血友病市場 - 市場シェア分析、市場規模、業界動向、数値データおよび成長率予測レポート（2019年〜2029年） ‐ 医薬品/生命科学市場

COVID-19のアウトブレイクは、血友病などの慢性的な医療条件を持つ個人に悪影響を及ぼしました。彼らはCOVID-19に感染するリスクを最小限に抑えるために追加の予防策を講じなければなりませんでした。この集団は感染症にかかりやすかったからです。さらに、2020年8月に『歯科および医学科学ジャーナル』に発表された研究によると、ロックダウン前の平均年間出血率は2.4でした。それに対し、ロックダウン中に、定期的な予防策を講じていた子供たちでは、8.2に大幅に増加しました。血友病治療センターに通えなかった理由として挙げられたさまざまな理由の中で、重要な理由は、交通手段の不在（97.5%）、コロナウイルス感染への恐怖（95%）、代替交通手段の高コスト（90%）でした。これにより、パンデミックの間に市場成長が鈍化しました。しかし、パンデミック制限が緩和され、血友病のための先進的な治療法の利用が増加したため、今後数年で市場は著しく成長する可能性があります。

血友病A型は病気タイプセグメントで重要な市場シェアを持つと期待されています

血友病Aは最も一般的な重度の出血障害です。失われた凝固因子を提供する置換療法は、予防的および出血治療の主な治療法でした。血友病Aは、血友病Aの症例の増加、啓蒙プログラムの増加、血友病Aに対するさまざまな治療法の急増などの要因により、予測期間中に重要な市場シェアを保持すると期待されています。例えば、2021年の世界血友病連盟の年次グローバル調査報告によると、2021年には世界中で233,577人が血友病を抱えており、そのうち185,318件が血友病Aでした。

さらに、血友病関連製品および治療法の承認と認可は、予測期間中の市場成長を促進することが期待されています。例えば、2022年10月にノボ ノルディスクは、18歳未満の血友病B患者における日常的予防のためのREBINYN（凝固因子IX（組換え）、ペグ化）の新しい適応に対して、カナダ保健省の承認を受けました。REBINYNは、血友病B（先天性因子IX欠乏症またはクリスマス病）を持つすべての年齢において、出血エピソードの頻度を防ぐまたは減少させるため、さらに周術期の出血を制御し防ぐための日常的予防に適応されています。

• 1 イントロダクション

  • 1.1 調査の前提条件と市場の定義

  • 1.2 本調査の範囲

• 2 調査手法

• 3 エグゼクティブサマリー

• 4 市場ダイナミクス

  • 4.1 市場概要

  • 4.2 市場の促進要因

    • 4.2.1 血友病の普及率拡大

    • 4.2.2 政府の前向きな取組み

    • 4.2.3 研究開発・新製品開発の活発化

  • 4.3 市場の抑制要因

    • 4.3.1 高い治療コスト

    • 4.3.2 先端技術に対する意識の低さ

  • 4.4 ポーターのファイブフォース分析

    • 4.4.1 サプライヤーの交渉力

    • 4.4.2 買い手・消費者の交渉力

    • 4.4.3 新規参入の脅威

    • 4.4.4 代替品の脅威

    • 4.4.5 競合・競争状況の激しさ

• 5 市場のセグメンテーション（市場規模、米ドル）

  • 5.1 疾病タイプ別

    • 5.1.1 ヘモフィリアA

    • 5.1.2 ヘモフィリアB

    • 5.1.3 その他

  • 5.2 治療別

    • 5.2.1 療法

    • 5.2.2 遺伝子治療

    • 5.2.3 その他

  • 5.3 製品タイプ別

    • 5.3.1 遺伝子組換え凝固因子濃縮製剤

    • 5.3.2 血漿由来凝固因子濃縮製剤

    • 5.3.3 その他

  • 5.4 地域別

    • 5.4.1 北米

      • 5.4.1.1 米国
      • 5.4.1.2 カナダ
      • 5.4.1.3 メキシコ

    • 5.4.2 ヨーロッパ

      • 5.4.2.1 ドイツ
      • 5.4.2.2 英国
      • 5.4.2.3 フランス
      • 5.4.2.4 イタリア
      • 5.4.2.5 スペイン
      • 5.4.2.6 その他のヨーロッパ

    • 5.4.3 アジア太平洋

      • 5.4.3.1 中国
      • 5.4.3.2 日本
      • 5.4.3.3 インド
      • 5.4.3.4 オーストラリア
      • 5.4.3.5 韓国
      • 5.4.3.6 その他のアジア太平洋

    • 5.4.4 中東・アフリカ

      • 5.4.4.1 GCC地域
      • 5.4.4.2 南アフリカ
      • 5.4.4.3 その他の中東・アフリカ

    • 5.4.5 南米

      • 5.4.5.1 ブラジル
      • 5.4.5.2 アルゼンチン
      • 5.4.5.3 その他の南米

• 6 競合情勢

  • 6.1 企業プロファイル

    • 6.1.1 Bayer AG

    • 6.1.2 BioMarin

    • 6.1.3 CSL Limited (CSL Behring)

    • 6.1.4 Kedrion S.p.A

    • 6.1.5 Novo Nordisk A/S

    • 6.1.6 Pfizer, Inc

    • 6.1.7 F. Hoffmann-La Roche AG (Chugai Pharmaceutical Co.)

    • 6.1.8 Sanofi (Genzyme Corporation)

    • 6.1.9 Takeda Pharmaceutical Company Limited (Shire Plc.)

    • 6.1.10 Grifols, S.A

    • 6.1.11 Medexus Pharmaceuticals, Inc

    • 6.1.12 Octapharma AG

• 7 市場機会と今後の動向

The hemophilia market is expected to register a CAGR of 5.7% during the forecast period.

The outbreak of COVID-19 adversely impacted individuals with chronic medical conditions, such as hemophilia. They had to take extra precautions to minimize the risk of contracting COVID-19, as this population group was more prone to infectious diseases. In addition, according to the study published in the Journal of Dental and Medical Sciences in August 2020, the mean annualized bleed rate before the lockdown was 2.4. In contrast, it significantly increased to 8.2 during the lockdown in children who were on regular prophylaxis earlier. Among various reasons cited by them for not attending a Hemophilia treatment center, the important reasons were non-availability of transport (97.5%), fear of coronavirus infection (95%), and high cost of alternate transport (90%). It slowed down the market growth during the pandemic. However, since the pandemic restriction was eased and the rise in availability of advanced therapies for hemophilia, the studied market is likely to grow notably in the coming years.

Certain factors driving the market growth include the growing prevalence of hemophilia, favorable government initiatives, and rising research and development. For instance, according to the World Federation of Hemophilia Global Report 2021, there were 386,966 people with bleeding disorders identified globally, of which 233,577 cases were hemophilia. Such a huge prevalence of hemophilia globally will likely lead to increased adoption of therapies for hemophilia, driving market growth.

Additionally, an increase in grants for research on hemophilia is expected to contribute to the market studied over the forecast period. For instance, in March 2022, a researcher from the Indiana University School of Medicine received USD 2 million from the National Heart Lung and Blood Institute. It is for developing three major themes in a gene therapy approach that could lead to safer and potentially curative treatments for hemophilia. It will lead to the development of novel therapies for hemophilia, thereby expected to drive market growth over the forecast period. Moreover, rising product approvals for the United States Food and Drug Administration are expected to propel the market growth. For instance, in March 2022, Freeline Therapeutics Holdings plc dosed the first patient in its Phase 1/2 B-LIEVE dose-confirmation clinical trial of FLT180a for treating hemophilia B. It is a debilitating genetic bleeding disorder caused by a clotting factor IX protein deficiency.

Thus, due to the rise in hemophilia cases and the increase in product launches coupled with research and development for hemophilia therapies, the studied market is expected to witness significant market growth over the forecast period. However, the high cost of hemophilia treatment and the need for more awareness among people regarding advanced technologies is expected to hinder the studied market.

Hemophilia Market Trends

Hemophilia A is Expected to Hold Significant Market Share in the Disease Type Segment

Hemophilia A is the most common severe bleeding disorder. Replacement therapy, providing the missing coagulation factor, was the mainstay of treatment both prophylactically and to treat bleeding. Hemophilia A is expected to hold a significant market share over the forecast period owing to factors such as an increase in the prevalence of hemophilia A cases, a rise in awareness programs, and a surge in different therapies for hemophilia A. For instance, according to the World Federation of Hemophilia Report on the Annual Global Survey 2021, 233,577 people were living with hemophilia in 2021 globally, out of which 185,318 cases were hemophilia A.

Furthermore, product approval, clearance, and launches will likely increase market opportunities over the forecast period. For instance, in August 2022, the European Commission (EC) granted conditional marketing authorization to valoctocogene roxaparvovec gene therapy under ROCTAVIAN. BioMarin includes multiple clinical studies underway in its comprehensive gene therapy program for treating severe hemophilia A. Similarly, in June 2022, the FDA granted Breakthrough Therapy designation to efanesoctocog alfa (BIVV001) for treating people with hemophilia A. It is a rare, life-threatening bleeding disorder based on pivotal XTEND-1 Phase 3 study data. Sanofi and Sobi collaborated on the development and commercialization of efanesoctocog alfa.

Moreover, research and development to advance the treatment for hemophilia A are widely carried out, which is expected to fuel the market growth over the forecast period. For instance, as per the article published in September 2022 in PubMed, various key players are working on an in vivo genome-editing program based on CRISPR/Cas9. This technique uses non-homologous end-joining to prevent the loss of the AAV vector due to hepatocyte growth. It is to enable the permanent chromosomal integration of a modified human B-domain-deleted FVIII at the albumin locus in liver cells. Young individuals with hemophilia A who are currently ineligible for traditional gene therapy may benefit greatly from such a method.

Therefore, due to the increase in hemophilia A cases and the rise in different pipeline studies and product approvals for hemophilia A, the studied segment is expected to include significant market growth over the forecast period.

North America Expected to Hold Significant Share in the Market and Expected to do Same Over the Forecast Period

North America is expected to hold a significant share of the market. It is expected to do the same over the forecast period due to factors such as the rise in hemophilia cases, increasing awareness about technologically advanced products, and favorable regional government initiatives. For instance, according to the CDC update in August 2022, about 33,000 people in the US contain hemophilia. Hemophilia typically affects men and people assigned male at birth (AMAB). As per the same source, the estimated prevalence of hemophilia in the United States is 12 cases per 100,000 US males for hemophilia A and 3.7 cases per 100,000 US males for hemophilia B.

Furthermore, according to the study published in Frontiers in Immunology in April 2021, in the United States, patients with hereditary disorders, such as hemophilia B, benefited from Adeno-associated virus (AAV)-mediated gene transfer by attaining long-term expression of the therapeutic transgene. Such studies further bolster gene therapies for hemophilia, thereby driving market growth. Moreover, rising initiatives from key market players are also expected to drive market growth in this region. For instance, in February 2022, BioMarin Pharmaceutical Inc. presented positive results from a two-year Phase 3 GENEr8-1 study analysis and an overall safety update of valoctocogene roxaparvovec. It is an investigational gene therapy for treating adults with severe hemophilia A at the 15th Annual Virtual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD).

Additionally, approvals and clearances for hemophilia-related products and therapies are expected to bolster market growth over the forecast period. For instance, in October 2022, Novo Nordisk received Health Canada approval for a new indication for REBINYN (Coagulation Factor IX (Recombinant), pegylated) for the treatment of routine prophylaxis in hemophilia B patients that are <18 years of age. REBINYN is now indicated in all ages with hemophilia B (congenital factor IX deficiency or Christmas disease) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes and control and prevent bleeding in a perioperative setting.

Thus, North America is expected to include a significant market share over the forecast period due to the increase in hemophilia cases and the rise in different therapies for hemophilia coupled with research and development.

Hemophilia Industry Overview

The hemophilia market is moderately competitive and consists of a few major players. The key players are also involved in various strategic alliances such as acquisitions, collaborations, and launching advanced products and research studies to secure their position in the global market. Some of the key market players in this market include Bayer AG, BioMarin, CSL Limited (CSL Behring), Novo Nordisk, Pfizer, Inc., Sanofi (Genzyme Corporation), Takeda Pharmaceutical Company Limited (Shire Plc.), among others.

Additional Benefits:

• The market estimate (ME) sheet in Excel format
• 3 months of analyst support

• 1 INTRODUCTION

  • 1.1 Study Assumptions and Market Definition

  • 1.2 Scope of the Study

• 2 RESEARCH METHODOLOGY

• 3 EXECUTIVE SUMMARY

• 4 MARKET DYNAMICS

  • 4.1 Market Overview

  • 4.2 Market Drivers

    • 4.2.1 Growing Prevalence of Hemophilia

    • 4.2.2 Favorable Government Initiatives

    • 4.2.3 Rising R&D and New Product Development

  • 4.3 Market Restraints

    • 4.3.1 High Cost of Treatment

    • 4.3.2 Lack of Awareness Regarding Advanced Technologies

  • 4.4 Porter's Five Forces Analysis

    • 4.4.1 Bargaining Power of Suppliers

    • 4.4.2 Bargaining Power of Buyers/Consumers

    • 4.4.3 Threat of New Entrants

    • 4.4.4 Threat of Substitute Products

    • 4.4.5 Intensity of Competitive Rivalry

• 5 MARKET SEGMENTATION (Market Size by Value - USD)

  • 5.1 By Disease Type

    • 5.1.1 Hemophilia A

    • 5.1.2 Hemophilia B

    • 5.1.3 Others

  • 5.2 By Therapy

    • 5.2.1 Replacement Therapy

    • 5.2.2 Gene Therapy

    • 5.2.3 Others

  • 5.3 By Product Type

    • 5.3.1 Recombinant Coagulation Factor Concentrates

    • 5.3.2 Plasma-derived Coagulation Factor Concentrates

    • 5.3.3 Others

  • 5.4 Geography

    • 5.4.1 North America

      • 5.4.1.1 United States
      • 5.4.1.2 Canada
      • 5.4.1.3 Mexico

    • 5.4.2 Europe

      • 5.4.2.1 Germany
      • 5.4.2.2 United Kingdom
      • 5.4.2.3 France
      • 5.4.2.4 Italy
      • 5.4.2.5 Spain
      • 5.4.2.6 Rest of Europe

    • 5.4.3 Asia-Pacific

      • 5.4.3.1 China
      • 5.4.3.2 Japan
      • 5.4.3.3 India
      • 5.4.3.4 Australia
      • 5.4.3.5 South Korea
      • 5.4.3.6 Rest of Asia-Pacific

    • 5.4.4 Middle East and Africa

      • 5.4.4.1 GCC
      • 5.4.4.2 South Africa
      • 5.4.4.3 Rest of Middle East and Africa

    • 5.4.5 South America

      • 5.4.5.1 Brazil
      • 5.4.5.2 Argentina
      • 5.4.5.3 Rest of South America

• 6 COMPETITIVE LANDSCAPE

  • 6.1 Company Profiles

    • 6.1.1 Bayer AG

    • 6.1.2 BioMarin

    • 6.1.3 CSL Limited (CSL Behring)

    • 6.1.4 Kedrion S.p.A

    • 6.1.5 Novo Nordisk A/S

    • 6.1.6 Pfizer, Inc

    • 6.1.7 F. Hoffmann-La Roche AG (Chugai Pharmaceutical Co.)

    • 6.1.8 Sanofi (Genzyme Corporation)

    • 6.1.9 Takeda Pharmaceutical Company Limited (Shire Plc.)

    • 6.1.10 Grifols, S.A

    • 6.1.11 Medexus Pharmaceuticals, Inc

    • 6.1.12 Octapharma AG

• 7 MARKET OPPORTUNITIES AND FUTURE TRENDS