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

Life Sciences | By ALEXANDER GAFFNEY, MS, RAC

Feb. 02, 2023

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 ( [email protected])
To contact the editor of this analysis, please email Kari Oakes ( [email protected])

Key Documents and Dates

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