FDA Regulatory News and Trends - February 14, 2024

FDA finalizes gene therapy product guidance.

• On January 29, 2024, FDA issued final guidance, entitled, “Human Gene Therapy Products Incorporating Human Genome Editing.”


• This guidance provides recommendations to sponsors developing human gene therapy products that incorporate genome editing (GE) of human cells. The Agency’s recommendations address information that should be included in an Investigational New Drug (IND) application, such as details related to product design, product manufacturing and testing, nonclinical safety assessment, and clinical trial design.


• The structure and content of the final guidance remains largely the same as in the draft version. However, the finalized document reflects various changes in response to industry comments, including clearer, more tailored terminology; information on efficacy endpoints and use of accelerated approval; broader language around techniques that can be used; component control strategy; and how to characterize potency.


• FDA’s Center for Biologics Evaluation and Research (CBER) will hold a virtual webinar for industry on February 29, 2024 to discuss the new guidance.
  
  

FDA releases final guidance on CAR T cell therapy products.

• On January 31, 2024, FDA issued final guidance, entitled, “Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products,” which is intended to assist sponsors developing ex vivo-manufactured CAR T cell products.


• In the guidance, FDA provides CAR T cell-specific recommendations regarding chemistry, manufacturing, and control (CMC); nonclinical considerations; pharmacology and toxicology; design of clinical studies for oncology indications; and analytical comparability studies.


• While the guidance specifically focuses on CAR T cell products, some of the information and recommendations provided may also apply to other genetically modified lymphocyte products, such as CAR Natural Killer cells or T cell receptor-modified T cells.


• To discuss considerations specific to these related products or non-oncology indications, FDA recommends sponsors communicate with CBER’s Office of Therapeutic Products (OTP) before submitting an IND.
  
  

FDA revises draft guidance on remote regulatory assessments.

• On February 2, 2024, FDA issued an update to its July 2022 draft guidance, titled, “Conducting Remote Regulatory Assessments Questions and Answers.” This update addresses public comments and provides new information on authority for mandatory records requests.


• The guidance defines remote regulatory assessments (RRAs) as "an examination of an FDA-regulated establishment and/or its records, conducted entirely remotely, to evaluate compliance with applicable FDA requirements." Recent updates to section 704(a)(4) of the Food Drug and Cosmetic Act hold that this mandatory records request authority applies to drug and device establishments and to sites, entities, or facilities subject to BIMO inspections.


• FDA will likely increase use of this authority to make onsite inspections more efficient by allowing a pre-review of records, or to completely replace onsite inspections, in some cases.


• Those in industry subject to FDA inspection are encouraged to review the guidance and consider what procedural or IT system changes may be needed to support an RRA.

Two Abbreviated New Drug Application updates.

• In January 2024, FDA finalized a guidance, entitled, “Revising ANDA Labeling Following Revision of the RLD Labeling Guidance for Industry,” which provides information on updating generic product labeling, current practices for monitoring reference listed drug labeling changes, and submitting new Abbreviated New Drug Application (ANDA) product labeling.


• This guidance, which finalizes a 2022 draft version, is the first finalized update to the policy on revising generic labeling since 2000.


• FDA also published a revised final guidance for ANDA applicants that seek to make an amendment or request for final approval on a tentatively approved application. The updates reflect GDUFA III commitments, with the majority of changes relating to patent certifications and exclusivities.


• Both of these guidance updates are part of FDA’s Drug Competition Action Plan (DCAP), an initiative through which the Agency aims to remove barriers to generic drug development and market entry, in an effort to spur competition and increase access to medicines for consumers.
  
  

Draft guidance on collection of race and ethnicity data in clinical trials and studies.

• On January 29, 2024, FDA published a draft guidance, entitled, “Collection of Race and Ethnicity Data in Clinical Trials and Clinical Studies for FDA-Regulated Medical Products.” This draft guidance updates the final guidance issued on October 26, 2016 by including both observational studies and interventional clinical trials.


• The draft guidance proposes to use standard terminologies for race and ethnicity to ensure data are collected and reported consistently when submitted to FDA for regulatory determinations. FDA emphasizes the importance of collecting data on race and ethnicity to identify population-specific signals, especially when different responses to medical products are captured in different populations.


• Interested parties should submit their comments on or before April 26, 2024.
  
  

CVM draft guidance on good manufacturing practice requirements for APIs in veterinary medicine.

• On January 24, 2024, FDA’s Center for Veterinary Medicine (CVM) published a new draft guidance, entitled, “Good Manufacturing Practice for Active Pharmaceutical Ingredients Used in Veterinary Medicinal Products.” This draft guidance was developed by the Veterinary International Conference on Harmonization (VICH) Expert Working Group, and the final draft will be recommended for adoption to the regulatory bodies of the European Union, Japan, and the US.


• The guidance provides a comprehensive scheme regarding good manufacturing practice (GMP) for the manufacturing of active pharmaceutical ingredients (APIs) and sets expectations for nearly all steps, such as facility design and equipment validation, among others. The guidance does not outline expectations for the sterilization and aseptic processing of sterile APIs.


• Interested parties should submit their comments on or before March 25, 2024.
  
  

New submission to FDA’s ISTAND Pilot Program.

• On January 23, 2024, FDA announced a new submission into the Innovative Science and Technology Approaches for New Drugs (ISTAND) Pilot Program.


• The submission, which has since been accepted, is for an automated depression and anxiety severity measurement tool that utilizes multiple behavioral and physiological indices of depression in a machine learning (ML) model to derive clinician-reported outcomes for depression and anxiety based on the Hamilton Depression Rating Scale (HAM-D) and Hamilton Anxiety Rating Scale (HAM-A) scores.


• The submission is the first artificial intelligence-based and digital health technology-based project, and the first project in neuroscience, to be accepted into ISTAND.


• The requestor will work with FDA on the qualification plan and, if qualified, the Full Qualification Package (FQP).
  
  

New video on tech-enabled traceability in the food industry.

• On January 22, 2024, FDA released the first video in its educational series on how food companies’ use of tech-enabled traceability can enhance food safety and protect consumers from foodborne illness.


• The episode, which addresses supply chain technology, features a panel from four companies discussing ways to adopt tracing technologies, harmonize tracing activities, and incentivize the development and implementation of scalable and cost-effective traceability systems.


• Building on key themes from the 2021 FDA New Era of Smarter Food Safety Low- or No-Cost Tech-Enabled Traceability Challenge, the video serves as a valuable resource for food producers, technology providers, and other stakeholders interested in advancing food traceability concepts and implementation.