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By Jasmine Laws
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Senator Ron Johnson has launched an investigation into the Food and Drug Administration (FDA) over the agency's recent denials of treatments for rare diseases, the Wisconsin Republican said in an interview with Bloomberg News.
The FDA has rejected several drugs intended to treat rare conditions in recent months. The agency has instead advised 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, according to The New York Times.
On Monday, Johnson told Bloomberg he plans 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.
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"The stories are so outrageous," Johnson told the outlet. "It just appears that they’re looking for excuses to say no."
A Department of Health and Human Services (HHS) spokesperson said that the number of FDA approvals and rejections under the Donald Trump administration have been "consistent with historical data over the last decade."
Newsweek has contacted the FDA, and Johnson's office outside of regular working hours via email for comment.
Why It Matters
The FDA has come under increasing pressure in recent months over its denials of certain rare disease treatments. According to The New York Times, the agency has issued roughly 20 denials over the past eight months. .
Pharmaceutical firms and rare disease advocates have said that the FDA's rejections show signs of inconsistencies around regulatory requirements, which is slowing down the process of getting treatments across the line for suffering Americans. The FDA has said that it is following the direction of scientists and data, and that approvals in general have not gone down.
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What To Know
Johnson said he's also considering asking FDA officials to testify before the Senate’s Permanent Subcommittee on Investigations, which he chairs.
Last week, the FDA asked the biotechnology company UniQure to conduct an additional clinical trial before its new gene therapy for Huntington's disease could be approved.
Huntington's disease is a genetic neurodegenerative disorder that is life-threatening. It can result in symptoms like difficulty swallowing, involuntary movements, memory loss, loss of coordination and many others, and it is thought that tens of thousands of Americans have the disease.
According to a March 2 UniQure report, the company said the FDA had stated that "it cannot agree that data from the Phase I/II studies, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application," and that it "strongly recommended uniQure conduct a prospective, randomized, double-blind, sham surgery-controlled study."
Commenting on the decision, Johnson said the move was "bureaucratic idiocy," and he told Bloomberg: "You’re expecting people to go through sham surgeries where they get holes drilled in their heads?"
"That’s just unbelievable," he added. The FDA has denied the claims, and an official told CNN the surgery would not involve study participants in the control group needing to get holes drilled in their heads, or having 10 hours worth of anesthesia as some reports have said, and instead that it would require "one to three nicks in the scalp" and 30 minutes of anesthesia.
In his interview with Bloomberg, Johnson also said that he couldn't understand the FDA's rejection of the drug for spinocerebellar ataxia, a group of genetic, progressive neurological disorders, manufactured by the company Biohaven Ltd in November, given that he said top scientists had asserted the drug had helped their patients.
Johnson has long advocated expanding access to experimental treatments for people with terminal or rare diseases, arguing they should be given “hope and the freedom to try” emerging therapies. In December, he praised the HHS for adding two rare diseases to the list of conditions screened for in newborns.
He also sponsored the Right to Try Act, which became law in 2018 and allows patients with life‑threatening illnesses to access experimental drugs outside of clinical trials. The FDA has frequently argued, however, that providing unproven treatments carries significant safety risks.
What People Are Saying
HHS spokesman Andrew Nixon told Newsweek: "The number of FDA approvals and rejections under this administration are consistent with historical data over the last decade. In fact, CBER [Center for Biologics Evaluation and Research] had a record number of approvals in December.
"The new plausible mechanism framework will accelerate approvals for individualized gene therapies as this FDA is doing more than any other time in history to help speed up the development of ultra-rare disease cures for the American people.
"Our obligation is to patients and public health and we remain committed to a regulatory approach that brings safe, effective individualized treatments to patients who urgently need them.
"The FDA stands by the scientists evaluating these applications and each rationale is explained in detail through CRL’s [Complete Response Letter], which for the first time, are made available to the public."
FDA Commissioner Marty Makary wrote in a February 23 opinion piece for the Wall Street Journal: "The Food and Drug Administration is announcing new guidance to implement the Plausible Mechanism framework. It will facilitate the development of bespoke therapies that target a specific problem, such as a genetic abnormality, that causes severely debilitating or life-threatening diseases. In these cases, no randomized trial will be required because it’s simply infeasible."
"The framework is modeled on a regulatory 'first' that the Food and Drug Administration achieved last year. Working with FDA scientists who processed their single-patient investigational new drug application within a week, researchers at the Children’s Hospital of Philadelphia treated a newborn who had a fatal genetic disorder with a first-ever personalized gene-editing therapy."
He also wrote in a post on X: "Collectively, 30 million Americans have a rare disease. The problem isn't rare — it's a call to action."
What Happens Next
The details of how Johnson's investigation will proceed are not yet clear. Newsweek has asked the FDA and Johnson's office for more information.
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