Global Orphan Diseases Drugs Market ExecutiveSummary
An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases. The Orphan Drug Designation program provides orphan status to drugs and biologics for rare diseases that meet certain criteria.
Market Size and Key Findings
The Global Orphan Diseases Drugs Market size is at around US $ xx Bn in 2021 and is projected to reach US $ xx Bn in 2030, exhibiting a CAGR of xx% during the forecast period.
Global Orphan Diseases Drugs Market Size (In USD Bn)
(2021-2030F)
Market Dynamics
Market Growth Drivers Analysis
The presence of a strong product pipeline and the imminent launches are expected to fuel market growth. Increasing approval and launch of new orphan drugs are anticipated to boost industry growth.
Market Restraints
The market growth could be hampered by the high cost associated with the research and development of pipeline candidates used for the treatment of rare diseases, which, in turn, contributes to the rise in drug prices. This can be attributed to factors such as the increased capital for conducting clinical trials through contract research organizations (CROs) and the risk of drug failure during the clinical trials.
Competitive Landscape
Key Players
Major players in Orphan Diseases Drugs Market are F. Hoffmann-La Roche Ltd.; Pfizer, Inc.; PTC Therapeutics; AstraZeneca; Novartis AG; Novo Nordisk; Bayer AG; AbbVie Inc.; Merck & Co. Inc.; Bristol Myers Squibb
TABLE OF CONTENT
1. Report Description of the Global Orphan Diseases Drugs Market
1.1 Research Scope and Assumption
1.2 Objective of the study
1.3 Research Methodology
1.4 Reason to buy the report
2. Global Orphan Diseases Drugs Market Executive Summary
2.1 Global Orphan Diseases Drugs Market– Industry Snapshot & key buying criteria, 2021-2030
2.2 Market Size, Growth Prospects, and Key findings
3. Market Dynamics of Global Orphan Diseases Drugs Market
3.1 Market Growth Drivers Analysis
3.2 Market Restrains Analysis
4. Global Orphan Diseases Drugs Market Segmentation
4.1 Therapeutic area
4.1.1 Cancer
4.1.2 Neurological condition
4.1.3 Cardiovascular condition
4.1.4 Musculoskeletal condition
4.1.4 Hematologic condition
4.1.5 Others
4.2 Route of administration
4.2.1 Oral
4.2.2 Injectable
4.2.3 Others
4.3 Drug type
4.3.1 Biologics
4.3.2 Biosimilar
4.3.3 Small Molecule
4.4 Distribution Channel
4.4.1 Hospital Pharmacy
4.4.2 Specialty Pharmacy
4.4.3 Online Pharmacy
5. Global Orphan Diseases Drugs Market Share
5.1 Market Analysis, Insights, and Forecast – By Revenue
6. Competitive Landscape
6.1 Major Market Players
7. Key Company Profiles
7.1 Company 1
7.1.1 Overview
7.1.2 Financial Performance
7.1.3 Product & Services
7.1.4 Strategic initiatives
7.2 Company 2
7.2.1 Overview
7.2.2 Financial Performance
7.2.3 Product & Services
7.2.4 Strategic initiatives
7.3 Company 3
7.3.1 Overview
7.3.2 Financial Performance
7.3.3 Product & Services
7.3.4 Strategic initiatives
7.4 Company 4
7.4.1 Overview
7.4.2 Financial Performance
7.4.3 Product & Services
7.4.4 Strategic initiatives
7.5 Company 5
7.5.1 Overview
7.5.2 Financial Performance
7.5.3 Product & Services
7.5.4 Strategic initiatives
8. Healthcare Policies and Regulatory Landscape
8.1 Healthcare Policies in Global
8.2 Regulatory Framework in Global
8.3 Pricing & Reimbursement Scenario in Global Orphan Diseases Drugs Market
9. Factors Driving Future Growth
9.1 New Trends & Development In Global Orphan Diseases Drugs Market
9.2 Future Opportunities
10. Strategic Recommendations
Segmentation
Orphan Diseases Drugs Market Segmentation
Therapeutic area
Cancer
Neurological condition
Cardiovascular condition
Musculoskeletal condition
Hematologic condition
Others
Route of administration
The injectable segment dominated the market and is expected to witness significant growth during the forecast period. This can be attributed to the launch of novel injectables for the treatment of rare diseases.
Oral
Injectable
Others
Drug Type
Biologics dominated the market and is expected to witness significant growth during the forecast period. The advancement in biotechnology and research techniques has facilitated the development of novel biologics. High target specificity and potential of biological drugs revolutionizing the treatments of several rare diseases are expected to fuel the segment growth.
Biologics
Biosimilars
Small molecule
Distribution Channel
The specialty pharmacy segment dominated the market. The dominance can be attributed to strategic initiatives undertaken by key players, such as the acquisition of specialty pharmacies for the distribution of their products.
Hospital Pharmacy
Specialty Pharmacy
Online Pharmacy
Methodology
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