The U.S. Food and Drug Administration (FDA) has faced an unusual convergence of political, scientific, legal and operational pressures in recent months, as it balances public health demands with intensifying scrutiny from the White House, Congress, industry and patient advocates.

This includes backlash over a White House–backed push around an autism treatment that some experts say lacks sufficient evidence, sweeping staff cuts, a plan to accelerate drug reviews, and most recently, outcries over its rejection of a number of rare-disease treatments.

“The FDA continues to apply the same high standards to all drugs and therapies, requiring clear evidence of clinical benefit for the American public,” an HHS spokesperson told Newsweek in response to the issues. “No FDA in history has produced as much positive regulatory change in such a short span as Commissioner Makary’s FDA. The American people voted for an FDA that works for them, not the industry, and the agency is delivering by refusing to rubber stamp approvals.”

Newsweek details below four major issues that have plagued the FDA in the past year.

1. The Use Of Leucovorin For Autism

In September 2025, at a White House event, FDA Commissioner Marty Makary said the drug leucovorin, a type of vitamin B also known as folinic acid, could have a potential benefit for “hundreds of thousands of kids” with autism. The administration cited small studies suggesting that a number of people with autism might have antibodies that interfere with how folate is transported in the body.

However, health experts and autism advocates raised concerns about Markary’s comments, arguing that the process departed from the agency’s typical approach and that evidence of effectiveness remains limited.

This also coincided with the administration warning pregnant women to only take the painkiller Tylenol when absolutely necessary, which prompted sharp criticism from scientists and health officials, including some outside the U.S.

The FDA has since approved leucovorin – on March 10, 2026 – as the first treatment for a rare genetic disorder, cerebral folate deficiency, but not for autism. Senior officials told reporters on Monday that after reviewing the strongest evidence, research only supported the drug’s use by patients with the rare mutation that impacts folate levels in the brain.

The Trump administration has been vocal about its stance on autism, with President Donald Trump and Health and Human Services (HHS) Secretary Robert F. Kennedy Jr both calling into question the high number of American children with the condition. Kennedy’s ‘Make America Healthy Again’ (MAHA) campaign has put finding the causes of autism at the forefront of its agenda.

But health experts have frequently warned that some administration rhetoric risks reviving long-debunked claims about autism—including assertions made without evidence about links to Tylenol and vaccines.

2. Leadership Upheaval And Workforce Pressures

On March 31, 2025, Dr. Peter Marks, who had led the FDA’s Center for Biologics Evaluation and Research (CBER) for eight years, resigned shortly after Makary’s private swearing-in.

The CBER regulates biological products to ensure their safety and effectiveness, which includes overseeing the approval process for vaccines.

His resignation alarmed lawmakers and industry leaders who then were concerned about a broader overhaul of vaccine policy, with Marks no longer at the driving seat of the CBER.

With Kennedy—a prominent vaccine skeptic—serving as health secretary, the move enhanced growing fears about how the administration would change guidance on vaccines.

Marks wrote in a letter to Acting FDA Commissioner Sara Brenner: “It has become clear that truth and transparency are not desired by the Secretary, but rather he wishes subservient confirmation of his misinformation and lies.”

Marks added in his letter that he was “willing to work” to address the concerns expressed by Kennedy about the safety of vaccinations, but he concluded that wasn’t possible.

A former FDA official familiar with the discussions said Marks was offered the choice of resigning or being fired by Kennedy. The official spoke to The Associated Press on condition of anonymity because they were not authorized to discuss the matter publicly

This also came after a broader restructuring of the agency was announced earlier in the year, with plans to reduce FDA staff by 3,500 positions.

While the HHS officials said this would have no impact on the running of the FDA, industry experts warned that the move would notably impact the agency’s ability to review and approve products.

3. The Commissioner’s National Priority Voucher

The FDA Commissioner’s National Priority Voucher (CNPV) program was designed as a pathway to “dramatically reduce review times for drug and biological product applications and manufacturing or efficacy supplements.” The program would mean certain drugs could be reviewed in as little as one month, instead of the traditional six to 10 months.

For the new program, the FDA said it would issue a limited number of “national priority vouchers” to companies “aligned with U.S. national priorities,” and those vouchers would give selected companies access to extra FDA communications, streamlined staff reviews and the ability to submit much of their product information in advance.

Health policy experts and FDA veterans have warned that aggressively shortening review timelines risks undermining the scientific rigor that underpins drug safety decisions. “The concept of doing a review in one to two months just does not have scientific precedent,” Dr. Aaron Kesselheim, a professor at Harvard Medical School, told AP, adding that the agency “cannot do the same detailed review that it does of a regular application in one to two months, and it doesn’t have the resources to do it.”

4. Drug Shortages And Rejections

The FDA allowed certain drugs from factories under import bans to continue entering the U.S. under exemptions, due to concerns about medication shortages, as reported by ProPublica.

A warning letter from the FDA issued to a factory in India revealed that it did not follow necessary regulations to avoid “microbiological contamination” of their drug products, among a host of other safety breaches, such as dirty water being allowed to leak through the ceiling into sterile areas.

Despite those concerns, ProPublica reported that an FDA branch allowed 20 foreign factories—mostly based in India—to receive special passes to continue sending drugs to the U.S. even after quality control breaches.

These drugs were given to a wide range of patients around the country who may not have been aware, including those with cancer and epilepsy, and records reviewed by ProPublica allegedly tied 70 hospitalizations and nine deaths to drugs from the initially banned factories.

Another issue facing the FDA, stemming from the shortage of medical treatments, is that rare-disease patients and advocates have been calling for more treatments to be made available, while the FDA has been rejecting a number of treatments for those very conditions.

The agency has instead been advising manufacturers to launch expensive and time-consuming studies into the treatments, delaying approval by years for drugs aimed at patients with life‑threatening conditions that often have no existing cure.

This has prompted notable backlash, as Senator Ron Johnson has said he will be launching an investigation into the FDA because of the treatment denials.

The Wisconsin Republican told Bloomberg on Monday he planned to seek copies of the FDA’s written denial letters to drugmakers and formally write to the agency to understand the rationale behind the decisions.

“The number of FDA approvals and rejections under this administration are consistent with historical data over the last decade,” HHS spokesman Andrew Nixon told Newsweek. “In fact, CBER had a record number of approvals in December.”

Together, the disputes have placed the FDA in an unusually fraught position, forced to defend its scientific standards even as it faces mounting political pressure to move faster, approve more treatments, and align more closely with White House priorities. How the agency navigates those competing demands may shape public trust in federal health regulation long after the current controversies subside.

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