东京——日本癌症和罕见病患者无法获得海外已有的治疗药物，因为美国在2021年至2025年间批准的药物中，超过一半至今尚未获得日本政府批准。
TOKYO -- Japanese patients of cancer and rare diseases are missing treatments available overseas, as more than half of the drugs approved in the U.S. between 2021 and 2025 have yet to receive government greenlight in Japan.
美国食品药品监督管理局和日本医药品医疗器械综合机构发布的清单显示，截至8月底，在美国这五年间批准的238种新药中，有127种（占53%）尚未在日本获批。
A review of lists published by the U.S. Food and Drug Administration and Japan's Pharmaceuticals and Medical Devices Agency found that 127 drugs, or 53% of the 238 new drugs approved in the U.S. over those five years, were unapproved in Japan as of the end of August.
获批产品中，生物药约占30%，小分子药物约占70%。包括细胞和基因疗法在内的再生医学产品未被纳入统计。
Biopharmaceuticals accounted for about 30% of the approved products, while small-molecule drugs made up roughly 70%. Regenerative medicine products, including cell and gene therapies, were excluded from the tally.
在日本尚未获批的药物中，约30%是癌症治疗药物，另有约30%针对日本认定的难治性疾病或其他罕见疾病。
About 30% of the drugs not approved in Japan were cancer treatments, while another 30% targeted conditions designated by Japan as intractable illnesses or other rare diseases.
至少约一半未获批的药物，无法确认在日本有开发计划。这些疗法不只是上市时间可能被推迟，甚至存在永远无法在日本上市的风险——日本将这种情况称为“药品流失”。
For at least around half of the unapproved drugs, no development plans in Japan could be confirmed. The launch of those treatments cannot just be delayed, but there is a risk that they will never become available in the country -- a situation called "drug loss" in Japan.
有关开发计划的信息，是通过查阅美国有关机构管理的临床试验数据库和日本临床试验注册中心，并联系各家公司后汇总而成的。
The information about development plans was compiled using clinical trial databases managed by American authorities and the Japan Registry of Clinical Trials, and also by reaching out to individual companies.
要在日本获得药品批准，企业通常需要提交涉及日本患者的临床试验数据。因此，要让新药在该国上市，海外制药企业必须尽早在日本开展试验。
To obtain drug approval in Japan, companies generally are required to submit clinical trial data involving Japanese patients. Making new medicines available therefore requires overseas drugmakers to conduct trials in the country at an early stage.
但是，未在日本设立业务据点的中小型制药公司常常面临复杂法规和语言障碍等壁垒，难以在该国积极推进开发。
But small and midsize pharmaceutical companies without a presence in Japan often face barriers including complicated regulations and language hurdles, making it difficult to pursue development aggressively in the country.
日本较低的药品价格也构成障碍。据美国药品研究制造商协会报告，经国内生产总值调整后，日本药品价格中位数比美国低约30%。
Japan's lower drug prices also pose an obstacle. The median drug price in Japan, adjusted for gross domestic product, is about 30% less than in the U.S., the Pharmaceutical Research and Manufacturers of America reports.
美国研究机构IQVIA估计，2025年美国药品支出达到9200亿美元，而日本市场规模仅相当于这一水平的约8%。
American research firm IQVIA estimates U.S. pharmaceutical spending reached $920 billion in 2025, while Japan's market amounted to only about 8% of that level.
在欧洲和美国，由初创企业开展药物研发已越来越普遍。在2021年至2025年获美国批准的药物开发者中，约40%的企业年销售额不足5亿美元，且获批时成立时间不足30年。日本尚未批准的药物中，约60%由这类企业研发。资金拮据的初创企业往往很难投入资源进入日本市场。
Drug discovery by startups is increasingly common in Europe and the U.S. Among developers of drugs approved in the U.S. between 2021 and 2025, roughly 40% had annual sales of less than $500 million and were less than 30 years old at the time of approval. About 60% of the drugs still unapproved in Japan were developed by such companies. Cash-strapped startups often find it difficult to devote resources to entering Japan.
普华永道董事宫崎克俊表示：“之所以出现药物流失，是因为越来越多的创新药物正由尚未在日本布局的海外生物技术初创企业开发，同时临床试验也难以招募到足够的患者。”
"Drug loss is occurring because more innovative drugs are being developed by overseas biotech startups without a presence in Japan, and because it is difficult to recruit enough patients for clinical trials," said Katsutoshi Miyazaki, a director at PwC.
西方初创企业研发的药物有时会在全球制药企业收购这些初创企业并开展日本临床试验后进入日本。在许多情况下，日本制药企业还会从海外企业手中取得这些药物在日本的开发权和营销权。
Drugs developed by Western startups sometimes reach Japan after global pharmaceutical companies acquire the startups and conduct Japanese clinical trials. Japanese drugmakers in many cases also have acquired domestic development and marketing rights.
例如，数种获欧洲和美国批准用于治疗杜氏肌营养不良症这一罕见遗传病的药物，尚未获日本批准。该国Nxera Pharma和JCR Pharmaceuticals已从海外企业手中取得其中部分药物的日本开发权，并正在推进开发。
For example, several drugs approved in Europe and the U.S. to treat Duchenne muscular dystrophy, a rare inherited disease, have yet to receive approval in Japan. The country's Nxera Pharma and JCR Pharmaceuticals have obtained Japanese development rights for some of those drugs from overseas companies and are pursuing development.
日本厚生劳动省将那些已获欧洲或美国批准、但尚未在日本获批且没有正在进行的研发项目的药品列为“高需求药品”，并敦促企业开展研发。在2016年至2020年间获美国批准但未获日本批准的86种药品中，已有5种开始在日本研发，厚生劳动省还对另外27种药品提出开发要求或正在寻找研发方。
Japan's Ministry of Health, Labor and Welfare identifies "high-need" drugs that have approval in Europe or the U.S. but remain unapproved in Japan with no active development, and has urged companies to develop them. Of 86 drugs approved in the U.S. but not in Japan between 2016 and 2020, development in Japan has begun for five, while the ministry has issued requests or sought applicants for 27 others.