A closer look at CDER’s new 2024 guidance agenda

The FDA’s Center for Drug Evaluation and Research, its drug review division, this week quietly updated its 2024 Guidance Agenda, a list of all draft guidance documents the agency is working on this calendar year. AgencyIQ analyzed the document to determine which documents were new, which had been carried over from the last edition of the agenda, and which documents had been removed. Our review found that CDER is planning several notable new guidances, including one on artificial intelligence.

BY RACHEL COE, MSC | FEB 2, 2024 9:53 PM CST

Quick Context

  • What is a guidance agenda? Under the FDA’s Good Guidance Practices regulation at 21 CFR 10.115, the agency is required to annually publish “a list of possible topics for future guidance document development or revision during the next year.” This list is known as the FDA’s “guidance agenda.” However, the FDA does not publish a singular guidance agenda; rather, it publishes separate agendas from each major product review center (CBER, CDER, CDRH, CFSAN), as well as some additional FDA offices (like its Office of the Chief Scientist).
  • FDA’s Center for Drug Evaluation and Research (CDER) usually publishes an initial guidance agenda at the start of the calendar year along with an updated version sometime between July and September. The same is true for the Center for Biologics Evaluation and Research (CBER) [Read Agency IQ’s breakdown of the 2024 CBER guidance agenda here]. FDA’s Center for Devices and Radiological Health (CDRH) departs from this practice and publishes its guidance agendas just once at the start of the fiscal year (each October). [Read Agency IQ’s breakdown of the 2024 CDRH guidance agenda here].

What’s new?

  • This week, CDER published its guidance agenda for 2024. AgencyIQ has reviewed the Guidance Agenda to determine what has changed since the time the agenda was released. In total, since August 2023, there have been 34 new guidances added to the agenda, 58 that have been carried over to the new calendar year, and 28 which were removed (most—but not all—of which were published since the last guidance agenda update).
  • Of particular interest: CDER is planning a new guidance on Artificial Intelligence to “support regulatory decision making for drugs and biological products.” This is a topic that the agency has been keenly aware of for years, but it’s not clear from the title if it will refer to how the FDA will use A.I. in its regulatory reviews, or whether it will refer to how industry can use A.I. to analyze data, generate summaries of certain information, or other tasks of interest to companies in the interest of efficiencies.
  • A new real-world evidence guidance on the list is meant to address the integration of randomized controlled trials for drug and biological products into routine clinical practice. This is a topic that seems highly related to a recent report from the Reagan-Udall Foundation for the FDA focused on “enhancing post-market evidence generation for medical products” that calls for a “learning health care system.”
  • Other guidances of note include one on the “study of sex differences in the clinical evaluation of medical products.” FDA first published a guidance on a similar topic in 1993 (“ Study and evaluation of gender differences”), but it’s not clear if the FDA plans to replace the existing guidance or publish one that is complimentary to it. Another guidance will focus on pharmacokinetics in pregnancy, likely replacing a 2004 guidance document that the FDA never finalized, while a guidance the creation of a “REMS Logic Model” framework is meant to “link program design with assessment” – something called for in the most recent PDUFA VII commitment letter.

CDER’s 2024 guidance agenda:

Title Category New or repeated from previous agenda?
Accelerated Approval of Drugs and Biologics Administrative/ Procedural New
Civil Monetary Penalties for Failure to Meet Accelerated Post Marketing Requirements Administrative/ Procedural Carried over from previous guidance agenda
Exclusivity for First Interchangeable Biosimilar Biological Products Administrative/ Procedural Carried over from previous guidance agenda
Institutional Review Board (IRB) Review of Individual Patient Expanded Access Requests for Investigational Drugs and Biological Products Administrative/ Procedural Carried over from previous guidance agenda
Key Information and Facilitating Understanding in Informed Consent Administrative/ Procedural New*
NDC Creation, Assignment, Listing and Appropriate Use for Human Drugs, Including Biological Products Administrative/ Procedural New*
Priority Review Voucher Programs Administrative/ Procedural Carried over from previous guidance agenda
Qualified Infectious Disease Product Designation—Questions and Answers Administrative/ Procedural New
Repackaging and Relabeling of Human Drugs: Labeling; Registration and Listing, Safety Reporting, Supply Chain Security, and Good Manufacturing Practice Administrative/ Procedural Carried over from previous guidance agenda
Responding to Form FDA 483 Observations at the Conclusion of a Drug CGMP Administrative/ Procedural Carried over from previous guidance agenda
Considerations in Demonstrating Interchangeability with a Reference Product: Update Biosimilars Carried over from previous guidance agenda
Pediatric Study Plans for Biosimilar Products Biosimilars Carried over from previous guidance agenda
Pharmacokinetics in Pregnancy — Study Design, Data Analysis, and Impact on Dosing and Labeling Clinical Pharmacology New
Chronic Pain: Developing Drugs for Treatment Clinical/ Medical Carried over from previous guidance agenda
Collection of Race and Ethnicity Data in Clinical Trials and Clinical Studies for FDA Regulated Medical Products Clinical/ Medical New, already published
Considerations for the Use of Artificial Intelligence to Support Regulatory Decision Making for Drugs and Biological Products Clinical/ Medical New
Developing Products for Chronic Weight Management; Revised Draft Clinical/ Medical Carried over from previous guidance agenda
Development of Animal-Derived Thyroid Products Clinical/ Medical New
Development of Non-Opioid Analgesics for Chronic Pain Clinical/ Medical New
Diabetes Mellitus: Efficacy Endpoints for Clinical Trials Investigating Antidiabetic Drugs and Biological Products Clinical/ Medical Carried over from previous guidance agenda
Drugs With Teratogenic Potential — Recommendations for Pregnancy Planning and Prevention Clinical/ Medical Carried over from previous guidance agenda
Early Alzheimer’s Disease: Developing Drugs for Treatment; Revised Draft Clinical/ Medical Carried over from previous guidance agenda
Endometriosis-Associated Pain: Establishing Effectiveness and Safety of Drugs for Management Clinical/ Medical New
Optical Imaging: Developing Drugs for Surgical Oncology Clinical/ Medical New
Pediatric Inflammatory Bowel Disease: Developing Drugs for Treatment Clinical/ Medical Carried over from previous guidance agenda
Protocol Deviations Clinical/ Medical Carried over from previous guidance agenda
Radiation Dosimetry for First-in-Human Studies of Positron Emission Tomography Drugs Clinical/ Medical New
Study of Sex Differences in the Clinical Evaluation of Medical Products Clinical/ Medical New
Use of Data Monitoring Committees in Controlled Clinical Trials Clinical/ Medical Carried over from previous guidance agenda
Compounded Drug Products That Are Essentially Copies of a Commercially Available Drug Product Under Section 503A of the Federal Food, Drug, and Cosmetic Act; Revised Draft Compounding Carried over from previous guidance agenda
Compounded Drug Products That Are Essentially Copies of Approved Drug Products Under Section 503B of the Federal Food, Drug, and Cosmetic Act; Revised Draft Compounding Carried over from previous guidance agenda
Nomination of Bulk Drug Substances for Use in Compounding Under Section 503B of the Federal Food, Drug, and Cosmetic Act Compounding Carried over from previous guidance agenda
Pharmacy Compounding of Human Drug Products Under Section 503A of the Federal Food, Drug, and Cosmetic Act; Revised Draft Compounding Carried over from previous guidance agenda
Safety Considerations for Container Labels and Carton Labeling Design to Minimize Medication Errors-Guidance for Outsourcing Facilities Under Section 503B of the FD&C Act Compounding Carried over from previous guidance agenda
Biomarker Qualification: Evidentiary Framework Drug Development Tools Carried over from previous guidance agenda
Development of a Shared System or Separate REMS; Revised Draft Drug Safety Carried over from previous guidance agenda
Purpose and Content of Use-Related Risk Analyses Drug Safety Carried over from previous guidance agenda
REMS Logic Model: A Framework to Link Program Design with Assessment Drug Safety New
“Open for Business” Definition Under 744B of the Federal Food, Drug and Cosmetic Act Generics Carried over from previous guidance agenda
180-Day Exclusivity: Questions and Answers; Revised Draft Generics Carried over from previous guidance agenda
30-Month Stay of Approval of a 505(b)(2) Application or an ANDA Generics Carried over from previous guidance agenda
ANDA Submissions – Refuse-to-Receive for DMF Facilities Deficiencies Generics Carried over from previous guidance agenda
ANDA Submissions – Refuse-to-Receive Standards: Questions and Answers Generics Carried over from previous guidance agenda
Assessing Adhesion with Transdermal Delivery Systems and Topical Patches for ANDAs; Revised Draft Generics New*
Assessing the Irritation and Sensitization Potential of Transdermal and Topical Delivery Systems for Abbreviated New Drug Applications; Revised Draft Generics New*
Bioequivalence Recommendations for Specific Products Generics New
Bioequivalence Studies With Pharmacokinetic Endpoints for Drugs Submitted Under an Abbreviated New Drug Application; Revised Draft Generics New
Content and Format of Composition Tables in NDAs and ANDAs And Corresponding Statement of Ingredients in Labeling Generics Carried over from previous guidance agenda
Data Integrity for In Vivo Bioavailability and Bioequivalence Studies Generics Carried over from previous guidance agenda
Determining Whether to Submit an ANDA or 505(b)(2) Application Generics Carried over from previous guidance agenda
Form FDA 3542a and Form FDA 3542: Questions and Answers Generics New
Handling and Retention of BA and BE Testing Samples Generics Carried over from previous guidance agenda
In Vitro Permeation Tests for Semisolid Topical Products Submitted in ANDAs; Revised Draft Generics New
In Vitro Release Tests for Semisolid Topical Products Submitted in ANDAs; Revised Draft Generics New
Mechanistic Modeling and Simulation Approaches for Non-Orally Administered (locally applied) Drug Products Generics New
New Clinical Investigation Exclusivity for Drug Products: Questions and Answers Generics Carried over from previous guidance agenda
Pediatric Exclusivity General Considerations for ANDAs Generics Carried over from previous guidance agenda
Physico-Structural (Q3) Characterization of Topical Dermatological Drug Products Submitted in ANDAs; Revised Draft Generics New
Submission of Patent Information for Listing in the Orange Book Generics New
E20 Adaptive Clinical Trials ICH Carried over from previous guidance agenda
E2D(R1) Post-Approval Safety Data Management: Definitions and Standards for Expedited Reporting ICH Carried over from previous guidance agenda
E2D(R1) Post-Approval Safety Data: Definitions and Standards for Management and Reporting of Individual Case Safety Reports ICH New
E6(R3) Good Clinical Practice Annex 2 ICH Carried over from previous guidance agenda
M13B Bioquivalence for Additional Solid, Oral Dosage Strengths Including Biowaiver Considerations ICH Carried over from previous guidance agenda
M14 General Principles on Planning and Designing Pharmacoepidemiological Studies that Utilize Real-World Data for Safety Assessment ICH Carried over from previous guidance agenda
M15 MIDD General Principles ICH New
M4Q(R2) Revision of M4Q(R1) CTD on Quality ICH Carried over from previous guidance agenda
Q1/Q5C Targeted Revisions of ICH Stability Guidelines ICH New
Q3E Impurity: Assessment and Control of Extractables and Leachables for Pharmaceuticals and Biologics ICH Carried over from previous guidance agenda
Q5A(R2) Biotechnology Products Derived from Cell Lines of Human or Animal Origin ICH Carried over from previous guidance agenda
Combined Hormonal Contraceptives for Prevention of Pregnancy-Labeling for Health Care Providers and Patients Labeling Carried over from previous guidance agenda
Drug Interaction Section of Labeling for Human Prescription Drug and Biological Products – Content and Format Labeling Carried over from previous guidance agenda
Impact of Identifying Group Purchasing Organizations on a Drug Label Labeling New*
Annual Reportable Labeling Changes for NDAs and ANDAs for Nonprescription Drug Products OTC Drugs Carried over from previous guidance agenda
Formal Dispute Resolution and Consolidated Proceedings: Requestor of OMUFA Products Appeals Above the Division Level OTC Drugs Carried over from previous guidance agenda
Approaches to Meeting CGMP Requirements for Distributed Manufacturing Pharmaceutical Quality/ CGMP Carried over from previous guidance agenda
Laboratory Testing of Drugs Held in Interstate Commerce: Compliance with CGMP Pharmaceutical Quality/ CGMP New
PET Drugs – Current Good Manufacturing Practice (CGMP); Revised Draft Pharmaceutical Quality/ CGMP Carried over from previous guidance agenda
ANDAs: Stability Testing of Drug Substances and Products Q&A Pharmaceutical Quality/ CMC Carried over from previous guidance agenda
Considerations for Complying with 21 CFR 211.110 Pharmaceutical Quality/ CMC Carried over from previous guidance agenda
Container Closure System and Component Changes: Glass Vials and Stoppers; Immediately in Effect Pharmaceutical Quality/ CMC New
Container Closure Systems for Drugs, Including Biological Products Pharmaceutical Quality/ CMC Carried over from previous guidance agenda
Control of Nitrosamine Impurities in Human Drugs; Immediately in Effect Upon Publication (Revision 2) Pharmaceutical Quality/ CMC Carried over from previous guidance agenda
Platform Technology Designation Program for Drug Development Pharmaceutical Quality/ CMC New
Post-approval Manufacturing Changes to Biosimilars and Interchangeable Biosimilars Questions and Answers Pharmaceutical Quality/ CMC Carried over from previous guidance agenda
Quality Management Maturity Program Draft Guidance Pharmaceutical Quality/ CMC New
Stability Considerations for Drug Substances and Drug Products in NDAs, ANDAs, and BLAs and Associated Labeling Statements for Drug Products Pharmaceutical Quality/ CMC Carried over from previous guidance agenda
Stability Recommendations for Additional Manufacturing Facilities in NDAs, ANDAs and BLAs, and Additional Drug Substance Sources in NDAs and ANDAs Pharmaceutical Quality/ CMC Carried over from previous guidance agenda
Considerations Regarding Non-Interventional Studies for Drug and Biological Products RWD/ RWE Carried over from previous guidance agenda
Integrating Randomized Controlled Trials for Drug and Biological Products Into Routine Clinical Practice RWD/ RWE New
Promotional Labeling and Advertising Considerations for Prescription Biological Reference, Biosimilar, and Interchangeable Products – Questions and Answers Promotional Labeling and Advertising New
Using Clinical Practice Data in Randomized Controlled Trials (RCT) for Regulatory Decision-Making for Drug and Biological Products RWD/ RWE Carried over from previous guidance agenda

A quick look at the guidances removed from the prior agenda:

Title Category Outcome
Classification Categories for Certain Supplements Under 351(k) of the PHS Act Administrative/ Procedural Published August 2023
Formal Meetings Between the FDA and Sponsors or Applicants of BsUFA Products; Revised Draft Administrative/ Procedural Published August 2023
Master Protocols for Drug Development and Biological Product Development Administrative/ Procedural Published December 2023
Porcine Derived Thyroglobulin Products Administrative/ Procedural  
Use of Generally Accepted Scientific Knowledge in Applications for Drugs and Biological Products: Nonclinical Information Administrative/ Procedural Published May 2023
Product Class-Specific Recommendations for Developing Biosimilar and Interchangeable Biological Products Biosimilars  
Clinical Pharmacogenomics: Premarket Evaluation in Early-Phase Clinical Studies and Recommendations for Labeling Clinical Pharmacology  
Clinical Pharmacology Considerations for Peptides Clinical Pharmacology Published September 2023
Early Lyme Disease as Manifested by Erythema Migrans: Developing Drugs for Treatment Clinical/Antimicrobial Published January 2023
Demonstrating Substantial Evidence Standard Based on One Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence Clinical/ Medical Published September 2023
Development of 351(a) Biologics License Applications for Thyroid Products Clinical/ Medical  
Development of Local Anesthetic Drug Products with Prolonged Duration of Effect Clinical/ Medical Published March 2023
Endogenous Cushing’s Syndrome: Developing Drugs for Treatment Clinical/ Medical Published September 2023
Interstitial Cystitis/Bladder Pain Syndrome: Establishing Drug Development Programs for Treatment Clinical/ Medical Published June 2023
Neovascular Age-Related Macular Degeneration: Developing Drugs for Treatment Clinical/ Medical Published February 2023
Psychedelic Drugs: Considerations for Scientific Investigations Clinical/ Medical Published June 2023
Stimulant Use Disorders: Developing Drugs for Treatment Clinical/ Medical Published October 2023
Identification of Medicinal Products: Implementation and Use Electronic Submissions Published March 2023
ANDA Submissions – Amendments and Requests for Final Approval to Tentatively Approved ANDAs Generics Published January 2024
ANDA Submissions – Amendments to Abbreviated New Drug Applications Under GDUFA; Revised Draft Generics  
Comparative Analyses and Related Comparative Use Human Factors Studies for a Drug-Device Combination Product Submitted in an ANDA; Revised Draft Generics Potentially addressed by another FDA guidance on human factors studies.
E6(R3) Good Clinical Practice Principles and Annex 1 ICH Published June 2023
Regulatory Considerations for Prescription Drug Use-Related Software Labeling Published September 2023
Advanced Manufacturing Technologies Designation Program Designated Technologies in Drug and Biological Products Pharmaceutical Quality/ CMC Published December 2023
Products With Benzene-Containing Carbomers: Recommendations for Reformulation Pharmaceutical Quality/ CMC Published December 2023
Quality Considerations for Topical Ophthalmic Drug Products Pharmaceutical Quality/ CMC Published December 2023
Use of Alternative Tools to Assess Manufacturing Facilities Named in Pending Applications Pharmaceutical Quality/ CMC Published September 2023
Translation of Nonclinical Toxicology Study Reports: Questions and Answers Pharmacology/ Toxicology Published November 2023

What’s next?

  • The guidance agenda is not binding, and there’s plenty of reason to be skeptical that we will see these new guidance documents by the end of 2024. Despite the title of FDA’s guidance agenda (“Documents Planned for Publication in Calendar Year 2024), it is under no legal obligation to follow the agenda.

To contact the author of this item, please email Rachel Coe ( rcoe@agencyiq.com).
To contact the editor of this item, please email Alexander Gaffney ( agaffney@agencyiq.com).

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