寒冷凝集素症市場 - 市場の洞察、疫学、市場予測：2034年

寒冷凝集素病は、赤血球の早期破壊（溶血）を特徴とするまれな自己免疫疾患です。CADでは、赤血球が早期に破壊され、骨髄における新しい細胞の産生速度がその損失を補うことができなくなります。寒冷凝集素病の症状は一般的に40～80歳で発症し、高齢者に多い疾患です。症状は溶血性貧血、深い疲労、息切れ、肢端チアノーゼ、黄疸、暗色尿などです。

寒冷凝集素病の治療は、臨床症状の重症度別異なります。コルチコステロイド、アルキル化剤、プリンヌクレオシドアナログ、そしてリツキシマブ単剤療法または併用療法（フルダラビンとリツキシマブ、ベンダムスチンとリツキシマブ）などの生物学的製剤が寒冷凝集素病治療の主要な治療ラインです。

主要7ヶ国における寒冷凝集素病の有病率は、2023年には約13,000人となりました。

2023年、主要7ヶ国の寒冷凝集素病の市場規模は合計で1億9,000万米ドルとなりました。2023年の主要7ヶ国における寒冷凝集素病の市場規模は米国が最も大きく、次いで日本でした。ENJAYMOは2034年までに米国で最大の寒冷凝集素病市場シェアを確保すると予想されます。EU4ヶ国および英国における寒冷凝集素病の市場規模は、ドイツが最も大きく、2023年には1,800万米ドルに達します。

結論として、現在の寒冷凝集素病治療市場の状況には適切な治療法がないにもかかわらず、新規のメカニズムを持つ多くの潜在的な治療法が寒冷凝集素病治療薬市場に参入し、切実なアンメットニーズを解決し、寒冷凝集素病患者の治療成績の大幅な改善につながると期待されています。したがって、新たな治療オプションの利用が可能になりつつあり、主要7ヶ国全体で医療支出が増加していることから、治療シナリオは予測期間中（2024年～2034年）に大きな成長を遂げると予想されます。

溶血性貧血の診断は、綿密な臨床評価、詳細な病歴、特徴的な症状の確認、およびヘモグロビン値や全血液量のうち赤血球が占める割合（ヘマトクリット値）を測定する血液検査などのさまざまな検査に基づいて疑われます。血液検査では、未熟な赤血球（網状赤血球）の値が上昇することもあります。これは、早期に破壊された赤血球を補うために、体が余分な赤血球を作らざるを得なくなった場合に起こります。溶血性貧血の人の中には、血液中のビリルビン値が上昇する人（高ビリルビン血症）もいます。

寒冷凝集素病の治療では、症状の程度に応じて生活習慣の改善や薬物療法を行い、緊急の場合には輸血が必要になることもあります。治療方針を決定するもう一つの要因は、患者が原発性寒冷凝集素病に罹患しているか（CADはそれ自体で起こる）、二次性CADに罹患しているか（CADは他の病気が原因で起こる）です。有効な治療法は病原性B細胞クローンまたは古典的補体経路に対するものであり、治療法の選択は個々に行うべきです。BTK阻害や補体指向性治療のさらなる開発など、新たな治療アプローチが準備中です。

当レポートでは、主要7ヶ国における寒冷凝集素症（CAD）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 CAD市場概要

第4章 寒冷凝集素症（CAD）のエグゼクティブサマリー

第5章 疫学と市場調査手法

第6章 疾患の背景と概要

• イントロダクション
• CADの原因
• CADの兆候と症状
• CADの合併症
• CADの臨床症状
• CADの病態生理学
• CADの発症機序

第7章 CADの診断

• 診断アルゴリズム
• 診断テスト
• 鑑別診断

第8章 診断ガイドライン

第9章 CADの治療

第10章 治療ガイドライン

第11章 疫学と患者人口

• 主な調査結果
• 仮定と根拠
• 主要7ヶ国における疫学シナリオ
• 米国における疫学シナリオ
• EU4ヶ国と英国における疫学シナリオ
• 日本における疫学シナリオ

第12章 患者動向

第13章 寒冷凝集素症の臨床試験における重要なエンドポイント

第14章 上市済み治療法

第15章 新たな治療法

第16章 CAD：主要7ヶ国分析

• 主な調査結果
• 主要な市場予測の前提条件
• 主要7ヶ国におけるCAD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17章 市場アクセスと償還

第18章 KOLの見解

第19章 SWOT分析

第20章 アンメットニーズ

第21章 付録

第22章 DelveInsightのサービス内容

第23章 免責事項

第24章 DelveInsightについて

List of Tables

• Table 1: Summary of CAD, Market, Epidemiology, and Key Events (2020-2034)
• Table 2: Total Prevalent Cases of CAD (2020-2034)
• Table 3: Total Prevalent Cases of CAD (2020-2034)
• Table 4: Total Diagnosed Cases of CAD (2020-2034)
• Table 5: Gender-specific Cases of CAD (2020-2034)
• Table 6: Type-specific Cases of CAD (2020-2034)
• Table 7: Total Treated Cases of CAD (2020-2034)
• Table 8: Total Prevalent Cases of CAD (2020-2034)
• Table 9: Total Diagnosed Cases of CAD (2020-2034)
• Table 10: Gender-specific Cases of CAD in EU4 and the UK (2020-2034)
• Table 11: Type-specific Cases of CAD in EU4 and the UK (2020-2034)
• Table 12: Treated Cases of CAD in EU4 and the UK (2020-2034)
• Table 13: Total Prevalent Cases of CAD (2020-2034)
• Table 14: Total Diagnosed Cases of CAD (2020-2034)
• Table 15: Gender-specific Cases of CAD (2020-2034)
• Table 16: Type-specific Cases of CAD (2020-2034)
• Table 17: Total Treated Cases of CAD (2020-2034)
• Table 18: Efficacy Results in Patients of CADENZA trial
• Table 19: Efficacy Results in Patients in CARDINAL trial
• Table 20: Comparison of Emerging Drugs Under Development
• Table 21: Iptacopan (LNP023), Clinical Trial Description, 2024
• Table 22: Parsaclisib (INCB050465), Clinical Trial Description, 2024
• Table 23: Total Market Size of CAD in the 7MM, USD million (2020-2034)
• Table 24: Total Market Size of CAD in the US, USD million (2020-2034)
• Table 25: Market Size of CAD by Therapies in the US, USD million (2020-2034)
• Table 26: Market Size of CAD in EU4 and the UK, in USD million (2020-2034)
• Table 27: Market Size of CAD by Therapies in EU4 and the UK, in USD millions (2020-2034)
• Table 28: Total Market Size of CAD in Japan, USD million (2020-2034)
• Table 29: Market Size of CAD by Therapies in Japan, USD million (2020-2034)
• Table 30: Key HTA Decisions for PNH

List of Figures

• Figure 1: Epidemiology and Market Methodology
• Figure 2: Antibody-mediated Hemolysis
• Figure 3: Signs and Symptoms of CAD
• Figure 4: Pathophysiology of CAD
• Figure 5: Diagnostic Algorithm for CAD
• Figure 6: Typical Acrocyanosis Exacerbated by Cold Exposure
• Figure 7: Diagnostic Test Using Nailfold Capillaroscopy
• Figure 8: Pharmacological Management of CAD
• Figure 9: Treatment Algorithm of CAD
• Figure 10: Total Prevalent Cases of CAD in the 7MM (2020-2034)
• Figure 11: Total Prevalent Cases of CAD in the US (2020-2034)
• Figure 12: Total Diagnosed Cases of CAD in the US (2020-2034)
• Figure 13: Gender-specific Cases of CAD in the US (2020-2034)
• Figure 14: Type-specific Cases of CAD in the US (2020-2034)
• Figure 15: Total Treated Cases of CAD in the US (2020-2034)
• Figure 16: Total Prevalent Cases of CAD in EU4 and the UK (2020-2034)
• Figure 17: Total Diagnosed Cases of CAD in EU4 and the UK (2020-2034)
• Figure 18: Gender-specific Cases of CAD in EU4 and the UK (2020-2034)
• Figure 19: Type-specific Cases of CAD in EU4 and the UK (2020-2034)
• Figure 20: Total treated cases of CAD in EU4 and the UK (2020-2034)
• Figure 21: Total Prevalent Cases of CAD in Japan (2020-2034)
• Figure 22: Total Diagnosed Cases of CAD in Japan (2020-2034)
• Figure 23: Gender-specific Cases of CAD in Japan (2020-2034)
• Figure 24: Type-specific Cases of CAD in Japan (2020-2034)
• Figure 25: Total Treated Cases of CAD in Japan (2020-2034)
• Figure 26: Total Market Size of CAD in the 7MM, USD million (2020-2034)
• Figure 27: Total Market Size of CAD in the US, USD million (2020-2034)
• Figure 28: Market Size of CAD by Therapies in the US, USD million (2020-2034)
• Figure 29: Market Size of CAD in EU4 and the UK, USD million (2020-2034)
• Figure 30: Market Size of CAD by Therapies in EU4 and the UK, USD millions (2020-2034)
• Figure 31: Total Market Size of CAD in Japan, USD million (2020-2034)
• Figure 32: Market Size of CAD by Therapies in Japan, USD million (2020-2034)

Key Highlights:

• Cold agglutinin disease is a rare autoimmune disorder characterized by the premature destruction of red blood cells (hemolysis). In CAD, the red blood cells are destroyed prematurely, and the production rate of new cells in the bone marrow can no longer compensate for their loss.
• Cold Agglutinin Disease symptoms typically develop in individuals between the ages of 40 and 80 and are more common in elder individuals. Symptoms may include hemolytic anemia, profound fatigue, and shortness of breath, acrocyanosis, jaundice, and dark urine.
• The Cold Agglutinin Disease treatment depends on the severity of the clinical symptoms. Corticosteroids, alkylating agents, purine nucleoside analogs, and majorly biologics, such as rituximab monotherapy or combination therapy (fludarabine and rituximab, bendamustine and rituximab) and others are the major therapeutic line of Cold Agglutinin Disease treatment.
• In the 7MM, the Cold Agglutinin Disease prevalence cases were approximately 13,000 in 2023.
• ENJAYMO, a complement C1s inhibitor monoclonal antibody, is the first FDA-approved drug for treating Cold Agglutinin Disease. This approval has impacted the Cold Agglutinin Disease treatment market landscape significantly, especially in patients with severe anemia.
• Other promising treatments under development for Cold Agglutinin Disease include iptacopan (Novartis), pegcetacoplan (Apellis and SOBI), SAR445088 (Sanofi), and INCB050465 (Incyte).The total Cold Agglutinin Disease drugs market of the US was ~USD 95 million in 2023.
• In 2023, the Cold Agglutinin Disease gender-specific cases were ~40% of males and ~60% cases of females, in US.
• In 2023, approximately 90% of Cold Agglutinin Disease cases in the US were classified as type-specific primary cases, while the remaining 10% were categorized as secondary Cold Agglutinin Disease.

Cold Agglutinin Disease Drugs Market: Report Summary

The Cold Agglutinin Disease Drugs Market 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 Cold Agglutinin Disease treatment market landscape and result in an overall market shift has been provided in the report.

The Cold Agglutinin Disease Drugs Market report also encompasses a comprehensive analysis of the Cold Agglutinin Disease therapeutics market, providing an in-depth examination of its historical and projected market size (2020 - 2034). It also includes the Cold Agglutinin Disease market share of therapies, detailed assumptions, and the underlying rationale for our methodology. The Cold Agglutinin Disease therapeutics market report also includes drug outreach coverage in the 7MM region.

The Cold Agglutinin Disease Drugs Market 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 Cold Agglutinin Disease therapeutics market.

Cold Agglutinin Disease Recent Developments

• On October 4, 2024, Sanofi struck a deal to sell Enjaymo, its treatment for cold agglutinin disease, to Recordati for a potential $1 billion. Under the agreement, the Italian pharmaceutical company will pay Sanofi $825 million upfront for global rights to the biologic, with the possibility of an additional $250 million in milestone payments. Enjaymo, a humanized monoclonal antibody, treats the rare autoimmune disorder by reducing the risk of hemolysis in patients.

Cold Agglutinin Disease Drugs Market

• Various key Cold Agglutinin Disease Companies are leading the Cold Agglutinin Disease treatment market landscape, such as Novartis, Incyte Corporation, Sanofi and others. The details of the country-wise and therapy-wise Cold Agglutinin Disease market size have been provided below.
• In 2023, the total Cold Agglutinin Disease market size in the 7MM was ~USD 190 million.
• The United States had the largest Cold Agglutinin Disease market size in the 7MM in 2023, followed by Japan.
• ENJAYMO is expected to secure the largest Cold Agglutinin Disease market share in the US by 2034.
• In 2023, the CAD market's largest Cold Agglutinin Disease market share within the EU4 and the UK was held by Germany, amounting to around USD 18 million.
• Germany accounted for the highest total Cold Agglutinin Disease market size in EU4 and the UK, i.e., ~USD 18 million in 2023.

Cold Agglutinin Disease Drugs Market Chapters

The section dedicated to drugs in the Cold Agglutinin Disease drugs market report provides an in-depth evaluation of late-stage pipeline drugs (Phase III, Phase II and Phase I) related to Cold Agglutinin Disease. The Cold Agglutinin Disease drugs market chapters section provides valuable information on various aspects related to Cold Agglutinin Disease clinical trials, 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 newsCold Agglutinin Disease updates and press releases on drugs targeting Cold Agglutinin Disease.

Cold Agglutinin Disease Marketed Therapies

• ENJAYMO (sutimlimab): Sanofi/Bioverativ

ENJAYMO (sutimlimab) is a humanized monoclonal IgG4 antibody directed against human complement factor C1s, a serine protease responsible for the enzymatic propagation of the classical complement pathway (CP), which is a part of the innate immune system. C1s is a part of the C1 complex, a multimeric protein assembly containing the pattern recognition receptor C1q and the C1s-activating serine protease, C1r. By binding to C1s, sutimlimab specifically inhibits the classical pathway, preventing the enzymatic action of the C1 complex on its substrates (C4 and C2), thereby blocking the formation of the pivotal enzyme, C3-convertase. Sutimlimab preserves the functions of the alternative and lectin complement pathways to mediate the humoral surveillance of pathogens. By selectively inhibiting the classical pathway upstream at C1s, sutimlimab did not alter C1q levels or inhibit the lectin and alternative complement pathways.

Cold Agglutinin Disease Emerging Therapies

LNP023 (iptacopan): Novartis

Iptacopan is an investigational, first-in-class, orally administered, small-molecule inhibitor of complement factor B (FB) with potential immunomodulatory activity and targeting one of the key drivers of several complement-driven renal diseases (CDRDs). Upon administration, iptacopan binds to FB and prevents the formation of the alternative pathway (AP) C3-convertase (C3bBb). This limits the cleavage of C3 to the active fragment C3b and may prevent C3b-mediated extravascular hemolysis in certain complement-driven disorders such as PNH and CAD.

Iptacopan is one of the most advanced assets in the Novartis nephrology pipeline and has the potential to become the first targeted therapy to delay the progression to dialysis in C3G. Discovered at the Novartis institute for biomedical research, iptacopan is currently in development for several CDRDs where significant unmet needs exist. To investigate the medication in individuals with primary CAD, Novartis Pharmaceuticals is presently doing a Phase II (NCT05086744) basket trial.

INCB050465 (parsaclisib): Incyte Corporation

Parsaclisib, a potent and selective inhibitor of the delta isoform of phosphoinositide-3 kinase (PI3K), demonstrates promising antineoplastic activity with reduced hepatotoxicity compared to first-generation inhibitors. By targeting PI3K-delta, primarily expressed in hematopoietic cells, it inhibits the PI3K/AKT pathway, decreasing proliferation and inducing cell death in PI3K-delta-overexpressing tumor cells. Parsaclisib's selective inhibition aims to spare normal cells' PI3K signaling. It directly blocks tumor cell proliferation and indirectly suppresses tumor growth by inhibiting regulatory T-cells, reducing immunosuppression. Currently, the drug is in Phase II (NCT03538041) clinical stage in patients with autoimmune hemolytic anemia. The trial is also ongoing in the EU clinical trials register (2017-003652-22) for autoimmune hemolytic anemia (AIHA) and includes patients with cold AIHA.

Cold Agglutinin Disease Market Outlook

The 'watch and wait' approach is appropriate in patients with compensated hemolysis or mild anemia, absent or tolerable cold-induced circulatory symptoms, and no unacceptable fatigue. Bendamustine plus rituximab is the preferred choice in moderately to severely affected patients. Rituximab monotherapy is used to maintain its role in frail patients and those with milder diseases. Sutimlimab, if available and affordable, should be preferred even in the first line in severely anemic patients who require a quick response, like in acute exacerbations that do not resolve spontaneously and in patients in whom chemo-immunotherapy is contraindicated.

Many new molecules with novel mechanisms, like cyclic peptide, Monoclonal antibody, Complement C3 inhibitor, Phosphatidylinositol 3 kinase delta inhibitors, among others, are being developed for the Cold Agglutinin Disease treatment by key Cold Agglutinin Disease Companies like Iptacopan (Novartis), and Parsaclisib (Incyte Corporation), among others.

In conclusion, despite the lack of appropriate treatment in the current Cold Agglutinin Disease treatment market landscape, many potential therapies with novel mechanisms are expected to enter the Cold Agglutinin Disease drugs market, resolving a dire unmet need and leading to significant improvement in the treatment outcome of CCold Agglutinin Disease patients. Hence, with the upcoming availability of new treatment options and increasing healthcare spending across the 7MM, the treatment scenario is expected to experience significant growth during the forecast period (2024-2034).

Cold Agglutinin Disease Overview

Cold Agglutinin Disease (CAD) is a rare autoimmune disorder characterized by premature destruction of red blood cells (hemolysis). Autoimmune diseases occur when one's immune system attacks healthy tissue; more specifically, CAD is a subtype of autoimmune hemolytic anemia. Autoimmune hemolytic anemia (AIHAs) are clinic-pathological entities characterized by the production of autoantibodies directed against surface antigens on red blood cells (RBCs). AIHAs are generally classified as warm, cold, or mixed type, depending on the optimum temperature at which the autoantibodies bind surface antigens. In this type of disorder, red blood cells are "tagged" by antibodies and are then destroyed by other types of immune cells. The disease is termed "cold" because the antibodies are active and cause hemolysis at cold temperatures, usually 3-4°C (37-39°F), which is not necessarily the case in other types of autoimmune hemolytic anemia.

Cold Agglutinin Disease Diagnosis

The diagnosis of hemolytic anemia may be suspected based on a thorough clinical evaluation, a detailed patient history, identification of characteristic symptoms, and a variety of tests such as blood tests that measure values of hemoglobin and the percentage of the total blood volume occupied by red blood cells (hematocrit). Blood tests May also show an elevated value of immature red blood cells (reticulocytes), which occurs when the body is forced to produce extra red blood cells to make up for those destroyed prematurely. Some individuals with hemolytic anemia have elevated bilirubin values in the blood (hyperbilirubinemia).

Hemolytic anemia also leads to increased lactate dehydrogenase values (LDH) in the blood, as it is released when red blood cells are destroyed. Haptoglobin is a hemoglobin scavenger that gets consumed when hemoglobin is released in the blood due to hemolysis; haptoglobin values are, therefore, low in hemolytic anemia. When hemolytic anemia is suspected to be autoimmune, specialized tests such as a Coombs test may be performed. This test is used to detect antibodies bound to red blood cells or other biological mediators, like complement (component 3, C3), which accompanies the binding of the immunoglobulin to their targets. A sample of blood is taken and then exposed to the Coombs reagent. A positive test is indicated when the red blood cells clump in the presence of the reagent.

Cold Agglutinin Disease Treatment

Cold Agglutinin Disease treatment involves the management of Cold Agglutinin Disease with lifestyle changes and medications, depending on the severity of symptoms, and in certain emergency cases, blood transfusions might be required. Another factor that decides treatment allocation is whether the patient has primary Cold Agglutinin Disease (CAD happens on its own) or secondary CAD (CAD caused due to another illness). Effective treatments are directed against the pathogenic B-cell clone or the classic complement pathway, and the choice of therapy should be made individually. Additional therapeutic approaches are in the pipeline, for example, BTK inhibition and further development of complement-directed therapies; therefore, patients with CAD requiring treatment should be considered for prospective trials.

Cold Agglutinin Disease Epidemiology

The Cold Agglutinin Disease epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by Total Cold Agglutinin Disease prevalent cases, total Cold Agglutinin Disease diagnosed cases, Cold Agglutinin Disease gender-specific cases, Cold Agglutinin Disease type-specific cases, total Cold Agglutinin Disease treated cases in the United States, EU4 countries (Germany, France, Italy, Spain) and the United Kingdom, and Japan from 2020 to 2034.

• In 2023, the Cold Agglutinin Disease prevalence cases were ~5,500 in the US.
• In 2023, approximately 90% of Cold Agglutinin Disease cases in the US were classified as type-specific primary cases, while the remaining 10% were categorized as secondary CAD.
• Among EU4 and the UK, the highest number of Cold Agglutinin Disease prevalence cases were in Germany.
• The Cold Agglutinin Disease gender-specific cases were ~1,800 and ~2,660 cases of males and females in 2023, respectively, in the US.

KOL Views

To stay abreast of the latest trends in the Cold Agglutinin Disease treatment 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 Cold Agglutinin Disease, including the evolving Cold Agglutinin Disease treatment market 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 KOL, etc., among others. By obtaining the opinions of these experts, we gained a better understanding of the current and emerging treatment patterns in the Cold Agglutinin Disease treatment market, which will assist our clients in analyzing the overall epidemiology and market scenario.

Qualitative Analysis

We perform Qualitative and Cold Agglutinin Disease Therapeutics 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 CAD, important primary endpoints are evaluation includes assessing hemoglobin levels, C3 levels, total bilirubin levels, and Chronic Illness Therapy (FACIT)-Fatigue Scale scores.

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, a final weightage score is decided, based on which the emerging therapies are ranked.

Cold Agglutinin Disease Treatment 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 Cold Agglutinin Disease treatment 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.

Cold Agglutinin Disease Treatment Market Report Insights

• Patient-based Cold Agglutinin Disease Market Forecasting
• Therapeutic Approaches
• Cold Agglutinin Disease Market Size
• Cold Agglutinin Disease Market Trends
• Existing Cold Agglutinin Disease Therapeutics Market Opportunity

Cold Agglutinin Disease Treatment Market Report Key Strengths

• 11 Years- Cold Agglutinin Disease Market Forecast
• The 7MM Coverage
• Cold Agglutinin Disease Epidemiology Segmentation
• Key Cross Competition

Cold Agglutinin Disease Treatment Market Report Assessment

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

Key Questions:

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

Table of Contents

1 Key Insights

2 Report Introduction

3 CAD Market Overview at a Glance

• 3.1 Market Share (%) Distribution of CAD by Drug class in 2020
• 3.2 Market Share (%) Distribution of CAD by Drug class by 2034

4 Executive Summary of Cold Agglutinin Disease (CAD)

• 4.1 Key Events

5 Epidemiology and Market Methodology

6 Disease Background and Overview

• 6.1 Introduction
• 6.2 Causes of CAD
• 6.3 Signs and Symptoms of CAD
• 6.4 Complications of CAD
• 6.5 Clinical Manifestations of CAD
• 6.6 Pathophysiology of CAD
• 6.7 Pathogenesis of CAD

7 Diagnosis of CAD

• 7.1 Diagnostic Algorithm
• 7.2 Diagnosis Tests
  • 7.2.1 Physical examination
  • 7.2.2 Imaging technique
  • 7.2.3 Laboratory tests
• 7.3 Differential Diagnosis

8 Diagnostic Guidelines

• 8.1 First International Consensus Meeting (2020) Recommendations for the Diagnosis of CAD
• 8.2 European British Committee for Standards in Hematology (BCSH) Recommendations for the Diagnosis of CAD

9 Treatment of CAD

• 9.1 Treatment Algorithm of CAD

10 Treatment Guidelines

• 10.1 European British Committee for Standards in Hematology (BCSH) Recommendations for the Treatment of CAD
• 10.2 First International Consensus Meeting (2020) Recommendations for the Treatment of CAD

11 Epidemiology and Patient Population

• 11.1 Key Findings
• 11.2 Assumptions and Rationale
• 11.3 Epidemiology Scenario in the 7MM
• 11.4 Epidemiology Scenario in the United States
  • 11.4.1 Total prevalent cases of CAD in the US
  • 11.4.2 Total diagnosed cases of CAD in the US
  • 11.4.3 Gender-specific cases of CAD in the US
  • 11.4.4 Type-specific cases of CAD in the US
  • 11.4.5 Total treated cases of CAD in the US
• 11.5 Epidemiology Scenario in EU4 and the UK
  • 11.5.1 Total prevalent cases of CAD in EU4 and the UK
  • 11.5.2 Total diagnosed cases of CAD in EU4 and the UK
  • 11.5.3 Gender-specific cases of CAD in EU4 and the UK
  • 11.5.4 Total type-specific cases of CAD in EU4 and the UK
  • 11.5.5 Total treated cases of CAD in EU4 and the UK
• 11.6 Epidemiology Scenario in Japan
  • 11.6.1 Total prevalent cases of CAD in Japan
  • 11.6.2 Total diagnosed cases of CAD in Japan
  • 11.6.3 Gender-specific cases of CAD in Japan
  • 11.6.4 Type-specific cases of CAD in Japan
  • 11.6.5 Total treated cases of CAD in Japan

12 Patient Journey

13 Key Endpoints in Cold Agglutinin Disease Clinical Trials

14 Marketed Therapies

• 14.1 Key Cross Competition
• 14.2 ENJAYMO (sutimlimab): Sanofi/Bioverativ
  • 14.2.1 Product description
  • 14.2.2 Regulatory milestones
  • 14.2.3 Other development activities
  • 14.2.4 Pivotal clinical trial

15 Emerging Therapies

• 15.1 Key Cross Competition
• 15.2 LNP023 (iptacopan): Novartis
  • 15.2.1 Product description
  • 15.2.2 Other development activities
  • 15.2.3 Clinical development
• 15.3 INCB050465 (parsaclisib): Incyte Corporation
  • 15.3.1 Product description
  • 15.3.2 Other development activities
  • 15.3.3 Clinical development
  • 15.3.4 Safety and efficacy

16 CAD: The 7MM Analysis

• 16.1 Key Findings
• 16.2 Key Market Forecast Assumptions
• 16.3 Total Market Size of CAD in the 7MM
• 16.4 The United States Market Size
  • 16.4.1 Total market size of CAD in the US
  • 16.4.2 The market size of CAD by therapies in the US
• 16.5 EU4 and the UK Market Size
  • 16.5.1 Total market size of CAD in EU4 and the UK
  • 16.5.2 Market size of CAD by therapies in EU4 and the UK
• 16.6 Japan Market Size
  • 16.6.1 Total market size of CAD in Japan
  • 16.6.2 The market size of CAD by therapies in Japan

17 Market Access and Reimbursement

• 17.1 Key HTA Decisions for PNH

18 KOL Views

19 SWOT Analysis

20 Unmet Needs

21 Appendix

• 21.1 Bibliography
• 21.2 Acronyms and Abbreviations
• 21.3 Report Methodology

22 DelveInsight Capabilities

23 Disclaimer

24 About DelveInsight