{"id":29019,"date":"2025-08-16T00:00:02","date_gmt":"2025-08-15T16:00:02","guid":{"rendered":"https:\/\/naipnews.naipo.com\/?p=29019"},"modified":"2025-08-19T10:17:22","modified_gmt":"2025-08-19T02:17:22","slug":"%e5%9b%bd%e9%99%85%e8%8d%af%e5%8e%82%e6%96%b0%e8%8d%af%e5%8a%a0%e9%80%9f%e4%b8%8a%e5%b8%82%e7%ad%96%e7%95%a5%ef%bc%9a%e5%ad%a4%e5%84%bf%e8%8d%af%e8%ae%a4%e5%ae%9a","status":"publish","type":"post","link":"https:\/\/naipnews.naipo.com\/zh-hans\/29019\/","title":{"rendered":"\u56fd\u9645\u836f\u5382\u65b0\u836f\u52a0\u901f\u4e0a\u5e02\u7b56\u7565\uff1a\u5b64\u513f\u836f\u8ba4\u5b9a"},"content":{"rendered":"<blockquote><p>\u5c3d\u7ba1\u533b\u5b66\u8fdb\u5c55\u65e5\u65b0\u6708\u5f02\uff0c\u4f46\u5168\u7403\u4ecd\u670995%\u7684\u7f55\u89c1\u75be\u75c5\uff08rare diseases\uff09\u4ecd\u5904\u4e8e\u65e0\u836f\u53ef\u533b\u7684\u72b6\u6001\u3002\u4e3a\u4e86\u7167\u987e\u7f55\u89c1\u75be\u75c5\u60a3\u8005\u7684\u9700\u6c42\uff0c\u9664\u4e86\u805a\u7126\u7279\u5b9a\u9057\u4f20\u6027\u7f55\u89c1\u75be\u75c5\u5f00\u53d1\u836f\u7269\u7684\u751f\u6280\u516c\u53f8\uff0c\u4e5f\u6709\u56fd\u9645\u5927\u578b\u836f\u5382\u805a\u7126\u7279\u5b9a\u7f55\u89c1\u75be\u75c5\u3002\u300c\u5b64\u513f\u836f\u300d\u6307\u7684\u662f\u7528\u6765\u6cbb\u7597\u3001\u9884\u9632\u6216\u8bca\u65ad\u7f55\u89c1\u75be\u75c5\u7684\u836f\u54c1\u3002\u7531\u4e8e\u8fd9\u4e9b\u75be\u75c5\u5f71\u54cd\u7684\u4eba\u53e3\u5f88\u5c11\uff0c\u6bcf\u4e00\u79cd\u75be\u75c5\u53ef\u80fd\u53ea\u6709\u51e0\u5343\u4eba\u6216\u66f4\u5c11\uff0c\u56e0\u6b64\u5e02\u573a\u56de\u62a5\u6709\u9650\uff0c\u5236\u836f\u516c\u53f8\u901a\u5e38\u7f3a\u4e4f\u5f00\u53d1\u52a8\u673a\u3002\u4e3a\u4e86\u89e3\u51b3\u8fd9\u4e2a\u95ee\u9898\uff0c\u653f\u5e9c\u900f\u8fc7\u300c\u5b64\u513f\u836f\u8ba4\u5b9a\u300d\uff08Orphan Drug Designation\uff0cODD\uff09\u7ed9\u4e88\u5f00\u53d1\u8fd9\u4e9b\u836f\u7269\u7684\u8bf1\u56e0\u3002<\/p><\/blockquote>\n<figure id=\"attachment_13063\" aria-describedby=\"caption-attachment-13063\" style=\"width: 1200px\" class=\"wp-caption aligncenter\"><img loading=\"lazy\" decoding=\"async\" class=\"size-full wp-image-13063\" src=\"https:\/\/naipnews.naipo.com\/wp-content\/uploads\/2025\/04\/2582977515_51M-scaled-e1755569806741.jpg\" alt=\"\" width=\"1200\" height=\"800\" \/><figcaption id=\"caption-attachment-13063\" class=\"wp-caption-text\">\u56fe\u7247\u6765\u6e90 : shutterstock\u3001\u8fbe\u5fd7\u5f71\u50cf<\/figcaption><\/figure>\n<p>\u5b64\u513f\u836f\u8ba4\u5b9a\u662f\u7531\u653f\u5e9c\u673a\u5173\u6240\u63d0\u4f9b\u7684\u7279\u6b8a\u6cd5\u89c4\u5236\u5ea6\uff0c\u65e8\u5728\u9f13\u52b1\u5236\u836f\u516c\u53f8\u7814\u53d1\u6cbb\u7597\u7f55\u89c1\u75be\u75c5\u7684\u836f\u7269\u3002\u4ee5\u7f8e\u56fd\u4e3a\u4f8b\uff0c\u836f\u54c1\u82e5\u8981\u83b7\u5f97\u5b64\u513f\u836f\u8ba4\u5b9a\uff0c\u5fc5\u987b\u7b26\u5408\u75be\u75c5\u5f71\u54cd\u7f8e\u56fd\u5c11\u4e8e20\u4e07\u4eba\uff1b\u6216\u662f\u867d\u7136\u60a3\u8005\u8d85\u8fc720 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id=\"attachment_29012\" aria-describedby=\"caption-attachment-29012\" style=\"width: 986px\" class=\"wp-caption aligncenter\"><img loading=\"lazy\" decoding=\"async\" class=\"wp-image-29012 size-full\" src=\"https:\/\/naipnews.naipo.com\/wp-content\/uploads\/2025\/08\/0812-2.jpg\" alt=\"\" width=\"986\" height=\"461\" srcset=\"https:\/\/naipnews.naipo.com\/wp-content\/uploads\/2025\/08\/0812-2.jpg 986w, https:\/\/naipnews.naipo.com\/wp-content\/uploads\/2025\/08\/0812-2-768x359.jpg 768w\" sizes=\"auto, (max-width: 986px) 100vw, 986px\" \/><figcaption id=\"caption-attachment-29012\" class=\"wp-caption-text\">\u56fe1. 2015\uff5e2024\u5e74FDA\u6838\u51c6\u7684\u5b64\u513f\u836f\u6570\u91cf\uff1b\u56fe\u7247\u6765\u6e90\uff1aFDA\/\u53f0\u6e7e\u5730\u533a\u751f\u6280\u4e2d\u5fc3\u6574\u7406\uff082025.01\uff09<\/figcaption><\/figure>\n<p>\u4e0d\u8fc7\uff0c\u7f55\u89c1\u75be\u75c5\u4ecd\u5177\u6709\u672a\u6ee1\u8db3\u533b\u7597\u9700\u6c42\uff0c\u6839\u636e\u7f8e\u56fd\u7f55\u89c1\u75be\u75c5\u7ec4\u7ec7\uff08National Organization for Rare Disease, NORD\uff09\u7edf\u8ba1\uff0c\u76ee\u524d\u5df2\u6709\u8d85\u8fc711,000\u79cd\u7f55\u89c1\u75be\u75c5\uff0c\u76ee\u524d\u7ea6\u67094%\uff5e6%\u7f55\u89c1\u75be\u75c5\u5df2\u6709\u81f3\u5c111\u79cd\u5b64\u513f\u836f\u53ef\u4ee5\u6cbb\u7597\uff0c\u4ea6\u5373\u4ecd\u670995%\u7684\u7f55\u89c1\u75be\u75c5\u4ecd\u5904\u4e8e\u65e0\u836f\u53ef\u533b\u7684\u72b6\u6001\u3002<\/p>\n<div id=\"ez-toc-container\" class=\"ez-toc-v2_0_69_1 counter-hierarchy ez-toc-counter ez-toc-grey ez-toc-container-direction\">\n<div class=\"ez-toc-title-container\">\n<p class=\"ez-toc-title\" style=\"cursor:inherit\">\u76ee\u9304<\/p>\n<span class=\"ez-toc-title-toggle\"><a href=\"#\" class=\"ez-toc-pull-right ez-toc-btn ez-toc-btn-xs ez-toc-btn-default ez-toc-toggle\" aria-label=\"Toggle Table of Content\"><span class=\"ez-toc-js-icon-con\"><span class=\"\"><span class=\"eztoc-hide\" style=\"display:none;\">Toggle<\/span><span class=\"ez-toc-icon-toggle-span\"><svg style=\"fill: #999;color:#999\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" class=\"list-377408\" width=\"20px\" height=\"20px\" viewBox=\"0 0 24 24\" fill=\"none\"><path d=\"M6 6H4v2h2V6zm14 0H8v2h12V6zM4 11h2v2H4v-2zm16 0H8v2h12v-2zM4 16h2v2H4v-2zm16 0H8v2h12v-2z\" fill=\"currentColor\"><\/path><\/svg><svg style=\"fill: #999;color:#999\" class=\"arrow-unsorted-368013\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"10px\" height=\"10px\" viewBox=\"0 0 24 24\" version=\"1.2\" baseProfile=\"tiny\"><path d=\"M18.2 9.3l-6.2-6.3-6.2 6.3c-.2.2-.3.4-.3.7s.1.5.3.7c.2.2.4.3.7.3h11c.3 0 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title=\"\u4ee5\u5b64\u513f\u836f\u8ba4\u5b9a\u52a0\u901f\u836f\u54c1\u4e0a\u5e02\">\u4ee5\u5b64\u513f\u836f\u8ba4\u5b9a\u52a0\u901f\u836f\u54c1\u4e0a\u5e02<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-3\" href=\"https:\/\/naipnews.naipo.com\/zh-hans\/29019\/#RMAT%E8%AE%A4%E5%AE%9A%E5%8A%A0%E9%80%9F%E4%B8%8A%E5%B8%82\" title=\"RMAT\u8ba4\u5b9a\u52a0\u901f\u4e0a\u5e02\">RMAT\u8ba4\u5b9a\u52a0\u901f\u4e0a\u5e02<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-4\" href=\"https:\/\/naipnews.naipo.com\/zh-hans\/29019\/#%E8%97%89%E7%94%B1%E3%80%8C%E8%AE%A4%E5%AE%9A%E3%80%8D%EF%BC%88Designation%EF%BC%89%E5%8A%A0%E9%80%9F%E4%BA%A7%E5%93%81%E5%BC%80%E5%8F%91%E8%BF%9B%E7%A8%8B\" title=\"\u85c9\u7531\u300c\u8ba4\u5b9a\u300d\uff08Designation\uff09\u52a0\u901f\u4ea7\u54c1\u5f00\u53d1\u8fdb\u7a0b\">\u85c9\u7531\u300c\u8ba4\u5b9a\u300d\uff08Designation\uff09\u52a0\u901f\u4ea7\u54c1\u5f00\u53d1\u8fdb\u7a0b<\/a><\/li><\/ul><\/nav><\/div>\n<h2><span class=\"ez-toc-section\" id=\"%E8%8D%AF%E5%8E%82%E5%88%87%E5%85%A5%E5%AD%A4%E5%84%BF%E8%8D%AF%E5%BC%80%E5%8F%91%E4%B9%8B%E7%AD%96%E7%95%A5\"><\/span><strong>\u836f\u5382\u5207\u5165\u5b64\u513f\u836f\u5f00\u53d1\u4e4b\u7b56\u7565<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n<p>\u4e3a\u4e86\u7167\u987e\u7f55\u89c1\u75be\u75c5\u60a3\u8005\u7684\u9700\u6c42\uff0c\u6709\u4e9b\u751f\u6280\u516c\u53f8\u4f1a\u805a\u7126\u7279\u5b9a\u9057\u4f20\u6027\u7f55\u89c1\u75be\u75c5\uff0c\u5f00\u53d1\u6cbb\u7597\u836f\u7269\uff1b\u4e5f\u6709\u5927\u578b\u836f\u5382\u805a\u7126\u7279\u5b9a\u7f55\u89c1\u75be\u75c5\u3002\u5bc7\u6021\u8861\u4e3e\u4f8b\uff0c\u7f8e\u56fd\u836f\u5382Vertex\u4e13\u6ce8\u7814\u7a76\u56ca\u6027\u7ea4\u7ef4\u5316\uff08CF\uff09\u3001\u5927\u578b\u533b\u7597\u4fdd\u5065\u5236\u9020\u5546Johnson &amp; Johnson\u5219\u4ee5\u591a\u53d1\u6027\u9aa8\u9ad3\u7624\uff08MM\uff09\u4e3a\u516c\u53f8\u91cd\u70b9\u53d1\u5c55\u9886\u57df\uff0c\u4e24\u5bb6\u516c\u53f8\u6240\u5f00\u53d1\u7684\u5b64\u513f\u836f\u90fd\u8fdb\u5165\u5168\u7403\u5b64\u513f\u836f\u9500\u552e\u524d\u5341\u540d\u3002<\/p>\n<h2><span class=\"ez-toc-section\" id=\"%E4%BB%A5%E5%AD%A4%E5%84%BF%E8%8D%AF%E8%AE%A4%E5%AE%9A%E5%8A%A0%E9%80%9F%E8%8D%AF%E5%93%81%E4%B8%8A%E5%B8%82\"><\/span><strong>\u4ee5\u5b64\u513f\u836f\u8ba4\u5b9a\u52a0\u901f\u836f\u54c1\u4e0a\u5e02<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n<p>\u56ca\u6027\u7ea4\u7ef4\u5316\u662f\u4e00\u79cd\u7f55\u89c1\u7684\u3001\u7f29\u77ed\u5bff\u547d\u7684\u9057\u4f20\u6027\u75be\u75c5\uff0c\u76ee\u524d\u7ea6\u670910.9\u4e07\u4eba\u60a3\u6709\u56ca\u6027\u7ea4\u7ef4\u5316\uff0c\u7f8e\u56fd\u3001\u6b27\u6d32\u3001\u6fb3\u6d32\u548c\u52a0\u62ff\u5927\u5360\u5168\u7403\u56ca\u6027\u7ea4\u7ef4\u5316\u4eba\u53e3\u7684\u7edd\u5927\u591a\u6570\u3002\u968f\u7740\u56ca\u6027\u7ea4\u7ef4\u5316\u8de8\u819c\u4f20\u5bfc\u8c03\u8282\u5668\uff08CFTR\uff09\u8c03\u8282\u7597\u6cd5\u7684\u51fa\u73b0\uff0c\u5f97\u4ee5\u89e3\u51b3\u75be\u75c5\u7684\u6839\u672c\u539f\u56e0\uff0c\u8ba9CF\u7684\u6cbb\u7597\u53d1\u751f\u4e86\u5de8\u5927\u53d8\u5316\u3002<\/p>\n<p>Vertex\u662f\u4e00\u5bb6\u603b\u90e8\u4f4d\u4e8e\u7f8e\u56fd\u6ce2\u58eb\u987f\u7684\u5236\u836f\u516c\u53f8\uff0c\u81f4\u529b\u4e8e\u8bbe\u8ba1\u3001\u5f00\u53d1\u6cbb\u7597CF\u7684\u836f\u7269\uff0c\u81f3\u4eca\u5df2\u83b7\u8bb8\u53ef\u7684\u7684\u4e94\u79cd\u836f\u7269\uff0c\u9002\u7528\u4e8e\u7ea690%\u7684CF\u60a3\u8005\uff0c\u6709\u8d85\u8fc7 75,000\u540d\u60a3\u8005\u6b63\u5728\u63a5\u53d7Vertex\u836f\u54c1\u7684\u6cbb\u7597\u3002\u9664\u7f8e\u56fd\u4ee5\u5916\uff0cVertex\u7684CF\u836f\u7269\u5df2\u9500\u5f80\u516d\u5927\u6d32\u768460\u591a\u4e2a\u56fd\u5bb6\u3002\u672a\u6765Vertex\u5c06\u4e3a\u90a3\u4e9b\u56e0\u75be\u75c5\u7279\u6b8a\u6027\u800c\u65e0\u6cd5\u4ece\u73b0\u6709\u7684CF\u836f\u7269\u4e2d\u53d7\u76ca\u7684\u5269\u4f59\u60a3\u8005\uff0c\u7ee7\u7eed\u63d0\u4f9b\u5148\u8fdb\u7684\u6cbb\u7597\u65b9\u6cd5<a href=\"#_ftn1\" name=\"_ftnref1\">[1]<\/a>\u3002\u5bc7\u6021\u8861\u6307\u51fa\uff0cVertex\u805a\u7126\u7814\u53d1\u6cbb\u7597\u4e25\u91cd\u75be\u75c5\u7684\u5c0f\u5206\u5b50\u836f\u7269\uff0c\u9884\u6d4b2028\u5e74\u5b64\u513f\u836f\u5360Vertex\u6574\u4f53\u836f\u54c1\u9500\u552e\u989d\u9ad8\u8fbe94%\u3002<\/p>\n<p>\u9664\u4e86\u805a\u7126\u4e8e\u7279\u5b9a\u7f55\u75c5\u7cbe\u8fdb\u836f\u7269\u5f00\u53d1\uff0c\u66f4\u591a\u6570\u7684\u5927\u578b\u836f\u5382\u662f\u5148\u4ee5\u7f55\u75c5\u52a0\u901f\u836f\u7269\u4e0a\u5e02\uff0c\u518d\u6269\u5c55\u81f3\u5e02\u573a\u8f83\u5927\u7684\u9002\u5e94\u75c7\uff0c\u4f8b\u5982\u7f8e\u56fd\u9ed8\u514b\u836f\u5382\uff08Merck\uff09\u5f00\u53d1\u7684\u514d\u75ab\u7597\u6cd5\u6297\u764c\u836f\u54c1Keytruda\uff0c\u5df2\u7ecf\u88abFDA\u6279\u51c6\u7528\u4e8e\u591a\u79cd\u764c\u75c7\u7684\u6cbb\u7597\uff0c\u4f462014\u5e74\u662f\u4ee5\u9ed1\u8272\u7d20\u7624\u9996\u6b21\u83b7\u4e0a\u5e02\u8bb8\u53ef\u3002\u5bc7\u6021\u8861\u8fdb\u4e00\u6b65\u89e3\u91ca\uff0c\u5b64\u513f\u836f\u8ba4\u5b9a\u7684\u4e3b\u8981\u4ef7\u503c\uff0c\u5728\u4e8e\u4e0a\u5e02\u540e\u53ef\u83b7\u5f97\u5e02\u573a\u4e13\u5c5e\u6743\u7684\u4fdd\u969c\uff08\u4f46\u4ec5\u9650\u4e8e\u83b7\u4e0a\u5e02\u8bb8\u53ef\u7684\u9002\u5e94\u75c7\uff09\uff0c\u5bf9\u4e8e\u836f\u5382\u800c\u8a00\uff0c\u53ef\u5229\u7528\u540c\u4e00\u5b64\u513f\u836f\u8ba4\u5b9a\u4e0b\u7684\u9002\u5e94\u75c7\u6216\u6269\u5c55\u5e94\u7528\uff0c\u4ee5\u5ef6\u7eed\u5e02\u573a\u7684\u4e13\u5c5e\u6743\u4fdd\u969c\u3002<\/p>\n<h2><span class=\"ez-toc-section\" id=\"RMAT%E8%AE%A4%E5%AE%9A%E5%8A%A0%E9%80%9F%E4%B8%8A%E5%B8%82\"><\/span><strong>RMAT<\/strong><strong>\u8ba4\u5b9a\u52a0\u901f\u4e0a\u5e02<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n<p>2016\u5e7412\u6708FDA\u589e\u52a0\u300c\u518d\u751f\u533b\u7597\u5148\u8fdb\u7597\u6cd5\u8ba4\u5b9a\u300d\uff08Regenerative Medicine Advanced Therapy Designation\uff0cRMAT\uff09\uff0c\u8ba9\u6cbb\u7597\u4e25\u91cd\u75be\u75c5\u7684\u518d\u751f\u533b\u5b66\u7597\u6cd5\u80fd\u52a0\u901f\u4e0a\u5e02\u3002RMAT\u662f\u4e00\u79cd\u7f8e\u56fdFDA\u4e3a\u65b0\u578b\u518d\u751f\u533b\u7597\u4ea7\u54c1\u8bbe\u7acb\u7684\u52a0\u901f\u5ba1\u67e5\u8d44\u683c\uff0c\u4f46\u4ec5\u9650\u4e8e\u518d\u751f\u533b\u7597\u4ea7\u54c1\uff0c\u5305\u62ec\u7ec6\u80de\u7597\u6cd5\u3001\u6cbb\u7597\u6027\u7ec4\u7ec7\u5de5\u7a0b\u4ea7\u54c1\u3001\u4eba\u4f53\u7ec6\u80de\u548c\u7ec4\u7ec7\u4ea7\u54c1\u3001\u6216\u7ec4\u5408\u4ea7\u54c1\u3002\u83b7\u5f97RMAT\u8ba4\u5b9a\u540e\uff0cFDA \u5c06\u4e3b\u52a8\u534f\u52a9\u4ea7\u54c1\u5f00\u53d1\u7b56\u7565\u3001\u4e34\u5e8a\u8bd5\u9a8c\u8bbe\u8ba1\uff0c\u5e76\u83b7\u5f97\u52a0\u901f\u4e0a\u5e02\u5ba1\u67e5\u673a\u4f1a\uff0c\u7f29\u77ed\u5f00\u53d1\u4e0e\u5ba1\u67e5\u65f6\u95f4\u3002<\/p>\n<p>\u4e0e\u7f8e\u56fd FDA\u300c\u5feb\u901f\u5ba1\u67e5\u300d\uff08Fast Track\uff09\u8ba4\u5b9a\u3001\u7a81\u7834\u6027\u7597\u6cd5\uff08Breakthrough Therapy\uff09\u7b49\u5176\u4ed6\u52a0\u901f\u901a\u9053\u76f8\u6bd4\uff0cRMAT\u8ba4\u5b9a\u7684\u5f3a\u5ea6\u66f4\u5f3a\uff0c\u4f46\u4ec5\u9650\u518d\u751f\u533b\u7597\u4ea7\u54c1\u3002\u5bc7\u6021\u8861\u6307\u51fa\uff0cRMAT\u7533\u8bf7\u6848\u9010\u5e74\u6210\u957f\uff0c\u81f32024\u5e74\u5e95\u7d2f\u79ef\u7533\u8bf7297\u6848\uff0c\u901a\u8fc7143\u6848\uff0cFDA\u6838\u51c6\u6bd4\u4f8b\u903e\u4e03\u6210\uff1b2025\u5e74\u81f36\u6708\u5e95\u5df2\u670971\u6848\u7533\u8bf7\u3001\u901a\u8fc741\u6848\uff0c\u76ee\u524d\u4e3a\u6b62\u5e76\u65e0RMAT\u901a\u8fc7\u540e\u64a4\u56de\u6216\u64a4\u9500\u7684\u6848\u4ef6\u3002<\/p>\n<figure id=\"attachment_29009\" aria-describedby=\"caption-attachment-29009\" style=\"width: 1000px\" class=\"wp-caption aligncenter\"><img loading=\"lazy\" decoding=\"async\" class=\"wp-image-29009 size-full\" src=\"https:\/\/naipnews.naipo.com\/wp-content\/uploads\/2025\/08\/0812-1.jpg\" alt=\"\" width=\"1000\" height=\"529\" \/><figcaption id=\"caption-attachment-29009\" class=\"wp-caption-text\">\u56fe2. \u5386\u5e74FDA RMAT\u8ba4\u5b9a\u60c5\u5f62\uff1b\u56fe\u7247\u6765\u6e90\uff1a\u6570\u636e\u6e90\uff1aFDA\uff082025.07\uff09\u3001\u5bc7\u6021\u8861\u6574\u7406<\/figcaption><\/figure>\n<h2><span class=\"ez-toc-section\" id=\"%E8%97%89%E7%94%B1%E3%80%8C%E8%AE%A4%E5%AE%9A%E3%80%8D%EF%BC%88Designation%EF%BC%89%E5%8A%A0%E9%80%9F%E4%BA%A7%E5%93%81%E5%BC%80%E5%8F%91%E8%BF%9B%E7%A8%8B\"><\/span><strong>\u85c9\u7531\u300c\u8ba4\u5b9a\u300d\uff08<\/strong><strong>Designation<\/strong><strong>\uff09\u52a0\u901f\u4ea7\u54c1\u5f00\u53d1\u8fdb\u7a0b<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n<p>\u7f8e\u56fdFDA\u4e8e\u4eca\u5e746\u6708\u5ba3\u5e03\u4e00\u9879\u65b0\u7684\u56fd\u5bb6\u4f18\u5148\u51ed\u8bc1\u8ba1\u5212\uff08Commissioner\u2019s National Priority Voucher\uff0cCNPV\uff09\uff0c\u63d0\u4f9b\u673a\u4f1a\u7ed9\u9650\u91cf\u7684\u836f\u7269\u548c\u751f\u7269\u5236\u5242\u5f00\u53d1\u5546\u53c2\u4e0e\u3002FDA\u9884\u8ba1\u5728CNPV\u8ba1\u5212\u5b9e\u65bd\u7684\u7b2c\u4e00\u5e74\uff0c\u5411\u7b26\u5408\u7f8e\u56fd\u56fd\u5bb6\u91cd\u70b9\u7684\u516c\u53f8\u63d0\u4f9b\u6709\u9650\u6570\u91cf\u7684\u5ba1\u6279\uff0c\u7b26\u5408\u7f8e\u56fd\u56fd\u5bb6\u4f18\u5148\u4e8b\u9879\u8fdb\u884c\u7814\u53d1\u7684\u5382\u5546\uff0c\u53ef\u83b7\u5f97\u52a0\u5f3a\u6c9f\u901a\u548c\u6eda\u52a8\u5ba1\u67e5\u7b49\u7279\u6b8a\u798f\u5229\uff1b\u5982\u679c\u4f7f\u7528\u5ba1\u6279\u7684\u4ea7\u54c1\u7b26\u5408\u52a0\u901f\u5ba1\u6279\u7684\u6cd5\u5f8b\u8981\u6c42\uff0cFDA\u53ef\u80fd\u4f1a\u6388\u4e88\u52a0\u901f\u5ba1\u6279\uff0c\u8ba9\u5ba1\u67e5\u836f\u7269\u548c\u751f\u7269\u5236\u5242\u7684\u5ba1\u6279\u65f6\u95f4\uff0c\u4ece10~12\u4e2a\u6708\u7f29\u77ed\u81f31~2\u4e2a\u6708<a href=\"#_ftn2\" name=\"_ftnref2\">[2]<\/a>\uff0c\u8fd9\u9879CNPV\u5148\u5bfc\u8ba1\u5212\u5df2\u4e8e2025\u5e747\u670822\u65e5\u5f00\u59cb\u5b9e\u65bd\u3002<\/p>\n<p>\u5bc7\u6021\u8861\u8ba4\u4e3a\uff0c\u5584\u7528\u300c\u8ba4\u5b9a\u300d\u53ef\u52a0\u901f\u65b0\u836f\u4ea7\u54c1\u5f00\u53d1\u8fdb\u7a0b\uff0c\u65b0\u836f\u5382\u5546\u5e94\u968f\u65f6\u66f4\u65b0\u5404\u9879\u8ba4\u5b9a\u53ca\u89c4\u8303\u4fe1\u606f\uff0c\u8ba9\u8ba4\u5b9a\u6210\u4e3a\u4ea7\u54c1\u5f00\u53d1\u91cd\u8981\u7684\u52a9\u529b\u3002<\/p>\n<p><strong>\u6570\u636e\u6e90\uff1a<\/strong><\/p>\n<ol>\n<li>2025\/7\/24\uff0c\u53f0\u6e7e\u5730\u533a\u8d44\u7b56\u4f1a\u79d1\u6280\u6cd5\u5f8b\u7814\u7a76\u6240\u4e3b\u529e\u300c\u521b\u65b0\u836f\u54c1\u52a0\u901f\u4e0a\u5e02\u6cd5\u5236\u4e0e\u4ea7\u4e1a\u7b56\u7565\u62a5\u544a\u53d1\u5e03\u4f1a\u300d\uff0c\u5bc7\u6021\u8861\u7b80\u62a5<\/li>\n<\/ol>\n<p><strong>\u5907\u6ce8\uff1a<\/strong><\/p>\n<p><a href=\"#_ftnref1\" name=\"_ftn1\">[1]<\/a> \u53c2\u8003\u6570\u636e\uff1aVertex: <a href=\"https:\/\/www.vrtx.com\/medicines\/cystic-fibrosis-facts-and-figures\/\">CF facts and figures<\/a>.<\/p>\n<p><a href=\"#_ftnref2\" name=\"_ftn2\">[2]<\/a> \u53c2\u8003\u6570\u636e\uff1aFDA: <a href=\"https:\/\/www.fda.gov\/industry\/commissioners-national-priority-voucher-cnpv-pilot-program\">Commissioner&#8217;s National Priority Voucher (CNPV) Pilot Program<\/a>.<br \/>\n<!-- \u4f5c\u8005\u4ecb\u7ecd\u5f00\u59cb --><\/p>\n<table border=\"0\" width=\"89%\" cellspacing=\"1\" cellpadding=\"8\">\n<tbody>\n<tr>\n<td bgcolor=\"#EFEFF8\">\n<table style=\"margin-bottom: 0px;\" border=\"0\" width=\"100%\" cellspacing=\"0\" cellpadding=\"3\">\n<tbody>\n<tr>\n<td class=\"Article_content_15px\" valign=\"top\" bgcolor=\"#DBDBEE\" width=\"13%\"><strong>\u4f5c\u8005\uff1a<\/strong><\/td>\n<td class=\"Article_content_15px\" bgcolor=\"#DBDBEE\" width=\"87%\">\u5434\u78a7\u5a25<\/td>\n<\/tr>\n<tr>\n<td class=\"Article_content_15px\" valign=\"top\"><strong>\u73b0\u4efb\uff1a<\/strong><\/td>\n<td class=\"Article_content_15px\">\u5317\u7f8e\u667a\u6743\u62a5\u4e3b\u7f16<\/td>\n<\/tr>\n<tr>\n<td class=\"Article_content_15px\" valign=\"top\" bgcolor=\"#DBDBEE\"><strong>\u5b66\u5386\uff1a<\/strong><\/td>\n<td class=\"Article_content_15px\" bgcolor=\"#DBDBEE\">\u653f\u6cbb\u5927\u5b66\u65b0\u95fb\u7814\u7a76\u6240<\/td>\n<\/tr>\n<tr>\n<td class=\"Article_content_15px\" valign=\"top\"><strong>\u7ecf\u5386\uff1a<\/strong><\/td>\n<td class=\"Article_content_15px\">\u5317\u7f8e\u667a\u6743\u62a5\u8d44\u6df1\u7f16\u8f91<br \/>\n\u9a85\u8baf\u7535\u5b50\u603b\u7ecf\u7406\u5ba4\u7279\u52a9<br \/>\n\u7ecf\u6d4e\u65e5\u62a5\u8d22\u7ecf\u7ec4\u8bb0\u8005<br \/>\n\u4e1c\u68ee\u8d2d\u7269\u603b\u7ecf\u7406\u5ba4\u7ecf\u8425\u4f01\u5212<\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n<\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n<p><!-- \u4f5c\u8005\u4ecb\u7ecd\u7ed3\u675f --><\/p>\n","protected":false},"excerpt":{"rendered":"<p>\u5c3d\u7ba1\u533b\u5b66\u8fdb\u5c55\u65e5\u65b0\u6708\u5f02\uff0c\u4f46\u5168\u7403\u4ecd\u670995%\u7684\u7f55\u89c1\u75be\u75c5\uff08rare diseases\uff09\u4ecd\u5904\u4e8e\u65e0\u836f\u53ef\u533b\u7684\u72b6\u6001\u3002\u4e3a\u4e86\u7167\u987e\u7f55 [&hellip;]<\/p>\n","protected":false},"author":10,"featured_media":13063,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[254,277],"tags":[1806,1808,1807,1809],"author_name1":[298],"class_list":{"0":"post-29019","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-254","8":"category-277","9":"tag-1806","10":"tag-1808","11":"tag-1807","12":"tag-1809","13":"author_name1--zh-hans"},"_links":{"self":[{"href":"https:\/\/naipnews.naipo.com\/zh-hans\/wp-json\/wp\/v2\/posts\/29019","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/naipnews.naipo.com\/zh-hans\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/naipnews.naipo.com\/zh-hans\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/naipnews.naipo.com\/zh-hans\/wp-json\/wp\/v2\/users\/10"}],"replies":[{"embeddable":true,"href":"https:\/\/naipnews.naipo.com\/zh-hans\/wp-json\/wp\/v2\/comments?post=29019"}],"version-history":[{"count":0,"href":"https:\/\/naipnews.naipo.com\/zh-hans\/wp-json\/wp\/v2\/posts\/29019\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/naipnews.naipo.com\/zh-hans\/wp-json\/wp\/v2\/media\/13063"}],"wp:attachment":[{"href":"https:\/\/naipnews.naipo.com\/zh-hans\/wp-json\/wp\/v2\/media?parent=29019"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/naipnews.naipo.com\/zh-hans\/wp-json\/wp\/v2\/categories?post=29019"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/naipnews.naipo.com\/zh-hans\/wp-json\/wp\/v2\/tags?post=29019"},{"taxonomy":"author_name1","embeddable":true,"href":"https:\/\/naipnews.naipo.com\/zh-hans\/wp-json\/wp\/v2\/author_name1?post=29019"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}