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镰状细胞疾病治疗市场规模和预测、全球和地区份额、趋势以及按治疗、给药途径和配销通路分類的成长机会分析

市场调查报告书

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镰状细胞疾病治疗市场规模和预测、全球和地区份额、趋势以及按治疗、给药途径和配销通路分類的成长机会分析

Sickle Cell Disease Treatment Market Size and Forecasts, Global and Regional Share, Trends, and Growth Opportunity Analysis By Treatment, Route of Administration, and Distribution Channel

出版日期: 2023年11月07日 | 出版商:

The Insight Partners | 英文 245 Pages | 订单完成后即时交付

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镰状细胞疾病治疗市场预计将从2022年的11.6亿美元成长到2030年的46.91亿美元；预计 2022 年至 2030 年CAGR为19.1%。由于镰状细胞病患疾病率不断增加，以及政府和私营部门提高人们对镰状细胞疾病认识的倡议，镰状细胞疾病治疗市场正在不断增长。此外，先进诊断工具的推出数量不断增加，以及提供有效治疗方法的研究活动不断增加，也进一步推动了市场的成长。然而，缺乏镰状细胞疾病治疗方案等因素限制了市场的成长。

镰状细胞疾病（SCD）的治疗方案可分为两类：维持健康和治疗併发症。镰状细胞性贫血具有基于血红蛋白基因独特突变的基因型和表型变异。该疾病在全球发病率很高，而且严重程度也在上升。据世界卫生组织称，全球约有5%的人口具有抑制血红蛋白疾病（如镰状细胞性贫血和地中海贫血）的基因。根据美国国家医学图书馆发表的一项关于镰状细胞疾病的研究，每年有超过 30 万名婴儿出生时患有严重的血红蛋白疾病；此外，到2050年，预计将有40万名新生儿患有镰状细胞疾病。此外，根据美国血液学会2023年发布的报告，预计美国患有SCD的人数约为70,000-100,000人。因此，全球不同地区疟疾和镰状细胞病患疾病率的上升推动了疟疾和镰状细胞疾病治疗市场的成长。

多年来，专门的儿科计画和研究措施显着改善了患者护理和预期寿命。以下是一些为解决美国和世界各地的 SCD 治疗问题筹集资金的基金会。

美国血液学会基金会寻求筹集 50 万美元的私人慈善医疗资金，以开发侧重于以下方面的具体计划：

提高人们对 SCD 的认识并鼓励采取行动

培训和教育治疗 SCD 患者的血液学家和其他医疗保健提供者

减少对 SCD 计划的资助

提高 SCD 患者的护理标准、研究计画和临床试验的机会

支持血液科医师和其他医疗保健专业人员在各个阶段（即从儿童期到成年期的早期诊断）对 SCD 患者进行最佳治疗

多丽丝杜克基金会 (Doris Duke Foundation) 拨款支持 SCD 的先进治疗方法，包括基因修饰和恢復血红蛋白功能的药物治疗。支援专案的年度直接成本为 150,000 美元至 300,000 美元，加上每三年 10% 的间接成本。该奖项旨在支持临床研究，这些研究将支持：

临床上针对镰状细胞疾病的先进基因疗法，例如基因添加和基因组编辑

建立珠蛋白调节机制来恢復红血球功能

推进骨髓移植程序，最大限度地减少死亡时间并改善结果

2021 年 8 月，部落事务部与 ICMR 合作，向各州发放了 720 万美元，寻求拨款来解决镰状细胞贫血症，包括筛检。部落事务部 (MoTA) 部落研究所 (TRI) 部门批准了甘加拉姆爵士医院关于镰状细胞疾病的研究计画。此外，还开展了一个名为「为镰状细胞性贫血和地中海贫血患者提供诊断、IEC 和 Nutri 支持」的计画。

2021 年 2 月，诺华与比尔及梅琳达盖兹基金会合作，确定并开发了一种可用于镰状细胞疾病的体内基因疗法。该伙伴关係的资金为728 万美元，旨在开发一种负担得起且足够简单的治疗方法，以便在SCD 发病率高的资源匮乏地区使用，特别是撒哈拉以南非洲地区，全世界约80%的受影响人群都在该地区位于。此次合作希望创造一种现成的治疗方法，可以绕过目前治疗 SCD 的基因治疗方法中涉及的一些体内步骤，这些步骤成本高昂、复杂，并且是针对个别患者设计的。

2022 年 3 月，一群国际研究人员获得了美国国立卫生研究院 (NIH) 的 300 万美元拨款，用于对加纳镰状细胞病儿童的整个遗传密码进行测序。透过分析 500 名加纳 SCD 儿童的整个 DNA 序列，研究人员希望找出疾病的潜在遗传修饰因素，有助于改善患者的管理和照护。这些孩子是非洲镰状细胞疾病基因组网络 (SickleGenAfrica) 的参与者。 Ofori-Acquah 领导的一个由 NIH 资助、耗资 540 万美元的国际计画也致力于了解遗传学如何影响非洲人的 SCD 进展。

基于治疗的见解

镰状细胞疾病治疗市场依治疗分为仿製药和原厂药。到 2022 年，原厂药细分市场占据最大的市场份额。镰状细胞疾病是一种以血红蛋白缺陷为特征的遗传性血液疾病。它抑制红血球中血红蛋白携带氧气的能力。

基于管理的洞察之路

根据给药途径，疟疾治疗市场分为口服、肠胃外和静脉注射。 2022年，口服细分市场占最大市场份额；然而，预计注射剂领域在预测期内的CAGR最高。口服给药是最优选和最合适的给药途径，因为它提供了高患者依从性、非侵入性、最少的无菌限制、成本效益、剂型设计的灵活性以及製造过程的简便性。易于给药和长期成本效益等优点是推动口服药物采用的主要因素。

基于配销通路的见解

根据分销管道，疟疾治疗市场分为直接招标、医院药房、零售药房、网路药房等。 2022年直接招标部分占据最大市场份额；然而，线上药局领域预计在预测期内CAGR最高。

在准备疟疾治疗市场报告时提到的一些主要一手和二手来源包括世界卫生组织 (WHO)、美国人口普查局和疾病预防控制中心等。

The sickle cell disease treatment market is expected to grow from US$ 1.160 billion in 2022 to US$ 4.691 billion by 2030; it is estimated to grow at a CAGR of 19.1% from 2022 to 2030. The market for sickle cell disease treatment is growing due to the increasing prevalence of sickle cell disease and the government and private sector's initiatives to raise awareness about sickle cell disease. Also, the increasing number of launches of advanced diagnostic tools and rising research activities to provide effective therapeutics are further driving the growth of the market. However, factors such as lack of sickle cell disease treatment options are restraining the market growth.

The treatment plan for sickle cell disease (SCD) can be divided into two categories: maintaining health and treating complications. Sickle cell anemia has genotypic and phenotypic variants that are based on unique mutations in hemoglobin genes. There is a high prevalence of the disease as well as a rise in its severity across the globe. According to the WHO, approximately 5% of the world's population has genes inhibiting hemoglobin diseases such as sickle cell anemia and thalassemia. According to a study on sickle cell disease published in the National Library of Medicine, more than 300,000 babies are born with severe hemoglobin disorders each year; also, 400,000 newborns are expected to have sickle cell disease by 2050. In addition, according to a published report in 2023 by the American Society of Hematology, the estimated number of people suffering from SCD in the US is approximately 70,000-100,000. Thus, the rising prevalence of malaria and sickle cell disease in different regions across the globe boosts the malaria and sickle cell disease treatment market growth.

Over the years, dedicated pediatric programs and research initiatives have significantly improved patient care and life expectancy. Following are a few foundations that have raised funds to address SCD treatment in the US and around the world.

American Society of Hematology Foundation seeks to raise US$ 500,000 in private charitable care to develop specific programs that focus on:

Raising awareness and encouraging action against SCD

Training and educating hematologists and other healthcare providers who treat SCD patients

Alleviating funding of SCD programs

Improving standards of care, research programs, and access to clinical trials for SCD people

Supporting workforce of hematologists and other health care professionals to optimally treat SCD patients throughout all stages-i.e., early diagnosis through childhood and into adulthood

Doris Duke Foundation grant awards to support advanced healing methods for SCD, including gene modification and drug therapies to restore hemoglobin function. The annual direct costs to support projects range from US$ 150,000-300,000 plus 10% indirect costs for each of three years. This award aims to support clinical research that will support:

Advanced gene therapies such as gene addition and genome editing for sickle cell disease in clinics

Build on globin regulatory mechanisms to restore red blood cell function

Advance bone marrow transplant procedures to minimize deadliness and improve outcomes

In August 2021, the Ministry of Tribal Affairs, in collaboration with ICMR, released US$ 7.2 million to the states seeking grants to tackle sickle cell anemia, including screening. The Tribal Research Institute (TRI) Division of the Ministry of Tribal Affairs (MoTA) sanctioned a research study program at Sir Ganga Ram Hospital on sickle cell disease. Also, a project called "'Diagnosis, IEC and Nutri support for sickle cell anemia and Thalassemia affected patients" was undertaken.

In February 2021, Novartis and the Bill & Melinda Gates Foundation collaborated to determine and develop an accessible in vivo gene therapy for sickle cell disease. The partnership, funded with US$ 7.28 million, aims to develop a treatment that is affordable and simple enough to be used in low-resource areas with a high prevalence of SCD, particularly sub-Saharan Africa, where about 80% of affected people worldwide are located. The collaboration hopes to create an off-the-shelf treatment that can bypass some of the in vivo steps involved in current gene therapy approaches to treat SCD, which are costly, complex, and crafted for individual patients.

In March 2022, a team of international researchers received a grant of US$ 3 million from the National Institutes of Health (NIH) to sequence the whole genetic code of children with sickle cell disease in Ghana. By analyzing the whole DNA sequence of 500 Ghanaian children with SCD, the researchers hope to identify potential genetic modifiers of the disease that will help improve the management and care of patients. These children are the participants in the Sickle Cell Disease Genomics Network of Africa (SickleGenAfrica). A US$ 5.4-million, NIH-funded international project led by Ofori-Acquah also focused on understanding how genetics influence SCD progression in Africans.

Treatment-Based Insights

The sickle cell disease treatment market is segmented based on treatment into generic drugs and originators. Originators segment held the largest market share in 2022. Sickle cell disease is an inherited blood disorder marked by defective hemoglobin. It inhibits the capability of hemoglobin in red blood cells to carry oxygen.

Route of Administration-Based Insights

Based on route of administration, the malaria treatment market is bifurcated into oral and parenteral & intravenous. The oral segment accounted for the largest share of the market in 2022; however, the parenteral segment is expected to register the highest CAGR during the forecast period. Oral drug delivery is the most preferred and suitable route of drug administration as it offers high patient compliance, non-invasiveness, least sterility constraints, cost-effectiveness, flexibility in the design of dosage form and ease in the manufacturing process. Benefits such as ease of administration and long-term cost efficiency are major factors fueling the adoption of oral drugs.

Distribution Channel-Based Insights

Based on distribution channels, the malaria treatment market is segmented into direct tender, hospital pharmacies, retail pharmacies, online pharmacies, and others. The direct tender segment accounted for the largest share of the market in 2022; however, the online pharmacies segment is expected to register the highest CAGR during the forecast period.

A few of the major primary and secondary sources referred to while preparing the report on the malaria treatment market are the World Health Organization (WHO), the US Census Bureau, and CDC, among others.

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1. Introduction

1.1 The Insight Partners Research Report Guidance
1.2 Market Segmentation

2. Executive Summary

2.1 Key Insights - Malaria
2.2 Key Insights – Sickle Cell Disease

3. Research Methodology

3.1 Coverage
3.2 Secondary Research
3.3 Primary Research

4. Sickle Cell Disease Treatment Market Landscape

4.1 Overview
4.2 PEST Analysis – Sickle Cell Disease Treatment

5. Sickle Cell Disease Treatment Market - Key Industry Dynamics

5.1 Key Market Drivers:
5.2 Market Restraints
5.2.1 Lack of Sickle Cell Disease Treatment Options
5.3 Market Opportunities
5.4 Future Trends
5.5 Impact Analysis 44

6. Sickle Cell Disease Treatment Market - Global Market Analysis

6.1 Sickle Cell Disease Treatment Market Revenue (US$ million), 2022 – 2030
6.2 Geography Analysis Market Revenue (US$ million), 2022 – 2030
6.3 Sickle Cell Disease
6.4 Fundings for Sickle Cell Disease (SCD)
6.5 Reimbursement Scenario for Sickle Cell Disease

7. Sickle Cell Disease Treatment Market – Revenue and Forecast to 2030 – by Treatment

7.1 Overview
7.2 Sickle Cell Disease Treatment Market Revenue Share, by Treatment 2022 & 2030 (%)
7.3 Originators
7.4 Generic Drugs

8. Sickle Cell Disease Treatment Market – Revenue and Forecast to 2030 – by Route of Administration

8.1 Overview
8.2 Sickle Cell Disease Treatment Market Revenue Share, by Route of Administration 2022 & 2030 (%)
8.3 Oral .
8.4 Parenteral

9. Sickle Cell Disease Treatment Market – Revenue and Forecast to 2030 – by Distribution Channel

9.1 Overview
9.2 Sickle Cell Disease Treatment Market Revenue Share, by Distribution Channel 2022 & 2030 (%)
9.3 Direct Tender
9.4 Hospital Pharmacies
9.5 Retail Pharmacies
9.6 Online Pharmacies
9.7 Others

10. Sickle Cell Disease Treatment Market - Geographical Analysis

10.1 North America Sickle Cell Disease Treatment Market, Revenue and Forecast To 2030
10.2 Europe Sickle Cell Disease Treatment Market, Revenue and Forecast To 2030
10.3 Asia Pacific Sickle Cell Disease Treatment Market, Revenue and Forecast To 2030
10.4 Middle East & Africa Sickle Cell Disease Treatment Market, Revenue and Forecast To 2030
10.5 South & Central America Sickle Cell Disease Treatment Market, Revenue and Forecast To 2030