This Week at FDA: Lawmakers ask about CURES 2.0, Califf wants more pragmatic clinical research

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 took the recommendation of its vaccine advisory committee and recommended an update to the fall 2024 COVID-19 vaccine, top agency officials want emphasis on more pragmatic clinical research, and US lawmakers want to hear about legislative solutions to bring new treatments.

CNN reported that FDA’s Vaccines and Related Biologic Products Advisory Committee (VRBPAC) unanimously recommended the agency update the 2024-2025 COVID-19 with a monovalent JN.1-lineage coronavirus vaccine composition. The decision was based on the fact the Centers for Disease Control and Prevention (CDC) reports that JN.1 and its descendants KP.2 and KP.3 are the most common variants of the coronavirus in the US currently.

Writing in the Journal of the American Medical Association (JAMA), FDA Commissioner Robert Califf and top agency policy advisors recommended an increased focus on pragmatic clinical research. They argued that such research should be leveraged to understand medical products in the context of real-world clinical practice.

Members of the House Energy and Commerce Committee (E&C), Reps. Diana DeGette, D-CO, and Larry Bucshon, R-IN, wrote letters to healthcare stakeholders about what Congress should consider including in a new 21st Century Cures Act to promote the next generation of treatments.

Emer Cooke, executive director of the European Medicines Agency (EMA), participated in a Q&A interview with FDA about her agency's duties and lessons learned from the COVID pandemic. She also emphasized the partnerships and work that EMA is doing with FDA and other regulators.

FDA has renewed two advisory committees – the Arthritis Advisory Committee and the Obstetrics, Reproductive and Urologic Drugs Advisory Committee. The agency said the arthritis committee will continue operating until 5 April 2026 and the urologic drugs committee will continue its work until 23 March 2026.

Drugs & Biologics

CBS News reported that FDA's Psychopharmacologic Drugs Advisory Committee (PDAC) voted 9-2 against recommending the psychedelic drug MDMA, also known as ecstasy or molly, for treating post-traumatic stress disorder (PTSD). Several panelist said they were concerned about gaps in the data from the studies that were submitted for review and some also were concerned about the way the studies were designed.

Pink Sheet reported that CDER Director Patrizia Cavazzoni said that a public-private partnership and additional resources may be needed to handle the growing number of premarket drug applications that include artificial intelligence and machine learning (AI/ML) components. The increased resources may be part of the agency's request when they go to the table to negotiate the next Prescription Drug User Fee Amendment (PDUFA) deal with the pharmaceutical industry.

Compound drugs that contain sulfite preservatives may cause life-threatening allergic reactions in some people, according to FDA. The agency issued an alert that it has received reports of patients suffering allergic reactions from such drugs and has asked compounders to note the presence of sulfites in the drug labeling and include a warning statement about the potential for allergic reactions.

Geron's cancer oligonucleotide telomerase inhibitor Rytelo (imetelstat) was approved by FDA to treat certain adults with low- to intermediate-1 risk myelodysplastic syndromes (MDS) who have not responded to or are ineligible for treatment with erythropoiesis-stimulating agents (ESAs). The drug was reviewed using the agency's voluntary Assessment Aid submission program and was granted orphan drug designation.

The Oncologic Drugs Advisory Committee is scheduled to meet in person and virtually on 25 July to discuss AstraZeneca's supplemental biologics license application (sBLA) for IMFINZI (durvalumab). The drug is being evaluated as a treatment for adults with resectable non-small cell lung cancer (NSCLC).

FDA wants to refuse approval of Vanda Pharmaceuticals' supplemental new drug application (sNDA) for Hetlioz (tasimelteon) as a treatment of insomnia characterized by difficulties with sleep initiation. The drug is already approved for non-24-hour sleep-wake disorder and Smith-Magenis syndrome in patients 16 years and older, but the agency said the data provided by the company for the new indication hasn't proven it is safe and effective.

The Center for Biologics Evaluation and Research (CBER) published an updated standard operating policy and procedures (SOPP) that details how its staff will post information on its website about when it approves or denies Premarket Approval Applications (PMA) and Humanitarian Device Exemption (HDE) applications. It also outlines what documents are needed for staff to comply with the federal mandates and how to post them on its website.

Medtech

Since FDA issued a warning in March not to use Chinese-manufactured plastic syringes unless absolutely necessary, the agency has stated it has sent a warning letter to Medline Industries for failing to meet quality system requirements and announced the company has initiated a recall for such products. This week it announced additional recalls initiated by Medline to stop using bulk unauthorized plastic syringes made in China.

The Center for Devices and Radiological Health (CDRH) published data on its 510(k) third-party performance metrics and accreditation status from the first two quarters of 2024. The program was developed under the 1997 FDA Modernization Act (FDAMA), and the Medical Device User Fee Amendments (MDUFA V) require the agency to publish performance data on accredited third parties.

Medtronic has initiated a class I recall of some of its StealthStation Systems with StealthStation Cranial software used to map the brain during surgery. According to FDA, the company has received complaints about software errors causing numbers and letters to be missing from the system display, which could lead to surgeons making incorrect measurements about where the biopsy tip needs to be.

This Week at FDA: Lawmakers ask about CURES 2.0, Califf wants more pragmatic clinical research

Related topics

Recommended content

Welcome to the new RAPS Digital Experience