由于缺少PBS列表, 患有罕见疾病的澳大利亚女性无法获得美国批准的药物。 Australian woman with rare disease can't access U.S.-approved drug due to lack of PBS listing.

多明尼克·罗思(Dominique Roth)是一名51岁的澳大利亚妇女,患有罕见的疾病CTX,尽管美国批准了一种药物Ctexli(chenodiol),可以减缓其疾病发展速度,但她仍面临有限的治疗选择。 Dominique Roth, a 51-year-old Australian woman with the rare disorder CTX, faces limited treatment options despite a U.S.-approved drug, Ctexli (chenodiol), being available to slow her disease’s progression. 该药物于2025年2月获得林业发展局批准,但在澳大利亚没有登记,也没有一份PBS清单,因此只能通过每年花费约500 000美元的昂贵特别准入计划获取。 The drug, approved by the FDA in February 2025, is not registered in Australia and lacks a PBS listing, making it accessible only through a costly Special Access Scheme that costs about $500,000 annually. 没有一家公司申请将PBS包括在内,而美国制造商Mirum制药公司没有计划在美国境外销售该药物。 No company has applied for PBS inclusion, and the U.S. manufacturer, Mirum Pharmaceuticals, has no plans to sell the drug outside the U.S. 倡导者呼吁PBS改革以改善罕见病治疗的可及性，而罗斯则通过NDIS获得重要支持。 Advocates urge PBS reform to improve access to rare disease treatments, while Roth receives essential support through the NDIS.