CDER unveils new guidance agenda for 2023, with 42 new policies planned

Life Sciences | By ALEXANDER GAFFNEY, MS, RAC

FDA’s Center for Drug Evaluation and Research (CDER) has unveiled a new guidance agenda covering the guidance documents it potentially plans to release in 2023. While many of the documents are holdovers from its 2022 guidance agenda, there are also 42 new additions to the list of 98.

Regulatory Background

FDA’s Center for Drug Evaluation and Research (CDER) publishes a guidance agenda every calendar year to inform the public of its planned activities. Similarly, other centers, the Center for Biologics Evaluation and Research (CBER) and the Center for Devices and Radiological Health (CDRH) publish their respective guidance agendas. This document is updated throughout the year as appropriate, but typically at least once.
CDER’s guidance agenda was last updated in July 2022 and typically includes about 100 documents planned for release. The agenda is aspirational – the center is under no obligation to release documents on the list and it often doesn’t release a significant number of them. Still, it is often useful to determine which subjects have risen to the level of attention that the center believes additional guidance would be necessary.
This week CDER quietly released an updated list of the guidance documents it plans to release in 2023. The list contains some holdovers from 2022 (and earlier), which AgencyIQ expected since the center was unable to release dozens of documents that had been on its list last year. However, the new agenda also includes a significant number of new documents as well.
AgencyIQ has reviewed the Guidance Agenda to determine which of the 98 guidance documents are new and which are carry-overs from last year. We identified 42 new guidance documents on the list, which are noted in the list below as “NEW.” There are also a handful of documents that have already been published but are set to be revised.

New and noteworthy on the agenda

Despite a massive amount of industry interest, there’s no new guidance on cannabis scheduled for this year – but there is a newly-planned guidance on psychedelic drugs. That guidance, Psychedelic drugs: Considerations for scientific investigation, is likely to be of interest to companies working on products like psilocybin or MDMA-derived drugs, and comes just as FDA is expecting to begin receiving submissions for more of the drugs for conditions like post-traumatic stress disorder.
Several new biosimilars guidance documents are on the way: Buried within the document (and not necessarily under the biosimilars category heading) are several titles of guidance documents that are likely to benefit the biosimilars industry. There’s one on classification categories for biosimilar supplemental applications; another on exclusivity for first interchangeable biosimilars (likely reflecting a recent legal change under the Food and Drug Omnibus Reform Act that permits multiple companies to obtain this exclusivity); another on product class-specific recommendations for developing biosimilars and interchangeable biological products; one on labeling for biosimilars and interchangeables; and a final document on post-approval manufacturing changes to biosimilars and interchangeables.
There’s quite a bit of new generic drug guidance planned as well, including one on how FDA intends to handle 30-month stays of approval for 505(b)(2) applications or an Abbreviated New Drug Application, a revised draft on amendments to ANDAs, a new guidance on making amendments to tentatively approved ANDAs, and a guidance on “determining whether to submit an ANDA or 505(b)(2) application.”
Another guidance on master protocols: To date, FDA has released a few different guidance documents that have explained how sponsors can take advantage of master protocols, including one released in final form in March 2022. Now CDER says it’s working on another one, entitled Master Protocols for Drug Development and Biological Product Development. The document title doesn’t give too much away, although since the prior guidance focused on oncology products, we expect this one to be more general in its scope (which industry had explicitly asked for in response to the last guidance).
A few guidance documents on hot topics: We get a lot of questions about FDA guidance here at AgencyIQ, and this list has plenty of new entries that would help answer questions that we get asked about often. There’s a new guidance on priority review vouchers. Another on pediatric drug development and qualifying for exclusivity. One on how IRBs can review individual patient expanded access requests, and another on how to notify FDA of permanently discontinued or interrupted drugs. Two others caught our eye as well: One on protocol deviations, and another on the purpose of use-related risk analyses. All of these topics tend to come up a lot, and from a wide range of companies.
One guidance new draft guidance on a much-anticipated topic will address efficacy endpoints for diabetes, and specifically “clinical trials investigating antidiabetic drugs and biological products.” This is an area of substantial development for the life sciences industry.

CDER’s 2023 Guidance Agenda

Document	Category	Notes
Civil Monetary Penalties for Failure to Meet Accelerated Post Marketing Requirements	Administrative, Procedural
Classification Categories for Certain Supplements Under 351(k) of the PHS Act	Administrative, Procedural	NEW
Electronic Records, and Electronic Signatures in Clinical Investigations: Questions and Answers	Administrative, Procedural
Exclusivity for First Interchangeable Biosimilar Biological Products	Administrative, Procedural	NEW
Fixed Dose Combinations and Single-Entity Versions of Previously Approved Antiretrovirals for the Treatment of Human Immunodeficiency Virus-1 Under President’s Emergency Plan for AIDS Relief (PEPFAR)	Administrative, Procedural
Formal Dispute Resolution and Administrative Hearings of Final Administrative Orders Under Section 505G of the Food, Drug, and Cosmetic Act	Administrative, Procedural
Formal Meetings Between the FDA and Sponsors or Applicants of BsUFA Products; Revised Draft	Administrative, Procedural	NEW; REVISED DRAFT
Institutional Review Board (IRB) Review of Individual Patient Expanded Access Requests for Investigational Drugs and Biological Products	Administrative, Procedural	NEW
Key Information and Facilitating Understanding in Informed Consent for FDA-Regulated Clinical Investigations	Administrative, Procedural
Master Protocols for Drug Development and Biological Product Development	Administrative, Procedural	NEW
Notification of a Permanent Discontinuance or Interruption in Manufacturing Under Section 506C of the FD&C Act	Administrative, Procedural	NEW
Pediatric Drug Development: Regulatory Considerations – Complying with the Pediatric Research Equity Act and Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for Children Act	Administrative, Procedural	NEW
Pediatric Drug Development Under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act: Scientific Considerations	Administrative, Procedural	NEW
Porcine Derived Thyroglobulin Products	Administrative, Procedural	NEW
Priority Review Voucher Programs	Administrative, Procedural	NEW
Responding to CGMP Observations on Form FDA 483	Administrative, Procedural
Use of Generally Accepted Scientific Knowledge in Applications for Drugs and Biological Products: Nonclinical Information	Administrative, Procedural	NEW
Acute Radiation Syndrome: Developing Drugs for Prevention and Treatment	Animal Rule
Product Class-Specific Recommendations for Developing Biosimilar and Interchangeable Biological Products	Biosimilars
Early Lyme Disease as Manifested by Erythema Migrans: Developing Drugs for Treatment	Clinical, Antimicrobial	ALREADY RELEASED
Chronic Pain: Developing Drugs for Treatment	Clinical, Medical
Decentralized Clinical Trials	Clinical, Medical
Demonstrating Substantial Evidence Standard Based on One Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence	Clinical, Medical
Development of 351(a) Biologics License Applications for Thyroid Products	Clinical, Medical	NEW
Development of Local Anesthetic Drug Products with Prolonged Duration of Effect	Clinical, Medical	NEW
Diabetes Mellitus: Efficacy Endpoints for Clinical Trials Investigating Antidiabetic Drugs and Biological Products	Clinical, Medical	NEW
Drugs With Teratogenic Potential — Recommendations for Pregnancy Planning and Prevention	Clinical, Medical	NEW
Endogenous Cushing’s Syndrome: Developing Drugs for Treatment	Clinical, Medical	NEW
Inborn Errors of Metabolism That Use Dietary Management: Considerations for Optimizing and Standardizing Diet in Clinical Trials for Drug Product Development	Clinical, Medical
Interstitial Cystitis/Bladder Pain Syndrome: Establishing Drug Development Programs for Treatment	Clinical, Medical	NEW
Migraine: Developing Drugs for Preventive Treatment	Clinical, Medical
Neovascular Age-Related Macular Degeneration: Developing Drugs for Treatment	Clinical, Medical
Pediatric Inflammatory Bowel Disease: Developing Drugs for Treatment	Clinical, Medical
Protocol Deviations	Clinical, Medical	NEW
Psychedelic Drugs: Considerations for Scientific Investigations	Clinical, Medical	NEW
Stimulant Use Disorders: Developing Drugs for Treatment	Clinical, Medical	NEW
Use of Data Monitoring Committees in Controlled Clinical Trials	Clinical, Medical
Clinical Pharmacology Considerations for Peptides	Clinical Pharmacology	NEW
Clinical Pharmacogenomics: Premarket Evaluation in Early-Phase Clinical Studies and Recommendations for Labeling	Clinical Pharmacology	NEW
Pharmacogenomic Data Submission	Clinical Pharmacology
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	REVISED DRAFT
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	REVISED DRAFT
Nomination of Bulk Drug Substances for Use in Compounding Under Section 503B of the Federal Food, Drug, and Cosmetic Act	Compounding
Pharmacy Compounding of Human Drug Products Under Section 503A of the Federal Food, Drug, and Cosmetic Act; Revised Draft	Compounding	REVISED DRAFT
Prohibition on Wholesaling Under Section 503B of the Federal Food, Drug, and Cosmetic Act	Compounding
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
Biomarker Qualification: Evidentiary Framework	Drug Development Tools
Postmarketing Studies and Clinical Trials: Determining Good Cause for Noncompliance with Section 505(o)(3)(E)(ii) of the Federal Food, Drug, and Cosmetic Act	Drug Safety
Development of a Shared System or Separate Comparable Risk Evaluation and Mitigation Strategies; Revised Draft	Drug Safety	REVISED DRAFT
Purpose and Content of Use-Related Risk Analyses	Drug Safety	NEW
Identification of Medicinal Products: Implementation and Use	Electronic Submissions
NDC Assignment of Human Drugs including Biological Products	Electronic Submissions
Group Purchasing Organization vs. Private Label Distributor	Electronic Submissions
Repackagers and Relabelers of Human Drugs: Labeling; Registration and Listing, Safety Reporting, Supply Chain Security, and Good Manufacturing Practice	Electronic Submissions
180-Day Exclusivity: Questions and Answers; Revised Draft	Generics	REVISED DRAFT
30-Month Stay of Approval of a 505(b)(2) Application or an ANDA	Generics	NEW
ANDA and NDA Submissions: Data Integrity for BA/BE Studies at Testing Sites	Generics
ANDA Submissions – Amendments to Abbreviated New Drug Applications Under GDUFA; Revised Draft	Generics	NEW, REVISED DRAFT
ANDA Submissions – Amendments and Requests for Final Approval to Tentatively Approved ANDAs	Generics	NEW
ANDA Submissions – Refuse-to-Receive for DMF Facilities Deficiencies	Generics
ANDA Submissions – Refuse-to-Receive Standards: Questions and Answers	Generics
Assessing Adhesion with Transdermal Delivery Systems and Topical Patches for ANDAs; Revised Draft	Generics	REVISED DRAFT
Assessing the Irritation and Sensitization Potential of Transdermal and Topical Delivery Systems for Abbreviated New Drug Applications; Revised Draft	Generics	REVISED DRAFT
Comparative Analyses and Related Comparative Use Human Factors Studies for a Drug-Device Combination Product Submitted in an ANDA; Revised Draft	Generics	REVISED DRAFT
Content and Format of Composition Tables in NDAs and ANDAs And Corresponding Statement of Ingredients in Labeling	Generics	REVISED TITLE
Determining Whether to Submit an ANDA or 505(b)(2) Application	Generics	NEW
Handling and Retention of BA and BE Testing Samples	Generics
“Open for Business” Definition Under 744B of the Federal Food, Drug and Cosmetic Act	Generics
Pediatric Exclusivity General Considerations for ANDAs	Generics
Product Specific Guidance Meetings Between FDA and ANDA Applicants under GDUFA	Generics
Three-Year Exclusivity Determinations for Drug Products	Generics
E2D(R1) Post-Approval Safety Data Management: Definitions and Standards for Expedited Reporting	ICH	NEW
E6(R3) Good Clinical Practice Principles and Annex 1	ICH	NEW
E20 Adaptive Clinical Trials	ICH	NEW
M4Q(R2) Revision of M4Q(R1) CTD on Quality	ICH	NEW
M14 General Principles on Planning and Designing Pharmacoepidemiological Studies that Utilize Real-World Data for Safety Assessment	ICH	NEW
Q3E Impurity: Assessment and Control of Extractables and Leachables for Pharmaceuticals and Biologics	ICH	NEW
Combined Hormonal Contraceptives for Prevention of Pregnancy — Labeling for Health Care Providers and Patients	Drug Labeling
Labeling for Biosimilar and Interchangeable Biosimilar Products	Drug Labeling
Regulatory Considerations for Prescription Drug Use-Related Software	Drug Labeling	NEW
Annual Reportable Labeling Changes for NDAs and ANDAs for Nonprescription Drug Products	OTC Drugs
Formal Dispute Resolution and Consolidated Proceedings: Requestor of OMUFA Products Appeals Above the Division Level	OTC Drugs
OTC Monographs Order Requests (OMORs) – Format and Content of Data Submissions	OTC Drugs
PET Drugs – Current Good Manufacturing Practice (CGMP); Revised Draft	CGMP	REVISED DRAFT
Advanced Manufacturing Technologies Designation Program Designated Technologies in Drug and Biological Products	Drug Quality, CMC	NEW
ANDAs: Stability Testing of Drug Substances and Products Q&A	Drug Quality, CMC	NEW
Potency Assay Considerations for Monoclonal Antibodies and Other Therapeutic Proteins Targeting Viral Pathogens	Drug Quality, CMC
Products With Benzene-Containing Carbomers: Recommendations for Reformulation	Drug Quality, CMC	NEW
Quality Considerations for Topical Ophthalmic Drug Products	Drug Quality, CMC
Stability Considerations for Drug Substances and Drug Products in NDAs, ANDAs, and BLAs and Associated Labeling Statements for Drug Products	Drug Quality, CMC
Stability Recommendations for Additional Manufacturing Facilities in NDAs, ANDAs and BLAs, and Additional Drug Substance Sources in NDAs and ANDAs	Drug Quality, CMC
Container Closure Systems for Drugs, Including Biological Products	Drug Quality, CMC	NEW
Post-approval Manufacturing Changes to Biosimilars and Interchangeable Biosimilars Questions and Answers	Drug Quality, CMC	NEW
Use of Alternative Tools to Assess Manufacturing Facilities Named in Pending Applications	Drug Quality, CMC	NEW
Translation of Nonclinical Toxicology Study Reports: Questions and Answers	Pharmacology/Toxicology	NEW
Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products	RWD/RWE
Considerations Regarding Non-Interventional Studies for Drug and Biological Products	RWD/RWE	NEW
Using Clinical Practice Data in Randomized Controlled Trials (RCT) for Regulatory Decision-Making for Drug and Biological Products	RWD/RWE

To contact the author of this analysis, please email Alec Gaffney ( agaffney@agencyiq.com)
To contact the editor of this analysis, please email Kari Oakes ( koakes@agencyiq.com)

CDER unveils new guidance agenda for 2023, with 42 new policies planned

Regulatory Background

New and noteworthy on the agenda

Key Documents and Dates

Get an insider’s view on regulatory movements.