FDA plans to optimize the approval process to help accelerate the market launch of drugs for rare diseases.

Robert F. Kennedy Jr., Secretary of Health and Human Services of the United States, publicly announced on Thursday that the Food and Drug Administration (FDA) is considering opening a "fast track" for the approval of new drugs for rare diseases, by streamlining the regulatory framework to shorten the process of bringing innovative therapies to market. During the FDA workshop on cell and gene therapy held on the same day, Kennedy explicitly stated: "We will build a more efficient regulatory pathway to ensure that the United States continues to lead global biotechnology innovation." According to reports, this closed-door meeting focused on regulatory reform for cutting-edge therapies. Some members of the development group expressed concern: "While Chinese innovative drug companies are advancing their pipeline at a staggering pace, our regulatory system is still examining every application under a microscope." The FDA has not yet announced specific reform plans, but the market widely expects systematic optimization of processes such as orphan drug designation, clinical trial design, and accelerated approval pathways, while ensuring safety. This regulatory reform not only affects the accessibility of drugs for rare disease patients but also reshapes the global landscape of biopharmaceutical innovation.