希少疾病用医薬品市場：疾患タイプ、製品タイプ、流通チャネル別-2025-2030年世界予測

希少疾病用医薬品市場は、2023年に1,918億6,000万米ドルと評価され、2024年には2,168億米ドルに達すると予測され、CAGR 13.39%で成長し、2030年には4,624億5,000万米ドルに達すると予測されています。

希少疾病用医薬品は、人口のごく一部が罹患する希少疾患の治療に特化したもので、アンメット・メディカル・ニーズへの対応において重要な役割を果たしています。これらの疾患は、その希少性から有効な治療法がないことが多く、希少疾病用医薬品は製薬業界において不可欠な焦点となっています。これらの医薬品市場は、希少疾患に対する治療法の提供が急務であり、市場独占権や税額控除などの規制上の優遇措置が増加していることを特徴としています。希少疾病用医薬品の主な用途は、遺伝性疾患、希少がん、その他の希少疾患にわたり、病院、診療所、専門薬局での最終用途が大きいです。市場の成長は、バイオテクノロジーの技術的進歩、規制機関からの積極的な支援、個別化医療への注目の高まりによって推進されています。とはいえ、高い開発コスト、長い承認プロセス、限られた患者プールといった課題が、進展の妨げになることもあります。未開拓のヘルスケアニーズを抱える新興国市場への進出や、研究開発活動のための戦略的提携など、市場は潜在的なビジネスチャンスで急成長しています。企業は、遺伝子治療、個別化医療、適応型臨床試験デザインに投資することで、医薬品開発に関連する時間とコストを削減し、その機会を活用することができます。しかし、企業は複雑な規制状況や倫理的配慮を乗り越えなければならないです。イノベーションは主に、創薬と開発を合理化するための精密医療と、AIとビッグデータ分析の統合に焦点を当てるべきです。また、希少疾患の新規経路やターゲットの探索にも重点を置いた調査が必要です。この市場の性質は、患者中心の医療を目指す傾向や、迅速な医薬品開発に対応した規制の枠組みなど、徐々にダイナミックになっています。こうした動向に適応し、戦略的パートナーシップに投資する企業は、ニッチではあるが急速に進化するこの分野で競合優位に立てる可能性が高いです。

市場力学：急速に進化する希少疾病用医薬品市場の主要市場インサイトを公開

希少疾病用医薬品市場は、需要と供給のダイナミックな相互作用によって変貌を遂げています。このような市場力学の進化を理解することで、企業は十分な情報に基づいた投資決定、戦略的意思決定、新たなビジネスチャンスの獲得を行うことができます。これらの動向を包括的に把握することで、企業は政治的、地理的、技術的、社会的、経済的な領域にわたる様々なリスクを軽減することができるとともに、消費者行動とそれが製造コストや購買動向に与える影響をより明確に理解することができます。

• 市場促進要因
  • 希少疾患の急増とヘルスケア支出の増加
  • 希少疾患治療薬の開発に対する政府・経済的優遇措置
  • 希少疾病用医薬品の独占権の有無
• 市場抑制要因
  • 希少疾病用医薬品に関連する高コスト
• 市場機会
  • 研究開発イニシアチブの増加と新規製品の開発
  • 新興諸国における希少疾病用医薬品の開発に対する規制上のインセンティブ
• 市場の課題
  • 希少疾病用医薬品に伴う副作用

ポーターの5つの力：希少疾病用医薬品市場をナビゲートする戦略ツール

ポーターの5つの力フレームワークは、希少疾病用医薬品市場の競合情勢を理解するための重要なツールです。ポーターのファイブフォース・フレームワークは、企業の競争力を評価し、戦略的機会を探るための明確な手法を提供します。このフレームワークは、企業が市場内の勢力図を評価し、新規事業の収益性を判断するのに役立ちます。これらの洞察により、企業は自社の強みを活かし、弱みに対処し、潜在的な課題を回避することができ、より強靭な市場でのポジショニングを確保することができます。

PESTLE分析：希少疾病用医薬品市場における外部からの影響の把握

外部マクロ環境要因は、希少疾病用医薬品市場の業績ダイナミクスを形成する上で極めて重要な役割を果たします。政治的、経済的、社会的、技術的、法的、環境的要因の分析は、これらの影響をナビゲートするために必要な情報を提供します。PESTLE要因を調査することで、企業は潜在的なリスクと機会をよりよく理解することができます。この分析により、企業は規制、消費者の嗜好、経済動向の変化を予測し、先を見越した積極的な意思決定を行う準備ができます。

市場シェア分析希少疾病用医薬品市場における競合情勢の把握

希少疾病用医薬品市場の詳細な市場シェア分析により、ベンダーの業績を包括的に評価することができます。企業は、収益、顧客ベース、成長率などの主要指標を比較することで、競争上のポジショニングを明らかにすることができます。この分析により、市場の集中、断片化、統合の動向が明らかになり、ベンダーは競争が激化する中で自社の地位を高める戦略的意思決定を行うために必要な知見を得ることができます。

FPNVポジショニング・マトリックス希少疾病用医薬品市場におけるベンダーのパフォーマンス評価

FPNVポジショニングマトリックスは、希少疾病用医薬品市場においてベンダーを評価するための重要なツールです。このマトリックスにより、ビジネス組織はベンダーのビジネス戦略と製品満足度に基づき評価することで、目標に沿った十分な情報に基づいた意思決定を行うことができます。4つの象限によってベンダーを明確かつ正確にセグメント化し、戦略目標に最適なパートナーやソリューションを特定することができます。

戦略分析と推奨希少疾病用医薬品市場における成功への道筋を描く

希少疾病用医薬品市場の戦略分析は、世界市場でのプレゼンス強化を目指す企業にとって不可欠です。主要なリソース、能力、業績指標を見直すことで、企業は成長機会を特定し、改善に取り組むことができます。このアプローチにより、競合情勢における課題を克服し、新たなビジネスチャンスを活かして長期的な成功を収めるための体制を整えることができます。

本レポートでは、主要な注目分野を網羅した市場の包括的な分析を提供しています：

1.市場の浸透度：現在の市場環境の詳細なレビュー、主要企業による広範なデータ、市場でのリーチと全体的な影響力の評価。

2.市場の開拓度：新興市場における成長機会を特定し、既存分野における拡大可能性を評価し、将来の成長に向けた戦略的ロードマップを提供します。

3.市場の多様化：最近の製品発売、未開拓の地域、業界の主要な進歩、市場を形成する戦略的投資を分析します。

4.競合の評価と情報：競合情勢を徹底的に分析し、市場シェア、事業戦略、製品ポートフォリオ、認証、規制当局の承認、特許動向、主要企業の技術進歩などを検証します。

5.製品開発およびイノベーション：将来の市場成長を促進すると期待される最先端技術、研究開発活動、製品イノベーションをハイライトしています。

また、利害関係者が十分な情報を得た上で意思決定できるよう、重要な質問にも答えています：

1.現在の市場規模と今後の成長予測は？

2.最高の投資機会を提供する製品、セグメント、地域はどこか？

3.市場を形成する主な技術動向と規制の影響とは？

4.主要ベンダーの市場シェアと競合ポジションは？

5.ベンダーの市場参入・撤退戦略の原動力となる収益源と戦略的機会は何か？

目次

第1章 序文

第2章 調査手法

第3章 エグゼクティブサマリー

第4章 市場の概要

第5章 市場洞察

• 市場力学
  • 促進要因
    • 希少疾患の急増とヘルスケア費の増加
    • 希少疾患の治療薬開発に対する政府および経済の有利なインセンティブ
    • 希少疾病用医薬品の独占権の可用性
  • 抑制要因
    • 希少疾病用医薬品に関連する高コスト
  • 機会
    • 研究開発の取り組みと新製品の開発の増加
    • 新興国における希少疾病用医薬品の開発に対する規制上のインセンティブ
  • 課題
    • 希少疾病用医薬品に関連する副作用
• 市場セグメンテーション分析
• ポーターのファイブフォース分析
• PESTEL分析
  • 政治的
  • 経済
  • 社交
  • 技術的
  • 法律上
  • 環境

第6章 希少疾病用医薬品市場：疾患タイプ別

• 胃腸
• 血液学
• 感染症
• 神経学
• 腫瘍学
• 肺

第7章 希少疾病用医薬品市場：製品タイプ別

• 生物学的
• 非生物学的

第8章 希少疾病用医薬品市場：流通チャネル別

• 病院薬局
• 小売薬局
• 専門薬局

第9章 南北アメリカの希少疾病用医薬品市場

• アルゼンチン
• ブラジル
• カナダ
• メキシコ
• 米国

第10章 アジア太平洋地域の希少疾病用医薬品市場

• オーストラリア
• 中国
• インド
• インドネシア
• 日本
• マレーシア
• フィリピン
• シンガポール
• 韓国
• 台湾
• タイ
• ベトナム

第11章 欧州・中東・アフリカの希少疾病用医薬品市場

• デンマーク
• エジプト
• フィンランド
• フランス
• ドイツ
• イスラエル
• イタリア
• オランダ
• ナイジェリア
• ノルウェー
• ポーランド
• カタール
• ロシア
• サウジアラビア
• 南アフリカ
• スペイン
• スウェーデン
• スイス
• トルコ
• アラブ首長国連邦
• 英国

第12章 競合情勢

• 市場シェア分析2023
• FPNVポジショニングマトリックス, 2023
• 競合シナリオ分析
• 戦略分析と提言

企業一覧

• Alexion Pharmaceuticals, Inc.
• AstraZeneca PLC
• Bayer AG
• Belite Bio Inc
• Biogen Inc.
• Dynacure SA
• Eisai Co. Ltd.
• Eli Lilly and Company
• F. Hoffmann-La Roche Ltd
• GlaxoSmithKline PLC
• Johnson & Johnson Services, Inc.
• MeiraGTx Limited
• Novartis AG
• Pfizer Inc.
• Sanofi S.A.

LIST OF FIGURES

• FIGURE 1. ORPHAN DRUGS MARKET RESEARCH PROCESS
• FIGURE 2. ORPHAN DRUGS MARKET SIZE, 2023 VS 2030
• FIGURE 3. GLOBAL ORPHAN DRUGS MARKET SIZE, 2018-2030 (USD MILLION)
• FIGURE 4. GLOBAL ORPHAN DRUGS MARKET SIZE, BY REGION, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 5. GLOBAL ORPHAN DRUGS MARKET SIZE, BY COUNTRY, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 6. GLOBAL ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2023 VS 2030 (%)
• FIGURE 7. GLOBAL ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 8. GLOBAL ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2023 VS 2030 (%)
• FIGURE 9. GLOBAL ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 10. GLOBAL ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2023 VS 2030 (%)
• FIGURE 11. GLOBAL ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 12. AMERICAS ORPHAN DRUGS MARKET SIZE, BY COUNTRY, 2023 VS 2030 (%)
• FIGURE 13. AMERICAS ORPHAN DRUGS MARKET SIZE, BY COUNTRY, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 14. UNITED STATES ORPHAN DRUGS MARKET SIZE, BY STATE, 2023 VS 2030 (%)
• FIGURE 15. UNITED STATES ORPHAN DRUGS MARKET SIZE, BY STATE, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 16. ASIA-PACIFIC ORPHAN DRUGS MARKET SIZE, BY COUNTRY, 2023 VS 2030 (%)
• FIGURE 17. ASIA-PACIFIC ORPHAN DRUGS MARKET SIZE, BY COUNTRY, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 18. EUROPE, MIDDLE EAST & AFRICA ORPHAN DRUGS MARKET SIZE, BY COUNTRY, 2023 VS 2030 (%)
• FIGURE 19. EUROPE, MIDDLE EAST & AFRICA ORPHAN DRUGS MARKET SIZE, BY COUNTRY, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 20. ORPHAN DRUGS MARKET SHARE, BY KEY PLAYER, 2023
• FIGURE 21. ORPHAN DRUGS MARKET, FPNV POSITIONING MATRIX, 2023

LIST OF TABLES

• TABLE 1. ORPHAN DRUGS MARKET SEGMENTATION & COVERAGE
• TABLE 2. UNITED STATES DOLLAR EXCHANGE RATE, 2018-2023
• TABLE 3. GLOBAL ORPHAN DRUGS MARKET SIZE, 2018-2030 (USD MILLION)
• TABLE 4. GLOBAL ORPHAN DRUGS MARKET SIZE, BY REGION, 2018-2030 (USD MILLION)
• TABLE 5. GLOBAL ORPHAN DRUGS MARKET SIZE, BY COUNTRY, 2018-2030 (USD MILLION)
• TABLE 6. ORPHAN DRUGS MARKET DYNAMICS
• TABLE 7. GLOBAL ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 8. GLOBAL ORPHAN DRUGS MARKET SIZE, BY GASTROINTESTINAL, BY REGION, 2018-2030 (USD MILLION)
• TABLE 9. GLOBAL ORPHAN DRUGS MARKET SIZE, BY HEMATOLOGY, BY REGION, 2018-2030 (USD MILLION)
• TABLE 10. GLOBAL ORPHAN DRUGS MARKET SIZE, BY INFECTIOUS DISEASES, BY REGION, 2018-2030 (USD MILLION)
• TABLE 11. GLOBAL ORPHAN DRUGS MARKET SIZE, BY NEUROLOGY, BY REGION, 2018-2030 (USD MILLION)
• TABLE 12. GLOBAL ORPHAN DRUGS MARKET SIZE, BY ONCOLOGY, BY REGION, 2018-2030 (USD MILLION)
• TABLE 13. GLOBAL ORPHAN DRUGS MARKET SIZE, BY PULMONARY, BY REGION, 2018-2030 (USD MILLION)
• TABLE 14. GLOBAL ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 15. GLOBAL ORPHAN DRUGS MARKET SIZE, BY BIOLOGIC, BY REGION, 2018-2030 (USD MILLION)
• TABLE 16. GLOBAL ORPHAN DRUGS MARKET SIZE, BY NON-BIOLOGIC, BY REGION, 2018-2030 (USD MILLION)
• TABLE 17. GLOBAL ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 18. GLOBAL ORPHAN DRUGS MARKET SIZE, BY HOSPITAL PHARMACIES, BY REGION, 2018-2030 (USD MILLION)
• TABLE 19. GLOBAL ORPHAN DRUGS MARKET SIZE, BY RETAIL PHARMACIES, BY REGION, 2018-2030 (USD MILLION)
• TABLE 20. GLOBAL ORPHAN DRUGS MARKET SIZE, BY SPECIALTY PHARMACIES, BY REGION, 2018-2030 (USD MILLION)
• TABLE 21. AMERICAS ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 22. AMERICAS ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 23. AMERICAS ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 24. AMERICAS ORPHAN DRUGS MARKET SIZE, BY COUNTRY, 2018-2030 (USD MILLION)
• TABLE 25. ARGENTINA ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 26. ARGENTINA ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 27. ARGENTINA ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 28. BRAZIL ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 29. BRAZIL ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 30. BRAZIL ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 31. CANADA ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 32. CANADA ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 33. CANADA ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 34. MEXICO ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 35. MEXICO ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 36. MEXICO ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 37. UNITED STATES ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 38. UNITED STATES ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 39. UNITED STATES ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 40. UNITED STATES ORPHAN DRUGS MARKET SIZE, BY STATE, 2018-2030 (USD MILLION)
• TABLE 41. ASIA-PACIFIC ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 42. ASIA-PACIFIC ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 43. ASIA-PACIFIC ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 44. ASIA-PACIFIC ORPHAN DRUGS MARKET SIZE, BY COUNTRY, 2018-2030 (USD MILLION)
• TABLE 45. AUSTRALIA ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 46. AUSTRALIA ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 47. AUSTRALIA ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 48. CHINA ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 49. CHINA ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 50. CHINA ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 51. INDIA ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 52. INDIA ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 53. INDIA ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 54. INDONESIA ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 55. INDONESIA ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 56. INDONESIA ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 57. JAPAN ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 58. JAPAN ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 59. JAPAN ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 60. MALAYSIA ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 61. MALAYSIA ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 62. MALAYSIA ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 63. PHILIPPINES ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 64. PHILIPPINES ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 65. PHILIPPINES ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 66. SINGAPORE ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 67. SINGAPORE ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 68. SINGAPORE ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 69. SOUTH KOREA ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 70. SOUTH KOREA ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 71. SOUTH KOREA ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 72. TAIWAN ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 73. TAIWAN ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 74. TAIWAN ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 75. THAILAND ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 76. THAILAND ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 77. THAILAND ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 78. VIETNAM ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 79. VIETNAM ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 80. VIETNAM ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 81. EUROPE, MIDDLE EAST & AFRICA ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 82. EUROPE, MIDDLE EAST & AFRICA ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 83. EUROPE, MIDDLE EAST & AFRICA ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 84. EUROPE, MIDDLE EAST & AFRICA ORPHAN DRUGS MARKET SIZE, BY COUNTRY, 2018-2030 (USD MILLION)
• TABLE 85. DENMARK ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 86. DENMARK ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 87. DENMARK ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 88. EGYPT ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 89. EGYPT ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 90. EGYPT ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 91. FINLAND ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 92. FINLAND ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 93. FINLAND ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 94. FRANCE ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 95. FRANCE ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 96. FRANCE ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 97. GERMANY ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 98. GERMANY ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 99. GERMANY ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 100. ISRAEL ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 101. ISRAEL ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 102. ISRAEL ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 103. ITALY ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 104. ITALY ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 105. ITALY ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 106. NETHERLANDS ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 107. NETHERLANDS ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 108. NETHERLANDS ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 109. NIGERIA ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 110. NIGERIA ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 111. NIGERIA ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 112. NORWAY ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 113. NORWAY ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 114. NORWAY ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 115. POLAND ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 116. POLAND ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 117. POLAND ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 118. QATAR ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 119. QATAR ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 120. QATAR ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 121. RUSSIA ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 122. RUSSIA ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 123. RUSSIA ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 124. SAUDI ARABIA ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 125. SAUDI ARABIA ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 126. SAUDI ARABIA ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 127. SOUTH AFRICA ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 128. SOUTH AFRICA ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 129. SOUTH AFRICA ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 130. SPAIN ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 131. SPAIN ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 132. SPAIN ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 133. SWEDEN ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 134. SWEDEN ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 135. SWEDEN ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 136. SWITZERLAND ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 137. SWITZERLAND ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 138. SWITZERLAND ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 139. TURKEY ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 140. TURKEY ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 141. TURKEY ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 142. UNITED ARAB EMIRATES ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 143. UNITED ARAB EMIRATES ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 144. UNITED ARAB EMIRATES ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 145. UNITED KINGDOM ORPHAN DRUGS MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 146. UNITED KINGDOM ORPHAN DRUGS MARKET SIZE, BY PRODUCT TYPE, 2018-2030 (USD MILLION)
• TABLE 147. UNITED KINGDOM ORPHAN DRUGS MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 148. ORPHAN DRUGS MARKET SHARE, BY KEY PLAYER, 2023
• TABLE 149. ORPHAN DRUGS MARKET, FPNV POSITIONING MATRIX, 2023

The Orphan Drugs Market was valued at USD 191.86 billion in 2023, expected to reach USD 216.80 billion in 2024, and is projected to grow at a CAGR of 13.39%, to USD 462.45 billion by 2030.

Orphan drugs, specifically designed to treat rare diseases affecting a small percentage of the population, play a critical role in addressing unmet medical needs. These conditions often lack effective treatments due to their rarity, making orphan drugs an essential focus in the pharmaceutical industry. The market for these drugs is driven by the urgent necessity to provide therapies for rare diseases and is characterized by an increasing number of regulatory incentives like market exclusivity and tax credits. The primary application of orphan drugs spans across genetic disorders, rare cancers, and other rare conditions, with significant end-use in hospitals, clinics, and specialty pharmacies. Market growth is propelled by technological advancements in biotechnology, active support from regulatory bodies, and the heightened focus on personalized medicine. Nonetheless, challenges such as high development costs, lengthy approval processes, and limited patient pools can hinder progress. The market is burgeoning with potential opportunities, namely the expansion into emerging markets with untapped healthcare needs, and strategic collaborations for research and development activities. Companies can capitalize on opportunities by investing in gene therapy, personalized medicine, and adaptive clinical trial designs which can reduce the time and cost associated with drug development. However, firms must navigate through complex regulatory landscapes and ethical considerations. Innovations should primarily focus on precision medicine and the integration of AI and big data analytics to streamline drug discovery and development. Research should also emphasize the exploration of novel pathways and targets for rare disorders. The nature of this market is progressively dynamic, with a trend toward patient-centric care and regulatory frameworks being more accommodating for expedited drug development. Businesses that adapt to these trends and invest in strategic partnerships are likely to gain a competitive edge in this niche but rapidly evolving sector.

Market Dynamics: Unveiling Key Market Insights in the Rapidly Evolving Orphan Drugs Market

The Orphan Drugs Market is undergoing transformative changes driven by a dynamic interplay of supply and demand factors. Understanding these evolving market dynamics prepares business organizations to make informed investment decisions, refine strategic decisions, and seize new opportunities. By gaining a comprehensive view of these trends, business organizations can mitigate various risks across political, geographic, technical, social, and economic domains while also gaining a clearer understanding of consumer behavior and its impact on manufacturing costs and purchasing trends.

• Market Drivers
  • Surge in prevalence of rare diseases and increasing healthcare expenditure
  • Favorable government and economic incentives for the development of drugs for rare diseases
  • Availability of exclusivity for orphan drugs
• Market Restraints
  • High cost associated with orphan drugs
• Market Opportunities
  • Increasing R&D initiatives and development of novel products
  • Regulatory incentives for the development of orphan drugs in emerging countries
• Market Challenges
  • Side effects associated with orphan drugs

Porter's Five Forces: A Strategic Tool for Navigating the Orphan Drugs Market

Porter's five forces framework is a critical tool for understanding the competitive landscape of the Orphan Drugs Market. It offers business organizations with a clear methodology for evaluating their competitive positioning and exploring strategic opportunities. This framework helps businesses assess the power dynamics within the market and determine the profitability of new ventures. With these insights, business organizations can leverage their strengths, address weaknesses, and avoid potential challenges, ensuring a more resilient market positioning.

PESTLE Analysis: Navigating External Influences in the Orphan Drugs Market

External macro-environmental factors play a pivotal role in shaping the performance dynamics of the Orphan Drugs Market. Political, Economic, Social, Technological, Legal, and Environmental factors analysis provides the necessary information to navigate these influences. By examining PESTLE factors, businesses can better understand potential risks and opportunities. This analysis enables business organizations to anticipate changes in regulations, consumer preferences, and economic trends, ensuring they are prepared to make proactive, forward-thinking decisions.

Market Share Analysis: Understanding the Competitive Landscape in the Orphan Drugs Market

A detailed market share analysis in the Orphan Drugs Market provides a comprehensive assessment of vendors' performance. Companies can identify their competitive positioning by comparing key metrics, including revenue, customer base, and growth rates. This analysis highlights market concentration, fragmentation, and trends in consolidation, offering vendors the insights required to make strategic decisions that enhance their position in an increasingly competitive landscape.

FPNV Positioning Matrix: Evaluating Vendors' Performance in the Orphan Drugs Market

The Forefront, Pathfinder, Niche, Vital (FPNV) Positioning Matrix is a critical tool for evaluating vendors within the Orphan Drugs Market. This matrix enables business organizations to make well-informed decisions that align with their goals by assessing vendors based on their business strategy and product satisfaction. The four quadrants provide a clear and precise segmentation of vendors, helping users identify the right partners and solutions that best fit their strategic objectives.

Strategy Analysis & Recommendation: Charting a Path to Success in the Orphan Drugs Market

A strategic analysis of the Orphan Drugs Market is essential for businesses looking to strengthen their global market presence. By reviewing key resources, capabilities, and performance indicators, business organizations can identify growth opportunities and work toward improvement. This approach helps businesses navigate challenges in the competitive landscape and ensures they are well-positioned to capitalize on newer opportunities and drive long-term success.

Key Company Profiles

The report delves into recent significant developments in the Orphan Drugs Market, highlighting leading vendors and their innovative profiles. These include Alexion Pharmaceuticals, Inc., AstraZeneca PLC, Bayer AG, Belite Bio Inc, Biogen Inc., Dynacure SA, Eisai Co. Ltd., Eli Lilly and Company, F. Hoffmann-La Roche Ltd, GlaxoSmithKline PLC, Johnson & Johnson Services, Inc., MeiraGTx Limited, Novartis AG, Pfizer Inc., and Sanofi S.A..

Market Segmentation & Coverage

This research report categorizes the Orphan Drugs Market to forecast the revenues and analyze trends in each of the following sub-markets:

• Based on Disease Type, market is studied across Gastrointestinal, Hematology, Infectious Diseases, Neurology, Oncology, and Pulmonary.
• Based on Product Type, market is studied across Biologic and Non-Biologic.
• Based on Distribution Channel, market is studied across Hospital Pharmacies, Retail Pharmacies, and Specialty Pharmacies.
• Based on Region, market is studied across Americas, Asia-Pacific, and Europe, Middle East & Africa. The Americas is further studied across Argentina, Brazil, Canada, Mexico, and United States. The United States is further studied across California, Florida, Illinois, New York, Ohio, Pennsylvania, and Texas. The Asia-Pacific is further studied across Australia, China, India, Indonesia, Japan, Malaysia, Philippines, Singapore, South Korea, Taiwan, Thailand, and Vietnam. The Europe, Middle East & Africa is further studied across Denmark, Egypt, Finland, France, Germany, Israel, Italy, Netherlands, Nigeria, Norway, Poland, Qatar, Russia, Saudi Arabia, South Africa, Spain, Sweden, Switzerland, Turkey, United Arab Emirates, and United Kingdom.

The report offers a comprehensive analysis of the market, covering key focus areas:

1. Market Penetration: A detailed review of the current market environment, including extensive data from top industry players, evaluating their market reach and overall influence.

2. Market Development: Identifies growth opportunities in emerging markets and assesses expansion potential in established sectors, providing a strategic roadmap for future growth.

3. Market Diversification: Analyzes recent product launches, untapped geographic regions, major industry advancements, and strategic investments reshaping the market.

4. Competitive Assessment & Intelligence: Provides a thorough analysis of the competitive landscape, examining market share, business strategies, product portfolios, certifications, regulatory approvals, patent trends, and technological advancements of key players.

5. Product Development & Innovation: Highlights cutting-edge technologies, R&D activities, and product innovations expected to drive future market growth.

The report also answers critical questions to aid stakeholders in making informed decisions:

1. What is the current market size, and what is the forecasted growth?

2. Which products, segments, and regions offer the best investment opportunities?

3. What are the key technology trends and regulatory influences shaping the market?

4. How do leading vendors rank in terms of market share and competitive positioning?

5. What revenue sources and strategic opportunities drive vendors' market entry or exit strategies?

Table of Contents

1. Preface

• 1.1. Objectives of the Study
• 1.2. Market Segmentation & Coverage
• 1.3. Years Considered for the Study
• 1.4. Currency & Pricing
• 1.5. Language
• 1.6. Stakeholders

2. Research Methodology

• 2.1. Define: Research Objective
• 2.2. Determine: Research Design
• 2.3. Prepare: Research Instrument
• 2.4. Collect: Data Source
• 2.5. Analyze: Data Interpretation
• 2.6. Formulate: Data Verification
• 2.7. Publish: Research Report
• 2.8. Repeat: Report Update

3. Executive Summary

4. Market Overview

5. Market Insights

• 5.1. Market Dynamics
  • 5.1.1. Drivers
    • 5.1.1.1. Surge in prevalence of rare diseases and increasing healthcare expenditure
    • 5.1.1.2. Favorable government and economic incentives for the development of drugs for rare diseases
    • 5.1.1.3. Availability of exclusivity for orphan drugs
  • 5.1.2. Restraints
    • 5.1.2.1. High cost associated with orphan drugs
  • 5.1.3. Opportunities
    • 5.1.3.1. Increasing R&D initiatives and development of novel products
    • 5.1.3.2. Regulatory incentives for the development of orphan drugs in emerging countries
  • 5.1.4. Challenges
    • 5.1.4.1. Side effects associated with orphan drugs
• 5.2. Market Segmentation Analysis
• 5.3. Porter's Five Forces Analysis
  • 5.3.1. Threat of New Entrants
  • 5.3.2. Threat of Substitutes
  • 5.3.3. Bargaining Power of Customers
  • 5.3.4. Bargaining Power of Suppliers
  • 5.3.5. Industry Rivalry
• 5.4. PESTLE Analysis
  • 5.4.1. Political
  • 5.4.2. Economic
  • 5.4.3. Social
  • 5.4.4. Technological
  • 5.4.5. Legal
  • 5.4.6. Environmental

6. Orphan Drugs Market, by Disease Type

• 6.1. Introduction
• 6.2. Gastrointestinal
• 6.3. Hematology
• 6.4. Infectious Diseases
• 6.5. Neurology
• 6.6. Oncology
• 6.7. Pulmonary

7. Orphan Drugs Market, by Product Type

• 7.1. Introduction
• 7.2. Biologic
• 7.3. Non-Biologic

8. Orphan Drugs Market, by Distribution Channel

• 8.1. Introduction
• 8.2. Hospital Pharmacies
• 8.3. Retail Pharmacies
• 8.4. Specialty Pharmacies

9. Americas Orphan Drugs Market

• 9.1. Introduction
• 9.2. Argentina
• 9.3. Brazil
• 9.4. Canada
• 9.5. Mexico
• 9.6. United States

10. Asia-Pacific Orphan Drugs Market

• 10.1. Introduction
• 10.2. Australia
• 10.3. China
• 10.4. India
• 10.5. Indonesia
• 10.6. Japan
• 10.7. Malaysia
• 10.8. Philippines
• 10.9. Singapore
• 10.10. South Korea
• 10.11. Taiwan
• 10.12. Thailand
• 10.13. Vietnam

11. Europe, Middle East & Africa Orphan Drugs Market

• 11.1. Introduction
• 11.2. Denmark
• 11.3. Egypt
• 11.4. Finland
• 11.5. France
• 11.6. Germany
• 11.7. Israel
• 11.8. Italy
• 11.9. Netherlands
• 11.10. Nigeria
• 11.11. Norway
• 11.12. Poland
• 11.13. Qatar
• 11.14. Russia
• 11.15. Saudi Arabia
• 11.16. South Africa
• 11.17. Spain
• 11.18. Sweden
• 11.19. Switzerland
• 11.20. Turkey
• 11.21. United Arab Emirates
• 11.22. United Kingdom

12. Competitive Landscape

• 12.1. Market Share Analysis, 2023
• 12.2. FPNV Positioning Matrix, 2023
• 12.3. Competitive Scenario Analysis
• 12.4. Strategy Analysis & Recommendation

Companies Mentioned

• 1. Alexion Pharmaceuticals, Inc.
• 2. AstraZeneca PLC
• 3. Bayer AG
• 4. Belite Bio Inc
• 5. Biogen Inc.
• 6. Dynacure SA
• 7. Eisai Co. Ltd.
• 8. Eli Lilly and Company
• 9. F. Hoffmann-La Roche Ltd
• 10. GlaxoSmithKline PLC
• 11. Johnson & Johnson Services, Inc.
• 12. MeiraGTx Limited
• 13. Novartis AG
• 14. Pfizer Inc.
• 15. Sanofi S.A.