Description
As part of FDA’s Real-World Evidence (RWE) Program, this draft guidance is intended to support the conduct of randomized controlled drug trials with streamlined protocols and procedures that focus on essential data collection, allowing integration of research into routine clinical practice. Such trials have sometimes been referred to as point of care trials or large simple trials. Like decentralized clinical trials, which aim to bring trial-related activities to patients’ homes or other convenient locations, such RCTs may improve convenience and accessibility for participants and allow for enrollment of more representative populations, resulting in more generalizable trial results. Leveraging established health care institutions and existing clinical expertise in the medical community can reduce startup times and speed up enrollment.
Scope & Applicability
Product Classes
1postmarketing studies for FDA-approved drugs
Stakeholders
7Entity responsible for submitting applications under section 524B
Responsible for qualifications, training, and trial conduct; Individual responsible for trial conduct and data governance at a site.; May delegate tasks but retains overall responsibility; Person responsible for the conduct of the clinical trial at a trial site; Responsible for trial conduct and participant safety; Responsible for trial conduct, data integrity, and investigational product management.; Individual responsible for trial conduct at a site and informing the institution.; maintaining
Veterinarian responsible for selecting anesthetic regimens and conducting field studies
HCPs providing trial-related activities at remote locations
Member of the investigation team
Source of safety information in clinical trials
Healthcare professionals performing trial activities locally
Regulatory Context
Attributes
3Data relating to patient health status/delivery of health care
Clinical evidence regarding usage and benefits/risks of a drug
identifying critical-to-quality factors (i.e., those that are likely to have a meaningful impact on participant's rights)
Related CFR Sections (13)
- 21CFR312.23§ 312.23 IND content and format.
(a) A sponsor who intends to conduct a clinical investigation subject to this part shall submit an “Investigational New Drug Application” (IND) including, in the following order:Read full regulation →
- 21CFR312.62§ 312.62 Investigator recordkeeping and record retention.
(a) Disposition of drug. An investigator is required to maintain adequate records of the disposition of the drug, including dates, quantity, and use by subjects. If the investigation is terminated, suspended, discontinued, or completed, the investigator shall return the unused supplies of the drug tRead full regulation →
- 21CFR312.57§ 312.57 Recordkeeping and record retention.
(a) A sponsor shall maintain adequate records showing the receipt, shipment, or other disposition of the investigational drug. These records are required to include, as appropriate, the name of the investigator to whom the drug is shipped, and the date, quantity, and batch or code mark of each such Read full regulation →
- 21CFR312.58§ 312.58 Inspection of sponsor's records and reports.
(a) FDA inspection. A sponsor shall upon request from any properly authorized officer or employee of the Food and Drug Administration, at reasonable times, permit such officer or employee to have access to and copy and verify any records and reports relating to a clinical investigation conducted undRead full regulation →
- 21CFR312.3§ 312.3 Definitions and interpretations.
(a) The definitions and interpretations of terms contained in section 201 of the Act apply to those terms when used in this part:Read full regulation →
- 21CFR312.60§ 312.60 General responsibilities of investigators.
An investigator is responsible for ensuring that an investigation is conducted according to the signed investigator statement, the investigational plan, and applicable regulations; for protecting the rights, safety, and welfare of subjects under the investigator's care; and for the control of drugs Read full regulation →
- 21CFR312.50§ 312.50 General responsibilities of sponsors.
Sponsors are responsible for selecting qualified investigators, providing them with the information they need to conduct an investigation properly, ensuring proper monitoring of the investigation(s), ensuring that the investigation(s) is conducted in accordance with the general investigational plan Read full regulation →
- 21CFR312.56§ 312.56 Review of ongoing investigations.
(a) The sponsor shall monitor the progress of all clinical investigations being conducted under its IND.Read full regulation →
- 21CFR50.20§ 50.20 General requirements for informed consent.
Except as provided in §§ 50.22 , 50.23 , and 50.24 , no investigator may involve a human being as a subject in research covered by these regulations unless the investigator has obtained the legally effective informed consent of the subject or the subject's legally authorized representative. An invesRead full regulation →
- 21CFR56.109§ 56.109 IRB review of research.
(a) An IRB shall review and have authority to approve, require modifications in (to secure approval), or disapprove all research activities covered by these regulations.Read full regulation →
- 21CFR56.111§ 56.111 Criteria for IRB approval of research.
(a) In order to approve research covered by these regulations the IRB shall determine that all of the following requirements are satisfied:Read full regulation →
- 21CFR312.66§ 312.66 Assurance of IRB review.
An investigator shall assure that an IRB that complies with the requirements set forth in part 56 will be responsible for the initial and continuing review and approval of the proposed clinical study. The investigator shall also assure that he or she will promptly report to the IRB all changes in thRead full regulation →
- 21CFR312.32§ 312.32 IND safety reporting.
(a) Definitions. The following definitions of terms apply to this section:Read full regulation →
Related Warning Letters (10)
- 2025-12-23
In Vivo Bioavailability-Bioequivalence Studies – Clinical
Maria A. Carballosa, M.D.
- 2025-12-09
Unapproved New Drug/Unlicensed Biological Product/Biologics License Application (BLA)
Celularity, Inc
- 2025-11-18
Sponsor/Investigator
Verdure Sciences, Inc.
- 2025-09-30
Clinical Investigator (Sponsor)
Pamela K. Den Besten, DDS, MS
- 2025-09-23
Clinical Investigator/Sponsor
Ralph A. DeFronzo, M.D.
- 2025-09-09
Clinical Investigator
Shirish M. Gadgeel, M.D.
- 2025-07-15
Clinical Investigator
Mark J. Savant, M.D
- 2025-07-01
Clinical Investigator
Peter Michael, M.D.
- 2025-06-10
Clinical Investigator (Sponsor)
American Behavioral Research Institute, LLC
- 2025-06-03
Bioresearch Monitoring Program
Amy Lightner, MD
See Also (8)
- Frequently Asked Questions — Developing Potential Cellular and Gene Therapy Products (Status: Draft)
- Institutional Review Boards Frequently Asked Questions: Guidance for Institutional Review Boards and Clinical Investigators (Status: Final)
- Content and Format of Investigational New Drug Applications (INDs) for Phase 1 Studies of Drugs, Including Well-Characterized, Therapeutic, Biotechnology-derived Products: Guidance for Industry (Status: Final)
- Guidance for Human Somatic Cell Therapy and Gene Therapy: Guidance for Industry (Status: Final)
- Drug Master Files for Bulk Antibiotic Drug Substances: Guidance for Industry (Status: Final)
- INDs for Phase 2 and Phase 3 Studies Chemistry, Manufacturing, and Controls Information: Guidance for Industry (Status: Final)
- Pharmacogenomic Data Submissions; Examples of Voluntary Submissions or Submissions Required Under 21 CFR 312, 314, or 601 (Status: Final)
- Pharmacogenomic Data Submissions: Guidance for Industry (Status: Final)