Global Rare Diseases Treatment Market Size study & Forecast, by Therapeutic Area by Route of Administration, by Drug Type, by Distribution Channel and Regional Analysis, 2022-2029

Description

List of Tables

Global Rare Diseases Treatment Market is valued at approximately USD 119.6 billion in 2021 and is anticipated to grow with a healthy growth rate of more than 12.8% over the forecast period 2022-2029. Rare Diseases are diseases that affect a small percentage of people. It is coded as ICD-11 by the world health organization, ICD-11 consists of around 5500 rare diseases. Rare disease can also be termed as an orphan disease, a condition that lacks a market size large enough to attract funding and resources for the development of remedies, barring the government from giving favorable economic terms for developing and commercializing such treatments. According to the US Food and Drug Administration, a rare disease is one that affects less than two lakh persons in the country. Increasing approval and launch of new orphan drugs, increasing prevalence of rare diseases, government support and initiatives for the treatment of rare diseases, increasing research and development in the sector, and favorable government policies are the factors contributing to the growth of the market.

According to the National Institutes of Health, One in ten Americans (about 30 million people) have a rare condition, and there are approximately 7,000 identified rare diseases in the US. Additionally, In January 2022, Amgen announced the approval of LUMAKRAS® drug in Japan for the treatment of rare cancer, and in October 2021, Scemblix drug was approved by the Food and Drug Administration for patients with chronic-phase (CP)-CML as a third line of treatment. Increasing approval and launch of new orphan drugs and increasing prevalence of rare diseases are the major factors driving the growth of the market. Furthermore, Increasing initiatives by the government and increasing awareness of rare diseases are the factors creating major market opportunities in the forecast period. However, the high cost of Rare Diseases Treatment stifles market growth throughout the forecast period of 2022-2029.

The key regions considered for the Global Rare Diseases Treatment Market study includes Asia Pacific, North America, Europe, Latin America, and Rest of the World. North America dominated the market in terms of revenue, owing to the dominance of rare diseases, easy accessibility to products, new product approval, and increasing government support and investment in the region. Asia Pacific is expected to grow with the highest CAGR during the forecast period, owing to factors such as rising government initiatives, increasing prevalence of orphan diseases, geographic expansion of key players, and active participation of government.

Major market players included in this report are:

F. Hoffmann-La Roche Ltd.
Pfizer, Inc
PTC Therapeutics
AstraZeneca
Novartis AG
Novo Nordisk Pharmatech A/S
Takeda Pharmaceutical Company Limited
Bayer AG
Merck & Co., Inc.
Bristol Myers Squibb Company

Recent Developments in the Market:

In July 2021, AstraZeneca acquired Alexion Pharmaceuticals, Inc. to enjoy a stronger scientific foothold in immunology and to continue to lead the way in the research and development of drugs for people with rare diseases through Alexion's cutting-edge complement-biology platform and a strong pipeline.
In November 2022, The FDA received a new Biologics License Application (BLA) for PRX-102 (pegunigalsidasealfa) from ProtalixBiotherapeutics Inc. and Chiesi Global Rare Diseases for the treatment of adult Fabry disease patients.

Global Rare Diseases Treatment Market Report Scope:

Historical Data 2019-2020-2021

Base Year for Estimation 2021

Forecast period 2022-2029

Report Coverage Revenue forecast, Company Ranking, Competitive Landscape, Growth factors, and Trends

Segments Covered Therapeutic Area, Route of Administration, Drug Type, Distribution Channel, Region

Regional Scope North America; Europe; Asia Pacific; Latin America; Rest of the World

Customization Scope Free report customization (equivalent up to 8 analyst's working hours) with purchase. Addition or alteration to country, regional & segment scope*

The objective of the study is to define market sizes of different segments & countries in recent years and to forecast the values to the coming years. The report is designed to incorporate both qualitative and quantitative aspects of the industry within countries involved in the study.

The report also caters detailed information about the crucial aspects such as driving factors & challenges which will define the future growth of the market. Additionally, it also incorporates potential opportunities in micro markets for stakeholders to invest along with the detailed analysis of competitive landscape and Therapeutic Area offerings of key players. The detailed segments and sub-segment of the market are explained below:

By Therapeutic Area:

Cancer
Neurological Conditions
Cardiovascular Conditions
Musculoskeletal Conditions
Hematologic Disorders
Infectious Diseases
Metabolic disorders
Endocrine disorders
Others

By Drug Type:

Biologics
Biosimilar
Small Molecule

By Route of Administration:

Oral
Injectable
Others

By Distribution Channel:

Hospital Pharmacy
Specialty Pharmacy
Online Pharmacy

By Region:

North America
U.S.
Canada
Europe
UK
Germany
France
Spain
Italy
ROE
Asia Pacific
China
India
Japan
Australia
South Korea
RoAPAC
Latin America
Brazil
Mexico
Rest of the World

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

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)