鎌状赤血球症市場 - 市場の洞察、疫学、市場予測：2032年

鎌状赤血球症（SCD）は、ヘモグロビンの異常により赤血球が鎌状になる遺伝性血液疾患です。この異常な形状は、慢性疼痛、臓器障害、感染症感受性の増加などの合併症を引き起こします。

SCDの主な原因は、ヘモグロビンの産生に影響を及ぼすHBB遺伝子の変異です。SCDの患者は、両親から2コピーの変異遺伝子を受け継ぐため、ヘモグロビンの産生に異常をきたします。異常赤血球は硬く粘着性になる傾向があり、血流を妨げ、鎌状赤血球クリーゼと呼ばれる激痛のエピソードを引き起こします。

ヒドロキシ尿素、非ステロイド性抗炎症薬、輸血、キレート剤、栄養補助食品、および広域抗生物質がSCDを治療します。さらに、米国FDAが鎌状赤血球症の治療薬として承認している治療薬には、ENDARI（L-グルタミン経口粉末）、ADAKVEO（crizanlizumab-tmca）、OXBRYTA（voxelotor）などがあります。

鎌状赤血球症の治療薬としては、Exa-cel（Vertex Pharmaceuticals/CRISPR Therapeutics）、Inclacumab（Global Blood Therapeutics/Pfizer）、Etavopivat（Forma Therapeutics/Novo Nordisk）などが研究されています。

2022年、米国における鎌状赤血球症の総患者数は104,900人と推定されており、2032年までに増加する見込みです。米国におけるSCDの市場規模は、2019年に1億4,200万米ドル程度と推定され、2032年までに大幅なCAGRで増加すると予測されています。

EU4ヶ国の中で、鎌状赤血球症の有病者数が最も多いのはフランスで、2022年には26,600人であり、2032年までに増加すると推定されています。

SCDはヘモグロビンの異常を特徴とする複雑な遺伝性血液疾患であり、合併症を引き起こし、生命予後を低下させる。しかし、治療法の進歩や現在進行中の研究努力により、管理方法の改善や治癒の可能性が期待されています。ヘルスケアサービスの強化、認知度の向上、SCD患者への支援は、SCDに関連する課題に取り組む上で不可欠なステップです。

鎌状赤血球貧血（SCD）の治療目標は、疼痛の緩和、感染症の予防、特に合併症の管理です。目標は症状のコントロールと合併症の管理です。治療はSCD患者の痛みを認識することから始まる。急性の激しい痛みが再発することがSCDの特徴です。

現在のSCDの治療パターンでは、完全な治癒の提供、それ自体が許容できる治療副作用、投与量や剤形の改善といった空白を埋める新たな治療法が可能です。今後、患者の負担は増加し、新薬の承認によって市場は拡大します。したがって、市場規模は全体的に拡大し、より良い投資治療分野となる可能性があります。

当レポートでは、主要6ヶ国における鎌状赤血球症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、2032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 鎌状赤血球症（SCD）市場概要

第4章 鎌状赤血球症（SCD）のエグゼクティブサマリー

第5章 主要な出来事

第6章 疾患の背景と概要

• イントロダクション
• 鎌状赤血球症の分類
• 原因
• 関連する危険因子
• 合併症
• 症状
• 病態生理学
• 診断
• 鎌状赤血球症の治療と管理
• 治療アルゴリズム
• 治療ガイドライン

第7章 調査手法

第8章 疫学と患者数

• 主な調査結果
• 仮定と根拠
• SCDの有病者数は合計600万人
• SCDと診断済み主要6ヶ国市場の症例
• 米国
• EU4ヶ国と英国

第9章 患者動向

第10章 上市済み薬剤

第11章 新たな治療法

第12章 市場分析

• 主な調査結果
• 主要な市場予測の前提条件
• コンジョイント分析
• 市場の見通し
• 主要6ヶ国市場分析
• 米国
• EU4ヶ国と英国

第13章 KOLのビュー

第14章 SWOT分析

第15章 アンメットニーズ

第16章 市場アクセスと償還

• 米国
• EU4ヶ国と英国

第17章 付録

第18章 DelveInsightのサービス内容

第19章 免責事項

List of Tables

• Table 1: Summary of SCD, Market, and Epidemiology (2019-2032)
• Table 2: Key Events
• Table 3: Laboratory Test Schedule for Patients With SCD
• Table 4: Pharmacological Management of Pain Using WHO Three-step "Analgesic Ladder"
• Table 5: Non-narcotic Analgesics for Mild Pain in Sickle Cell Disease
• Table 6: Outline of Management of Acute Pain in Opioid Naive Adults
• Table 7: Outline of Management of Acute Pain in Opioid Naive Adults
• Table 8: Management of Acute Pain in Opioid Naive Adults
• Table 9: A German Consensus Recommendation
• Table 10: Current situation and future perspectives.
• Table 11: Recommendations from a Pan-European Consensus Conference.
• Table 12: A British Society for Haematology Guideline
• Table 13: 6MM Total Prevalent Cases of SCD (2019-2032)
• Table 14: 6MM Total Diagnosed Cases of SCD (2019-2032)
• Table 15: Total Prevalent Cases of SCD Trait in the United States (2019-2032)
• Table 16: Total Prevalent Cases of SCD in the United States (2019-2032)
• Table 17: Total Diagnosed Cases of SCD in the United States (2019-2032)
• Table 18: Age-specific Cases of SCD in the United States (2019-2032)
• Table 19: Type-specific of SCD in the United States (2019-2032)
• Table 20: Total Prevalent Cases of SCD Trait in EU4 and the UK (2019-2032)
• Table 21: Total Prevalent Cases of SCD in EU4 and the UK (2019-2032)
• Table 22: Total Diagnosed Cases of SCD in EU4 and the UK (2019-2032)
• Table 23: Age-specific Cases of SCD in EU4 and the UK (2019-2032)
• Table 24: Type-specific Cases of SCD in EU4 and the UK (2019-2032)
• Table 25: Key Cross (Marketed Therapies)
• Table 26: ENDARI (L-glutamine), Clinical Trial Description, 2023
• Table 27: Results From the ENDARI Clinical Trial in Sickle Cell Disease
• Table 28: ADAKVEO (crizanlizumab), Clinical Trial Description, 2023
• Table 29: Efficacy Results from SUSTAIN Trial in Sickle Cell Disease
• Table 30: OXBRYTA (voxelotor), Clinical Trial Description, 2023
• Table 31: Adjusted Mean Change From Baseline to Week 24 in Hemoglobin and Clinical Measures of Hemolysis
• Table 32: Key Cross (Emerging Therapies)
• Table 33: Exa-cel (CTX001), Clinical Trial Description, 2023
• Table 34: Lovo-cel, Clinical Trial Description, 2023
• Table 35: GBT-601, Clinical Trial Description, 2023
• Table 36: Mitapivat, Clinical Trial Description, 2023
• Table 37: EDIT-301, Clinical Trial Description, 2023
• Table 38: Inclacumab, Clinical Trial Description, 2023
• Table 39: Etavopivat, Clinical Trial Description, 2023
• Table 40: NDec (EPI 01), Clinical Trial Description, 2023
• Table 41: Rifaximin, Clinical Trial Description, 2023
• Table 42: Key Market Forecast Assumptions for Exa-cel
• Table 43: Key Market Forecast Assumptions for Inclacumab
• Table 44: Key Market Forecast Assumptions for Etavopivat
• Table 45: Key Market Forecast Assumptions for Lovo-cel
• Table 46: Key Market Forecast Assumptions for GBT601
• Table 47: Key Market Forecast Assumptions for Mitapivat
• Table 48: Key Market Forecast Assumptions for EDIT-301
• Table 49: Total Market Size of SCD in the 6MM, in USD Millions (2019-2032)
• Table 50: Market Size of SCD by therapies in the 6MM, in USD Millions (2019-2032)
• Table 51: Total Market Size of SCD in the United States, in USD Millions (2019-2032)
• Table 52: Market Size of SCD by therapies in the United States, in USD Millions (2019-2032)
• Table 53: Total Market Size of SCD in EU4 and United Kingdom, in USD Millions (2019-2032)
• Table 54: Market Size of SCD by therapies in EU4 and United Kingdom, in USD Millions (2019-2032)

List of Figures

• Figure 1: Sickle Cell Disease Classification
• Figure 2: Sickle Cell Disease Complications
• Figure 3: Common Symptoms Associated With Sickle Cell Disease
• Figure 4: HbS Polymerization and Erythrocyte Deformation
• Figure 5: Mechanism in Sickle Cell disease
• Figure 6: Measure of Pain Intensity and Location
• Figure 7: Treatment Algorithm
• Figure 8: 6MM Total Prevalent Cases of SCD (2019-2032)
• Figure 9: 6MM Total Diagnosed Cases of SCD (2019-2032)
• Figure 10: Total Prevalent Cases of SCD Trait in the United States (2019-2032)
• Figure 11: Total Prevalent Cases of SCD in the United States (2019-2032)
• Figure 12: Total Diagnosed Cases of SCD in the United States (2019-2032)
• Figure 13: Age-specific Cases of SCD in the United States (2019-2032)
• Figure 14: Type-specific of SCD in the United States (2019-2032)
• Figure 15: Total Prevalent Cases of SCD Trait in EU4 and the UK (2019-2032)
• Figure 16: Total Prevalent Cases of SCD in EU4 and the UK (2019-2032)
• Figure 17: Total Diagnosed Cases of SCD in EU4 and the UK (2019-2032)
• Figure 18: Age-specific Cases of SCD in EU4 and the UK (2019-2032)
• Figure 19: Type-specific Cases of SCD in EU4 and the UK (2019-2032)
• Figure 20: Total Market Size of SCD in the 6MM, in USD Millions (2019-2032)
• Figure 21: Market Size of SCD by therapies in the 6MM, in USD Millions (2019-2032)
• Figure 22: Total Market Size of SCD in the United States, in USD Millions (2019-2032)
• Figure 23: Market Size of SCD by therapies in the United States, in USD Millions (2019-2032)
• Figure 24: Total Market Size of SCD in EU4 and United Kingdom, in USD Millions (2019-2032)
• Figure 25: Market Size of SCD by therapies in EU4 and United Kingdom, in USD Millions (2019-2032)
• Figure 26: Unmet Needs
• 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

Key Highlights:

• Sickle cell disease (SCD) is a genetic blood disorder characterized by abnormal hemoglobin, causing red blood cells to become sickle-shaped. This abnormal shape leads to complications, including chronic pain, organ damage, and increased infection susceptibility.
• The primary cause of SCD is a mutation in the HBB gene, which affects the production of hemoglobin. Individuals with SCD inherit two copies of the mutated gene, one from each parent, resulting in abnormal hemoglobin production. The abnormal red blood cells tend to become rigid and sticky, obstructing blood flow and causing episodes of intense pain called sickle cell crises.
• Hydroxyurea, NSAIDs, blood transfusions, chelating agents, nutritional supplements, and broad-spectrum antibiotics treat SCD. Moreover, a few therapies that the US FDA approves for the treatment of sickle cell disease include ENDARI (L-glutamine oral powder), ADAKVEO (crizanlizumab-tmca), and OXBRYTA (voxelotor).
• A few therapies that are being investigated for sickle cell disease include Exa-cel (Vertex Pharmaceuticals/CRISPR Therapeutics), Inclacumab (Global Blood Therapeutics/Pfizer), Etavopivat (Forma Therapeutics/Novo Nordisk) and others.
• In 2022, the total prevalent cases of Sickle Cell Disease in the United States were found to be ~104,900 which as per DelveInsight's estimates, will increase by 2032.
• Among the EU4 countries, the highest number of prevalent cases of Sickle Cell Disease were found in France, i.e., ~26,600 cases in the year 2022, which are estimated to increase by the year 2032.
• SCD is a complex genetic blood disorder characterized by abnormal hemoglobin, leading to complications and reduced life expectancy. However, advancements in treatment approaches and ongoing research efforts offer hope for improved management and potential cures. Enhancing healthcare services, raising awareness, and supporting individuals with SCD are essential steps toward addressing the challenges associated with this disease.

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 SCD market, providing an in-depth examination of its historical and projected market size (2019-2032). It also includes the market share of therapies, detailed assumptions, and the underlying rationale for our methodology.
• 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 journeys, and treatment preferences that help shape and drive the United States SCD market.

The table given below further depicts the key segments provided in the report:

• Study Period: 2019-2032
• Forecast Period: 2023-2032
• Geographies Covered: United States, EU4 and United Kingdom

Epidemiology Segmented by:

• By SCD Trait
• By Prevalent Cases of SCD
• By Diagnosed Cases of SCD
• By Age
• By Type

Market Segmented by:

• Market size in the 6MM
• Therapies

Market Analysis * KOL Views

• SWOT Analysis
• Unmet Needs

Market

Various key players currently leading the treatment landscape of SCD include Novartis Pharmaceuticals, Global Blood Therapeutics/Pfizer, and Emmaus Life Sciences. Moreover, various key players are investigating their therapeutic candidates in the late phases of clinical development. Vertex Pharmaceuticals/CRISPR Therapeutics, Global Blood Therapeutics/Pfizer, Forma Therapeutics/Novo Nordisk, Bluebird Bio, etc. The details of the country-wise and therapy-wise market size have been provided below.

• The market of SCD in the US is estimated to be around ~USD 142 million in 2019, which is expected to increase by 2032 with a significant CAGR.
• Among the 6MM current market, Hydroxyurea accounted for the largest market size of ~USD 65 million in 2022, while OXBRYTA seems to have captured a market size of ~USD 200 million in 2022.

Market Share 2022 Market Size (in USD million) (%)

• Hydroxyurea 12%
• ENDARI (L-glutamine) 4%
• ADAKVEO (crizanlizumab) 33%
• OXBRYTA (voxelotor) 37%
• Iron Chelating agents 4%
• Opioids/NSAIDs 3%
• Others 8%

Sickle Cell Disease (SCD) Drug Chapters

The section dedicated to drugs in the SCD report provides an in-depth evaluation of marketed therapies and the late-stage pipeline drugs (Phase III and Phase II) related to SCD.

The drug chapters section provides valuable information on various aspects related to clinical trials of SCD, including specific details, 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 SCD.

Marketed Therapies

ENDARI: Emmaus Life Sciences

ENDARI (L-glutamine) is an oral-administered pharmaceutical grade L-glutamine (PGLG), an amino acid formulation to relieve pain, swelling, and other complications of sickle cell anemia in adults and children 5 years and older.

ADAKVEO (crizanlizumab): Novartis Pharmaceuticals

ADAKVEO (crizanlizumab) is a humanized anti-P selectin monoclonal antibody that binds a molecule called P-selectin on the surface of endothelial cells and platelets in the blood vessels, causing the blockade of P-selectin; it drives the vaso-occlusive process. The therapeutic blockade of P-selectin can prevent painful vaso-occlusion in small blood vessels and maintain blood flow.

OXBRYTA (voxelotor): Global Blood Therapeutics/Pfizer

OXBRYTA is an oral, once-a-day tablet developed by Global Blood Therapeutics. It is designed to block sickle hemoglobin polymerization, the root cause of sickle cell disease. This results in poor blood flow that affects the oxygenation of tissues, ultimately leading to painful vaso-occlusive crises. By increasing hemoglobin's affinity to oxygen, OXBRYTA halts this process.

Note: Detailed current therapies assessment will be provided in the full report of Sickle Cell Disease

Emerging Therapies

Exa-cel: Vertex Pharmaceuticals/CRISPR Therapeutics

Exa-cel (CTX001) is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patient's hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. Currently, it is being evaluated in Phase III for treating SCD patients.

Inclacumab: Global Blood Therapeutics/Pfizer

Inclacumab is a novel, fully human monoclonal antibody that selectively targets P-selectin. This protein mediates cell adhesion and is clinically validated to reduce pain due to VOCs in people with SCD. Preclinical results suggest that inclacumab can be a best-in-class option for reducing VOCs in people with SCD. The therapy is estimated to launch by 2025 and has a slow-medium uptake.

Etavopivat: Forma Therapeutics/Novo Nordisk

Etavopivat is an orally available, small-molecule allosteric activator of the selective red blood cell (RBC) isoform of pyruvate kinase (PK-R), with the potential to improve symptoms in sickle cell disease (SCD) patients. Currently being evaluated in Phase II/III clinical development, the therapy is estimated to launch by 2026 and attain its peak in 7 years.

Therapy Name Company Indication Approval Year

Exa-cel Vertex Pharmaceuticals/CRISPR Therapeutics Sickle Cell Disease 2024

Inclacumab Global Blood Therapeutics/Pfizer Sickle Cell Disease 2025

Etavopivat Forma Therapeutics/Novo Nordisk Sickle Cell Disease 2026

Note: Detailed assessment will be provided in the final report of Sickle Cell Disease (SCD)…

Sickle Cell Anemia (SCD) Market Outlook

The treatment goals for SCD aim to relieve pain, prevent infections, and specifically manage complications. The goals are for symptom control and management of disease complications. The treatment starts with the recognition of pain in SCD patients. Recurrent episodes of acute, severe pain are the hallmark of SCD.

Initial management aims at providing rapid pain control. Many drug regimens have effectively treated acute pain in SCD. Pain management should follow the three-step "analgesic ladder" recommended by the World Health Organization for treating cancer-related pain. The choice of analgesic and dosage should be based on the severity of pain in the individual patient.

The treatment pattern currently consists of different approaches classified into pharmacologic and nonpharmacological therapies. The pharmacological therapies, including hydroxyurea, ENDARI (L-glutamine), ADAKVEO (crizanlizumab), and OXBRYTA (voxelotor), are approved. Pain management agents are segregated into opioids, nonsteroidal anti-inflammatory drugs (NSAIDs), acetaminophen, corticosteroids, etc. Acute vaso-occlusive crisis is generally managed using opioids and nonsteroidal anti-inflammatory drugs (NSAIDs). Further, nonpharmacological therapies include cognitive behavioral therapy, biofeedback, relaxation techniques, acupuncture, hypnosis, etc.

The current SCD treatment patterns allow emerging therapies to fill the voids, such as providing a complete cure, tolerable treatment side effects per se, and improved dosing and dosage forms. The patient burden will increase in the coming times, and approval of the emerging drug will expand the market. Therefore, the overall market size will increase, which could be a better investment therapy area.

Sickle Cell Disease (SCD) Understanding and Treatment

Sickle Cell Disease (SCD) Overview

Sickle Cell Disease (SCD) is a group of lifelong inherited conditions that affect hemoglobin. It is characterized as a chronic hemolytic disorder marked by the tendency of hemoglobin molecules within red blood cells to polymerize and deform the red cell into sickle (or crescent) shape (Hb S), resulting in characteristic vaso-occlusive events and accelerated hemolysis.

Sickle cell disease is inherited in an autosomal fashion, whether in the homozygous or double heterogeneous state. Sickle cell disease is called sickle cell anemia (SCA) when there is an inheritance in the homozygous state. Other known SCD genotypes include hemoglobin SC disease, sickle beta plus thalassemia, sickle beta zero thalassemia (which has similar severity with sickle cell anemia), hemoglobin SD Punjab disease, hemoglobin SO Arab disease, and others. Hemoglobin S (Hb S) differs from normal hemoglobin (Hb A) because of the substitution of valine for glutamic acid in the sixth position in the B-globin gene.

Sickle cell anemia can lead to many complications, including stroke, acute chest syndrome, pulmonary hypertension, organ damage, splenic sequestration, leg ulcers, gallstones, priapism, deep vein thrombosis, and others.

Sickle Cell Disease (SCD) diagnosis

SCD diagnosis starts with a blood test that is analyzed for defective genes or hemoglobin cells. Various screening programs also help in the early diagnosis of the disease during the prenatal or infancy period.

Further details related to country-based variations are provided in the report…

Sickle Cell Disease (SCD) treatment

The primary objective of early intervention is to relieve pain. Numerous medication plans have successfully addressed sudden pain in sickle cell disease (SCD). For effective pain relief, it is advisable to adhere to the three-stage "analgesic ladder" endorsed by the World Health Organization, which is commonly used for managing cancer-related pain. The selection of the appropriate painkiller and its dosage needs to be determined by the intensity of pain experienced by each patient.

Oxygen therapy is often used routinely in managing vaso-occlusive crises, despite a lack of evidence supporting the effectiveness of these measures in all patients. Fluids should be administered in a quantity sufficient to correct existing deficits and replace ongoing losses to maintain a normal state. Moreover, antibiotics should be considered if there is evidence of infection.

Hydroxyurea is often used in patients with severe complications who can reliably follow the regimen. Hydroxyurea reduces the frequency of painful crises and the need for blood transfusions in patients with recurrent painful crises. Lastly, the only chance for a cure for Sickle Cell Disease is bone marrow or stem cell transplantation. The bone marrow nurtures stem cells, which are early cells that mature into red and white blood cells and platelets.

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

Sickle Cell Disease (SCD) Epidemiology

The Sickle Cell Disease (SCD) epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by Total Prevalent Cases of SCD Trait, Total Prevalent Cases of SCD, Diagnosed Cases of SCD, Age-specific Cases of SCD, and Type-specific Cases of SCD in the 6MM from 2019 to 2032.

• In 2022, the total prevalent cases of Sickle Cell Disease in the United States were found to be ~104,900 which as per DelveInsight's estimates, will increase by 2032, with a mild CAGR.
• In 2022, Italy accounted for ~7,500 cases of diagnosed cases of SCD, accounting for around 5% of the total diagnosed cases in the 6MM,
• In 2022, Germany accounted for ~2,100 cases of diagnosed cases of SCD, which was approximately 1% of the total diagnosed cases of SCD in the 6MM.
• As per DelveInsight's estimates sickle cell disease was most prevalent in the 18-44 years of age group. In 2022, the age-specific cases of SCD in the France were ~4,700; ~6,900; ~5,900, and ~4,400 in the age groups of 0-17 years, 18-44 years, 45-64 years, and 65 years and above, respectively, which will increase by 2032.

Further details related to epidemiology will be provided in the report…

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 SCD, 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 United States.

Our team of analysts at DelveInsight connected with more than 10 KOLs in the United. We contacted institutions such as the Brooklyn Hospital Center, the Department of Pediatrics, Duke University, and others. By obtaining the opinions of these experts, we gained a better understanding of the current and emerging treatment patterns in the SCD market, which will assist our clients in analyzing the overall epidemiology and market scenario.

The opinion of experts from various regions has been provided below:

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 SCD, one of the most important primary endpoints was the proportion of subjects who have not experienced any severe vaso-occlusive crisis, frequency, and severity of collected adverse events (AEs). 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.

Sickle Cell Disease (SCD) Report Insights

• Patient Population
• Therapeutic Approaches
• Sickle Cell Disease (SCD) Market Size and Trends
• Existing Market Opportunity

Sickle Cell Disease (SCD) Report Key Strengths

• Ten Year Forecast
• The United States Coverage
• Sickle Cell Disease (SCD) Epidemiology Segmentation
• Key Cross Competition

Sickle Cell Disease (SCD) Report Assessment

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

Key Questions

• Would there be any changes in the treatment approach that can cater to the needs of old patients, too, as over 50% of sickle cell disease patients belong to the older age group?
• Will there be any improvements in management recommendations for the pain in sickle cell disease?
• Would research and development advances pave the way for future tests and therapies for sickle cell disease?
• Would the diagnostic testing space experience a significant impact and lead to a positive shift in the treatment landscape of sickle cell disease?
• What kind of uptake will the new therapies witness in the coming years in sickle cell disease patients?

Table of Contents

1. Key Insights

2. Report Introduction

3. Sickle Cell Disease (SCD) Market Overview at a Glance

• 3.1. Market Share (%) Distribution of SCD by Therapies in the 6MM in 2019
• 3.2. Market Share (%) Distribution of SCD by Therapies in the 6MM in 2032

4. Executive Summary of Sickle Cell Disease (SCD)

5. Key events

6. Disease Background and Overview

• 6.1. Introduction
• 6.2. Classification of Sickle Cell Disease
• 6.3. Causes
• 6.4. Associated Risk Factors
• 6.5. Complications
• 6.6. Symptoms
• 6.7. Pathophysiology
• 6.8. Diagnosis
  • 6.8.1. Differential Diagnosis
• 6.9. Treatment and Management of Sickle Cell Disease
  • 6.9.1. Vaso-occlusive Crisis
  • 6.9.2. Chronic Disease
• 6.10. Treatment Algorithm
• 6.11. Treatment Guidelines
  • 6.11.1. American Society of Hematology 2021 Guidelines for Sickle Cell Disease: Stem Cell Transplantation
  • 6.11.2. American Society of Hematology 2020 Guidelines for Sickle Cell Disease: Management of Acute and Chronic Pain
  • 6.11.3. American Society of Hematology 2020 Guidelines for Sickle Cell Disease: Prevention, Diagnosis, and Treatment of Cerebrovascular Disease in Children and Adults
  • 6.11.4. American Society of Hematology 2020 Guidelines for Sickle Cell Disease: Transfusion Support
  • 6.11.5. American Society of Hematology 2019 Guidelines for Sickle Cell Disease: Cardiopulmonary and Kidney Disease
  • 6.11.6. Transition in Sickle Cell Disease (SCD): A German Consensus Recommendation
  • 6.11.7. Management of children with sickle cell disease in Europe: current situation and future perspectives.
  • 6.11.8. Newborn screening for sickle cell disease in Europe: recommendations from a Pan-European Consensus Conference.
  • 6.11.9. Management of sickle cell disease in pregnancy. A British Society for Haematology Guideline

7. Methodology

8. Epidemiology and Patient Population

• 8.1. Key Findings
• 8.2. Assumptions and Rationale
• 8.3. 6MM Total Prevalent Cases of SCD
• 8.4. 6MM Diagnosed Cases of SCD
• 8.5. The United States
  • 8.5.1. Total Prevalent Cases of SCD Trait in the United States
  • 8.5.2. Total Prevalent Cases of SCD in the United States
  • 8.5.3. Diagnosed Cases of SCD in the United States
  • 8.5.4. Age-specific Prevalent Cases of SCD in the United States
  • 8.5.5. Type-specific Prevalence of SCD in the United States
• 8.6. EU4 and the UK
  • 8.6.1. Total Prevalent Cases of SCD Trait in the EU4 and the UK
  • 8.6.2. Total Prevalent Cases of SCD in the EU4 and the UK
  • 8.6.3. Diagnosed Cases of SCD in the EU4 and the UK
  • 8.6.4. Age-specific Prevalent Cases of SCD in EU4 and the UK
  • 8.6.5. Type-specific Prevalent Cases of SCD in EU4 and the UK

9. Patient Journey

10. Marketed Therapies

• 10.1. Key Cross Competition
• 10.2. ENDARI (L-glutamine): Emmaus Life Sciences
  • 10.2.1. Product Description
  • 10.2.2. Regulatory Milestones
  • 10.2.3. Other Developmental Activities
  • 10.2.4. Ongoing Pipeline Activity
  • 10.2.5. Safety and Efficacy
• 10.3. ADAKVEO (crizanlizumab): Novartis Pharmaceuticals
  • 10.3.1. Product Description
  • 10.3.2. Regulatory Milestones
  • 10.3.3. Other Developmental Activities
  • 10.3.4. Ongoing Pipeline Activity
  • 10.3.5. Safety and Efficacy
• 10.4. OXBRYTA (voxelotor): Global Blood Therapeutics/Pfizer
  • 10.4.1. Product Description
  • 10.4.2. Regulatory Milestones
  • 10.4.3. Other Developmental Activities
  • 10.4.4. Ongoing Pipeline Activity
  • 10.4.5. Safety and Efficacy

11. Emerging Therapies

• 11.1. Key Cross Competition
• 11.2. Exa-cel: Vertex Pharmaceuticals/CRISPR Therapeutics
  • 11.2.1. Product Description
  • 11.2.2. Other Developmental Activities
  • 11.2.3. Clinical Developmental Activities
  • 11.2.4. Safety and Efficacy
• 11.3. Lovo-cel: Bluebird Bio
  • 11.3.1. Product Description
  • 11.3.2. Other Developmental Activities
  • 11.3.3. Clinical Developmental Activities
  • 11.3.4. Safety and Efficacy
• 11.4. GBT-601: Global Blood Therapeutics/Pfizer
  • 11.4.1. Product Description
  • 11.4.2. Other Developmental Activities
  • 11.4.3. Clinical Developmental Activities

12. Market Analysis

• 12.1. Key Findings
• 12.2. Key Market Forecast Assumptions
• 12.3. Conjoint Analysis
• 12.4. Market Outlook
• 12.5. Six Major Market Analysis
  • 12.5.1. Total Market Size of SCD in the 6MM
  • 12.5.2. Market Size of SCD by therapies in the 6MM
• 12.6. The United States
  • 12.6.1. Total Market Size of SCD in the United States
  • 12.6.2. Market Size of SCD by therapies in the United States
• 12.7. The EU4 and United Kingdom
  • 12.7.1. Total Market Size of SCD in EU4 and United Kingdom
  • 12.7.2. Market Size of SCD by therapies in EU4 and United Kingdom

13. KOL Views

14. SWOT Analysis

15. Unmet Needs

16. Market Access and Reimbursement

• 16.1. The United States
  • 16.1.1. Center for Medicare & Medicaid Services (CMS)
  • 16.1.2. OXBRYTA
  • 16.1.3. ENDARI
  • 16.1.4. ADAKVEO
• 16.2. EU4 and the UK
  • 16.2.1. Germany
  • 16.2.2. France
  • 16.2.3. Italy
  • 16.2.4. Spain
  • 16.2.5. United Kingdom

17. Appendix

• 17.1. Bibliography
• 17.2. Report Methodology

18. DelveInsight Capabilities

19. Disclaimer