FDA Regulatory News and Trends - July 7, 2023

Welcome to FDA Regulatory News and Trends, designed to help you identify significant legal developments and navigate the evolving business, legal, and regulatory world.

First-ever draft guidance on patient-matched guides to orthopedic implants.

• In addition to supporting the safety and efficacy of an implant, patient-matched guides are accessories to medical devices intended to facilitate pre-surgical planning and efficiency in an implantation procedure. While FDA has issued guidance related to patient-matched devices, its latest release is the first-ever guidance on the guides. 
  
  
• The guidance is “intended to promote clarity and transparency” regarding the Agency’s expectations for regulatory submissions and certain considerations when developing design processes for patient-matched guides, specifically those involving orthopedic implants. FDA’s recommendations span a variety of topics, including indications for use, device description, software, biocompatibility, sterility, shelf life and packaging, performance testing, labeling, and modifications.
  
  
• While this draft guidance incorporates recommendations related to design aspects, it is not intended to represent a comprehensive list of considerations for meeting the quality system regulation requirements of 21 CFR 820, and manufacturers and developers must still establish procedures (including process validation of patient-matched guides) to ensure that their devices will perform as intended. The comment period is set to close August 28, 2023.
  
  

Final guidance on quantitative efficacy and risk information for DTC promotional materials.

• On June 27, 2023, FDA finalized its final guidance, “Presenting Quantitative Efficacy and Risk Information in Direct-to-Consumer (DTC) Promotional Labeling and Advertisements,” based on comments and recent research. While the final guidance is largely consistent with the 2018 draft guidance, there are certain changes that may influence how companies present quantitative efficacy and risk data. 
  
  
• The final guidance includes a new section regarding the disclosure of quantitative efficacy or risk information from the control group in a clinical study. Among other changes within the guidance, FDA advises companies to prominently disclose the absolute probability measure and “in direct conjunction” with the relative frequency measure. Finally, FDA cautions that the presentation of quantitative probability information about a particular risk should be done in a manner that does not minimize the severity of the risk.
  
  
• In addition to the new recommendations, FDA deleted its statement that the guidance applies to all DTC promotional materials “regardless of the medium” and replaced it with more nuanced language: The Agency recommends that companies consider the amount of space or time available in determining whether and how to present quantitative efficacy or risk information in their DTC promotional communications.  
  
  

CDER update on the first year of the Accelerating Rare disease Cures (ARC) Program.

• On June 13, 2023, CDER provided an update on the first anniversary of its Accelerating Rare disease Cures (ARC) Program, which aims to bridge the gap between rare disease drug development and the pressing needs of patients. Under the program, CDER launched various outreach and education initiatives to better understand the patients’ perspectives on rare diseases and hurdles in bringing rare disease products to market. ARC additionally supports various scientific and regulatory initiatives, such as Rare Disease Endpoint Advancement (RDEA) Pilot Program, to accelerate rare disease drug development.
  
  
• The update provided ARC’s year-two plan, which includes creating and disseminating educational materials through the LEADER 3D initiative, partnering with CDER’s Patient-Focused Drug Development Program to develop patient materials, sharing what the industry has learned from the use of novel endpoints for rare disease therapies in the RDEA Pilot Program, and participating in patient listening sessions with rare disease advocates.
  
  
• On the scientific front, ARC plans to work on bolstering the use, development, and knowledge of frameworks such as complex innovative designs, including Bayesian statistical approaches, and supporting platforms that facilitate rigorous use of data from natural history studies and other real-world data. 
  
  

New pilot program for oncology laboratory-developed tests.

• On June 20, 2023, FDA launched a pilot program intended to reduce risks associated with using laboratory-developed tests (LDT) in targeted oncology therapies. The details of the voluntary pilot program are described in the draft guidance, “Oncology Drug Products Used with Certain In Vitro Diagnostic Tests: Pilot Program.” Specifically, FDA intends to establish minimum performance standards for in vitro diagnostic tests used with oncology drug products. 
  
  
• This is the most recent Agency action to move away from the “one-drug-one-diagnostic” model, which has been the mainstay of targeted cancer therapies in the past decade but has failed to serve patients well, according to FDA. The pilot program also signals the Agency’s renewed effort to regulate LDTs.
  
  
• The guidance has been implemented without prior comment. Interested parties should submit comments in accordance with the Agency’s good guidance practices. Stay tuned for detailed analysis in our forthcoming client alert. 
  
  

Final guidance on content of premarket submissions for device software functions.

• To keep pace with evolving regulatory considerations for device software functions, “Content of Premarket Submissions for Device Software Functions” replaces FDA’s “Guidance for the Content of Premarket Submissions for Software Contained in Medical Devices,” issued on May 11, 2005.
  
  
• The guidance sets out FDA’s latest thinking related to recommended documentation for inclusion in premarket submissions, including a two-tier documentation system: Basic or Enhanced. 
  
  
• On July 20, 2023, FDA will host a webinar to discuss the newly published guidance, which will be implemented on August 13, 2023.
  
  

Draft guidance on formal dispute resolution for OTC drugs.

• On June 23, 2023, FDA announced a new draft guidance entitled, “Formal Dispute Resolution and Administrative Hearings of Final Administrative Orders Under Section 505G of the Federal Food, Drug, and Cosmetic Act.”
  
  
• The draft guidance provides recommendations for industry and review staff on the formal dispute resolution (FDR) and administrative hearings procedures for resolving scientific and/or medical disputes between the Center for Drug Evaluation and Research and sponsors of drugs that will be subject to a final administrative order under section 505G of the Food, Drug, and Cosmetic Act. 
  
  
• The draft guidance covers the FDR process for over-the-counter (OTC) drugs without approved new drug applications, governed by the provisions of section 505G, or OTC monograph drugs.  
  
  
• The “Over-the-Counter Monograph User Fee Program Performance Goals and Procedures” document (or the “OMUFA commitment letter”) specified that FDA would (1) revise previous guidance to include circumstances and procedures under which FDR may be used with respect to final orders under section 505G and (2) issue guidance describing best practices for consolidated proceedings for appeals.  For administrative efficiency, FDA aimed to accomplish both commitments in this draft guidance. 
• The comment period for this draft guidance closes on August 22, 2023.  
  
  

Draft guidance on clinical investigations for psychedelic drugs.

• On June 26, 2023, FDA announced a new draft guidance entitled, “Psychedelic Drugs: Considerations for Clinical Investigations.”
  
  
• The draft guidance is intended to present foundational aspects for sponsors to consider when developing psychedelic drugs for treatment of medical conditions (eg, psychiatric disorders, substance use disorders).  The draft guidance applies to clinical trials conducted under an investigational new drug application (IND), including clinical trials (eg, research or academic studies) that are not intended to support marketing applications. 
  
  
• FDA recognizes that designing clinical studies to evaluate the safety and effectiveness of psychedelic drugs poses unique challenges, and notes that the draft guidance provides foundational constructs for sponsors to take into account.
  
  
• The draft guidance includes general considerations for drug development programs evaluating the therapeutic potential of psychedelic drugs in various disciplines, including chemistry, manufacturing, and controls (CMC); nonclinical; clinical pharmacology; abuse potential assessment; and clinical. 
  
  
• The comment period for this draft guidance closes on August 25, 2023.