{"id":1247,"date":"2018-09-19T00:00:40","date_gmt":"2018-09-18T16:00:40","guid":{"rendered":"https:\/\/clarivate.com\/tw\/2018\/09\/19\/%e7%bd%95%e8%a6%8b%e7%97%85%e8%97%a5%e7%89%a9%e7%9a%84%e6%8c%91%e6%88%b0%e8%88%87%e6%a9%9f%e6%9c%83-%e6%9a%a8-%e5%b0%8d%e4%b8%ad%e5%9c%8b%e8%97%a5%e4%bc%81%e7%9a%84%e5%95%9f%e7%a4%ba\/?lang=zh-hant"},"modified":"2024-10-24T00:27:46","modified_gmt":"2024-10-24T00:27:46","slug":"challenges-and-opportunities-for-developing-orphan-drugs-in-china","status":"publish","type":"post","link":"https:\/\/clarivate.com\/life-sciences-healthcare\/zh\/blog\/challenges-and-opportunities-for-developing-orphan-drugs-in-china\/","title":{"rendered":"\u7f55\u898b\u75c5\u85e5\u7269\u7684\u6311\u6230\u8207\u6a5f\u6703"},"content":{"rendered":"<p>\u7b2c\u4e03\u5c46\u4e2d\u570b\u7f55\u898b\u75c5\u9ad8\u5cf0\u8ad6\u58c7\u65bc2018\u5e749\u670814-16\u65e5\u5728\u4e0a\u6d77\u8209\u8fa6\uff0c\u6b64\u8ad6\u58c7\u7684\u76ee\u7684\u5728\u65bc\u589e\u52a0\u5c0d\u65bc\u7f55\u898b\u75c5\u7684\u7406\u89e3\u548c\u8a8d\u8b58\u3002\u5728\u6b64\u4e4b\u524d\uff0c\u79d1\u777f\u552f\u5b89\u751f\u547d\u79d1\u5b78\u89e3\u6c7a\u65b9\u6848\u9867\u554f \u8d99\u5b87\u8587 \u65bc8\u6708\u521d\u7684 webinar\u4e2d\u5c0d\u7f55\u898b\u75c5\u53ca\u7f55\u898b\u75c5\u85e5\u7269\uff08\u4e5f\u6307\u5b64\u5152\u85e5\uff0c\u5373orphan drug\uff09\u5728\u4e2d\u570b\u7684\u767c\u5c55\u73fe\u6cc1\u9032\u884c\u8a0e\u8ad6\uff0c\u672c\u6587\u7bc0\u9304\u8a72\u6b21webinar\u5167\u5bb9\uff0c\u4e14\u7279\u5225\u611f\u8b1d\u79d1\u777f\u552f\u5b89\u751f\u547d\u79d1\u5b78\u8cc7\u6df1\u7de8\u8f2f Shyama Ghosh\u5973\u58eb\u5171\u540c\u5b8c\u6210\u672c\u6587\u3002<\/p>\n<p>&nbsp;<\/p>\n<h3><strong>\u5168\u7403\u4e3b\u8981\u85e5\u653f\u5e02\u5834\u7f55\u898b\u75c5\u85e5\u7269\u653f\u7b56<\/strong><\/h3>\n<p>\u5728\u4eca\u5e74(2018)\u76845\u670822\u65e5\uff0c\u4e2d\u570b\u516c\u5e03\u4e86\u7b2c\u4e00\u6279\u7f55\u898b\u75c5\u76ee\u9304\uff0c\u5176\u4e2d\u4e00\u5171\u5217\u51fa121\u500b\u7f55\u898b\u75c5\uff1b\u6b64\u8209\u88ab\u8996\u70ba\u4e2d\u570b\u653f\u5e9c\u91cd\u8996\u7f55\u898b\u75c5\u53ca\u76f8\u61c9\u7f55\u898b\u75c5\u85e5\u7269\u5e02\u5834\u9700\u6c42\u7684\u7b2c\u4e00\u6b65\u3002\u5982\u57161\u6240\u793a\uff0c\u5728\u4e2d\u570b\u76ee\u524d\u53ef\u7d93\u7531\u7279\u6b8a\u5be9\u6279\u7684\u5feb\u901f\u901a\u9053\u52a0\u901f\u7f55\u898b\u75c5\u85e5\u4e0a\u5e02 \uff08\u53c3\u80032017\u5e74126\u865f\u6587\uff09\uff0c\u5176\u4ed6\u4e3b\u8981\u7684\u85e5\u653f\u5e02\u5834\u5982\u7f8e\u570b\u3001\u6b50\u76df\u53ca\u6fb3\u6d32\u7b49\uff0c\u4e5f\u6709\u76f8\u61c9\u63a8\u52d5\u65b0\u85e5\uff0c\u7279\u5225\u662f\u7f55\u898b\u75c5\u85e5\u7269\u4e0a\u5e02\u7684\u7279\u6b8a\u5be9\u67e5\u53ca\u52a0\u901f\u6a5f\u5236\u3002<\/p>\n<div id=\"attachment_5672\" style=\"width: 650px\" class=\"wp-caption aligncenter\"><img loading=\"lazy\" decoding=\"async\" aria-describedby=\"caption-attachment-5672\" class=\"wp-image-5672 size-large\" src=\"http:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-1-1024x620.png\" alt=\"\u57161\uff1a\u5168\u7403\u4e3b\u8981\u5e02\u5834\u52a0\u901f\u85e5\u7269\u4e0a\u5e02\uff08\u542b\u7f55\u898b\u75c5\uff09\u7684\u7279\u6b8a\u5be9\u67e5\u6a5f\u5236\uff08\u8cc7\u6599\u4f86\u6e90\uff1aCortellis\u6cd5\u898f\u60c5\u5831\uff09\" width=\"640\" height=\"388\" srcset=\"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-1-1024x620.png 1024w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-1-300x182.png 300w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-1-768x465.png 768w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-1-1536x930.png 1536w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-1-66x40.png 66w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-1.png 1611w\" sizes=\"auto, (max-width: 640px) 100vw, 640px\" \/><p id=\"caption-attachment-5672\" class=\"wp-caption-text\">\u57161\uff1a\u5168\u7403\u4e3b\u8981\u5e02\u5834\u52a0\u901f\u85e5\u7269\u4e0a\u5e02\uff08\u542b\u7f55\u898b\u75c5\uff09\u7684\u7279\u6b8a\u5be9\u67e5\u6a5f\u5236\uff08\u8cc7\u6599\u4f86\u6e90\uff1a<a href=\"https:\/\/clarivate.com\/cortellis\/zh-hant\/solutions\/regulatory-intelligence-solutions\/\" target=\"_blank\" rel=\"noopener\">Cortellis\u6cd5\u898f\u60c5\u5831<\/a>\uff09<\/p><\/div>\n<p>&nbsp;<\/p>\n<p>\u88681\u5217\u8209\u4e86\u7f8e\u570b\u3001\u6b50\u6d32\u53ca\u65e5\u672c\u4e09\u500b\u4e3b\u8981\u5e02\u5834\u767c\u5c55\u7f55\u898b\u75c5\u85e5\u7269\u7684\u6cd5\u898f\u53ca\u9f13\u52f5\u63aa\u65bd\u3002<\/p>\n<table class=\"table\">\n<caption>\u88681\uff1a\u7f8e\u570b\u3001\u6b50\u6d32\u53ca\u65e5\u672c\u767c\u5c55\u7f55\u898b\u75c5\u85e5\u7269\u7684\u6cd5\u898f\u53ca\u9f13\u52f5\u63aa\u65bd \uff08\u8cc7\u6599\u4f86\u6e90\uff1a<a href=\"https:\/\/clarivate.com\/cortellis\/zh-hant\/solutions\/regulatory-intelligence-solutions\/\" target=\"_blank\" rel=\"noopener\">Cortellis \u6cd5\u898f\u60c5\u5831<\/a>\uff09<\/caption>\n<tbody>\n<tr>\n<th><strong>\u570b\u5bb6<\/strong><\/th>\n<th><strong>\u7f8e\u570b<\/strong><\/th>\n<th><strong>\u6b50\u76df<\/strong><\/th>\n<th><strong>\u65e5\u672c<\/strong><\/th>\n<\/tr>\n<tr>\n<th><strong>\u6cd5\u6e90\u57fa\u790e<\/strong><\/th>\n<td>21 CFR part 316<\/p>\n<p>\u5b64\u5152\u85e5\u6cd5\u6848<\/td>\n<td>Regulation (EC) No 141\/2000<\/td>\n<td>\u85e5\u4e8b\u6cd5\u7b2c77-2\u689d<\/td>\n<\/tr>\n<tr>\n<th><strong>\u4e3b\u7ba1\u6a5f\u95dc<\/strong><\/th>\n<td>FDA;<\/p>\n<p>\u5b64\u5152\u85e5\u7522\u54c1\u958b\u767c\u8fa6\u516c\u5ba4 (Office of Orphan Products &amp; Development)<\/td>\n<td>EMA;<\/p>\n<p>\u5b64\u5152\u7528\u85e5\u59d4\u54e1\u6703 (Committee for Orphan Medicinal Products, COMP)<\/td>\n<td>\u539a\u751f\u52de\u52d5\u7701 (MHLW);<\/p>\n<p>\u85e5\u4e8b\u98df\u54c1\u885b\u751f\u7701\u8b70\u6703 (Pharmaceutical Affairs &amp; Food Sanitation Council )<\/td>\n<\/tr>\n<tr>\n<th><strong>\u7f55\u898b\u75c5\u5b9a\u7fa9<\/strong><\/th>\n<td>\u5c11\u65bc20\u842c\u4eba<\/td>\n<td>\u4f4e\u65bc1\/2000\uff08\u7d04\u5c11\u65bc25\u842c\u4eba\uff09<\/td>\n<td>\u5c11\u65bc5\u842c\u4eba<\/td>\n<\/tr>\n<tr>\n<th><strong>\u653f\u7b56\u734e\u52f5<\/strong><\/th>\n<td>\n<ul>\n<li>7\u5e74\u7368\u8ce3<\/li>\n<li>\u7a05\u6536\u62b5\u51cf<\/li>\n<li>\u5feb\u901f\u901a\u9053\/ \u512a\u5148\u5be9\u8a55\/ \u52a0\u901f\u5be9\u8a55<\/li>\n<li>\u6280\u8853\u6307\u5c0e<\/li>\n<li>PDUFA\u8cbb\u7528\u8c41\u514d<\/li>\n<\/ul>\n<\/td>\n<td>\n<ul>\n<li>\u8a66\u9a57\u65b9\u6848\u5354\u52a9\/ \u79d1\u5b78\u6307\u5c0e(scientific advice)<\/li>\n<li>\u9032\u5165\u96c6\u4e2d\u7a0b\u5e8f\u5be9\u6279(centralized procedure access)<\/li>\n<li>10\u5e74\u7368\u8ce3<\/li>\n<li>\u8cbb\u7528\u51cf\u514d<\/li>\n<\/ul>\n<\/td>\n<td>\n<ul>\n<li>\u539a\u751f\u7701\u3001NIBIO\u3001PDMA\u7684\u6307\u5c0e<\/li>\n<li>\u7a05\u6536\u62b5\u51cf<\/li>\n<li>\u512a\u5148\u5be9\u8a55<\/li>\n<li>10\u5e74\u5e02\u5834\u7368\u8ce3<\/li>\n<li>\u570b\u5bb6\u5065\u5eb7\u4fdd\u96aa\u5b9a\u50f9\u512a\u60e0<\/li>\n<\/ul>\n<\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n<p>&nbsp;<\/p>\n<h3><strong>\u7f55\u898b\u75c5\u85e5\u7269\u5728\u7f8e\u570bFDA\u7684\u7372\u6279\u8da8\u52e2<\/strong><\/h3>\n<p>\u7f55\u898b\u75c5\u6216\u8a31\u6bd4\u60a8\u6211\u60f3\u50cf\u7684\u66f4\u666e\u904d\uff0c\u6839\u64da\u7d71\u8a08\uff0c\u76ee\u524d\u7d04\u6709 7000 \u7a2e\u7f55\u898b\u75c5<sup><a id=\"ref1\" href=\"#fn1\">1<\/a><\/sup>\uff0c\u5168\u7403\u7f79\u60a3\u7f55\u898b\u75c5\u7684\u4eba\u53e3\u7d04\u67093\u5104\u4eba\uff0c80%\u7684\u7f55\u898b\u75c5\u548c\u57fa\u56e0\u6709\u95dc\uff0c\u800c50%\u7684\u7f55\u898b\u75c5\u60a3\u8005\u662f\u5152\u7ae5\u3002\u7f8e\u570bFDA\u57281983\u5e74\u9812\u5e03\u4e86\u5b64\u5152\u85e5\u6cd5\u6848\u5f8c\uff0c\u8fc4\u4eca\u7d04\u6709450\u500b\u7f55\u898b\u75c5\u85e5\u7269\u7372\u51c6\u4e0a\u5e02\uff0c\u5176\u4e2d\u90e8\u5206\u7684\u85e5\u7269\u662f\u900f\u904e\u53d6\u5f97\u7f55\u898b\u75c5\u85e5\u7269\u8cc7\u683c\u4e0a\u5e02\uff0c\u800c\u6709\u90e8\u5206\u85e5\u7269\u5247\u662f\u4ee5\u9069\u61c9\u75c7\u62d3\u5c55\u7b49\u65b9\u5f0f\u7372\u5f97\u7f55\u898b\u75c5\u85e5\u7269\u8cc7\u683c\u3002\u57162\u662fFDA\u516c\u544a1983\u5e74\u4ee5\u4f86\u7f55\u898b\u75c5\u85e5\u7269\u7372\u51c6\u53d6\u5f97\u4e0a\u5e02\u8a31\u53ef\u7684\u7d71\u8a08\u7d50\u679c\uff0c\u6839\u64daFDA\u7684\u5206\u6790\uff0c2017\u5e74\u5171\u670977\u500b\u7f55\u898b\u75c5\u85e5\u7269\u7372\u51c6\u4e0a\u5e02\uff0c\u4e0a\u5e02\u6210\u529f\u7387\u9054100%\uff0c\u6db5\u84cb\u816b\u7624 (58%)\u3001\u5176\u4ed6 (10%)\u53ca\u4ee3\u8b1d\u75be\u75c5 (7%)\u3001\u795e\u7d93\u75be\u75c5 (7%) \u7b49\u4e3b\u8981\u6cbb\u7642\u9818\u57df\u3002\u7136\u800c\uff0c\u5373\u4f7f\u5728\u9ad8\u7372\u51c6\u7387\u7684\u60c5\u52e2\u4e4b\u4e0b\uff0c\u4ecd\u670995%\u7684\u7f55\u898b\u75c5\u7121\u85e5\u53ef\u6cbb<sup><a id=\"ref2\" href=\"#fn2\">2<\/a><\/sup>\u3002<\/p>\n<div id=\"attachment_5674\" style=\"width: 650px\" class=\"wp-caption aligncenter\"><img loading=\"lazy\" decoding=\"async\" aria-describedby=\"caption-attachment-5674\" class=\"wp-image-5674 size-large\" src=\"http:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-2-1024x479.png\" alt=\"\" width=\"640\" height=\"299\" srcset=\"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-2-1024x479.png 1024w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-2-300x140.png 300w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-2-768x359.png 768w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-2-86x40.png 86w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-2.png 1435w\" sizes=\"auto, (max-width: 640px) 100vw, 640px\" \/><p id=\"caption-attachment-5674\" class=\"wp-caption-text\">\u57162\uff1a1983\u5e74\u4ee5\u4f86FDA\u6279\u51c6\u7f55\u898b\u75c5\u85e5\u7269\u7684\u7d71\u8a08\u7d50\u679c \uff08\u8cc7\u6599\u4f86\u6e90\uff1aInsights into Rare Disease Drug Approval 2017, FDA\uff09<\/p><\/div>\n<p>&nbsp;<\/p>\n<p><a href=\"#_ftnref1\" name=\"_ftn1\"><\/a><\/p>\n<h3><strong> \u7f55\u898b\u75c5\u85e5\u7269\u7684\u81e8\u5e8a\u6210\u529f\u7387<\/strong><\/h3>\n<p>\u65e2\u7136\u5982FDA\u516c\u5e03\u7684\u57282017\u5e74\u7f55\u898b\u75c5\u85e5\u7269\u7372\u6279\u7684\u6210\u529f\u7387\u662f100%\uff0c\u90a3\u9ebd\u5728\u81e8\u5e8a\u8a66\u9a57\u4e0a\u7684\u6210\u529f\u7387\u5462\uff1f\u5f9e2018\u5e74Wong et al.\u7684\u7814\u7a76\u6307\u51fa\uff0c\u5f9e\u81e8\u5e8a\u4e00\u671f\u8d77\u7b97\u5230\u53d6\u5f97\u4e0a\u5e02\u8a31\u53ef\uff0c\u7f55\u898b\u75c5\u85e5\u7269\u5e73\u5747\u7684\u6210\u529f\u7387\u662f6.2%\uff0c\u7565\u4f4e\u65bc\u5176\u4ed6\u9069\u61c9\u75c7\u7528\u85e5\u7684\u6210\u529f\u7387(6.9%)\u3002\u82e5\u628a\u816b\u7624\u76f8\u95dc\u7684\u9069\u61c9\u75c7\u6392\u9664\u5f8c\uff0c\u7f55\u898b\u75c5\u85e5\u7269\uff08\u4e0d\u542b\u816b\u7624\u7528\u85e5\uff09\u7684\u6210\u529f\u7387\u5247\u589e\u52a0\u81f313.6% \uff08\u898b\u57163\uff09\u3002<\/p>\n<div id=\"attachment_5675\" style=\"width: 650px\" class=\"wp-caption aligncenter\"><img loading=\"lazy\" decoding=\"async\" aria-describedby=\"caption-attachment-5675\" class=\"wp-image-5675 size-large\" src=\"http:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-3-1024x483.png\" alt=\"\" width=\"640\" height=\"302\" srcset=\"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-3-1024x483.png 1024w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-3-300x141.png 300w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-3-768x362.png 768w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-3-85x40.png 85w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-3.png 1194w\" sizes=\"auto, (max-width: 640px) 100vw, 640px\" \/><p id=\"caption-attachment-5675\" class=\"wp-caption-text\">\u57163\uff1a\u81e8\u5e8a\u8a66\u9a57\u7684\u6210\u529f\u7387(\u6240\u6709\u9069\u61c9\u75c7\u3001\u9069\u7528\u7f55\u898b\u75c5\u7684\u7f55\u898b\u75c5\u85e5\u7269\u4ee5\u53ca\u6392\u9664\u816b\u7624\u7528\u85e5\u7684\u7f55\u898b\u75c5\u85e5\u7269) (\u5716\u8868\u4f9d\u64daWong et al., Biostatistics (2018) 00, 00, pp. 1\u201314\u88fd\u4f5c)<\/p><\/div>\n<p>&nbsp;<\/p>\n<h3><strong>\u7f55\u898b\u75c5\u85e5\u7269\u5931\u6557\u539f\u56e0\u5206\u6790<\/strong><\/h3>\n<p>\u57164\u63cf\u7e6a\u4e86\u5728\u6b50\u6d32\u7f55\u898b\u75c5\u85e5\u7269\u672a\u80fd\u53d6\u5f97\u4e0a\u5e02\u8a31\u53ef\uff0c\u6216\u5728\u7814\u767c\u904e\u7a0b\u4e2d\u88ab\u653e\u68c4\u5c0e\u81f3\u4e2d\u65b7\u7684\u539f\u56e0\uff08\u57164\u5de6\uff09\uff0c\u4ee5\u53ca\u9032\u4e00\u6b65\u5206\u6790\u56e0\u5b89\u5168\u6027\/\u6709\u6548\u6027\u5931\u6557\u7684\u7f55\u898b\u75c5\u85e5\u7269\u7684\u6cbb\u7642\u9818\u57df\uff08\u57164\u53f3\uff09\u3002\u8207\u5176\u4ed6\u5728\u7814\u767c\u904e\u7a0b\u4e2d\u5931\u6557\u7684\u85e5\u7269\u76f8\u4f3c\uff0c\u7f55\u898b\u75c5\u85e5\u7269\u5931\u6557\u7684\u4e3b\u8981\u539f\u56e0\u9084\u662f\u5728\u5b89\u5168\u6027\/\u6709\u6548\u6027(efficacy\/safety issue)\u3002\u5728\u672a\u80fd\u53d6\u5f97\u85e5\u8b49\u7684\u7f55\u898b\u75c5\u85e5\u7269\u7576\u4e2d\uff0c\u524d\u4e09\u5927\u7684\u5931\u6557\u539f\u56e0\u5206\u5225\u662f\uff1a\u5b89\u5168\u6027\/\u6709\u6548\u6027(52%)\u3001\u6578\u64da\u4e0d\u5145\u5206(insufficient data) (24%)\u4ee5\u53ca\u54c1\u8cea(quality issue)(14%)\uff1b50%\u7684\u5931\u6557\u662f\u767c\u751f\u5728\u81e8\u5e8a\u4e8c\u671f\u3002\u9032\u4e00\u6b65\u5206\u6790\u56e0\u5b89\u5168\u6027\/\u6709\u6548\u6027\u5931\u6557\u7684\u7f55\u898b\u75c5\u85e5\u7269\u7684\u6cbb\u7642\u9818\u57df\uff0c\u5206\u5225\u662f\u816b\u7624\u3001 \u907a\u50b3\u6027\u4ee3\u8b1d\u75be\u75c5(inborn errors of metabolism diseases)\u3001\u795e\u7d93\u53ca\u7cbe\u795e\u75be\u75c5\u3001\u4ee5\u53ca\u5fc3\u8840\u7ba1\u53ca\u547c\u5438\u76f8\u95dc\u75be\u75c5\u3002<\/p>\n<div id=\"attachment_5676\" style=\"width: 650px\" class=\"wp-caption aligncenter\"><img loading=\"lazy\" decoding=\"async\" aria-describedby=\"caption-attachment-5676\" class=\"wp-image-5676 size-large\" src=\"http:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-4-1024x431.png\" alt=\"\" width=\"640\" height=\"269\" srcset=\"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-4-1024x431.png 1024w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-4-300x126.png 300w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-4-768x323.png 768w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-4-1536x646.png 1536w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-4-95x40.png 95w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-4.png 1787w\" sizes=\"auto, (max-width: 640px) 100vw, 640px\" \/><p id=\"caption-attachment-5676\" class=\"wp-caption-text\">\u57164\uff1a\u5728\u6b50\u6d32\u7f55\u898b\u75c5\u85e5\u7269\u672a\u80fd\u53d6\u5f97\u4e0a\u5e02\u8a31\u53ef\u6216\u5728\u7814\u767c\u904e\u7a0b\u4e2d\u88ab\u653e\u68c4\u5c0e\u81f3\u4e2d\u65b7\u7684\u539f\u56e0(\u57164\u5de6)\uff0c\u4ee5\u53ca\u9032\u4e00\u6b65\u5206\u6790\u56e0\u5b89\u5168\u6027\/\u6709\u6548\u6027\u5931\u6557\u7684\u7f55\u898b\u75c5\u85e5\u7269\u7684\u6cbb\u7642\u9818\u57df(\u57164\u53f3) (\u8cc7\u6599\u4f86\u6e90: Source: BMJ Open 2017;7:e017358)<\/p><\/div>\n<p>&nbsp;<\/p>\n<h3><strong>\u7814\u767c\u6210\u672c\u53ca\u7f55\u898b\u75c5\u6cbb\u7642\u6210\u672c<\/strong><\/h3>\n<p>\u7f55\u898b\u75c5\u85e5\u7269\u8207\u975e\u7f55\u898b\u75c5\u85e5\u7269\u7684\u7814\u767c\u6210\u672c\u5dee\u8ddd\u5728\u65bc\u81e8\u5e8a\u8a66\u9a57\uff0c\u5982\u88682\u6240\u793a\uff0c\u7f55\u898b\u75c5\u85e5\u7269\u7684\u81e8\u5e8a\u8a66\u9a57\u6210\u672c\u5728\u6263\u9664\u7a05\u52d9\u62b5\u51cf\u524d\u662f\u975e\u7f55\u898b\u75c5\u85e5\u7269\u768450% <sup><a id=\"ref3\" href=\"#fn3\">3<\/a><\/sup>\uff1bLinda Martin\u7684\u7814\u7a76\u6307\u51fa\uff0c\u57f7\u884c\u975e\u7f55\u898b\u75c5\u85e5\u7269\u5168\u7403\u81e8\u5e8a\u8a66\u9a57\u7684\u6210\u672c\u4e2d\u4f4d\u6578\u7d04\u662f3,340\u842c\u7f8e\u5143<sup><a id=\"ref4\" href=\"#fn4\">4<\/a><\/sup>\uff0c\u56e0\u6b64\uff0c\u82e5\u8003\u616e\u81e8\u5e8a\u8a66\u9a57\u6210\u672c\u53ca\u7a05\u52d9\u62b5\u51cf\uff0c\u7f55\u898b\u75c5\u85e5\u7269\u7684\u81e8\u5e8a\u8a66\u9a57\u6210\u672c\u4e2d\u4f4d\u6578\u7d04\u662f\u5728835\u842c\u7f8e\u5143\u3002<\/p>\n<div id=\"attachment_5677\" style=\"width: 650px\" class=\"wp-caption aligncenter\"><img loading=\"lazy\" decoding=\"async\" aria-describedby=\"caption-attachment-5677\" class=\"wp-image-5677 size-large\" src=\"http:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-5-1024x435.png\" alt=\"\" width=\"640\" height=\"272\" srcset=\"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-5-1024x435.png 1024w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-5-300x127.png 300w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-5-768x326.png 768w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-5-1536x652.png 1536w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-5-94x40.png 94w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-5.png 1714w\" sizes=\"auto, (max-width: 640px) 100vw, 640px\" \/><p id=\"caption-attachment-5677\" class=\"wp-caption-text\">\u88682\uff1a\u7814\u767c\u7f55\u898b\u75c5\u85e5\u7269\u53ca\u975e\u7f55\u898b\u75c5\u85e5\u7269\u7684\u6210\u672c\u6bd4\u8f03 (\u672c\u8868\u57fa\u65bc Sara Smith\u7684\u8ad6\u6587\u53caNature Reviews Drug Discovery volume 16, pages 381\u2013382 (2017)\u6574\u7406)<\/p><\/div>\n<p>&nbsp;<\/p>\n<p>\u88683\u5217\u8209\u4e86\u90e8\u5206\u6cbb\u7642\u56e0\u57fa\u56e0\u7570\u5e38\u5c0e\u81f4\u7684\u907a\u50b3\u6027\u7f55\u898b\u75c5\u7684\u85e5\u7269\u6cbb\u7642\u6210\u672c\uff08\u4ee5\u6bcf\u500b\u75c5\u60a3\u6bcf\u5e74\u7684\u7528\u85e5\u8cbb\u7528\u70ba\u8a08\u7b97\u57fa\u790e\uff09\uff0c\u5728\u7f8e\u570b\u7684\u5e73\u5747\u6cbb\u7642\u6210\u672c\u662f147,308\u7f8e\u5143\uff0c\u800c\u975e\u7f55\u898b\u75c5\u60a3\u8005\u7684\u7528\u85e5\u6210\u672c\u662f30,708\u7f8e\u5143<sup><a id=\"ref5\" href=\"#fn5\">5<\/a><\/sup>\u3002\u96d6\u7136\u7f55\u898b\u75c5\u60a3\u8005\u7684\u6578\u91cf\u6709\u9650\uff0c\u82e5\u5c07\u90e8\u5206\u7f55\u898b\u75c5\u7528\u85e5\u7684\u9ad8\u85e5\u50f9\u7d0d\u5165\u8003\u91cf\uff0c\u9019\u4e9b\u85e5\u7269\u7684\u4ecd\u80fd\u9032\u5165\u91cd\u78c5\u85e5\u7269 (blockbuster)\u540d\u55ae\uff08\u5e74\u92b7\u552e\u984d\u9054\u621010\u5104\u7f8e\u5143\uff09\uff0c\u4f8b\u5982\u7531Alexion Pharmaceuticals\u958b\u767c\u53ca\u92b7\u552e\u7684Soliris\u00ae\u3002<\/p>\n<div id=\"attachment_5678\" style=\"width: 650px\" class=\"wp-caption aligncenter\"><img loading=\"lazy\" decoding=\"async\" aria-describedby=\"caption-attachment-5678\" class=\"wp-image-5678 size-large\" src=\"http:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-6-1024x671.png\" alt=\"\" width=\"640\" height=\"419\" srcset=\"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-6-1024x671.png 1024w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-6-300x197.png 300w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-6-768x504.png 768w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-6-61x40.png 61w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-6.png 1176w\" sizes=\"auto, (max-width: 640px) 100vw, 640px\" \/><p id=\"caption-attachment-5678\" class=\"wp-caption-text\">\u88683\uff1a\u90e8\u5206\u56e0\u57fa\u56e0\u7570\u5e38\u5c0e\u81f4\u7684\u907a\u50b3\u6027\u7f55\u898b\u75c5\u85e5\u7269\u7684\u6cbb\u7642\u6210\u672c(\u4ee5\u6bcf\u500b\u75c5\u60a3\u6bcf\u5e74\u7684\u7528\u85e5\u8cbb\u7528\u70ba\u8a08\u7b97\u57fa\u790e) (\u8a3b6)<\/p><\/div>\n<p>&nbsp;<\/p>\n<h3><strong>\u7f55\u898b\u75c5\u85e5\u7269\u4e2d\u7684\u91cd\u78c5\u85e5\u7269<\/strong><\/h3>\n<p>\u5168\u7403\u7f55\u898b\u75c5\u85e5\u7269\u7684\u5e74\u92b7\u552e\u984d\u4f30\u8a08\u5c07\u5f9e2018\u5e74\u76841,380\u5104\u7f8e\u5143\u6210\u9577\u81f32024\u5e74\u76842,620\u5104\u7f8e\u5143\uff08\u5e74\u8907\u5408\u6210\u9577\u7387\u70ba11.38%\uff09<sup><a id=\"ref7\" href=\"#fn7\">7<\/a><\/sup>\uff0c\u90e8\u5206\u7f55\u898b\u75c5\u85e5\u7269\u751a\u81f3\u53ef\u4ee5\u9032\u5165\u5e74\u92b7\u552e\u984d\u905410\u5104\u7f8e\u5143\u7684\u91cd\u78c5\u85e5\u7269\u540d\u55ae\uff0c\u4f8b\u5982\u7528\u65bc\u6cbb\u7642\u514b\u9686\u6c0f\u75c7(Crohn\u2019s disease)\u7684Remicade\u00ae\u3001\u6cbb\u7642\u591a\u767c\u6027\u786c\u5316\u75c7(multiple sclerosis)\u7684Copaxone\u00ae\u4ee5\u53ca\u7528\u65bc\u6cbb\u7642\u975e\u4f55\u6770\u91d1\u6c0f\u6dcb\u5df4\u7624(non-Hodgkin\u2019s B-cell lymphoma)\u7684Rituxan\u00ae\uff0c\u5176\u4e2d\u6709\u90e8\u5206\u85e5\u7269\u81ea\u9996\u6b21\u4e0a\u5e02\u8ddd\u4eca\u5df2\u8d85\u904e20\u5e74\u4ecd\u80fd\u9ad8\u5c45\u91cd\u78c5\u85e5\u7269\u4e4b\u4f4d\u3002\u5982\u88684\u6240\u793a\uff0c\u4e09\u7a2e\u91cd\u78c5\u85e5\u7269\u4e2d\u7684Remicade\u00ae\u548cRituxan\u00ae\u4e5f\u5728\u4e2d\u570b\u4e0a\u5e02\u3002<\/p>\n<p>&nbsp;<\/p>\n<table class=\"table\">\n<caption>\u88684\uff1a\u91cd\u78c5\u7f55\u898b\u75c5\u85e5\u7269\u53ca\u5176\u76f8\u61c9\u7684\u9069\u61c9\u75c7\u3001\u9996\u6b21\u4e0a\u5e02\u6642\u9593\u30012017\u5e74\u7684\u92b7\u552e\u984d\u4ee5\u53ca\u5728\u4e2d\u570b\u4e0a\u5e02\u8207\u5426 \uff08\u8cc7\u6599\u4f86\u6e90\uff1a<a href=\"https:\/\/clarivate.com\/cortellis\/zh-hant\/solutions\/competitive-intelligence-and-analytics\/\" target=\"_blank\" rel=\"noopener\">Cortellis\u7af6\u722d\u60c5\u5831<\/a>\uff09<\/caption>\n<tbody>\n<tr>\n<td><strong>Drug name<\/strong><\/td>\n<td><strong>Remicade\u00ae<\/strong><\/td>\n<td><strong>Copaxone\u00ae<\/strong><\/td>\n<td><strong>Rituxan\u00ae<\/strong><\/td>\n<\/tr>\n<tr>\n<td style=\"width: 385.6px\"><strong>First approved indication (US)<\/strong><\/td>\n<td>Crohn&#8217;s disease<\/td>\n<td>multiple sclerosis<\/td>\n<td>non-Hodgkin&#8217;s B-cell<\/p>\n<p>lymphoma<\/td>\n<\/tr>\n<tr>\n<td><strong>Launched year (US)<\/strong><\/td>\n<td>1998<\/td>\n<td>1997<\/td>\n<td>1994<\/td>\n<\/tr>\n<tr>\n<td style=\"width: 385.6px\"><strong>Sales in 2017 ($ M)<\/strong><\/td>\n<td>7,755<\/td>\n<td>3,801<\/td>\n<td>7,504<\/td>\n<\/tr>\n<tr>\n<td><strong>Launched in China, Year, Indication<\/strong><\/td>\n<td>Yes, 2007,<\/p>\n<p>Crohn\u2019s disease<\/td>\n<td>No<\/td>\n<td>Yes, 2002,<br \/>\nFollicle center lymphoma<\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n<p>&nbsp;<\/p>\n<p>\u53e6\u5916\uff0c\u6839\u64da <a href=\"https:\/\/clarivate.com\/zh-hant\/news\/news-releases-2018-0323\/\">Drugs to Watch 2018<\/a> \u7684\u7d71\u8a08\uff0c\u7f85\u6c0f\u6700\u8fd1\u525b\u88abFDA\u6279\u51c6\u4e0a\u5e02\u3001\u7528\u65bc\u6cbb\u7642A\u578b\u8840\u53cb\u75c5\u7684Hemlibra\u00ae\uff0c\u57282019\u5e74\u5c07\u53ef\u9054\u5230\u91cd\u78c5\u7d1a\u5225\uff0c\u9810\u8a08\u5e74\u92b7\u552e\u984d\u905414.57\u5104\u7f8e\u5143\u3002\u53e6\u5916\uff0c\u7528\u65bc\u6cbb\u7642\u907a\u50b3\u6027\u5bb6\u65cf\u6027\u8f49\u7532\u72c0\u817a\u7d20\u985e\u6fb1\u7c89\u86cb\u767d\u8b8a\u6027 (hereditary transthyretin-mediated amyloidosis, hATTR amyloidosis)\u7684Patisiran\uff0c\u662f\u9996\u500b(first-in-class) RNA\u5e72\u64fe(RNAi)\u85e5\u7269 \uff0c\u525b\u65bc2018\u5e748\u6708\u7531FDA\u6279\u51c6\u4e0a\u5e02\uff0c\u4f30\u8a08\u57282021\u5e74\u53ef\u9054\u91cd\u78c5\u7d1a\u5225\uff0c\u5e74\u92b7\u552e\u984d11.04\u5104\u7f8e\u5143\u3002<\/p>\n<p>&nbsp;<\/p>\n<h3><strong>\u6b66\u7530\u88fd\u85e5\u5de8\u984d\u6536\u8cfc\u7f55\u898b\u75c5\u85e5\u7269\u9f8d\u982d<\/strong><strong>Shire<\/strong><\/h3>\n<p>\u57282018\u5e74\u521d\uff0c\u65e5\u672c\u7684\u6b66\u7530\u88fd\u85e5 (Takeda) \u958b\u50f9623\u5104\u7f8e\u5143\u6536\u8cfc\u7f55\u898b\u75c5\u85e5\u7269\u9f8d\u982d Shire\u3002Shire \u76ee\u524d\u5728\u7f55\u898b\u75c5\u5171\u670933\u500b\u5df2\u4e0a\u5e02\u7684\u85e5\u7269\u4e14\u5176\u7522\u54c1\u7dda\u4e0a\u9084\u6709\u8d85\u904e100\u500b\u85e5\u7269\u5728\u7814\u3002\u900f\u904e Cortellis\u7684\u865b\u64ec\u4f75\u8cfc (virtual merger) \u5206\u6790\uff0c\u5728\u6b66\u7530\u4f75\u8cfcShire\u5f8c\u53ef\u4ee5\u5f3a\u5316\u81e8\u5e8a\u5f8c\u671f\u7684\u7523\u54c1\u7dda\uff0c\u4e14\u5408\u4f75\u5f8c\u7684\u516c\u53f8\u5c07\u5115\u8eab\u5168\u7403\u524d10\u5927\u85e5\u5ee0\u4e4b\u5217\u3002<\/p>\n<div id=\"attachment_5680\" style=\"width: 650px\" class=\"wp-caption aligncenter\"><img loading=\"lazy\" decoding=\"async\" aria-describedby=\"caption-attachment-5680\" class=\"wp-image-5680 size-large\" src=\"http:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-7-1024x735.png\" alt=\"\u57165\uff1a\u6b66\u7530\u4f75\u8cfcShire\u5f8c\u7684\u7522\u54c1\u7dda (\u8cc7\u6599\u4f86\u6e90: Cortellis\u7af6\u722d\u60c5\u5831)\" width=\"640\" height=\"459\" srcset=\"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-7-1024x735.png 1024w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-7-300x215.png 300w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-7-768x551.png 768w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-7-56x40.png 56w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-7.png 1034w\" sizes=\"auto, (max-width: 640px) 100vw, 640px\" \/><p id=\"caption-attachment-5680\" class=\"wp-caption-text\">\u57165\uff1a\u6b66\u7530\u4f75\u8cfcShire\u5f8c\u7684\u7522\u54c1\u7dda (\u8cc7\u6599\u4f86\u6e90: Cortellis\u7af6\u722d\u60c5\u5831)<\/p><\/div>\n<p>&nbsp;<\/p>\n<h3><strong>\u7f55\u898b\u75c5\u85e5\u7269\u5728\u4e2d\u570b\u7684\u53ef\u53ca\u6027<\/strong><\/h3>\n<p>\u516c\u5e03\u7b2c\u4e00\u6279\u7f55\u898b\u75c5\u540d\u55ae\u958b\u5553\u4e86\u5728\u4e2d\u570b\u767c\u5c55\u7f55\u898b\u75c5\u7528\u85e5\u7684\u7b2c\u4e00\u6247\u9580\uff0c\u5728\u4e2d\u570b\u53d6\u5f97\u5df2\u4e0a\u5e02\u7f55\u898b\u75c5\u85e5\u7269\u4e5f\u662f\u5728\u6b64\u9818\u57df\u767c\u5c55\u7684\u91cd\u8981\u8b70\u984c\u3002\u6839\u64daGong et al.\u57282012\u5e74\u7684\u8abf\u67e5\u5831\u544a <sup><a id=\"ref8\" href=\"#fn8\">8<\/a><\/sup> \u4e2d\u6307\u51fa\uff0c\u5728\u7f8e\u570b\u3001\u65e5\u672c\u3001\u6b50\u6d32\u5df2\u6838\u51c6\u7684408\u500b\u7f55\u898b\u75c5\u85e5\u7269\u4e2d\uff0c\u50c5\u670940.4% \uff08408\u500b\u7f55\u898b\u75c5\u85e5\u7269\u4e2d\u7684165\u500b\uff09\u53ef\u4ee5\u5728\u8abf\u67e5\u4e2d\u768424\u9593\u4e09\u7532\u91ab\u9662\u8cfc\u5f97\u85e5\u7269\uff08\u898b\u57166\uff09\uff0c\u5176\u4e2d18.9%\u662f\u539f\u5ee0\u85e5\uff0c45.8%\u662f\u5b78\u540d\u85e5\uff0c\u53c8165\u500b\u4e2d\u7684120\u500b\u7f55\u898b\u75c5\u85e5\u7269\u53ef\u7531\u91ab\u4fdd\u7d66\u4ed8\uff1b\u5e73\u5747\u7684\u6cbb\u7642\u8cbb\u7528\u70ba4,843.5\u7f8e\u5143\uff0c\u76f8\u7576\u65bc\u4e2d\u7b49\u6536\u5165\u7684\u57ce\u5e02\u5c45\u6c11505.6\u5929\u7684\u6536\u5165\u3002\u6839\u64daGong\u7684\u8abf\u67e5\uff0c\u5927\u90e8\u5206\u53ef\u53d6\u5f97\u7684\u7f55\u898b\u75c5\u85e5\u7269\u4ecd\u662f\u8d85\u904e\u4e00\u822c\u6c11\u773e\u7684\u8ca0\u8377\u3002<\/p>\n<div id=\"attachment_5681\" style=\"width: 947px\" class=\"wp-caption aligncenter\"><img loading=\"lazy\" decoding=\"async\" aria-describedby=\"caption-attachment-5681\" class=\"wp-image-5681 size-full\" src=\"http:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-8.png\" alt=\"\" width=\"937\" height=\"620\" srcset=\"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-8.png 937w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-8-300x199.png 300w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-8-768x508.png 768w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-8-60x40.png 60w\" sizes=\"auto, (max-width: 937px) 100vw, 937px\" \/><p id=\"caption-attachment-5681\" class=\"wp-caption-text\">\u57166\uff1a\u7f8e\u570b\u3001\u6b50\u6d32\u4ee5\u53ca\u65e5\u672c\u5df2\u6838\u51c6\u4e0a\u5e02\u7684\u7f55\u898b\u75c5\u85e5\u7269\u5728\u4e2d\u570b\u7684\u53ef\u53ca\u6027 (\u672c\u5716\u4f9d\u64daGong et al.\u7684\u7814\u7a76\u6574\u7406\uff0c\u53ef\u53ca\u6027\u4f9dGong et al. \u57282012\u5e74\u8abf\u67e5\u768424\u5bb6\u4e09\u7532\u91ab\u9662\uff0c\u5728\u8abf\u67e5\u7576\u6642\u53ef\u8cfc\u5f97\u85e5\u7269\u505a\u70ba\u53ef\u53ca\u6027\u7684\u8a08\u7b97\u57fa\u790e)<\/p><\/div>\n<p>&nbsp;<\/p>\n<p>\u767c\u751f\u5728\u4e2d\u570b\u7684\u4e3b\u8981\u7f55\u898b\u75c5\u5305\u62ec\u82ef\u916e\u5c3f\u75c7 (phenylketonuria), (thalassemia)\u3001\u91cd\u578b\u6d77\u6d0b\u6027\u8ca7\u8840 (thalassemia)\u3001\u6210\u9aa8\u4e0d\u5168\u75c7 (osteogenesis imperfecta)\u3001\u9ad8\u8840\u6c28\u75c7 (hyperammonemia)\u3001\u6709\u6a5f\u9178\u8840\u75c7 (organic academia) \u4ee5\u53ca\u5a01\u723e\u68ee\u6c0f\u75c7 (Wilson&#8217;s disease)\u3001\u91cd\u75c7\u808c\u7121\u529b (Myasthenia gravis) \u3001\u7d50\u7bc0\u6027\u786c\u5316\u75c7 (tuberous sclerosis complex)\u3001\u767d\u5316\u75c5(albinism)\u7b49\u3002\u5176\u4ed6\u7684\u7f55\u898b\u75c5\u5982\u8840\u53cb\u75c5 (hemophilia)\u3001\u9f90\u8c9d\u6c0f\u75c7 (Pompe disease) \u548c\u56e0\u91a3\u8102\u8cea\u7121\u6cd5\u5206\u89e3\u800c\u5806\u7a4d\u5728\u9ad4\u5167\u9020\u6210\u7684\u6cd5\u5e03\u745e\u6c0f\u75c7 (Fabry disease)\uff0c\u5176\u4e2d\u5728\u4e2d\u570b\u88ab\u8a3a\u65b7\u51fa\u6cd5\u5e03\u745e\u6c0f\u75c7\u7684\u75c5\u60a3\u4e0d\u52305%\u3002\u767c\u751f\u5728\u5152\u7ae5\u7684\u5247\u6709\u96c4\u6fc0\u7d20\u4e0d\u654f\u611f\u75c7 (androgen insensitivity syndrome)\u3001\u808b\u819c\u80ba\u6bcd\u7d30\u80de\u7624 (pleuropulmonary blastoma)\u3001\u6a6b\u7d0b\u808c\u8089\u7624(rhabdomyosarcoma\uff0c\u542b\u817a\u6ce1\u578b(alveolar)\u53ca\u80da\u80ce\u578b(embryonal)\u4e8c\u7a2e\u5b50\u578b)\u3001Blau\u75c7\u5019\u7fa4 (Blau syndrome)\u3001Bardet-Biedl\u6c0f\u75c7\u5019\u7fa4(Bardet-Biedl Syndrome) \u4ee5\u53ca\u5148\u5929\u6027\u773c\u7403\u9707\u986b(Infantile nystagmus syndrome)\u2026\u7b49\u5152\u79d1\u7f55\u898b\u75c5\u3002<\/p>\n<p>\u7f55\u898b\u75c5\u6216\u6cd5\u5f8b\u5b9a\u7fa9\u4e0a\u7684\u5b64\u5152\u75be\u75c5 (orphan disease) \u7d04\u5f71\u97ff\u4e2d\u570b1600\u842c\u4eba\uff0c\u4f30\u8a08\u7684\u767c\u75c5\u7387\u7d04\u662f\u6bcf30-50\u842c\u4eba\u7d04\u67091\u540d\u75c5\u60a3\uff0c\u76ee\u524d\u5728\u4e2d\u570b\u7684\u516c\u5e03\u7f55\u898b\u75c5\u6578\u64da\u5c1a\u4e0d\u5b8c\u6574\uff0c\u4e5f\u6c92\u6709\u8db3\u591f\u7684\u8ca1\u52d9\u652f\u63f4\u7f55\u898b\u75c5\u6cbb\u7642\uff0c\u5176\u4e2d\u50c5\u6709\u90e8\u5206\u7684\u60a3\u8005\u80fd\u5f97\u5230\u6cbb\u7642\u3002\u76ee\u524d\u4e2d\u570b\u570b\u5bb6\u85e5\u54c1\u76e3\u7763\u7ba1\u7406\u5c40 (NMPA) \u5c1a\u672a\u516c\u5e03\u95dc\u65bc\u7f55\u898b\u75c5\u5728\u4e2d\u570b\u7684\u7279\u5225\u6cd5\u53ca\u76f8\u61c9\u689d\u6b3e\uff0c\u4f7f\u5f97\u7f55\u898b\u75c5\u60a3\u8005\u63a5\u53d7\u6cbb\u7642\u7684\u53ef\u53ca\u6027\u53d7\u5230\u5f71\u97ff\u3002<\/p>\n<p>\u591a\u6578\u60c5\u51b5\u4e0b\uff0c\u4e3b\u8981\u85e5\u653f\u5e02\u5834\u7684\u76e3\u7ba1\u55ae\u4f4d\u754c\u5b9a\u7f55\u898b\u75c5\u6703\u7528\u5169\u500b\u6a19\u6e96\uff0c\u4e00\u500b\u662f\u7d55\u5c0d\u6578\u91cf\uff0c\u6bd4\u5982\u4f4e\u65bc25\u842c\u4eba\u53e3\uff0c\u5c31\u53ef\u4ee5\u5b9a\u7fa9\u7f55\u898b\u75c5\u3002\u4e00\u500b\u662f\u767e\u5206\u6bd4\uff0c\u6bd4\u5982\u4f4e\u65bc\u842c\u5206\u4e4b\u4e00\u7b49\uff0c\u505a\u70ba\u7f55\u898b\u75c5\u7684\u57fa\u790e\u5b9a\u7fa9\u3002<\/p>\n<p>\u7136\u800c\uff0c\u4e2d\u570b\u6709\u845713\u5104\u7684\u4eba\u53e3\u57fa\u6578\uff0c\u9019\u4e8c\u500b\u6a19\u6e96\uff0c\u7121\u8ad6\u7528\u54ea\u500b\uff0c\u5c07\u90fd\u662f\u4e00\u500b\u503c\u5f97\u95dc\u6ce8\u7684\u6578\u5b57\uff0c\u9019\u6703\u5c0d\u652f\u4ed8\u90e8\u9580\uff08\u91ab\u4fdd\u6216\u8005\u4fdd\u96aa\u516c\u53f8\uff09\u5e36\u4f86\u5de8\u5927\u8ca0\u64d4\u3002\u7b2c\u4e00\u6279\u7f55\u898b\u75c5\u76ee\u9304\u7684121\u7f55\u898b\u75c5\uff0c\u5b83\u65e2\u4e0d\u770b\u767c\u75c5\u7387\u60a3\u75c5\u7387\uff0c\u4e5f\u4e0d\u770b\u7d55\u5c0d\u6578\u5b57\uff0c\u800c\u662f\u5efa\u8a2d\u6027\u7d66\u51fa\u4e00\u500b\u76ee\u9304\uff0c\u76f8\u4fe1\u662f\u5fb5\u6c42\u4e86\u81e8\u5e8a\u91ab\u5e2b\u9700\u6c42\u610f\u898b\u5f8c\u7d66\u51fa\u7684\u3002\u8003\u91cf\u4e2d\u570b\u81ea\u8eab\u7684\u9996\u8981\u9700\u6c42\uff0c\u7387\u5148\u63a8\u9032\u4e00\u6279\u7f55\u898b\u75c5\u7684\u7814\u767c\u548c\u6cbb\u7642\u3002<\/p>\n<p>&nbsp;<\/p>\n<h3><strong>\u975e\u816b\u7624\u7f55\u898b\u75c5\u7528\u85e5\u5728\u4e2d\u570b\u7684\u81e8\u5e8a\u8a66\u9a57\u8da8\u52e2<\/strong><\/h3>\n<p>\u89c0\u5bdf\u7b2c\u4e00\u6279\u7f55\u898b\u75c5\u76ee\u9304\u7684121\u500b\u7f55\u898b\u75c5\u662f\u5c6c\u65bc\u975e\u816b\u7624\u7684\u7f55\u898b\u75c5\uff0c\u8acb\u53c3\u8003\u57167\u548c\u57168\uff0c\u9032\u4e00\u6b65\u5206\u6790\u8fd110\u5e74\u5728\u4e2d\u570b\u7684\u81e8\u5e8a\u8a66\u9a57\u53ca\u975e\u816b\u7624\u7f55\u898b\u75c5\u7528\u85e5\u7684\u81e8\u5e8a\u8a66\u9a57\u8da8\u52e2\u3002\u5e73\u5747\u800c\u8a00\uff0c\u5728\u4e2d\u570b\u7684\u57f7\u884c\u7684\u81e8\u5e8a\u8a66\u9a57\u4e2d\uff0c4.3%\u7232\u975e\u816b\u7624\u7f55\u898b\u75c5\u7684\u81e8\u5e8a\u8a66\u9a57\u3002\u6392\u9664\u81e8\u5e8a\u8a66\u9a57\u671f\u5225\u7121\u6cd5\u78ba\u5b9a\u7684\u6578\u91cf\u5f8c\uff0c\u7576\u4e2d\u53c8\u4ee5\u81e8\u5e8a\u4e8c\u671f\uff08\u901a\u5e38\u88ab\u8996\u70ba\u6a1e\u7d10\u8a66\u9a57 pivotal trials\uff09\u7684\u6578\u91cf\u6700\u591a\uff0c\u7dca\u63a5\u8457\u7684\u662f\u81e8\u5e8a\u56db\u671f\uff08\u4e0a\u5e02\u5f8c\u7684\u85e5\u54c1\u76e3\u63a7\u8a66\u9a57\uff09\u4ee5\u53ca\u81e8\u5e8a\u4e09\u671f\u7684\u8a66\u9a57\uff1b\u5728\u4e2d\u570b\u57f7\u884c\u7684\u591a\u7232\u5f8c\u671f\u7684\u81e8\u5e8a\u8a66\u9a57\uff0c\u4f30\u8a08\u548c\u4e0a\u5e02\u7b56\u7565\u6216\u8207\u76e3\u7ba1\u6cd5\u898f\u8981\u6c42\u6709\u95dc\u3002<\/p>\n<div id=\"attachment_5682\" style=\"width: 650px\" class=\"wp-caption aligncenter\"><img loading=\"lazy\" decoding=\"async\" aria-describedby=\"caption-attachment-5682\" class=\"wp-image-5682 size-large\" src=\"http:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-9-1024x500.png\" alt=\"\u57167\uff1a2008\u5e74\u52302018\u5e74\u6240\u6709\u9069\u61c9\u75c7\u53ca\u975e\u816b\u7624\u7f55\u898b\u75c5\u5728\u4e2d\u570b\u57f7\u884c\u81e8\u5e8a\u8a66\u9a57\u6578\u91cf\u6bd4\u8f03 (\u8cc7\u6599\u4f86\u6e90: Cortellis \u81e8\u5e8a\u60c5\u5831\uff0c\u4f9d\u64daCortellis\u4e2d\u7f55\u898b\u75c5\u7684\u7d22\u5f15\u6a19\u7c64(rare disease)\u6574\u7406\uff0c\u8cc7\u6599\u7372\u53d6\u65e5\u671f:2018\u5e747\u670830\u65e5)\" width=\"640\" height=\"313\" srcset=\"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-9-1024x500.png 1024w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-9-300x146.png 300w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-9-768x375.png 768w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-9-1536x750.png 1536w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-9-82x40.png 82w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-9.png 1755w\" sizes=\"auto, (max-width: 640px) 100vw, 640px\" \/><p id=\"caption-attachment-5682\" class=\"wp-caption-text\">\u57167\uff1a2008\u5e74\u52302018\u5e74\u6240\u6709\u9069\u61c9\u75c7\u53ca\u975e\u816b\u7624\u7f55\u898b\u75c5\u5728\u4e2d\u570b\u57f7\u884c\u81e8\u5e8a\u8a66\u9a57\u6578\u91cf\u6bd4\u8f03 (\u8cc7\u6599\u4f86\u6e90: <a href=\"https:\/\/clarivate.com\/cortellis\/zh-hant\/solutions\/trials-intelligence-analytics\/\" target=\"_blank\" rel=\"noopener\">Cortellis \u81e8\u5e8a\u60c5\u5831<\/a>\uff0c\u4f9d\u64daCortellis\u4e2d\u7f55\u898b\u75c5\u7684\u7d22\u5f15\u6a19\u7c64(rare disease)\u6574\u7406\uff0c\u8cc7\u6599\u7372\u53d6\u65e5\u671f:2018\u5e747\u670830\u65e5)<\/p><\/div>\n<p>&nbsp;<\/p>\n<div id=\"attachment_5683\" style=\"width: 650px\" class=\"wp-caption aligncenter\"><img loading=\"lazy\" decoding=\"async\" aria-describedby=\"caption-attachment-5683\" class=\"wp-image-5683 size-large\" src=\"http:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-10-1024x511.png\" alt=\"\u57168\uff1a2008\u5e74\u52302018\u5e74\u6240\u6709\u9069\u61c9\u75c7\u53ca\u975e\u816b\u7624\u7f55\u898b\u75c5\u5728\u4e2d\u570b\u57f7\u884c\u81e8\u5e8a\u8a66\u9a57\u671f\u5225\u6bd4\u8f03 (\u8cc7\u6599\u4f86\u6e90: Cortellis \u81e8\u5e8a\u60c5\u5831\uff0c\u4f9d\u64daCortellis\u4e2d\u7f55\u898b\u75c5\u7684\u7d22\u5f15\u6a19\u7c64(rare disease)\u6574\u7406\uff0c\u8cc7\u6599\u7372\u53d6\u65e5\u671f:2018\u5e747\u670830\u65e5)\" width=\"640\" height=\"319\" srcset=\"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-10-1024x511.png 1024w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-10-300x150.png 300w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-10-768x383.png 768w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-10-1536x767.png 1536w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-10-80x40.png 80w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-10.png 1743w\" sizes=\"auto, (max-width: 640px) 100vw, 640px\" \/><p id=\"caption-attachment-5683\" class=\"wp-caption-text\">\u57168\uff1a2008\u5e74\u52302018\u5e74\u6240\u6709\u9069\u61c9\u75c7\u53ca\u975e\u816b\u7624\u7f55\u898b\u75c5\u5728\u4e2d\u570b\u57f7\u884c\u81e8\u5e8a\u8a66\u9a57\u671f\u5225\u6bd4\u8f03 (\u8cc7\u6599\u4f86\u6e90: <a href=\"https:\/\/clarivate.com\/cortellis\/zh-hant\/solutions\/trials-intelligence-analytics\/\" target=\"_blank\" rel=\"noopener\">Cortellis \u81e8\u5e8a\u60c5\u5831<\/a>\uff0c\u4f9d\u64daCortellis\u4e2d\u7f55\u898b\u75c5\u7684\u7d22\u5f15\u6a19\u7c64(rare disease)\u6574\u7406\uff0c\u8cc7\u6599\u7372\u53d6\u65e5\u671f:2018\u5e747\u670830\u65e5)<\/p><\/div>\n<p>&nbsp;<\/p>\n<p>\u6574\u9ad4\u800c\u8a00\uff0c2015\u5e74\u5f8c\u516c\u958b\u7684\u81e8\u5e8a\u8a66\u9a57\u6578\u91cf\u6709\u660e\u986f\u6210\u9577\uff0c\u5982\u57169\u6240\u793a\uff0c\u7b2c\u4e00\u6279\u7f55\u898b\u75c5\u540d\u55ae\u7684121\u500b\u9069\u61c9\u75c7\u5728\u4e2d\u570b\u7684\u81e8\u5e8a\u8a66\u9a57\uff0c\u4e5f\u662f\u5f8c\u671f\u7684\u81e8\u5e8a\u8a66\u9a57\u7684\u6578\u91cf\u6700\u591a\u3002<\/p>\n<div id=\"attachment_5684\" style=\"width: 650px\" class=\"wp-caption aligncenter\"><img loading=\"lazy\" decoding=\"async\" aria-describedby=\"caption-attachment-5684\" class=\"wp-image-5684 size-large\" src=\"http:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-11-1024x469.png\" alt=\"\u57169\uff1a2008\u5e74\u52302018\u5e74\u7b2c\u4e00\u6279\u7f55\u898b\u75c5\u540d\u55ae\u5728\u4e2d\u570b\u7684\u81e8\u5e8a\u8a66\u9a57\u671f\u5225\u6bd4\u8f03 (\u8cc7\u6599\u4f86\u6e90: Cortellis \u81e8\u5e8a\u60c5\u5831\uff0c\u4f9d\u64daCortellis\u4e2d\u9069\u61c9\u75c7\u540d\u7a31\u7684\u7d22\u5f15\u6a19\u7c64\u6574\u7406\uff0c\u8cc7\u6599\u7372\u53d6\u65e5\u671f:2018\u5e747\u670830\u65e5)\" width=\"640\" height=\"293\" srcset=\"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-11-1024x469.png 1024w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-11-300x137.png 300w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-11-768x351.png 768w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-11-1536x703.png 1536w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-11-87x40.png 87w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-11.png 1720w\" sizes=\"auto, (max-width: 640px) 100vw, 640px\" \/><p id=\"caption-attachment-5684\" class=\"wp-caption-text\">\u57169\uff1a2008\u5e74\u52302018\u5e74\u7b2c\u4e00\u6279\u7f55\u898b\u75c5\u540d\u55ae\u5728\u4e2d\u570b\u7684\u81e8\u5e8a\u8a66\u9a57\u671f\u5225\u6bd4\u8f03 (\u8cc7\u6599\u4f86\u6e90: Cortellis \u81e8\u5e8a\u60c5\u5831\uff0c\u4f9d\u64daCortellis\u4e2d\u9069\u61c9\u75c7\u540d\u7a31\u7684\u7d22\u5f15\u6a19\u7c64\u6574\u7406\uff0c\u8cc7\u6599\u7372\u53d6\u65e5\u671f:2018\u5e747\u670830\u65e5)<\/p><\/div>\n<p>&nbsp;<\/p>\n<p>\u9032\u4e00\u6b65\u5206\u6790\u7b2c\u4e00\u6279\u7f55\u898b\u75c5\u540d\u55ae\u4e2d\u7684\u81e8\u5e8a\u8a66\u9a57\uff0c\u5982\u571610\u6240\u793a\uff0c\u6bd4\u4f8b\u6700\u9ad8\u7684\u662f\u795e\u7d93\u75be\u75c5\u76f8\u95dc\u7684\u7f55\u898b\u75c5\uff0c\u4f5440%\uff0c\u5176\u6b21\u662f\u8840\u6db2\u76f8\u95dc\u7684\u7f55\u898b\u75c5\uff08\u4f5429%\uff09\uff0c\u5305\u62ec\u7b2c\u516b\u51dd\u8840\u56e0\u5b50\u7f3a\u4e4f (factor VIII deficiency)\u3001\u7b2c\u4e5d\u51dd\u8840\u56e0\u5b50\u7f3a\u4e4f (Factor IX deficiency)\u4ee5\u53ca\u8840\u53cb\u75c5\uff0c\u8207\u524d\u8ff0\u975e\u816b\u7624\u7f55\u898b\u75c5\u7684\u81e8\u5e8a\u8a66\u9a57\u76f8\u4f3c\uff0c\u4e5f\u662f\u5f8c\u671f\u7684\u81e8\u5e8a\u8a66\u9a57\u5c45\u591a\u6578\u3002<\/p>\n<div id=\"attachment_5685\" style=\"width: 650px\" class=\"wp-caption aligncenter\"><img loading=\"lazy\" decoding=\"async\" aria-describedby=\"caption-attachment-5685\" class=\"wp-image-5685 size-large\" src=\"http:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-12-1024x502.png\" alt=\"\u571610\uff1a2008\u52302018\u5e74\u7b2c\u4e00\u6279\u7f55\u898b\u75c5\u540d\u55ae\u4e2d\u7684121\u500b\u9069\u61c9\u75c7\u5728\u4e2d\u570b\u7684\u81e8\u5e8a\u8a66\u9a57\u53ca\u671f\u5225 (\u8cc7\u6599\u4f86\u6e90: Cortellis \u81e8\u5e8a\u60c5\u5831\uff0c\u4f9d\u64daCortellis\u4e2d\u9069\u61c9\u75c7\u540d\u7a31\u7684\u7d22\u5f15\u6a19\u7c64\u6574\u7406\uff0c\u8cc7\u6599\u7372\u53d6\u65e5\u671f:2018\u5e747\u670830\u65e5)\" width=\"640\" height=\"314\" srcset=\"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-12-1024x502.png 1024w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-12-300x147.png 300w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-12-768x376.png 768w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-12-1536x753.png 1536w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-12-82x40.png 82w, https:\/\/clarivate.com\/life-sciences-healthcare\/wp-content\/uploads\/sites\/4\/2018\/09\/challenges-orphen-drugs-in-china-12.png 1749w\" sizes=\"auto, (max-width: 640px) 100vw, 640px\" \/><p id=\"caption-attachment-5685\" class=\"wp-caption-text\">\u571610\uff1a2008\u52302018\u5e74\u7b2c\u4e00\u6279\u7f55\u898b\u75c5\u540d\u55ae\u4e2d\u7684121\u500b\u9069\u61c9\u75c7\u5728\u4e2d\u570b\u7684\u81e8\u5e8a\u8a66\u9a57\u53ca\u671f\u5225 (\u8cc7\u6599\u4f86\u6e90: Cortellis 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et al.\u7684\u7814\u7a76\uff0c\u5728\u7f8e\u570b\u3001\u6b50\u6d32\u548c\u65e5\u672c\u6838\u51c6\u4e0a\u5e02\u7684\u7f55\u898b\u75c5\u85e5\u7269\u4e2d\uff0c\u5206\u5225\u50c5\u670937.8%\u300124.6%\u548c52.4%\u80fd\u5728\u4e2d\u570b\u7684\u4e09\u7532\u91ab\u9662\u8cfc\u85e5\u3002\u76f8\u8f03\u65bc\u4e0a\u8ff0\u570b\u5bb6\uff0c\u4e2d\u570b\u5c1a\u672a\u516c\u5e03\u4f9d\u64da\u4eba\u53e3\u6216\u60a3\u75c5\u7387\u800c\u5b9a\u7684\u7f55\u898b\u75c5\u7684\u5b98\u65b9\u5b9a\u7fa9\uff0c\u4e5f\u9084\u6c92\u516c\u5e03\u91dd\u5c0d\u7f55\u898b\u75c5\u4e0a\u5e02\u7684\u7279\u5225\u6cd5\u6216\u512a\u60e0\u3002NMPA\u53ef\u4f9d\u7533\u8acb\u4eba\u7684\u8acb\u6c42\u8c41\u514d\u81e8\u5e8a\u8a66\u9a57\u6216\u51cf\u5c11\u53d7\u8a66\u8005\u4eba\u6578\uff1b\u5728\u5176\u4ed6\u570b\u5bb6\u5df2\u6838\u51c6\u4e0a\u5e02\u7684\u7f55\u898b\u75c5\u85e5\u7269\uff0cNMPA\u53ef\u80fd\u8003\u616e\u6709\u689d\u4ef6\u7684\u6279\u51c6\u4e0a\u5e02\uff0c\u4e26\u8981\u6c42\u7533\u8acb\u4eba\u5728\u4e0a\u5e02\u5f8c\u88dc\u5145\u8a66\u9a57\u6578\u64da\u3002<\/p>\n<p>\u5c31\u73fe\u6709\u7684\u7f55\u898b\u75c5\u540d\u55ae\u4f86\u770b\uff0c\u8eab\u70ba\u5168\u7403\u7b2c\u4e8c\u5927\u91ab\u85e5\u5e02\u5834\u7684\u4e2d\u570b\uff0c\u76ee\u6a19\u662f\u52a0\u901f\u7f55\u898b\u75c5\u7684\u8a3a\u65b7\u53ca\u65b0\u85e5\u4e0a\u5e02\uff1b\u7f55\u898b\u75c5\u540d\u55ae\u7684\u516c\u5e03\u5c0d\u65bc\u5f8c\u7e8c\u7684\u653f\u7b56\u65b9\u5411\u63d0\u4f9b\u91ab\u7642\u7167\u8b77\u3001\u8ca1\u52d9\u652f\u6301\u7684\u57fa\u790e\u3002\u4e2d\u570b\u653f\u5e9c\u4e5f\u91cd\u8996\u5efa\u7acb\u975e\u76c8\u5229\u7684\u7f55\u898b\u75c5\u7814\u7a76\u6a5f\u69cb\uff0c\u4f8b\u5982\uff1a\u4e2d\u570b\u7f55\u898b\u75c5\u767c\u5c55\u4e2d\u5fc3 (Chinese Organization for Rare Disorders, CORD) \u6210\u7acb\u65bc2013\u5e74\uff0c\u662f\u4e00\u5bb6\u5c08\u6ce8\u65bc\u7f55\u898b\u75c5\u9818\u57df\u7684\u975e\u71df\u5229\u7d44\u7e54\u3002\u7f55\u898b\u75c5\u5728\u4e2d\u570b\u7684\u767c\u5c55\u53ef\u4ee5\u9810\u671f\u7684\u662f\u82e5\u7531\u570b\u5bb6\u8a08\u5283\u4fdd\u8b49\u8986\u84cb\u660e\u78ba\u7684\u7f55\u898b\u75c5\uff0c\u5c07\u63a8\u52d5\u4e2d\u570b\u7f55\u898b\u75c5\u60a3\u8005\u7684\u91ab\u7642\u4fdd\u969c\u7167\u9867\uff0c\u5c0d\u65bc\u5df2\u5728\u5176\u4ed6\u570b\u5bb6\u4e0a\u5e02\u7684\u7f55\u898b\u75c5\u85e5\u7269\uff0c NMPA\u53ef\u80fd\u4ee5\u7279\u5225\u6cd5\u898f\u7bc4\u5176\u4e0a\u5e02\u53ca\u92b7\u552e\uff0c\u4f7f\u5f97\u75c5\u60a3\u80fd\u5f97\u5230\u59a5\u5584\u6cbb\u7642\u53ca\u51cf\u5c11\u76e3\u7ba1\u5197\u9918\u53ca\u7d93\u6fdf\u4e0a\u7684\u8ca0\u64d4\u3002\u5176\u4ed6\u7684\u95dc\u9375\u63aa\u65bd\u53ef\u80fd\u9084\u5305\u62ec\uff1a\u6bd4\u7167\u5176\u4ed6\u85e5\u653f\u5e02\u5834\u5141\u8a31\u7f55\u898b\u75c5\u4ee5\u5feb\u901f\u901a\u9053\u7684\u4e0a\u5e02\u8def\u5f91\u3001\u514d\u9664\u5927\u898f\u6a21\u81e8\u5e8a\u8a66\u9a57\u3001\u5c0d\u7f55\u898b\u75c5\u85e5\u7269\u7684\u7279\u5225\u8a31\u53ef\u4ee5\u53ca\u914d\u5408\u5efa\u7acb\u75c5\u60a3\u7db2\u7d61\u548c\u6cbb\u7642\u8cbb\u7528\u5831\u92b7\u7b49\u65b9\u5f0f\u3002 \u4e0a\u8ff0\u7a2e\u7a2e\u5c07\u4f7f\u4e2d\u570b\u5728\u7f55\u898b\u75c5\u7684\u91ab\u7642\u7167\u8b77\u548c\u793e\u6703\u8ca0\u64d4\u4e0a\uff0c\u4f4d\u65bc\u4e00\u500b\u6709\u5229\u4f4d\u7f6e\u3002<\/p>\n<p>&nbsp;<\/p>\n<p>&nbsp;<\/p>\n<p>\u53c3\u8003\u8cc7\u6599\uff1a<\/p>\n<p><sup id=\"fn1\">1.<\/sup> GlobalGenes.org<\/p>\n<p><sup id=\"fn2\">2.<\/sup> GlobalGenes.org<\/p>\n<p><sup id=\"fn3\">3.<\/sup> Sara H D Smith, Trinity College, Orphan Drug Development: Incentives Under the Orphan Drug Act (2016)<\/p>\n<p><sup id=\"fn4\">4.<\/sup> Nature Reviews Drug Discovery volume 16, pages 381\u2013382 (2017)<\/p>\n<p><sup id=\"fn5\">5.<\/sup> Drug Discovery Today. 2018 Jan;23 (1):187-195)<\/p>\n<p><sup id=\"fn6\">6.<\/sup> Drug Discovery Today. 2018 Jan;23 (1):187-195)<\/p>\n<p><sup id=\"fn7\">7.<\/sup> Evaluate Pharma, Orphan Drug report (2018))<\/p>\n<p><sup id=\"fn8\">8.<\/sup> Gong et al. 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