Description
This guideline is intended to assist an applicant in presenting the clinical and statistical data required as part of an application under 21 CFR 314.50. The guideline describes an acceptable format for organizing the clinical and statistical sections and presenting the clinical and statistical information and accompanying statistical documentation of a clinical trial. With respect to documenting the results of individual studies, the guideline describes a fully integrated clinical and statistical report rather than two separate reports.
Scope & Applicability
Product Classes
7Requirements for controlled clinical trials
control groups that are identified in regulations are... historical control
control groups that are identified in regulations are... active treatment concurrent control
control groups that are identified in regulations are placebo concurrent control
Products combining drug, device, or biological constituents; Generally recommended for Enhanced Documentation; Requires 14971-based framework incorporating ICH Q9; A drug-device combination where the device constituent part detects ingestion.
application is submitted for a drug product involving two or more drug substances
A situation requiring additional pediatric safety data collection.
Stakeholders
10Guidance for Sponsors, Investigators, and Institutional Review Boards
Roles and responsibilities of any data-monitoring board
Sponsor of the NDA
sponsor is requested to consult the biostatistical reviewer
statistical reviewers in CDER either treated all tumors as incidental
Required list for study documentation; Required list in Section II.A of the application
A group that may perform interim analyses of trial data.
Independent entity to assess safety and efficacy
If the study had an organizational structure in addition to the investigator (...data-monitoring committee...)
If the study had an organizational structure in addition to the investigator (safety committee...)
Regulatory Context
Attributes
10Determined during the planning of a research study
Determined by statistical considerations and experimental design.
Relevant characteristic of the study population
Particular variables that are considered primary end-points
Variable DIA77 recorded to nearest integer
Criteria for diagnosing anaphylaxis in infants, children, and adults.
Flat file containing primarily efficacy data
characterizing the safety of individual drugs
Evidence of benefit from controlled clinical trials.
data category for demographic information
Identified Hazards
Hazards
3Drug-related safety concern requiring specific exposure duration
risk assessment for pharmaceuticals affecting the immune system
Potential harm from overdose due to dosing schedule deviation
Related CFR Sections (6)
- 21CFR314.50§ 314.50 Content and format of an NDA.
NDAs and supplements to approved NDAs are required to be submitted in the form and contain the information, as appropriate for the particular submission, required under this section. Three copies of the NDA are required: An archival copy, a review copy, and a field copy. An NDA for a new chemical enRead full regulation →
- 21CFR314.80§ 314.80 Postmarketing reporting of adverse drug experiences.
(a) Definitions. The following definitions of terms apply to this section:Read full regulation →
- 21CFR314.125§ 314.125 Refusal to approve an NDA.
(a) The Food and Drug Administration will refuse to approve the NDA and for a new drug give the applicant written notice of an opportunity for a hearing under § 314.200 on the question of whether there are grounds for denying approval of the NDA under section 505(d) of the Federal Food, Drug, and CoRead full regulation →
- 21CFR314.101§ 314.101 Filing an NDA and receiving an ANDA.
(a) Filing an NDA.Read full regulation →
- 21CFR314.126§ 314.126 Adequate and well-controlled studies.
(a) The purpose of conducting clinical investigations of a drug is to distinguish the effect of a drug from other influences, such as spontaneous change in the course of the disease, placebo effect, or biased observation. The characteristics described in paragraph (b) of this section have been develRead full regulation →
- 21CFR300.50§ 300.50 Fixed-combination prescription drugs for humans.
The Food and Drug Administration's policy in administering the new-drug, antibiotic, and other regulatory provisions of the Federal Food, Drug, and Cosmetic Act regarding fixed combination dosage form prescription drugs for humans is as follows:Read full regulation →
Related Warning Letters (10)
- 2025-09-09
Clinical Investigator
Shirish M. Gadgeel, M.D.
- 2025-07-15
Clinical Investigator
Mark J. Savant, M.D
- 2025-05-20
Bioresearch Monitoring Program/Institutional Review Board (IRB)
United Health Products, Inc.
- 2025-03-25
Clinical Investigator
Americo F. Padilla, M.D.
- 2024-12-03
Sponsor
Applied Therapeutics, Inc.
- 2024-11-05
Institutional Review Board (IRB)
Armstrong County Memorial Hospital
- 2024-10-22
Clinical Investigator
Namita A. Goyal, M.D.
- 2024-10-08
Institutional Review Board (IRB)
Louisiana State University Health Science Center IRB
- 2024-07-16
Bioresearch Monitoring Program/IRB
Massachusetts Institute of Technology MIT
- 2024-06-18
Clinical Investigator (Sponsor)
Angela D. Ritter, M.D.
See Also (8)
- Guidance for Review Staff and Industry Good Review Management Principles and Practices for PDUFA Products (Status: Draft)
- Labeling for Bronchodilators: Cold, Cough, Allergy, Bronchodilator, And Antiasthmatic Drug Products for Over-the-Counter Human Use (Small Entity Compliance Guide): Guidance for Industry (Status: Final)
- Nasal Spray and Inhalation Solution, Suspension, and Spray Drug Products--Chemistry, Manufacturing, and Controls Documentation: Guidance for Industry (Status: Final)
- Food-Effect Bioavailability and Fed Bioequivalence Studies: Guidance for Industry (Status: Final)
- Changes to an Approved NDA or ANDA: Guidance for Industry (Status: Final)
- FDA Review of Vaccine Labeling Requirements for Warnings, Use Instructions, and Precautionary Information: Guidance for Industry (Status: Final)
- Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments (Status: Final)
- Annual Reportable Labeling Changes for New Drug Applications and Abbreviated New Drug Applications for Nonprescription Drug Products (Status: Draft)