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1576097

希少疾患治療市場：薬剤タイプ別、患者別、治療領域別、投与形態別、流通チャネル別、地域別、機会、予測、2017年～2031年

Rare Disease Treatment Market Assessment, By Drug Type, By Patient, By Therapeutic Area, Mode of Administration, By Distribution Channel, By Region, Opportunities and Forecast, 2017-2031F
出版日: | 発行: Market Xcel - Markets and Data | ページ情報: 英文 237 Pages | 納期: 3～5営業日

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価格
価格表記: USDを日本円(税抜)に換算
本日の銀行送金レート: 1USD=150.96円
希少疾患治療市場：薬剤タイプ別、患者別、治療領域別、投与形態別、流通チャネル別、地域別、機会、予測、2017年～2031年
出版日: 2024年10月24日
発行: Market Xcel - Markets and Data
ページ情報: 英文 237 Pages
納期: 3～5営業日
  • 全表示
  • 概要
  • 図表
  • 目次
概要

世界の希少疾患治療の市場規模は、2024年～2031年の予測期間中に10.10％のCAGRで拡大し、2023年の1,825億4,000万米ドルから2031年には3,941億5,000万米ドルに成長すると予測されています。同市場は、疾病負担の増大、診断法の改善、希少疾病用治療薬（オーファンドラッグ）の指定を受けて上市される新規治療薬によって、より速いペースで成長すると予測されています。希少疾患治療市場は、医学研究の進歩、認知度の向上、規制環境の整備など、さまざまな要因によって大きな成長を遂げています。

人口のごく一部が罹患する疾患は、一般的に20万人未満が罹患する疾患と定義され、希少疾患に分類され、そのような疾患の治療に用いられる薬剤は希少疾病用治療薬と呼ばれます。個別化医療、ゲノミクス、バイオテクノロジーの発展により、希少疾病を患う患者特有の遺伝子プロファイルに対応した治療法の発見や標的治療の機会が生まれつつあります。潜在的な医薬品候補に対する規制当局の承認は、希少疾患に対する治療の選択肢の幅を広げています。例えば、2024年3月、Cabaletta Bio, Inc.は、米国食品医薬品局（USFDA）がCABA-201に希少疾病用医薬品の指定（ODD）を与えたと発表しました。CABA-201は、全身性硬化症（SSc）の治療を目的とした、4-1BBを含む完全ヒトT細胞治験薬です。CABA-201は、B細胞によって駆動される自己免疫疾患の治療薬として開発されています。

当レポートでは、世界の希少疾患治療市場について調査し、市場の概要とともに、薬剤タイプ別、患者別、治療領域別、投与形態別、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 エグゼクティブサマリー

第4章 世界の希少疾患治療市場の見通し、2017年～2031年

  • 市場規模の分析と予測
  • 市場シェア分析と予測
  • 市場マップ分析、2023年
    • 薬剤タイプ別
    • 患者別
    • 治療領域別
    • 投与形態別
    • 流通チャネル別
    • 地域別

第5章 北米の希少疾患治療市場の見通し、2017年～2031年

第6章 欧州の希少疾患治療市場の見通し、2017年～2031年

第7章 アジア太平洋の希少疾患治療市場の見通し、2017年～2031年

第8章 南米の希少疾患治療市場の見通し、2017年～2031年

第9章 中東・アフリカの希少疾患治療市場の見通し、2017年～2031年

第10章 需要供給分析

第11章 バリューチェーン分析

第12章 ポーターのファイブフォース分析

第13章 PESTLE分析

第14章 価格分析

第15章 市場力学

第16章 市場の動向と発展

第17章 規制枠組みとイノベーション

第18章 特許の情勢

第19章 ケーススタディ

第20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
  • 市場参入企業トップ10の情勢
    • Amgen Inc.
    • Bayer AG
    • Novartis AG
    • F. Hoffmann-La Roche Ltd.
    • Pfizer, Inc.
    • AbbVie Inc.
    • Merck & Co., Inc.
    • Bristol-Myers Squibb Company
    • AstraZeneca PLC
    • PTC Therapeutics Inc.

第21章 戦略的提言

第22章 お問い合わせと免責事項

図表

List of Tables

  • Table 1. Pricing Analysis of Products from Key Players
  • Table 2. Competition Matrix of Top 5 Market Leaders
  • Table 3. Mergers & Acquisitions/ Joint Ventures (If Applicable)
  • Table 4. About Us - Regions and Countries Where We Have Executed Client Projects

List of Figures

  • Figure 1. Global Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 2. Global Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 3. Global Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 4. Global Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 5. Global Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 6. Global Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 7. Global Rare Disease Treatment Market Share (%), By Region, 2017-2031F
  • Figure 8. North America Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 9. North America Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 10. North America Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 11. North America Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 12. North America Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 13. North America Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 14. North America Rare Disease Treatment Market Share (%), By Country, 2017-2031F
  • Figure 15. United States Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 16. United States Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 17. United States Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 18. United States Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 19. United States Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 20. United States Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 21. Canada Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 22. Canada Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 23. Canada Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 24. Canada Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 25. Canada Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 26. Canada Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 27. Mexico Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 28. Mexico Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 29. Mexico Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 30. Mexico Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 31. Mexico Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 32. Mexico Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 33. Europe Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 34. Europe Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 35. Europe Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 36. Europe Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 37. Europe Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 38. Europe Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 39. Europe Rare Disease Treatment Market Share (%), By Country, 2017-2031F
  • Figure 40. Germany Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 41. Germany Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 42. Germany Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 43. Germany Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 44. Germany Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 45. Germany Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 46. France Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 47. France Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 48. France Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 49. France Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 50. France Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 51. France Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 52. Italy Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 53. Italy Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 54. Italy Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 55. Italy Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 56. Italy Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 57. Italy Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 58. United Kingdom Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 59. United Kingdom Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 60. United Kingdom Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 61. United Kingdom Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 62. United Kingdom Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 63. United Kingdom Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 64. Russia Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 65. Russia Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 66. Russia Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 67. Russia Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 68. Russia Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 69. Russia Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 70. Netherlands Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 71. Netherlands Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 72. Netherlands Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 73. Netherlands Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 74. Netherlands Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 75. Netherlands Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 76. Spain Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 77. Spain Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 78. Spain Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 79. Spain Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 80. Spain Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 81. Spain Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 82. Turkey Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 83. Turkey Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 84. Turkey Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 85. Turkey Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 86. Turkey Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 87. Turkey Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 88. Poland Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 89. Poland Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 90. Poland Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 91. Poland Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 92. Poland Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 93. Poland Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 94. South America Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 95. South America Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 96. South America Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 97. South America Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 98. South America Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 99. South America Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 100. South America Rare Disease Treatment Market Share (%), By Country, 2017-2031F
  • Figure 101. Brazil Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 102. Brazil Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 103. Brazil Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 104. Brazil Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 105. Brazil Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 106. Brazil Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 107. Argentina Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 108. Argentina Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 109. Argentina Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 110. Argentina Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 111. Argentina Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 112. Argentina Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 113. Asia-Pacific Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 114. Asia-Pacific Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 115. Asia-Pacific Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 116. Asia-Pacific Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 117. Asia-Pacific Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 118. Asia-Pacific Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 119. Asia-Pacific Rare Disease Treatment Market Share (%), By Country, 2017-2031F
  • Figure 120. India Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 121. India Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 122. India Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 123. India Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 124. India Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 125. India Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 126. China Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 127. China Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 128. China Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 129. China Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 130. China Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 131. China Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 132. Japan Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 133. Japan Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 134. Japan Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 135. Japan Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 136. Japan Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 137. Japan Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 138. Australia Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 139. Australia Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 140. Australia Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 141. Australia Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 142. Australia Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 143. Australia Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 144. Vietnam Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 145. Vietnam Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 146. Vietnam Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 147. Vietnam Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 148. Vietnam Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 149. Vietnam Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 150. South Korea Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 151. South Korea Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 152. South Korea Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 153. South Korea Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 154. South Korea Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 155. South Korea Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 156. Indonesia Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 157. Indonesia Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 158. Indonesia Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 159. Indonesia Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 160. Indonesia Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 161. Indonesia Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 162. Philippines Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 163. Philippines Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 164. Philippines Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 165. Philippines Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 166. Philippines Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 167. Philippines Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 168. Middle East & Africa Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 169. Middle East & Africa Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 170. Middle East & Africa Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 171. Middle East & Africa Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 172. Middle East & Africa Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 173. Middle East & Africa Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 174. Middle East & Africa Rare Disease Treatment Market Share (%), By Country, 2017-2031F
  • Figure 175. Saudi Arabia Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 176. Saudi Arabia Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 177. Saudi Arabia Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 178. Saudi Arabia Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 179. Saudi Arabia Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 180. Saudi Arabia Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 181. UAE Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 182. UAE Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 183. UAE Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 184. UAE Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 185. UAE Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 186. UAE Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 187. South Africa Rare Disease Treatment Market, By Value, In USD Billion, 2017-2031F
  • Figure 188. South Africa Rare Disease Treatment Market Share (%), By Drug Type, 2017-2031F
  • Figure 189. South Africa Rare Disease Treatment Market Share (%), By Patient, 2017-2031F
  • Figure 190. South Africa Rare Disease Treatment Market Share (%), By Therapeutic Area, 2017-2031F
  • Figure 191. South Africa Rare Disease Treatment Market Share (%), By Mode of Administration, 2017-2031F
  • Figure 192. South Africa Rare Disease Treatment Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 193. By Drug Type Map-Market Size (USD Billion) & Growth Rate (%), 2023
  • Figure 194. By Patient Map-Market Size (USD Billion) & Growth Rate (%), 2023
  • Figure 195. By Therapeutic Area Map-Market Size (USD Billion) & Growth Rate (%), 2023
  • Figure 196. By Mode of Administration Map-Market Size (USD Billion) & Growth Rate (%), 2023
  • Figure 197. By Distribution Channel Map-Market Size (USD Billion) & Growth Rate (%), 2023
  • Figure 198. By Region Map-Market Size (USD Billion) & Growth Rate (%), 2023
目次
Product Code: MX12144

Global rare disease treatment market is projected to witness a CAGR of 10.10% during the forecast period 2024-2031, growing from USD 182.54 billion in 2023 to USD 394.15 billion in 2031. The market is anticipated to grow at a faster pace owing to the growing disease burden, improved diagnostics, and novel therapies being launched under orphan drug designation. The rare disease treatment market is experiencing significant growth, driven by various factors, including advancements in medical research, increased awareness, and supportive regulatory environments.

The medical conditions affecting a very small portion of the population, typically defined as those that impact fewer than 200,000 individuals, are categorized as rare diseases, and the drugs used to treat such diseases are called orphan drugs. Developments in personalized medicine, genomics, and biotechnology are initiating opportunities for therapeutic discovery and targeted therapies catering to the unique genetic profiles of patients suffering from rare diseases. Regulatory approvals to potential drug candidates expand the plethora of treatment options for rare diseases. For instance, in March 2024, Cabaletta Bio, Inc. announced that the U.S. Food and Drug Administration (USFDA) granted an Orphan Drug Designation (ODD) to CABA-201. It is a fully human T cell investigational therapy containing 4-1BB, intended to treat systemic sclerosis (SSc). CABA-201 is being developed as a potential treatment for autoimmune diseases that are driven by B cells.

Rising Prevalence of Rare Diseases to Drive Market Growth

Increasing cases of rare diseases are being recognized as an important and emerging public health issue, with approximately 300 million affected people worldwide. The increase in diagnostics is due to improvements in diagnostic technologies and increased awareness of the healthcare professional workforce, which works with the increasing access to genetic testing. As diagnostic capabilities emerge, more cases are found whose condition had not been diagnosed prior, which tends to lead to an apparent rise in prevalence. Increased prevalence will automatically reflect an increased demand for orphan drugs, which are specifically developed to handle such sets of rare diseases. As per the data provided by the National Organization for Rare Disorders, Inc., there are more than 7000 known rare diseases around the globe, and around 25-30 million Americans live with different rare diseases. The number is anticipated to rise in the future due to enhanced diagnostics facilities, which enable more patients to be treated promptly.

Market Exclusivity of Orphan Drugs to Increase Market Growth

The market exclusivity of orphan drugs, protected by several patents, gives an extra advantage to market players as it influences the growth of the rare disease treatment market. This is because market exclusivity serves as one of the major incentives that encourage pharmaceutical companies to develop treatments for rare diseases since such diseases have smaller patient populations and correspondingly low revenues. Market exclusivity bars generic versions of the drug from entering the market during the exclusivity period. Therefore, companies can maintain higher prices. Exclusivity shapes the competitive landscape. With fewer competitors, orphan drugs should be able to support higher prices, which ought to translate into higher revenues for the manufacturers. For instance, in 2023, the USFDA gave 12 orphan approvals (new BLAs), including drugs such as Casgevy, Lyfgenia, Adzynma, Balfaxar, Cyfendus, and others. As per the Orphan Drug Act, these drugs get a seven-year market exclusivity period after they receive orphan designation.

Oncology Segment to Dominate the Rare Disease Treatment Market Share

Oncology is the most common therapy area in rare disease treatment for several reasons, primarily driven by the unique characteristics of rare cancers, historical legislative support, and the evolving landscape of drug development. Approximately 20% of all cancers are classified as rare, with hundreds of distinct types existing. The prevalence has led to a concentrated effort in developing therapies specifically for these conditions, as they often share similar challenges with other rare diseases, including limited treatment options and a lack of specialized knowledge among healthcare providers. For instance, in August 2024, Adaptimmune Therapeutics plc announced that TECELRA (afamitresgene autoleucel) received accelerated approval from the U.S. Food and Drug Administration. It is intended for the treatment of adults with unresectable or metastatic synovial sarcoma who have undergone previous chemotherapy. TECELRA marks the first approved engineered cell therapy for a solid tumor cancer to be approved in the United States. It represents the first new therapy option for synovial sarcoma in over a decade.

North America to Dominate Rare Disease Treatment Market Share

North America is anticipated to dominate the rare disease treatment market owing to several factors, such as a comparatively high diagnosis rate of rare diseases in the region and robust healthcare infrastructure. The region has a significant number of individuals affected by rare diseases, with around 30 million Americans and 1 in 12 Canadians impacted annually. This high prevalence drives demand for effective treatments. There are substantial government incentives aimed at promoting research and development in rare disease treatments. Initiatives, such as the Orphan Drug Act, provide financial benefits to companies developing therapies for rare conditions, fostering innovation. Additionally, the rise of precision medicine, which tailors treatments to individual genetic profiles, has been particularly impactful in North America. Regulatory approvals from the USFDA further enhance market growth. For instance, USFDA approved four orphan drugs to treat different rare diseases from January 2024 to August 2024.

Future Market Scenario (2024 - 2031F)

Biologics are revolutionizing the treatment landscape for rare diseases, offering innovative therapies that target specific genetic and molecular mechanisms. These biologics include monoclonal antibodies, gene therapies, and enzyme replacement therapies, which emerged as critical options for conditions previously deemed untreatable. Developing biosimilars-biologics that are highly similar to already approved products has further enhanced accessibility, allowing more patients to benefit from these advanced treatments. Regulatory bodies such as the FDA and EMA have established pathways to facilitate the approval of these therapies, ensuring that safety and efficacy are maintained while promoting competition in the market. For instance, in March 2024, Merck & Co., Inc. received FDA approval for WINREVAIR (sotatercept-csrk), a first-in-class treatment for adults with pulmonary arterial hypertension (PAH), a rare, progressive, and ultimately life-threatening disease. This biologic therapy aims to enhance exercise capacity and reduce the risk of clinical worsening in patients. WINREVAIR is a biologic administered subcutaneously every three weeks and represents a novel approach targeting activin signaling in PAH treatment, expanding options for patients suffering from life-threatening conditions.

Key Players Landscape and Outlook

The rare disease treatment market landscape is dynamic, with several notable players such as Novartis and Merck. The market has witnessed many business activities, such as strategic mergers, acquisitions, and collaborations in recent times. In March 2024, Merck & Co., Inc. completed its acquisition of Harpoon Therapeutics, Inc., enhancing its oncology pipeline with innovative treatments. The key focus is on MK-6070, a T-cell engager targeting delta-like ligand 3 (DLL3), which has received an Orphan Drug Designation from the FDA for small cell lung cancer (SCLC), a rare disease. This designation aims to facilitate the development of therapies for rare diseases. The acquisition includes other promising candidates, such as HPN217 for multiple myeloma, reinforcing Merck's commitment to advancing cancer treatment options.

In January 2024, Sanofi S.A. signed an agreement to acquire Inhibrx, Inc., a clinical-stage biopharmaceutical company, for approximately USD 1.7 billion. The acquisition adds INBRX-101, a potential best-in-class rare disease medication for Alpha-1 Antitrypsin Deficiency (AATD), to Sanofi's pipeline. INBRX-101 has completed a Phase 1 trial and is enrolling in a Phase 2 trial. It could significantly improve treatment options and quality of life for AATD patients.

Table of Contents

1. Project Scope and Definitions

2. Research Methodology

3. Executive Summary

4. Global Rare Disease Treatment Market Outlook, 2017-2031F

  • 4.1. Market Size Analysis & Forecast
    • 4.1.1. By Value
  • 4.2. Market Share Analysis & Forecast
    • 4.2.1. By Drug Type
      • 4.2.1.1. Biologics
      • 4.2.1.2. Biosimilars
      • 4.2.1.3. Small Molecules
    • 4.2.2. By Patient
      • 4.2.2.1. Adult
      • 4.2.2.2. Pediatric
      • 4.2.2.3. Geriatric
    • 4.2.3. By Therapeutic Area
      • 4.2.3.1. Oncology
      • 4.2.3.2. Hematology
      • 4.2.3.3. Neurology
      • 4.2.3.4. Endocrinology
      • 4.2.3.5. Cardiovascular
      • 4.2.3.6. Respiratory
      • 4.2.3.7. Immunotherapy
      • 4.2.3.8. Infectious Diseases
      • 4.2.3.9. Others
    • 4.2.4. Mode of Administration
      • 4.2.4.1. Oral
      • 4.2.4.2. Injectable
      • 4.2.4.3. Others
    • 4.2.5. By Distribution Channel
      • 4.2.5.1. Hospital Pharmacy
      • 4.2.5.2. Retail Pharmacy
      • 4.2.5.3. Online Pharmacy
    • 4.2.6. By Region
      • 4.2.6.1. North America
      • 4.2.6.2. Europe
      • 4.2.6.3. Asia-Pacific
      • 4.2.6.4. South America
      • 4.2.6.5. Middle East and Africa
    • 4.2.7. By Company Market Share Analysis (Top 5 Companies and Others - By Value, 2023)
  • 4.3. Market Map Analysis, 2023
    • 4.3.1. By Drug Type
    • 4.3.2. By Patient
    • 4.3.3. By Therapeutic Area
    • 4.3.4. By Mode of Administration
    • 4.3.5. By Distribution Channel
    • 4.3.6. By Region

5. North America Rare Disease Treatment Market Outlook, 2017-2031F*

  • 5.1. Market Size Analysis & Forecast
    • 5.1.1. By Value
  • 5.2. Market Share Analysis & Forecast
    • 5.2.1. By Drug Type
      • 5.2.1.1. Biologics
      • 5.2.1.2. Biosimilars
      • 5.2.1.3. Small Molecules
    • 5.2.2. By Patient
      • 5.2.2.1. Adult
      • 5.2.2.2. Pediatric
      • 5.2.2.3. Geriatric
    • 5.2.3. By Therapeutic Area
      • 5.2.3.1. Oncology
      • 5.2.3.2. Hematology
      • 5.2.3.3. Neurology
      • 5.2.3.4. Endocrinology
      • 5.2.3.5. Cardiovascular
      • 5.2.3.6. Respiratory
      • 5.2.3.7. Immunotherapy
      • 5.2.3.8. Infectious Diseases
      • 5.2.3.9. Others
    • 5.2.4. Mode of Administration
      • 5.2.4.1. Oral
      • 5.2.4.2. Injectable
      • 5.2.4.3. Others
    • 5.2.5. By Distribution Channel
      • 5.2.5.1. Hospital Pharmacy
      • 5.2.5.2. Retail Pharmacy
      • 5.2.5.3. Online Pharmacy
    • 5.2.6. By Country Share
      • 5.2.6.1. United States
      • 5.2.6.2. Canada
      • 5.2.6.3. Mexico
  • 5.3. Country Market Assessment
    • 5.3.1. United States Rare Disease Treatment Market Outlook, 2017-2031F*
      • 5.3.1.1. Market Size Analysis & Forecast
        • 5.3.1.1.1. By Value
      • 5.3.1.2. Market Share Analysis & Forecast
        • 5.3.1.2.1. By Drug Type
          • 5.3.1.2.1.1. Biologics
          • 5.3.1.2.1.2. Biosimilars
          • 5.3.1.2.1.3. Small Molecules
        • 5.3.1.2.2. By Patient
          • 5.3.1.2.2.1. Adult
          • 5.3.1.2.2.2. Pediatric
          • 5.3.1.2.2.3. Geriatric
        • 5.3.1.2.3. By Therapeutic Area
          • 5.3.1.2.3.1. Oncology
          • 5.3.1.2.3.2. Hematology
          • 5.3.1.2.3.3. Neurology
          • 5.3.1.2.3.4. Endocrinology
          • 5.3.1.2.3.5. Cardiovascular
          • 5.3.1.2.3.6. Respiratory
          • 5.3.1.2.3.7. Immunotherapy
          • 5.3.1.2.3.8. Infectious Diseases
          • 5.3.1.2.3.9. Others
        • 5.3.1.2.4. Mode of Administration
          • 5.3.1.2.4.1. Oral
          • 5.3.1.2.4.2. Injectable
          • 5.3.1.2.4.3. Others
        • 5.3.1.2.5. By Distribution Channel
          • 5.3.1.2.5.1. Hospital Pharmacy
          • 5.3.1.2.5.2. Retail Pharmacy
          • 5.3.1.2.5.3. Online Pharmacy
    • 5.3.2. Canada
    • 5.3.3. Mexico

All segments will be provided for all regions and countries covered

6. Europe Rare Disease Treatment Market Outlook, 2017-2031F

  • 6.1. Germany
  • 6.2. France
  • 6.3. Italy
  • 6.4. United Kingdom
  • 6.5. Russia
  • 6.6. Netherlands
  • 6.7. Spain
  • 6.8. Poland

7. Asia-Pacific Rare Disease Treatment Market Outlook, 2017-2031F

  • 7.1. India
  • 7.2. China
  • 7.3. Japan
  • 7.4. Australia
  • 7.5. Vietnam
  • 7.6. South Korea
  • 7.7. Indonesia
  • 7.8. Philippines

8. South America Rare Disease Treatment Market Outlook, 2017-2031F

  • 8.1. Brazil
  • 8.2. Argentina

9. Middle East and Africa Rare Disease Treatment Market Outlook, 2017-2031F

  • 9.1. Saudi Arabia
  • 9.2. UAE
  • 9.3. South Africa
  • 9.4. Israel

10. Demand Supply Analysis

11. Value Chain Analysis

12. Porter's Five Forces Analysis

13. PESTLE Analysis

14. Pricing Analysis

15. Market Dynamics

  • 15.1. Market Drivers
  • 15.2. Market Challenges

16. Market Trends and Developments

17. Regulatory Framework and Innovation

  • 17.1. Clinical Trials
  • 17.2. Regulatory Approvals

18. Patent Landscape

19. Case Studies

20. Competitive Landscape

  • 20.1. Competition Matrix of Top 5 Market Leaders
  • 20.2. SWOT Analysis for Top 5 Players
  • 20.3. Key Players Landscape for Top 10 Market Players
    • 20.3.1. Amgen Inc.
      • 20.3.1.1. Company Details
      • 20.3.1.2. Key Management Personnel
      • 20.3.1.3. Products and Services
      • 20.3.1.4. Financials (As Reported)
      • 20.3.1.5. Key Market Focus and Geographical Presence
      • 20.3.1.6. Recent Developments/Collaborations/Partnerships/Mergers and Acquisition
    • 20.3.2. Bayer AG
    • 20.3.3. Novartis AG
    • 20.3.4. F. Hoffmann-La Roche Ltd.
    • 20.3.5. Pfizer, Inc.
    • 20.3.6. AbbVie Inc.
    • 20.3.7. Merck & Co., Inc.
    • 20.3.8. Bristol-Myers Squibb Company
    • 20.3.9. AstraZeneca PLC
    • 20.3.10. PTC Therapeutics Inc.

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

21. Strategic Recommendations

22. About Us and Disclaimer