This Week at FDA: Makary teases plan for single-trial drug approvals, Høeg named acting CDER chief, 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, the FDA Commissioner Marty Makary announced the agency will shift its evidence standards for drugs to require a single pivotal trial – rather than two studies – for drugs by default.

FDA has traditionally required two well-controlled clinical trials to meet the “substantial evidence” requirement for approval for most drugs – though the agency often requires only a single pivotal trial, along with other confirmatory evidence, for rare or serious diseases. However, in an interview with STAT News, Makary said the agency will soon default to requiring a single clinical trial for most products and will begin updating its policies and guidance to reflect the change. Makary said the updates could be done within three to six months.

“You can achieve the same statistical power with one trial as you would with two trials when it’s designed and controlled appropriately,” Makary told STAT. The change aims to expedite the review and approval process. Two anonymous sources from FDA’s drug center informed STAT that Makary’s push for the one trial standard contributed to the decision of top drug regulator Richard Pazdur to retire after being named director of FDA’s Center for Drug Evaluation and Research (CDER) less than a month prior.

To fill the post vacated by Pazdur, FDA on Wednesday announced that Tracy Beth Høeg has been named acting director of CDER. Høeg, a physician and epidemiologist, was a visiting scholar at the Massachusetts Institute of Technology Sloan School of Management and practiced physical and interventional spine and sports medicine before joining the FDA as senior advisor for clinical sciences in the Office of the Commissioner and the Center for Biologics Evaluation and Research (CBER).

An industry representative has raised concerns about the ongoing leadership turmoil at FDA. In a statement, John Crowley, president and CEO of the Biotechnology Innovation Organization (BIO) expressed concerns about Pazdur’s departure, noting that the “departure raises serious concerns about the repeated turnover in key leadership occurring at the FDA. We need organizational strength and stability at the Agency. This constant turmoil is undermining America's leadership in biotechnology, creating unprecedented regulatory instability and unpredictability, and risks ceding this critical sector to China.”

Twelve former FDA commissioners and high-ranking former officials published a scathing article in the New England Journal of Medicine this week, criticizing the agency’s recent tightening of vaccine policies advocated by Center for Biologics Evaluation and Research (CBER) director Vinay Prasad. The officials said they are “deeply concerned” by vaccine proposals that would “undermine a regulatory model designed to ensure that vaccines are safe, effective, and available when the public needs them most.”

The officials take issue with the shift away from the agency’s long-standing reliance on immunobridging studies for well-understood vaccines with extensive safety data. FDA leadership has cited the deaths of 10 children as the motivation for these sweeping changes, claiming that these fatalities were caused by COVID-19 vaccines. However, these assertions regarding the deaths have not been substantiated.

Prasad addressed the criticism of his leadership during an investor conference on Thursday, as reported by STAT. He attributed this criticism to “misleading media narratives.” During the meeting, Prasad defended his policy of requiring manufacturers to conduct longer and larger studies before updating vaccines.

FDA has removed the Theresa Michele from her position as director of the Office of Nonprescription Drugs on Wednesday, agency sources told STAT. Michele has been with FDA since at least 2015. During an internal meeting on Wednesday, she announced she would be transitioning to a leadership role in FDA’s medical devices center. FDA announced that Karen Murry will replace her as ONPD director.

FDA on Monday announced the launch of agentic AI capabilities for all agency employees. FDA announced that “Agentic AI refers to advanced artificial intelligence systems designed to achieve specific goals by planning, reasoning, and executing multi-step actions. These systems incorporate built-in guidelines — including human oversight —to ensure reliable outcomes. The tool is entirely optional for FDA staff and is used voluntarily.” Agentic AI will join an LLM-based tool called Elsa, voluntarily used by more than 70% of staff, according to internal agency data.

On Friday, CDC’s Advisory Committee on Immunization Practices (ACIP) voted 8 to 3 to end the decades-long recommendation that all newborns be immunized at birth against hepatitis B, according to the New York Times. This decision is considered a victory for Health Secretary Robert F. Kennedy Jr., who has been pushing to revise the childhood vaccine schedule.

Drugs & Biologics

FDA launched a new website dedicated to providing Information related to providing information related to its preparation for the seventh reauthorization of the Prescription Drug User Fe Act (PDUFA). The current legislative authority for PDUFA VII expires in September 2027. At that time, new legislation will be required for FDA to continue collecting prescription drug user fees in future fiscal years. The webpage includes minutes posted from past meetings, where FDA presented a proposal to establish fee incentives for domestic drug development.

FDA has published final guidance to assist applicants in including information related to heart rate-corrected QT (QTc) interval prolongation in the labeling of non-antiarrhythmic human prescription drugs and biological products.

The agency sent an untitled letter to Daiichi Sankyo in Basking Ridge, NJ regarding claims that a video promoting its Turalio (pexidartinib) capsules is false or misleading. The video implies that treatment with Turalio will enable all patients to return to their pre-diagnosis quality of life after being diagnosed with their tenosynovial giant cell tumor (TGCT), a claim that has not been substantiated.

FDA on Tuesday posted a warning letter to Rhyz Analytical Labs in Provo, UT, due to its insufficient handling of multiple test results that failed microbiological specifications. Additionally, the letter highlighted the quality unit’s (QU) failure to provide adequate oversight over contract testing operations.

FDA also posted a warning letter to Turbare Manufacturing, of Conway, AK, due to “serious deficiencies” in their sterile drug production practices that posed a risk to patients. The agency also noted that on 18 February 2025, the company initiated a voluntary recall of Bevacizumab 1.25mg/0.05mL in 0.25mL syringe.

FDA has approved Bristol Meyers Squibb’s Breyanzi (lisocabtagene maraleucel), the first chimeric antigen receptor (CAR T-cell) therapy for marginal zone lymphoma in the US for adult patients. This approval is for patients who have failed treatment with or relapsed after two or more prior lines of therapy. Marginal zone lymphoma is a rare slow-growing cancer of the lymphatic system.

FDA on Tuesday announced the approval of Eli Lully’s Jaypirca (pirtobrutinib) for adults with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) who have previously been treated with a covalent BTK inhibitor. In 2023, FDA granted accelerated approval of this drug.

A draft guidance titled "New Clinical Investigation Exclusivity (3-Year Exclusivity) for Drug Products: Questions and Answers" is currently pending review by the Office of Management and Budget (OMB).

The FDA has published the third-year report of its Accelerating Rare Disease Cures (ARC) program. This program aims to speed up the availability of treatments for patients with rare diseases and unmet medical needs.

Two Republican lawmakers reportedly were invited to attend a meeting at the FDA regarding the potential expanded approval of a drug supported by billionaire Patrick Soon-Shiong. However, the plan was ultimately abandoned due to internal opposition within the agency, according to Bloomberg. Lawmakers are generally not allowed to attend drug approval meetings to avoid any appearance of political interference.

Medtech

The FDA's Center for Devices and Radiological Health (CDRH) has issued a final guidance document regarding the eCopy program for medical device submissions. This updated guidance provides clarifications on how the eCopy Program intersects with the eSTAR Program and outlines the availability of the CDRH Portal. Additionally, it includes minor updates to align with current technical standards.

FDA’s CDRH has announced an early alert regarding issues with Abbott’s glucose monitor sensors. Abbott’s Diabetes Care has notified distributors, health care providers, and affected customers, advising them to remove certain glucose monitor sensors from use or sale. These include the FreeStyle Libre 3 Sensor, the FreeStyle Libre 3 Plus Sensor. As of 14 November, Abbott has reported 736 serious injuries, and seven deaths associated with this issue.

FDA is increasing its scrutiny of wearable devices, indicating that features such as blood-pressure monitoring devices may fall under medical-device rules rather than “wellness” exemptions, according to Reuters. This stricter regulation could raise costs for smaller firms like WHOOP, as they may be required to conduct clinical studies, cybersecurity checks and upgrade quality systems.

This Week at FDA: Makary teases plan for single-trial drug approvals, Høeg named acting CDER chief, and more

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