Wisconsin Republican Sen. Ron Johnson reportedly is launching an investigation into the Food and Drug Administration's recent denials of treatments for rare diseases.
Johnson, who chairs the Senate Permanent Subcommittee on Investigations, said he plans to seek documentation explaining why the FDA rejected several experimental treatments and may call top agency officials, including FDA Commissioner Marty Makary, to testify before Congress.
According to Bloomberg News, the senator wants access to the FDA's written rejection notices to drugmakers, known as "complete response letters," which outline why therapies are denied.
"The stories are so outrageous," Johnson told Bloomberg. "It just appears that they're looking for excuses to say no."
The investigation comes after the FDA rejected or delayed multiple therapies for rare diseases, often requesting additional studies before granting approval.
Those decisions have angered patient advocates who argue the agency's demands can add years of delay for treatments targeting conditions with few or no existing options.
Johnson sharply criticized the FDA's request that uniQure NV conduct a new trial for its experimental gene therapy for Huntington's disease, calling the demand "bureaucratic idiocy."
The senator objected to trial designs that critics say could require invasive procedures for patients in control groups, though the FDA disputes that characterization.
The agency, which is overseen by the Department of Health and Human Services, has defended its record.
A spokesman for HHS told Bloomberg that the number of drug approvals and rejections under the Trump administration remains consistent with historical trends over the past decade.
The FDA says decisions are largely guided by agency scientists and that its rejection letters now provide detailed explanations.
Still, the controversy highlights growing tensions over the approval of therapies for rare diseases, which affect about 30 million Americans, according to federal estimates.
In recent months, the FDA has rejected about 20 drug applications or declined to review them, including several gene therapies for rare conditions, according to The New York Times.
Patient advocates and biotech companies say the agency has at times changed requirements late in the process, after companies spent years and hundreds of millions of dollars developing those treatments.
Johnson has long argued the FDA is overly cautious when dealing with experimental therapies for patients facing terminal illnesses. He previously sponsored the Right to Try Act, a 2018 law designed to give seriously ill patients expanded access to experimental medicines that have not yet received full FDA approval.
The agency, however, maintains that its cautious approach protects patients from unsafe or ineffective treatments.
Officials say rigorous clinical trials remain the gold standard for determining whether new therapies work.
At the same time, the FDA recently proposed guidelines aimed at accelerating the development of customized treatments for rare genetic diseases.
The draft framework would create a pathway for certain therapies tested in only a small number of patients, reflecting the difficulty of conducting large trials for extremely rare conditions, according to The Associated Press.
For Johnson and other critics, however, the agency's recent decisions suggest a deeper problem.
"They're moving the goalposts," Johnson said, arguing that desperate patients should have faster access to promising treatments rather than facing years of regulatory delays.
Charlie McCarthy ✉
Charlie McCarthy, a writer/editor at Newsmax, has nearly 40 years of experience covering news, sports, and politics.
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