U.S. Health and Human Services Secretary Robert F. Kennedy Jr. announced plans to streamline the approval process for drugs treating rare diseases, aiming to bring new therapies to patients faster and solidify the U.S. as a leader in biotechnology innovation.
Speaking at a Food and Drug Administration (FDA) meeting focused on cell and gene therapies, Kennedy emphasized the urgent need to reduce regulatory delays that currently slow down access to potentially life-changing treatments for patients with rare and serious conditions.
“We are committed to finding new ways to accelerate approvals for rare disease drugs and treatments,” Kennedy said. “Our goal is to ensure that America remains the global hub for biotechnology breakthroughs.”
The FDA panel, which included experts from industry, academia, and regulatory agencies, discussed concerns that the U.S. risks falling behind countries like China, where drug development and clinical trials are moving rapidly.
Experts warned that lengthy approval processes could drive companies to conduct research and trials overseas, delaying new treatments for American patients.
Vinay Prasad, the FDA’s head of the Center for Biologics Evaluation and Research, pledged to support rapid access to promising therapies, stating the agency will act swiftly at the first signs of biomedical success to bring treatments to patients.
Faster approvals could significantly impact people living with rare diseases, many of whom currently face limited treatment options and long waits for new drugs to reach the market. Investors in companies developing gene and cell therapies reacted positively, with shares in several biotech firms rising following the announcement.
Experts hope these regulatory changes will boost innovation while maintaining rigorous safety standards, ultimately providing faster relief to patients with unmet medical needs.
