再生不良性貧血市場 - 市場の洞察、疫学、市場予測：2034年

主要7ヶ国における再生不良性貧血の総発生症例数は、2023年には約2,500例であり、予測期間中に増加すると予測されます。主要7ヶ国Mの中で再生不良性貧血の罹患数が最も多いのは日本であり、2023年には800例でした。推計によると、再生不良性貧血は高齢者に多く、2020年の主要7ヶ国の罹患者数は60歳未満が60％を占め、60歳未満が40％でした。2023年、EU4ヶ国と英国の中で再生不良性貧血症例数が最も多いのはドイツであり、スペインは最下位です。

主要7ヶ国における再生不良性貧血の総市場規模は、2023年に約2億7,000万米ドルとなっており、予測期間中（2024年～2034年）に拡大すると予測されます。米国が約1億9,000万米ドルで最大となっており、EU4ヶ国と英国においては、2023年の市場規模はドイツが最大で、英国は最下位です。治療薬の中では、プロマクタが2034年までに主要7ヶ国で最も高い収益を上げると予想されます。

再生不良性貧血は、初期造血細胞の自己免疫破壊を特徴とする稀で重篤な非悪性疾患です。世界の罹患率は人口100万人当たり年間0.7～7.4人で、欧米に比べアジアでの罹患率が高くなっています。主な治療法は骨髄移植と免疫抑制療法です。最も一般的に使用される免疫抑制剤は、抗胸腺細胞グロブリン（ATG）とシクロスポリンであり、単独または併用で投与されます。40歳以上の患者では、フロントラインISTが一般的な標準治療です。逆に、SAAと診断された小児や若年成人、特にMSDを有する患者に対しては、依然としてBMTが望ましい治療法です。

再生不良性貧血とは、傷害による慢性的な原発性造血不全の症候群を指し、骨髄中の造血前駆体の減少または消失、およびそれに伴う汎血球減少をもたらします。再生不良性貧血の症状は、骨髄が十分な血球を産生できないために起こります。軽度の症状で長年安定している人もいれば、重篤な症状で生命を脅かす合併症にまで進行する人もいます。貧血では、疲れやすい、睡眠不足、脱力感、ふらつき、めまい、神経過敏、頭痛、皮膚の青白さ、呼吸困難、胸痛などの心臓症状がみられることがあります。

再生不良性貧血はまれな疾患であり、他の骨髄不全症候群との重複もあるため、診断に難渋します。出血や感染症に関連する所見を除き、検査では主にリンパ節腫脹がないこと、脾臓や肝臓の腫大がないこと、他の臓器に浸潤がないことなどの陰性所見を示します。再生不良性貧血の診断には、骨髄吸引と生検が不可欠です。細胞遺伝学的異常は、典型的な再生不良性貧血患者の最大12-15%で検出されるため、すべての再生不良性貧血患者に対して系統的な細胞遺伝学的検査が不可欠です。次世代シーケンシング（NGS）を含む分子解析は、疾患の病態生理を理解する上でますます重要となってきています。

当レポートは、主要7ヶ国市場における再生不良性貧血について調査し、過去の疫学と予測、再生不良性貧血市場動向を詳細に紹介しています。また、現在の治療法、新薬、個々の治療法の市場シェア、2020年から2034年までの主要7ヶ国の再生不良性貧血市場規模の現状と予測を提供しています。現在の再生不良性貧血の治療法/アルゴリズムやアンメットメディカルニーズも網羅し、最良の機会を発掘し、市場の可能性を評価します。

目次

第1章 重要な洞察

第2章 レポートのイントロダクション

第3章 再生不良性貧血のエグゼクティブサマリー

第4章 再生不良性貧血市場概要

• 治療法別市場シェア（%）主要7ヶ国における2020年の再生不良性貧血の分布
• 治療法別市場シェア（%）主要7ヶ国における2034年の再生不良性貧血の分布

第5章 主要な出来事

第6章 疫学と市場予測調査手法

第7章 疾患の背景と概要

• イントロダクション
• 再生不良性貧血の兆候と症状
• 再生不良性貧血の危険因子
• 再生不良性貧血の病因
• 再生不良性貧血の診断

第8章 再生不良性貧血の治療と管理

• 治療アルゴリズム
• 再生不良性貧血の治療ガイドライン

第9章 疫学と患者数

• 主な調査結果
• 仮定と根拠
• 主要7ヶ国における再生不良性貧血の総発生件数
• 米国
• EU4ヶ国と英国
• 日本

第10章 患者動向

• 概要

第11章 上市済み治療法

第12章 新興薬剤

第13章 網膜色素変性症：市場分析

• 主な調査結果
• 市場の見通し
• コンジョイント分析
• 主要な市場予測の前提条件
• 主要7ヶ国における再生不良性貧血の総市場規模
• 米国の市場規模
• EU4ヶ国と英国の市場規模
• 日本市場規模

第14章 アンメットニーズ

第12章 SWOT分析

第13章 KOLのビュー

第14章 市場アクセスと償還

• 米国
• EU4ヶ国と英国
• 日本
• 再生不良性貧血における償還シナリオ

第15章 付録

第16章 DelveInsightのサービス内容

第17章 免責事項

第18章 DelveInsightについて

List of Tables

• Table 1: Summary of Aplastic Anemia Market and Epidemiology (2020-2034)
• Table 2: Summary of aplastic anemia pathogenesis.
• Table 3: Key recommendation for treatment of severe aplastic anemia:
• Table 4: Total Incident Cases of Aplastic Anemia in the 7MM (2020-2034)
• Table 5: Total Incident Cases of Aplastic Anemia in the US (2020-2034)
• Table 6: Severity-specific Cases of Aplastic Anemia in the US (2020-2034)
• Table 7: Age-specific Cases of Aplastic Anemia in the US (2020-2034)
• Table 8: Total Incident Cases of Aplastic Anemia in the 7MM (2020-2034)
• Table 9: Severity-specific Cases of Aplastic Anemia in EU4 and the UK (2020-2034)
• Table 10: Age-specific Cases of Aplastic Anemia in EU4 and the UK (2020-2034)
• Table 11: Total Incident Cases of Aplastic Anemia in Japan (2020-2034)
• Table 12: Severity-specific Cases of Aplastic Anemia in Japan (2020-2034)
• Table 13: Age-specific Cases of Aplastic Anemia in Japan (2020-2034)
• Table 14: Comparison of Marketed Drugs
• Table 16: Eltrombopag olamine, Clinical Trial Description, 2024
• Table 17: Eltrombopag choline, Clinical Trial Description, 2024
• Table 18: Romiplostim, Clinical Trial Description, 2024
• Table 19: Comparison of Emerging Drugs
• Table 20: REGN7257, Clinical Trial Description, 2024
• Table 21: OMISIRGE (OMIDUBICEL), Clinical Trial Description, 2024
• Table 22: Key Market Forecast Assumption of Aplastic Anemia in the United States
• Table 23: Key Market Forecast Assumption of Aplastic Anemia in EU4 and the UK
• Table 24: Key Market Forecast Assumption of Aplastic Anemia in Japan
• Table 25: Total Market Size of Aplastic Anemia in the 7MM, USD million (2020-2034)
• Table 26: Total Market Size of Aplastic Anemia in the US, USD million (2020-2034)
• Table 27: Market Size of Aplastic Anemia by Therapies in the US, USD million (2020-2034)
• Table 28: Total Market Size of Aplastic Anemia in EU4 and the UK, USD million (2020-2034)
• Table 29: Market Size of Aplastic Anemia by Therapies in EU4 and the UK, USD million (2020-2034)
• Table 30: Total Market Size of Aplastic Anemia in Japan, USD million (2020-2034)
• Table 31: Market Size of Aplastic Anemia by Therapies in Japan, USD million (2020-2034)
• Table 32: SMR Values of Drugs for Aplastic Anemia
• Table 33: ASMR Values of Drugs for Aplastic Anemia
• Table 34: AIFA Assessment for Aplastic Anemia Therapies

List of Figures

• Figure 1: Difference Between Normal Blood Cells and Aplastic Cells
• Figure 2: Symptoms of Aplastic Anemia
• Figure 3: Risk Factors of Aplastic Anemia
• Figure 4: Stimulating and Inhibitory Factors Affecting Aplastic Bone Marrow
• Figure 5: Pathophysiology of Immune Dysfunction
• Figure 6: T Lymphocytes and Their Secreted Cytokines Pathology of Aplastic Anemia
• Figure 7: Stepwise Diagnostic Phases of Acquired Aplastic Anemia.
• Figure 8: A Stepwise Approach to Screening and Diagnosing of Aplastic Anemia
• Figure 9: Treatment Algorithms for Patients With Immune Aplastic Anemia
• Figure 10: Total Incident Cases of Aplastic Anemia in the 7MM (2020-2034)
• Figure 11: Total Incident Cases of Aplastic Anemia in the US (2020-2034)
• Figure 12: Severity-specific Cases of Aplastic Anemia in the US (2020-2034)
• Figure 13: Age-specific Cases of Aplastic Anemia in the US (2020-2034)
• Figure 14: Total Incident Cases of Aplastic Anemia in EU4 and the UK (2020-2034)
• Figure 15: Severity-specific Cases of Aplastic Anemia in EU4 and the UK (2020-2034)
• Figure 16: Age-specific Cases of Aplastic Anemia in EU4 and the UK (2020-2034)
• Figure 17: Total Incident Cases of Aplastic Anemia in Japan (2020-2034)
• Figure 18: Severity-specific Cases of Aplastic Anemia in Japan (2020-2034)
• Figure 19: Age-specific Cases of Aplastic Anemia in Japan (2020-2034)
• Figure 20: Total Market Size of Aplastic Anemia in the 7MM (2020-2034)
• Figure 21: Total Market Size of Aplastic Anemia in the US (2020-2034)
• Figure 22: Market Size of Aplastic Anemia by Therapies in the US (2020-2034)
• Figure 23: Total Market Size of Aplastic Anemia in EU4 and the UK (2020-2034)
• Figure 24: Market Size of Aplastic Anemia by Therapies in EU4 and the UK (2020-2034)
• Figure 25: Total Market Size of Aplastic Anemia in Japan (2020-2034)
• Figure 26: Market Size of Aplastic Anemia by Therapies in Japan (2020-2034)
• Figure 27: Health Technology Assessment
• Figure 28: Reimbursement Process in Germany
• Figure 29: Reimbursement Process in France
• Figure 30: Reimbursement Process in Italy
• Figure 31: Reimbursement Process in Spain
• Figure 32: Reimbursement Process in the United Kingdom
• Figure 33: Reimbursement Process in Japan

Key Highlights:

• In the 7MM, United States accounted for the largest market size in 2023, with approximately USD ~190 million.
• Aplastic anemia is a rare and severe nonmalignant disease characterized by autoimmune destruction of early hematopoietic cells. Its global incidence rate ranges from 0.7-7.4 cases per million inhabitants per year, with higher rates in Asia compared to Europe and the United States.
• The two main forms of specific treatment are bone marrow transplantation and immunosuppressive therapies. The two most commonly used immunosuppressive agents, given alone or in combination, are antithymocyte globulin (ATG) and cyclosporine. In the context of patients aged 40 years and above, frontline IST stands as the prevailing standard of care. Conversely, BMT remains the preferred therapeutic avenue for children and young adults diagnosed with SAA, particularly those possessing an MSD.
• THYMOGLOBULINE (rabbit ATG) was developed by Sanofi and approved in Japan in 2008 for moderate to VSAA.
• The landscape of therapies for SAA witnessed a transformative moment in 2014 with the approval of PROMACTA (eltrombopag), a thrombopoietin receptor agonist, by the FDA for SAA cases inadequately responsive to initial IST, and later got approved in Europe and Japan in 2015 and 2017, respectively, under the brand name REVOLADE. Subsequently, in 2018, the US FDA approved PROMACTA as a first-line treatment for SAA.
• Another Tpo receptor agonist, ROMIPLATE, was granted approval in Japan in June 2019 for aplastic anemia cases where conventional treatments have proven insufficient. Subsequently, in September 2023, Kyowa Kirin disclosed the expanded approval of ROMIPLATE as a first-line treatment for aplastic anemia in Japan.
• Key players, such as Regeneron Pharmaceuticals, are working on REGN7257 for refractory or relapsed SAA after immunosuppressive therapy, and Gamida Cell is developing umbilical cord blood-derived stem and progenitor cells to enhance engraftment and improve transplant outcomes in unrelated umbilical cord blood transplantation for SAA.
• PROMACTA held the leading market share in the 7MM in 2023, boasting a market size of around USD ~160 million. This figure is projected to experience further growth by the year 2034.
• Despite the advancements in treating aplastic anemia, there remain challenges in finding effective and well-tolerated treatments, especially for patients who do not respond to standard therapies or for those who may not be suitable candidates for more aggressive interventions.
• The unmet need in aplastic anemia continues to be addressed through novel therapies like eltrombopag and ROMIPLATE, but the limited emerging pipeline underscores the necessity for further research and development to expand treatment options for this challenging condition.

DelveInsight's "Aplastic Anemia - Market Insights, Epidemiology and Market Forecast - 2034" report delivers an in-depth understanding of the aplastic anemia, historical and forecasted epidemiology as well as the aplastic anemia market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The aplastic anemia market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM aplastic anemia market size from 2020 to 2034. The report also covers current aplastic anemia treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's potential.

Geography Covered:

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Study Period: 2020-2034

Aplastic Anemia Disease Understanding and Treatment Algorithm

Aplastic Anemia Overview

Aplastic anemia refers to the syndrome of chronic primary hematopoietic failure from injury leading to diminished or absent hematopoietic precursors in the bone marrow and attendant pancytopenia. The symptoms of aplastic anemia occur because the bone marrow fails to produce enough blood cells. Some individuals may have mild symptoms that remain stable for many years; others may have serious symptoms that can progress to life-threatening complications. Individuals with anemia may experience tiredness, increased need for sleep, weakness, lightheadedness, dizziness, irritability, headaches, pale skin color, difficulty breathing, and cardiac symptoms like chest pain.

Aplastic Anemia Diagnosis

The rarity of aplastic anemia, coupled with its overlap with other bone marrow failure syndromes, poses a diagnostic challenge.Except for findings related to bleeding or infections, the examination presents mainly negative characteristics: absence of lymphadenopathy, no enlarged spleen or liver, and no infiltration of any other organ. Bone marrow (BM) aspiration and biopsy are essential procedures for diagnosing aplastic anemia. Cytogenetic abnormalities can be detected in up to 12-15% of otherwise typical aplastic anemia patients, making systematic cytogenetic investigations essential for all aplastic anemia patients. Molecular analysis, including next-generation sequencing (NGS), has become increasingly instrumental in comprehending the pathophysiology of diseases.

Aplastic Anemia Treatment

Treatment of aplastic anemia varies, depending upon the individual's age, general health, and the severity of aplastic anemia. Treatment aims to correct the bone marrow failure, as well as to treat the patient's immediate signs and symptoms. The two main forms of specific treatment are bone marrow transplantation and immunosuppressive therapies. Initial treatment of acquired aplastic anemia may be directed toward improving the symptoms that may result from low blood counts. Such treatment consists of giving red blood cell transfusions to correct anemia, platelet transfusions to treat or prevent serious bleeding, and antibiotics to treat or prevent infections. Supportive care is a critical component in the management of aplastic anemia, aiming to address symptoms, complications, and overall patient well-being. Patients often require regular blood transfusions to alleviate anemia and manage low platelet counts.

Further details related to diagnosis and treatment are provided in the report…

Aplastic Anemia Epidemiology

As the market is derived using a patient-based model, the aplastic anemia epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total incident cases of aplastic anemia, severity-specific cases of aplastic anemia, and age-specific cases of aplastic anemia in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan from 2020 to 2034.

• The total incident cases of aplastic anemia in the 7MM comprised approximately ~2,500 cases in 2023 and are projected to increase during the forecast period.
• Among the 7MM, the Japan accounted for the highest number of incident cases of aplastic anemia, i.e., ~800 cases in 2023.
• According to estimates, aplastic anemia is more common in the older population; the highest incident cases were in age group of =60 years, accounting for ~60% cases, and ~40% were <60 in 2020 in the 7MM.
• In 2023, Among EU4 and the UK, Germany accounted for the largest number of aplastic anemia cases, whereas Spain occupied the bottom of the ladder.

Aplastic Anemia Drug Chapters

The drug chapter segment of the aplastic anemia report encloses a detailed analysis of aplastic anemia marketed drugs and emerging pipeline drugs. It also helps understand the aplastic anemia pivotal clinical trial details, recent and expected market approvals, patent details, each drug's advantages and disadvantages, the latest news, and recent deals and collaborations.

Marketed Drug

PROMACTA/REVOLADE (eltrombopag): Novartis

PROMACTA/REVOLADE (eltrombopag) tablets contain eltrombopag olamine, a small molecule thrombopoietin (TPO) receptor agonist for oral administration. Eltrombopag, sold under the brand name PROMACTA, among others, is a medication used to treat thrombocytopenia and severe aplastic anemia (SAA). Eltrombopag is sold under the brand name REVOLADE outside the US and is marketed by Novartis. In August 2014, the US FDA approved a sNDA for the once-daily use of PROMACTA in patients with severe aplastic anemia (SAA) who had an insufficient response to immunosuppressive therapy. And later in November 2018, the us FDA expanded the label for PROMACTA (eltrombopag) to include first-line treatment for adults and pediatric patients 2 years and older with SAA in combination with IST.

ROMIPLATE (romiplostim; AMG531): Kyowa Kirin/Amgen

ROMIPLATE is composed of recombinant protein acting on the thrombopoietin receptor, which has been licensed from Amgen (K-A) to Kyowa Kirin. It was launched as a drug for idiopathic thrombocytopenic purpura (ITP) in April 2011 and for aplastic anemia in patients who had an inadequate response to conventional therapy in June 2019 in Japan. The drug is composed of recombinant protein stimulating hematopoiesis via acting on the thrombopoietin receptors. It acts directly on megakaryocytic progenitor cells and exerts platelet hematopoietic effects.

Emerging Drug

REGN7257: Regeneron Pharmaceuticals

REGN7257 is a gc cytokine receptor antibody that targets the common g chain (gc; IL-2RG) found in interleukin (IL) receptors for various gc cytokines, including IL-2, IL-4, IL-7, IL-9, IL-15, and IL-21. It was developed to investigate the potential of targeting gc cytokines for the prevention and treatment of T-cell-mediated diseases. By blocking the signaling of gc cytokines, REGN7257 aims to ameliorate T-cell-mediated pathogenesis. The drug REGN7257 is presently undergoing evaluation in a Phase I/II clinical trial to assess its safety and tolerability in patients with severe aplastic anemia (SAA) who have either relapsed after immunosuppressive therapy (IST) or are refractory to IST.

OMISIRGE (omidubicel): Gamida Cell

OMISIRGE (omidubicel-onlv) is a cryopreserved nicotinamide-modified allogeneic hematopoietic progenitor cell therapy derived from cord blood and manufactured utilizing a proprietary NAM-based technology producing enriched HPCs. It is indicated for use in adults and pediatric patients aged 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning. Omisirge has received approval for use in hematological malignancies and is currently undergoing evaluation in an ongoing investigator-sponsored Phase I/II study focusing on patients with severe aplastic anemia.

Aplastic Anemia Market Outlook

In real-world scenarios, the approach to treating NSAA differs significantly from SAA/VSAA. Unlike SAA/VSAA, hematopoietic stem cell transplantation (HSCT) is not employed in NSAA cases. The criteria for determining when to initiate treatment for NSAA are less clearly defined, and there is a lack of comprehensive recommendations for clinical evaluation and therapeutic strategies. The ultimate management of NSAA predominantly relies on the expertise of individual medical centers. Historically, the primary modalities for treating aplastic anemia have been IST and BMT in eligible patients. For individuals with SAA/VSAA deemed suitable for transplant-based interventions, age emerges as a pivotal determinant influencing survival post-matched sibling donor (MSD) allogeneic transplantation. Notably, older patients, often having undergone prior IST, presenting with additional comorbidities, diminished performance status, and an extended duration between diagnosis and BMT, exhibit distinctive challenges in outcomes, with fludarabine-containing regimens offering more promising results in this demographic, albeit based on limited retrospective analyses. In the realm of second-line treatments, patients with aplastic anemia are considered eligible for transplantation in cases of refractory status following first-line IST. IST in the form of horse ATG + CyA has been extensively researched regimen in Europe, Asia, and the United States over several decades. Recent endeavors to enhance the efficacy of horse ATG have involved augmenting immunosuppression with agents such as mycophenolate, sirolimus, rabbit ATG, alemtuzumab, and cyclophosphamide.

Approximately a decade ago, notable progress was made with the approval of thrombopoietin receptor agonists for immune thrombocytopenia, effectively inducing platelet count recovery in refractory cases. Distinguishing itself from erythropoietin and G-CSF, the Tpo receptor is expressed in stem cells. Tpo receptor knockout models revealed significant reductions not only in megakaryocytes and platelets but also in HSCs. In 2014, the US FDA approved PROMACTA for the treatment of aplastic anemia, which then approved in Europe and Japan in 2015 and 2017, respectively, under the brand name REVOLADE. Later, another Tpo receptor agonist, ROMIPLATE, was granted approval in Japan in June 2019 for aplastic anemia cases where conventional treatments have proven insufficient.

With respect to the emerging pipeline, there is a scarcity of advancements in SAA treatment, with only two companies in the 7MM actively engaged in developing therapies-Regeneron Pharmaceuticals with REGN7257 and Gamida Cell with omidubicel. This underscores the crucial need for additional research and development to broaden the spectrum of treatment options for this complex condition.

Detailed market assessment will be provided in the final report

Key Findings

As per DelveInsight's estimates, the potential drugs that can mark a significant change in the forecast period include xx, xx, and others.

• The total market size of aplastic anemia in the 7MM is approximately USD ~270 million in 2023 and is projected to increase during the forecast period (2024-2034).
• Among EU4 and the UK, Germany accounted for the maximum market size in 2023, while the UK occupies the bottom of the ladder.
• Among the therapies, PROMACTA is expected to generate the highest revenue in the 7MM by 2034.

Aplastic Anemia Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2020-2034. A medium uptake is expected by REGN7257.

Further detailed analysis of therapies drug uptake will be provided in the final report …

Aplastic Anemia Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III and Phase II. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for aplastic anemia emerging therapy.

KOL Views

To keep up with current market trends, we take KOLs and SMEs' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry experts contacted for insights on aplastic anemia evolving treatment landscape, patient reliance on conventional therapies, patient's therapy switching acceptability, and drug uptake, along with challenges related to accessibility, include Medical/scientific writers; Professors; MD, FACS, Head of the Departments, Universities, and others.

Delveinsight's analysts connected with 30+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. Centers such as the East Tennessee State University, Dusseldorf Clinics Association, Duke University School of Medicine, etc. were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or aplastic anemia market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Analyst views. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

The analyst analyzes multiple emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry.

In efficacy, the trial's primary and secondary outcome measures are evaluated. Further, the therapies' safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials.

Market Access and Reimbursement

Reimbursement is a crucial factor affecting the drug's market access. Often, the decision to reimburse comes down to the price of the drug relative to the benefit it produces in treated patients. To reduce the healthcare burden of these high-cost therapies, payers and other industry insiders are considering many payment models.

Patients prescribed PROMCATA might be eligible to receive a free 14-day supply by mail while beginning therapy. Additionally, Novartis also provides eligible patients with a temporary supply of PROMACTA through its Voucher Program. Through its Universal Copay Card, eligible patients with private insurance may pay USD 0 per month, with Novartis paying the remaining copay up to USD 15,000 per calendar year. Patients facing financial hardship and/or having no third-party insurance coverage might be eligible to receive PROMACTA for free through the Novartis Patient Assistance Foundation. About 99% of the patients being prescribed PROMACTA have it included in their health coverage, with more than 70% of Medicare and commercially insured patients paying less than USD 10,000 in out-of-pocket costs.

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Scope of the Report:

• The report covers a segment of key events, an executive summary, descriptive overview of Aplastic Anemia, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight into the epidemiology segments and forecasts, disease progression, and treatment guidelines has been provided.
• Additionally, an all-inclusive account of the current and emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current treatment landscape.
• A detailed review of the aplastic anemia market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help shape and drive the 7MM aplastic anemia market.

Aplastic Anemia Report Insights

• Patient Population
• Therapeutic Approaches
• Aplastic Anemia Pipeline Analysis
• Aplastic Anemia Market Size and Trends
• Existing and future Market Opportunity

Aplastic Anemia Report Key Strengths

• Eleven Years Forecast
• The 7MM Coverage
• Aplastic Anemia Epidemiology Segmentation
• Key Cross Competition
• Drugs Uptake and Key Market Forecast Assumptions

Aplastic Anemia Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)

FAQs

• What was the aplastic anemia total market size, the market size by therapies, and market share (%) distribution in 2020, and what would it look like in 2034? What are the contributing factors for this growth?
• Which class is going to be the largest contributor in 2034?
• What will be the market size of PROMACTA in 2025?
• What are the pricing variations among different geographies for approved and off-label therapies?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?
• What are the disease risks, burdens, and unmet needs of aplastic anemia? What will be the growth opportunities across the 7MM concerning the patient population of aplastic anemia?
• What is the historical and forecasted aplastic anemia patient pool in the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan?
• What are the current options for the treatment of aplastic anemia? What are the current guidelines for treating aplastic anemia in the US and Europe?
• What are the recent novel therapies, targets, mechanisms of action, and technologies being developed to overcome the limitations of existing therapies?
• What key designations have been granted for the emerging therapies for aplastic anemia?
• Patient acceptability in terms of preferred treatment options as per real-world scenarios?

Reasons to Buy:

• The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving the aplastic anemia market.
• Insights on patient burden/disease incidence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the Analyst view section to provide visibility around leading classes.
• Highlights of Access and Reimbursement policies of current therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.
• To understand Key Opinion Leaders' perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.

Table of Contents

1. Key Insights

2. Report Introduction

3. Executive Summary of Aplastic Anemia

4. Aplastic Anemia Market Overview at a Glance

• 4.1. Market Share by Therapies (%) Distribution of Aplastic Anemia in 2020 in the 7MM
• 4.2. Market Share by Therapies (%) Distribution of Aplastic Anemia in 2034 in the 7MM

5. Key Events

6. Epidemiology and Market Forecast Methodology

7. Disease Background and Overview

• 7.1. Introduction
• 7.2. Signs and Symptoms of Aplastic Anemia
• 7.3. Risk Factors of Aplastic Anemia
• 7.4. Pathogenesis of Aplastic Anemia
  • 7.4.1. Deficiencies in Hematopoietic Stem and Progenitor Cells (Hspcs)
  • 7.4.2. Abnormal Bone Marrow Microenvironment
  • 7.4.3. Immune Dysfunction
  • 7.4.4. Genetic Background
• 7.5. Diagnosis of Aplastic Anemia
  • 7.5.1. Diagnosis Confirmation
  • 7.5.2. Diagnostic Algorithms
  • 7.5.3. British Society of Haemotology Guidelines for the diagnosis and management of adult aplastic anemia:

8. Treatment and Management of Aplastic Anemia

• 8.1. Treatment Algorithm
• 8.2. Treatment Guidelines for Aplastic Anemia
  • 8.2.1. The European Society for Blood and Marrow Transplantation (EBMT) Handbook: Hematopoietic Stem Cell Transplantation and Cellular Therapies Guidelines
  • 8.2.2. British Society of Hematology Guidelines for the diagnosis and management of adult aplastic anemia:

9. Epidemiology and Patient Population

• 9.1. Key Findings
• 9.2. Assumptions and Rationale
• 9.3. Total Incident Cases of Aplastic Anemia in the 7MM
• 9.4. The United States
  • 9.4.1. Total Incident Cases of Aplastic Anemia in the US
  • 9.4.2. Severity-specific Cases of Aplastic Anemia in the United States
  • 9.4.3. Age-specific Cases of Aplastic Anemia in the United States
• 9.5. EU4 and the UK
  • 9.5.1. Total Incident Cases of Aplastic Anemia in EU4 and the UK
  • 9.5.2. Severity-specific Cases of Aplastic Anemia in EU4 and the UK
  • 9.5.3. Age-specific Cases of Aplastic Anemia in EU4 and the UK
• 9.6. Japan
  • 9.6.1. Total Incident Cases of Aplastic Anemia in Japan
  • 9.6.2. Severity-specific Cases of Aplastic Anemia in Japan
  • 9.6.3. Age-specific Cases of Aplastic Anemia in Japan

10. Patient Journey

• 10.1. Description

11. Marketed Therapies

• 11.1. Key Cross of Marketed Therapies
• 11.2. PROMACTA/REVOLADE (eltrombopag olamine): Novartis
  • 11.2.1. Product Description
  • 11.2.2. Regulatory Milestones
  • 11.2.3. Other Developmental Activities
  • 11.2.4. Clinical Development
  • 11.2.5. Safety and Efficacy
• 11.3. ALVAIZ (eltrombopag choline): Teva Pharmaceuticals
  • 11.3.1. Product Description
  • 11.3.2. Regulatory Milestones
  • 11.3.3. Clinical Development
  • 11.3.4. Safety and Efficacy
• 11.4. ROMIPLATE (romiplostim; AMG531): Kyowa Kirin/Amgen
  • 11.4.1. Product Description
  • 11.4.2. Regulatory Milestones
  • 11.4.3. Other Developmental Activites
  • 11.4.4. Clinical Development
  • 11.4.5. Safety and Efficacy

12. Emerging Drugs

• 12.1. Key Cross of Emerging Therapies
• 12.2. REGN7257: Regeneron Pharmaceuticals
  • 12.2.1. Product Description
  • 12.2.2. Other Developmental Activities
    • 12.2.2.1. Clinical Trial Information
• 12.3. OMISIRGE (omidubicel): Gamida Cell
  • 12.3.1. Product Description
  • 12.3.2. Other Developmental Activities
    • 12.3.2.1. Clinical Trial Information
  • 12.3.3. Safety and Efficacy

13. Retinitis Pigmentosa: Market Analysis

• 13.1. Key Findings
• 13.2. Market Outlook
• 13.3. Conjoint Analysis
• 13.4. Key Market Forecast Assumptions
• 13.5. Total Market Size of Aplastic Anemia in the 7MM
• 13.6. United States Market Size
  • 13.6.1. Total Market Size of Aplastic Anemia in the United States
  • 13.6.2. Market Size of Aplastic Anemia by Therapies in the United States
• 13.7. EU4 and the UK Market Size
  • 13.7.1. Total Market Size of Aplastic Anemia in EU4 and the UK
  • 13.7.2. Market Size of Aplastic Anemia by Therapies in EU4 and the UK
• 13.8. Japan Market Size
  • 13.8.1. Total Market Size of Aplastic Anemia in Japan
  • 13.8.2. Market Size of Aplastic Anemia by Therapies in Japan

14. Unmet Needs

12. SWOT Analysis

13. KOL Views

14. Market Access and Reimbursement

• 14.1. United States
  • 14.1.1. Centre for Medicare and Medicaid Services (CMS)
• 14.2. EU4 and the UK
  • 14.2.1. Germany
  • 14.2.2. France
  • 14.2.3. Italy
  • 14.2.4. Spain
  • 14.2.5. United Kingdom
• 14.3. Japan
  • 14.3.1. MHLW
• 14.4. Reimbursement Scenario in Aplastic Anemia

15. Appendix

• 15.1. Bibliography
• 15.2. Report Methodology

16. DelveInsight Capabilities

17. Disclaimer

18. About DelveInsight