When a 19‑year‑old woman from South Carolina was diagnosed with glioblastoma, her family faced a grim prognosis. After surgery failed to stop the aggressive tumor, she flew to London for a personalized immunotherapy that her doctors credit with clearing the cancer. The treatment, developed by an American company, was not available in the United States because the Food and Drug Administration (FDA) has not granted even limited trial approval.
Why the current system leaves patients stranded
Under existing FDA rules, physicians must obtain formal permission before prescribing investigational drugs, even when a patient’s condition is terminal. The process can take months, and many patients die before a decision is reached. Critics argue that this approach prioritizes bureaucratic caution over the constitutional right of individuals to make medical choices for themselves.
Trump’s “right‑to‑try” legislation offers a foundation
President Trump signed the federal Right‑to‑Try Act, which allows terminally ill patients to request access to unapproved therapies after they have completed Phase 1 clinical trials. While the law was a step forward, advocates say it remains too narrow and that the FDA continues to impose delays that undermine its intent.
Three concrete steps the administration can take
- Adopt a “plausible success” standard for rare diseases. When compelling scientific evidence suggests a therapy could work, the FDA should permit patients to try it without waiting for full approval.
- Expand compassionate‑use authority. Treat physicians’ requests to prescribe promising investigational treatments as notifications rather than permission requests, reducing paperwork and speeding access.
- Recognize foreign approvals. If a drug has been cleared by a reputable regulator in another developed nation, the FDA should allow U.S. patients to use it under a distinct designation, leaving the final decision to the patient and their doctor.
Economic and moral arguments
Allowing patients to access American‑developed therapies at home keeps medical innovation and its benefits within the United States. It also spares families the financial burden of international travel and the emotional toll of seeking care abroad. Moreover, patients who enroll in such programs provide valuable real‑world data that can accelerate future approvals.
Administration’s response
White House officials have reiterated their commitment to “Make America Healthy Again.” They note that the Right‑to‑Try Act was a historic achievement and that the Department of Health and Human Services is reviewing ways to streamline compassionate‑use requests. The administration argues that these reforms will preserve safety while honoring individual liberty.
What’s at stake
Every day that a promising therapy remains inaccessible is a day that patients with terminal illnesses lose. By acting now, the Trump administration can demonstrate that it values both innovation and the constitutional principle of personal choice in healthcare.
Original reporting: Herald-Standard – latest news for Uniontown, Pennsylvania — read the source article.