Rare Disease Treatment Market Insights and Growth by 2031
【研究报告】2022年罕见病治疗市场规模为1.9352亿美元，预计到2030年将达到4.9606亿美元；预计 2022 年至 2030 年复合年增长率为 11.70%。
见解和分析师观点：
大约 7000 种罕见疾病影响着美国超过 3000 万人，危及生命。罕见疾病的药物、生物制剂和设备开发等治疗具有挑战性，例如复杂的生物学以及对许多罕见疾病的自然史缺乏了解——罕见疾病的患病率不断增加，而且患有罕见疾病的患者人数本来就很少。罕见疾病。美国FDA已批准了数百种治疗罕见疾病的药物，但大多数罕见疾病没有FDA批准的治疗方法。
增长动力和限制：
罕见疾病患病率增加
根据美国食品和药物管理局的数据，一种罕见疾病影响美国不到 20 万人，并且根据 2022 年 Cheisi 全球罕见疾病文章，根据每年每名患者 (PPPY) 计算，罕见疾病发病率比大众市场疾病发病率高出约 10 倍。此外，欧盟公共卫生委员会表示，2000 年欧盟境内受该病影响的人数不超过 1 人。在加拿大，罕见病影响的人数不到万分之五。同样，根据rarediseaseday.org 2022年2月的更新，约有3亿人患有罕见疾病，这是一个很高的数字。因此，由于沉重的负担和不断提高的认识，预计对罕见疾病有效和先进的治疗解决方案的需求将会增加。预计这将推动预测期内研究市场的增长。由于罕见病治疗采用多种分销渠道，如网上药房、专科药房和医院药房，对罕见病治疗的需求激增。
此外，罕见疾病病例数量的增加和有利于疫苗接种的倡议、新疗法和药物的增加以及新药上市数量的增加等因素有利的政府政策正在推动整体市场的增长。
药物批准数量不断增加，推动罕见病治疗市场增长
根据药物研究和美国制造商表示，到 2021 年，大约有 791 种潜在的孤儿罕见病候选药物正在进行临床试验。在所有临床孤儿药中，罕见癌症168个，罕见血癌120个，神经系统疾病56个，遗传性疾病192个，血液疾病54个，自身免疫性疾病51个，感染性疾病36个。 2021 年，药物评价与研究中心 (CDER) 批准了约 26 种新型孤儿药用于治疗罕见病。其中一些批准的产品包括 Amondys 4、Bylvay、Welireg、Lumakras、Scemblix、Welireg、Cytalux、Besremi 、Empaveli、Evkeeza、Exkivity、fexinidazol 和 Zynlonta 等。因此，罕见病药物审批的增加和新孤儿药的推出预计将促进行业增长。
战略见解
报告细分和范围：
罕见病治疗市场按方法细分为给药途径 (ROA) ，即口服、注射剂等。注射剂细分市场在 2022 年占据主导地位。市场还按药物类型细分，即生物制剂、小分子和生物仿制药。生物制品细分市场在 2022 年占据最大的市场份额。预计该细分市场从 2022 年至 2030 年的复合年增长率最高，因为与其他产品相比，该细分市场因其功效而受到欢迎。生物技术和研发的不断进步促进了新型生物制剂的开发。罕见病治疗市场按治疗领域分为肿瘤学、神经系统疾病、心血管疾病、肌肉骨骼疾病、传染病等。肿瘤学领域在 2022 年占据最大的市场份额，预计 2022 年至 2030 年复合年增长率最高。该细分市场的主导地位可归因于罕见癌症适应症的高患病率和复发率。罕见病治疗市场按分销渠道分为医院药房、网上药房和专科药房。 2022年，专业药品领域占据最大市场份额；然而，在线药店预计在 2022 年至 2030 年期间复合年增长率最高。全球越来越多的医院药房以及在线药房因其易用性而出现，正在推动该细分市场的增长。
罕见病治疗市场根据地理位置进行细分北美（美国、加拿大和墨西哥）、欧洲（德国、法国、意大利、英国、俄罗斯和欧洲其他地区）、亚太地区（澳大利亚、中国、日本、印度、韩国和其他地区）亚太地区）、中东和非洲（南非、沙特阿拉伯、阿联酋以及中东和非洲其他地区）以及南美洲和中美洲（巴西、墨西哥以及 SCAM 其他地区）。
细分分析：
罕见病治疗市场按药物类型分为生物制剂、小分子、和生物仿制药。生物制品领域在 2022 年占据最大的市场份额。预计该领域在 2022 年至 2030 年期间复合年增长率最高。生物技术公司的不断进步促进了新型生物制品的开发：高靶点特异性和生物药物改变稀有药物的潜力疾病治疗市场。例如，2021年，美国FDA批准了Zolgensma，这是一种用于治疗脊髓性肌萎缩症的基因疗法。此外，预计越来越多的孤儿生物制剂专利到期将为新公司铺平道路，导致用于治疗各种罕见疾病的药品价格下降，从而推动整体细分市场的增长。
2022 年和 2030 年罕见疾病治疗市场 ROA
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罕见病治疗市场按给药途径（ROA）分为口服、注射和其他。注射剂细分市场在 2022 年占据最大市场份额，预计 2022-2030 年复合年增长率最高。这可以归因于越来越多的公司参与新推出的治疗罕见疾病的新型注射剂。例如，2021年2月，美国 FDA 批准了 Sarepta Therapeutics 的治疗方案。 AMONDYS 45（卡西默森注射液）用于治疗杜氏肌营养不良症（DMD）。口服部分紧随注射部分之后。市场上的主要巨头也参与了新产品创新。例如，2022 年 7 月，辉瑞公司的 Xalkori（克唑替尼）获得美国 FDA 批准，用于治疗 ALK 阳性炎性肌纤维母细胞瘤（IMT）儿童和成人患者，该药物口服剂量为 250 mg，直至疾病进展
区域分析：
根据地理位置，全球罕见病治疗市场进行细分分为五个关键区域：北美、欧洲、亚太地区、南美和北美。中美洲、中东和非洲非洲。 2022年，北美在全球罕见病治疗市场规模中占据最大份额。据估计，2022年至2030年，亚太地区的复合年增长率最高。由于接受罕见病治疗的肥胖人口不断增加以及北美地区罕见病的流行，北美的罕见病治疗市场在过去几年中经历了大幅增长。美国和罕见疾病治疗的进步。 2022年，北美在全球罕见病治疗市场中占据最大份额。由于罕见病负担不断加重，北美罕见病治疗市场在过去几年经历了大幅增长。亚太地区是全球罕见病治疗市场增长最快的地区。这一增长归因于多种因素，由于未满足的医疗保健需求增加、罕见疾病患病率增加以及对罕见疾病治疗的需求，未开发和新兴市场的罕见疾病治疗潜力巨大。
此外，由于对增强医疗服务的需求增加、对改善医疗基础设施的大量投资以及医疗保健创新的发展，罕见疾病市场的新兴经济体正在以显着的速度发展行业。对罕见疾病治疗的需求不仅限于新兴国家，在发展中国家也存在。企业在罕见病治疗市场的贡献等因素进一步推动罕见病治疗市场的增长。例如，2021年10月，阿斯利康的 Tezepelumab 获得美国 FDA 的孤儿药资格认定，用于治疗嗜酸性粒细胞性食管炎（EoE）。这些产品可以提高患者的依从性，从而扩大消费者基础并增加市场收入。
行业发展和未来机遇：
全球罕见病治疗市场主要参与者采取的各种举措如下：
- 2021 年 8 月，Zevra Therapeutics 以 9100 万美元收购了 Acer，将自己打入商业罕见病生态系统。
- 2021 年，Orphan Technologies 以 9000 万美元收购了美国生物技术公司 Retropin Inc.。 。该安排包括OT-58，一种全新的酶替代疗法。该公司正在进行治疗经典同型半胱氨酸尿症的 1/2 期试验。
- 2021 年 10 月，UCB 和 Ra Pharmaceuticals Inc. 签订了一项合并协议，根据该协议，UCB 收购了 Ra Pharma 以改善重症肌无力和其他罕见疾病患者的治疗选择。
- 2023 年 7 月，Alexion 与阿斯利康罕见病就一系列临床前基因治疗项目和辉瑞公司的支持技术。该协议的目的是通过增加互补的管道资产和创新技术来推进下一代基因组药物。此次收购价值 133 亿美元，还为市场提供了高增长机会，从而增强了公司的罕见
Report Coverage
Revenue forecast, Company Analysis, Industry landscape, Growth factors, and Trends
Segment Covered
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Regional Scope
North America, Europe, Asia Pacific, Middle East & Africa, South & Central America
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- F. Hoffmann-La Roche Ltd.
- Pfizer, Inc.
- PTC Therapeutics
- Novartis AG
- Takeda Pharmaceutical Company
- Bayer AG
- AbbVie Inc.
- Merck & Co. Inc.
- Bristol Myers Squibb
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