This Week: Health officials want to cut red tape for CGTs, FDA’s budget advances, 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, top US health officials said they want to reduce regulatory barriers and quickly approve promising cell and gene therapy (CGT) products, FDA’s FY 2026 budget bill advanced in the House, and the Senate confirmed a controversial HHS nominee.

FDA organized a roundtable this week with top stakeholders from academia and advocacy organizations to talk about the potential barriers to developing CGTs. At the start of the meeting FDA Commissioner Marty Makary talked about how the agency is evolving and wants to listen to and learn from stakeholders on how to reduce barriers and cut waste while challenging established norms.

"We are asking ourselves, can certain materials be submitted in advance, so approvals are quicker," said Makary. "Can we look at a product in a way the patient or parent looks at the product?"

"Can we use post-approval monitoring and the assurance of post-approval monitoring in the actual decision-making process?" he added.

He was joined by Vinay Prasad, director of the Center for Biologics Evaluation and Research (CBER), who emphasized that FDA wants to be transparent about its thinking.

"FDA will consider surrogate endpoints and overall survival," said Prasad. "We will rapidly make therapies available at the first sign of biomedical success or action, but we will also follow up on quality of life to ensure we're accomplishing what we think we're accomplishing."

He also said the agency is interested in leveraging randomized clinical trials (RCT), parachute trials, and real-world data (RWD).

Prasad’s comments echoed those he made at the National Organization for Rare Disorders (NORD) Rare Disease Scientific Symposium earlier in the week. According to Endpoints, he said that the agency would allow surrogate endpoints to get new drugs to patients with rare diseases before they receive full marketing authorization.

Makary and Prasad were later joined by other HHS officials, including HHS Secretary Robert Kennedy Jr., who said FDA and the whole healthcare system were beholden to a “bundle of perverse incentives where people make money by doing bad things.” He said the incentives were part of every level of the healthcare system, including doctors, hospitals, pharmaceutical companies, and insurance companies, who were making money while keeping the American public sick.

“We need to fundamentally change that so that we are focusing on curing disease and making people better, and the way we're going to do that is by unraveling those incentives and focusing on curing disease,” said Kennedy. “We want to make sure we're getting rid of the regulatory impediments that stop you from getting your drugs to market.”

He said the administration would find new ways to accelerate approvals for treatments for rare diseases and make the US a hub of biotechnology innovation.

Kennedy also mentioned President Donald Trump’s deregulatory executive order to remove 10 regulations, rules, or guidances for every new one published. He said that in order to get helpful regulations published, they need to remove other regulations and asked the attendees to send HHS lists of guidances that they would like to see removed.

“If you can provide us with that list, we will look at it, and we will do everything that we can to get rid of the bad ones and put in place the good ones and get you the funding," said Kennedy.

He also said that the administration hasn’t taken away funding from FDA but rather cut administrative costs and redundancies. He said that he would reapply the savings to unleash medical innovation.

Politico reported that House Agriculture, Rural Development, Food and Drug Administration Subcommittee of the House Appropriations Committee has voted to advance an FY 2026 budget bill that would give FDA $6.8 billion, including $3.2 billion in appropriations and the rest from user fees. The proposal would slash $300 million from FDA's FY 2025 enacted funding level and mirrors the level proposed in Trump’s budget request.

Jim O'Neill, former CEO of the Thiel Foundation and a close associate of Trump funder Peter Thiel, was confirmed deputy HHS secretary down party lines this week in the Senate. A controversial libertarian who has espoused the view that FDA should approve drugs solely on safety and let patients decide if they are effective was under consideration for the position of FDA commissioner during the first Trump presidency.

The Government Accountability Office (GAO) published a report this week calling for a coordinated national approach for diagnostic testing for pandemic threats. The agency made four specific recommendations and noted that HHS struggled with national testing efforts early in the COVID-19 pandemic, which ultimately required coordination with public and private stakeholders.

Federal News Network reported that the Office of Personnel Management has proposed a rule that would make it easier for the Trump administration to fire federal employees accused of misconduct. If finalized, the rule would allow the fast-tracking of firings due to an employee's failure to meet suitability and fitness standards.

Drugs & biologics

Sen. Josh Hawley (R-MO) published a letter from Makary on X, stating that the FDA commissioner has committed to reviewing the abortion drug mifepristone. The letter echoes what Makary said to lawmakers during his Senate confirmation hearings.

Over the past few weeks, HHS has gotten pushback for taking steps limiting who the COVID-19 vaccines are intended for and the people who should get the vaccines. To address the concerns, Kennedy posted on X that: "Moderna has agreed to a true placebo-controlled trial of the new vaccine, which is similar to the existing mRNA vaccine but uses a smaller protein... FDA will scrutinize every aspect of the trial."

Prasad sat down with Makary for a podcast earlier this week in which he addressed the controversy surrounding limiting the COVID-19 vaccines. He said that the FDA does not regulate clinical practice and reiterated that decisions to take the vaccine should be between patients and their physicians.

FDA approved Bayer's cancer drug Nubeqa (darolutamide) to treat metastatic castration-sensitive prostate cancer (mCSPC). The drug was previously only approved in combination with docetaxel to treat mCSPC.

Medtech

FDA announced recalls for several products, including a Class I recall for Medtronic's Shiley Adult Flexible Tracheostomy Tube with TaperGuard Cuff Reusable Inner Cannula. The agency said the flange could disconnect from the device cannula, which could prevent the patient from breathing or could cause a blockage of their airway that could lead to serious adverse events, including death.

Baxter also issued a Class I recall for its Novum IQ Large Volume infusion pump due to concerns about underinfusion after the use of the "standby mode" on the device or if it is powered off with the set loaded. The company has provided updated instructions for use, including instructions on monitoring patients and the device to ensure they are operating correctly and notifying healthcare providers about the issue.

This Week: Health officials want to cut red tape for CGTs, FDA’s budget advances, and more

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