孤兒藥市場規模、佔有率和成長分析（按藥物類型、疾病類型、分銷管道和地區）：產業預測（2024-2031）

2022年全球孤兒藥市場規模估值為1,750.6億美元，從2023年的1957.6億美元成長到2031年的4784.9億美元，在預測期內（2024年）預計2019年至2031年複合年成長率為11.82 % ）。

根據世界衛生組織 (WHO) 的定義，孤兒藥市場主要是由解決每 10,000 人中影響不到約 10 人的罕見疾病的迫切需求推動的。對於這些疾病，只有十分之一的治療分子有潛力被證明是有效的，並且開發通常需要大約 10 年和數千萬歐元，製藥公司不願意遵循標準開發通訊協定。自2019年鼓勵開發罕見疾病治療方法的《孤兒藥法案》生效以來，已有378種孤兒藥核准，Kalydeco就是一個顯著的例子。美國在孤兒藥開發方面持續處於領先地位，目前有300多種藥物處於臨床試驗階段，其中癌症佔30%以上。目前，美國國立衛生研究院認可的大約美國7,000 種罕見疾病，主要是遺傳和神經系統疾病，影響了約 2500 至 3000 萬美國。美國孤兒藥市場正在經歷顯著成長，每年報告約 250 種新的罕見疾病，預計遺傳學的進步可能會導致更多常見疾病被歸類為獨特的罕見疾病。市場分析師預計未來幾年將出現持續的複合年成長率，凸顯了孤兒藥在應對罕見疾病帶來的獨特挑戰方面所發揮的關鍵作用。

目錄

介紹

• 分析目的
• 定義
• 市場範圍

分析方法

• 資訊採購
• 二手資料來源和主要資料來源
• 市場規模預測
• 市場假設與限制

執行摘要

• 市場概況及展望
• 供需趨勢分析
• 按細分市場的機會分析

市場動態及展望

• 市場動態
  • 促進因素
  • 機會
  • 抑制因素
  • 任務
• 波特的分析

主要市場考察

• 關鍵成功因素
• 競爭程度
• 主要投資機會
• 市場生態系統
• 價值鏈分析
• 技術分析
• 專利分析
• 管道分析
• 監管環境

孤兒藥市場：依藥物類型

• 市場概況
• 生物
• 非生物的

孤兒藥市場：依疾病類型

• 市場概況
• 腫瘤學
• 血液學
• 神經病學
• 內分泌學
• 心血管
• 呼吸系統
• 免疫療法
• 感染疾病
• 其他

孤兒藥市場：依通路分類

• 市場概況
• 醫院
• 藥局
• 網上銷售
• 其他

孤兒藥市場：依地區

• 市場概況
• 北美洲
  • 美國
  • 加拿大
• 歐洲
  • 德國
  • 西班牙
  • 法國
  • 英國
  • 義大利
  • 其他歐洲國家
• 亞太地區
  • 中國
  • 印度
  • 日本
  • 韓國
  • 其他亞太地區
• 拉丁美洲
  • 巴西
  • 其他拉丁美洲
• 中東和非洲 (MEA)
  • 波灣合作理事會(GCC) 國家
  • 南非
  • 其他中東/非洲

競爭格局

• 前5名企業對比
• 主要企業市場定位（2023年）
• 主要市場參與者所採取的策略
• 近期市集活動
• 主要企業市場佔有率（2023年）

主要企業簡介

• 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