On mobile, the table below is best viewed by scrolling sideways.
| Title of guidance | Type and level of guidance | About the guidance | Source |
|---|---|---|---|
| Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments | Draft guidance | This guidance (Guidance 3) is the third in a series of four methodological patient-focused drug development (PFDD) guidance documents that describe how stakeholders (patients, caregivers, researchers, medical product developers, and others) can collect and submit patient experience data and other relevant information from patients and caregivers to be used for medical product development and regulatory decision-making. When finalized, Guidance 3 will represent the current thinking of CDER, CBER, and CDRH on this topic. You can view a guidance snapshot here and listen to a guidance recap podcast here. | FDA |
| Bladder Cancer: Developing Drugs and Biologics for Adjuvant Treatment | Final guidance | This guidance provides recommendations to sponsors regarding the development of drugs and biological products regulated by CDER and CBER for the adjuvant treatment of muscle-invasive bladder cancer. The guidance includes recommendations regarding eligibility criteria, choice of comparator, follow-up imaging assessments, determination of disease recurrence, analyses of disease-free survival (DFS), and interpretation of trial results. Although FDA may consider endpoints other than DFS for the adjuvant treatment of muscle-invasive bladder cancer, this guidance is focused on clinical trials with DFS as the primary efficacy endpoint. | FDA |
| Renal Cell Carcinoma: Developing Drugs and Biologics for Adjuvant Treatment | Final guidance | This guidance provides recommendations to sponsors regarding the development of drugs and biological products regulated by CDER and CBER for the adjuvant treatment of renal cell carcinoma. The guidance includes recommendations regarding eligibility criteria, choice of comparator, follow-up imaging assessments, determination of disease recurrence, analyses of disease-free survival (DFS), and interpretation of trial results. Although FDA may consider endpoints other than DFS for the adjuvant treatment of renal cell carcinoma, this guidance is focused on clinical trials with DFS as the primary efficacy endpoint. | FDA |
| Providing Regulatory Submissions in Alternate Electronic Format Guidance for Industry | Final, Level 2 revised guidance | This guidance provides recommendations on an alternate electronic format for submissions covered under an exemption from or a waiver of the requirements of section 745A(a) of the FD&C Act). These recommendations pertain to the format of content contained in new drug applications (NDAs), abbreviated new drug applications (ANDAs), certain drug master files (DMFs), certain biologics license applications (BLAs), and certain investigational new drug applications (INDs) submitted to CDER or to CBER. Sponsors and applicants who receive an exemption or a waiver from filing in eCTD format under section 745A(a) of the FD&C Act should still provide those exempted or waived submissions electronically. This recommendation is consistent with the efforts of Federal Agencies to transition their business processes and recordkeeping to a fully electronic environment. | FDA |
| Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics | Draft guidance | This guidance provides recommendations: · to assist industry in the development of oligonucleotide therapeutics under section 505 of the FD&C Act and 21 CFR parts 312 and 314. Specifically, this guidance represents the FDA’s recommendations for certain evaluations including pharmacokinetic, pharmacodynamic, and safety assessments during oligonucleotide therapeutic development, including: (1) characterizing the potential for QTc interval prolongation, (2) performing immunogenicity risk assessment, (3) characterizing the impact of hepatic and renal impairment, and (4) assessing the potential for drug-drug interactions. · on when to conduct these assessments and what types of assessments are suitable to address these questions. The recommendations in this guidance generally apply to oligonucleotide therapeutics that use an RNA-centric mechanism of action. Providing recommendations based on any specific characteristics (e.g., backbone modification, specific conjugation) is beyond the scope of this guidance. This guidance is based on the knowledge gained in the development of oligonucleotide therapeutics submitted to the Agency in new drug applications (NDA) as of the date of this guidance. As the development of oligonucleotide therapeutics evolve (e.g., chemical modifications to the base and/or backbone, structure, delivery strategy), sponsors should contact appropriate review Divisions for questions related to the topics in Sections II.A through D of this guidance. | FDA |
| Non-Penicillin Beta-Lactam Drugs: A CGMP Framework for Preventing Cross-Contamination | Final, Level 1 revised guidance | This guidance: · describes methods, facility design elements, and controls that are important in preventing drugs from being cross-contaminated with compounds containing a beta-lactam ring. · also provides information regarding the relative health risk of, and the potential for, cross-reactivity in the classes of non-penicillin beta-lactam antibacterial drugs and non-antibacterial beta-lactam compounds. · recommends complete and comprehensive separation of the manufacturing operations of non-penicillin beta-lactam antibacterial drugs from the manufacturing operations of other drugs. · provides (for manufacturers of non-antibacterial beta-lactam compounds), recommendations on cross-contamination prevention strategies, including examples of relevant design features and control approaches for those seeking to justify a cross-contamination prevention strategy other than complete and comprehensive separation when appropriate. | FDA |
| Considerations for Rescinding Breakthrough Therapy Designation | Draft guidance | This guidance explains how, during its evaluation of a drug development program, FDA may consider whether to rescind a breakthrough therapy designation (BTD). This guidance is consistent with, and supplements the information on BTD contained in the guidance for industry Expedited Programs for Serious Conditions––Drugs and Biologics and other BTD policies and procedures of CDER and CBER. | FDA |
| Assessing the Effects of Food on Drugs in INDs and NDAs – Clinical Pharmacology Considerations | Final guidance | This guidance provides recommendations to sponsors planning to conduct food-effect (FE) studies for orally administered drug products under investigational new drug applications (INDs) to support new drug applications (NDAs) and supplements to these applications for drugs being developed under section 505 of the FD&C Act (21 U.S.C. 355). This guidance revises and replaces part of the 2002 FDA guidance entitled Food Effect Bioavailability and Fed Bioequivalence Studies (December 2002). Information on fed bioequivalence (BE) studies to be submitted in abbreviated new drug applications (ANDAs) is now found in the FDA guidance entitled Bioequivalence Studies with Pharmacokinetic Endpoints for Drugs Submitted Under an ANDA (August 2021).3 Specific recommendations concerning fed comparability studies are now described in the FDA guidance entitled Bioavailability Studies Submitted in NDAs or INDs — General Considerations (April 2022). Here you can view a snapshot of the guidance or listen to a guidance re3cap podcast. | FDA |
| Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies | Draft guidance | This guidance describes a standards recognition program for regenerative medicine therapies (SRP-RMT) at FDA’s CBER designed to identify and recognize Voluntary Consensus Standards (VCS) to facilitate the development and assessment of regenerative medicine therapy (RMT) products regulated by CBER when such standards are appropriate. CBER encourages the use of appropriate standards in the development of CBER-regulated products. The use of recognized VCS can assist stakeholders in more efficiently meeting regulatory requirements and increasing regulatory predictability for RMT products. This program is modeled after the formal standards and conformity assessment program or S-CAP for medical devices. | FDA |
| Development of Therapeutic Protein Biosimilars: Comparative Analytical Assessment and Other Quality-Related Considerations Guidance for Industry | Draft guidance | This guidance describes the Agency’s recommendations on the design and evaluation of comparative analytical studies intended to support a demonstration that a proposed therapeutic protein product is biosimilar to a reference product licensed under section 351(a) of the Public Health Service Act (PHS Act). Additionally, this guidance is intended to provide recommendations to sponsors on the scientific and technical information for the chemistry, manufacturing, and controls (CMC) portion of a marketing application for a proposed product submitted under section 351(k) of the PHS Act. This guidance is one in a series of guidances that FDA is developing to facilitate implementation of the Biologics Price Competition and Innovation Act of 2009 (BPCI Act). | FDA |
| Q9(R1) Quality Risk Management | Draft guidance | The purpose of this document is to offer a systematic approach to quality risk management for better, more informed, and timely decisions. It serves as a foundation or resource document that is independent of, yet supports, other ICH Quality documents and complements existing quality practices, requirements, standards, and guidelines within the pharmaceutical industry and regulatory environment. It specifically provides guidance on the principles and some of the tools of quality risk management that can enable more effective and consistent risk based decisions, both by regulators and industry, regarding the quality of drug substances and drug (medicinal) products across the product lifecycle. It is not intended to create any new expectations beyond the current regulatory requirements. | FDA |
| Advanced Prostate Cancer: Developing Gonadotropin-Releasing Hormone Analogues Guidance for Industry | Final, Level 1 guidance | This guidance describes the FDA’s current recommendations regarding the overall development program to establish the effectiveness and safety of gonadotropin-releasing hormone (GnRH) analogues for treating advanced prostate cancer. The recommendations in section III.C., Registrational Trial Considerations, apply to drug product development programs for GnRH analogues in advanced prostate cancer for all dosage forms and routes of administration (e.g., tablets, capsules, injectable suspensions, injectable emulsions, subcutaneous implants). Other sections apply only to extended-release injectable dosage forms. | FDA |
| Antibacterial Therapies for Patients With an Unmet Medical Need for the Treatment of Serious Bacterial Diseases – Questions and Answers (Revision 1) | Draft guidance | This draft guidance: · is intended to assist sponsors in the clinical development of new antibacterial drugs. Specifically, the guidance explains the FDA’s current thinking about possible development programs and clinical trial designs for antibacterial drugs to treat serious bacterial diseases in patients with an unmet medical need, including patients with a serious bacterial disease for which effective antibacterial drugs are limited or lacking. Antibacterial drugs that are active against only a single species or few species within a genus of bacteria can be developed for the treatment of serious bacterial diseases in patients with an unmet medical need. For products that have the potential to address an unmet medical need, a more flexible development program may be acceptable to facilitate development. · revises the guidance for industry Antibacterial Therapies for Patients With an Unmet Medical Need for the Treatment of Serious Bacterial Diseases (August 2017). After it has been finalized, this draft guidance will replace the August 2017 guidance. Significant changes in this draft guidance from the 2017 version include the possibility to conduct noninferiority trials that include subjects with infections caused by certain drug-resistant pathogens since effective active controls are now available. More detail is also provided for the currently used noninferiority trial designs that may be used with a wider noninferiority margin, including cases for which the trial population is enriched for subjects with infections caused by certain drug-resistant organisms. · does not contain discussion of the general issues of statistical analysis or clinical trial design. Those topics are addressed in the ICH guidances for industry E9 Statistical Principles for Clinical Trials (September 1998) and E10 Choice of Control Group and Related Issues in Clinical Trials (May 2001), respectively. | FDA |
| Risk Management Plans to Mitigate the Potential for Drug Shortages | Draft guidance | This guidance is intended to help stakeholders develop, maintain, and implement risk management plans (RMPs) to proactively assist in the prevention of human drug product and biological product shortages. RMPs can provide stakeholders with a framework to proactively identify, prioritize, and implement strategies to mitigate hazards that can cause a supply disruption. Such a supply disruption may lead to a drug shortage. Effective quality risk management can facilitate better, more informed decisions; can provide FDA with greater assurance that stakeholders understand and can manage the associated risks; and can potentially affect the extent and level of direct regulatory oversight. Based on recent publications and reports, the majority of drug shortages are associated with quality issues. This guidance describes a framework for stakeholders to consider when developing RMPs that aligns with principles stated in the International Council for Harmonisation (ICH) guidance for industry Q9 Quality Risk Management (June 2006). In addition, FDA also recommends risk factors to consider when developing the content of the RMPs. | FDA |
| E2B(R3) Electronic Transmission of Individual Case Safety Reports (ICSRs) Implementation Guide – Data Elements and Message Specification | Final guidance | This document is a guide for implementing the standard adopted by the ICH1 for electronic transmission of Individual Case Safety Reports (ICSRs) according to the ICH E2B(R3) message standard. Conceptually, an ICSR is a report of information describing adverse event(s) / reaction(s) experienced by an individual patient. This ICH IG focuses on medicinal products and therapeutic biologics for human use. This IG is also intended to support the implementation of software and tools for creating, editing, sending and receiving electronic ICSR messages This IG is not intended to serve as a reference for proper pharmacovigilance practices nor is it intended to explain the underlying scientific or medical issues that support the collation, categorisation or analysis of medicinal product safety information. It is also not intended to explain the rationale that underlies proper case safety reporting. The focus of this ICH IG is on technical implementation. Thus, the intended audience includes system developers, IT professionals, system implementers and system users who need to understand the technical requirements for constructing and using valid electronic messages to transmit ICSRs. This IG provides the information necessary to support the development of adequate informatics tools (e.g. forms and interfaces for end user data entry) as well as technical requirements to design style sheets, conduct data transformations and code well-formed messages. However, this IG does not provide or infer guidance or recommendations for any particular database technology or software platform. Instead, this IG describes the technical requirement to generate valid XML code according to the standard outlined in this IG. Subsequent sections of this IG provide explanatory text concerning the business context for electronic ICSR messaging, including ICH documentation, and application to pharmacovigilance transactions. | FDA |
| Appendix I (B) to the ICH E2B(R3) ICSRs Implementation Guide Backwards and Forwards Compatibility | Final guidance | This document is an appendix to the Implementation Guide (IG) for the ‘International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) Electronic Transmission of Individual Case Safety Reports (ICSRs). This Appendix is intended to assist reporters and recipients (including pharmaceutical companies, authorities and non-commercial sponsors) in implementing systems with special focus on the recommendations for conversion back and forth between the previous standard, i.e., E2B(R2) and this standard, i.e., E2B(R3). The evolution of the guideline, from E2B(R2) to E2B(R3), has the consequence that ICSRs cannot be perfectly converted from one standard version to the other (either backwards or forwards). Repeated conversion could result in transformation or loss of information. Therefore receivers need to evaluate contents carefully. This document presents the recommendations for conversion agreed within ICH so as to provide a reference to system providers, and a common understanding on the way to convert ICSRs and ICSR acknowledgments (ICSR ACKs) between E2B(R2) and E2B(R3). | FDA |
| Safety Considerations for Container Labels and Carton Labeling Design to Minimize Medication Errors | Final guidance | The purpose of this guidance is to help human prescription drug and biological product sponsors, application holders, and applicants minimize medication errors associated with their products. This guidance: · focuses on safety aspects of the application holder’s container label and carton labeling design. It provides a set of principles and recommendations for ensuring that critical elements of a product’s container label and carton labeling are designed to promote safe dispensing, administration, and use of the product. · applies to human prescription drug and biological products, including the following: · Prescription drug products marketed under an approved new drug application or abbreviated new drug application · Prescription drugs marketed without an approved application · Biological products marketed under an approved biologics license application | FDA |
| Assessing User Fees Under the Generic Drug User Fee Amendments of 2017 | Final Level 1 revised guidance | This guidance provides stakeholders information regarding FDA’s implementation of the Generic Drug User Fee Amendments of 2017 (GDUFA II) under Title III of the FDA Reauthorization Act of 2017.2 Because GDUFA II created changes to the user fee program, this guidance serves to provide an explanation about the new fee structure and types of fees for which entities are responsible. This guidance: · describes the types of user fees authorized by GDUFA II, the process for submitting payments to FDA, the consequences for failing to pay generic drug user fees, and the process for requesting a reconsideration of a user fee assessment. · also describes how FDA determines affiliation for purposes of assessing generic drug user fees. FDA will issue separate guidance documents regarding GDUFA II non-user fee requirements and processes. · does not address how FDA determines and adjusts fees for each fiscal year, nor does it address FDA’s implementation of other user fee programs (e.g., under the PDUFA or BSUFA. Throughout this guidance, references to user fees or the user fee program indicate generic drug user fees assessed and collected under section 744B of the FD&C Act. | FDA |
| Considerations for Waiver Requests for pH Adjusters in Generic Drug Products Intended for Parenteral, Ophthalmic, or Otic Use | Draft guidance for industry | This guidance: · describes how the FDA intends to evaluate a request for a waiver, with regard to a pH adjuster, under 21 CFR 314.99(b) (hereinafter waiver) of the requirement in CFR 314.94(a)(9)(iii) and (iv) that a drug product intended for parenteral, ophthalmic, or otic use generally “must contain the same inactive ingredients and in the same concentration as the reference listed drug identified by the applicant.” · also provides recommendations regarding the timing and process for requesting such a waiver of the requirement in § 23 314.94(a)(9)(iii) and (iv) (waiver request). · is intended to assist abbreviated new drug application (ANDA)2 25 applicants that reference a reference listed drug (RLD) intended for parenteral, ophthalmic, or otic use but are seeking approval of a drug that is qualitatively (Q1) different or quantitatively (Q2) different from the RLD with respect to a pH adjuster(s). · is intended to identify the type of information FDA may generally consider in evaluating a waiver request for pH adjusters in generic drug products intended for parenteral, ophthalmic, or otic use and provide recommendations to ANDA applicants regarding the submission and content of such a waiver request. The recommendations in this guidance are limited to inactive ingredients in ANDAs that adjust he pH of a drug product intended for parenteral, ophthalmic, or otic use, and do not apply to other inactive ingredients. | FDA |
| Investigating Out-of-Specification (OOS) Test Results for Pharmaceutical Production – Level 2 revision | Final Level 2 revised guidance | This guidance: · for industry provides the Agency’s current thinking on how to evaluate out-of specification (OOS) test results. For the purposes of this document, the term OOS results includes all test results that fall outside the specifications or acceptance criteria established in drug applications, drug master files (DMFs), official compendia, or by the manufacturer. The term also applies to all in-process laboratory tests that are outside of established specifications. · applies to chemistry-based laboratory testing of drugs regulated by CDER. It is directed toward traditional drug testing and release methods. These laboratory tests are performed on active pharmaceutical ingredients, excipients and other components, in-process materials, and finished drug products3 to the extent that current good manufacturing practice (CGMP) regulations (21 CFR parts 210 and 211) and the FD&C Act (section 501(a)(2)(B)) apply. The principles in this guidance also apply to in-house testing of drug product components that are purchased by a firm. This guidance can also be used by contract firms performing production and/or laboratory testing responsibilities. Specifically, the guidance discusses how to investigate OOS test results, including the responsibilities of laboratory personnel, the laboratory phase of the investigation, additional testing that may be necessary, when to expand the investigation outside the laboratory, and the final evaluation of all test results. | FDA |
| Fostering Medical Device Improvement: FDA Activities andEngagement with the Voluntary Improvement Program | Draft guidance (Medical Devices) | The FDA’s CDRH is issuing this draft guidance to describe its policy regarding FDA’s participation in the Voluntary Improvement Program (VIP). The VIP is a voluntary program facilitated through the Medical Device Innovation Consortium (MDIC) that evaluates the capability and performance of a medical device manufacturer’s practices using third-party appraisals, and is intended to guide improvement to enhance the quality of devices. The VIP builds on the framework piloted through FDA’s 2018 Case for Quality Voluntary Medical Device Manufacturing and Product Quality Pilot Program (CfQ Pilot Program)1 and incorporates some of the successes and learnings from the pilot. This voluntary program is currently only available to eligible manufacturers of medical devices regulated by CDRH and whose marketing applications are reviewed under the applicable provisions of the FD&C Act (including under sections 510(k), 513, 515, and 520). | FDA |
| Benefit-Risk Considerations for Product Quality Assessments | Draft guidance | This guidance is not intended to address the review by other disciplines or sections of a marketing application (e.g., clinical, nonclinical, biostatistics, pharmacology). Sections II and III of this guidance focus on product quality assessment in the context of FDA’s review of an NDA or BLA. Although not specifically addressed in sections II and III, product quality assessments are also done for ANDAs. However, the product quality assessment of an ANDA can be different to the extent that the ANDA relieson FDA’s finding that the reference listed drug (RLD) identified is safe and effective. As with NDAs and BLAs, an ANDA will not be approved if the applicant’s product development studies, manufacturing process, and control strategy will not consistently result in a finished product of acceptable quality when manufactured at the facilities named in the application. Section IV of this guidance, which discusses how unresolved product quality issues may be handled in the context of regulatory decision-making, specifically addresses how FDA may handle such issues as part of its review of an ANDA. | FDA |
| Electronic Submission of IND Safety Reports Technical Conformance Guide | Final Level 2 Guidance | This Technical Conformance Guide (Guide) provides specifications, recommendations, and general considerations on how to submit electronic investigational new drug application (IND) safety reports to the Center for Drug Evaluation and Research (CDER) or the Center for Biologics Evaluation and Research (CBER). This Guide supplements the draft guidance for industry Providing Regulatory Submissions in Electronic Format: IND Safety Reports (October 2019), which implements the electronic submission requirements of section 745A(a) of the FD&C Act with respect to electronic submissions for certain IND safety reports submittedto CDER or CBER. This Guide discusses the format for the submission of IND safety reports required under 21 CFR 312.32(c)(1)(i) as individual case safety reports (ICSRs) to the FDA Adverse Event Reporting System (FAERS) and provides recommendations to sponsors who elect to submit such ICSRs to FAERS before the requirement for submission to FAERS is in effect for applicable INDs. This Guide discusses the format for the submission of IND safety reports required under 21 CFR 312.32(c)(1)(i) as individual case safety reports (ICSRs) to the FDA Adverse Event Reporting System (FAERS) and provides recommendations to sponsors who elect to submit such ICSRs to FAERS before the requirement for submission to FAERS is in effect for applicable INDs. This Guide provides general information for sponsors of commercial and noncommercial INDs pertaining to electronic submission of IND safety reports and attachments in electronic format to FAERS. It also provides information to sponsors on the format for submission of IND safety reports required under section 312.32(c)(1) that should not be submitted to FAERS and should continue to be submitted in electronic common technical document (eCTD) format. | FDA |
| FDA Regional Implementation Guide for E2B(R3) Electronic Transmission of Individual Case Safety Reports for Drug and Biological Products Technical Specifications Document | Final Guidance | The purpose of this technical specifications document is to assist submitters transmitting electronic individual case safety reports (ICSRs) and ICSR attachments to the FDA Adverse Event Reporting System (FAERS) database. An ICSR is a description of an adverse experience related to an individual patient or subject. FDA adopted the International Council for Harmonisation (ICH) Implementation Guide (IG) for Electronic Transmission of Individual Case Safety Reports (ICSRs): E2B(R3) Data Elements and Message Specification (ICH ICSR IG) in February 2014, and published the guidance for industry E2B(R3) Electronic Transmission of Individual Case Safety Reports: Implementation Guide — Data Elements and Message Specification (E2B(R3) Electronic Transmission of ICSRs IG) and an appendix to the guidance entitled Appendix I (B) to the ICH E2B(R3) ICSRs Implementation Guide — Backwards and Forwards Compatibility. This document describes FDA’s technical approach for submitting ICSRs, for incorporating its regionally controlled terminology , and for adding FAERS regional data elements that are not addressed in the E2B(R3) Electronic Transmission of ICSRs IG for the following FDA-regulated products: • Drug products marketed for human use with approved new drug applications (NDAs) or abbreviated new drug applications (ANDAs) • Prescription drug products marketed for human use without approved applications, including prescription drug products that are compounded by facilities registered as outsourcing facilities under section 503B of the FD&C Act (21 U.S.C. 353b) • Nonprescription drug products marketed for human use without approved NDAs or ANDAs • CDER regulated therapeutic biological products with approved BLAs • Combination products with approved NDAs, ANDAs, or BLAs • Drug and biological products studied under INDs or IND-exempt bioavailability/bioequivalence (BA/BE) studies This document does not apply to the following: • Postmarketing safety reports for vaccines • Whole blood or blood components • Combination products with a drug or biological product constituent part marketed under a device application • Human cellular and tissue-based products regulated solely under section 361 of the Public Health Service Act (42 U.S.C. 264) | FDA |
| E2B(R3) Electronic Transmission of Individual Case Safety Reports Implementation Guide — Data Elements and Message Specification; and Appendix to the Implementation Guide — Backwards and Forwards Compatibility | Final guidance | This ICH IG focuses on medicinal products and therapeutic biologics for human use. However, the ICH is aware of other regional applications of the messaging standard that have a wider scope, such as pharmacovigilance activities related to vaccines, herbal products, cosmetics, veterinary products or medical devices. The primary ICH application is for the exchange of pharmacovigilance information between and among the pharmaceutical industry and regulatory authorities. This IG is also intended to support the implementation of software and tools for creating, editing, sending and receiving electronic ICSR messages. This IG is not intended to serve as a reference for proper pharmacovigilance practices nor is it intended to explain the underlying scientific or medical issues that support the collation, categorisation or analysis of medicinal product safety information. It is also not intended to explain the rationale that underlies proper case safety reporting. The focus of this ICH IG is on technical implementation. Thus, the intended audience includes system developers, IT professionals, system implementers and system users who need to understand the technical requirements for constructing and using valid electronic messages to transmit ICSRs. | FDA |
E2B(R3) The Electronic Transmission of Individual Case Safety Reports Implementation Guide —Appendix to the Implementation Guide — Backwards and Forwards Compatibility | Final | FDA | |
| Crohn’s Disease: Developing Drugs for Treatment | Draft guidance | The purpose of this guidance is to help sponsors in the clinical development of drugs to treat adults with Crohn’s disease (CD). It addresses the (FDA’s current recommendations on clinical trials for drugs being developed under section 505 of the FD&C Act, section 351 of the PHSA Act and 21 CFR parts 312, 314, and 601for treating CD. Specifically, this guidance addresses FDA’s current thinking about the necessary attributes of clinical trials for drugs being developed for treating CD, including trial population, trial designs, efficacy considerations, and safety assessments. This guidance does not address extraintestinal manifestations of CD, stricturing or fistulizing disease, pediatric drug development, or the treatment or prevention of long-term complications of CD. | FDA |
| Ulcerative Colitis: Developing Drugs for Treatment | Draft guidance | The purpose of this guidance is to help sponsors in the clinical development of drugs to treat adults with ulcerative colitis (UC). It addresses the FDA’s current recommendations on clinical trials for drugs being developed under section 505 of the FD&C Act), section 351 of the PHSA Act and 21 CFR parts 312, 314, and 601 for treating UC. Specifically, this guidance addresses FDA’s current thinking about the necessary attributes of clinical trials for drugs being developed for treating UC, including trial population, trial designs, efficacy considerations, and safety assessments. This guidance does not address extraintestinal manifestations of UC, pediatric drug development, or the treatment or prevention of long-term complications of UC (e.g., this guidance is not intended to discuss endpoints for prevention or reduction in risk of colorectal cancer). | FDA |
| Providing Submissions in Electronic Format — Postmarketing Safety Reports | Final guidance | This guidance: • is one in a series of guidance documents intended to assist industry when making certain regulatory submissions in electronic format to FDA’s CDER and CBER. • provides general information on the electronic submission of postmarketing safety reports under the various provisions listed. However, this guidance does not apply to the following: • Vaccines • Whole blood or blood components • Combination products with a drug or biological product constituent part marketed under a device application • Lot distribution reports • Human cells, tissues, and cellular tissue-based products (HCT/Ps) regulated solely under section 361 of the Public Health Service Act | FDA |
| Drug Products, Including Biological Products, that Contain Nanomaterials – Guidance for Industry | Nanotechnology: • can be used in a broad array of FDA-regulated products, such as human drugproducts, including those that are biological products. • may be used to create drug products in which nanomaterials (as explained in section II of this document), serve a variety of functions, as active ingredients or inactive ingredients, including carriers loaded with an active ingredient. The inclusion of such materials may result in product attributes that differ from those of products that do not contain such materials, and thus may merit particular examination. This document provides guidance on the development of human drug products, including those that are biological products, in which a nanomaterial is present in the finished dosage form. Note that FDA does not categorically judge all products containing nanomaterials or otherwise involving the use of nanotechnology as intrinsically benign or harmful. Rather, for all products (nanotechnology-derived or otherwise), FDA considers the characteristics of the product and its safety and effectiveness for its use. FDA issued a guidance document to industry on the Agency’s considerations related to applications of nanotechnology in FDA-regulated products (referred to as “FDA’s nanotechnology considerations guidance”). FDA’s consideration of the use of nanomaterials in drug products, including those that are biological products, in this document is consistent with FDA’s nanotechnology considerations guidance, and with the broader federal guidance on regulatory oversight of emerging technologies and nanotechnology. | FDA | |
| Bioavailability Studies Submitted in NDAs or INDs – General Considerations | Final guidance | This guidance: • provides recommendations to sponsors and applicants submitting bioavailability (BA) information for drug products in INDs, NDAs, and NDA supplements. • contains recommendations on how to meet the BA requirements set forth in 21 CFR part 320 as they apply to dosage forms intended for oral administration. These dosage forms include tablets, capsules, solutions, suspensions, conventional (e.g., immediate-release (IR) drug products) and modified-release (MR) (e.g., extended-release (ER), delayed-release (DR)) drug products. • is also applicable to non-orally administered drug products when it is appropriate to rely on systemic exposure measures to determine the BA of a drug (e.g., transdermal delivery systems and certain vaginal, rectal, and nasal drug products). • provides recommendations on conducting BA studies during the investigational period for a drug intended to be submitted for approval in an NDA and bioequivalence (BE) studies during the post-approval period for certain changes to drug products with an approved NDA. • finalizes the FDA guidance entitled Bioavailability Studies Submitted in NDAs or INDs – General Considerations (February 2019). This guidance: • does not discuss information for demonstrating BE for drug products in ANDAs and ANDA supplements. In August 2021, the FDA issued a separate draft guidance on this topic entitled Bioequivalence Studies with Pharmacokinetic Endpoints for Drugs SubmittedUnder an ANDA. • does not provide recommendations on studies conducted in support of demonstrating comparability or biosimilarity for biological products licensed under section 351 of the Public Health Service Act . You can view a snapshot of the guidance here. You can also view a presentation on this guidance here and listen to a podcast here. | FDA |
| Considerations for Waiver Requests for pH Adjusters in Generic Drug Products Intended for Parenteral, Ophthalmic, or Otic Use | Draft guidance | This guidance: • describes how FDA intends to evaluate a request for a waiver, with regard to a pH adjuster, under 21 CFR 314.99(b) (hereinafter waiver) of the requirement in 21 CFR 314.94(a)(9)(iii) and (iv) that a drug product intended for parenteral, ophthalmic, or otic use generally “must contain the same inactive ingredients and in the same concentration as the reference listed drug identified by the applicant.” • also provides recommendations regarding the timing and process for requesting such a waiver of the requirement in § 314.94(a)(9)(iii) and (iv) (waiver request). • is intended to assist ANDA applicants that reference a reference listed drug (RLD) intended for parenteral, ophthalmic, or otic use but are seeking approval of a drug that is qualitatively (Q1) different or quantitatively (Q2) different from the RLD with respect to a pH adjuster(s). • is intended to identify the type of information FDA may generally consider in evaluating a waiver request for pH adjusters in generic drug products intended for parenteral, ophthalmic, or otic use and provide recommendations to ANDA applicants regarding the submission and content of such a waiver request. The recommendations in this guidance are limited to inactive ingredients in ANDAs that adjust the pH of a drug product intended for parenteral, ophthalmic, or otic use, and do not apply to other inactive ingredients. | FDA |
| Diversity Plans to Improve Enrollment of Participants From Underrepresented Racial and Ethnic Populations in Clinical Trials; Draft Guidance for Industry; Availability | Draft guidance | The purpose of this guidance is to provide recommendations to sponsors developing medical products on the approach for developing a Race and Ethnicity Diversity Plan (henceforth referred to as the “Plan”) to enroll representative numbers of participants from underrepresented racial and ethnic populations in the United States, such as Black or African American, Hispanic/Latino, Indigenous and Native American, Asian, Native Hawaiian and Other Pacific Islanders, and other persons of color, in clinical trials. Individuals from these populations are frequently underrepresented in biomedical research despite having a disproportionate disease burden for certain diseases relative to their proportional representation in the general population. Adequate representation of these populations in clinical trials and studies supporting regulatory submissions helps ensure that the data generated in the development program reflect the racial and ethnic diversity of the population expected to use the medical product if approved, and may potentially identify effects on safety or efficacy outcomes that may be associated with, or occur more frequently within these populations. | FDA |
| Providing Regulatory Submissions in Electronic and Non-Electronic Format—Promotional Labeling and Advertising Materials for Human Prescription Drugs | Final guidance | This guidance: • concerns submissions of promotional materials for human prescription drugs (drugs) to the FDA made by manufacturers, packers, and distributors (firms), whether the applicant, or an entity acting on behalf of the applicant. • Specifically concerns submissions made to the Office of Prescription Drug Promotion (OPDP) in CDER and the Advertising and Promotional Labeling Branch (APLB) in CBER. • also explains certain aspects of electronic submission of promotional materials in module 1 of the electronic common technical document (eCTD), using version 3.3 or higher of the us-regional-backbone file. For the purpose of this guidance, the term promotional materials collectively refers to promotional labeling and advertising materials regardless of the format, manner, or medium by which they are presented. Promotional materials may include, but are not limited to, television advertisements (ads), brochures, booklets, detailing pieces, internet websites, print ads, exhibits, sound recordings, and radio ads. | FDA |
| E8(R1) General considerations for clinical studies | Final guidance | This guidance: • describes internationally accepted principles and practices in the design and conduct of clinical studies of drug and biological products. • is intended to assist sponsors and other parties that design clinical studies, and to promote the quality of the studies submitted to regulatory authorities, while allowing for flexibility. • revises the ICH guidance E8 General Considerations for Clinical Trials issued in December 1997. Significant changes from the 1997 version include the following: (1) addresses study quality to ensure the protection of study participants and the generation of reliable and meaningful results, while promoting study efficiency; (2) addresses a broad range of study designs and data sources; and (3) provides updated cross-referencing to other relevant ICH guidances that inform the design, planning, and conduct of clinical research. | FDA |
| M7(R2) Addendum: Application of the principles of the ICH M7 guideline to calculation of compound specific acceptable intakes | Draft, Level 1 guidance | This document contains only the list of the revisions to the M7(R1) Guideline as well as the new monographs for the 7 new compounds Acetaldehyde, Dibromoethane, Epichlorohydrin, Ethyl Bromide, Formaldehyde, Styrene, and Vinyl Acetate, which are submitted for public consultation. Further to reaching Step 4, these revisions would be integrated into a complete M7(R2) Guideline and Addendum documents. | FDA |