducted in phases. The trials at each phase have a different purpose and help scientists answer different questions:
- In Phase I trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
- In Phase II trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
- In Phase III trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
- In Phase IV trials, post marketing studies delineate additional information including the drug’s risks, benefits, and optimal use.
Most human use of investigational new drugs takes place in controlled clinical trials conducted to assess safety and efficacy of new drugs. Data from the trials can serve as the basis for the drug marketing application. Sometimes, patients do not qualify for these carefully-controlled trials because of other health problems, age, or other factors. For patients who may benefit from the drug use but don’t qualify for the trials, FDA regulations enable manufacturers of investigational new drugs to provide for “expanded access” use of the drug. For example, a treatment IND (Investigational New Drug application) or treatment protocol is a relatively unrestricted study.
The primary intent of a treatment IND/protocol is to provide for access to the new drug for people with a life-threatening or serious disease for which there is no good alternative treatment. A secondary purpose for a treatment IND/protocol is to generate additional information about the drug, especially its safety. Expanded access protocols can be undertaken only if clinical investigators are actively studying the new treatment in well-controlled studies, or all studies have been completed. There must be evidence that the drug may be an effective treatment in patients like those to be treated under the protocol. The drug cannot expose patients to unreasonable risks given the severity of the disease to be treated.
Some investigational drugs are available from pharmaceutical manufacturers through expanded access programs listed in ClinicalTrials.gov. Expanded access protocols are generally managed by the manufacturer, with the investigational treatment administered by researchers or doctors in office-based practice. If you or a loved one are interested in treatment with an investigational drug under an expanded access protocol listed in ClinicalTrials.gov, review the protocol eligibility criteria and location information and inquire at the Contact Information number.
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Clinical Trial Resources
For more information about Expanded Access in the USA, please visit FDA’s educational page. For European readers please visit EMA’s page.
The above information is provided by clinicaltrials.gov, a service of the National Institute of Health. Additional resources for information and frequently asked questions about clinical trials for patients and their families can be found at:
www.clinicaltrials.gov/ct2/about-studies/learn
www.webmd.com
www.centerwatch.com
www.cancer.gov
rarediseases.info.nih.gov
www.acrpnet.org
www.foxtrialfinder.org
www.centerwatch.com/clinical-trials/listings/
www.coronaclinicaltrials.com
Questions to Ask
People who are in a clinical trial or are thinking about joining a clinical trial have the right to ask the study staff any questions they may have about the study.
The questions below are useful for patients, potential study volunteers, and their family members or friends, to ask the research staff – which can include doctors, nurses, study coordinators and other people who help conduct the trial.
For a printable version of these questions, click here to download the PDF and take with you to your appointment.
- What is the main purpose of this study?
- Does the study involve a placebo or a treatment that is already on the market?
- How will the treatment be given to me?
- How long is the study going to last and what will I be asked to do as a participant?
- What has been learned about the study treatment and are any study results published?
- Do I have to pay for any part of the study? Will my insurance cover these costs?
- Is there any reimbursement for travel costs or childcare?
- Will I be able to see my own doctor?
- If the treatment works for me, can I keep using it after the study?
- Can anyone find out whether I’m participating in the clinical trial?
- Will I receive any follow-up care after the study has ended?
- What will happen to my medical care if I stop participating in the study?
- Does the physician/investigator have any financial or special interest in the clinical study?
- What are the credentials and research experience of the physician and study staff?
- When and how will the results of the clinical trial be provided to me?
- Will I learn my treatment assignment?
CISCRP was featured in a Wall Street Journal Supplement: “Your Guide to Understanding the Need for Clinical Research and Participation” with its feature article, Understanding Clinical Trials: Appreciating Medical Heroes.