US FDA guidance for Industry, Oct – Dec 2023

DateTitle of guidance and link to documentType and level
of guidance
About the guidance
28 Dec 2023Potency Assurance for Cellular and Gene Therapy ProductsDraftThis draft guidance provides recommendations for developing a science- and risk-based strategy to help assure the potency of a human cellular therapy or gene therapy (CGT) product. A potency assurance strategy is a multifaceted approach that reduces risks to the potency of a product through manufacturing process design, manufacturing process control, material control, in-process testing, and potency lot release assays. The goal of a potency assurance strategy is to ensure that every lot of a product released will have the specific ability or capacity to achieve the intended therapeutic effect.
28 Dec 2023Quality Considerations for Topical Ophthalmic Drug ProductsDraft, Level 1This guidance discusses certain quality considerations for ophthalmic drug products (i.e., gels, ointments, creams, and liquid formulations such as solutions, suspensions, and emulsions) intended for topical delivery in and around the eye. Specifically, the guidance discusses:
• Microbiological considerations.
• Approaches to evaluating visible particulate matter, extractables and leachables, and impurities and degradation products.
• Use of in vitro drug release/dissolution testing as an optional quality control strategy for 25 certain ophthalmic dosage forms.
• Recommendations for design, delivery, and dispensing features of container closure systems (CCSs).
• Recommendations for stability studies.

This guidance:
• provides information regarding quality considerations for ophthalmic drug products consistent with the current good manufacturing practice (CGMP) requirements outlined in section 501(a)(2)(B) of the FD&C Act and 21 CFR 35 parts 210 and 211 for all drug products, part 601 for biological products, and part 4 for combination products.
• provides for ophthalmic drug products with a United States Pharmacopeia (USP) monograph, information about applicable criteria from the USP.
• also provides recommendations to industry on the documentation that should be submitted in the CMC section of NDAs, ANDAs, and BLAs, including BLAs for biosimilar and interchangeable biosimilar products.
• does not apply to biological products regulated by CBER.
• revises the draft guidance of the same name issued in October 2023. This revision adds microbiological considerations related to product sterility for all ophthalmic drug products and the prevention of contamination of ophthalmic drug products packaged in multidose containers.
27 Dec 2023Reformulating Drug Products That Contain Carbomers Manufactured With BenzeneFinal, Level 1This guidance:
• provides recommendations for applicants and manufacturers on what tests should be performed and what documentation should be submitted or available to FDA to support the reformulation of drug products that use carbomers manufactured with benzene. Certain USP carbomer monographs currently allow for unacceptable levels of benzene, which raises safety concerns. FDA has requested that the USP omit (or remove) these monographs, and applicants and manufacturers may need to reformulate their drug products to avoid using these carbomers.
• provides recommendations for testing and documentation related to reformulation, taking into consideration the various routes of administration and dosage forms of affected drug products.
• for application holders, also recommends appropriate submission types to notify the Agency of such changes.
26 Dec 2023Direct-to-Consumer Prescription Drug Advertisements: Presentation of the Major Statement in a Clear, Conspicuous, and Neutral Manner in Advertisements in Television and Radio Format Final Rule Questions and AnswersFinalThis guidance is intended to help small entities understand and comply with the standards established in the final rule, “Direct-to-Consumer Prescription Drug Advertisements: Presentation of the Major Statement in a Clear, Conspicuous, and Neutral Manner in Advertisements in Television and Radio Format” (CCN Final Rule) (88 FR 80958, November 21, 2023). Section 502(n) of the FD&C Act, as amended by the Food and Drug Administration Amendments Act of 2007 (FDAAA), requires that human prescription drug advertisements presented directly to consumers (DTC) in television or radio format that state the name of the drug and its conditions of use (DTC TV/radio ads) present the major statement relating to side effects and contraindications (“major statement”) in a clear, conspicuous, and neutral manner. The CCN Final Rule modifies 21 CFR 202.1(e)(1) to reflect this requirement and establishes standards to help ensure the major statement in these advertisements is presented in the manner required.

The major statement provides information relating to the major side effects and contraindications of an advertised prescription drug. Note that this is a selected presentation of the major side effects and contraindications of the drug and not a listing of every risk. DTC TV/radio ads are required to include a major statement.
21 Dec 2023Rare Diseases: Considerations for the Development of Drugs and Biological ProductsFinal, Level 1The purpose of this guidance is to assist sponsors of drugs for the treatment of rare diseases in conducting efficient and successful drug development programs. The statutory requirements for marketing approval for drugs to treat rare and common diseases are the same and issues discussed in this guidance are encountered in other drug development programs. These issues are frequently more difficult to address in the context of a rare disease for which there is often limited medical and scientific knowledge, poorly understood natural history data, sample size constraints, and lack of drug development experience.

This guidance does not contain discussion of the general issues of statistical analysis. Those topics are addressed in other documents
21 Dec 2023Data Standards for Drug and Biological Product Submissions Containing Real-World DataFinal, Level 1This guidance:
• provides recommendations to sponsors for complying with section 745A(a) of the FD&C Act (21 U.S.C. 379k-1(a)) when submitting RWD as study data in applicable drug submissions. FDA is issuing this guidance as part of its RWE Program and to satisfy, in part, the mandate under section 505F of the FD&C Act (21 U.S.C. 355g) to issue guidance on the use of RWE in regulatory decision-making.
• addresses considerations for the use of data standards currently supported by FDA in applicable drug submissions containing study data derived from RWD sources. For the purposes of this guidance, FDA defines RWD as data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources. Examples of RWD include data from electronic health records (EHRs); medical claims data; data from product and disease registries;8 patient-generated data (including data from in-home-use settings); and data gathered from other sources that can inform on health status, such as mobile devices.
20 Dec 2023Real-World Data: Assessing Registries To Support Regulatory Decision-Making for Drug and Biological ProductsFinal, Level 1FDA is issuing this guidance as part of its RWE Program and to satisfy, in part, the mandate under section 505F of the FD&C Act to issue guidance on the use of RWE in regulatory decision-making.

Topics covered in this guidance include:
• Considerations regarding a registry’s fitness-for-use in regulatory decision-making, focusing on attributes of a registry that support the collection of relevant and reliable data
• Considerations when linking a registry to another data source for supplemental information, such as data from medical claims, electronic health records (EHRs), digital health technologies,7 or other registries
• Considerations for supporting FDA review of submissions that include registry data
21 Dec 2023Master Protocols for Drug and Biological Product DevelopmentThis guidance document provides recommendations on the design and analysis of trials conducted under a master protocol as well as guidance on the submission of documentation to support regulatory review.

For the purpose of this guidance, the term master protocol sponsor refers to the person or organization who takes responsibility for and initiates the master protocol.3 In many instances individual drugs chosen for evaluation in the master protocol will also be evaluated under separate Investigational New Drug Applications (INDs) independent of the master protocol. A sponsor responsible for the investigation of an individual drug evaluated under the separate IND is referred to as the individual drug sponsor. The master protocol sponsor and the individual drug sponsor may or may not be the same entity. This guidance uses the term sponsor when providing general recommendations that may be relevant to both the master protocol sponsor and individual drug sponsors.

The primary focus of this guidance is on randomized umbrella and platform trials that are intended to contribute to a demonstration of safety and substantial evidence of effectiveness of a drug. The concepts discussed may also be useful to consider for early-phase or exploratory umbrella and platform trials as well as those conducted to satisfy post marketing commitments or requirements. The recommendations and considerations in this guidance do not apply to master protocols evaluating first-in-human drugs given the unique attributes from both a trial 56 design and regulatory perspective that must be considered.

You can view a snapshot of the guidance here.
For further information you can listen to the guidance recap podcast and/or read the podcast transcript here.
19 Dec 2023Development of Monoclonal Antibody Products Targeting SARS-CoV-2 for Emergency Use AuthorizationFinal, Level 1FDA is issuing this guidance to provide recommendations to sponsors on the development of monoclonal antibody (mAb) products targeting SARS-CoV-2 intended for the prevention or treatment of COVID-19, including recommendations on addressing the impact of emerging variants.

This guidance does not address the information and data necessary to support the licensure of mAb products under section 351 of the Public Health Service Act (42 U.S.C. 262).

Given the need to ensure that sponsors are aware of our current recommendations to facilitate timely development of monoclonal antibody products targeting SARS-CoV-2, FDA is issuing this guidance for immediate implementation without initially seeking prior comment because the Agency has determined that prior public participation is not feasible or appropriate (see 21 CFR 10.115(g)(2) and section 701(h)(1)(C)(i) of the FD&C Act (21 U.S.C. 371(h)(1)(C)(i))). This guidance document is being implemented immediately, but it remains subject to comment in accordance with the Agency’s good guidance practices. (see 21 CFR 10.115(g)(3)).
13 Dec 2023Clinical Pharmacology Considerations for Peptide Drug Products

Source: FDA
Draft, Level 2This guidance provides recommendations to assist industry in the development of peptide drug products. Specifically, this guidance, when finalized, will describe the FDA’s current thinking regarding the impact of clinical pharmacology considerations, including hepatic impairment, drug-drug interactions (DDIs), QTc prolongation risk, and immunogenicity risk on a peptide drug product’s pharmacokinetics (PK), safety, and efficacy.

This guidance specifically outlines clinical pharmacology considerations for development programs for proposed peptide drug products submitted in a new drug application (NDA) under section 505(b) of the FD&C Act and references other relevant guidances when appropriate. The term peptide, for purposes of this guidance, refers to any polymer composed of 40 or fewer amino acids. In general, if a peptide meets the definition of a drug and does not otherwise meet the statutory definition of a “biological product” or a “device,” it would be regulated as a drug under the FD&C Act and be subject to all the “drug” requirements under the FD&C Act and FDA’s regulations, including the requirement that new drugs must be approved under section 505(c) of the FD&C Act before they can be marketed in interstate commerce. However, peptide drug products can have product characteristics that may be similar, in certain respects, to biological products, and as such, this guidance includes references to other FDA guidances on biological products that discuss scientific principles that could also be applicable to peptide drug products.
12 Dec 2023Advanced Manufacturing Technologies Designation Program

Source: FDA
DraftAdvanced manufacturing is a term for an innovative pharmaceutical manufacturing technology or approach that has the potential to improve the reliability and robustness of the manufacturing process and supply chain and increase timely access to quality medicines for the American public. Advanced manufacturing can integrate novel technological approaches, use established techniques in an innovative way, or apply production methods in a new domain where there are no defined best practices or experience. Advanced manufacturing can potentially be used for new or currently marketed small molecule drugs or biological products.

This guidance provides recommendations to persons and organizations interested in participating in FDA’s Advanced Manufacturing Technologies Designation Program, which is intended to facilitate the development of drugs, including biological products, manufactured using an AMT that has been designated as such under the program (hereinafter designated AMT). The guidance outlines the eligibility criteria for AMT designation, the submission and assessment process for requests, and the benefits of receiving an AMT designation and includes a questions and answers section to cover additional details about key concepts important for program utilization.
Specifically, the guidance describes:
The process for submitting an AMT designation request, including a description of eligibility criteria and the data and other information to be included.
• When and how FDA will communicate receipt of and provide advice on an AMT designation request.
• When and how FDA will assess AMT designation requests.
• The process by which FDA will engage with holders of designated AMTs and applicants for drugs manufactured using, referencing, or relying upon a designated AMT
• Potential benefits related to drug development and application assessment.
12 Dec 2023Study Data Technical Conformance Guide – Technical Specifications Document

Source: FDA
FinalThis Study Data Technical Conformance Guide (Guide) provides specifications, recommendations, and general considerations on how to submit standardized study data using FDA-supported data standards located in the FDA Data Standards Catalog (Catalog). The Guide supplements the guidance for industry Providing Regulatory Submissions in Electronic Format — Standardized Study Data (eStudy Data). The eStudy Data guidance implements the electronic submission requirements of section 745A(a) of the FFD&C Act with respect to standardized study data contained in certain INDs, NDAs; ANDAs; and certain BLAs that are submitted to the CDER or the CBER).
7 Dec 2023Verification Systems Under the Drug Supply Chain Security Act for Certain Prescription Drugs

Source: FDA
Final, Level 1FDA is issuing this guidance to describe FDA’s interpretation of the requirements of section 582 of the FD&C Act regarding verification systems.
· This guidance provides recommendations for robust verification systems for the determination, quarantine, and investigation of suspect products, as well as the quarantine, notification, and disposition of illegitimate products.
· The guidance also addresses the manner in which FDA recommends that trading partners submit cleared product notifications.
· Finally, this guidance addresses the statutory requirements for verification, including verification of saleable returns, at the package level for product identifiers on packages and homogenous cases intended to be introduced in a transaction into commerce.
30 Nov 2023Demonstrating Substantial Evidence of Effectiveness With One Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence

Source: FDA
Draft, Level 1This guidance (the Confirmatory Evidence guidance) complements the draft guidance for industry Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products (December 2019) (the 2019 Effectiveness draft guidance) and the guidance for industry Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products (May 1998) (the 1998 Effectiveness guidance). This guidance provides recommendations for sponsors to consider when planning a drug development program.
27 Nov 2023Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies

Source: FDA
FinalThis 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.
21 Nov 2023


Direct-to-Consumer Prescription Drug Advertisements: Presentation of the Major Statement in a Clear, Conspicuous, and Neutral Manner in Advertisements in Television and Radio Format
Final RuleThis rule is effective May 20, 2024. The compliance date of this rule is November 20, 2024.

The FDA is issuing a final rule to amend its regulations concerning direct-to-consumer (DTC) advertisements (ads) for human prescription drugs presented in television or radio format and stating the name of the drug and its conditions of use (DTC TV/radio ads). Specifically, the final rule implements a requirement of the FD&C Act, added by the Food and Drug Administration Amendments Act of 2007 (FDAAA), that in such DTC TV/radio ads, the major statement relating to side effects and contraindications must be presented in a clear, conspicuous, and neutral manner. As directed by FDAAA, FDA is establishing standards to determine whether the major statement in DTC TV/radio ads is presented in a clear, conspicuous, and neutral manner.
21 Nov 2023Translation of Good Laboratory Practice Study Reports: Questions and Answers

Source: FDA
DraftThis guidance provides information to sponsors and nonclinical laboratories regarding the language translation of study reports for studies conducted in compliance with good laboratory practice (GLP) regulations (21 CFR part 58). GLP studies include, but are not limited to nonclinical toxicology studies, safety pharmacology studies, and device safety studies received by different FDA Centers. When study reports of GLP studies are translated from their original language into English, adequate documentation is critical to ensure accurate and complete study data are submitted to FDA. This question-and-answer document is intended to clarify FDA’s recommendations concerning the translation of study reports from a non-English language into English for studies conducted in compliance with GLP regulations. We expect that the recommendations for translating GLP study reports described in this guidance will increase our stakeholders’ understanding of the documentation needed to ensure study reports translated from the original language into English are clear, accurate, complete, and truthful.

This draft guidance does not address the reliable translation of other study reports submitted to support a marketing authorization, including studies that are not conducted in compliance with GLP regulations, but the concepts described in the guidance may be informative for the translation of study reports from those studies that are intended for submission to FDA to support a marketing authorization. FDA may issue guidance regarding questions & answers for the translation of other study reports submitted to FDA in support of marketing authorizations as appropriate.

Here you can view the guidance snapshot, listen to the guidance recap podcast and read the podcast transcript.
9 Nov 2023E6(R2) Good Clinical Practice: Integrated Addendum to ICH E6(R1

Source: FDA
FinalSince the development of the ICH GCP Guidance, the scale, complexity, and cost of clinical trials have increased. Evolutions in technology and risk management processes offer new opportunities to increase efficiency and focus on relevant activities. When the original ICH E6(R1) text was prepared, clinical trials were performed in a largely paper-based process. Advances in use of electronic data recording and reporting facilitate implementation of other approaches. For example, centralized monitoring can now offer a greater advantage, to a broader range of trials than is suggested in the original text. Therefore, this guidance has been amended to encourage implementation of improved and more efficient approaches to clinical trial design, conduct, oversight, recording, and reporting while continuing to ensure human subject protection and reliability of trial results. Standards regarding electronic records and essential documents intended to increase clinical trial quality and efficiency have also been updated.

This ICH GCP Guidance Integrated Addendum provides a unified standard for the European
Union, Japan, the United States, Canada, and Switzerland to facilitate the mutual acceptance
of data from clinical trials by the regulatory authorities in these jurisdictions. In the event of
any conflict between the E6(R1) text and the E6(R2) addendum text, the E6(R2) addendum
text should take priority.
8 Nov 2023Study Data Technical Conformance Guide – Technical Specifications Document

Source: FDA
FinalThis Study Data Technical Conformance Guide (Guide) provides specifications, recommendations, and general considerations on how to submit standardized study data using FDA-supported1 data standards located in the FDA Data Standards Catalog (Catalog). The Guide supplements the guidance for industry Providing Regulatory Submissions in Electronic Format — Standardized Study Data (eStudy Data). The eStudy Data guidance implements the electronic submission requirements of section 745A(a) of the FD&C Act with respect to standardized study data contained in certain INDs, NDAs, ANDAs and certain BLAs that are submitted to CDER or CBER.

This Guide provides technical recommendations to sponsors4 for the submission of animal and human study data and related information in a standardized electronic format in INDs, NDAs, ANDAs, and BLAs. The Guide is intended to complement and promote interactions between sponsors and FDA review divisions. However, it is not intended to replace the need for sponsors to communicate directly with review divisions regarding implementation approaches or issues relating to data standards.
6 Nov 2023Real-Time Oncology Review (RTOR

FDA
FinalThe purpose of this guidance is to provide recommendations to applicants on the process for submission of selected NDAs and BLAs with oncology indications for review under Real-Time Oncology Review (RTOR).

This guidance does not address FDA’s expedited programs such as the Fast Track Designation, Breakthrough Therapy Designation, or Priority Review Designation. Additional information on these expedited programs can be found in the guidance for industry Expedited Programs for Serious Conditions – Drugs and Biologics (May 2014) RTOR is separate from the Split Time Application Review (STAR) pilot program which was established under the Prescription Drug User Fee Act (PDUFA) VII commitments.
3 Nov 2023Submitting Patient-Reported Outcome Data in Cancer Clinical Trials

Source: FDA
Final, Level 2This document:
· provides technical specifications for submitting patient-reported outcome (PRO) data collected in cancer clinical trials to support a marketing application for a medical product in oncology, where a PRO is a type of clinical outcome assessment (COA) used to collect patient experience data. The FDA Patient Focused Drug Development (PFDD) Glossary defines a PRO as a measurement based on a report that comes directly from the patient (i.e., study subject) about the status of a patient’s health condition without interpretation of the patient’s response by a clinician or anyone else, where a PRO can be measured by self-report or by interview, provided that the interviewer records only the patient’s response. This technical specifications document supplements FDA’s draft guidance for industry Core Patient-Reported Outcomes in Cancer Clinical Trials (June 2021)4 and the PFDD Guidance series.
· provides specifications for the submission of Clinical Data Interchange Standards Consortium (CDISC) Study Data Tabulation Model (SDTM) and Analysis Data Model (ADaM) datasets and specifications for recommended tables and figures. These technical specifications aim to provide general guidelines for (1) standardized dataset content and structure and (2) recommended tables and figures to facilitate FDA review of the marketing application that the submitted data and analysis outputs are intended to support. The SDTM and ADaM specifications outlined in section 3.0 Overview of Dataset Specifications are not prescriptive and do not include an exhaustive list of all datasets, variables, and controlled terminologies to be submitted for FDA review. Further, the recommended tables and figures do not comprise all information needed to support FDA review of a marketing application. The dataset specifications and specifications for tables and figures are pursuant to discussions with FDA and may vary by clinical drug development program and clinical trial therein. These specifications are intended to be applicable to any PRO data used to inform the evaluation of (1) safety and tolerability or (2) clinical benefit in randomized studies (i.e., improvement in disease symptoms) within a cancer clinical trial.
· does not pertain to submissions needed to support FDA review of the PRO measure itself or the proposed interpretation and use of scores generated by the PRO measure within the context of a specific clinical trial. Agreement on the PRO measure(s) used to collect study data and analyses of the resulting PRO data should be discussed with FDA as early as possible in a medical product development program, for example, prior to trial initiation. Sponsors are strongly encouraged to use the resources described in section 1.3 Relationships to Other Documents and to seek Agency input for confirmation and clarification as needed. Sponsors should consult with the Agency to determine which requested displays defined in section 4.0 Specifications for Tables and Figures apply to the PRO measure used within the marketing application. Lastly, although this guidance focuses on PRO measures, some of these recommendations may be relevant to other COAs (i.e., clinician-reported, observer reported, and performance outcome measures) in cancer clinical trials.
3 Nov 2023Submitting Clinical Trial Datasets and Documentation for Clinical Outcome Assessments Using Item Response Theory

Source: FDA
Final, Level 2This document provides technical specifications for the submission of clinical outcome assessment (COA) data that use Item Response Theory (IRT) and supplements the FDA CDER Patient-Focused Drug Development (PFDD) Methodological Guidance Series. As described in the Biomarkers, EndpointS, and other Tools (BEST) Resource glossary, a COA refers to the assessment of a clinical outcome made through a report by a clinician, a patient, a non-clinician observer, or through a performance-based assessment; thus, there are four types of COAs: clinician-reported outcome (ClinRO), patientreported outcome (PRO), observer-reported outcome (ObsRO), and performance outcome (PerfO) measures.

IRT is a family of mathematical models that describes the functional relationship between item performance, item characteristics, and the patient’s status on the construct being measured. COAs that use IRT include static, fixed-form COAs that are developed and/or scored using IRT, or COAs that are administered using IRT-based Computerized Adaptive Testing (CAT). CAT is a sequential form of individual testing administered by a computer in which successive items in the COA measure are selected for administration based primarily on the item’s psychometric properties and content in relation to the patient’s responses to previous items . COAs that use IRT can leverage an item bank for item selection. An item bank represents the total set of items from which a subset is selected during COA measure development or selected for the patient during adaptive testing.
1 Nov 2023Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers

Source: FDA
DraftThis guidance provides information for industry, researchers, physicians, institutional review boards (IRBs), and patients about the implementation of FDA’s regulations on expanded access to investigational drugs for treatment use under an IND (21 CFR part 312, subpart I), which went into effect on October 13, 2009. FDA received numerous questions concerning implementation of the regulatory requirements for expanded access. As a result, FDA issued the guidance for industry Expanded Access to Investigational Drugs for Treatment Use — Questions and Answers (June 2016, updated October 2017) (the 2017 guidance), providing recommendations in a question-and-answer format, addressing the most frequently asked questions. Since 2017, FDA has received additional questions concerning implementation of the regulatory and statutory requirements of expanded access to investigational drugs, including those added by the 21st Century Cures Act and the FDA Reauthorization Act of 2017 (FDARA). When finalized, this guidance will replace the 2017 guidance. Significant changes from the 2017 version include additional recommendations related to IRB review, informed consent, and new requirements established by the Cures Act and FDARA related to sponsors making their policies for evaluating and responding to expanded access requests (i.e., expanded access policy) public and readily available.

The Informed Consent Template is included as an example in the above draft guidance titled to assist physicians and institutions in developing the informed consent for approval by Institutional Review Boards (IRBs). For additional information about informed consent see FDA’s final guidance for IRBs, clinical investigators and sponsors titled Informed Consent.
25 Oct 2023Remote Interactive Evaluations of Drug Manufacturing and Bioresearch Monitoring Facilities

Source: FDA
Draft, Level 1FDA conducts inspections for many purposes and programs, ensuring necessary oversight of FDA-regulated products and assessing facilities’ compliance with the FD&C Act. FDA may use alternative tools, such as Remote Regulatory Assessments (RRAs) , in advance or in lieu of an inspection or to support an inspection of a facility and assess compliance with applicable laws and regulations. For instance, if a program office determines that an inspection is not necessary, feasible, or practical, FDA may instead conduct an RRA. An RRA is an examination of an FDA-regulated establishment and/or its records, conducted entirely remotely, to evaluate compliance with applicable FDA requirements. RRAs may consist of (or include) a request to conduct voluntary remote interactive evaluations.

FDA is issuing this guidance to describe how it requests and conduct voluntary remote interactive evaluations at facilities where drugs are manufactured, processed, packed, compounded, or held, and at drug facilities covered under FDA’s bioresearch monitoring (BIMO) program. FDA may consider the use of a remote interactive evaluation for any of the inspection program areas described in Section III of this guidance.
24 Oct 2023Topical Dermatologic Corticosteroids: In Vivo Bioequivalence

Source: FDA
DraftThis guidance:
· is intended to assist applicants who submit ANDAs for topical dermatologic corticosteroid products of all potency group, hereinafter referred to as topical corticosteroids.
· describes recommendations for in vivo studies to demonstrate the bioequivalence of topical corticosteroids.
· provides recommendations for the study design, method qualification, data analysis, and data reporting for the pilot dose-duration vasoconstrictor response study and pivotal vasoconstrictor bioequivalence study used to demonstrate bioequivalence of topical corticosteroids. The guidance also discusses considerations and approaches for estimating key
study parameters (e.g., dose corresponding to half the maximal vasoconstrictor response (ED50))
30 and sample size for the pivotal vasoconstrictor bioequivalence study).
20 Oct 2023Benefit-Risk Assessment for New Drug and Biological Products

Source: FDA
Final, Level 1The intent of this guidance is to clarify for drug sponsors and other stakeholders how considerations about a drug’s benefits, risks, and risk management options factor into certain premarket and postmarket regulatory decisions that the FDA makes about NDAs submitted under section 505(b) of the FD&C Act (21 U.S.C. 355(b) as well as BLAs submitted under section 351(a) of the PHS Act. This guidance first articulates important considerations that factor into the CDER and the CBER benefit-risk assessments, including how patient experience data4 can be used to inform the benefit-risk assessment. It then discusses how sponsors can inform FDA’s benefit risk assessment through the design and conduct of a development program, as well as how they may present benefit and risk information in the marketing application. It also discusses opportunities for interaction between FDA and sponsors to discuss benefit-risk considerations in connection with the development of an NDA or BLA. This guidance concludes with additional considerations on benefit-risk assessments that inform regulatory decision-making in the postmarket setting.

This guidance:
· pertains to benefit-risk assessments made to support certain regulatory decisions about NDAs or BLAs, from premarket approval through the postmarket setting. This includes decisions regarding any regulatory requirements for approval, such as inclusion of a boxed warning in approved labeling, postmarketing study requirements and commitments, and risk
evaluation and mitigation strategies (REMS). These regulatory decisions are made in accordance with specific, applicable legal and regulatory authorities and criteria.
· touches on some of these authorities but does not attempt to list or address them all.
· does not directly address other regulatory decisions that may occur throughout the drug development lifecycle, such as decisions regarding first-in-human trials of an investigational new drug and expanded access applications, which also may require FDA to consider information about the benefits and risks of an investigational or marketed drug for its proposed use. However, the concepts discussed in this guidance may also be relevant to these other types of decisions.
13 Oct 2023Quality Considerations for Topical Ophthalmic Drug Products

Source: FDA
Draft, Level 1This guidance provides information regarding quality considerations for ophthalmic drug products consistent with the current good manufacturing practice (CGMP) requirements outlined in section 501(a)(2)(B) of the FD&C Act and 21 CFR parts 210 and 211 for all drug products, part 601 for biological products, and part 4 for combination products. For ophthalmic drug products with a United States Pharmacopeia (USP) monograph, this guidance provides information about applicable criteria from the USP. This guidance also provides recommendations to industry on the documentation that should be submitted in the chemistry, manufacturing, and controls (CMC) section of NDAs, ANDAs, and BLAs, including BLAs for biosimilar and interchangeable biosimilar products. The CMC section of NDAs, ANDAs, and BLAs must be included as required by 21 CFR 314.50, 21 CFR 314.94, and 21 CFR part 601, respectively. Relevant records and other information that demonstrate compliance with CGMP requirements must be made available for FDA review during an inspection conducted under section 704(a)(1) of the FD&C Act or when requested by FDA in advance or in lieu of an inspection as described in section 704(a)(4) of the FD&C Act. This guidance does not apply to biological products regulated by CBER
10 Oct 2023Communications From Firms to Health Care Providers Regarding Scientific Information on Unapproved Uses of Approved/Cleared Medical Products Questions and Answers

Source: FDA
Draft, Level 1This revised draft guidance, when finalized, will provide FDA’s current thinking on common questions regarding certain communications by firms to health care providers (HCPs) of scientific information on unapproved use(s) (SIUU) of approved/cleared medical products. Specifically, this guidance relates to firms sharing the following types of communications with HCPs:

• Published scientific or medical journal articles (reprints)
• Published clinical reference resources, as follows:
– Clinical practice guidelines (CPGs)
– Scientific or medical reference texts (reference texts)
– Materials from independent clinical practice resources
• Firm-generated presentations of scientific information from an accompanying published reprint
4 Oct 2023Considerations for the Conduct of Clinical Trials of Medical Products During Major Disruptions Due to Disasters and Public Health Emergencies

Source: FDA
Final, Level 1Disasters and public health emergencies (PHEs) have the potential to cause major disruptions in the conduct of clinical trials for medical products. Such events can include (but are not limited to) hurricanes, earthquakes, military conflicts, infectious disease outbreaks, or bioterrorist attacks. FDA is issuing this guidance to provide general considerations to assist sponsors, institutional review boards (IRBs), and clinical investigators in assuring the safety of trial participants, maintaining compliance with good clinical practice (GCP), and minimizing risks to trial integrity during disasters and PHEs that may lead to major disruption of clinical trial conduct and operations. The appendix to this guidance further explains these general considerations in a question-and-answer format.