FDA Justified In Controversial Decisions To Reject Two Rare Disease Drugs, Letters To Companies Show
Patients can access experimental drugs through the “compassionate use” provision, but the law requires a higher standard for full approval
Critics say the Food and Drug Administration (FDA) is keeping life-saving medicines from patients with rare, deadly diseases. The agency rejected an immunotherapy shot for advanced melanoma even though a third of patients who had not responded to prior treatments showed strong and durable responses, noted the Wall Street Journal, with tumors shrinking in nearly all patients and few serious side effects. They also say the FDA has delayed a therapy for Barth syndrome, a rare genetic disorder that causes fatal heart failure in children, despite parents pleading that their children have no other options. In both cases, the charge is that regulators are hiding behind higher scientific standards and demanding randomized trials that would take years, while patients die waiting.