2022年儿科疾病治疗分析与药物研究.pdf

?https:/?https:/?02?03?01?04?05Contents?https:/?900?2016?研?务?群?群?39.2%39.2%?20.5%20.5%?17.6%17.6%?15.8%15.8%?务?2019?研?2019?0 0-1515?2497724977?17.8%17.8%?16-59?89640?64.0%?60?副?25388?18.1%?务?65?副?17603?12.6%?17.80%64%18.10%2019?0-15?16-59?60?务?务?300-400?2024?2.69?(?研)?溶?3?/?9%?研?研?60%60%?研?5%5%?17.8%17.8%?2011?2011?384?384?副?副?2019?2019?872?872?10.80%10.80%?务?10.69%?39.20%20.50%17.60%15.80%?6.81%?https:/?研?1?2?研务?务?90%90%?54.46%41.44%4.10%?1 1?6.3%12.9%24.4%0.0%5.0%10.0%15.0%20.0%25.0%30.0%?1 10.1%0.1%研NMPA?研?6000?10?0.1%?0 0-1 1?1 1-2424?3 3-5 5?6 6-1414?1.84?930?5%?群?90%?1?https:/?研?研?研?研?研?导?试?试?试?6000?10?告?90%?副?75%?副?3 3500500+?60+60+?诱?https:/?模?模?务?;?务?告?模?模?4?的?研?https:/?CFDA?够?11?20032003201620162013201320112011?(CFDA)?(?3尼)?20042004?务?(2011?2020?)?的?务?20122012CFDA?够?研?研?吐?20152015?溶?告?资?告?确?2021202120222022?务?,?12?https:/?模?务?(?ICH)?ICH?E11/S11?/M11?2014?务?够?务?2016?研?研?务?研?研?研?研?研?2017?溶?务?研?务?CDE?https:/?FDA?模?1979?FDA?1994?1997?FDA?务?6?2002?&2003?吐?PBCA?PREA?2?2007?FDA?2007?吐?2012?FDA?II?够?够?研?2019?FDA?2-12?12-17?FDA?够?务?62%85%?2005?2015?600600?务?2810531522699?BPCA?PREA?BPCA?PREA2007-2020?2007-2012?2012-2020?00001747352002040602012 2013 2014 2015 2016 2017 2018 20192012-2019?(?)?1979?务?2003?https:/?%?4?71116.76?4?63516.49?-?3?86813.76?2?97710.59?2?7289.70?1?1163.97?1?0563.76?E?1?0083.59?9273.30?9243.29?23?95085.19-?%?220?89048.92?副?群100?14522.18?38?2738.48?18?1174.01?9?6342.13?7?6161.69?7?0571.56?5?9801.32?5?6921.26?-?4?7201.05?418?12492.61-?1.?10?2.?10?务?务?0 0 1818?20102010 20162016?务?务?https:/?研?95%?%?186?96273.11?54?60621.35?4?4831.75?4?3661.71?1?7980.70?1?0740.42?8470.33?6050.24?4420.17?2790.11?255?46299.90?%?副?群113?08146.09?31?95913.02?22?4269.14?22?2049.05?14?6535.97?13?9185.67?9?2083.75?4?4831.83?3?8871.58?3?0171.23?238?83697.34-?3.?10?4.?10?https:/?/?30%13%57%?研?3030%?1313%?5757%?务?2.2?2.5%?/?/?务?务?OTCOTC?研?研?https:/?7 7?5 5?(?4 4%)?3 3%?副?30%?副?20%20%?10%10%?30%?2022?副?OS?12%?OS?65%?儿童肿瘤类型多且复杂，中美儿童抗肿瘤可用药存在差距?务?务?务?务?D?D?Mylotarg?Inotuzumab?Daratumumab?Pembrolizumab?Blinatumomab?D?https:/?导?*?得?*?导?*?*?*?导?*MSI-H+?TMB-H?-号?*?群?-?*?B?-*?2021?9?29?5?*?2021?务?号?务?务?吗?https:/?+?研务?研?2017-2020?2020?3.62?0.004%?3.63.6?2017-2020?(?%)?研?2019?务?RSV?CMV?+?A?腺?https:/?Spinraza?SMA?SMN1?SMN?SMN1?SMN?SMA?SMN2?SMN?SMN2?7?7?90%?研?DOI:10.1001/archneurol.2011.74.Spinraza?intrathecal injection?Spinraza?2019?2?5q?SMA?95%?SMA?SMASMA?SpinrazaSpinraza?研务?研务?SMA?务?SMA?3-5?盛?SMA?Spinraza?70?模?3?2021?11?20?SMA?副?Spinraza?https:/?除助听器或人工耳蜗外，基因疗法可为先天性耳聋提供创新药解决方案?1 1/500500?1 1/10001000?研务?2003?副?20572057?16.79?3 3?群?群?务?50%?副?GJB2GJB2?GJB2?GJB2?;?SLC26A4SLC26A4?SLC26A4?的?14%?12s?RNA?A1555G12s?RNA?A1555G?mtDNA A1555G?1993?Presant?mtDNA 12Sr RNA?1555?AG?务?https:/?64%64%36%36%务?%?务?务?研?研?务?研?务?务?务?研?务?研?6?12?21?38?17?https:/?研?1?Ph3?TJ101?1?Ph3?AK0529?1.1?Ph3?群ACT001?1?Ph1?2.2/2.4?Ph2?5?Ph2?XW001?1?Ph1?群?1?Ph1?KY0467?1?Ph1?2.2?Ph1?2022?副?12?务?2022/2/24?3-18?2022/3/3?2022/3/11?2022/3/11?导?2022/3/24?3?2022/3/24?3?NTRK?2022/4/13?2022/5/7?https:/TOP?1?6.28%2?翘?9?3.82%3?D诱?AD诱?3.42%4?2.72%5?2.61%6?D诱?AD诱?2.35%7资?2.25%8?务?1.94%9?1.93%10?1.79%TOP?1?岁5.74%2?D诱?岁5.27%3?岁2.90%4?岁2.81%5?群?2.72%6?群?岁2.23%7?岁2.14%8?AD诱?岁2.09%9?岁1.93%10?1.88%?2020?top102020?top10?研?https:/?+?2021?(600566)204.9?10.16?10.3?603439?46.55?6399?8.79?000915?96.99?3.046?AD诱?号?9?2020?002737?99.81?4.012?翘?4.5?2.5?600557?77.05?2.111?5?300723?67.84?1.546?4.4?000999?379.6?14.33?-?腺?002412?28.13?7888?-?(600351)43.89?1.004?4.19?2019?务?(600750)90.79?3.923?务?3.5?2020?https:/?1994?务?务?务?10?210?110?2016-2021?2019?2020?务?+?2005?副?+?2020?务?务?60.43%60.43%?28.01%28.01%?务?+?务?2g/6?务?务?24.33?务?43.89?https:/?AAV?&?AAVAAV?AAV?A A V M e t aA A V M e t a?溶?AAVAAV-ieieAAVAAV-WM09WM09AAVAAV-WM01WM01?6?2021.12?/?Science?AAV?AAV?AAV?(AAV)?AAVAAV?https:/?务?务?Minoryx?Leriglitazone?X-?副?(X-ALD)?2019?A?吗?吗?https:/?RSV?副?9?务?ziresovir?III?RSV?群?Ziresovir?AK0529?F?8?ziresovir?研?ziresovir溶?9?模?II?RSV?1-24?III?2022?NMPA?ziresovir?NMPA?务?务?3 3?3030%?RSV?务?5555%?p0.001?5 5?7777%?p=0.006?6?研?6?9.0%vs.26.3%?p=0.02?务?ZiresovirZiresovir?https:/?patch?2.2?505b(2)?模?确?务?务?2022?务?patch?010102020303010102020303010102020303?https:/?务?务?务?试?务?务?务?务?导?30?20?10?副?导?务?https:/SOWTStrength?Weakness?/?模?研?Opportunity?OTC/?2?Threat?模?+?SWOT?