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Regulation, Safety, and Ethics of Clinical Trials

Table of Contents:
Regulatory Oversight
The Rights and Safety of Clinical Trial Participants

Regulatory Oversight of Clinical Trials

The Food and Drug Administration (FDA)

The FDA’s mission is to “protect public health by ensuring the safety, efficacy, and security of human and veterinary drugs, biological products, and medical devices.” The FDA does not develop new treatments or conduct clinical trials; they oversee the researchers and organizations that do.

What is the role of the FDA in clinical trials?

The FDA's Center for Drug Evaluation and Research (CDER) is responsible for ensuring the safety and effectiveness of all drugs marketed in the country. The FDA and the CDER protect the integrity and reliability of clinical research by ensuring clinical trials are designed and conducted in a manner that promotes the safety and ethical protection of study participants.


Office for Human Research Protections (OHRP)

The Office for Human Research Protections (OHRP) is a division of the U.S. Department of Health and Human Services (DHHS) that protects the rights, welfare, and well-being of people involved in clinical trials. The OHRP is also responsible for ensuring that clinical research follows 45 CFR 46 (Protection of Human Subjects) law. This law serves to protect human subjects from risks in research studies that any federal agency or department has a part in.


Institutional Review Boards (IRBs)

An IRB is a committee composed of physicians, statisticians, researchers, and community members that reviews all research projects to ensure they are ethical. IRBs approve or reject initial applications from the researchers before the study begins and periodically monitor and review the clinical trial for the duration of the study.

What are the roles and responsibilities of an IRB?

The primary responsibility of IRBs is to protect the rights of human research participants. IRBs use a group process to review the clinical trial protocol and all related materials, such as informed consent forms, to ensure the rights and welfare of the participants are protected.

Why are IRBs required?

The National Research Act of 1974 was signed into law in response to the unethical, inhumane Tuskegee Syphilis Study. This law required the establishment of IRBs at any Institute that conducts research involving human participants and receives federal support.

Watch this video to learn more about an institutional review board (IRB) and how IRBs protect people who participate in research.

Data Safety and Monitoring Boards (DSMBs)

Data Safety and Monitoring Boards (DSMBs) are a group of statisticians and clinical investigators, who are not directly involved with the study, that monitor the safety and scientific integrity of a clinical trial. All members of a DSMB are prohibited from having direct involvement in the conduct of the study. DSMBs are responsible for performing periodic analyses of the clinical trial data in order to decide whether or not to continue, modify, or stop a trial.

The DSMB can recommend to the sponsor that the trial be stopped if:

  • It is not effective,

  • It poses clear harm to participants, or

  • It is unlikely to serve its scientific purpose.

Overall, DSMBs provide an additional layer of oversight to clinical trials to ensure the safety and welfare of study participants.


The Rights and Safety of Clinical Trial Participants

Good Clinical Practice

The FDA defines Good Clinical Practice (GCP) as “an international ethical and scientific quality standard for designing, conducting, recording and reporting trials that involve the participation of human subjects.” GCP guidelines ensure that the rights and well-being of patients are protected when conducting clinical research.

The following video briefly explains Good Clinical Practice (GCP) guidelines.


Informed Consent

Informed consent is a process used by researchers to communicate information about the clinical study to participants so they can make an informed decision about whether or not to enroll or continue to participate in the study. The goal of the process is to protect participants by ensuring they understand all of the details of the study, including the risks and benefits before they agree to participate.

Thus, “informed consent” means that the research participants must be fully informed and understand all aspects of the research, including: 

  • Nature and objectives of the study

  • The study’s treatments and tests 

  • Alternative treatment options that could be available to them

  • Potential risks and benefits of participating in the study

  • How the data will be collected and stored

  • Who will have access to the data, and how long the data will be stored

  • Methodology of the actual study

Before enrolling in a study, potential participants must sign an informed consent form to confirm that they have been fully informed about all aspects of the study.

Sample Informed Consent Form:

Informed_Consent Template
Download PDF • 109KB

In summary, protecting research participants and ensuring the integrity of the data collected are the guiding principles for clinical trial oversight and regulation. The multiple forms of federal regulation of clinical trials we outlined above exist to significantly protect the health, safety, and welfare of all human research subjects and the patients who will take the drugs resulting from clinical trials.

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