世界の希少疾患治療市場は、2021年に約1,196億米ドルと評価され、予測期間の2022年から2029年には12.8%以上の健全な成長率で成長すると予測されます。
希少疾患とは、ごく一部の人にしか発症しない疾病のことです。世界保健機関によってICD-11としてコード化されており、ICD-11は約5500の希少疾患から構成されています。希少疾患は、治療法開発のための資金や資源を集めるのに十分な市場規模がなく、そのような治療法の開発や商業化に対して政府が有利な経済条件を与えることを禁じている状態であるとも言えます。米国食品医薬品局（FDA）によると、希少疾患とは、国内で20万人未満しか罹患していない疾病のことを指します。新しい希少疾患用医薬品の承認・発売の増加、希少疾患の有病率の増加、希少疾患の治療に対する政府の支援や取り組み、この分野における研究開発の増加、政府の有利な政策などが、市場の成長に寄与する要因となっています。
米国国立衛生研究所によると、アメリカ人の10人に1人（約3,000万人）が希少疾患を抱えており、アメリカでは約7,000種類の希少疾患が確認されています。さらに、2022年1月、Amgen社は日本での希少がん治療薬 LUMAKRAS® の承認を発表し、2021年10月には、Scemblix薬が慢性期（CP）の慢性骨髄性白血病（CML）患者を対象に3次治療として食品医薬品局から承認されました。新規希少疾患用医薬品の承認・上市の増加、希少疾患の有病率の増加が、市場成長を促進する主な要因となっています。さらに、政府によるイニシアチブの増加や希少疾患に対する意識の高まりは、予測期間中に大きな市場機会を創出する要因となっています。しかし、希少疾患治療の高額な費用が、2022年から2029年の予測期間を通じて市場の成長を阻害しています。
希少疾患治療の世界市場の調査対象地域は、アジア太平洋地域、北アメリカ、ヨーロッパ、ラテンアメリカ、その他の地域です。北米は、希少疾患の優位性、製品へのアクセスの容易さ、新製品の承認、政府の支援と投資の増加により、売上高で市場を支配しています。 アジア太平洋地域は、政府のイニシアチブの高まり、希少疾患の増加、主要企業の地理的拡大、政府の積極的な参加などの要因により、予測期間中に最も高いCAGRで成長すると予想されます。
本調査の目的は、近年における様々なセグメント・国の市場規模を定義し、今後数年間の価値を予測することです。本レポートは、調査対象国における産業の質的・量的な側面を取り込むように設計されています。
さらに、市場の今後の成長を決定づける推進要因や課題などの重要な側面に関する詳細情報も提供しています。さらに、主要企業の競争環境と製品提供の詳細な分析とともに、利害関係者が投資するためのミクロ市場の潜在的な機会も組み込んでいます。
目次
Table of Contents
Chapter 1. Executive Summary
1.1. Market Snapshot
1.2. Global & Segmental Market Estimates & Forecasts, 2019-2029 (USD Billion)
1.2.1. Rare Diseases Treatment Market, by Region, 2019-2029 (USD Billion)
1.2.2. Rare Diseases Treatment Market, by Therapeutic Area, 2019-2029 (USD Billion)
1.2.3. Rare Diseases Treatment Market, by Drug Type, 2019-2029 (USD Billion)
1.2.4. Rare Diseases Treatment Market, by Route of Administration, 2019-2029 (USD Billion)
1.2.5. Rare Diseases Treatment Market, by Distribution Channel, 2019-2029 (USD Billion)
1.3. Key Trends
1.4. Estimation Methodology
1.5. Research Assumption
Chapter 2. Global Rare Diseases Treatment Market Definition and Scope
2.1. Objective of the Study
2.2. Market Definition & Scope
2.2.1. Scope of the Study
2.2.2. Industry Evolution
2.3. Years Considered for the Study
2.4. Currency Conversion Rates
Chapter 3. Global Rare Diseases Treatment Market Dynamics
3.1. Rare Diseases Treatment Market Impact Analysis (2019-2029)
3.1.1. Market Drivers
3.1.1.1. Increasing approval and launch of new orphan drugs
3.1.1.2. Increasing prevalence of rare diseases
3.1.2. Market Challenges
3.1.2.1. High Cost of Rare Diseases Treatment
3.1.3. Market Opportunities
3.1.3.1. Increasing initiatives by the government
3.1.3.2. Increasing awareness of rare diseases
Chapter 4. Global Rare Diseases Treatment Market Industry Analysis
4.1. Porter’s 5 Force Model
4.1.1. Bargaining Power of Suppliers
4.1.2. Bargaining Power of Buyers
4.1.3. Threat of New Entrants
4.1.4. Threat of Substitutes
4.1.5. Competitive Rivalry
4.2. Futuristic Approach to Porter’s 5 Force Model (2019-2029)
4.3. PEST Analysis
4.3.1. Political
4.3.2. Economical
4.3.3. Social
4.3.4. Technological
4.4. Investment Adoption Model
4.5. Analyst Recommendation & Conclusion
4.6. Top investment opportunity
4.7. Top winning strategies
Chapter 5. Risk Assessment: COVID-19 Impact
5.1. Assessment of the overall impact of COVID-19 on the industry
5.2. Pre COVID-19 and post COVID-19 Market scenario
Chapter 6. Global Rare Diseases Treatment Market, by Therapeutic Area
6.1. Market Snapshot
6.2. Global Rare Diseases Treatment Market by Therapeutic Area, Performance - Potential Analysis
6.3. Global Rare Diseases Treatment Market Estimates & Forecasts by Therapeutic Area 2019-2029 (USD Billion)
6.4. Rare Diseases Treatment Market, Sub Segment Analysis
6.4.1. Cancer
6.4.2. Neurological Conditions
6.4.3. Cardiovascular Conditions
6.4.4. Musculoskeletal Conditions
6.4.5. Hematologic Disorders
6.4.6. Infectious Diseases
6.4.7. Metabolic disorders
6.4.8. Endocrine disorders
6.4.9. Others
Chapter 7. Global Rare Diseases Treatment Market, by Drug Type
7.1. Market Snapshot
7.2. Global Rare Diseases Treatment Market by Drug Type, Performance - Potential Analysis
7.3. Global Rare Diseases Treatment Market Estimates & Forecasts by Drug Type 2019-2029 (USD Billion)
7.4. Rare Diseases Treatment Market, Sub Segment Analysis
7.4.1. Biologics
7.4.2. Biosimilar
7.4.3. Small Molecule
Chapter 8. Global Rare Diseases Treatment Market, by Route of Administration
8.1. Market Snapshot
8.2. Global Rare Diseases Treatment Market by Route of Administration, Performance - Potential Analysis
8.3. Global Rare Diseases Treatment Market Estimates & Forecasts by Route of Administration 2019-2029 (USD Billion)
8.4. Rare Diseases Treatment Market, Sub Segment Analysis
8.4.1. Oral
8.4.2. Injectable
8.4.3. Others
Chapter 9. Global Rare Diseases Treatment Market, by Distribution Channel
9.1. Market Snapshot
9.2. Global Rare Diseases Treatment Market by Distribution Channel, Performance - Potential Analysis
9.3. Global Rare Diseases Treatment Market Estimates & Forecasts by Distribution Channel 2019-2029 (USD Billion)
9.4. Rare Diseases Treatment Market, Sub Segment Analysis
9.4.1. Hospital Pharmacy
9.4.2. Specialty Pharmacy
9.4.3. Online Pharmacy
Chapter 10. Global Rare Diseases Treatment Market, Regional Analysis
10.1. Rare Diseases Treatment Market, Regional Market Snapshot
10.2. North America Rare Diseases Treatment Market
10.2.1. U.S. Rare Diseases Treatment Market
10.2.1.1. Therapeutic Area breakdown estimates & forecasts, 2019-2029
10.2.1.2. Drug Type breakdown estimates & forecasts, 2019-2029
10.2.1.3. Route of Administration breakdown estimates & forecasts, 2019-2029
10.2.1.4. Distribution Channel breakdown estimates & forecasts, 2019-2029
10.2.2. Canada Rare Diseases Treatment Market
10.3. Europe Rare Diseases Treatment Market Snapshot
10.3.1. U.K. Rare Diseases Treatment Market
10.3.2. Germany Rare Diseases Treatment Market
10.3.3. France Rare Diseases Treatment Market
10.3.4. Spain Rare Diseases Treatment Market
10.3.5. Italy Rare Diseases Treatment Market
10.3.6. Rest of Europe Rare Diseases Treatment Market
10.4. Asia-Pacific Rare Diseases Treatment Market Snapshot
10.4.1. China Rare Diseases Treatment Market
10.4.2. India Rare Diseases Treatment Market
10.4.3. Japan Rare Diseases Treatment Market
10.4.4. Australia Rare Diseases Treatment Market
10.4.5. South Korea Rare Diseases Treatment Market
10.4.6. Rest of Asia Pacific Rare Diseases Treatment Market
10.5. Latin America Rare Diseases Treatment Market Snapshot
10.5.1. Brazil Rare Diseases Treatment Market
10.5.2. Mexico Rare Diseases Treatment Market
10.6. Rest of The World Rare Diseases Treatment Market
Chapter 11. Competitive Intelligence
11.1. Top Market Strategies
11.2. Company Profiles
11.2.1. F. Hoffmann-La Roche Ltd.
11.2.1.1. Key Information
11.2.1.2. Overview
11.2.1.3. Financial (Subject to Data Availability)
11.2.1.4. Product Summary
11.2.1.5. Recent Developments
11.2.2. Pfizer, Inc
11.2.3. PTC Therapeutics
11.2.4. AstraZeneca
11.2.5. Novartis AG
11.2.6. Novo Nordisk Pharmatech A/S
11.2.7. Takeda Pharmaceutical Company Limited
11.2.8. Bayer AG
11.2.9. Merck & Co., Inc.
11.2.10. Bristol Myers Squibb Company
Chapter 12. Research Process
12.1. Research Process
12.1.1. Data Mining
12.1.2. Analysis
12.1.3. Market Estimation
12.1.4. Validation
12.1.5. Publishing
12.2. Research Attributes
12.3. Research Assumption
List of Tables
TABLE 1. Global Rare Diseases Treatment Market, report scope
TABLE 2. Global Rare Diseases Treatment Market estimates & forecasts by Region 2019-2029 (USD Billion)
TABLE 3. Global Rare Diseases Treatment Market estimates & forecasts by Therapeutic Area 2019-2029 (USD Billion)
TABLE 4. Global Rare Diseases Treatment Market estimates & forecasts by Drug Type 2019-2029 (USD Billion)
TABLE 5. Global Rare Diseases Treatment Market estimates & forecasts by Route of Administration 2019-2029 (USD Billion)
TABLE 6. Global Rare Diseases Treatment Market estimates & forecasts by Distribution Channel 2019-2029 (USD Billion)
TABLE 7. Global Rare Diseases Treatment Market by segment, estimates & forecasts, 2019-2029 (USD Billion)
TABLE 8. Global Rare Diseases Treatment Market by region, estimates & forecasts, 2019-2029 (USD Billion)
TABLE 9. Global Rare Diseases Treatment Market by segment, estimates & forecasts, 2019-2029 (USD Billion)
TABLE 10. Global Rare Diseases Treatment Market by region, estimates & forecasts, 2019-2029 (USD Billion)
TABLE 11. Global Rare Diseases Treatment Market by segment, estimates & forecasts, 2019-2029 (USD Billion)
TABLE 12. Global Rare Diseases Treatment Market by region, estimates & forecasts, 2019-2029 (USD Billion)
TABLE 13. Global Rare Diseases Treatment Market by segment, estimates & forecasts, 2019-2029 (USD Billion)
TABLE 14. Global Rare Diseases Treatment Market by region, estimates & forecasts, 2019-2029 (USD Billion)
TABLE 15. Global Rare Diseases Treatment Market by segment, estimates & forecasts, 2019-2029 (USD Billion)
TABLE 16. Global Rare Diseases Treatment Market by region, estimates & forecasts, 2019-2029 (USD Billion)
TABLE 17. U.S. Rare Diseases Treatment Market estimates & forecasts, 2019-2029 (USD Billion)
TABLE 18. U.S. Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 19. U.S. Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 20. Canada Rare Diseases Treatment Market estimates & forecasts, 2019-2029 (USD Billion)
TABLE 21. Canada Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 22. Canada Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 23. UK Rare Diseases Treatment Market estimates & forecasts, 2019-2029 (USD Billion)
TABLE 24. UK Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 25. UK Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 26. Germany Rare Diseases Treatment Market estimates & forecasts, 2019-2029 (USD Billion)
TABLE 27. Germany Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 28. Germany Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 29. France Rare Diseases Treatment Market estimates & forecasts, 2019-2029 (USD Billion)
TABLE 30. France Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 31. France Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 32. Italy Rare Diseases Treatment Market estimates & forecasts, 2019-2029 (USD Billion)
TABLE 33. Italy Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 34. Italy Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 35. Spain Rare Diseases Treatment Market estimates & forecasts, 2019-2029 (USD Billion)
TABLE 36. Spain Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 37. Spain Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 38. RoE Rare Diseases Treatment Market estimates & forecasts, 2019-2029 (USD Billion)
TABLE 39. RoE Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 40. RoE Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 41. China Rare Diseases Treatment Market estimates & forecasts, 2019-2029 (USD Billion)
TABLE 42. China Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 43. China Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 44. India Rare Diseases Treatment Market estimates & forecasts, 2019-2029 (USD Billion)
TABLE 45. India Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 46. India Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 47. Japan Rare Diseases Treatment Market estimates & forecasts, 2019-2029 (USD Billion)
TABLE 48. Japan Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 49. Japan Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 50. South Korea Rare Diseases Treatment Market estimates & forecasts, 2019-2029 (USD Billion)
TABLE 51. South Korea Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 52. South Korea Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 53. Australia Rare Diseases Treatment Market estimates & forecasts, 2019-2029 (USD Billion)
TABLE 54. Australia Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 55. Australia Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 56. RoAPAC Rare Diseases Treatment Market estimates & forecasts, 2019-2029 (USD Billion)
TABLE 57. RoAPAC Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 58. RoAPAC Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 59. Brazil Rare Diseases Treatment Market estimates & forecasts, 2019-2029 (USD Billion)
TABLE 60. Brazil Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 61. Brazil Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 62. Mexico Rare Diseases Treatment Market estimates & forecasts, 2019-2029 (USD Billion)
TABLE 63. Mexico Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 64. Mexico Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 65. RoLA Rare Diseases Treatment Market estimates & forecasts, 2019-2029 (USD Billion)
TABLE 66. RoLA Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 67. RoLA Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 68. Row Rare Diseases Treatment Market estimates & forecasts, 2019-2029 (USD Billion)
TABLE 69. Row Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 70. Row Rare Diseases Treatment Market estimates & forecasts by segment 2019-2029 (USD Billion)
TABLE 71. List of secondary sources, used in the study of global Rare Diseases Treatment Market
TABLE 72. List of primary sources, used in the study of global Rare Diseases Treatment Market
TABLE 73. Years considered for the study
TABLE 74. Exchange rates considered
List of tables and figures and dummy in nature, final lists may vary in the final deliverable
List of figures
FIG 1. Global Rare Diseases Treatment Market, research methodology
FIG 2. Global Rare Diseases Treatment Market, Market estimation techniques
FIG 3. Global Market size estimates & forecast methods
FIG 4. Global Rare Diseases Treatment Market, key trends 2021
FIG 5. Global Rare Diseases Treatment Market, growth prospects 2022-2029
FIG 6. Global Rare Diseases Treatment Market, porters 5 force model
FIG 7. Global Rare Diseases Treatment Market, pest analysis
FIG 8. Global Rare Diseases Treatment Market, value chain analysis
FIG 9. Global Rare Diseases Treatment Market by segment, 2019 & 2029 (USD Billion)
FIG 10. Global Rare Diseases Treatment Market by segment, 2019 & 2029 (USD Billion)
FIG 11. Global Rare Diseases Treatment Market by segment, 2019 & 2029 (USD Billion)
FIG 12. Global Rare Diseases Treatment Market by segment, 2019 & 2029 (USD Billion)
FIG 13. Global Rare Diseases Treatment Market by segment, 2019 & 2029 (USD Billion)
FIG 14. Global Rare Diseases Treatment Market, regional snapshot 2019 & 2029
FIG 15. North America Rare Diseases Treatment Market 2019 & 2029 (USD Billion)
FIG 16. Europe Rare Diseases Treatment Market 2019 & 2029 (USD Billion)
FIG 17. Asia pacific Rare Diseases Treatment Market 2019 & 2029 (USD Billion)
FIG 18. Latin America Rare Diseases Treatment Market 2019 & 2029 (USD Billion)
FIG 19. Global Rare Diseases Treatment Market, company Market share analysis (2021)
List of tables and figures and dummy in nature, final lists may vary in the final deliverable
