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Why Are Clinical Trials Important?
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By: Robert L. Ferris, MD, PhD

 

Clinical trials are a key research tool for advancing medical knowledge and patient care. Clinical research is

 
 Clinical trials are a key research tool.
done only if doctors do not know whether a new approach works well in people and is safe, and which treatments or strategies work best for certain illnesses or groups of people.

 


Clinical trials are important for discovering new treatments for diseases, as well as new ways to detect, diagnose, and reduce the chance of developing the disease. Clinical trials can show researchers what does and doesn’t work in humans that cannot be learned in the laboratory or in animals. Clinical trials also help doctors decide if the side effects of a new treatment are acceptable when weighed against the potential benefits. Researchers don’t know what the results of clinical trials will be. (If they did, they wouldn’t have to do the trials!) This uncertainty can make it hard for a patient to decide to participate in a clinical trial. While in rare cases, patient volunteers have been hurt by the treatment or procedure on a clinical trial, millions of people have been helped because other people before them chose to participate in a trial that resulted in a new, more effective treatment.


While clinical trials are important, the choice to participate in one is very personal and depends on your unique situation. You and your doctor need to weigh the benefits against the risks and decide what’s best for you, when presented with a clinical trial. According to the American Cancer Society, about 1,000 potential medicines are tested before one makes it to clinical trials. On average, new cancer treatments have been studied for at least six years (and sometimes many more) before a clinical trial is started. Usually by the time a treatment makes it to the stage of a clinical trial, it has been found to be safe and to have some chance of being effective. In some cases, a treatment is safe and already FDA-approved and standard practice in one disease, and the clinical trial is testing it in another type of disease.

 

How Clinical Trials Work


If you take part in a clinical trial, you may get tests or treatments in a hospital, clinic, or doctor’s office.


In some ways, taking part in a clinical trial is different from having regular care from your own doctor. For example, you may have more tests and medical exams than you would otherwise.


The purpose of clinical trials is research, so the studies follow strict scientific standards. These standards protect patients and help produce reliable study results.

 

Clinical Trial Protocol


Each clinical trial has a master plan called a protocol, which explains how the trial will work. The trial is led by a principal investigator (PI), who usually is a doctor. The PI prepares the protocol for the clinical trial.


The protocol outlines what will be done during the clinical trial and why. Each medical center that does the study uses the same protocol, which is reviewed and approved by various committees. The committees are in place to ensure patient protections and some potential of learning new information or benefitting patients with the disease.

 

 Clinical trials can show researchers what does and doesn’t work in humans that cannot be learned in the laboratory or in animals.

Key information in a protocol includes how many patients will take part in the clinical trial; who is eligible to take part in the clinical trial; what tests patients will get and how often they will get them; what type of data will be collected during the clinical trial; and detailed information about the treatment plan.

 


The researchers doing clinical trials take steps to avoid bias. “Bias” means that human choices or other factors not related to the protocol affect the trial’s results.

 

Comparison Groups


In most clinical trials, researchers use comparison groups. This means the patients taking part in a trial are assigned to one of two or more similar groups. Each group will receive different medical strategies. For example, one group may get the current standard treatment for a condition, while another group gets a new treatment. Researchers can then compare the results to see whether one group has better outcomes than the other.


Using comparison groups also ensures that no one in a study is left without treatment for the sake of research. Sometimes, when no accepted standard treatment exists for a condition, people in one group may receive a placebo (inactive pill that looks like the test product). You’ll be told if a placebo will be used in a study before you agree to take part.

 

Randomization


Some clinical trials that have comparison groups use randomization. This involves assigning patients to different comparison groups by chance, rather than choice. This method helps ensure that any differences observed during a trial are due to the different strategies being used, not to preexisting differences between the patients. Usually a computer program makes the group assignments.

 

Possible Benefits and Risks of Clinical Trials


Taking part in a clinical trial can have many benefits. For example, you may gain access to new treatments before they’re widely available. If a new treatment is proven to work and you’re in the group getting it, you might be among the first to benefit. If you’re in a clinical trial and don’t get the new strategy being tested, you may receive the current standard care for your condition. This treatment might be as good as, or better than, the new approach. You also will have the support of a team of health care providers, who will likely monitor your health closely.


In late-phase clinical trials, possible benefits or risks of a treatment can be identified earlier than they would be in general medical practice. This is because late-phase trials have large groups of similar patients taking the same treatment the same way. These patients are closely watched by Data and Safety Monitoring Boards.


Even if you don’t directly benefit from the results of the clinical trial you take part in, the information gathered can help others and add to scientific knowledge. People who take part in clinical trials are vital to the process of improving medical care. Many people volunteer because they want to help others.


Clinical trials do have risks and some downsides, such as the following:


• The new strategies and treatments being studied aren’t always better than current standard care.


• Even if a new approach benefits some participants, it may not work for you.


• A new treatment may have side effects or risks that doctors don’t know about or expect. This is especially true during phase I and phase II clinical trials. The risk of side effects might be even greater for trials with cutting-edge approaches, such as gene therapy or new biological treatments.


• Health insurance and health care providers don’t always cover all patient care costs for clinical trials. Find out ahead of time about costs and coverage.


• You should learn about the risks and benefits of any clinical trial before you agree to take part in the trial.


• Talk with your doctor about specific trials you’re interested in.

 

Scientific Oversight


Institutional Review Board


Institutional review boards (IRBs) help provide scientific oversight for clinical trials. An IRB is an independent committee created by the institution that sponsors a clinical trial. IRB members are doctors, statisticians, and community members.


The IRB’s purpose is to ensure that clinical trials are ethical and that the participants’ rights are protected. The IRB reviews the trial’s protocol before the study begins.


An IRB will only approve research that deals with medically important questions in a scientific and responsible way. The IRB also checks on results during the trial. All U.S. clinical trials are required to have an IRB.


Office for Human Research Protections


The U.S. Department of Health and Human Services’ (HHS’) Office for Human Research Protections (OHRP) oversees all research done or supported by HHS. The OHRP helps protect the rights, welfare, and well-being of research participants. It provides guidance and oversight to the IRBs, develops educational programs and materials, and offers advice on research-related issues.


Data Safety Monitoring Board


Every National Institutes of Health (NIH) phase III clinical trial is required to have a Data and Safety Monitoring Board (DSMB). This board consists of a group of research and study topic experts. The NIH also requires DSMBs for large trials comparing alternative strategies for diagnosis or treatment. In addition, the NIH requires DSMBs for some earlier phase trials that involve high-risk procedures (such as gene therapy) or vulnerable patients (such as children).


A DSMB’s role is to review data from a clinical trial for safety problems or differences in results among different groups. The DSMB also reviews research results from other relevant studies. These results may reveal unknown patient risks, or they may even answer the NIH study’s research question. Scientific oversight informs decisions about a trial while it’s under way. For example, some trials are stopped early if benefits from a strategy or treatment are obvious. That way, wider access to the new strategy can occur sooner. Sponsors also may stop a trial, or part of a trial, early if the strategy or treatment is having harmful effects.


Food and Drug Administration


In the United States, the Food and Drug Administration (FDA) provides oversight for clinical trials that are testing new medicines or medical devices. The FDA reviews applications for new medicines and devices before any testing on humans is done, and checks to make sure that the proposed studies have proper informed consent (see below) and protection for human subjects.


The FDA also provides oversight and guidance at various stages throughout the studies. For example, before large-scale phase III trials begin, the FDA provides input on how these studies should be done.


Patient Rights: Informed Consent

 
 Informed consent includes details about the treatments and tests you may receive, and the benefits and risks they may have.

 


Informed consent is the process of giving clinical trial participants all of the facts about a trial. This happens before they agree to take part and during the course of the trial. Informed consent includes details about the treatments and tests you may receive and the benefits and risks they may have.


Before you decide whether to enroll in a clinical trial, a doctor or nurse will give you an informed consent form that presents the key facts of the study. If you agree to take part in the trial, you’ll be asked to sign the form.


You can and should ask questions about the trial to make sure you understand what’s involved. Here are some questions to ask before enrolling in a clinical trial:


• What is the purpose of the study?

• Who is sponsoring the study, and who has reviewed and approved it?
• What kinds of tests, medicines, surgery, or devices are involved? Are any procedures painful?
• What are the possible risks, side effects, and benefits of taking part in the study?
• How might this trial affect my daily life? Will I have to be in the hospital?
• How long will the trial last?
• Who will pay for the tests and treatments I receive?
• Will I be reimbursed for other expenses (for example, travel and child care)?
• Who will be in charge of my care?
• What will happen after the trial?


The informed consent document is not a contract. You have the right to withdraw from a study at any time, for any reason. Also, during the trial, you have the right to learn about new risks or findings that emerge. If researchers learn that a treatment harms you, you’ll be removed from the study.


Taking part in a clinical trial is your decision. Talk with your doctor about all of your treatment options. Together, you can make the best choice for you.


Editor’s Note: Any clinical trial posted by the Oley Foundation has been reviewed by our research committee and deemed appropriate for home parenteral and enteral consumers.


Reprinted with permission from “News from SPOHNC” (October 2016), the Support for People with Oral and Head and Neck Cancer’s newsletter, with slight changes. Robert L. Ferris, MD, PhD, is the UPMC Endowed Professor, Vice-Chair and Chief of Head and Neck Surgery, and Fellowship Director in Head and Neck Oncologic Surgery.

 


LifelineLetter, March/April 2017

This website is an educational resource. It is not intended to provide medical advice or recommend a course of treatment. You should discuss all issues, ideas, suggestions, etc. with your clinician prior to use. Clinicians in a relevant field have reviewed the medical information; however, the Oley Foundation does not guarantee the accuracy of the information presented, and is not liable if information is incorrect or incomplete. If you have questions please contact Oley staff.

 

Updated in 2015 with a generous grant from Shire, Inc. 

 

This website was updated in 2015 with a generous grant from Shire, Inc. This website is an educational resource. It is not intended to provide medical advice or recommend a course of treatment. You should discuss all issues, ideas, suggestions, etc. with your clinician prior to use. Clinicians in a relevant field have reviewed the medical information; however, the Oley Foundation does not guarantee the accuracy of the information presented, and is not liable if information is incorrect or incomplete. If you have questions please contact Oley staff.
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