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21 November 20238 minute read

FDA Regulatory News and Trends - November 21, 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.

FDA issues guidance to extend policy for certain device modifications without regulatory submission.

  • On November 2, 2023, the US Food and Drug Administration (FDA) issued a guidance entitled, “Enforcement Policy for Certain Supplements for Approved Premarket Approval (PMA) or Humanitarian Device Exemption (HDE) Submissions.”

  • Generally, following approval of a premarket approval application (PMA) or humanitarian device exemption (HDE), applicants must submit a supplement for FDA review and approval before making any changes affecting device safety or effectiveness. In light of the COVID-19 public health emergency (PHE), however, FDA released a guidance in 2020 that announced a policy for adapting manufacturing or design due to supply chain disruptions and accommodating manufacturing personnel safety through social distancing. The November 2023 guidance supersedes that guidance, which was revised in 2022 and earlier in 2023.

  • The latest guidance reflects the Agency’s intent to foster continued availability of medical devices outside the context of the COVID-19 public health emergency (PHE) where potential shortages or limitations persist.

  • According to the guidance, FDA intends to continue monitoring the situation and may make further revisions to this policy, withdraw the guidance, or pursue other regulatory actions, as appropriate.

CDER releases guidance on technical specifications for Clinical Outcome Assessments.

  • On November 6, 2023, the Center for Drug Evaluation and Research (CDER) published a guidance document titled, “Submitting Clinical Trial Datasets and Documentation for Clinical Outcome Assessments Using Item Response Theory.” This guidance is a continuation of the center’s Patient-Focused Drug Development Methodological Guidance Series.

  • The guidance elaborates on technical specifications for Clinical Outcome Assessments (COAs) that specifically use Item Response Theory (IRT). IRT is a mathematical model describing the functional relationship between item performance, item characteristics, and the patient’s status. A goal of these specifications is to standardize content and structure of COAs and facilitate FDA review.

  • Pharmaceutical companies may consider this guidance when designing their study to ensure they collect the appropriate types and levels of evidence for clinical outcome assessments. CDER encourages companies to consult them early during the Investigational New Drug Application (IND) phase for guidance.

CDER, CBER, and CVM release guidance on a remote alternative to on-site inspections.

  • On October 25, 2023, CDER, the Center for Biologics Evaluation and Research (CBER), and the Center for Veterinary Medicine (CVM) released a joint guidance document titled, “Remote Interactive Evaluations of Drug Manufacturing and Bioresearch Monitoring Facilities.” 

  • The guidance solidifies alternative methods for inspection that the centers previously used to adapt to travel restrictions during the COVID-19 pandemic. It explains that Remote Interactive Evaluations (RIEs) involve tools such as teleconferencing, livestream video, screen sharing, and sharing of electronic data and documents. RIEs can replace on-site inspection if FDA is able to complete a sufficient review, but it is possible that an on-site inspection would still be needed.

  • If FDA is considering conducting an RIE, the Agency will contact the facility and confirm willingness and ability (ie, IT capabilities) to participate in the voluntary program. The guidance states that these evaluations will be considered for five types of inspection: (1) preapproval and prelicense inspections, (2) postapproval inspections, (3) surveillance inspections, (4) follow-up and compliance inspections, and (5) Bioresearch Monitoring (BIMO) inspections.

  • Companies subject to inspection are encouraged to review the guidance document for awareness of what FDA would expect in an REI, as well as assess internal IT capabilities to determine whether they can agree to participate in one, should FDA request it.

FDA releases guidance for clinical electronic thermometers.

  • This immediately effective guidance makes permanent the enforcement policy for use of clinical electronic thermometers that FDA set during the COVID-19 PHE. 

  • The guidance provides clarification on FDA enforcement policies and expectations for premarket review of clinical electronic thermometers after the PHE ends.

  • Along with this guidance, FDA has proposed clinical electronic thermometers without telethermography or continuous temperature measurement functions to be exempt from premarket notification requirements. At this time, FDA is not proposing these devices to be exempt from other statutory and regulatory requirements. FDA intends to withdraw this guidance after any final exemption notice has been published in the Federal Register.

Final guidance on patient-reported outcome data in cancer clinical trials.

  • On November 3, 2023, FDA issued a final guidance document, “Submitting Patient-Reported Outcome Data in Cancer Clinical Trials.” This final guidance supplements FDA’s draft guidance, “Core Patient-Reported Outcomes in Cancer Clinical Trials,” dated June 2021, and the Agency’s Patient-Focused Drug Development Guidance Series.

  • This 48-page guidance provides technical specifications for submitting patient-reported outcome (PRO) data collected in cancer clinical trials. This data is used to support marketing applications for medical products in oncology. FDA’s specifications provide general guidelines for (1) standardized dataset content and structure and (2) recommended tables and figures to facilitate FDA review of the marketing application.

  • Sponsors are encouraged to review the examples included in the Study Data Tabulation Models (SDTM) and Analysis Data Model (ADaM) questionnaires datasets when preparing new drug applications or biological license applications. 

Updated final guidance on process to request review of denials of certain export certificates for devices.

  • FDA has updated its final guidance on requesting review of denials of certain export certificates for devices, outlining its latest processes for issuing denials and responding to requests for review.

  • FDA export certificates allow medical device manufacturers to certify to entities outside the US that a device complies with FDA regulations.

  • The guidance replaces a 2020 version, incorporating legislative changes that replace the Certificate for Devices Not Exported from the US (CDNEs) with new Certificates to Foreign Government for Devices Not Exported from the US (CFG-NE).

FDA announces completion of the second collaborative assessment under the ICMRA pilot program.

  • On November 2, 2023, FDA announced the completion of the second collaborative assessment for a drug product with the European Medicines Agency (EMA). The assessment, which centered around a powder for oral suspension, took place in the context of the International Coalition of Medicines Regulatory Authorities (ICMRA) pilot program.

  • The ICMRA is a global medicines regulatory group comprised of voluntary, executive-level authorities who work to harmonize regulatory assessments and inspection procedures worldwide. In response to the stress that COVID-19 imposed on the global drug supply chain, the ICMRA launched two pilot programs to enhance international cooperation in the pharmaceutical sector. The programs allow regulatory authorities from different countries to collaborate on post-approval changes and hybrid inspections.

  • On July 28, 2023, FDA and EMA announced the completion of the first collaborative assessment of a proposed post-approval change. This involved adding new manufacturing and quality control sites for a critical oncology biologic, with the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) serving as an observer.

CDER publishes environmental impact review.

  • CDER published a webpage addressing its evaluation of the environmental impacts of pharmaceuticals and biologics under the National Environmental Policy Act of 1969.

  • FDA regulations require submissions of environmental assessments (EAs) for certain Agency actions, including certain new drug applications and applications for marketing approval of biologic products and their supplements, unless an action qualifies for a categorical exclusion. CDER’s webpage outlines these exclusions and how they may be claimed. Failure to submit either an EA or an exclusion is sufficient grounds for refusal to file or approve an application.

  • Organizations filing applications or petitions requesting Agency action can visit the site to learn about these requirements, access resources including industry guidance, and find contact information for the CDER team.