希少疾病用医薬品（オーファンドラッグ）の世界市場：治療分野別、薬剤タイプ別、流通チャネル別、地域別、機会、予測、2018年～2032年

世界の希少疾病用医薬品（オーファンドラッグ）の市場規模は、2025年～2032年の予測期間中に15.07%のCAGRで拡大し、2024年の2,083億2,000万米ドルから2032年には6,402億1,000万米ドルに成長すると予測されています。世界の希少疾病用医薬品市場は、希少疾病の有病率の上昇と社会的・臨床的認知度の向上によって牽引されています。規制当局の支援と財政的インセンティブが、製薬会社の特殊治療への投資を後押ししています。さらに、がん領域における技術革新と、強力なインフラによる北米の優位性が、市場の上昇軌道を形成しています。

例えば、2024年3月、Terns Pharmaceuticalsは、FDAが慢性骨髄性白血病の治療薬としてTERN-701に希少疾病用医薬品の指定を与えたと発表しました。TERN-701はアロステリックBCR-ABLチロシンキナーゼ阻害薬で、現在第1相臨床開発中です。

当レポートでは、世界の希少疾病用医薬品（オーファンドラッグ）市場について調査し、市場の概要とともに、治療分野別、薬剤タイプ別、流通チャネル別、地域別動向、および市場に参入する企業のプロファイルなどを提供しています。

目次

第1章 プロジェクトの範囲と定義

第2章 調査手法

第3章 米国の関税の影響

第4章 エグゼクティブサマリー

第5章 世界の希少疾病用医薬品市場の見通し、2018年～2032年

• 市場規模分析と予測
• 市場シェア分析と予測
  • 治療分野別
    • 腫瘍学
    • 血液学
    • 神経学
    • 内分泌学
    • 心血管系
    • 呼吸器
    • 免疫療法
    • 感染症
    • その他
  • 薬剤タイプ別
    • 生物学的製剤
    • 非生物学的製剤
  • 流通チャネル別
    • 病院薬局
    • 小売薬局とドラッグストア
    • オンライン薬局
  • 地域別
    • 北米
    • 欧州
    • アジア太平洋
    • 南米
    • 中東・アフリカ
  • 企業別市場シェア分析（上位5社およびその他- 金額別、2024年）
• 2024年の市場マップ分析

第6章 北米の希少疾病用医薬品市場の見通し、2018年～2032年

• 市場規模分析と予測
• 市場シェア分析と予測
• 国別市場評価
  • 米国
  • カナダ
  • メキシコ

第7章 欧州の希少疾病用医薬品市場の見通し、2018年～2032年

• ドイツ
• フランス
• イタリア
• 英国
• ロシア
• オランダ
• スペイン
• トルコ
• ポーランド

第8章 アジア太平洋の希少疾病用医薬品市場の見通し、2018年～2032年

• インド
• 中国
• 日本
• オーストラリア
• ベトナム
• 韓国
• インドネシア
• フィリピン

第9章 南米の希少疾病用医薬品市場の見通し、2018年～2032年

• ブラジル
• アルゼンチン

第10章 中東・アフリカの希少疾病用医薬品市場の見通し、2018年～2032年

• サウジアラビア
• アラブ首長国連邦
• 南アフリカ

第11章 需要供給分析

第12章 バリューチェーン分析

第13章 ポーターのファイブフォース分析

第14章 PESTLE分析

第15章 価格分析

第16章 市場力学

• 市場の促進要因
• 市場の課題

第17章 市場動向と発展

第18章 規制枠組みとイノベーション

• 規制当局の承認
• 臨床試験

第19章 特許の情勢

第20章 ケーススタディ

第21章 競合情勢

• 市場リーダートップ5の競合マトリックス
• 参入企業トップ5のSWOT分析
• 市場の主要企業トップ10の情勢
  • Novartis AG
  • Amgen, Inc.
  • Bristol-Myers Squibb Company
  • AstraZeneca plc
  • Pfizer Inc.
  • Novo Nordisk A/S
  • Sanofi S.A.
  • Johnson & Johnson Services, Inc.
  • F. Hoffmann-La Roche Ltd
  • Vertex Pharmaceuticals Incorporated

第22章 戦略的提言

第23章 調査会社について・免責事項

List of Tables

• Table 1. Competition Matrix of Top 5 Market Leaders
• Table 2. Mergers & Acquisitions/ Joint Ventures (If Applicable)
• Table 3. About Us - Regions and Countries Where We Have Executed Client Projects

List of Figures

• Figure 1. Global Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 2. Global Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 3. Global Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 4. Global Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 5. Global Orphan Drugs Market Share (%), By Region, 2018-2032F
• Figure 6. North America Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 7. North America Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 8. North America Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 9. North America Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 10. North America Orphan Drugs Market Share (%), By Country, 2018-2032F
• Figure 11. United States Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 12. United States Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 13. United States Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 14. United States Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 15. Canada Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 16. Canada Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 17. Canada Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 18. Canada Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 19. Mexico Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 20. Mexico Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 21. Mexico Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 22. Mexico Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 23. Europe Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 24. Europe Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 25. Europe Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 26. Europe Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 27. Europe Orphan Drugs Market Share (%), By Country, 2018-2032F
• Figure 28. Germany Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 29. Germany Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 30. Germany Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 31. Germany Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 32. France Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 33. France Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 34. France Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 35. France Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 36. Italy Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 37. Italy Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 38. Italy Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 39. Italy Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 40. United Kingdom Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 41. United Kingdom Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 42. United Kingdom Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 43. United Kingdom Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 44. Russia Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 45. Russia Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 46. Russia Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 47. Russia Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 48. Netherlands Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 49. Netherlands Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 50. Netherlands Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 51. Netherlands Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 52. Spain Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 53. Spain Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 54. Spain Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 55. Spain Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 56. Turkey Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 57. Turkey Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 58. Turkey Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 59. Turkey Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 60. Poland Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 61. Poland Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 62. Poland Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 63. Poland Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 64. South America Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 65. South America Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 66. South America Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 67. South America Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 68. South America Orphan Drugs Market Share (%), By Country, 2018-2032F
• Figure 69. Brazil Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 70. Brazil Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 71. Brazil Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 72. Brazil Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 73. Argentina Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 74. Argentina Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 75. Argentina Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 76. Argentina Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 77. Asia-Pacific Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 78. Asia-Pacific Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 79. Asia-Pacific Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 80. Asia-Pacific Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 81. Asia-Pacific Orphan Drugs Market Share (%), By Country, 2018-2032F
• Figure 82. India Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 83. India Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 84. India Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 85. India Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 86. China Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 87. China Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 88. China Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 89. China Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 90. Japan Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 91. Japan Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 92. Japan Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 93. Japan Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 94. Australia Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 95. Australia Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 96. Australia Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 97. Australia Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 98. Vietnam Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 99. Vietnam Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 100. Vietnam Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 101. Vietnam Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 102. South Korea Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 103. South Korea Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 104. South Korea Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 105. South Korea Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 106. Indonesia Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 107. Indonesia Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 108. Indonesia Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 109. Indonesia Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 110. Philippines Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 111. Philippines Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 112. Philippines Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 113. Philippines Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 114. Middle East & Africa Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 115. Middle East & Africa Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 116. Middle East & Africa Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 117. Middle East & Africa Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 118. Middle East & Africa Orphan Drugs Market Share (%), By Country, 2018-2032F
• Figure 119. Saudi Arabia Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 120. Saudi Arabia Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 121. Saudi Arabia Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 122. Saudi Arabia Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 123. UAE Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 124. UAE Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 125. UAE Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 126. UAE Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 127. South Africa Orphan Drugs Market, By Value, In USD Billion, 2018-2032F
• Figure 128. South Africa Orphan Drugs Market Share (%), By Therapy Area, 2018-2032F
• Figure 129. South Africa Orphan Drugs Market Share (%), By Drug Type, 2018-2032F
• Figure 130. South Africa Orphan Drugs Market Share (%), By Distribution Channel, 2018-2032F
• Figure 131. By Therapy Area Map-Market Size (USD Billion) & Growth Rate (%), 2024
• Figure 132. By Drug Type Map-Market Size (USD Billion) & Growth Rate (%), 2024
• Figure 133. By Distribution Channel Map-Market Size (USD Billion) & Growth Rate (%), 2024
• Figure 134. By Region Map-Market Size (USD Billion) & Growth Rate (%), 2024

Global orphan drugs market is projected to witness a CAGR of 15.07% during the forecast period 2025-2032, growing from USD 208.32 billion in 2024 to USD 640.21 billion in 2032. The global orphan drugs market is being driven by a rising prevalence of rare diseases and increasing public and clinical awareness. Regulatory support and financial incentives are encouraging pharmaceutical companies to invest in specialized treatments. Additionally, targeted innovation in oncology and the dominance of North America due to strong infrastructure are shaping the market's upward trajectory.

For instance, in March 2024, Terns Pharmaceuticals announced that the FDA has granted Orphan Drug Designation to TERN-701 for the treatment of chronic myeloid leukemia. TERN-701, which is an allosteric BCR-ABL tyrosine kinase inhibitor, is currently undergoing Phase 1 clinical development.

Rising Prevalence of Rare Diseases and Growing Awareness Are Boosting Demand for Orphan Drugs

The growing global prevalence of rare diseases, which impact over 300 million individuals worldwide, has emerged as a key factor driving the orphan drugs market. Although these diseases are rare on an individual basis, they collectively present a considerable challenge to healthcare systems. The absence of effective treatments for many of these conditions has garnered the attention of healthcare providers and policymakers, resulting in increased awareness and earlier diagnoses. Additionally, advocacy groups for rare diseases and international health organizations have intensified their efforts to inform both the public and healthcare professionals. The rise in rare disease registries and genetic databases further facilitates the development of personalized orphan drugs. Furthermore, the demand for equitable access to healthcare has encouraged pharmaceutical companies to invest in treatments for smaller patient populations. For instance, in May 2023, Amgen announced that the U.S. FDA had granted Orphan Drug Designation (ODD) to tarlatamab, a bispecific T-cell engager being evaluated for small cell lung cancer, a rare and aggressive disease. This reflects how rare disease awareness and regulatory incentives stimulate innovation.

Favorable Regulatory Support and Incentives Are Accelerating Market Expansion

Governments and regulatory authorities have implemented policies and financial incentives to promote the development of orphan drugs, which has significantly influenced the growth of the market. These measures include tax credits for clinical trials, periods of market exclusivity, grant funding, and expedited approval processes. For example, the U.S. Orphan Drug Act and comparable regulations in the EU and Japan have motivated pharmaceutical companies to invest in treatments for rare diseases, even with small patient populations. Furthermore, streamlined regulatory processes reduce barriers for new therapies. These frameworks have been essential in mitigating the financial risks associated with research and development, thus fostering innovation. The rise in designations from key regulatory bodies illustrates the effectiveness of this system. As a recent example, the FDA has recently announced that it has awarded rare pediatric disease designation to AOC 1044, an investigational therapy developed by Avidity Biosciences for treating Duchenne muscular dystrophy (DMD) in patients with mutations suitable for exon 44 skipping (DMD44).

Oncology Segment Dominates the Market Due to High R&D Activity and Unmet Needs

Among therapy areas, oncology continues to hold the largest share in the orphan drugs market, owing to the high incidence of rare cancers and substantial investments in oncology R&D. Rare cancers often lack effective treatment options, making them prime candidates for orphan drug development. With pharmaceutical companies heavily focusing on targeted therapies and immunotherapies, the oncology segment has seen an influx of orphan drug approvals. Additionally, the complexity of rare cancers encourages the use of precision medicine and companion diagnostics, driving segment-specific innovation. The high clinical unmet need also supports premium pricing for oncology-related orphan drugs. For instance, in July 2023, Bristol Myers Squibb announced the U.S. FDA approval of Reblozyl for the treatment of anemia in adults with lower-risk myelodysplastic syndromes-a rare type of blood cancer-marking an important addition to orphan oncology drugs. This reflects how oncology continues to be the most lucrative and active therapeutic area in the orphan drug domain.

North America Maintains Market Leadership

North America, especially the United States, commands the largest share of the global orphan drugs market, propelled by robust regulatory frameworks, a sophisticated biopharmaceutical sector, and substantial healthcare investments. The region's prominence in rare disease research and development, coupled with extensive patient support systems and advocacy groups, has fueled market expansion. Additionally, the U.S. Food and Drug Administration (FDA) offers advantageous pathways, including Fast Track and Breakthrough Therapy Designations, encouraging pharmaceutical companies to create treatments for rare diseases. Key biotech centers throughout the U.S. continue to draw venture capital and collaborations in the orphan drug field. In December 2023, Vertex Pharmaceuticals, headquartered in Boston, received FDA approval for CASGEVY, the first CRISPR-based gene-editing therapy for sickle cell disease-a rare genetic disorder-demonstrating North America's innovation edge. This milestone reflects how North American policies and investments are positioning the region as a global leader in orphan drug development.

Future Market Scenario (2025-2032F)

The worldwide orphan drugs market is set for significant expansion, fueled by heightened awareness of rare diseases, progress in genetic and personalized medicine, and supportive regulatory environments that provide incentives such as tax credits and market exclusivity. Increased investments from leading pharmaceutical companies in specialized therapeutic fields, especially oncology and neurology, are further driving innovation. Moreover, the incorporation of artificial intelligence in drug discovery and the growth of newborn screening initiatives are facilitating earlier diagnosis and treatment. With patient advocacy groups and international health organizations advocating for broader access and affordability, the market is anticipated to broaden beyond affluent nations. Emerging markets and the development of biosimilars are also expected to be crucial in enhancing global access to orphan drugs in the years ahead.

Key Players Landscape and Outlook

The key players in the market are significantly investing in the development of orphan drugs and are utilizing strategies such as mergers, acquisitions, partnerships, and new product launches to improve their services and competitiveness. Such efforts will propel significant growth in the market, allowing large-cap industry players to increase their presence and, therefore, find new opportunities in this market.

For example, in September 2024, Agios Pharmaceuticals revealed that the FDA has awarded orphan drug designation to tebapivat (AG-946) for the treatment of myelodysplastic syndromes (MDS). This designation underscored the necessity for innovative oral therapies for anemia in patients with lower-risk MDS. Agios is focused on creating the first oral treatment that addresses ineffective erythropoiesis in MDS.

Table of Contents

1. Project Scope and Definitions

2. Research Methodology

3. Impact of U.S. Tariffs

4. Executive Summary

5. Global Orphan Drugs Market Outlook, 2018-2032F

• 5.1. Market Size Analysis & Forecast
  • 5.1.1. By Value
• 5.2. Market Share Analysis & Forecast
  • 5.2.1. By Therapy Area
    • 5.2.1.1. Oncology
    • 5.2.1.2. Hematology
    • 5.2.1.3. Neurology
    • 5.2.1.4. Endocrinology
    • 5.2.1.5. Cardiovascular
    • 5.2.1.6. Respiratory
    • 5.2.1.7. Immunotherapy
    • 5.2.1.8. Infectious Diseases
    • 5.2.1.9. Others
  • 5.2.2. By Drug Type
    • 5.2.2.1. Biologics
    • 5.2.2.2. Non-Biologics
  • 5.2.3. By Distribution Channel
    • 5.2.3.1. Hospital Pharmacies
    • 5.2.3.2. Retail Pharmacies and Drug Stores
    • 5.2.3.3. Online Pharmacies
  • 5.2.4. By Region
    • 5.2.4.1. North America
    • 5.2.4.2. Europe
    • 5.2.4.3. Asia-Pacific
    • 5.2.4.4. South America
    • 5.2.4.5. Middle East and Africa
  • 5.2.5. By Company Market Share Analysis (Top 5 Companies and Others - By Value, 2024)
• 5.3. Market Map Analysis, 2024
  • 5.3.1. By Therapy Area
  • 5.3.2. By Drug Type
  • 5.3.3. By Distribution Channel
  • 5.3.4. By Region

6. North America Orphan Drugs Market Outlook, 2018-2032F

• 6.1. Market Size Analysis & Forecast
  • 6.1.1. By Value
• 6.2. Market Share Analysis & Forecast
  • 6.2.1. By Therapy Area
    • 6.2.1.1. Oncology
    • 6.2.1.2. Hematology
    • 6.2.1.3. Neurology
    • 6.2.1.4. Endocrinology
    • 6.2.1.5. Cardiovascular
    • 6.2.1.6. Respiratory
    • 6.2.1.7. Immunotherapy
    • 6.2.1.8. Infectious Diseases
    • 6.2.1.9. Others
  • 6.2.2. By Drug Type
    • 6.2.2.1. Biologics
    • 6.2.2.2. Non-Biologics
  • 6.2.3. By Distribution Channel
    • 6.2.3.1. Hospital Pharmacies
    • 6.2.3.2. Retail Pharmacies and Drug Stores
    • 6.2.3.3. Online Pharmacies
  • 6.2.4. By Country Share
    • 6.2.4.1. United States
    • 6.2.4.2. Canada
    • 6.2.4.3. Mexico
• 6.3. Country Market Assessment
  • 6.3.1. United States Orphan Drugs Market Outlook, 2018-2032F*
    • 6.3.1.1. Market Size Analysis & Forecast
      • 6.3.1.1.1. By Value
    • 6.3.1.2. Market Share Analysis & Forecast
      • 6.3.1.2.1. By Therapy Area
        • 6.3.1.2.1.1. Oncology
        • 6.3.1.2.1.2. Hematology
        • 6.3.1.2.1.3. Neurology
        • 6.3.1.2.1.4. Endocrinology
        • 6.3.1.2.1.5. Cardiovascular
        • 6.3.1.2.1.6. Respiratory
        • 6.3.1.2.1.7. Immunotherapy
        • 6.3.1.2.1.8. Infectious Diseases
        • 6.3.1.2.1.9. Others
      • 6.3.1.2.2. By Drug Type
        • 6.3.1.2.2.1. Biologics
        • 6.3.1.2.2.2. Non-Biologics
      • 6.3.1.2.3. By Distribution Channel
        • 6.3.1.2.3.1. Hospital Pharmacies
        • 6.3.1.2.3.2. Retail Pharmacies and Drug Stores
        • 6.3.1.2.3.3. Online Pharmacies
  • 6.3.2. Canada
  • 6.3.3. Mexico

All segments will be provided for all regions and countries covered

7. Europe Orphan Drugs Market Outlook, 2018-2032F

• 7.1. Germany
• 7.2. France
• 7.3. Italy
• 7.4. United Kingdom
• 7.5. Russia
• 7.6. Netherlands
• 7.7. Spain
• 7.8. Turkey
• 7.9. Poland

8. Asia-Pacific Orphan Drugs Market Outlook, 2018-2032F

• 8.1. India
• 8.2. China
• 8.3. Japan
• 8.4. Australia
• 8.5. Vietnam
• 8.6. South Korea
• 8.7. Indonesia
• 8.8. Philippines

9. South America Orphan Drugs Market Outlook, 2018-2032F

• 9.1. Brazil
• 9.2. Argentina

10. Middle East and Africa Orphan Drugs Market Outlook, 2018-2032F

• 10.1. Saudi Arabia
• 10.2. UAE
• 10.3. South Africa

11. Demand Supply Analysis

12. Value Chain Analysis

13. Porter's Five Forces Analysis

14. PESTLE Analysis

15. Pricing Analysis

16. Market Dynamics

• 16.1. Market Drivers
• 16.2. Market Challenges

17. Market Trends and Developments

18. Regulatory Framework and Innovation

• 18.1. Regulatory Approvals
• 18.2. Clinical Trials

19. Patent Landscape

20. Case Studies

21. Competitive Landscape

• 21.1. Competition Matrix of Top 5 Market Leaders
• 21.2. SWOT Analysis for Top 5 Players
• 21.3. Key Players Landscape for Top 10 Market Players
  • 21.3.1. Novartis AG
    • 21.3.1.1. Company Details
    • 21.3.1.2. Key Management Personnel
    • 21.3.1.3. Products and Services
    • 21.3.1.4. Financials (As Reported)
    • 21.3.1.5. Key Market Focus and Geographical Presence
    • 21.3.1.6. Recent Developments/Collaborations/Partnerships/Mergers and Acquisition
  • 21.3.2. Amgen, Inc.
  • 21.3.3. Bristol-Myers Squibb Company
  • 21.3.4. AstraZeneca plc
  • 21.3.5. Pfizer Inc.
  • 21.3.6. Novo Nordisk A/S
  • 21.3.7. Sanofi S.A.
  • 21.3.8. Johnson & Johnson Services, Inc.
  • 21.3.9. F. Hoffmann-La Roche Ltd
  • 21.3.10. Vertex Pharmaceuticals Incorporated

Companies mentioned above DO NOT hold any order as per market share and can be changed as per information available during research work.

22. Strategic Recommendations

23. About Us and Disclaimer