This Week at FDA: New guidances address cancer PROs, neurodevelopmental safety studies, and more

Welcome to another installment of This Week at FDA, your weekly source for updates – big and small – on FDA, drug, and medical device regulation, and what we’re reading from around the web. This week, FDA issued several draft and final guidance documents, including guidance on patient-reported outcomes (PROs) in cancer clinical trials, long-term clinical neurodevelopmental safety studies for neonatal products, and requests for reconsideration at the division level for generic drug sponsors.

In the aftermath of Hurricane Helene last month, the Department of Health and Human Services (HHS) on Friday announced that Baxter has been able to distribute more than 450 truckloads of products from its storm-damaged North Carolina facility and has begun importing products from its foreign facilities, following regulatory assessments by FDA, to improve the supply of IV and peritoneal fluids, with the first shipments of products expected to arrive on 19 October. Despite the disruption caused by the storm, the company said it expects to reach customer allocations of 90-100% of historical levels by the end of 2024.

Earlier this week, FDA’s Accelerating Rare Disease Cures (ARC) program released its second annual report, outlining its activities in 2024, including public outreach, education, and scientific and regulatory initiatives.

Drugs & biologics

Among the several guidances issued this week, FDA finalized its guidance on core PROs in cancer clinical trials. FDA said it revised the guidance to recommend companies consult with it when selecting adverse events for reporting and to include hematological malignancies in the guidance.

FDA also finalized its recent guidance on long-term clinical neurodevelopmental safety studies for neonatal products. The final guidance features several changes from the draft version, including a discussion of minimum duration of follow-up in the background section, the impact of route of administration on the need for long-term neurodevelopmental safety evaluations, and to note that protocols should include a plan for clinical referral and support services if developmental problems are identified.

Additionally, FDA finalized its guidance on requests for reconsideration at the division level under the Generic Drug User Fee Amendments (GDUFA). The agency said it received no comments on the draft version and has made only editorial changes to the final version.

The agency also issued another GDUFA-related final guidance providing information and recommendations on the review of drug master files in advance of certain abbreviated new drug application (ANDA) submissions. According to FDA, the only change from the 2022 draft version is the addition of a footnote to “clarify the potential impact of unsolicited amendments submitted after the prior assessment is granted.”

FDA also issued two draft guidances this week, including one on developing drugs for preventing postoperative nausea and vomiting and another offering recommendations for the development of blood collection, processing, and storage systems for the manufacture of blood components using the buffy coat method.

Reuters reports that Gilead will voluntarily withdraw its accelerated approval for its drug Trodelvy (sacituzumab govitecan-hziy) to treat metastatic urothelial cancer after a confirmatory trial failed to show improved survival and observed more deaths related to adverse events with the drug than with other therapies in the control arm.

Fierce Pharma reports that FDA has approved Astellas’ Vyloy (zolbetuximab-clzb) as first-line therapy for adults with locally advanced or unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma that are CLDN18.2 positive.

At a public meeting organized by the Reagan-Udall Foundation earlier this week, top FDA officials, including FDA Principal Deputy Commissioner Namandjé Bumpus, Center for Drug Evaluation and Research (CDER) Director Patrizia Cavazzoni, and Center for Biologics Evaluation and Research (CBER) Director Peter Marks, discussed the agency’s new Rare Disease Innovation Hub. In an opinion piece published in STAT News, former FDA Associate Commissioner and President of the Center for Medicine in the Public Interest Peter Pitts wrote that the hub’s success will hinge on internal and external stakeholders ability to communicate and stay on the same page.

Medical devices

FDA has enrolled 54 devices to its Total Product Life Cycle Advisory Program (TAP) pilot, just shy of the 60-participant cap for the program in FY 2024. In FY 2025, participation is capped at 125 devices.

In response to a request from stakeholders, FDA said it will extend the comment period on its recent three draft guidances related to its Accreditation Scheme for Conformity Assessment (ASCA) program by 30 days. Stakeholders will now have until 23 December 2024 to submit their comments before the deadline.

FDA’s Center for Devices and Radiological Health (CDRH) this week announced it will conduct a pilot to provide medical device clinical trial participation snapshots to highlight information about medical devices and the diversity of trial participants for consumers. So far, the agency has posted snapshots for 5 devices authorized in 2024.

Earlier this week, FDA approved Novocure GmbH’s Optune Lua, which produces alternating electrical fields within a patient’s body to treat metastatic non-small cell lung cancer alongside concurrent pharmaceutical therapy in adults who have progressed on or after a platinum-based chemotherapy.

This Week at FDA: New guidances address cancer PROs, neurodevelopmental safety studies, and more

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