US FDA issued guidance for industry, April – June 2023

DateTitle of guidance and link to documentType and level
of guidance		About the guidance
27 Jun 2023Prohibition on Wholesaling Under Section 503B of the FD&C Act

Source: FDA		DraftUnder section 503B of the FD&C Act (21 U.S.C.
353b), a drug compounded by an outsourcing facility qualifies for exemptions from certain statutory requirements if, among other conditions, the drug “will not be sold or transferred by an entity other than the outsourcing facility that compounded such drug.” However, this provision “does not prohibit administration of a drug in a health care setting or dispensing a drug pursuant to a prescription executed in accordance with section 503(b)(1).”
This guidance:
• describes FDA’s interpretation of, and policies concerning, the prohibition on wholesaling in section 503B of the FD&C Act.
• also describes examples of how FDA intends to apply section 503B’s wholesaling provision.
27 June 2023Presenting Quantitative Efficacy and Risk Information in Direct-to-Consumer (DTC) Promotional Labeling and Advertisements

Source: FDA		Final This guidance provides recommendations for presenting quantitative efficacy and risk information in direct-to-consumer (DTC) promotional labeling and advertisements for prescription human drug and biological products and prescription animal drugs and in DTC promotional labeling for over-the-counter animal drugs (collectively, promotional communications). For the purposes of this guidance, quantitative efficacy and risk information refers to information that numerically addresses the likelihood or magnitude of a drug’s efficacy or risks.

The guidance outlines FDA’s recommendations for how firms that include quantitative efficacy or risk information in DTC promotional communications for their drugs can make the language and presentation more consumer-friendly. It covers the following topics:
• Providing quantitative efficacy or risk information for the control group, when applicable
• Presenting probability information in terms of absolute frequencies, percentages, and relative frequencies
• Formatting quantitative efficacy or risk information
• Using visual aids to illustrate quantitative efficacy or risk information
22 Jun 2023Formal Dispute Resolution and Administrative Hearings of Final Administrative Orders Under Section 505G of the FD&C Act

Source: FDA		Draft, Level 1This guidance provides recommendations for industry and review staff on the formal dispute resolution and administrative hearings procedures for resolving scientific and/or medical disputes between CDER and requestors and sponsors drugs that will be subject to a final administrative order (final order) under section 505G of the FD&C Act (21 U.S.C. 355h). The drugs that this guidance covers are nonprescription drugs without approved new drug applications, which are governed by the provisions of section 505G (hereafter referred to as over-the-counter (OTC) monograph drugs). Specifically, this guidance describes the CDER formal dispute resolution (FDR) procedures for eligible requestors or sponsors that wish to appeal a scientific and/or medical issue related to a final order. This guidance also outlines the procedures for an administrative hearing (hearing) related to a final order. Finally, this guidance describes the procedures for consolidated proceedings for FDR and hearings to resolve scientific and/or medical disputes related to final orders.
8 Jun 2023 Assessing User Fees Under the Generic Drug User Fee Amendments of 2022

Source: FDA		Final, Level 2This guidance:
• provides information to stakeholders regarding FDA’s implementation of the GDUFA III under Title III of the FDA User Fee Reauthorization Act of 2022. Because GDUFA III created changes to the user fee structure, this guidance serves to provide an explanation about the new fee structure and types of fees for which entities are responsible.
• describes the types of user fees authorized by GDUFA III, and the processes 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 previously developed under earlier GDUFA authorizations. This guidance also describes how FDA determines affiliation
for purposes of assessing generic drug user fees. FDA will issue separate guidance documents regarding GDUFA III non-user fee requirements and processes. This guidance 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 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).
5 June 2023E6(R3) Good Clinical Practice (GCP)

Source: FDA		Draft, Level 1Good Clinical Practice (GCP) is an international, ethical, scientific and quality standard for the conduct of trials that involve human participants. Clinical trials conducted in accordance with this standard will help to assure that the rights, safety and well-being of trial participants are protected; that the conduct is consistent with the principles that have their origin in the Declaration of Helsinki; and that the clinical trial results are reliable. The term “trial conduct” in this document includes processes from planning to reporting, including planning, initiating, performing, recording, oversight, evaluation, analysis and reporting activities as appropriate.

The objective of this ICH GCP Guideline is to provide a unified standard to facilitate the mutual acceptance of clinical trial data for ICH member countries and regions by applicable regulatory authorities.

This guideline builds on key concepts outlined in ICH E8(R1) General Considerations for Clinical Studies. This includes fostering a quality culture and proactively designing quality into clinical trials and drug development planning, identifying factors critical to trial quality, and engaging stakeholders, as appropriate, using a proportionate risk-based approach.
5 Jun 2023Requests for Reconsideration at the Division Level Under GDUFA Guidance for Industry

Source: FDA		DraftThis guidance:
• provides recommendations for industry on the procedures for resolving scientific and/or regulatory issues or matters between FDA and applicants of abbreviated new drug applications (ANDAs) that wish to pursue a request for reconsideration within the review discipline at the division level or original signatory authority.
• does not describe the formal dispute resolution procedures for resolving scientific and/or regulatory disputes between FDA and sponsors or applicants that cannot be resolved through the request for reconsideration process at the division level.
• also does not describe the procedures for resolving administrative matters, such as disputes regarding user fee assessments.
5 Jun 2023Cover Letter Attachments for Controlled Correspondences and ANDA Submissions

Source: FDA		Final, Level 1This guidance is intended to assist prospective applicants, applicants, and holders of ANDAs with optional attachments that can be used when preparing cover letters that accompany controlled correspondence, original ANDAs, amendments to ANDAs, and supplements to approved ANDAs submitted to FDA. These attachments do not replace the recommendations for the content of cover letters provided in other FDA guidances.
2 Jun 2023Nonclinical Evaluation of the Immunotoxic Potential of Pharmaceuticals

SOurce: FDA		Final, Level 1The purpose of this guidance is to assist sponsors in the nonclinical evaluation of the
immunotoxic potential of pharmaceuticals. Immunotoxicity is, for the purposes of this guidance, defined as unintended immunosuppression or stimulation (including hypersensitivity), which can include adverse effects of exaggerated pharmacology of pharmaceuticals that are intended to act
as immunomodulators. This guidance applies to drug products, including small molecule drugs and oligonucleotides, as well as certain biological products such as biotechnology-derived therapeutic proteins (referred to herein as biopharmaceuticals). For the purposes of this
guidance, the term pharmaceutical will be used as a general term that encompasses all of these
product types.

Cell and gene therapies, adjuvanted vaccines, and blood products are not within the scope of this guidance.
2 Jun 2023Drug-Drug Interaction Assessment for Therapeutic Proteins Guidance for Industry

Source: FDA		Final, Level 1The purpose of this guidance is to help sponsors of investigational new drug (IND) applications and applicants of biologic license applications (BLAs) determine the need for drug-drug interaction (DDI) studies for a therapeutic protein by providing recommendations for a systematic, risk-based approach.

For this guidance, a therapeutic protein refers to a protein that is being developed for licensure, or is licensed, as a biological product under section 351 of the Public Health Service Act (42 U.S.C. 262).
Therapeutic proteins:
• include purified monoclonal antibodies, cytokines, enzymes, and other novel proteins for in vivo use.
• do not include proteins intended to act as vaccines or allergenic products, cellular and gene therapy products, and/or human cells, tissues, and cellular and tissue-based products.

Although this guidance applies to therapeutic proteins, many of the general principles may be applicable to other biological products, such as novel products regulated by CBER (e.g., cellular and gene therapies). Due to the evolving knowledge of novel products, sponsors should consult corresponding review divisions for detailed information regarding a specific DDI assessment
1 Jun 2023Interstitial Cystitis/Bladder Pain Syndrome: Establishing Drug Development Programs for Treatment

Source: FDA		Draft, Level 1This guidance provides recommendations for clinical drug development for drugs intended to treat patients with interstitial cystitis/bladder pain syndrome (IC/BPS). This guidance incorporates advice FDA received at a December 2017 advisory committee meeting2 19 on appropriate patient selection criteria and trial design features, including enrollment criteria and acceptable efficacy endpoints for drugs intended to treat IC/BPS.
1 Jun 2023Migraine: Developing Drugs for Preventive Treatment

Source: FDA		DraftThe purpose of this guidance is to assist sponsors in the clinical development of drugs for the preventive treatment of migraine. Pharmacological approaches to the treatment of migraine include drugs to abort migraine attacks as they arise (acute treatment of migraine) and drugs to reduce the frequency of migraine attacks (preventive treatment). Specifically, this guidance addresses the FDA’s current thinking regarding the overall development program and clinical trial designs to support approval of drugs for the preventive
treatment of migraine.

This guidance does not address the development of drugs indicated for the acute treatment of migraine. Such development has been addressed in the guidance for industry Migraine: Developing Drugs for Acute Treatment (February 2018).
31 May Medication Guides: Patient Medication InformationProposed rule published in the FDA register)The FDA is proposing to amend its human prescription drug product labeling regulations for Medication Guides (FDA-approved written prescription drug product information distributed to patients).
•This action, if finalized, will require applicants to create a new type of Medication Guide, referred to as Patient Medication Information (PMI), for prescription drug products, including biological products, used, dispensed, or administered on an outpatient basis and for blood and blood components transfused in an outpatient setting.
•PMI would be a one-page document with standardized format and content that would be submitted to FDA for approval.
•This proposed rule is intended to improve public health by providing patients with clear, concise, accessible, and useful written prescription drug product information delivered in a consistent and easily understood format to help patients use their prescription drug products safely and effectively.

Either electronic or written comments on the proposed rule must be submitted by November 27, 2023.
26 May 2023Diabetes Mellitus: Efficacy Endpoints for Clinical Trials Investigating Antidiabetic Drugs and Biological Products

Source: FDA		Draft, Level 1This guidance :
• is intended to help sponsors develop antidiabetic drugs for adults and children with type 1 diabetes mellitus (T1D) and/or type 2 diabetes mellitus (T2D). In this guidance, antidiabetic drugs refer to drugs intended to improve glycemic control, including drugs intended to reduce diabetes-related hyperglycemia (i.e., antihyperglycemic drugs) and drugs intended to mitigate iatrogenic hypoglycemia associated with diabetes management.
• replaces, in part, the draft guidance for industry Diabetes Mellitus: Developing Drugs and Therapeutic Biologics for Treatment and Prevention, (73 FR 11420) published in February 2008. In March 2020, FDA withdrew the February 2008 draft guidance for industry because its recommendations for safety assessment were outdated. At the same time, FDA issued the draft guidance for industry Type 2 Diabetes Mellitus: Evaluating the Safety of New Drugs for
Improving Glycemic Control March 10, 2020 (85 FR 13903). When finalized, this guidance will address the FDA’s current recommendations regarding defining efficacy endpoints for clinical trials investigating antidiabetic drugs and replace the relevant sections in the withdrawn February 2008 draft guidance for industry.
• does not address the following topics: endpoints related to clinical complications of diabetes (e.g., cardiovascular risk reduction), endpoints related to the prevention or delay of T1D, or the use of hypoglycemia efficacy endpoints in trials of conditions other than diabetes mellitus (e.g., hypoglycemia related to bariatric surgery, congenital hyperinsulinism).
25 May 2023Adjusting for Covariates in Randomized Clinical Trials for Drugs and Biological Products

Source: FDA		Final, Level 1This guidance describes FDA’s current recommendations regarding adjusting for covariates in the statistical analysis of randomized clinical trials in drug development programs. This guidance provides recommendations for the use of covariates in the analysis of randomized, parallel group clinical trials that are applicable to both superiority trials and noninferiority trials. The main focus of the guidance is on the use of prognostic baseline covariates to improve statistical efficiency for estimating and testing treatment effects. This guidance does not address use of covariates to control for confounding variables in non-randomized trials, the use of covariates in models to account for missing outcome data (National Research Council 2010), the use of covariate adjustment for analyzing longitudinal repeated measures data, the use of Bayesian methods for covariate adjustment, or the use of machine learning methods for covariate adjustment.

You can view a guidance snapshot here, listen to a guidance recap podcast here and view the podcast transcript here.
24 May 2023Study Data Technical Conformance Guide – Technical Specifications Document

Source: FDA		FinalThis 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.
• 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.
24 May 2023Use of Whole Slide Imaging in Nonclinical Toxicology Studies: Questions and Answers

Source: FDA		Final, Level 1This guidance:
• provides information to sponsors and nonclinical laboratories regarding the use and management of whole slide images used during histopathology assessment and/or pathology peer review performed for good laboratory practice (GLP)-compliant nonclinical toxicology studies using non-human specimens.
• does not cover the use of whole slide imaging for clinical applications. When whole slide imaging is used as part of a nonclinical study conducted in compliance with the GLP regulations (21 CFR part 58), adequate documentation is critical. The FDA’s expectations regarding documentation practices during generation, use, and retention of whole slide images have not been clearly defined and vary among nonclinical testing facilities.
is intended to clarify FDA’s recommendations concerning the management, documentation, and use of whole slide imaging in histopathology assessment and/or pathology peer review for nonclinical studies conducted in compliance with the GLP regulations.

Link to Guidance Recap Podcast
23 May 2023Generally Accepted Scientific Knowledge in Applications for Drug and Biological Products: Nonclinical Information

Source: FDA		Draft, Level 1FDA has received an increasing number of questions regarding the extent to which generally accepted scientific knowledge (GASK) may be relied on for drug or biological product approval.

This guidance:
• describes instances in which it may be appropriate to rely on GASK to meet certain nonclinical safety requirements for new drug applications (NDAs) submitted under section 505(b) of the FD&C Act (21 U.S.C. 355(b)) and BLAs under section 351 of the PHS Act (42 U.S.C. 262(a)). The information that supports the nonclinical safety of a drug and that must be submitted in the application can include references to GASK, when appropriate, instead of or in addition to, specific studies conducted with respect to the drug. In such cases, therefore, it might be unnecessary to conduct certain nonclinical studies.
• focuses on two examples of GASK that could be appropriate to fulfill certain legal and regulatory requirements applicable to the drug or biological product in question. The examples discussed in this guidance reflect areas where we have previously determined that it was appropriate to rely on GASK to meet certain requirements for approval. Notably, while the examples provided here are for illustrative purposes, determinations regarding the appropriateness of data submitted for any application, including GASK, are fact-specific, and the question of whether certain information can be considered GASK and the purpose such information would serve in an application will be considered in the context of a particular application.
16 May 2023Pediatric Drug Development: Regulatory Considerations — Complying With the Pediatric Research Equity Act and Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for Children Act

Source: FDA		Draft, Level 1This guidance is intended to assist industry developing drug products to comply with the pediatric study requirements under the Pediatric Research Equity Act (PREA), and to describe the process for qualifying for pediatric exclusivity and the protections that pediatric exclusivity offers under the Best Pharmaceuticals for Children Act (BPCA) In 2010, the Biologics Price Competition and Innovation Act of 2009 extended provisions of the BPCA to biological products.

This guidance, along with the draft guidance for industry Pediatric Drug Development Under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act: Scientific Considerations, revises and replaces the draft guidance for industry How to Comply With the Pediatric Research Equity Act. In addition to addressing the PREA topics covered in the earlier draft guidance (i.e., the pediatric assessment, pediatric plan, waivers and deferrals, compliance issues, and pediatric exclusivity provisions), this guidance addresses statutory changes relating to adverse event reporting, pediatric study plans (PSPs), deferral extensions, and noncompliance.
16 May 2023Pediatric Drug Development Under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act: Scientific Considerations

Source: FDA		Draft, Level 1The purpose of this guidance is to assist industry in developing data and obtaining information needed to support approval of drug products in pediatric populations.
This guidance:
• addresses selected clinical, scientific, and ethical issues regarding the development of drugs for pediatric use when such drugs are subject to the Pediatric Research Equity Act (PREA)3 23 and/or the Best Pharmaceuticals for Children Act (BPCA). In 2010, the Biologics Price Competition and Innovation Act of 2009 extended provisions of the BPCA to biological products.
• does not address the clinical development of drugs that are not subject to either PREA or the BPCA.
• along with the draft guidance for industry Pediatric Drug Development: Regulatory Considerations — Complying With the Pediatric Research Equity Act and Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for Children Act (May 2023) (draft Regulatory Considerations guidance), revises and replaces the draft guidance for industry How to Comply With the Pediatric Research Equity Act.
• also addresses certain additional topics that FDA has not previously addressed in guidance.
• only addresses briefly the Food and Drug Administration Reauthorization Act of 2017’s (FDARA’s) amendments to PREA8 36 relating to requirements that sponsors of certain adult oncology drugs with molecular targets that are determined to be substantially relevant to the growth or progression of a pediatric cancer submit reports on molecularly targeted pediatric cancer investigations.
10 May 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).
• 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 FD&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.
• provides technical recommendations to sponsors for the submission of animal and human study data and related information in a standardized electronic format in INDs, NDAs, ANDAs, and BLAs.
• 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.
3 May 2023Q9(R1) Quality Risk Management
Source: FDA		FinalThe purpose of this document is to offer a systematic approach to quality risk management that leads to 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.
1 May 2023Decentralized Clinical Trials for Drugs, Biological Products, and Devices

Source: FDA		Draft Level 1This draft guidance provides recommendations for sponsors, investigators, and other stakeholders regarding the implementation of decentralized clinical trials (DCTs) for drugs,
biological products, and devices. In this guidance, a DCT refers to a clinical trial where some or all of the trial-related activities occur at locations other than traditional clinical trial sites.

In fully decentralized clinical trials, all activities take place at locations other than traditional trial sites. These trial-related activities may take place at the homes of trial participants or in local health care facilities that are convenient for trial participants. In hybrid DCTs, some trial activities involve in-person visits by trial participants to traditional clinical trial sites, and other activities are conducted at locations other than traditional clinical trial sites, such as participants’ homes.

FDA’s regulatory requirements for investigations of medical products are the same for DCTs and
traditional site-based clinical trials. Section 3606(a) of the Food and Drug Omnibus Reform Act (FDORA) directs FDA to “issue or revise draft guidance that includes recommendations to clarify and advance the use of decentralized clinical studies to support the development of drugs and devices,” not later than December 9, 2023.

This guidance provides recommendations related to FDA’s requirements for investigations of medical products when applied to DCTs and
fulfils the requirement set forth in section 3606(a)(1) of FDORA. The content described in
section 3606(b) of FDORA is further addressed through this guidance’s reference to the draft
guidance for industry, investigators, and other stakeholders entitled Digital Health Technologies for Remote Data Acquisition in Clinical Investigations (December 2021).

More here on additional steps taken by the FDA to enhance decentralised clinical trials.
27 Apr 2023S12 Nonclinical Biodistribution Considerations for Gene Therapy Products

Source: FDA		Final Level 1The objective of this guidance is to provide harmonized recommendations for the conduct of nonclinical biodistribution (BD) studies in the development of gene therapy (GT) products. This document provides recommendations for the overall design of nonclinical BD assessments. Considerations for interpretation and application of the BD data to support a nonclinical development program and the design of clinical trials are also provided. The recommendations in this guidance endeavor to facilitate the development of GT products while avoiding unnecessary use of animals, in accordance with the 3Rs (reduce/refine/replace) principles
12 Apr 2023Assessing the Irritation and Sensitization Potential of Transdermal and Topical Delivery Systems for ANDAs

Source: FDA		DraftThis guidance:
• provides recommendations for the design and conduct of studies to evaluate the in vivo skin irritation and sensitization (I/S) potential of a proposed transdermal or topical delivery system (collectively referred to as TDS). The recommendations in this guidance relate to studies submitted in support of an abbreviated new drug application (ANDA).
• revises the draft guidance for industry Assessing the Irritation and Sensitization Potential of Transdermal and Topical Delivery Systems for ANDAs (October 2018). This revision provides the following updates to the original draft guidance:
(1) Clarifies recommendations for the design and conduct of studies to evaluate the in vivo skin I/S potential of a proposed TDS.
(2) Clarifies when an in vivo study to assess the sensitization potential of a TDS product may not be needed.
12 Apr 2023Over-the-Counter Monograph Order Requests: Format and Content

Source: FDA		Draft Level 1This guidance:
• is intended to assist requestors in preparing over-the-counter (OTC) monograph order requests (OMORs) for submission to FDA under section 505G of the FD&C Act (21 U.S.C. 355h).
• provides FDA’s recommendations on the format and content of the information that requestors should provide in an OMOR and identifies relevant guidance documents to assist requestors in preparing their OMORs.
• provides an overview of the information that FDA may recommend for a sufficiently complete OMOR.
• is not intended to indicate the studies and related information that a requestor must submit in a specific OMOR.
11 Apr 2023Study Data Technical Conformance Guide – Technical Specifications Document

Source: FDA		FinalThis technical specifications document represents the FDA’s current thinking on this topic. It does not create or confer any rights for or on any person and does not operate to bind FDA or the public. You can use an alternative approach if the approach satisfies the requirements of the applicable statutes and regulations.
11 Apr 2023A Risk-Based Approach to Monitoring of Clinical Investigations Questions and Answers

Source: FDA		FinalThis guidance:
• provides information on risk-based approaches to monitoring the conduct of clinical investigations of human drug and biological products, medical devices, and
combination products. Clinical investigation monitoring is a quality control tool for
determining whether investigation activities are being carried out as planned.
• contains recommendations on planning a monitoring approach, developing the content of a monitoring plan, and addressing and communicating monitoring results.
• expands on the guidance for industry Oversight of Clinical Investigations – A Risk-Based Approach to Monitoring (August 2013) (the 2013 RBM guidance) by providing additional information to facilitate sponsors’ implementation of risk-based monitoring.
5 Apr 2023Notifying FDA of a Discontinuance or Interruption in Manufacturing of Finished Products or Active Pharmaceutical Ingredients Under Section 506C of the FD&C Act

Source: FDA		DraftFDA is issuing this guidance to assist applicants and manufacturers in providing FDA timely, informative notifications about changes in the production of certain finished drugs and biological products as well as certain active pharmaceutical ingredients (API) that may, in turn, help the Agency in its efforts to prevent and mitigate shortages.

The guidance discusses the notification requirements under section 506C of the FD&C Act (21 U.S.C. 356c) and FDA’s regulations. Generally, section 506C of the FD&C Act requires applicants and manufacturers of certain finished drugs and biological products to notify FDA of:
(1) a permanent discontinuance in the manufacture of such products
(2) an interruption in the manufacture of such products that is likely to lead to a meaningful disruption in supply of those products in the United States
(3) a permanent discontinuance in the manufacture of API for such products, or
(4) an interruption in the manufacture of API for such products that is likely to lead to a meaningful disruption in the supply of the API for those products.
This guidance recommends that applicants and manufacturers provide additional details and follow additional procedures to ensure FDA has the specific information it needs to help prevent or mitigate shortages. In addition, the guidance explains how FDA communicates information about products in shortage to the public. When finalized, this guidance will replace the March 2020 guidance for industry Notifying FDA of a Permanent Discontinuance or Interruption in Manufacturing Under Section 506C of the FD&C Act.
5 Apr 2023Patient-Focused Drug Development: Incorporating Clinical Outcome Assessments Into Endpoints for Regulatory Decision-Making

Source: FDA		DraftThis guidance (Guidance 4) is the fourth 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. The topics that each guidance document addresses are described below:

• Methods to collect patient experience data that are accurate and representative of the intended patient population (Guidance 1)
• Approaches to identifying what is most important to patients with respect to their experience as it relates to burden of disease/condition and burden of treatment (Guidance 2)
• Approaches to selecting, modifying, developing, and validating clinical outcome assessments (COAs) to measure outcomes of importance to patients in clinical trials (Guidance 3)
• Methods, standards, and technologies for collecting and analyzing COA data for regulatory decision-making, including selecting the COA-based endpoint and determining clinically meaningful change in that endpoint (Guidance 4; current guidance)