本態性血小板血症（ET）市場 - 市場の洞察、疫学、市場予測：2034年

本態性血小板血症（ET）は、血液中の血小板数の増加を特徴とする慢性骨髄増殖性新生物（MPN）です。50歳以上の女性に多く発症します。ETの主な原因は、JAK2、CALR、またはMPL遺伝子の変異別造血細胞の過剰産生です。これらの遺伝子は、骨髄増殖性新生物の開発における役割から「ドライバー変異」として知られています。成人の90％がJAK2、CALR、またはMPLの変異を有するが、分子トリプルワイルドタイプの小児を認めることも珍しくありません。

ET患者の多くは無症状です。そのため、血液検査で血小板数が多いことが判明した後、定期検診の一環として診断されることが多いです。症状がある場合、疲労を伴うこともあれば、小血管障害や大血管障害、出血に関連することもあります。ET患者の生存期間の中央値は20年です。しかし、発症の年齢別異なり、その結果、60歳未満の患者の生存期間の中央値は33年に近づいています。罹患率および死亡率の最も一般的な原因は血栓症であり、ET患者の20％にみられるのに対し、出血性合併症は10％にみられます。現在の市場シナリオでは、本態性血小板血症の治療は、血栓のリスクを低減し、個々のリスクレベルに合わせた様々な治療オプションで症状を管理することに重点を置いています。

アスピリンは血栓形成の予防に役立つため、低リスク、中リスク、高リスクの患者に一般的に推奨されています。第一選択治療では、血小板数の減少や合併症の予防を目的としたヒドロキシ尿素、アナグレリド、インターフェロンαなどの細胞還元療法にアスピリンを併用することが多いです。細胞還元療法は、血小板数が100万/μLを超える患者の出血リスクを低下させるために行われます。場合別は、患者の状態や他の治療に対する反応に応じて、ブスルファン、クロピドグレル、抗凝固薬などの追加薬が処方されることもあります。

現在、ETの治療に特化して承認されている薬剤は1種類のみで、欧州でのみ認可されています。一方、米国ではETの治療薬としてFDAの承認を受けているものはありません。

2024年8月、pharmaand GmbH（pharma&）は、ECが成人のET患者に対する単剤治療薬としてPEGASYS（peginterferon alfa-2a）のタイプIIバリエーションを販売承認したと発表しました。

主要7ヶ国における本態性血小板血症の総市場規模では、米国が最も高い市場シェアを占め、2024年には約60%を占め、次いで日本が続きます。EU4ヶ国と英国の中では、フランスと英国が2024年に最も高い市場規模を占めています。米国は2024年に約2億5,000万米ドルの市場規模を占めました。新興治療薬の中では、BESREMIとBomedemstatがET市場を一変させる可能性のある薬剤とみられています。2034年までに、新興治療薬の中で最も高い収益を上げるのはBESREMIと予想されます。

当レポートでは、主要7ヶ国における本態性血小板血症（ET）市場について調査し、市場の概要とともに、疫学、患者動向、新たな治療法、2034年までの市場規模予測、および医療のアンメットニーズなどを提供しています。

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 本態性血小板血症市場概要

第4章 調査手法

第5章 エグゼクティブサマリー

第6章 重要な出来事

第7章 疾患の背景と概要

• イントロダクション
• 病因
• リスク要因
• 兆候と症状
• 病因
• 病態生理学
• 予後
• ETにおけるMPNドライバー変異の有病率と表現型のケーススタディ
• 診断

第8章 ETの治療と管理

• 治療の概要
• 治療アルゴリズム
• 治療ガイドライン

第9章 疫学と患者人口

• 主な調査結果
• 仮定と根拠
• 主要7ヶ国におけるETの診断された有病症例の総数
• 主要7ヶ国におけるETの治療症例総数
• 米国
• EU4ヶ国と英国
• 日本

第10章 患者動向

第11章 上市済み治療薬

第12章 新たな治療薬

第13章 本態性血小板血症（ET）：主要7ヶ国市場分析

• 主な調査結果
• 市場見通し
• 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ETの市場規模
• 米国
• EU4ヶ国と英国
• 日本

第14章 KOLの見解

第15章 SWOT分析

第16章 アンメットニーズ

第17章 市場アクセスと償還

• 米国
• EU4ヶ国と英国
• 日本

第18章 付録

第19章 DelveInsightのサービス内容

第20章 免責事項

List of Tables

• Table 1: Summary of ET Market and Epidemiology (2020-2034)
• Table 2: IPSET Thrombosis Score
• Table 3: Current Prognostic Models for Overall Survival in ET
• Table 4: International Consensus Classification Diagnostic Criteria for ET and Post-ET MF
• Table 5: Total Diagnosed Prevalent Cases of ET in 7MM (2020-2034)
• Table 6: Total Treated Cases of ET in 7MM (2020-2034)
• Table 7: Total Diagnosed Prevalent Cases of ET in the United States (2020-2034)
• Table 8: Symptom-specific Cases of ET in the United States (2020-2034)
• Table 9: Gender-specific Cases of ET in the United States (2020-2034)
• Table 10: Mutation-specific Cases of ET in the United States (2020-2034)
• Table 11: Risk-specific Cases of ET in the United States (2020-2034)
• Table 12: Age-specific Cases of ET in the United States (2020-2034)
• Table 13: Total Treated Cases of ET in the United States (2020-2034)
• Table 14: Total Diagnosed Prevalent Cases of ET in EU4 and the UK (2020-2034)
• Table 15: Gender-specific cases of ET in EU4 and the UK (2020-2034)
• Table 16: Gender-specific cases of ET in EU4 and the UK (2020-2034)
• Table 17:Mutation-specific Cases of ET in EU4 and the UK (2020-2034)
• Table 18:Risk-specific Cases of ET in EU4 and the UK (2020-2034)
• Table 19: Age-specific Cases of ET in EU4 and the UK (2020-2034)
• Table 20: Total Treated Cases of ET in EU4 and the UK (2020-2034)
• Table 21:Total Diagnosed Prevalent Cases of ET in Japan (2020-2034)
• Table 22:Symptom-specific Cases of ET in the United States (2020-2034)
• Table 23:Gender-specific Cases of ET in Japan (2020-2034)
• Table 24: Mutation-specific Cases of ET in Japan (2020-2034)
• Table 25: Risk-specific Cases of ET in Japan (2020-2034)
• Table 26: Age-specific Cases of ET in Japan (2020-2034)
• Table 27: Total Treated Cases of ET in Japan (2020-2034)
• Table 28: PEGASYS (peginterferon alfa-2a) Clinical Trial Description, 2025
• Table 29: Key Cross (Emerging Therapies)
• Table 30: BESREMi (Ropeginterferon Alfa-2b) Clinical Trial Description, 2025
• Table 31: Bomedemstat (MK-3543/IMG-7289): Clinical Trial Description, 2025
• Table 32: Key Market Forecast Assumption of ET in the United States
• Table 33: Key Market Forecast Assumption of ET in EU4 and the UK
• Table 34: Key Market Forecast Assumption of ET in Japan
• Table 35: Total Market Size of ET in the 7MM, in USD Million (2020-2034)
• Table 36: Total Market Size of ET in the United States, in USD Million (2020-2034)
• Table 37: Market Size of ET by Therapies in the United States, in USD Million (2020-2034)
• Table 38: Total Market Size of ET in EU4 and the UK, in USD Million (2020-2034)
• Table 39: Market Size of ET by Therapies in EU4 and the UK, in USD million (2020-2034)
• Table 40: Total Market Size of Essential Thrombocythemia in Japan, in USD Million (2020-2034)
• Table 41: Market Size of ET by Therapies in the United States, in USD Million (2020-2034)

List of Figures

• Figure 1: Risk Factors Associated With ET
• Figure 2: Signs and Symptoms of ET
• Figure 3: Symptom of ET, Enlarged Spleen
• Figure 4: AAA Survival Model in ET. Overall Survival Data Among 598 MayoClinic Patients Stratified by Age, ANC, and ALC, Median Follow-up of 8.4 Years
• Figure 5: Thrombosis Risk Stratification in ET
• Figure 6: Practical Diagnostic Algorithm for MPNs
• Figure 7: Diagnostic Algorithm of ET
• Figure 8: Current Treatment Algorithm for ET
• Figure 9: NCCN Guidelines Version 2024
• Figure 10: NCCN Guidelines Version 2024
• Figure 11: Total Diagnosed Prevalent Cases of ET in 7MM (2020-2034)
• Figure 12: Total Treated Cases of ET in 7MM (2020-2034)
• Figure 13: Total Diagnosed Prevalent Cases of ET in the United States (2020-2034)
• Figure 14: Symptom-specific cases of ET in the United States (2020-2034)
• Figure 15: Gender-specific Cases of ET in the United States (2020-2034)
• Figure 16: Mutation-specific Cases of ET in the United States (2020-2034)
• Figure 17: Risk-specific Cases of ET in the United States (2020-2034)
• Figure 18: Age-specific Cases of ET in the United States (2020-2034)
• Figure 19: Total Treated Cases of ET in the United States (2020-2034)
• Figure 20: Total Diagnosed Prevalent Cases of ET in EU4 and the UK (2020-2034)
• Figure 21: Symptom-specific cases of ET in EU4 and the UK (2020-2034)
• Figure 22: Gender-specific Cases of ET in EU4 and the UK (2020-2034)
• Figure 23: Mutation-specific Cases of ET in EU4 and the UK (2020-2034)
• Figure 24: Risk-specific Cases of ET in EU4 and the UK (2020-2034)
• Figure 25: Age-specific Cases of ET in EU4 and the UK (2020-2034)
• Figure 26: Total Treated Cases of ET in EU4 and the UK (2020-2034)
• Figure 27: Total Diagnosed Prevalent Cases of ET in Japan (2020-2034)
• Figure 28: Symptom-specific cases of ET in Japan (2020-2034)
• Figure 29: Gender-specific Cases of ET in Japan (2020-2034)
• Figure 30: Mutation-specific Cases of ET in Japan (2020-2034)
• Figure 31: Risk-specific Cases of ET in Japan (2020-2034)
• Figure 32: Age-specific Cases of ET in Japan (2020-2034)
• Figure 33: Total Treated Cases of ET in Japan (2020-2034)
• Figure 34: Total Market Size of ET in the 7MM, in USD Million (2020-2034)
• Figure 35: Total Market Size of ET in the United States, in USD Million (2020-2034)
• Figure 36: Market Size of ET by Therapies in the United States, in USD Million (2020-2034)
• Figure 37: Total Market Size of ET in EU4 and the UK, in USD Million (2020-2034)
• Figure 38: Market Size of ET by Therapies in EU4 and the UK, in USD Million (2020-2034)
• Figure 39: Total Market Size of ET in Japan, in USD Million (2020-2034)
• Figure 40: Market Size of ET by Therapies in Japan, in USD Million (2020-2034)
• Figure 47: Health Technology Assessment
• Figure 48: Reimbursement Process in Germany
• Figure 49: Reimbursement Process in France
• Figure 50: Reimbursement Process in Italy
• Figure 51: Reimbursement Process in Spain
• Figure 52: Reimbursement Process in the United Kingdom
• Figure 53: Reimbursement Process in Japan

Key Highlights:

• Essential Thrombocythemia (ET) is a chronic myeloproliferative neoplasm (MPN) characterized by an increased number of platelets in the blood. Most commonly diagnosed in women over the age of 50.
• The primary cause of ET is the overproduction of hematopoietic cells due to the mutations of the JAK2, CALR, or MPL genes. These genes are known as 'driver mutations due to their role in developing a myeloproliferative neoplasm. Though 90% of adults have JAK2, CALR, or MPL mutations, it is not unusual to find children with a molecular triple wildtype status.
• Many ET patients are asymptomatic. Consequently, the disease is often diagnosed as part of a routine check-up after a blood test reveals a high platelet count. When symptoms are present, they may include fatigue or may be related to small or large vessel disturbance or bleeding.
• The median estimate of survival among ET patients is 20 years. However, depending on the age of presentation, this varies and as a result, median survival of patients younger than 60 years of age approaches 33 years.
• The most common cause of morbidity and mortality is thrombosis, which occurs among 20% of ET patients, compared to bleeding complications in 10% of this population.
• As per the current market scenario, treatment for essential thrombocythemia focuses on reducing the risk of blood clots and managing symptoms with a range of therapeutic options tailored to individual risk levels.
• Aspirin is commonly recommended in low, intermediate, as well as high-risk patients, as it helps prevent clot formation. First-line treatment often includes a combination of aspirin along with cytoreductive therapies such as hydroxyurea, anagrelide, and interferon-alpha, which aim to reduce platelet counts and prevent complications.
• Cytoreductive therapy is utilized to lower the risk of hemorrhage in patients with platelet counts exceeding 1 million/µL. In certain cases, additional medications such as busulfan, clopidogrel, and anticoagulants may be prescribed, depending on the patient's condition and response to other treatments.
• Currently, there is only one drug approved specifically for the treatment of ET, and it is authorized exclusively in Europe. In contrast, no therapies have received FDA approval for the treatment of ET in the United States.
• In August 2024, pharmaand GmbH (pharma&) announced that the EC had granted marketing authorization for a Type II variation for PEGASYS (peginterferon alfa-2a) as a monotherapy treatment for adults with ET.
• Beyond PEGASYS, the treatment landscape for ET is evolving with several promising assets in the pipeline. These include BESREMi (ropeginterferon alfa-2b), Bomedemstat (MK-3543/IMG-7289), Pelabresib (CPI-0610), Dencatistat (STP 938), INCA033989, and VAC85135.

Report Summary

• The report offers extensive knowledge regarding the epidemiology segments and predictions, presenting a deep understanding of the potential future growth in diagnosis rates, disease progression, and treatment guidelines. It provides comprehensive insights into these aspects, enabling a thorough assessment of the subject matter.
• Additionally, an all-inclusive account of the current management techniques and emerging therapies and the elaborative profiles of late-stage (Phase III and Phase II) and prominent therapies that would impact the current treatment landscape and result in an overall market shift has been provided in the report.
• The report also encompasses a comprehensive analysis of the ET market, providing an in-depth examination of its historical and projected market size (2020-2034). It also includes the market share of therapies, detailed assumptions, and the underlying rationale for our methodology. The report also includes drug outreach coverage in the 7MM region.
• The report includes qualitative insights that provide an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, including experts from various hospitals and prominent universities, patient journey, and treatment preferences that help shape and drive the 7MM ET market.

Essential Thrombocythemia Market

Various key players are leading the treatment landscape of essential thrombocythemia, such as pharma&, AOP Orphan Pharmaceuticals AG, Merck Sharp and Dohme, Novartis, Incyte Corporation, and Others. The details of the country-wise and therapy-wise market size have been provided below.

• In the total market size of Essential Thrombocythemia in the 7MM, the United States accounted for the highest market share, i.e. ~60% in 2024, followed by Japan.
• Among EU4 and the UK, France and the United Kingdom accounted for the highest market size in 2024.
• The United States accounted for approximately USD 250 million in 2024.
• Among the emerging therapies, BESREMI and Bomedemstat appear to be the drugs that can potentially transform the ET market.
• By 2034, among the emerging therapies, the highest revenue is expected to be generated by BESREMI.

Essential Thrombocythemia (ET) Drug Chapters

The section dedicated to drugs in the Essential Thrombocythemia report provides an in-depth evaluation of late-stage pipeline drugs (Phase III and Phase II) related to Essential Thrombocythemia. The drug chapters section provides valuable information on various aspects related to clinical trials of Essential Thrombocythemia, such as the pharmacological mechanisms of the drugs involved, designations, approval status, patent information, and a comprehensive analysis of the pros and cons associated with each drug. Furthermore, it presents the most recent news updates and press releases on drugs targeting Essential Thrombocythemia.

Marketed Therapies

PEGASYS (peginterferon alfa-2a): pharma&

PEGASYS is a Type I interferon; it is made when interferon alfa-2a undergoes the process of pegylation in which one or more chains of PEG are attached to another molecule. PEGASYS was previously approved by the European Commission (EC) for the treatment of Chronic Hepatitis B (CHB) in adults and children aged 3 years and older or Chronic Hepatitis C (CHC) in adults and children aged 5 years and older in combination with other medicinal products in adults or ribavirin in children.

In August 2024, pharma& announced that the EC had granted marketing authorization for a Type II variation for PEGASYS (peginterferon alfa-2a) as a monotherapy treatment for adults with essential thrombocythemia.

Emerging Therapies

BESREMi (ropeginterferon alfa-2b/P1101): AOP Orphan Pharmaceuticals AG/PharmaEssentia

Ropeginterferon alfa-2b is a novel, site-specific, monopegylated, stable IFN-a analog. The unique single isoform differentiates ropeginterferon alfa-2b from earlier generation polypegylated IFN, which utilized random pegylation methods and, therefore, contains many isoforms, with each Polyethylene Glycol (PEG) conjugate having its own activity and stability properties.

PharmaEssentia conducts clinical trials to study the efficacy and safety of ropeginterferon alfa-2b, while AOP Orphan often collaborates in the European market for regulatory approvals and distribution. Ropeginterferon alfa-2b is currently an FDA-approved treatment for patients with polycythemia vera. The company plans to pursue regulatory discussion with the FDA to seek a label expansion to include ET. They anticipate regulatory submission by the end of 2025.

Bomedemstat (MK-3543/IMG-7289): Merck Sharp and Dohme

Bomedemstat (MK-3543) is an investigational small molecule, irreversible LSD1 inhibitor being developed by Merck. LSD1 regulates the proliferation of hematopoietic stem cells, playing an essential role in cell differentiation and maturation. Bomedemstat is being evaluated in a wide range of MPNs, including ET, MF, and PV.

Bomedemstat has Orphan Drug Designation (ODD), and Fast Track Designations (FTDs) for the treatment of ET and myelofibrosis (MF), ODD for the treatment of Acute Myeloid Leukemia (AML), and Priority Medicines scheme designation by the European Medicines Agency for the treatment of MF. Merck presented updated data from the Phase IIb Shorespan-003 trial, including first-time genomic data, at the American Society of Hematology (ASH) Annual Meeting in December 2023.

Essential Thrombocythemia (ET) Market Outlook

Essential thrombocythemia is a rare blood disorder where the bone marrow produces an excessive number of platelets. This overproduction can increase the risk of clot formation, potentially leading to serious complications like stroke, heart attack, or pulmonary embolism. It is characterized by elevated platelet counts and an increased clotting tendency.

As per the current market scenario, treatment for essential thrombocythemia focuses on reducing the risk of blood clots and managing symptoms with a range of therapeutic options tailored to individual risk levels. Aspirin is commonly recommended in low, intermediate, as well as high-risk patients, as it helps prevent clot formation. First-line treatment often includes a combination of aspirin along with cytoreductive therapies such as hydroxyurea, anagrelide, and interferon-alpha, which aim to reduce platelet counts and prevent complications. Further, cytoreductive therapy is utilized to lower the risk of hemorrhage in patients with platelet counts exceeding 1 million/µL. In certain cases, additional medications such as busulfan, clopidogrel, and anticoagulants may be prescribed, depending on the patient's condition and response to other treatments. Regular monitoring is crucial to adjust therapy and minimize risks.

The development of new therapies for essential thrombocythemia is currently steady, with few new drugs on the horizon. novel agents are being explored, such as BESREMi (Ropeginterferon Alfa-2b), which offers long-acting, sustained platelet-lowering effects with a promising safety profile. Additionally, drugs like Bomedemstat (MK-3543/IMG-7289), targeting specific molecular pathways in the disease, are advancing in clinical trials, potentially offering a more targeted approach to treatment.

In a nutshell, many potential therapies are being investigated to manage Essential Thrombocythemia. Even though it is too soon to comment on the above-mentioned promising candidate to enter the market during the forecast period (2024-2034), it is safe to assume that the future of this market is bright. Eventually, these drugs will create a significant difference in the landscape of ET in the coming years. The treatment space is expected to experience a significant positive shift in the coming years owing to the improvement in healthcare spending worldwide.

Essential Thrombocythemia (ET) Disease Understanding and Treatment

Essential Thrombocythemia (ET) Overview

Essential Thrombocythemia (ET) is a chronic Myeloproliferative Neoplasm (MPN) characterized by an increased number of platelets in the blood. Most commonly diagnosed in women over the age of 50, ET is associated with a proliferation of platelet precursors in the bone marrow, and complications frequently include blood clotting and/or bleeding. Less common consequences in the later stages of ET include a transformation to myelofibrosis (marrow scarring) or acute leukemia.

The exact cause of ET and other MPNs remains unknown. Although ET, like many MPNs, is not considered a genetically inherited condition, some patients may have a familial predisposition. Researchers have identified mutations in many individuals with ET that affect proteins involved in signaling pathways, which play a crucial role in regulating cell growth and development. Many ET patients are asymptomatic. Consequently, the disease is often diagnosed as part of a routine check-up after a blood test reveals a high platelet count. When symptoms are present, they may include fatigue or may be related to small or large vessel disturbance or bleeding.

Essential Thrombocythemia (ET) Diagnosis

Diagnosis of ET should be based on a composite assessment of clinical, morphological, and laboratory features. In this regard, it should be noted that the overwhelming majority of thrombocytosis cases in routine clinical practice are non-clonal and associated with a spectrum of unrelated conditions such as infections, inflammation, post-surgical state, splenectomy, and iron deficiency. On the contrary, while the detection of JAK2V617F, CALR, or MPL mutations confirms the presence of an underlying MPN, their absence does not rule out the possibility of ET since up to 20% of patients might be triple-negative (i.e., negative for all three mutations). It is also important to note that other JAK2/CALR/MPL-mutated MPN (or MDS/MPN) can mimic ET in their presentation; these include pre-fibrotic PMF and MDS/MPN with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T). However, in the presence of JAK2/CALR/MPL mutation, the main distinction is with prefibrotic PMF. Bone marrow examination is necessary to make an accurate morphologic diagnosis of ET and distinguish it from other myeloid neoplasms, especially from prefibrotic PMF.

Essential Thrombocythemia (ET) Treatment

Median survival in young patients with ET and PV exceeds 35 years and is not that much worse for older patients. Therefore, it is very important to avoid non-evidence-based therapeutic adventures that might shorten life expectancy and increase the rate of fibrotic or leukemic transformations, as has been previously reported with chlorambucil, radiophosphorus, pipobroman, and anagrelide. To date, drug therapy has not been shown to improve survival or prevent leukemic/fibrotic transformation in either ET or polycythemia vera; therefore, treatment is instead directed at preventing thrombotic complications. In this regard, the decision to institute drug therapy should take into consideration the individual risk of thrombosis, the availability of controlled evidence of value, and potential harm to the patient, both short-term and long-term. In the latter regard, drug-induced alteration of host immunity and impact on clonal evolution are particularly highlighted in the context of opportunistic infections, induction of second malignancies, and leukemic or fibrotic transformation.

Essential Thrombocythemia (ET) Epidemiology

The Essential Thrombocythemia epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by * Total diagnosed prevalent cases, symptom-specific cases, gender-specific cases, mutation-specific cases, risk-specific cases, age-specific cases and total treated cases in the United States, EU4 countries (Germany, France, Italy, Spain) and the United Kingdom, and Japan from 2020 to 2034.

• The total diagnosed cases of Essential Thrombocythemia in the US were around 167,450 cases in 2024.
• In the United States, the highest number of age-specific cases were recorded for > 60 yrs of age, i.e., ~108,800 cases in 2024. Followed by the age group of 40-60 yrs and < 40yrs.
• Germany had the highest diagnosed prevalent cases of ET among the EU4 and the UK, accounting for more than 20% of cases in 2024.
• JAK2 was responsible for the majority of ET cases, accounting for 75% of them.
• In 2024, the total treated cases of Essential Thrombocythemia in the US were ~85,000 for the first-line treated patients.

KOL Views

To stay abreast of the latest trends in the market, we conduct primary research by seeking the opinions of Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) who work in the relevant field. This helps us fill any gaps in data and validate our secondary research.

We have reached out to industry experts to gather insights on various aspects of Essential Thrombocythemia, including the evolving treatment landscape, patients' reliance on conventional therapies, their acceptance of therapy switching, drug uptake, and challenges related to accessibility. The experts we contacted included medical/scientific writers, professors, and researchers from prestigious universities in the US, Europe, the UK, and Japan.

Our team of analysts at Delveinsight connected with more than 15 KOLs across the 7MM. We contacted institutions such as the University of Munich, the University of Tokyo, MD Anderson Cancer Center, Johns Hopkins Kimmel Cancer Center, etc., among others. By obtaining the opinions of these experts, we gained a better understanding of the current and emerging treatment patterns in the Essential Thrombocythemia market, which will assist our clients in analyzing the overall epidemiology and market scenario.

Qualitative Analysis

We perform Qualitative and Market Intelligence analysis using various approaches, such as SWOT analysis and Conjoint Analysis. 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.

Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, designation, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

In efficacy, the trial's primary and secondary outcome measures are evaluated; for instance, in trials for Essential Thrombocythemia, one of the most important primary endpoints was achieving hemolysis control, LDH normalization, etc. Based on these, the overall efficacy is 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. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

Market Access and Reimbursement

Because newly authorized drugs are often expensive, some patients escape receiving proper treatment or use off-label, less expensive prescriptions. Reimbursement plays a critical role in how innovative treatments can enter the market. The cost of the medicine, compared to the benefit it provides to patients who are being treated, sometimes determines whether or not it will be reimbursed. Regulatory status, target population size, the setting of treatment, unmet needs, the number of incremental benefit claims, and prices can all affect market access and reimbursement possibilities.

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.

Essential Thrombocythemia (ET) Report Insights

• Patient Population
• Therapeutic Approaches
• Essential Thrombocythemia Market Size and Trends
• Existing Market Opportunity

Essential Thrombocythemia (ET) Report Key Strengths

• Ten-year Forecast
• The 7MM Coverage
• Essential Thrombocythemia Epidemiology Segmentation
• Key Cross Competition

Essential Thrombocythemia (ET) Report Assessment

• Current Treatment Practices
• Reimbursements
• Market Attractiveness
• Qualitative Analysis (SWOT, Conjoint Analysis, Unmet needs)

Key Questions:

• Would there be any changes observed in the current treatment approach?
• Will there be any improvements in Essential Thrombocythemia management recommendations?
• Would research and development advances pave the way for future tests and therapies for Essential Thrombocythemia?
• Would the diagnostic testing space experience a significant impact and lead to a positive shift in the treatment landscape of Essential Thrombocythemia?
• What kind of uptake will the new therapies witness in the coming years in Essential Thrombocythemia patients?

Table of Contents

1. Key Insights

2. Report Introduction

3. Essential Thrombocythemia Market Overview at a Glance

• 3.1. Market Share (%) Distribution of ET in 2024 in the 7MM
• 3.2. Market Share (%) Distribution of ET in 2034 in the 7MM

4. Methodology

5. Executive Summary

6. Key Events

7. Disease Background and Overview

• 7.1. Introduction
• 7.2. Etiology
• 7.3. Risk Factors
• 7.4. Signs and Symptoms
• 7.5. Pathogenesis
• 7.6. Pathophysiology
• 7.7. Prognosis
  • 7.7.1. Survival Risk Models in ET
• 7.8. Case Study of Prevalence and Phenotype of MPN Driver Mutations in ET
• 7.9. Diagnosis
  • 7.9.1. Life Expectancy
  • 7.9.2. Diagnostic Algorithm
  • 7.9.3. Differential Diagnosis
  • 7.9.4. Diagnosis Guidelines

8. Treatment and Management of ET

• 8.1. Treatment Overview
  • 8.1.1. More on Risk-adapted Therapy: Very Low or Low Risk With or Without Extreme Thrombocytosis
  • 8.1.2. More on Risk-adapted Therapy: High- or Intermediate-risk Disease
• 8.2. Treatment Algorithm
• 8.3. Treatment Guidelines
  • 8.3.1. Treatment for Very-low-risk or Low-risk or Intermediate-risk ETa
  • 8.3.2. Treatment for High-risk ETa

9. Epidemiology and Patient Population

• 9.1. Key Findings
• 9.2. Assumptions and Rationale
  • 9.2.1. The United States
  • 9.2.2. EU4 and the UK
  • 9.2.3. Japan
• 9.3. Total Diagnosed Prevalent Cases of ET in 7MM
• 9.4. Total Treated Cases of ET in 7MM
• 9.5. The United States
  • 9.5.1. Total Diagnosed Prevalent Cases of ET in the United States
  • 9.5.2. Symptom-specific Cases of ET in the United States
  • 9.5.3. Gender-specific Cases of ET in the United States
  • 9.5.4. Mutation-specific Cases of ET in the United States
  • 9.5.5. Risk-specific Cases of ET in the United States
  • 9.5.6. Age-specific Cases of ET in the United States
  • 9.5.7. Treated Cases of ET in the United States
• 9.6. EU4 and the UK
  • 9.6.1. Total Diagnosed Prevalent Cases of ET in EU4 and the UK
  • 9.6.2. Symptom-specific Cases of ET in EU4 and the UK
  • 9.6.3. Gender-specific Cases of ET in EU4 and the UK
  • 9.6.4. Mutation-specific Cases of ET in EU4 and the UK
  • 9.6.5. Risk-specific Cases of ET in EU4 and the UK
  • 9.6.6. Age-specific Cases of ET in EU4 and the UK
  • 9.6.7. Total Treated Cases of ET in EU4 and the UK
• 9.7. Japan
  • 9.7.1. Total Diagnosed Prevalent Cases of ET Japan
  • 9.7.2. Symptom-specific Cases of ET in Japan
  • 9.7.3. Gender-specific Cases of ET in Japan
  • 9.7.4. Mutation-specific Cases of ET in Japan
  • 9.7.5. Risk-specific Cases of ET in Japan
  • 9.7.6. Age-specific Cases of ET in Japan
  • 9.7.7. Treated Cases of ET in Japan

10. Patient Journey

11. Marketed Therapies

• 11.1. PEGASYS (peginterferon alfa-2a): pharma&
  • 11.1.1. Product Description
  • 11.1.2. Regulatory Milestones
  • 11.1.3. Other Developmental Activities
  • 11.1.4. Clinical Development
  • 11.1.5. Safety and Efficacy

12. Emerging Therapies

• 12.1. Key Cross
• 12.2. BESREMi (ropeginterferon alfa-2b/P1101): AOP Orphan Pharmaceuticals AG/PharmaEssentia
  • 12.2.1. Product Description
  • 12.2.2. Other Developmental Activities
  • 12.2.3. Clinical Developmental Activities
  • 12.2.4. Safety and Efficacy
  • 12.2.5. Analyst View
• 12.3. Bomedemstat (MK-3543/IMG-7289): Merck Sharp and Dohme
  • 12.3.1. Product Description
  • 12.3.2. Other Developmental Activities
  • 12.3.3. Clinical Developmental Activities
  • 12.3.4. Safety and Efficacy
  • 12.3.5. Analyst View

13. Essential Thrombocythemia (ET): 7 Major Market Analysis

• 13.1. Key Findings
• 13.2. Market Outlook
• 13.3. Conjoint Analysis
• 13.4. Key Market Forecast Assumptions
  • 13.4.1 . Cost Assumptions and Rationale
  • 13.4.2. Pricing Trends
  • 13.4.3. Analogue Assessment
  • 13.4.4. Launch Year and Therapy uptake
• 13.5. Market Size of ET in the 7MM
• 13.6. The United States
  • 13.6.1. Total Market Size of ET in the United States
  • 13.6.2. Market Size of ET by Therapies in the United States
• 13.7. EU4 and the UK
  • 13.7.1. Total Market Size of ET in EU4 and the UK
  • 13.7.2. Market Size of ET by Therapies in EU4 and the UK
• 13.8. Japan
  • 13.8.1. Total Market Size of ET in Japan
  • 13.8.2. Market Size of Essential Thrombocythemia by Therapies in Japan

14. KOL Views

15. SWOT Analysis

16. Unmet Needs

17. Market Access and Reimbursement

• 17.1. United States
  • 17.1.1. Centre for Medicare and Medicaid Services (CMS)
• 17.2. EU4 and the UK
  • 17.2.1. Germany
  • 17.2.2. France
  • 17.2.3. Italy
  • 17.2.4. Spain
  • 17.2.5. United Kingdom
• 17.3. Japan
  • 17.3.1. MHLW

18. Appendix

• 18.1. Bibliography
• 18.2. Report Methodology

19. DelveInsight Capabilities

20. Disclaimer