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Global Rare Disease Treatment Market Outlook 2037

Published Jun 26, 2025
Length 274 Pages
SKU # RENE20451228

Description

The global rare disease treatment market was valued at USD 232.2 billion in 2024 and is expected to grow significantly, reaching approximately USD 792.8 billion by 2037. This growth reflects a projected compound annual growth rate (CAGR) of 10.3% over the forecast period from 2025 to 2037. By the end of 2025, the market size is anticipated to reach around USD 243.1 billion. The rare disease treatment market is witnessing strong momentum due to scientific breakthroughs in genetics, cell therapy, and mRNA technology. With thousands of rare conditions lacking effective treatment, pharma companies are fast-tracking orphan drug development, supported by regulatory incentives such as the U.S. Orphan Drug Act and the EU’s PRIME scheme. The growing adoption of precision medicine and advanced diagnostics is enabling earlier intervention and targeted therapies. Meanwhile, biopharmaceutical investments and cross-border collaborations are expanding access to novel treatments, especially for ultra-rare and pediatric conditions.

Table of Contents

274 Pages
Rare Disease Treatment Market
Introduction
Study Objective
Scope of the report
Market Taxonomy
Study Assumptions and Abbreviations
Research Methodology
Secondary Research
Primary Research
SPSS Approach
Data Triangulation
Executive Summary
Global Industry Overview
Market Overview
Regional Synopsis
Industry Supply Chain Analysis
DROT
Driver
Restraint
Opportunities
Threat
Government Regulation: How they would Aid the Business?
Competitive Landscape
Alexion Pharmaceuticals, Inc. (AstraZeneca)
AMGEN INC.
BioMarin Pharmaceutical Inc.
F. Hoffmann-La Roche Ltd
Novartis AG
PFIZER INC.
Sanofi S.A.
Takeda Pharmaceutical Company Limited
Vertex Pharmaceuticals Incorporated
Ongoing Advanced Method
Price Benchmarking
SWOT Analysis
Drug Type
Patents Filed in the Industry
Government Initiatives
Recent News Analysis
Analysis of Rare Disease
Analysis of Rare Disease Disorder
Industry Risk Assessment
Global Outlook and Projections
Global Overview
Market Value (USD Million), Current and Future Projections, 2019-2037
Increment $ Opportunity Assessment, 2019-2037
Global Segmentation (USD Million), 2019-2037, By
Drug Type, Value (USD Million)
Biologics
Non-biologics
Diseases, Value (USD Million)
Cardiovascular Disorder
Cancer
Metabolic Disorder
Hematologic Disorder
Endocrine Disorder
Others
Patient, Value (USD Million)
Pediatric
Adults
Route of Administration, Value (USD Million)
Injectable
Oral
Distribution Channel, Value (USD Million)
Hospital Pharmacy
Others
Regional Synopsis (USD Million), 2019-2037
North America, Value (USD Million)
Europe, Value (USD Million)
Asia Pacific, Value (USD Million)
Latin America, Value (USD Million)
Middle East and Africa, Value (USD Million)
Cross Analysis of Drug Type w.r.t. Disease (USD Million), 2019-2037
North America Market
Overview
Market Revenue by Value (USD Million), Current and Future Projections, 2019-2037
Segmentation (USD Million), 2019-2037, By
Drug Type, Value (USD Million)
Biologics
Non-biologics
Diseases, Value (USD Million)
Cardiovascular Disorder
Cancer
Metabolic Disorder
Hematologic Disorder
Endocrine Disorder
Others
Patient, Value (USD Million)
Pediatric
Adults
Route of Administration, Value (USD Million)
Injectable
Oral
Distribution Channel, Value (USD Million)
Hospital Pharmacy
Others
Country Level Analysis
U.S.
Canada
Mexico
Cross Analysis of Drug Type w.r.t. Disease (USD Million), 2019-2037
Europe Market
Overview
Market Revenue by Value (USD Million), Current and Future Projections, 2019-2037
Segmentation (USD Million), 2019-2037, By
Drug Type, Value (USD Million)
Biologics
Non-biologics
Diseases, Value (USD Million)
Cardiovascular Disorder
Cancer
Metabolic Disorder
Hematologic Disorder
Endocrine Disorder
Others
Patient, Value (USD Million)
Pediatric
Adults
Route of Administration, Value (USD Million)
Injectable
Oral
Distribution Channel, Value (USD Million)
Hospital Pharmacy
Others
Country Level Analysis
UK
Germany
France
Italy
Spain
Russia
Netherlands
Switzerland
Poland
Belgium
Rest of Europe
Cross Analysis of Drug Type w.r.t. Disease (USD Million), 2019-2037
Asia Pacific Market
Overview
Market Revenue by Value (USD Million), Current and Future Projections, 2019-2037
Segmentation (USD Million), 2019-2037, By
Drug Type, Value (USD Million)
Biologics
Non-biologics
Diseases, Value (USD Million)
Cardiovascular Disorder
Cancer
Metabolic Disorder
Hematologic Disorder
Endocrine Disorder
Others
Patient, Value (USD Million)
Pediatric
Adults
Route of Administration, Value (USD Million)
Injectable
Oral
Distribution Channel, Value (USD Million)
Hospital Pharmacy
Others
Country Level Analysis
China
Japan
India
South Korea
Australia
Indonesia
Taiwan
Thailand
Singapore
Philippines
Vietnam
New Zealand
Malaysia
Rest of Asia Pacific
Cross Analysis of Drug Type w.r.t. Disease (USD Million), 2019-2037
Latin America Market
Overview
Market Revenue by Value (USD Million), Current and Future Projections, 2019-2037
Segmentation (USD Million), 2019-2037, By
Drug Type, Value (USD Million)
Biologics
Non-biologics
Diseases, Value (USD Million)
Cardiovascular Disorder
Cancer
Metabolic Disorder
Hematologic Disorder
Endocrine Disorder
Others
Patient, Value (USD Million)
Pediatric
Adults
Route of Administration, Value (USD Million)
Injectable
Oral
Distribution Channel, Value (USD Million)
Hospital Pharmacy
Others
Country Level Analysis
Brazil
Argentina
Mexico
Rest of Latin America
Cross Analysis of Drug Type w.r.t. Disease (USD Million), 2019-2037
Middle East and Africa Market
Overview
Market Revenue by Value (USD Million), Current and Future Projections, 2019-2037
Segmentation (USD Million), 2019-2037, By
Drug Type, Value (USD Million)
Biologics
Non-biologics
Diseases, Value (USD Million)
Cardiovascular Disorder
Cancer
Metabolic Disorder
Hematologic Disorder
Endocrine Disorder
Others
Patient, Value (USD Million)
Pediatric
Adults
Route of Administration, Value (USD Million)
Injectable
Oral
Distribution Channel, Value (USD Million)
Hospital Pharmacy
Others
Country Level Analysis
Saudi Arabia
UAE
Israel
Qatar
Kuwait
Oman
South Africa
Rest of Middle East & Africa
Cross Analysis of Drug Type w.r.t. Disease (USD Million), 2019-2037
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