Wisconsin Republican Senator Ron Johnson has launched a congressional investigation into the Food and Drug Administration’s recent denials of experimental treatments for rare diseases, claiming the agency is looking for “excuses to say no.”
Johnson, who chairs the Senate Permanent Subcommittee on Investigations, plans to request documentation explaining why the FDA rejected several therapies and may call top officials, including FDA Commissioner Marty Makary, to testify before Congress. He specifically criticized the agency’s requirement that biotech company uniQure NV repeat a trial for its Huntington’s disease gene therapy as “bureaucratic idiocy.”
The senator noted that treatments for rare conditions affect an estimated 30 million Americans and that the FDA’s decisions often delay access to life-saving therapies. Johnson has previously sponsored the Right to Try Act, which provides expanded access to experimental medicines for seriously ill patients.
The FDA maintains that its rejections are based on scientific review and detailed explanations in rejection letters, though patient advocates argue the agency’s actions have caused years of delays for patients with few or no treatment options.
