희귀의약품 시장 규모, 점유율, 성장 분석 : 치료 유형별, 적응증별, 약제 유형별, 판매별, 유통 채널별, 지역별 - 산업 예측(2025-2032년)

Orphan Drug Market size was valued at USD 195.76 billion in 2023 and is poised to grow from USD 218.86 billion in 2024 to USD 534.2 billion by 2032, growing at a CAGR of 11.8% during the forecast period (2025-2032).

The orphan drug market has gained significant traction, particularly following the Orphan Drug Act of 2019, which aims to spur the development of treatments for rare diseases, defined by the WHO as those affecting less than 6.5 to 10 out of 10,000 individuals. Historically, the path to commercialization of these drugs is lengthy and costly, with a mere 10% success rate from molecule discovery to therapeutic application. Currently, over 378 orphan drugs have been approved, with the U.S. leading in clinical trials, particularly in oncology, which accounts for over 30% of these efforts. With approximately 7,000 rare diseases impacting 25-30 million people in the U.S. alone-and around 250 new conditions identified annually-the demand for orphan drugs continues to rise, poised for further market evolution.

Top-down and bottom-up approaches were used to estimate and validate the size of the Orphan Drug market and to estimate the size of various other dependent submarkets. The research methodology used to estimate the market size includes the following details: The key players in the market were identified through secondary research, and their market shares in the respective regions were determined through primary and secondary research. This entire procedure includes the study of the annual and financial reports of the top market players and extensive interviews for key insights from industry leaders such as CEOs, VPs, directors, and marketing executives. All percentage shares split, and breakdowns were determined using secondary sources and verified through Primary sources. All possible parameters that affect the markets covered in this research study have been accounted for, viewed in extensive detail, verified through primary research, and analyzed to get the final quantitative and qualitative data.

Orphan Drug Market Segments Analysis

Global Orphan Drug Market is segmented by Therapy Type, Indication Type, Drug Type, Sale, Distribution Channel and region. Based on Therapy Type, the market is segmented into Oncology, Hematology, Neurology, Endocrinology, Cardiovascular, Respiratory, Immunotherapy, Infectious Diseases and Others. Based on Indication Type, the market is segmented into Non-Hodgkin Lymphoma, Acute Myeloid Leukemia, Cystic Fibrosis, Glioma, Pancreatic Cancer, Ovarian Cancer, Multiple Myeloma, Duchenne Muscular Dystrophy, Renal Cell Carcinoma, Graft Vs Host Disease and Others. Based on Drug Type, the market is segmented into Biologics and Non-biologics. Based on Sale, the market is segmented into Generics and Prescribed. Based on Distribution Channel, the market is segmented into Hospital Pharmacy, Retail Pharmacy, Online Pharmacy and Others. Based on region, the market is segmented into North America, Europe, Asia Pacific, Latin America and Middle East & Africa.

Driver of the Orphan Drug Market

The Orphan Drug market is being significantly driven by advancements in technology, particularly in genomics and gene sequencing, which have facilitated research and development for rare diseases. These innovations have paved the way for novel treatment approaches, including gene therapy and antisense therapy, allowing for targeted interventions that were once deemed unfeasible. Consequently, the combination of these technological breakthroughs has enhanced the capacity for diagnosing and treating uncommon genetic disorders, making them more manageable and attractive to pharmaceutical companies. As a result, the landscape for orphan medications is evolving, ushering in a new era of possibilities for both patients and developers.

Restraints in the Orphan Drug Market

The orphan drug market faces significant constraints primarily due to the inability of many orphan medications to meet established health technology assessment standards, especially regarding cost-effectiveness. These challenges, exacerbated by the high cost of these drugs and insufficient data supporting their value, can limit patient access significantly. The high price tags associated with these treatments often lead to scrutiny from health payers and regulators who seek value for money, making it difficult for healthcare providers to justify their use. Consequently, the combination of cost and data limitations creates substantial barriers within the orphan drug market.

Market Trends of the Orphan Drug Market

The Orphan Drug Market is experiencing a notable upward trend due to the strategic adoption of auxiliary or surrogate endpoints in clinical trials, which allows companies to demonstrate treatment effectiveness with less stringent criteria. This paradigm shift not only streamlines the research and development process but also significantly lowers time and cost investment in bringing orphan drugs to market. Consequently, pharmaceutical companies are increasingly incentivized to develop therapies for rare diseases, further propelling market growth. As regulatory bodies continue to support these innovations, the orphan drug sector is poised for continued expansion, addressing unmet medical needs while driving profitability for developers.

Table of Contents

Introduction

• Objectives of the Study
• Scope of the Report
• Definitions

Research Methodology

• Information Procurement
• Secondary & Primary Data Methods
• Market Size Estimation
• Market Assumptions & Limitations

Executive Summary

• Global Market Outlook
• Supply & Demand Trend Analysis
• Segmental Opportunity Analysis

Market Dynamics & Outlook

• Market Overview
• Market Size
• Market Dynamics
  • Drivers & Opportunities
  • Restraints & Challenges
• Porters Analysis
  • Competitive rivalry
  • Threat of substitute
  • Bargaining power of buyers
  • Threat of new entrants
  • Bargaining power of suppliers

Key Market Insights

• Key Success Factors
• Degree of Competition
• Top Investment Pockets
• Market Ecosystem
• Market Attractiveness Index, 2024
• PESTEL Analysis
• Macro-Economic Indicators
• Value Chain Analysis
• Pricing Analysis

Global Orphan Drug Market Size by Therapy Type & CAGR (2025-2032)

• Market Overview
• Oncology
• Hematology
• Neurology
• Endocrinology
• Cardiovascular
• Respiratory
• Immunotherapy
• Infectious Diseases
• Others

Global Orphan Drug Market Size by Indication Type & CAGR (2025-2032)

• Market Overview
• Non-Hodgkin Lymphoma
• Acute Myeloid Leukemia
• Cystic Fibrosis
• Glioma
• Pancreatic Cancer
• Ovarian Cancer
• Multiple Myeloma
• Duchenne Muscular Dystrophy
• Renal Cell Carcinoma
• Graft Vs Host Disease
• Others

Global Orphan Drug Market Size by Drug Type & CAGR (2025-2032)

• Market Overview
• Biologics
• Non-biologics

Global Orphan Drug Market Size by Sale & CAGR (2025-2032)

• Market Overview
• Generics
• Prescribed

Global Orphan Drug Market Size by Distribution Channel & CAGR (2025-2032)

• Market Overview
• Hospital Pharmacy
• Retail Pharmacy
• Online Pharmacy
• Others

Global Orphan Drug Market Size & CAGR (2025-2032)

• North America (Therapy Type, Indication Type, Drug Type, Sale, Distribution Channel)
  • US
  • Canada
• Europe (Therapy Type, Indication Type, Drug Type, Sale, Distribution Channel)
  • Germany
  • Spain
  • France
  • UK
  • Italy
  • Rest of Europe
• Asia Pacific (Therapy Type, Indication Type, Drug Type, Sale, Distribution Channel)
  • China
  • India
  • Japan
  • South Korea
  • Rest of Asia-Pacific
• Latin America (Therapy Type, Indication Type, Drug Type, Sale, Distribution Channel)
  • Brazil
  • Rest of Latin America
• Middle East & Africa (Therapy Type, Indication Type, Drug Type, Sale, Distribution Channel)
  • GCC Countries
  • South Africa
  • Rest of Middle East & Africa

Competitive Intelligence

• Top 5 Player Comparison
• Market Positioning of Key Players, 2024
• Strategies Adopted by Key Market Players
• Recent Developments in the Market
• Company Market Share Analysis, 2024
• Company Profiles of All Key Players
  • Company Details
  • Product Portfolio Analysis
  • Company's Segmental Share Analysis
  • Revenue Y-O-Y Comparison (2022-2024)

Key Company Profiles

• Pfizer Inc. (United States)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• AbbVie Inc. (United States)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Novartis AG (Switzerland)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Amgen Inc. (United States)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• GSK plc (United Kingdom)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Johnson & Johnson (United States)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Sanofi (France)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• F. Hoffmann-La Roche Ltd (Switzerland)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Takeda Pharmaceutical Company Limited (Japan)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Bristol-Myers Squibb Company (United States)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• AstraZeneca plc (United Kingdom)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Biogen Inc. (United States)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• BioMarin Pharmaceutical Inc. (United States)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Swedish Orphan Biovitrum AB (Sobi) (Sweden)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Amicus Therapeutics (United States)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Ultragenyx Pharmaceutical Inc. (United States)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• PTC Therapeutics (United States)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Apellis Pharmaceuticals (United States)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Kamada Ltd. (Israel)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments

Conclusion & Recommendations