Executive Summary
According to Azoth Analytics research report, the Global Rare Disease Drugs was valued at USD 147.12 Billion in the year 2021. The increasing focus on research activities and the development of novel therapeutic drugs has facilitated the growth of the Rare Disease Drugs market. Additionally, growing product developments is leading to the implementation of technologically driven innovative devices in the diagnosis of rare genetic disorders.
Moreover, favourable government policies aiming at creating awareness pertaining to the Rare Disease treatment are leading to increased adoption of effective treatments of rare disorders. However, there are several factors hindering the efficient and effective clinical trials of drug development of rare disorders including low patient numbers, limited understanding of pathology and progression, and lack of established endpoints. Therefore, several regulatory bodies are engaged in implementing regulatory standards for drug approval for Rare diseases to ensure patient safety and efficacy of drugs.
Furthermore, improved detection and increased rates of diagnosis of Rare diseases are likely to contribute to rising costs. While the humanitarian benefits of Rare Disease therapies are indisputable, concerns regarding high treatment costs play a meaningful role in how payers administer and manage healthcare benefits that ultimately dictate patient access to orphan drugs. Moreover, the biological drugs are used for treating Rare diseases such as cancer with fewer side effects that have a high prevalence rate in the developed world.
Scope of the Report
• The report presents the analysis of the Rare Disease Drugs market for the historical period of 2017-2021 and the forecast period of 2022-2027.
• The report analyses the Rare Disease Drugs Market by Value (USD Billion).
• The report analyses the Rare Disease Drugs Market by Drug Type (Biologics, Non-Biologics).
• The report analyses the Rare Disease Drugs Market by Therapeutic Area (Infectious Disease, Oncology, Neurology, Metabolic, Others).
• The report analyses the Rare Disease Drugs Market by Patients (Pediatric, Adults).
• The report analyses the Rare Disease Drugs Market by Route of Administration (Oral, Injectable).
• The report analyses the Rare Disease Drugs Market by Distribution Channel (Hospital & Retail Pharmacies, Online Pharmacies).
• The Global Rare Disease Drugs Market has been analysed By Region (North America, Europe, Asia Pacific and LAMEA).
• The Global Rare Disease Drugs Market has been analysed By Country (United States, Canada, Germany, United Kingdom, France, Italy, China, Japan, India, South Korea).
• The attractiveness of the market has been presented by region, by Drug Type, by Therapeutic area, by patients, by Route of Administration and by Distribution Channel
• Also, the major opportunities, trends, drivers and challenges of the industry has been analysed in the report.
• The report tracks competitive developments, strategies, mergers and acquisitions and new product development.
• The companies analysed in the report include Takeda Pharmaceutical Company Limited, Sanofi, Merck KGaA, F. Hoffman-La Roche Ltd., Pfizer, Novartis AG, Bayer Group, Biogen, GlaxoSmithKline, AbbVie Inc.
Key Target Audience
• Rare Disease Drugs Manufacturers
• Healthcare and Pharmaceutical Companies
• Consulting and Advisory Firms
• Government and Policy Makers
• Regulatory Authorities
1. Report Scope and Methodology
1.1 Scope of the Report
1.2 Research Methodology
1.3 Executive Summary
2. Strategic Recommendations
3. Rare Disease Drugs Market: Product Outlook
4. Global Rare Disease Drugs Market: Sizing and Forecast
4.1 Global Rare Disease Drugs Market Size, By Value, 2017-2027
4.2 Impact of COVID-19 on Global Rare Disease Drugs Market
5. Global Rare Disease Drugs Market Segmentation - By Drug Type, By Therapeutic Area, By Patients, By Route of Administration, By Distribution Channel
5.1 Competitive Scenario of Global Rare Disease Drugs Market: By Drug Type
5.1.1 Biologics - Market Size and Forecast (2017-2027)
5.1.2 Non-Biologics - Market Size and Forecast (2017-2027)
5.2 Competitive Scenario of Global Rare Disease Drugs Market: By Therapeutic Area
5.2.1 Infectious disease - Market Size and Forecast (2017-2027)
5.2.2 Oncology- Market Size and Forecast (2017-2027)
5.2.3 Neurological - Market Size and Forecast (2017-2027)
5.2.4 Metabolic - Market Size and Forecast (2017-2027)
5.2.5 Others - Market Size and Forecast (2017-2027)
5.3 Competitive Scenario of Global Rare Disease Drugs Market: By Patients
5.3.1 Paediatric - Market Size and Forecast (2017-2027)
5.3.2 Adults - Market Size and Forecast (2017-2027)
5.4 Competitive Scenario of Global Rare Disease Drugs Market: By Route of Administration
5.4.1 Oral - Market Size and Forecast (2017-2027)
5.4.2 Injectable - Market Size and Forecast (2017-2027)
5.5 Competitive Scenario of Global Rare Disease Drugs Market: By Distribution Channel
5.5.1 Hospitals & Retail Pharmacies- Market Size and Forecast (2017-2027)
5.5.2 Online Pharmacies - Market Size and Forecast (2017-2027)
6. Global Rare Disease Drugs Market: Regional Analysis
6.1 Competitive Scenario of Global Rare Disease Drugs Market: By Region
7. North America Rare Disease Drugs Market: An Analysis (2017-2027)
7.1 North America Rare Disease Drugs Market: Size and Forecast (2017-2027), By Value
7.2 North America Rare Disease Drugs Market - Prominent Companies
7.3 Market Segmentation By Drug Type (Biologics, Non-Biologics)
7.4 Market Segmentation By Therapeutic Area (Infectious Disease, Oncology, Neurological, Metabolic and Others)
7.5 Market Segmentation By Patients Type (Pediatric, Adults)
7.6 Market Segmentation By Route of Administration (Oral, Injectable)
7.7 Market Segmentation By Distribution Channel (Hospitals & Retail Pharmacies, Online Pharmacies)
7.8 North America Rare Disease Drugs Market: Country Analysis
7.9 Market Opportunity Chart of North America Rare Disease Drugs Market - By Country, By Value, 2027
7.10 Competitive Scenario of North America Rare Disease Drugs Market: By Country
7.11 United States Rare Disease Drugs Market: Size and Forecast (2017-2027), By Value
7.12 United States Rare Disease Drugs Market Segmentation - By Drug Type, By Therapeutic Area, By Patients, By Route of Administration, By Distribution Channel (2017-2027)
7.13 Canada Rare Disease Drugs Market: Size and Forecast (2017-2027), By Value
7.14 Canada Rare Disease Drugs Market Segmentation - By Drug Type, By Therapeutic Area, By Patients, By Route of Administration, By Distribution Channel (2017-2027)
8. Europe Rare Disease Drugs Market: An Analysis (2017-2027)
8.1 Europe Rare Disease Drugs Market: Size and Forecast (2017-2027), By Value
8.2 Europe Rare Disease Drugs Market - Prominent Companies
8.3 Market Segmentation By Drug Type (Biologics, Non-Biologics)
8.4 Market Segmentation By Therapeutic Area (Infectious Disease, Oncology, Neurological, Metabolic, Others)
8.5 Market Segmentation By Patients Type (Pediatric, Adults)
8.6 Market Segmentation By Route of Administration (Oral, Injectable)
8.7 Market Segmentation By Distribution Channel (Hospitals & Retail Pharmacies, Online Pharmacies)
8.8 Europe Rare Disease Drugs Market: Country Analysis
8.9 Market Opportunity Chart of Europe Rare Disease Drugs Market - By Country, By Value, 2027
8.10 Competitive Scenario of Europe Rare Disease Drugs Market: By Country
8.11 Germany Rare Disease Drugs Market: Size and Forecast (2017-2027), By Value
8.12 Germany Rare Disease Drugs Market Segmentation - By Drug Type, By Therapeutic Area, By Patients, By Route of Administration, By Distribution Channel (2017-2027)
8.13 United Kingdom Rare Disease Drugs Market: Size and Forecast (2017-2027), By Value
8.14 United Kingdom Rare Disease Drugs Market Segmentation - By Drug Type, By Therapeutic Area, By Patients, By Route of Administration, By Distribution Channel (2017-2027)
8.15 France Rare Disease Drugs Market: Size and Forecast (2017-2027), By Value
8.16 France Rare Disease Drugs Market Segmentation - By Drug Type, By Therapeutic Area, By Patients, By Route of Administration, By Distribution Channel (2017-2027)
8.17 Italy Rare Disease Drugs Market: Size and Forecast (2017-2027), By Value
8.18 Italy Rare Disease Drugs Market Segmentation - By Drug Type, By Therapeutic Area, By Patients, By Route of Administration, By Distribution Channel (2017-2027)
9. Asia Pacific Rare Disease Drugs Market: An Analysis (2017-2027)
9.1 Asia Pacific Rare Disease Drugs Market: Size and Forecast (2017-2027), By Value
9.2 Asia Pacific Rare Disease Drugs Market - Prominent Companies
9.3 Market Segmentation By Drug Type (Biologics, Non-Biologics)
9.4 Market Segmentation By Therapeutic Area (Infectious Disease, Oncology, Neurological, Metabolic, Others)
9.5 Market Segmentation By Patients Type (Pediatric, Adults)
9.6 Market Segmentation By Route of Administration (Oral, Injectable)
9.7 Market Segmentation By Distribution Channel (Hospitals & Retail Pharmacies, Online Pharmacies)
9.8 Asia Pacific Rare Disease Drugs Market: Country Analysis
9.9 Market Opportunity Chart of Asia Pacific Rare Disease Drugs Market - By Country, By Value, 2027
9.10 Competitive Scenario of Asia Pacific Rare Disease Drugs Market: By Country
9.11 China Rare Disease Drugs Market: Size and Forecast (2017-2027), By Value
9.12 China Rare Disease Drugs Market Segmentation - By Drug Type, By Therapeutic Area, By Patients, By Route of Administration, By Distribution Channel (2017-2027)
9.13 Japan Rare Disease Drugs Market: Size and Forecast (2017-2027), By Value
9.14 Japan Rare Disease Drugs Market Segmentation - By Drug Type, By Therapeutic Area, By Patients, By Route of Administration, By Distribution Channel (2017-2027)
9.15 India Rare Disease Drugs Market: Size and Forecast (2017-2027), By Value
9.16 India Rare Disease Drugs Market Segmentation - By Drug Type, By Therapeutic Area, By Patients, By Route of Administration, By Distribution Channel (2017-2027)
9.17 South Korea Rare Disease Drugs Market: Size and Forecast (2017-2027), By Value
9.18 South Korea Rare Disease Drugs Market Segmentation - By Drug Type, By Therapeutic Area, By Patients, By Route of Administration, By Distribution Channel (2017-2027)
10. Global Rare Disease Drugs Market Dynamics
10.1 Drivers
10.2 Restraints
10.3 Trends
11. Market Attractiveness
11.1 Market Attractiveness Chart of Global Rare Disease Drugs Market - By Drug Class, 2027
11.2 Market Attractiveness Chart of Global Rare Disease Drugs Market - By Therapeutic Area, 2027
11.3 Market Attractiveness Chart of Global Rare Disease Drugs Market - By Patients, 2027
11.4 Market Attractiveness Chart of Global Rare Disease Drugs Market - By Route of Administration, 2027
11.5 Market Attractiveness Chart of Global Rare Disease Drugs Market - By Distribution Channel, 2027
11.6 Market Attractiveness Chart of Global Rare Disease Drugs Market - By Region, 2027
12. Competitive Landscape
12.1 Market Share Analysis
13. Company Analysis
13.1 Takeda Pharmaceutical Company Limited
13.2 Sanofi
13.3 Merck KGaA
13.4 F. Hoffman-La Roche Ltd.
13.5 Pfizer
13.6 Novartis AG
13.7 Bayer Group
13.8 Biogen
13.9 GlaxoSmithKline
13.10 AbbVie Inc.
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