Partners in Research

Volunteer Patients and the Clinical Center

The National Institutes of Health (NIH) is

one of the nation’s foremost centers for biomedical research. NIH is divided into an

extramural part, which handles biomedical

research throughout the country, and an

intramural part, based mainly in Bethesda,

Maryland. NIH is composed of various

Institutes, each of which conducts research

on health and disease. Studies involving volunteer subjects (research patients and clinical research volunteers) are carried out in

the Warren Grant Magnuson Clinical Center. 

The Clinical Center is the Federal government’s premier biomedical research hospital,

where clinical studies of the highest quality

are performed. Because the research is funded by the Federal government, there is no

cost for care received at the Clinical Center.

Volunteer subjects at the Clinical Center

become partners in a special relationship

with members of the research teams in the

search for better ways to treat disease.

What does it mean to be a volunteer?

The health of millions has been improved

because of advances in science and technology, and the willingness of thousands of individuals like you to take part in clinical

research. The role of volunteer subjects as

partners in clinical research is crucial in the

quest for knowledge that will improve the

health of future generations. Without your

help, the research studies at the Clinical

Center cannot be accomplished.

This information is prepared to help you

understand what is involved in participating

in clinical research. Before you take part in

any study, we encourage you to read the

booklet and be sure that you have satisfactory answers to the list of questions on the last

page. First decide which of the following

categories best describes your situation:

• Clinical research volunteer: A volunteer

subject with no known significant health

problems who participates in research to

test a new drug, device, or intervention.

The clinical research volunteer may be a

member of the community, an NIH

investigator or other employee, or family members of a patient volunteer.

Research procedures with these volunteers are designed to develop new

knowledge, not to provide direct benefit

to study participants.

Clinical research volunteers have always

played a vital role in medical research.

We need to study healthy volunteers for

several reasons: When developing a new

technique such as a blood test or imaging device, we need clinical research

volunteers to help us define the limits of

“normal.” These volunteers are recruited

to serve as controls for patient groups.

They are often matched to patients on

such characteristics as age, gender, or

family relationship. They are then given

the same test, procedure, or drug the

patient group receives. Investigators

learn about the disease process by comparing the patient group to the clinical

research volunteers.

Patient Information Publications

Warren Grant Magnuson Clinical Center

National Institutes of Health

Patient Information Publications 1 Partners in Research
Some studies require a major commitment

in time and effort on the part of the volunteer, or they may involve some discomfort. The research procedure may also

carry some risk. The consent process for

clinical research volunteers involves a

detailed discussion of all the study’s procedures and tests. After this discussion, he

or she will be given a consent document

to read. This document details the study

being considered. It must be read carefully and signed only when the clinical

research volunteer understands what is

involved and is prepared to accept the

potential risk, discomforts, and inconvenience involved.

• Patient volunteer: A volunteer subject

with a known health problem, who participates in research to better understand,

diagnose, treat, or cure a particular disease or condition. Research procedures

with a patient volunteer help develop new

knowledge. Such procedures may or may

not benefit individual study participants.

The following should help you better understand how the research is planned and carried

out, how to weigh the risks, and how your

safety and rights are safeguarded.

What is clinical research?

Patient volunteers have a particular illness or

condition that can help research doctors and

scientists better understand, diagnose, prevent,

treat, or cure it. The research is planned to

help others and may not benefit them directly. 

As a patient volunteer, you may be involved

in studies similar to those described in the

section on clinical research volunteers.

Benefits of such research may be indirect for

you, but may help others.

These studies involve drugs, devices, or interventions designed to prevent, treat, or cure

disease. It is important to remember, however,

that although such studies may provide direct

benefit to patient volunteers, the main aim is

to prove, by scientific means, the effects and

limitations of the experimental treatment. This

may mean that some patients serve as controls

by not taking the test drug, or they receive

test doses of the drug large enough only to

show that it is present, but not at a level that

can treat the condition.

What is a clinical trial?

Clinical trials are a means of developing new

treatments and medications for diseases and

conditions. There are strict rules for clinical

trials, which are monitored nationwide by the

NIH and the FDA, especially when they

involve new drugs. There are three types of

study:

• The phase 1 study is used to learn the

“maximum tolerated dose” of a drug that

does not produce unacceptable side

effects. Patient volunteers are followed

primarily for side effects, and not for how

the drug affects their disease. The first few

volunteer subjects receive low doses of

the trial drug to see how the drug is tolerated and to learn how it acts in the body.

The next group of volunteer subjects

Patient Information Publications 2 Partners in Research
Patient Information Publications 3 Partners in Research

receives larger amounts. Phase 1 studies

typically offer little or no benefit to the volunteer subjects.

• The phase 2 study involves a drug whose

dose and side effects are well known. Many

more volunteer subjects are tested, to define

side effects, learn how it is used in the body,

and learn how it helps the condition under

study. Some volunteer subjects may benefit

from a phase 2 study.

• The phase 3 study compares the new drug

against a commonly used drug. Some volunteer subjects will be given the new drug and

some the commonly used drug. The trial is

designed to find where the new drug fits in

managing a particular condition.

Determining the true benefit of a drug in a clinical trial is difficult. Medical research is dogged

by the “placebo effect”—the real or apparent

improvement in a patient’s condition due to

wishful thinking by the investigator or the

patient. Medical techniques use three ways to

rid clinical trials of this problem. These methods have helped discredit some previously

accepted treatments and validate new ones.

Methods used are the following: randomization,

single-blind or double-blind studies, and the use

of a placebo.

• Randomization is when two or more alternative treatments are selected by chance, not

by choice. The treatment chosen is given

with the highest level of professional care

and expertise, and the results of each treatment are compared. Analyses are done at

intervals during a trial, which may last

years. As soon as one treatment is found to

be definitely superior, the trial is stopped.

In this way, the fewest number of patients

receive the less beneficial treatment.

• In single- or double-blind studies, the participants don’t know which medicine is

being used, and they can describe what happens without bias. Blind studies are

designed to prevent anyone (doctors, nurses,

or patients) from influencing the results.

This allows scientifically accurate conclusions. In single-blind (“single-masked”)

studies, only the patient is not told what is

being given. In a double-blind study, only

the pharmacist knows; the doctors, nurses,

patients, and other health care staff are not

informed. If medically necessary, however,

it is always possible to find out what the

patient is taking.

• Placebos are harmless, inactive substances

made to look like the real medicine used in

the clinical trial. Placebos allow the investigators to learn whether the medicine being

given works better or no better than ordinary treatment. In many studies, there are

successive time periods, with either the

placebo or the real medicine. In order not to

introduce bias, the patient, and sometimes

the staff, are not told when or what the

changes are. 

If a placebo is part of a study, you will

always be informed in the consent form

given to you before you agree to take part in

the study.
Patient Information Publications 4 Partners in Research

When you read the consent form, be sure that

you understand what research approach is being

used in the study you are entering. Remember,

even with a clinical trial, there is no guarantee

that the new approach will be effective for you.

Are there risks involved in participating in clinical

research?

Risks are involved in clinical research, as in

routine medical care and activities of daily living. In thinking about the risks of research, it is

helpful to focus on two things: the degree of

harm that could result from taking part in the

study, and the chance of any harm occurring.

Most clinical studies pose risks of minor discomfort, lasting only a short time. Some volunteer subjects, however, experience complications that require medical attention. In rare

cases, volunteer subjects have received serious

injuries or died of complications resulting from

their participation in trials of experimental therapies.

The specific risks associated with any research

protocol are described in detail in the consent

document, which you are asked to sign before

taking part in research. In addition, the major

risks of participating in a study will be

explained to you by a member of the research

team, who will answer your questions about the

study. Before deciding to participate, you

should carefully weigh these risks. Although

you may not receive any direct benefit as a

result of participating in research, the knowledge developed may help others. The following

section describes safeguards to protect the safety and rights of volunteer subjects.

Protocol review

As in any medical research facility, all new protocols produced at NIH must be approved by an

institutional review board (IRB) before they can

begin. The IRB, which consists of medical specialists, statisticians, nurses, social workers, and

medical ethicists, is the advocate of the volunteer subject. The IRB will only approve protocols that address medically important questions

in a scientific and responsible manner.

Informed consent

Your participation in any Clinical Center

research protocol is voluntary. For every study

in which you intend to participate, you will

receive a document called “Consent to

Participate in a Clinical Research Study” that

explains the study in straightforward language.

A member of the research team will discuss the

protocol with you, explain its details, and

answer your questions. Reading the consent

document and understanding the protocol is

your responsibility. You may discuss the protocol with family and friends. You will not be

hurried into making a decision, and you will be

asked to sign the document only after you

understand the nature of the protocol and agree

to the commitment. 

At any time after signing the consent document,

you are free to change your mind and decide

not to participate further. This means that you

are free to withdraw from the study completely,

or to refuse particular treatments or tests.

Sometimes, however, this will make you ineligible to continue the study. If you are no longer

eligible or no longer wish to continue the study,

you will return to the care of the doctor who

referred you to NIH.
5 Patient Information Publications Partners in Research

Patient representative

The Patient Representative acts as a link

between the patient and the hospital. The Patient

Representative makes every effort to assure that

patients are informed of their rights and responsibilities, and that they understand what the

Clinical Center is, what it can offer, and how it

operates. We realize that this setting is unique

and may generate questions about the patient’s

role in the research process. 

As in any large and complex system, communication can be a problem and misunderstandings

can occur. If you have an unanswered question

or feel there is a problem you'd like to discuss,

call the Patient Representative. The sooner your

concerns are known, the easier they are to

address.

Bill of Rights 

Finally, whether you are a clinical research or a

patient volunteer subject, you are protected by

the Clinical Center Patients’ Bill of Rights. This

document is adapted from the one made by the

American Hospital Association for use in all

hospitals in the country. The bill of rights concerns the care you receive, privacy, confidentiality, and access to medical records. Your patient

handbook contains this information.

Questions to ask before agreeing to participate in a

research protocol

1. What is the purpose of the study?

2. What is required of me?

3. What is my role in the study—am I a clinical research volunteer or a patient volunteer? 

4. Will the study directly benefit me?

5. Will the study benefit others?

6. Are there risks? If so, what are they and

what are the chances that they will occur?

7. What discomforts are involved?

8. What is the total time involved?

9. Are there other inconveniences?

10. Have I discussed participation in the study

with those who are important to me, such

as family and friends?

11. Do I wish to participate in this study?

12. When can I decide not to participate in all or

part of the study? What will be the consequences?

Where applicable, brand names of commercial

products are provided only as illustrative examples of acceptable products, and do not imply

endorsement by NIH; nor does the fact that a

particular brand name product is not identified

imply that such product is unsatisfactory.

This information is prepared specifically for

patients participating in clinical research at the

Warren Grant Magnuson Clinical Center at the

National Institutes of Health and is not necessarily applicable to individuals who are

patients elsewhere. If you have questions

about the information presented here, talk to

a member of your healthcare team. 2003

National Institutes of Health

Warren Grant Magnuson Clinical Center

Bethesda, MD 20892

Questions about the Clinical Center? 

OCCC@cc.nih.gov