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鐮狀細胞疾病治療市場規模和預測、全球和地區佔有率、趨勢以及按治療、給藥途徑和配銷通路分類的成長機會分析

市場調查報告書

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1389687

鐮狀細胞疾病治療市場規模和預測、全球和地區佔有率、趨勢以及按治療、給藥途徑和配銷通路分類的成長機會分析

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 | 訂單完成後即時交付

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鐮狀細胞疾病治療市場預計將從2022年的11.6億美元成長到2030年的46.91億美元；預計 2022 年至 2030 年CAGR為19.1%。由於鐮狀細胞病患疾病率不斷增加，以及政府和私營部門提高人們對鐮狀細胞疾病認知的舉措，鐮狀細胞疾病治療市場正在不斷成長。此外，先進診斷工具的推出數量不斷增加，以及提供有效治療方法的研究活動不斷增加，也進一步推動了市場的成長。然而，缺乏鐮狀細胞疾病治療方案等因素限制了市場的成長。

鐮狀細胞疾病（SCD）的治療方案可分為兩類：維持健康和治療併發症。鐮狀細胞性貧血具有基於血紅蛋白基因獨特突變的基因型和表現型變異。該疾病在全球發病率很高，而且嚴重程度也在上升。據世界衛生組織稱，全球約有5%的人口具有抑制血紅蛋白疾病（如鐮狀細胞性貧血和地中海貧血）的基因。根據美國國家醫學圖書館發表的一項關於鐮狀細胞疾病的研究，每年有超過 30 萬名嬰兒出生時患有嚴重的血紅蛋白疾病；此外，到2050年，預計將有40萬名新生兒患有鐮狀細胞疾病。此外，根據美國血液學會2023年發布的報告，預計美國患有SCD的人數約為70,000-100,000人。因此，全球不同地區瘧疾和鐮狀細胞病患疾病率的上升推動了瘧疾和鐮狀細胞疾病治療市場的成長。

多年來，專門的兒科計畫和研究措施顯著改善了患者護理和預期壽命。以下是一些為解決美國和世界各地的 SCD 治療問題籌集資金的基金會。

美國血液學會基金會尋求籌集 50 萬美元的私人慈善醫療資金，以開發側重於以下方面的具體計劃：

提高人們對 SCD 的認知並鼓勵採取行動

培訓和教育治療 SCD 患者的血液學家和其他醫療保健提供者

減少對 SCD 計劃的資助

提高 SCD 患者的護理標準、研究計畫和臨床試驗的機會

支持血液科醫師和其他醫療保健專業人員在各個階段（即從兒童期到成年期的早期診斷）對 SCD 患者進行最佳治療

多麗絲杜克基金會 (Doris Duke Foundation) 撥款支持 SCD 的先進治療方法，包括基因修飾和恢復血紅蛋白功能的藥物治療。支援專案的年度直接成本為 150,000 美元至 300,000 美元，加上每三年 10% 的間接成本。該獎項旨在支持臨床研究，這些研究將支持：

臨床上針對鐮狀細胞疾病的先進基因療法，例如基因添加和基因組編輯

建立珠蛋白調節機制來恢復紅血球功能

推進骨髓移植程序，最大限度地減少死亡時間並改善結果

2021 年 8 月，部落事務部與 ICMR 合作，向各州發放了 720 萬美元，尋求撥款來解決鐮狀細胞貧血症，包括篩檢。部落事務部 (MoTA) 部落研究所 (TRI) 部門批准了甘加拉姆爵士醫院關於鐮狀細胞疾病的研究計畫。此外，還開展了一個名為「為鐮狀細胞性貧血和地中海貧血患者提供診斷、IEC 和 Nutri 支持」的計畫。

2021 年 2 月，諾華與比爾及梅琳達蓋茲基金會合作，確定並開發了一種可用於鐮狀細胞疾病的體內基因療法。該夥伴關係的資金為728 萬美元，旨在開發一種負擔得起且足夠簡單的治療方法，以便在SCD 發病率高的資源匱乏地區使用，特別是撒哈拉以南非洲地區，全世界約80%的受影響人群都在該地區位於。此次合作希望創造一種現成的治療方法，可以繞過目前治療 SCD 的基因治療方法中涉及的一些體內步驟，這些步驟成本高昂、複雜，並且是針對個別患者設計的。

2022 年 3 月，一群國際研究人員獲得了美國國立衛生研究院 (NIH) 的 300 萬美元撥款，用於對加納鐮狀細胞病兒童的整個遺傳密碼進行測序。透過分析 500 名加納 SCD 兒童的整個 DNA 序列，研究人員希望找出疾病的潛在遺傳修飾因素，有助於改善患者的管理和照護。這些孩子是非洲鐮狀細胞疾病基因組網路 (SickleGenAfrica) 的參與者。 Ofori-Acquah 領導的一個由 NIH 資助、耗資 540 萬美元的國際計畫也致力於了解遺傳學如何影響非洲人的 SCD 進展。

基於治療的見解

鐮狀細胞疾病治療市場依治療分為學名藥和原廠藥。到 2022 年，原廠藥細分市場佔據最大的市場佔有率。鐮狀細胞疾病是一種以血紅蛋白缺陷為特徵的遺傳性血液疾病。它抑制紅血球中血紅蛋白攜帶氧氣的能力。

基於管理的洞察之路

根據給藥途徑，瘧疾治療市場分為口服、腸胃外和靜脈注射。 2022年，口服細分市場佔最大市場佔有率；然而，預計注射劑領域在預測期內的CAGR最高。口服給藥是最優選和最合適的給藥途徑，因為它提供了高患者依從性、非侵入性、最少的無菌限制、成本效益、劑型設計的靈活性以及製造過程的簡便性。易於給藥和長期成本效益等優點是推動口服藥物採用的主要因素。

基於配銷通路的見解

根據分銷管道，瘧疾治療市場分為直接招標、醫院藥房、零售藥房、網路藥房等。 2022年直接招標部分佔據最大市場佔有率；然而，線上藥局領域預計在預測期內CAGR最高。

在準備瘧疾治療市場報告時提到的一些主要一手和二手來源包括世界衛生組織 (WHO)、美國人口普查局和疾病預防控制中心等。

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