What is the FDA? What Does FDA Do in Clinical Trials?

FDA in clinical trials refers to the regulatory role played by the U.S. Food and Drug Administration (FDA) in ensuring the safety, integrity, and ethical conduct of drug development in the United States.

Clinical trial sponsors operate in a complex regulatory environment. Understanding the FDA’s role in clinical trials is crucial for success. The U.S. Food and Drug Administration (FDA) oversees drug development to ensure patient safety and data integrity. Sponsors must navigate requirements like filing an Investigational New Drug (IND) application, adhering to Good Clinical Practice (GCP) guidelines, and meeting all regulatory milestones on the path to FDA approval.

FDA’s Role in Clinical Trials

The FDA plays a pivotal role in every U.S. clinical trial, acting as the guardian of participant safety and the credibility of trial data. By law, any new drug must be authorized by the FDA before it can be tested in humans or distributed across state lines. A sponsor cannot legally ship an investigational drug to trial sites without an approved IND – the IND serves as an exemption allowing the trial to proceed. FDA oversight begins at the moment a drug is ready to enter human testing. From that point on, the FDA’s mission is to ensure that the trial is conducted ethically and that the evidence collected will reliably demonstrate the drug’s safety and efficacy.

How does the FDA enforce these standards? Primarily through a framework of regulations and guidance. The FDA requires sponsors and investigators to comply with applicable statutes and regulations intended to protect the rights, safety, and welfare of participants and to ensure data quality. Key FDA regulations cover everything from informed consent and Institutional Review Boards (IRBs) to IND applications and safety reporting.

IND Application – The First Step toward FDA Approval

For sponsors, the journey toward FDA approval officially begins with the Investigational New Drug (IND) application. The IND is a comprehensive dossier submitted to the FDA before starting any clinical trial of a new drug or biologic. Its purpose is to demonstrate that it’s reasonable to proceed with human testing.

What does an IND include?

• Preclinical Data (Animal Pharmacology and Toxicology)
• Manufacturing Information
• Clinical Protocols and Investigator Information

Once the IND is submitted, a sponsor must wait 30 days before initiating the trial. During this 30-day FDA review period, the agency evaluates the IND for safety. If there are serious concerns, the FDA can issue a clinical hold.

Pro Tip: Engage with the FDA early. The FDA encourages sponsors to use its Pre-IND Consultation Program, which allows for early communication with FDA reviewers. Studying relevant FDA guidance documents is invaluable.

GCP Compliance and Sponsor Responsibilities

Filing an IND is just the beginning. Once your trial is underway, Good Clinical Practice (GCP) principles govern the conduct of the study. GCP is an international ethical and scientific quality standard for designing, conducting, recording, and reporting trials.

Key sponsor responsibilities under FDA regulations:

• Select qualified investigators
• Provide necessary information to investigators
• Ensure proper monitoring of the trial
• Conduct the study according to protocol
• Maintain an effective IND
• Inform the FDA and investigators of new adverse effects or risks

GCP compliance also includes ensuring that informed consent is obtained and that an Institutional Review Board (IRB) reviews and approves the study.

As a sponsor, ensuring that trial participants are appropriately pre-screened and referred can significantly impact the quality and compliance of your study. DecenTrialz enables a structured pre-screening and referral workflow that aligns with regulatory expectations. Protocol criteria are transformed into a guided format, participants complete eConsent digitally, and a registered nurse follows up to validate study-related details. Only qualified individuals progress, resulting in a more efficient, compliant, and site-ready handoff that supports your regulatory and operational goals.

From IND to FDA Approval: Regulatory Pathway

1. Clinical Trial Phases (Phase 1–3): Conducted with FDA oversight, these studies evaluate safety, dosage, and efficacy. Sponsors must submit annual IND reports and monitor for adverse events throughout.
2. Ongoing FDA Oversight: Includes required updates such as protocol amendments, safety reports, and communication with FDA through formal milestone meetings (e.g., End-of-Phase 2).
3. New Drug Application (NDA) or Biologics License Application (BLA): After completing Phase 3, sponsors submit an NDA (for drugs) or BLA (for biologics). This comprehensive application includes all preclinical and clinical trial data, proposed labeling, safety updates, and manufacturing information. The NDA is mandatory for gaining FDA marketing approval in the U.S.
4. FDA Review and Decision: The FDA thoroughly evaluates the NDA or BLA over a 10–12 month review period. This includes expert analysis of trial data, inspection of manufacturing sites, and assessment of risk-benefit profiles. The FDA may approve, issue a Complete Response Letter (CRL), or request additional information.

Proactive sponsors stay engaged with FDA officials and use formal meetings (e.g., Pre-IND, End-of-Phase2) to align with regulatory expectations.

Navigating Your Clinical Trial Roadmap

Successfully navigating FDA requirements is a challenging but essential task for sponsors. Understanding the FDA’s role, meeting IND and GCP requirements, and preparing for each regulatory milestone will help ensure your clinical trial is conducted ethically, efficiently, and effectively. Stay informed with the latest FDA guidance documents, and consult regulatory experts when needed to strengthen your path to approval.

Successfully navigating FDA requirements is a challenging but essential task for sponsors. Understanding the FDA’s role, meeting IND and GCP requirements, and preparing for each regulatory milestone will help ensure your clinical trial is conducted ethically, efficiently, and effectively. Stay informed with the latest FDA guidance documents, and consult regulatory experts when needed to strengthen your path to approval, or explore FDA’s Drug Development Resources for deeper insights.