最近FDA发表了加速批准用于治疗绝症患者药物的新程序,同时对其安全性和有效性仍注意合适保证。新程序旨在加速医治危及生命和致残疾病药物的发展、评价和上市。所谓“危及生命”是指如果不中止病程就有高度可能致死的疾病(如艾滋病和癌症)以及对具有潜在致命结果的疾病或状态进行以生存期作为分析终末指标的临床试验。所谓“致残”是指能导致较严重的不可逆病态(如致盲或神经退变等)的疾病或 Recently, the FDA published a new procedure for accelerating the approval of drugs for the treatment of terminally ill patients. At the same time, it still pays due attention to its safety and effectiveness. The new procedures aim to accelerate the development, evaluation, and marketing of medicines that are life threatening and disabling. The so-called “life-threatening” refers to diseases that are highly likely to cause death if they do not stop the disease (such as AIDS and cancer), and clinical trials that use survival as a final indicator of the analysis for diseases or conditions that have potentially fatal consequences. The so-called “disabling” refers to diseases that can cause more serious irreversible diseases (such as blinding or neurodegeneration, etc.) or