Brief Summaries of:

·         OHRP Brochure (English)

·         OHRP Brochure (Spanish)

Types of Research

These are some of the different types of research but note that people may categorize or define research in other ways. Types are not mutually exclusive and may overlap or be used in combination with each other.

·         Basic Research – Basic scientists seek answers to fundamental questions in the biology or pathogenesis of a phenomenon, process or disease. Basic science research provides the broad base of knowledge that underlies and is necessary for developing solutions to recognized medical problems. It is often conducted in controlled, laboratory settings. It may be undertaken without looking for long-term benefits other than the advancement of knowledge or to make discoveries in specified areas that will have applied value.

·         Clinical – Clinical research is often defined as research conducted with human subjects and which utilizes both a randomized selection process and a control group. However, it actually includes any research that involves interaction with human subjects in any way and is designed to change human behavior or outcomes.  It can be conducted in a variety of settings, not just in a controlled laboratory. There are many different types of clinical research.

·         Clinical Trials – Clinical trials are designed to determine whether new or current interventions are both safe and effective for changing outcomes in humans.  They use existing knowledge gained from basic research or practical experience for the purpose of creating new or improved products, processes, or practices. There are different types of clinical trials, although they all generally utilize randomized selection and a control group (RCT). Clinical trials are conducted in four phases, which are:

·          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.

·         Epidemiological Research – Epidemiology is the study of the distribution and determinants of health-related states, disease, and events in populations and the application of this study to the control of health problems. It answers questions about who gets diseases, why, and determinants of risk. It focuses on groups of people rather than individuals, and studies those who are healthy as well as those who are sick in order to determine crucial differences between the two groups.

·         Health Services Research – Health services research identifies healthcare needs and studies the provision and use of health services to meet these needs. It is a multidisciplinary field of inquiry, both basic and applied, that examines access, utilization patterns, costs, patient and provider relationships, social and behavioral epidemiology, quality, delivery, organization, financing and outcomes of health care services. It is designed to provide new knowledge about the structure, processes and effects of health services for individuals, employees, and employers.

·         Policy Analysis – Policy analysis refers to research on a fundamental social problem and its related public policies. Analysts are interested in the process by which policies are adopted as well as their effects and usually provide policymakers with briefings and pragmatic, action-oriented recommendations.

·         Evaluation Research – A Process Evaluation assesses the quality of the implementation of a project. An Outcome Evaluation assesses how well a project worked.

·         Survey Research – Survey research is a broad area of research in which people are asked to answer questions, either through standardized questionnaires, in-depth interviews, or focus groups. Surveys are generally administered to a scientifically selected sample, although sometimes they are taken from an entire population, as in the case of the decennial U.S. census. Surveys are used to ascertain beliefs, attitudes, opinions, behaviors, needs, abilities, social status, and nearly every other aspect of peoples' lives. Data collected through surveys are used to describe individuals, estimate population parameters, and discern relationships between what people think, how they act, and the circumstances in which they live.

·         Analysis of Secondary Data – Secondary data analyses use data that have already been collected; that the researcher was not responsible for collecting or it was collected for a different problem than the one currently under analysis. Secondary data may be available which are entirely appropriate and wholly adequate to draw conclusions and answer a research question. Sometimes primary data collection is not necessary. Secondary data analyses can be cheaper, less time-consuming, and more accurate than collecting primary data.

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Why participate in a clinical trial?

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.

Who can participate in a trial?

All clinical trials have guidelines about who can participate. Using inclusion/exclusion criteria is an important principle of medical research that helps to produce reliable results. The factors that allow someone to participate in a clinical trial are called "inclusion criteria" and 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. Some research studies seek participants with illnesses or conditions to be studied in the clinical trial, while others need healthy participants. It is important to note that inclusion and exclusion criteria are not used to reject people personally. Instead, the criteria are used to identify appropriate participants and keep them safe. The criteria help ensure that researchers will be able to answer the questions they plan to study.

What happens during a clinical trial?

The clinical trial process depends on the kind of trial being conducted. (See What are the different types of clinical trials?) The clinical trial team includes doctors and nurses as well as social workers and other health care professionals. They check the health of the participant at the beginning of the trial, give specific instructions for participating in the trial, monitor the participant carefully during the trial, and stay in touch after the trial is completed.

Some clinical trials involve more tests and doctor visits than the participant would normally have for an illness or condition. For all types of trials, the participant works with a research team. Clinical trial participation is most successful when the protocol is carefully followed and there is frequent contact with the research staff.

What is informed consent?

Informed consent is the process of learning the key facts about a clinical trial before deciding whether or not to participate. It is also a continuing process throughout the study to provide information for participants. To help someone decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. If the participant's native language is not English, translation assistance can be provided. 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.

What are the benefits & risks of participating in a clinical trial?

Benefits

Clinical trials that are well-designed and well-executed are the best approach for eligible participants to:

·         Play an active role in their own health care.

·         Gain access to new research treatments before they are widely available.

·         Obtain expert medical care at leading health care facilities during the trial.

·         Help others by contributing to medical research.

Risks

There are risks to clinical trials.

·         There may be unpleasant, serious or even life-threatening side effects to experimental treatment.

·         The experimental 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.

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What are side effects and adverse reactions?

Side effects are any undesired actions or effects of the experimental 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.

How is the safety of the participant protected?

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 (See Confidentiality Regarding Trial Participants).

What should people consider before participating in a clinical trial?

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 experimental treatment being tested may be effective? Has it been tested before?

·         What kinds of tests and experimental 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 experimental 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 experimental treatment is working?

·         Will results of the trials be provided to me?

·         Who will be in charge of my care?

What kind of preparation should a potential participant make for the meeting with the research coordinator or doctor?

·         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.

Does a participant continue to work with a primary health care provider while in a trial?

Yes. Most clinical trials provide short-term treatments related to a designated illness or condition, but do not provide extended or complete primary health care. In addition, by having the health care provider work with the research team, the participant can ensure that other medications or treatments will not conflict with the protocol.

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Can a participant leave a clinical trial after it has begun?

Yes. A participant can leave a clinical trial, at any time. When withdrawing from the trial, the participant should let the research team know about it, and the reasons for leaving the study.

Where do the ideas for trials come from?

Ideas for clinical trials usually come from researchers. After researchers test new therapies or procedures in the laboratory and in animal studies, the experimental treatments with the most promising laboratory results are moved into clinical trials. During a trial, more and more information is gained about an experimental treatment, its risks and how well it may or may not work.

Who sponsors 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.

What is a 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.

What is a 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.

What is a control or control group?

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.

What are the different 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.
 

What is an "expanded access" protocol?

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 be cause 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 experimental 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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