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市场调查报告书
商品编码
1914674
出血性疾病治疗市场-全球产业规模、份额、趋势、机会及预测(按药物类型、疾病类型、分销管道、最终用户、地区和竞争格局划分,2021-2031年)Bleeding Disorders Treatment Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, Segmented By Drug Type, By Disease Type, By Distribution Channel, By End User, By Region & Competition, 2021-2031F |
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全球出血性疾病治疗市场预计将保持强劲成长,从2025年的178.1亿美元成长到2031年的298.2亿美元,复合年增长率(CAGR)为8.97%。该市场涵盖多种药物干预措施,包括因子替代疗法和非因子製剂,这些疗法对于治疗先天性疾病(例如A型血友病、 B型血友病和血管性血友病)至关重要。市场扩张的主要驱动力是遗传性凝血障碍诊断率的提高以及政府主导的旨在扩大患者获得关键治疗机会的倡议。此外,半衰期延长产品的推出也是重要的推动因素,因为这些治疗方法可以减轻频繁给药的负担,并有助于患者长期坚持预防性治疗方案。
| 市场概览 | |
|---|---|
| 预测期 | 2027-2031 |
| 市场规模:2025年 | 178.1亿美元 |
| 市场规模:2031年 | 298.2亿美元 |
| 复合年增长率:2026-2031年 | 8.97% |
| 成长最快的细分市场 | 血浆衍生凝血因子浓缩物 |
| 最大的市场 | 亚太地区 |
儘管取得了这些积极进展,但终身治疗的高昂费用仍然是一大障碍,限制了医疗预算有限、报销体係不完善的开发中国家获得治疗的机会。这一经济障碍影响着全球范围内的病例发现和疾病管理的一致性。世界血友病联盟于2024年发布的《2023年全球监测报告》强调了持续治疗支持的迫切需求,该报告估计,全球累计确诊出血性疾病患者总数已达465,327例,凸显了需要持续治疗的患者群体庞大。
随着基因疗法和新型生物製药的商业化进程加速,市场正在经历变革,治疗重点也从慢性病管理转向潜在的治癒性治疗。这项变革包括核准单剂量疗法,这些疗法能够纠正潜在的遗传因素,提供长期保护,无需频繁输注。这些先进治疗方法价格不菲,并为罕见疾病药物的报销标准树立了新的标竿。例如,在2024年4月发布的新闻稿《美国FDA核准辉瑞的BEQVEZ™》中,辉瑞宣布其基因疗法核准用于治疗中重度B型血友病成人患者,定价为350万美元。这标誌着罕见出血性疾病治疗领域在临床和经济方面都迎来了一个重要的转捩点。
同时,皮下注射和长效半衰期疗法的出现正迅速将治疗模式从按需治疗转向预防性护理。患者越来越多地接受这些新型药物,它们能够维持较高的凝血水平,保护关节健康,同时减少传统治疗带来的后勤挑战。这些疗法的快速普及体现了其商业性影响力。根据赛诺菲于2024年10月发布的“2024年第三季度财务业绩”,仅第三季度,ALTUVIIIO的销售额就达到了1.72亿欧元。同样,罗氏于2024年2月发布的「2023年年度报告」显示,其抗体药物Hemlibra的年销售额达到了41亿瑞士法郎,这证实了非因子疗法在预防性管理方面的优势。
终身治疗的高昂费用是全球出血性疾病治疗市场发展的一大障碍。凝血因子浓缩物和先进的非因子替代疗法的高成本使得预算有限的医疗系统难以负担这些重要的药物。在公共健保体係不完善的地区,这种经济压力阻碍了标准预防性照护的广泛应用。因此,市场难以将相当一部分确诊患者转化为治疗产品的积极使用者,从而限制了新兴经济体的商机。
这些经济限制因素导致全球市场渗透率和产品利用率有显着差异。根据世界血友病联盟的数据,特别是2024年发布的《2023年全球调查报告》,高所得国家人均VIII因子消费量为6.64国际单位(IU),而低收入国家则为0.00 IU。这种显着差异凸显了不断上涨的治疗成本如何直接阻碍发展中地区的市场规模和商业性扩张,从而使大部分经济价值集中在已开发国家。
新型阻碍要素的研发,正在创造一种新的治疗模式,其重点在于抗凝血途径而非单纯补充不足的凝血因子。与传统的因子替代疗法或双特异性抗体不同,抗组织因子途径抑制剂(抗TFPI)和小干扰RNA(siRNA)疗法等药物透过抑制天然抗凝血因子来重建止血功能。对于存在标准疗法阻碍要素的患者而言,这种机制尤其重要,因为它提供了一种便捷的预防性旁路疗法。诺和诺德在2025年7月发布的新闻稿「FDA批准Alhemo上市」中宣布,其抗TFPI疗法在explorer8 III期临床试验中,使无阻碍要素的B型血友病患者的年出血率显着降低了79%,核准了该方法的临床价值。
同时,CRISPR-Cas9基因编辑技术的应用正突破第一代病毒载体基因疗法的限制。目前的方法主要依赖腺结合病毒(AAV)载体进行基因递送,而CRISPR技术能够对患者的基因组进行体外或体内的精确编辑,从而有望实现更持久、更永久的功能性治愈,并降低免疫抗原性风险。这一趋势针对的是导致凝血障碍的特定基因突变,并且正迅速从临床前研究过渡到人体临床试验。例如,在2025年7月题为「Bio宣布首例患者给药」的新闻稿中,B BioPharma宣布将推进其自主研发的CRISPR-Cas9 B细胞疗法BE-101进入临床评估阶段,计划在I/II期临床试验中招募24名B型血友病成年患者。
The Global Bleeding Disorders Treatment Market is projected to experience robust growth, increasing from a valuation of USD 17.81 Billion in 2025 to USD 29.82 Billion by 2031, reflecting a compound annual growth rate of 8.97%. This sector encompasses a range of pharmacological interventions, including clotting factor replacement therapies and non-factor agents, which are essential for managing congenital conditions such as hemophilia A, hemophilia B, and von Willebrand disease. The market's expansion is primarily fueled by rising diagnosis rates for genetic coagulation defects and government initiatives designed to enhance patient access to critical care. Furthermore, the introduction of extended-half-life products acts as a major catalyst, as these therapies alleviate the burden of frequent administration and promote long-term adherence to prophylactic treatment plans.
| Market Overview | |
|---|---|
| Forecast Period | 2027-2031 |
| Market Size 2025 | USD 17.81 Billion |
| Market Size 2031 | USD 29.82 Billion |
| CAGR 2026-2031 | 8.97% |
| Fastest Growing Segment | Plasma-Derived Coagulation Factor Concentrates |
| Largest Market | Asia Pacific |
Despite these positive developments, the exorbitant cost of lifelong therapy presents a significant obstacle, restricting access in developing nations where healthcare budgets are limited and reimbursement structures are inadequate. This financial barrier impacts both the identification of patients and the consistency of disease management on a global scale. Highlighting the magnitude of the need for sustained therapeutic support, the World Federation of Hemophilia's Report on the Annual Global Survey 2023, published in 2024, recorded a cumulative total of 465,327 individuals diagnosed with bleeding disorders worldwide, underscoring the vast population requiring ongoing care.
Market Driver
The market is being transformed by the accelerated commercialization of gene therapies and novel biologics, which are shifting the focus from chronic management to potential cures. This evolution involves the approval of one-time treatments that correct underlying genetic causes, providing long-term protection without the need for frequent infusions. These advanced therapies command premium prices, establishing new reimbursement benchmarks for orphan drugs. For example, Pfizer Inc. announced in an April 2024 press release titled "U.S. FDA Approves Pfizer's BEQVEZ(TM)" that regulators had approved their gene therapy for adults with moderate to severe hemophilia B, assigning it a list price of $3.5 million, marking a significant clinical and economic shift in managing rare bleeding disorders.
Simultaneously, there is a strong movement from on-demand care to prophylactic regimens, driven by the availability of subcutaneous and extended-half-life therapies. Patients are increasingly adopting these novel agents because they maintain higher coagulation levels and protect joint health while reducing the logistical challenges associated with traditional treatments. The commercial impact of these options is demonstrated by their rapid adoption; according to Sanofi's "Q3 2024 Earnings" presentation in October 2024, sales of ALTUVIIIO reached €172 million in just the third quarter. Similarly, Roche reported in their "Annual Report 2023" from February 2024 that the antibody Hemlibra achieved full-year sales of CHF 4.1 billion, confirming the dominance of non-factor therapies in prophylactic management.
Market Challenge
The prohibitive expense associated with lifelong treatment acts as a substantial hurdle to the growth of the Global Bleeding Disorders Treatment Market. The high costs of clotting factors and advanced non-factor replacement therapies make these vital medications unaffordable for healthcare systems operating with restricted budgets. In areas lacking robust public reimbursement schemes, this financial pressure hinders the widespread implementation of standard prophylactic care. As a result, the market struggles to convert a significant proportion of the diagnosed patient population into active users of therapeutic products, thereby constraining revenue opportunities in emerging economies.
This economic limitation fosters a sharp divide in global market penetration and product usage. Data from the World Federation of Hemophilia, specifically the Report on the Annual Global Survey 2023 published in 2024, indicated that while the median per capita consumption of Factor VIII was 6.64 International Units in high-income nations, it remained at 0.00 International Units in low-income countries. This stark disparity underscores how elevated treatment costs directly obstruct market volume and commercial expansion in developing regions, effectively concentrating the majority of economic value within developed nations.
Market Trends
The development of novel rebalancing agents for inhibitor management is creating a new therapeutic paradigm by focusing on anticoagulation pathways instead of merely replacing missing clotting factors. Distinct from traditional factor replacements or bispecific antibodies, agents such as anti-tissue factor pathway inhibitors (anti-TFPI) and small interfering RNA (siRNA) therapies work by suppressing natural anticoagulants to re-establish hemostasis. This mechanism is especially crucial for patients with inhibitors to standard treatments, offering a simplified bypass solution for prophylaxis. The clinical value of this approach was evidenced when Novo Nordisk announced in a July 2025 press release titled "FDA approves Alhemo" that their anti-TFPI therapy achieved a statistically significant 79% reduction in the annualized bleeding rate for Hemophilia B patients without inhibitors during the explorer8 Phase 3 trial.
In parallel, advancements in CRISPR-Cas9 gene editing applications are evolving beyond the limitations of first-generation viral vector gene therapies. While current approaches largely depend on adeno-associated virus (AAV) vectors for gene addition, CRISPR technology facilitates precise ex vivo or in vivo editing of the patient's genome, offering the potential for a more durable, permanent functional cure with lower immunogenicity risks. This trend is rapidly transitioning from preclinical studies to human trials, targeting the specific genetic mutations causing coagulation deficits. For instance, Be Biopharma announced in a July 2025 press release titled "Be Bio Announces First Participant Dosed" that it had moved its proprietary CRISPR-Cas9 engineered B cell medicine, BE-101, into clinical evaluation with a Phase 1/2 study aiming to enroll 24 adults with Hemophilia B.
Report Scope
In this report, the Global Bleeding Disorders Treatment Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:
Company Profiles: Detailed analysis of the major companies present in the Global Bleeding Disorders Treatment Market.
Global Bleeding Disorders Treatment Market report with the given market data, TechSci Research offers customizations according to a company's specific needs. The following customization options are available for the report: