Clinical Trials

A clinical trial is a research study in human volunteers to answer specific health questions. Carefully conducted clinical trials are the safest and fastest way to find treatments that work in people and new ways to improve health.

Types of Clinical Trials

  • Treatment trials test experimental 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, vaccines, vitamins, 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.

Need for Trials

  1. Many clinical trials are done to see if a new drug or device is safe and effective for people to use.
  2. Clinical trials are also done for other reasons. Some compare existing treatments to determine which is better.
  3. The current, approved treatments are called “standard treatments.” Sometimes clinical trials are used to study different ways to use the standard treatments so they will be more effective, easier to use, and/or decrease side effects
  4. Sometimes, studies are done to learn how to best use the treatment in a different population, such as children, in whom the treatment was not previously tested.
  5. For most trials, researchers, doctors, and other health professionals administer the clinical trials according to strict rules set by the Food and Drug Administration (FDA). FDA sets the rules to make sure that people who agree to be in studies are treated as safely as possible.

Potential Benefits

Participating in well-designed and well-executed clinical trials is one approach for eligible patients/volunteers to:

  • Get actively involved in their health care.
  • Gain access to potentially new research treatments
  • Have access to expert medical care for the condition being studied, since investigators are often specialists in the disease area being studied.
  • Help others by contributing to medical research.

Possible Risks

There are generally known and unknown risks associated with clinical trials, such as:

  • There may be unpleasant, serious, or even life-threatening side effects resulting from the treatment.
  • The treatment may not be effective for the participant.
  • The protocol may require more of the participant’s time and attention than a standard treatment. (Participants may need to visit the study site on a regular basis, be subjected to additional tests, get more treatments than are normally necessary, stay in the hospital and/or follow)

Participate

All clinical trials have guidelines about who can participate.

  • Inclusion/exclusion criteria is an important principle of medical research that helps to produce reliable results. 

Inclusion Criteria

The factors that allow someone to participate in a clinical trial are called “Inclusion Criteria” 

Exclusion Criteria

Those that disallow someone from participating are called “Exclusion Criteria”.

These criteria are based on such factors as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions. Before joining a clinical trial, a participant must qualify for the study.

Informed Consent

Informed consent is the process of learning the key facts about a clinical trial before deciding whether or not to participate. Then the research team provides an informed consent document that includes details about the study, such as its purpose, duration, required procedures, and key contacts. Risks and potential benefits are explained in the informed consent document. The participant then decides whether or not to sign the document. Informed consent is not a contract, and the participant may withdraw from the trial at any time.

Protocol

A protocol is a study plan on which all clinical trials are based. The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical trial, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.

Control Group

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.

SAFE Clinical Trials

The FDA works to protect participants in clinical trials and to ensure that people have reliable information as they decide whether to join a clinical trial. The federal government has regulations and guidelines for clinical research to protect participants from unreasonable risks.

Safety of the Participant

The ethical and legal codes that govern medical practice also apply to clinical trials. In addition, most clinical research is federally regulated with built-in safeguards to protect the participants. The trial follows a carefully controlled protocol, a study plan which details what researchers will do in the study. As a clinical trial progresses, researchers report the results of the trial at scientific meetings, to medical journals, and to various government agencies. Individual participants’ names will remain secret and will not be mentioned in these reports.

Phases of Clinical Trials

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 an experimental 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 experimental 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 experimental 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 experimental 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.

Expanded Access

Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.

Sponsors Of Clinical Trials

Clinical trials are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups, and pharmaceutical companies, in addition to federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD), and the Department of Veteran’s Affairs (VA). Trials can take place in a variety of locations, such as hospitals, universities, doctors’ offices, or community clinics.

Placebo

A placebo is an inactive pill, liquid, or powder that has no treatment value. In clinical trials, experimental treatments are often compared with placebos to assess the experimental treatment’s effectiveness. In some studies, the participants in the control group will receive a placebo instead of an active drug or experimental treatment.

People SHOULD think before joining a Clinical Trial

People should learn as much as possible about the clinical trials that interest them. They should also feel comfortable discussing their questions and concerns with members of the health care team. Prospective participants should understand what happens during the trial, the type of health care they will receive, and any costs to them – which may or may not include the cost of the product, costs associated with administering the product, etc.
Anyone considering a clinical trial should also know that there are benefits and risks associated with participating.

Role of the FDA in approving New Drugs & Medical Treatments

The Food and Drug Administration’s job is to make sure medical treatments are safe and effective for people to use. However, FDA does not develop new therapies, or conduct clinical trials to demonstrate safety and effectiveness. FDA staff members meet with researchers and perform inspections of clinical trial study sites to protect the rights of participants and to verify the quality and integrity of the data.

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