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市場調查報告書
商品編碼
1914674
出血性疾病治療市場-全球產業規模、佔有率、趨勢、機會及預測(按藥物類型、疾病類型、分銷管道、最終用戶、地區和競爭格局分類,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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全球出血性疾病治療市場預計將保持強勁成長,從2025年的178.1億美元成長到2031年的298.2億美元,複合年成長率(CAGR)為8.97%。該市場涵蓋多種藥物干預措施,包括因子替代療法和非因子製劑,這些療法對於治療先天性疾病(例如A型血友病、 B型血友病和血管性血友病)至關重要。市場擴張的主要驅動力是遺傳性凝血障礙診斷率的提高以及政府主導的旨在擴大患者獲得關鍵治療機會的舉措。此外,半衰期延長產品的推出也是重要的推動因素,因為這些治療方法可以減輕頻繁給藥的負擔,並有助於患者長期堅持預防性治療方案。
| 市場概覽 | |
|---|---|
| 預測期 | 2027-2031 |
| 市場規模:2025年 | 178.1億美元 |
| 市場規模:2031年 | 298.2億美元 |
| 複合年成長率:2026-2031年 | 8.97% |
| 成長最快的細分市場 | 血漿衍生凝血因子濃縮物 |
| 最大的市場 | 亞太地區 |
儘管取得了這些積極進展,但終身治療的高昂費用仍然是一大障礙,限制了醫療預算有限、報銷體係不完善的開發中國家獲得治療的機會。這一經濟障礙影響著全球範圍內的病例發現和疾病管理的一致性。世界血友病聯盟於2024年發布的《2023年全球監測報告》強調了持續治療支持的迫切需求,該報告估計,全球累計確診出血性疾病患者總數已達465,327例,凸顯了需要持續治療的患者群體龐大。
隨著基因療法和新型生物製藥的商業化進程加速,市場正在經歷變革,治療重點也從慢性病管理轉向潛在的治癒性治療。這項變革包括核准單劑量療法,這些療法能夠糾正潛在的遺傳因素,提供長期保護,無需頻繁輸注。這些先進治療方法價格不菲,並為罕見疾病藥物的報銷標準樹立了新的標竿。例如,在2024年4月發布的新聞稿《美國FDA核准輝瑞的BEQVEZ™》中,輝瑞宣布其基因療法核准用於治療中重度B型血友病成人患者,定價為350萬美元。這標誌著罕見出血性疾病治療領域在臨床和經濟方面都迎來了一個重要的轉捩點。
同時,皮下注射和長效半衰期療法的出現正迅速將治療模式從按需治療轉向預防性護理。患者擴大接受這些新型藥物,它們能夠維持較高的凝血水平,保護關節健康,同時減少傳統治療帶來的後勤挑戰。這些療法的快速普及體現了其商業性影響力。根據賽諾菲於2024年10月發布的“2024年第三季度財務業績”,僅第三季度,ALTUVIIIO的銷售額就達到了1.72億歐元。同樣,羅氏於2024年2月發布的「2023年年度報告」顯示,其抗體藥物Hemlibra的年銷售額達到了41億瑞士法郎,這證實了非因子療法在預防性管理方面的優勢。
終身治療的高昂費用是全球出血性疾病治療市場發展的一大障礙。凝血因子濃縮物和先進的非因子替代療法的高成本使得預算有限的醫療系統難以負擔這些重要的藥物。在公共健保體係不完善的地區,這種經濟壓力阻礙了標準預防性照護的廣泛應用。因此,市場難以將相當一部分確診患者轉化為治療產品的積極使用者,從而限制了新興經濟體的商機。
這些經濟限制因素導致全球市場滲透率和產品利用率有顯著差異。根據世界血友病聯盟的數據,特別是2024年發布的《2023年全球調查報告》,高所得國家人均VIII因子消費量為6.64國際單位(IU),而低收入國家則為0.00 IU。這種顯著差異凸顯了不斷上漲的治療成本如何直接阻礙發展中地區的市場規模和商業性擴張,從而使大部分經濟價值集中在已開發國家。
新型阻礙要素的研發,正在創造一種新的治療模式,其重點在於抗凝血途徑而非單純補充不足的凝血因子。與傳統的因子替代療法或雙特異性抗體不同,抗組織因子途徑抑制劑(抗TFPI)和小干擾RNA(siRNA)療法等藥物透過抑制天然抗凝血因子來重建止血功能。對於存在標準療法阻礙要素的患者而言,這種機制尤其重要,因為它提供了一種便捷的預防性旁路療法。諾和諾德在2025年7月發布的新聞稿「FDA批准Alhemo上市」中宣布,其抗TFPI療法在explorer8 III期臨床試驗中,使無阻礙要素的B型血友病患者的年出血率顯著降低了79%,核准了該方法的臨床價值。
同時,CRISPR-Cas9基因編輯技術的應用正突破第一代病毒載體基因療法的限制。目前的方法主要依賴腺結合病毒(AAV)載體進行基因遞送,而CRISPR技術能夠對患者的基因組進行體外或體內的精確編輯,從而有望實現更持久、更永久的功能性治愈,並降低免疫抗原性風險。這一趨勢針對的是導致凝血障礙的特定基因突變,並且正迅速從臨床前研究過渡到人體臨床試驗。例如,在2025年7月題為「Bio宣布首例患者給藥」的新聞稿中,B BioPharma宣布將推進其自主研發的CRISPR-Cas9 B細胞療法BE-101進入臨床評估階段,計劃在I/II期臨床試驗中招募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: