세계의 후천성 희귀 혈액질환 치료제 시장 - 산업규모, 점유율, 동향, 기회, 예측, 요법별, 적응 질환별, 유통 채널별, 지역별 부문, 경쟁(2020-2030년)
Acquired Orphan Blood Diseases Therapeutics Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, Segmented By Therapy, By Disease Indication, By Distribution Channel, By Region & Competition, 2020-2030F
상품코드:1691705
리서치사:TechSci Research
발행일:2025년 03월
페이지 정보:영문 180 Pages
라이선스 & 가격 (부가세 별도)
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한글목차
세계의 후천성 희귀 혈액질환 치료제 시장은 2024년에 79억 5,000만 달러로 평가되었고, 2030년까지 CAGR은 7.80%를 나타낼 전망입니다.
후천성 희귀 혈액 질환은 혈액과 그 성분(적혈구, 백혈구, 혈소판 등)에 영향을 미치고 유전성이 아니라 평생 동안 후천적으로 발생하는 희귀하고 종종 생명을 위협하는 병리학 그룹을 포괄하는 용어입니다. 이 혈액 질환은 출생시 존재하거나 유전에 의해 상속되지 않습니다. 그 대신, 많은 경우에, 여러가지 유인이나 기초적인 건강상태에 기인하여 사람이 살아 있는 동안에 개발되거나 후천적으로 발병합니다. 후천성 희소 혈액 질환의 진단, 치료 및 관리에는 혈액 전문의와 희소 혈액 질환에 대한 전문 지식을 가진 다른 건강 관리 제공업체의 전문 치료가 필요할 수 있습니다. 치료 접근법은 수혈, 면역억제 요법, 표적 요법, 경우에 따라서는 골수 이식 등을 포함합니다.
시장 개요
예측 기간
2026-2030년
시장 규모 : 2024년
79억 5,000만 달러
시장 규모 : 2030년
123억 8,000만 달러
CAGR : 2025-2030년
7.80%
급성장 부문
병원 약국
최대 시장
북미
유전체학, 면역학, 분자 생물학 등 의학 연구의 지속적인 발전으로 이러한 질병의 병인에 대한 이해가 깊어지고 있습니다. 이러한 지식은 표적 치료제와 치료 혁신의 개발을 지원합니다. 희소질환용 의약품의 지정에 의해 제공되는 시장독점권 연장, 세액공제, 개발비용 절감 등 규제상 인센티브는 제약회사가 희소질환의 연구개발에 투자하는 것을 뒷받침하고 있습니다. 환자 지원 단체와 조직은 연구를 추진하고, 인지도를 높이고, 치료에 대한 액세스 개선을 제창하는 데 있어 매우 중요한 역할을 하고 있습니다. 그들의 노력은 후천성 희소 혈액 질환 분야의 진보를 가속화하는 데 도움이 됩니다. 유전자 치료, 단클론항체, 면역조절제와 같은 새로운 치료법의 출현으로 후천성 희소 혈액 질환의 치료 옵션의 폭이 넓어지고 있습니다. 진단 기술과 기술의 진보로 후천성 희소 혈액 질환의 진단은 더욱 정확하고 시기 적절합니다. 이를 통해 조기 개입과 치료가 가능해졌습니다.
Global Acquired Orphan Blood Diseases Therapeutics Market was valued at USD 7.95 billion in 2024 and is anticipated to witness an impressive growth in the forecast period with a CAGR of 7.80% through 2030. Acquired orphan blood diseases is a term that encompasses a group of rare and often life-threatening medical conditions that affect the blood and its components (e.g., red blood cells, white blood cells, platelets) and are not inherited but acquired during a person's lifetime. These blood diseases are not present at birth or inherited through genetics. Instead, they develop or are acquired during a person's life, often due to various triggers or underlying health conditions. Diagnosis, treatment, and management of acquired orphan blood diseases often require specialized care from hematologists or other healthcare providers with expertise in rare blood disorders. Treatment approaches may include blood transfusions, immunosuppressive therapies, targeted therapies, and, in some cases, bone marrow transplantation.
Market Overview
Forecast Period
2026-2030
Market Size 2024
USD 7.95 Billion
Market Size 2030
USD 12.38 Billion
CAGR 2025-2030
7.80%
Fastest Growing Segment
Hospital Pharmacy
Largest Market
North America
Ongoing advancements in medical research, including genomics, immunology, and molecular biology, have deepened our understanding of the pathogenesis of these diseases. This knowledge supports the development of targeted therapies and treatment innovations. Regulatory incentives provided by orphan drug designations, such as extended market exclusivity, tax credits, and reduced development costs, encourage pharmaceutical companies to invest in research and development for rare diseases. Patient advocacy groups and organizations play a pivotal role in driving research, raising awareness, and advocating for improved access to treatments. Their efforts help accelerate progress in the field of acquired orphan blood diseases. The emergence of novel treatment modalities, including gene therapies, monoclonal antibodies, and immunomodulatory agents, has expanded the range of therapeutic options available for acquired orphan blood diseases. Advances in diagnostic techniques and technologies have led to more accurate and timely diagnoses of acquired orphan blood diseases. This enables early intervention and treatment.
Key Market Drivers
Advancements in Medical Research
Medical research has deepened our understanding of the underlying mechanisms and pathophysiology of acquired orphan blood diseases. This knowledge helps identify novel therapeutic targets and pathways for intervention. For instance, in February 2025, a new 3D lung model will revolutionize respiratory medicine research. This advanced model replicates lung structure and function, enabling more accurate drug testing and disease modeling. It offers a powerful tool for studying conditions like asthma and COPD, accelerating the development of innovative treatments and improving patient outcomes. Research efforts have led to the development of targeted therapies that address the specific molecular and cellular abnormalities associated with these diseases. Targeted treatments often offer better efficacy and safety profiles compared to traditional therapies. Research has contributed to the identification of biomarkers and genetic markers associated with these diseases. Biomarkers enable early diagnosis, disease monitoring, and personalized treatment approaches. Ongoing research supports drug discovery and development programs aimed at creating innovative and more effective therapeutics. This includes the development of monoclonal antibodies, gene therapies, and small molecule drugs. Advances in clinical trial design and methodology, including adaptive trials and innovative endpoints, facilitate the evaluation of potential therapies. Clinical trials are essential for assessing treatment safety and efficacy. Medical research has led to the creation of patient registries and databases for rare diseases. These registries facilitate data collection, epidemiological studies, and clinical trial recruitment.
Key Market Challenges
Access to Treatment
Rare diseases, including acquired orphan blood diseases, often have a limited number of treatment options available due to their low prevalence. This scarcity of therapies can make it difficult for patients to access appropriate treatments. Many orphan drugs and emerging therapies are associated with high treatment costs. The rarity of these diseases and the costs of research and development often result in expensive therapies, making them financially burdensome for patients and healthcare systems. Acquired orphan blood diseases are not always well-known among healthcare providers and the public. This lack of awareness can lead to delayed diagnoses and difficulty in accessing specialized treatments. Access to advanced medical treatments can vary significantly based on geographic location. Patients in remote or underserved areas may face challenges in accessing specialized healthcare facilities and therapies. In some regions, the healthcare infrastructure may not be adequately equipped to diagnose and manage acquired orphan blood diseases. This can lead to delays in treatment initiation. The availability and extent of insurance coverage for orphan blood disease therapies can vary widely. In some cases, insurance plans may not fully cover the cost of treatment, leaving patients with high out-of-pocket expenses. Pharmaceutical companies may encounter challenges in obtaining reimbursement approvals for orphan drugs, leading to delays in patients' access to treatment. Specialized treatment centers and healthcare providers with expertise in acquired orphan blood diseases may be concentrated in urban areas, making it challenging for patients in rural regions to access care.
Key Market Trends
Expanded Access Programs
(Expanded access programs) EAPs prioritize the needs of patients who have limited or no alternative treatment options. These programs provide access to potentially life-saving therapies, reflecting a patient-centered approach to healthcare. EAPs allow patients to access investigational or emerging therapies that are still in the clinical trial or regulatory approval phases. This can be especially valuable for patients with rare and life-threatening diseases like acquired orphan blood diseases. EAPs are driven by ethical considerations, recognizing the urgency of providing treatments to patients who may not qualify for clinical trials or cannot wait for regulatory approvals. They provide hope and potential benefits to those in need. Patients who do not respond to standard therapies or who have contraindications to traditional treatments may find EAPs as a viable option for accessing novel treatments tailored to their specific condition. EAPs allow for the collection of real-world data and evidence regarding the safety and efficacy of therapies in diverse patient populations. This information can complement clinical trial data and inform treatment decisions. EAPs involve close collaboration between physicians, patients, pharmaceutical companies, and regulatory authorities. This collaboration ensures that patients receive the most appropriate and personalized care.
Key Market Players
Alexion Pharmaceuticals, Inc.
Amgen, Inc.
Celgene Corporation
Eli Lilly and Company
Sanofi S.A.
GlaxoSmithKline plc,
Cyclacel Pharmaceuticals, Inc.
Onconova Therapeutics, Inc.
Incyte Corporation,
CTI BioPharma Corp
Report Scope:
In this report, the Global Acquired Orphan Blood Diseases Therapeutics Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:
Acquired Orphan Blood Diseases Therapeutics Market, By Therapy:
Recombinant Factor
Immunoglobulin Infusion Therapy
Activated Prothrombin Complex Concentrate
Thrombopoietin Receptor Agonists
Others
Acquired Orphan Blood Diseases Therapeutics Market, By Disease Indication:
Acquired Agranulocytosis
Acquired Hemophilia
Acquired Von Willebrand Syndrome
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Myelodysplastic Syndrome
Others
Acquired Orphan Blood Diseases Therapeutics Market, By Distribution Channel:
Hospital Pharmacy
Retail Pharmacy
Others
Acquired Orphan Blood Diseases Therapeutics Market, By region:
North America
United States
Canada
Mexico
Asia-Pacific
China
India
South Korea
Australia
Japan
Europe
Germany
France
United Kingdom
Spain
Italy
South America
Brazil
Argentina
Colombia
Middle East & Africa
South Africa
Saudi Arabia
UAE
Competitive Landscape
Company Profiles: Detailed analysis of the major companies present in the Global Acquired Orphan Blood Diseases Therapeutics Market.
Available Customizations:
Global Acquired Orphan Blood Diseases Therapeutics 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:
Company Information
Detailed analysis and profiling of additional market players (up to five).
Table of Contents
1. Product Overview
1.1. Market Definition
1.2. Scope of the Market
1.2.1. Markets Covered
1.2.2. Years Considered for Study
1.2.3. Key Market Segmentations
2. Research Methodology
2.1. Objective of the Study
2.2. Baseline Methodology
2.3. Key Industry Partners
2.4. Major Association and Secondary Sources
2.5. Forecasting Methodology
2.6. Data Triangulation & Validation
2.7. Assumptions and Limitations
3. Executive Summary
3.1. Overview of the Market
3.2. Overview of Key Market Segmentations
3.3. Overview of Key Market Players
3.4. Overview of Key Regions/Countries
3.5. Overview of Market Drivers, Challenges, Trends
4. Voice of Customer
5. Global Acquired Orphan Blood Diseases Therapeutics Market Outlook
