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市場調査レポート
商品コード
1353499
鎌状赤血球症治療の世界市場規模、シェア、産業動向分析レポート:治療別、エンドユース別、地域別展望と予測、2023年~2030年Global Sickle Cell Disease Treatment Market Size, Share & Industry Trends Analysis Report By Treatment, By End-Use, By Regional Outlook and Forecast, 2023 - 2030 |
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鎌状赤血球症治療の世界市場規模、シェア、産業動向分析レポート:治療別、エンドユース別、地域別展望と予測、2023年~2030年 |
出版日: 2023年08月31日
発行: KBV Research
ページ情報: 英文 203 Pages
納期: 即納可能
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鎌状赤血球症治療の市場規模は2030年までに73億米ドルに達すると予測され、予測期間中のCAGRは27.9%の市場成長率で上昇する見込みです。
KBV Cardinalのマトリックスに掲載された分析によると、ファイザーが同市場におけるトップランナーです。Novo Nordisk A/S社、Novartis AG社、Bristol Myers Squibb Company社などの企業は、市場における主要なイノベーターです。2019年10月、Novo Nordisk A/Sは、遺伝性疾患のためのin vivoゲノム編集治療法を開発するためにbluebird bio, Incとパートナーシップを締結しました。この提携により、ノボノルディスクは疾患修飾薬の開発により有利な立場に立つことになります。
COVID-19影響分析
ヘルスケア業界のワークフローは、世界中でCOVID-19の大流行によって妨げられました。ヘルスケアのいくつかのサブドメインを含むいくつかの産業は、病気の流行により一時的な閉鎖を余儀なくされました。市場拡大を制限するさらなる要因は、COVID-19の際に鎌状赤血球症治療薬のサプライチェーンが混乱したことです。さらに、病院の閉鎖は入院に悪影響を及ぼし、市場に影響を与えました。パンデミックの後期には、集団予防接種の増加、厳格なCOVID-19ガイドラインの実施、渡航制限の解除により、輸血および骨髄移植産業の成長は正常に戻っています。したがって、パンデミックは市場に大きなマイナスの影響を与えました。
市場成長要因
新しい治療法の開発
以前は、輸血と骨髄移植がSCDの唯一の治療法でした。SCDの有病率と認知度が高まるにつれ、代替となる効果的な治療法の需要が高まっています。主要企業は、市場の需要に応えるため、この疾患を治療する新製品の導入に重点を置くようになっています。エンダリは、数十年ぶりに承認されたブランド薬であり、それ以前は、この疾患の治療法を考案する上で技術革新に重点を置くことは限られていました。適切なドナーを持たない鎌状赤血球症患者は、自身の造血幹細胞を変化させる遺伝子治療によって治癒する可能性があります。このような市場開拓は、鎌状赤血球症に対する利用可能な治療法の数を増やし、多くの患者をカバーする可能性があるため、市場の成長を助長します。
臨床試験と製薬研究の近代化
製薬研究業界のリーダーたちは現在、研究開発プロセスの効率的な実施方法を模索しており、機械学習はその有望なソリューションと思われます。世界のトップ製薬会社は、新しい治療法を発見するヒット・アンド・ミスのプロセスを改善するために機械学習を利用しています。さらに、多くの大手製薬会社は、創薬プロセスをスピードアップするためのビッグデータの可能性を調査しています。これは、全体として、より小規模で、より迅速で、より安価な臨床試験につながると思われます。より安全性を高めるために、MLは遠隔モニタリングやリアルタイムのデータアクセスにも利用できます。そのため、より迅速で効果的な医薬品開発を可能にするMLを活用した製薬分野の臨床試験や研究開発の発展は、市場の成長を促進すると思われます。
市場抑制要因
SCDの治療選択肢が少ない
鎌状赤血球症の治療に用いられる薬物療法は、ヒドロキシ尿素と少数のブランド薬です。多くのヘルスケア専門家がヒドロキシ尿素を第一選択薬として推奨しています。しかし、世界中の国や地域で治療の選択肢がないことが、大きな制約要因となっています。WHOの小児用必須医薬品モデルリストに含まれているにもかかわらず、ヒドロキシ尿素はこの地域ではまだ入手できないです。さらに、アフリカではこの物質は高価すぎると考えられています。さらに、新興経済諸国では国民の間でこの病気に対する認識が低いことも、予測期間中の市場開拓の妨げになる可能性があります。
治療法の展望
治療法によって、市場は輸血、薬物療法、骨髄移植に分類されます。骨髄移植セグメントは、2022年の市場でかなりの収益シェアを占めています。このセグメントは、新しいブランド医薬品の出現と、これらの医薬品を早期に上市するための政府のインセンティブが高まっているため、急速な拡大を経験しています。政府は鎌状赤血球症の研究を支援するため、希少疾病用医薬品、ファスト・トラック、優先審査などの資金援助や指定を行っています。
最終用途別の展望
最終用途に基づき、市場は病院、専門クリニック、その他に分類されます。2022年には、病院セグメントが市場で最大の売上シェアを占める。病気の診断と治療の主な中心は病院です。輸血や骨髄移植のほとんどは病院で行われます。加えて、優れたヘルスケアサービス、大規模な患者プール、充実した医療インフラが、このセグメントの市場シェアが大きい主な理由の1つです。
地域別展望
地域別に見ると、市場は北米、欧州、アジア太平洋、LAMEAで分析されます。2022年には、北米地域が最も高い収益シェアを獲得して市場をリードしました。同地域の拡大は、SCD治療薬の増加や有望なパイプライン候補に起因しています。米国の強力な政府支援は市場成長をさらに促進すると思われます。同地域の市場は、鎌状赤血球症の罹患率の増加、臨床試験件数の増加、製品上市件数の増加により、平均よりも急速に成長すると予測されます。
List of Figures
The Global Sickle Cell Disease Treatment Market size is expected to reach $7.3 billion by 2030, rising at a market growth of 27.9% CAGR during the forecast period.
According to a study by NCBI in 2021, the disease can expose patients to up to 18 VOCs per year. The increasing prevalence of SCD and the efficacy of the products used to treat the disease in decreasing the risk of VOCs are boosting the product's usage among the patient population. Therefore, The Bone Marrow Transplant segment would capture 1/5thshare in the market by 2030, as the incidence of SCD disease has been rising more quickly. Vaso-occlusive crisis is a frequent and excruciating complication of sickle cell disease in adolescents and adults. By replacing the patient's diseased bone marrow with healthy donor marrow, this procedure can effectively eliminate the root cause of SCD, which is the production of abnormal hemoglobin. It causes episodes of acute, excruciating pain causing patients, including infants and adults, to seek urgent medical care.
The major strategies followed by the market participants are Partnerships as the key developmental strategy to keep pace with the changing demands of end users. For instance, In June, 2022, Novartis AG came into partnership with Precision BioSciences, Inc, to develop solutions for in vivo gene editing. Through the partnership, Novartis AG would be better positioned to provide effective treatment for patients with hemoglobinopathies. Additionally, In November, 2021, Emmaus Life Sciences, Inc. partnered with UpScript IP Holdings, LLC, to provide telehealth solutions for patients with sickle cell disease.
Based on the Analysis presented in the KBV Cardinal matrix; Pfizer Inc. is the forerunner in the Market. Companies such as Novo Nordisk A/S, Novartis AG, Bristol Myers Squibb Company are some of the key innovators in the Market. In October, 2019, Novo Nordisk A/S signed a partnership with bluebird bio, Inc, to develop in vivo genome editing treatments for inherited diseases. Through the partnership, Novo Nordisk would be better positioned to develop disease-modifying medicines.
COVID-19 Impact Analysis
Workflows in the healthcare industry were hampered by the COVID-19 pandemic all over the world. Several industries, including several sub-domains of healthcare, were compelled to close temporarily due to the disease outbreak. A further factor limiting market expansion was the disruption of the sickle cell disease drug supply chain during COVID-19. Additionally, the lockdown had a detrimental effect on hospital admissions, affecting the market. In the later phases of the pandemic, the growth of the blood transfusion and bone marrow transplant industries returned to normal due to a rise in population vaccination, the implementation of stringent COVID-19 guidelines, and the lifting of travel restrictions. Therefore, the pandemic had a significant negative impact on the market.
Market Growth Factors
Development of new treatment methods
Earlier, blood transfusions and bone marrow transplants were the only treatments for SCD. The increasing prevalence and awareness of the disease boosted the demand for alternative, effective treatment types. Major players have increased their emphasis on introducing new products to treat this disease to meet market demand. Endari was the first branded drug to be approved in decades, before which there was a limited emphasis on innovation in devising a treatment for the disease. Sickle cell disease patients without a suitable donor may be cured by genetic therapies that alter their own hematopoietic stem cells. Such development increases the number of available treatments for SCD and may cover a large proportion of patient base, and thus, aids in the growth of the market.
Modernization of clinical trials and pharmaceutical research
Leaders in the pharmaceutical research industry are now exploring to implement efficient ways of doing the RD processes, for which machine learning appear to be a promising solution. The top pharmaceutical companies in the world are using machine learning to better the hit-and-miss process of discovering new treatments. In addition, many large pharmaceutical companies are investigating the potential of big data to speed up the drug discovery process. This would lead to smaller, quicker, and more affordable trials overall. For greater safety, ML can also be used for remote monitoring and real-time data access. Therefore, the advancement of clinical trials and R&D in the pharma sector with ML, which enables faster and more effective drug development, will propel the market growth.
Market Restraining Factors
Minimal treatment options available for SCD
Hydroxyurea and a few branded medications are the pharmacotherapy used to treat sickle cell disease. Numerous healthcare professionals recommend hydroxyurea as the first-line treatment for the disease. However, the absence of treatment options in countries and regions around the world stands out as a significant constraining factor. Despite its inclusion on the WHO Model List of Essential Medicines for Children, hydroxyurea is still unavailable in the region. In addition, the substance is considered too costly in Africa. In addition, the lack of awareness of the disease among the population in developing economies can hinder the development of the market during the forecast period.
Treatment Outlook
By treatment, the market is categorized into blood transfusion, pharmacotherapy, and bone marrow transplant. The bone marrow transplant segment covered a considerable revenue share in the market in 2022. The segment has experienced rapid expansion due to the emergence of new, branded pharmaceutical medications and growing government incentives to launch these drugs early. The government provides funding and designations such as orphan pharmaceuticals, fast track, and priority review, among others, to support sickle cell disease research.
End-Use Outlook
Based on end-use, the market is classified into hospitals, specialty clinics, and others. In 2022 the hospitals segment witnessed the largest revenue share in the market. The primary centres for disease diagnosis and treatment are hospitals. Most blood transfusions and bone marrow transplants are performed in hospitals. In addition, superior healthcare services, a large patient pool, and an enhanced healthcare infrastructure are some of the primary reasons for the segment's large market share.
Regional Outlook
Region wise, the market is analysed across North America, Europe, Asia Pacific, and LAMEA. In 2022 the North America region led the market by generating the highest revenue share. The expansion in the region can be attributed to the increased availability of SCD treatments and promising pipeline candidates. Strong government support in the United States will further promote market growth. The region's market is anticipated to grow faster than average due to the increasing incidence of sickle cell disease, the rising number of clinical trials, and the increasing number of product launches.
The market research report covers the analysis of key stakeholders of the market. Key companies profiled in the report include Novartis AG, Pfizer Inc., bluebird bio, Inc., CSL Limited, Emmaus Life Sciences, Inc., Bristol Myers Squibb Company, CRISPR Therapeutics AG, Agios Pharmaceuticals, Inc. (LES LABORATOIRES SERVIER SAS), Novo Nordisk A/S and Medunik USA (Duchesnay Inc.)
Recent Strategies Deployed in Sickle Cell Disease Treatment Market
Partnerships, Collaborations & Agreements:
Jun-2022: Novartis AG came into partnership with Precision BioSciences, Inc., a biotechnology company based in the United States, to develop solutions for in vivo gene editing. Through the partnership, Novartis AG would be better positioned to provide effective treatment for patients with hemoglobinopathies.
Nov-2021: Emmaus Life Sciences, Inc. partnered with UpScript IP Holdings, LLC, a pharmaceutical company, to provide telehealth solutions for patients with sickle cell disease. The partnership would allow Emmaus Life Sciences to provide patients with access to its Endari treatment.
Jul-2021: Bluebird Bio, Inc. signed a partnership with National Resilience, Inc., a biotechnology company, to develop solutions for cell therapies. The partnership comes in line with Bluebird's ambition for the development of effective cell therapies.
Apr-2021: CRISPR Therapeutics AG entered into a partnership with Vertex Pharmaceuticals Incorporated, a US-based biotechnology company, to market its CTX001 gene editing therapy for sickle cell disease treatment. The partnership would extend the global reach of CRISPR's CTX001 gene editing therapy.
Feb-2021: Novartis AG announced a partnership with Bill & Melinda Gates Foundation, an American private foundation, to develop solutions for vivo gene therapy for sickle cell disease treatment. The partnership would pave the way for effective sickle cell disease treatment for patients around the world.
Mergers & Acquisition:
Oct-2022: Pfizer Inc. acquired Global Blood Therapeutics, Inc., a biopharmaceutical company. The acquisition strengthens Pfizer's capabilities to provide sickle cell disease treatment solutions.
Sep-2022: Novo Nordisk A/S announced the acquisition of Forma Therapeutics, a biotech company specializing in sickle cell disease (SCD) treatment. The acquisition enhances Novo Nordisk's sickle cell disease treatment offerings.
Product Launches and Product Expansions:
Apr-2022: Emmaus Life Sciences, Inc. announced the launch of a full-service telehealth solution. The solutions feature improved access to Endari sickle disease treatment drugs. The launch extends Endari's reach to a wider population of patients with sickle cell disease.
Trials and Approvals:
Jul-2023: The Oman Ministry of Health approved Emmaus Life Sciences' Endari, an Oral medicine used for the treatment of sickle cell disease.
Dec-2021: Medunik USA received approval from The Food and Drug Administration (FDA) for new applications of its Siklos, a sickle cell anemia treatment solution.
Nov-2020: Agios Pharmaceuticals, Inc. received approval from the U.S. Food and Drug Administration (FDA) for its mitapivat, a pyruvate kinase R (PKR) activator used for the treatment of sickle cell disease.
Oct-2020: Novartis AG received approval from the European Commission (EC) for its Adakveo, a sickle cell disease treatment used for the prevention of recurrent vaso-occlusive crises (VOCs).
Market Segments covered in the Report:
By Treatment
By End-Use
By Geography
Companies Profiled
Unique Offerings from KBV Research