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Clinical Trial Resource Guide
Understanding Clinical Trials
Clinical Trial Resource Guide
Understanding Clinical Trials
What Are Clinical Trials?
What Are Clinical Trials?
Clinical trials are research studies that evaluate the safety and effectiveness of new medical approaches in people. These studies are essential for developing new ways to prevent, detect, diagnose, or treat diseases. Clinical trials represent the final stages of a long research process and are the primary way researchers determine if new treatments are safe and effective for people.
Types of clinical trials include:
Prevention trials: Testing new approaches to prevent disease
Screening trials: Evaluating new ways to detect diseases or health conditions
Diagnostic trials: Studying procedures or tests for diagnosing particular diseases
Treatment trials: Testing new treatments, drugs, or surgical procedures
Behavioral trials: Evaluating ways to promote health-related behavioral changes
Quality of life trials: Exploring ways to improve comfort and quality of life for people with chronic conditions
Clinical trials are research studies that evaluate the safety and effectiveness of new medical approaches in people. These studies are essential for developing new ways to prevent, detect, diagnose, or treat diseases. Clinical trials represent the final stages of a long research process and are the primary way researchers determine if new treatments are safe and effective for people.
Types of clinical trials include:
Prevention trials: Testing new approaches to prevent disease
Screening trials: Evaluating new ways to detect diseases or health conditions
Diagnostic trials: Studying procedures or tests for diagnosing particular diseases
Treatment trials: Testing new treatments, drugs, or surgical procedures
Behavioral trials: Evaluating ways to promote health-related behavioral changes
Quality of life trials: Exploring ways to improve comfort and quality of life for people with chronic conditions
How Clinical Trials Work
How Clinical Trials Work
Clinical trials follow a careful plan called a protocol, which describes:
The goal of the study
Who can participate
Protections for participants
Details about tests, procedures, and treatments
Expected duration of the trial
Information to be collected
Clinical trials are conducted in phases:
Phase I: Testing in a small group (20-80 people) to evaluate safety, identify side effects, and determine safe dosage
Phase II: Testing in a larger group (100-300 people) to further evaluate safety and effectiveness
Phase III: Testing in large groups (1,000-3,000 people) to confirm effectiveness, monitor side effects, and compare with standard treatments
Phase IV: Post-market studies used to gather information on long-term effects and use in the general population
Clinical trials follow a careful plan called a protocol, which describes:
The goal of the study
Who can participate
Protections for participants
Details about tests, procedures, and treatments
Expected duration of the trial
Information to be collected
Clinical trials are conducted in phases:
Phase I: Testing in a small group (20-80 people) to evaluate safety, identify side effects, and determine safe dosage
Phase II: Testing in a larger group (100-300 people) to further evaluate safety and effectiveness
Phase III: Testing in large groups (1,000-3,000 people) to confirm effectiveness, monitor side effects, and compare with standard treatments
Phase IV: Post-market studies used to gather information on long-term effects and use in the general population
Why Consider Participating in a Clinical Trial?
Why Consider Participating in a Clinical Trial?
Access to new treatments before they are widely available
Receive regular and careful medical attention from research teams
Play an active role in your own healthcare
Help others by contributing to medical research and advancing science
Potentially improve your own condition or disease
Access to new treatments before they are widely available
Receive regular and careful medical attention from research teams
Play an active role in your own healthcare
Help others by contributing to medical research and advancing science
Potentially improve your own condition or disease
Your Rights and Safety
Your Rights and Safety
Informed Consent
Before joining a clinical trial, you'll receive an informed consent document that ensures you understand all aspects of the trial before deciding to participate. It explains:
The purpose of the study
Required procedures and time commitment
Potential risks and benefits
Your rights as a participant, including the right to leave the study at any time
Safety Oversight
Clinical trials are carefully regulated to protect participants:
Institutional Review Boards (IRBs) review and approve study protocols
Data and Safety Monitoring Boards independently review ongoing studies
The FDA inspects study sites and records
Researchers must report any adverse events or new information
Informed Consent
Before joining a clinical trial, you'll receive an informed consent document that ensures you understand all aspects of the trial before deciding to participate. It explains:
The purpose of the study
Required procedures and time commitment
Potential risks and benefits
Your rights as a participant, including the right to leave the study at any time
Safety Oversight
Clinical trials are carefully regulated to protect participants:
Institutional Review Boards (IRBs) review and approve study protocols
Data and Safety Monitoring Boards independently review ongoing studies
The FDA inspects study sites and records
Researchers must report any adverse events or new information
Diversity in Clinical Trials
Diversity in Clinical Trials
The Current State of Diversity
Clinical trials have historically lacked diversity among participants:
According to FDA data, in 2020, 75% of clinical trial participants were White, while only
11% were Hispanic, 8% were Black, and 6% were Asian
Women, older adults, and rural populations are also frequently underrepresented
Certain medical conditions disproportionately affect minority populations, yet these
groups are often underrepresented in relevant trials
Why Diversity Is Essential
Medical Relevance: Different populations may respond differently to treatments due to genetic factors, metabolism differences, and other biological variations
Health Equity: Including diverse populations helps reduce healthcare disparities by
ensuring treatments work for everyone
Scientific Validity: For results to be applicable to the general U.S. population, study
participants should reflect the diversity of that population
Regulatory Requirements: The FDA increasingly emphasizes the importance of
representative trial populations when evaluating new treatments
When clinical trials lack diversity, the resulting treatments may be less effective for underrepresented groups or may have unexpected side effects in these populations.
The Current State of Diversity
Clinical trials have historically lacked diversity among participants:
According to FDA data, in 2020, 75% of clinical trial participants were White, while only
11% were Hispanic, 8% were Black, and 6% were Asian
Women, older adults, and rural populations are also frequently underrepresented
Certain medical conditions disproportionately affect minority populations, yet these
groups are often underrepresented in relevant trials
Why Diversity Is Essential
Medical Relevance: Different populations may respond differently to treatments due to genetic factors, metabolism differences, and other biological variations
Health Equity: Including diverse populations helps reduce healthcare disparities by
ensuring treatments work for everyone
Scientific Validity: For results to be applicable to the general U.S. population, study
participants should reflect the diversity of that population
Regulatory Requirements: The FDA increasingly emphasizes the importance of
representative trial populations when evaluating new treatments
When clinical trials lack diversity, the resulting treatments may be less effective for underrepresented groups or may have unexpected side effects in these populations.
What to Ask Before Joining a Clinical Trial
What to Ask Before Joining a Clinical Trial
What is the purpose of the study?
What treatments will I receive?
What are the potential risks and benefits?
What tests and procedures are involved?
How long will the trial last?
How will the trial affect my daily life?
Will I have any costs related to participation?
Will I receive compensation for my time or travel expenses?
How will my privacy be protected?
Who can I contact if I have questions during the trial?
What is the purpose of the study?
What treatments will I receive?
What are the potential risks and benefits?
What tests and procedures are involved?
How long will the trial last?
How will the trial affect my daily life?
Will I have any costs related to participation?
Will I receive compensation for my time or travel expenses?
How will my privacy be protected?
Who can I contact if I have questions during the trial?
The Importance of Healthy Volunteers in Clinical Trials
The Importance of Healthy Volunteers in Clinical Trials
Many clinical trials need both patients with specific conditions and healthy volunteers. Healthy volunteers play a crucial role in medical research for several reasons:
Establishing "Normal" Baselines: Healthy volunteers help researchers define what's
"normal" in terms of how drugs are absorbed, distributed, and eliminated from the
body.
Comparison Groups: They provide a baseline against which patient groups can be
compared, allowing researchers to better understand disease processes and treatment effects.
Early Safety Testing: In Phase I trials, healthy volunteers help determine if new treatments are safe and identify potential side effects before they're given to patients.
Advancing Medical Knowledge: By participating, healthy volunteers contribute to scientific progress even when they don't personally benefit from the treatment being studied.
Healthy volunteer studies typically involve activities like:
Blood draws and other biological samples
Physical examinations
Questionnaires and interviews
Testing of vital signs and body functions
Sometimes short hospital stays
While these studies may not directly benefit the volunteer's health, they provide valuable data that can help develop treatments for various conditions and diseases.
Many clinical trials need both patients with specific conditions and healthy volunteers. Healthy volunteers play a crucial role in medical research for several reasons:
Establishing "Normal" Baselines: Healthy volunteers help researchers define what's
"normal" in terms of how drugs are absorbed, distributed, and eliminated from the
body.
Comparison Groups: They provide a baseline against which patient groups can be
compared, allowing researchers to better understand disease processes and treatment effects.
Early Safety Testing: In Phase I trials, healthy volunteers help determine if new treatments are safe and identify potential side effects before they're given to patients.
Advancing Medical Knowledge: By participating, healthy volunteers contribute to scientific progress even when they don't personally benefit from the treatment being studied.
Healthy volunteer studies typically involve activities like:
Blood draws and other biological samples
Physical examinations
Questionnaires and interviews
Testing of vital signs and body functions
Sometimes short hospital stays
While these studies may not directly benefit the volunteer's health, they provide valuable data that can help develop treatments for various conditions and diseases.
Finding Clinical Trials
Finding Clinical Trials
Clinical trials are conducted at hospitals, universities, doctors' offices, and community clinics.
To find a clinical trial:
Ask your healthcare provider
Use our FindMyClinicalTrial website search tools
Clinical trials are conducted at hospitals, universities, doctors' offices, and community clinics.
To find a clinical trial:
Ask your healthcare provider
Use our FindMyClinicalTrial website search tools
Glossary of Clinical Trial Terms
Glossary of Clinical Trial Terms
Randomization: The process by which treatments are assigned to participants by chance rather than choice. This prevents bias in treatment assignment.
Placebo: An inactive substance that looks like the medicine being tested but contains no
active ingredient. Placebos are not used if they would put participants at risk by denying
effective therapy.
Blinded Studies: Studies designed to prevent bias by keeping certain information from
participants and/or researchers:
In single-blind studies, participants don't know which treatment they're receiving
In double-blind studies, neither participants nor researchers know which treatment is being given
Adverse Event: Any unfavorable change in health that happens during a clinical study.
Baseline: Information collected at the beginning of a study against which later data are
compared.
Biomarker: A biological molecule found in blood, other body fluids, or tissues that can be
measured and indicates a normal or abnormal process, condition, or disease.
Clinical Research Coordinator: A health professional who manages the day-to-day clinical trial activities at a research site.
Dosage: The amount of medicine or treatment given to a participant.
Efficacy: The ability of a treatment to produce the desired beneficial effect.
Eligibility Criteria: Requirements that must be met for an individual to participate in a clinical trial.
Endpoint: A measurable outcome defined in the trial protocol that is used to determine whether the treatment being studied is beneficial.
Exclusion Criteria: Factors that prevent a person from participating in a clinical trial.
Inclusion Criteria: Factors that allow a person to participate in a clinical trial.
Institutional Review Board (IRB): A committee that reviews and monitors research involving human subjects to ensure their protection.
Intervention: A treatment, drug, device, or procedure being studied.
Protocol: The written description of a clinical trial that includes the trial's objectives, design, and methods.
Principal Investigator (PI): The person responsible for the scientific and technical direction of the clinical trial.
Sponsor: The individual, company, institution, or organization responsible for initiating,
managing, or financing the clinical trial.
Standard of Care: The treatment that medical experts accept as appropriate for a certain type of disease and that healthcare professionals regularly provide.
Washout Period: A period during a clinical study when participants do not receive any
treatment to eliminate the effects of a previous treatment.
Randomization: The process by which treatments are assigned to participants by chance rather than choice. This prevents bias in treatment assignment.
Placebo: An inactive substance that looks like the medicine being tested but contains no
active ingredient. Placebos are not used if they would put participants at risk by denying
effective therapy.
Blinded Studies: Studies designed to prevent bias by keeping certain information from
participants and/or researchers:
In single-blind studies, participants don't know which treatment they're receiving
In double-blind studies, neither participants nor researchers know which treatment is being given
Adverse Event: Any unfavorable change in health that happens during a clinical study.
Baseline: Information collected at the beginning of a study against which later data are
compared.
Biomarker: A biological molecule found in blood, other body fluids, or tissues that can be
measured and indicates a normal or abnormal process, condition, or disease.
Clinical Research Coordinator: A health professional who manages the day-to-day clinical trial activities at a research site.
Dosage: The amount of medicine or treatment given to a participant.
Efficacy: The ability of a treatment to produce the desired beneficial effect.
Eligibility Criteria: Requirements that must be met for an individual to participate in a clinical trial.
Endpoint: A measurable outcome defined in the trial protocol that is used to determine whether the treatment being studied is beneficial.
Exclusion Criteria: Factors that prevent a person from participating in a clinical trial.
Inclusion Criteria: Factors that allow a person to participate in a clinical trial.
Institutional Review Board (IRB): A committee that reviews and monitors research involving human subjects to ensure their protection.
Intervention: A treatment, drug, device, or procedure being studied.
Protocol: The written description of a clinical trial that includes the trial's objectives, design, and methods.
Principal Investigator (PI): The person responsible for the scientific and technical direction of the clinical trial.
Sponsor: The individual, company, institution, or organization responsible for initiating,
managing, or financing the clinical trial.
Standard of Care: The treatment that medical experts accept as appropriate for a certain type of disease and that healthcare professionals regularly provide.
Washout Period: A period during a clinical study when participants do not receive any
treatment to eliminate the effects of a previous treatment.