孤儿药市场规模、份额和成长分析（按药物类型、疾病类型、分销管道和地区）：产业预测（2024-2031）

2022年全球孤儿药市场规模估值为1,750.6亿美元，从2023年的1957.6亿美元成长到2031年的4784.9亿美元，在预测期内（2024年）预计2019年至2031年复合年增长率为11.82 % ）。

根据世界卫生组织 (WHO) 的定义，孤儿药市场主要是由解决每 10,000 人中影响不到约 10 人的罕见疾病的迫切需求推动的。对于这些疾病，只有十分之一的治疗分子有潜力被证明是有效的，并且开发通常需要大约 10 年和数千万欧元，製药公司不愿意遵循标准开发通讯协定。自2019年鼓励开发罕见疾病治疗方法的《孤儿药法案》生效以来，已有378种孤儿药核准，Kalydeco就是一个显着的例子。美国在孤儿药开发方面持续处于领先地位，目前有300多种药物处于临床试验阶段，其中癌症占30%以上。目前，美国国立卫生研究院认可的大约美国7,000 种罕见疾病，主要是遗传和神经系统疾病，影响了约 2500 至 3000 万美国。美国孤儿药市场正在经历显着增长，每年报告约 250 种新的罕见疾病，预计遗传学的进步可能会导致更多常见疾病被归类为独特的罕见疾病。市场分析师预计未来几年将出现持续的复合年增长率，凸显了孤儿药在应对罕见疾病带来的独特挑战方面所发挥的关键作用。

目录

介绍

• 分析目的
• 定义
• 市场范围

分析方法

• 资讯采购
• 二手资料来源和主要资料来源
• 市场规模预测
• 市场假设与限制

执行摘要

• 市场概况及展望
• 供需趋势分析
• 按细分市场的机会分析

市场动态及展望

• 市场动态
  • 促进因素
  • 机会
  • 抑制因素
  • 任务
• 波特的分析

主要市场考察

• 关键成功因素
• 竞争程度
• 主要投资机会
• 市场生态系统
• 价值链分析
• 技术分析
• 专利分析
• 管道分析
• 监管环境

孤儿药市场：依药物类型

• 市场概况
• 生物
• 非生物的

孤儿药市场：依疾病类型

• 市场概况
• 肿瘤学
• 血液学
• 神经病学
• 内分泌学
• 心血管
• 呼吸系统
• 免疫疗法
• 感染疾病
• 其他的

孤儿药市场：依通路分类

• 市场概况
• 医院
• 药局
• 网上销售
• 其他的

孤儿药市场：依地区

• 市场概况
• 北美洲
  • 美国
  • 加拿大
• 欧洲
  • 德国
  • 西班牙
  • 法国
  • 英国
  • 义大利
  • 其他欧洲国家
• 亚太地区
  • 中国
  • 印度
  • 日本
  • 韩国
  • 其他亚太地区
• 拉丁美洲
  • 巴西
  • 其他拉丁美洲
• 中东和非洲 (MEA)
  • 波湾合作理事会(GCC) 国家
  • 南非
  • 其他中东/非洲

竞争格局

• 前5名企业对比
• 主要企业市场定位（2023年）
• 主要市场参与者所采取的策略
• 近期市集活动
• 主要企业市场占有率（2023年）

主要企业简介

• Novartis AG
• Pfizer Inc.
• Sanofi
• Bristol-Myers Squibb Company
• GSK plc
• AbbVie Inc.
• Merck & Co., Inc.
• Johnson & Johnson
• F. Hoffmann-La Roche Ltd
• Vertex Pharmaceuticals
• BioMarin Pharmaceutical
• Takeda Pharmaceutical Company Limited
• Roche
• Amgen
• Ultragenyx Pharmaceutical
• CSL Behring
• Grifols
• Sobi (Swedish Orphan Biovitrum)
• Ipsen
• Jazz Pharmaceuticals
• AstraZeneca

Global Orphan Drugs Market size was valued at USD 175.06 billion in 2022 and is poised to grow from USD 195.76 billion in 2023 to USD 478.49 billion by 2031, growing at a CAGR of 11.82% during the forecast period (2024-2031).

The orphan drug market is gaining significant momentum, primarily driven by the pressing need to address rare diseases that affect fewer than approximately 10 people per 10,000, as defined by the World Health Organization (WHO). These illnesses often deter pharmaceutical companies from following standard development protocols due to the high costs and lengthy timeline, typically taking around ten years and requiring tens of millions of euros, with only one in ten molecules potentially proving effective. Since the implementation of the Orphan Drug Act in 2019, which incentivizes the development of treatments for rare diseases, a remarkable 378 orphan drugs have obtained approval, with Kalydeco standing out as a notable example. The U.S. remains the leader in orphan drug development, with over 300 drugs currently in clinical trials, especially for cancer, which represents more than 30% of these trials. Presently, approximately 7,000 rare diseases are recognized by the National Institute of Health, impacting an estimated 25-30 million Americans, primarily genetic and neurological disorders. As about 250 new rare diseases are documented annually, and with ongoing genetic advancements that will likely categorize more common conditions into distinct rare diseases, the U.S. orphan drug market is poised for substantial growth. Market analysts anticipate a sustainable compound annual growth rate (CAGR) for the coming years, underscoring the critical role orphan drugs will play in addressing the unique challenges posed by rare diseases.

Top-down and bottom-up approaches were used to estimate and validate the size of the Global Orphan Drugs 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.

Global Orphan Drugs Market Segmental Analysis

Global Orphan Drugs Market is segmented by Drug Type, Disease Type, Distribution Channel, and Region. Based on Drug Type, the market is segmented into Biological, and Non-biological. Based on Disease Type, the market is segmented into O Hematology, Neurology, Endocrinology, Cardiovascular, Respiratory, Immunotherapy, Infectious Diseases, and Others. Based on Distribution Channel, the market is segmented into Hospital, Pharmacy, Online sales, Others. Based on region, the market is segmented into North America, Europe, Asia Pacific, Latin America and Middle East & Africa.

Driver of the Global Orphan Drugs Market

The rapid advancement of technology, particularly in genomics and gene sequencing, is a key driver for the Global Orphan Drugs market. These innovations facilitate the identification and understanding of rare diseases, enabling the development of targeted therapies such as gene and antisense therapies. With these technologies, pharmaceutical companies can diagnose and treat uncommon genetic disorders more effectively than ever before. Additionally, attractive incentives, such as accelerated development timelines and favorable regulatory landscapes, render the development of orphan drugs economically viable, despite the small patient populations. Consequently, this synergistic effect of technological progress and supportive frameworks is propelling growth in the orphan drugs market.

Restraints in the Global Orphan Drugs Market

The Global Orphan Drugs market faces significant restraints primarily due to the stringent health technology assessment criteria that orphan medications often fail to meet, particularly concerning cost-effectiveness. High costs and inadequate clinical trial data hinder access and adoption of these therapies. Moreover, the recruitment of qualified patients for clinical trials presents a considerable challenge, given the rarity of the diseases targeted by orphan drugs. Efficient patient targeting requires a deep understanding of the underlying biology of these uncommon disorders, further complicating the development process. These barriers can limit market growth and the overall availability of orphan drugs for patients in need.

Market Trends of the Global Orphan Drugs Market

The Global Orphan Drugs market is experiencing a robust growth trend, driven by the increasing use of auxiliary or surrogate endpoints in clinical trials, which significantly streamline the research and development process. This innovative approach reduces both time and costs associated with bringing new therapies to market, enabling companies to respond swiftly to the demand for effective treatments for rare diseases. Heightened patient advocacy and expansion in R&D efforts are fostering the introduction of advanced therapeutics, leading to the emergence of blockbuster drugs across various categories, including oncology and immune disorders. As a result, the market is poised for continued expansion and innovation.

Table of Contents

Introduction

• Objectives of the Study
• Definitions
• Market Scope

Research Methodology

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

Executive Summary

• Market Overview Outlook
• Supply Demand Trend Analysis
• Segmental Opportunity Analysis

Market Dynamics & Outlook

• Market Dynamics
  • Drivers
  • Opportunities
  • Restraints
  • Challenges
• Porters Analysis
  • Competitive rivalry
  • Threat of Substitute Products
  • Bargaining Power of Buyers
  • Threat of New Entrants
  • Bargaining Power of Suppliers

Key Market Insights

• Key Success Factor
• Degree of Competition
• Top Investment Pockets
• Ecosystem of the Market
• Value Chain Analysis
• Technology Analysis
• Patent Analysis
• Pipeline Analysis
• Regulatory Landscape

Orphan Drug Market, By Drug Type

• Market Overview
• Biological
• Non-biological

Orphan Drug Market, By Disease Type

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

Orphan Drug Market, By Distribution Channel

• Market Overview
• Hospital
• Pharmacy
• Online sales
• Others

Orphan Drug Market Size by Region

• Market Overview
• North America
  • USA
  • Canada
• Europe
  • Germany
  • Spain
  • France
  • UK
  • Italy
  • Rest of Europe
• Asia Pacific
  • China
  • India
  • Japan
  • South Korea
  • Rest of Asia-Pacific
• Latin America
  • Brazil
  • Rest of Latin America
• Middle East & Africa (MEA)
  • GCC Countries
  • South Africa
  • Rest of MEA

Competitive Landscape

• Top 5 Player Comparison
• Market Positioning of Key Players, 2023
• Strategies Adopted by Key Market Players
• Recent Activities in the Market
• Key Companies Market Share (%), 2023

Key Company Profiles

• Novartis AG
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Pfizer Inc.
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Sanofi
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Bristol-Myers Squibb Company
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• GSK plc
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• AbbVie Inc.
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Merck & Co., Inc.
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Johnson & Johnson
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• F. Hoffmann-La Roche Ltd
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Vertex Pharmaceuticals
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• BioMarin Pharmaceutical
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Takeda Pharmaceutical Company Limited
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Roche
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Amgen
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Ultragenyx Pharmaceutical
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• CSL Behring
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Grifols
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Sobi (Swedish Orphan Biovitrum)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Ipsen
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Jazz Pharmaceuticals
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• AstraZeneca
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments