FDA Guidance Documents

Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers

FDA's Center for Drug Evaluation and Research issued guidance (Docket FDA-2013-D-0446) clarifying expanded access regulations for investigational drugs under 21 CFR part 312, subpart I, addressing frequently asked questions from industry, researchers, physicians, institutional review boards, and patients. The guidance incorporates statutory requirements from the 21st Century Cures Act and FDA Reauthorization Act of 2017. The document is available for ongoing public comment through the regulations.gov docket system.

Draft Compliance Policy for NIOSH-Approved Respirators Under 21 CFR 878.4040 and 880.6260

FDA's Center for Devices and Radiological Health (CDRH) has issued draft guidance establishing a proposed compliance policy for respirators approved by CDC NIOSH under 42 CFR part 84. The draft applies to surgical N95 respirators and other NIOSH-approved air-purifying respirators, including PAPRs, non-powered FFRs, elastomeric respirators, and FFRs for general public use in public health emergencies. FDA seeks public comments on the compliance policy by June 19, 2026.

Study Data Technical Conformance Guide Technical Specifications Document

FDA issued technical specifications for study data conformance to guide sponsors and applicants on formatting study data submissions for drug and biologics regulatory review. The guidance applies to regulated product submissions under FDA jurisdiction. It represents current FDA thinking on technical data standards without creating new legal obligations.

Safety Assessment of Genome Editing Human Gene Therapy Products Using Next-Generation Sequencing

FDA's Center for Biologics Evaluation and Research has issued draft guidance recommending next-generation sequencing (NGS)-based methods for assessing safety of human genome editing gene therapy products in nonclinical studies. The guidance addresses evaluation of off-target editing risks and genome integrity for products supporting IND and BLA applications. Comments are being accepted via regulations.gov under docket FDA-2026-D-1255.

Bioanalytical Method Validation for Biomarker Concentrations Guidance

FDA's Center for Drug Evaluation and Research issued guidance FDA-2017-D-6821 to help sponsors validate bioanalytical methods used to evaluate biomarker concentrations in drug and biologic development programs. The guidance applies to sponsors of investigational new drug applications (INDs), new drug applications (NDAs), biologics license applications (BLAs), and abbreviated new drug applications (ANDAs). It addresses method validation for biomarker analysis in both clinical study samples and nonclinical study samples.

Guidance on Incorporating Voluntary Patient Preference Information

The FDA has issued updated guidance on incorporating voluntary patient preference information (PPI) into medical device submissions. This guidance provides recommendations on methods and contexts for collecting and submitting PPI to aid FDA decision-making throughout the total product lifecycle for biologics and medical devices.

FDA Guidance on Pyrogen and Endotoxin Testing

The FDA has issued guidance for biological product, drug, and device firms regarding pyrogen and endotoxin testing. This document provides recommendations based on current FDA thinking concerning USP and AAMI standards for bacterial endotoxin and pyrogen tests.

FDA Final Guidance on Topical Drug Product Characterization for ANDAs

The FDA has issued final guidance for industry on the physicochemical and structural characterization of topical drug products submitted in Abbreviated New Drug Applications (ANDAs). This guidance assists applicants in identifying generic topical products and supporting bioequivalence demonstrations. It finalizes the draft guidance issued in October 2022.

FDA Guidance on Medical Devices for Weight Loss

The FDA has issued guidance for medical devices intended for weight loss, outlining recommendations for non-clinical testing and clinical study design to support premarket submissions. This guidance aims to promote consistency and facilitate efficient review of submissions for devices related to weight loss, reduction, management, or obesity treatment.

FDA Guidance: Flavored ENDS Premarket Applications

The FDA has issued guidance to assist with premarket tobacco product applications (PMTAs) for flavored electronic nicotine delivery systems (ENDS). This guidance, intended to improve submission and review efficiency, focuses on considerations related to youth risk. The document is non-binding and aims to inform potential future rulemaking.

FDA Q&A on Biosimilar Development and BPCI Act

The FDA has reissued its final guidance on biosimilar development and the BPCI Act, solely to withdraw three specific Q&As (I.8, I.10, and I.19). The agency is continuing to evaluate other Q&As for potential updates.

FDA Draft Q&As on Biosimilar Development and BPCI Act

The FDA has issued a draft guidance document providing updated answers to common questions regarding biosimilar development and the Biologics Price Competition and Innovation Act (BPCI Act). This revision replaces a previous draft and focuses on specific Q&As related to the abbreviated licensure pathway for biological products.

Draft Guidance: Responding to FDA Form 483 Observations for Drug Manufacturers

The FDA has issued draft guidance for drug manufacturers on how to respond to observations noted on FDA Form 483 following CGMP inspections. This guidance is intended to assist domestic and foreign manufacturers in assessing conformity with CGMP requirements.

FDA Guidance on Real-World Data for Medicine Safety Assessment

The FDA has issued final guidance on using real-world data for medicine safety assessments, replacing a previous draft and withdrawing an older guidance. This document provides principles for planning, designing, analyzing, and reporting non-interventional studies utilizing real-world data for drugs and biological products.

FDA Final Guidance on Post-Approval Safety Data Reporting

The FDA has issued final guidance on post-approval safety data reporting, updating standards for managing and reporting individual case safety reports. This guidance clarifies the use of new data sources and replaces previous versions from 2003 and a draft from March 2024.

FDA Draft Guidance on 3-Year Exclusivity for Drug Products

The FDA has issued draft guidance to assist applicants requesting 3-year exclusivity for drug products. The guidance, available for public comment, clarifies the statutory and regulatory criteria for eligibility and provides recommendations on request content and format. This aims to support the Drug Competition Action Plan.

FDA Draft Guidance on Medical Gases Certification Process

The FDA has issued draft guidance for industry on the certification process for designated medical gases. This guidance explains the certification process and annual reporting requirements, aiming to reduce regulatory burden for the medical gas industry. Comments are due by April 13, 2026.

FDA Guidance: Plausible Mechanism Framework for Individualized Genetic Therapies

The FDA has issued draft guidance outlining a plausible mechanism framework for developing individualized genetic therapies. This framework provides recommendations for generating substantial evidence of effectiveness and safety for these novel treatments. The comment period for this draft guidance closes on April 27, 2026.

FDA Draft Guidance Q&A on Food Traceability Records

The FDA has issued draft guidance in a Q&A format to help industry understand the requirements for additional traceability records for certain foods, as established by a final rule in 2022. Comments on this draft guidance are due by May 21, 2026.

FDA Guidance on Antimicrobial Drug Durations for Food Animals

The FDA's Center for Veterinary Medicine has issued guidance for sponsors of new animal drug applications concerning medically important antimicrobial drugs used in food-producing animals. The guidance aims to facilitate voluntary changes to define durations of use for these drugs to mitigate antimicrobial resistance.