希少疾病用医薬品の世界市場 - 2024年～2032年

世界の希少疾病用医薬品市場は、2023年に2,237億6,000万米ドルに達し、2032年には4,865億1,000万米ドルに達すると予測され、予測期間2024年のCAGRは9.1%で成長します。

希少疾病用医薬品は、希少疾病の治療、予防、診断に適応される医薬品です。米国食品医薬品局（FDA）によると、希少疾病とは国内の患者数が20万人未満の希少な疾患です。欧州医薬品庁（European Medicine Agency）によれば、EU全体で1万人に5人未満しか罹患しない疾患を希少疾患と定義しています。

世界保健機関（WHO）は、10万人あたり65人未満を希少疾病または希少疾病と定義しています。Orphanetによれば、希少疾病指定とは、希少疾病に対する治療可能性のある医薬品を、ヒトへの初投与前または臨床開発中に指定することを認める法的手続きです。

しかし、希少疾病用医薬品の開発は、患者数が限られており、市場の可能性も低いため、製薬会社にとって経済的な魅力に乏しいです。このような新規治療法の開発を促進するため、世界中の政府や規制機関は、税額控除、市場独占権、迅速な薬事承認などのインセンティブを提供しています。

市場力学：

促進要因と抑制要因

新薬開発活動の活発化と希少疾病用医薬品の指定

希少疾患患者の間で先進的な治療薬に対する需要が高まり続けていることから、メーカーはアンメットニーズの高い新薬の開発に積極的に取り組んでいます。世界中の規制機関は、高いアンメットニーズを考慮し、これらの新薬に希少疾病用医薬品の指定や迅速な承認を提供し、メーカーを継続的に支援しています。

例えば、2024年2月、米国食品医薬品局（FDA）はIonis Pharmaceuticals, Inc.が開発したオレザルセンに希少疾病用医薬品の指定を与えました。オレザルセンは家族性カイロミクロン血症症候群（FCS）の治療を目的とした治験薬で、極めて高いトリグリセリド値と再発性急性膵炎（AP）を特徴とする稀な遺伝性疾患です。イオニスは、オレザルセンが承認されれば、米国におけるFCS治療の標準薬となる可能性があるとしています。2023年1月、同剤はすでに米国FDAからファストトラック指定を受けた。

さらに2024年10月、カインド・ファーマシューティカルズLLCは、米国食品医薬品局（FDA）が鎌状赤血球症（SCD）の治療薬としてAND017を希少疾病用医薬品（オーファンドラッグ）に指定したと発表しました。AND017は、赤血球（RBC）のライフサイクルの複数の段階を標的とするファーストインクラスのヘモグロビン上昇薬です。カインド薬品は、AND017の希少疾病用医薬品指定は、SCD患者を安全かつ効果的に治療するための新しい治療法、特に経口薬に対する緊急の医療上の必要性を強調するものであるとしています。

このような製品開発活動の活発化と希少疾病用医薬品の指定は、アンメットニーズの高い様々な治療領域で大きな牽引力となり、市場全体の成長を促進すると期待されています。

希少疾病用医薬品の高コストが市場成長を抑制する可能性

希少疾病用医薬品は、いくつかの希少疾患の治療シナリオに革命をもたらすもの、その高コストが、特に中低所得層の患者における採用率を抑制する可能性があります。例えば、Drugs.comによると、Spinrazaの定価は初年度が75万米ドル、それ以降は35万米ドルです。Spinrazaは、脊髄性筋萎縮症（SMA）患者の治療薬としてZolgensmaに代わる薬剤です。患者の大多数が適切な医療保険に加入していないインドでは、同薬の薬価は約700万インドルピーです。

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目次

第1章 調査手法と調査範囲

第2章 定義と概要

第3章 エグゼクティブサマリー

第4章 市場力学

• 影響要因
  • 促進要因
    • 新薬開発活動と希少疾病用医薬品指定の増加
    • 希少疾患を抱える未治療患者層からの需要増加
  • 抑制要因
    • 治療費の高騰
  • 機会
  • 影響分析

第5章 産業分析

• ポーターのファイブフォース分析
• サプライチェーン分析
• 疫学
• 価格分析
• パイプライン分析
• 特許分析
• 規制分析

第6章 製品タイプ別

• 小分子
• 生物学的製剤
  • 遺伝子治療
  • モノクローナル抗体
  • その他
• その他

第7章 治療領域別

• 腫瘍学
• 血液疾患
• 中枢神経系
• 心臓血管
• 内分泌
• 呼吸器
• 免疫疾患
• 胃腸
• 筋骨格
• 皮膚科
• その他

第8章 地域別

• 北米
  • 米国
  • カナダ
  • メキシコ
• 欧州
  • ドイツ
  • 英国
  • フランス
  • スペイン
  • イタリア
  • その他欧州地域
• 南米
  • ブラジル
  • アルゼンチン
  • その他南米
• アジア太平洋地域
  • 中国
  • インド
  • 日本
  • 韓国
  • その他アジア太平洋地域
• 中東・アフリカ

第9章 競合情勢

• 競合シナリオ
• 市況・シェア分析
• M&A分析

第10章 企業プロファイル

• Johnson & Johnson Services, Inc.
  • 会社概要
  • 製品タイプポートフォリオと説明
  • 財務概要
  • 主な発展
• AbbVie Inc.
• Vertex Pharmaceuticals Incorporated
• AstraZeneca.
• Incyte.
• Biogen.
• Novartis AG
• F. Hoffmann-La Roche Ltd
• Pfizer Inc.
• Bristol-Myers Squibb Company

第11章 付録

The global orphan drugs market reached US$ 223.76 billion in 2023 and is expected to reach US$ 486.51 billion by 2032, growing at a CAGR of 9.1% during the forecast period 2024-2032.

Orphan drugs are the medication indicated to treat, prevent, or diagnose an orphan disease. As per the U.S. Food and Drug Administration (FDA), orphan disease is a rare condition with less than 200,000 patients in the country. According to the European Medicine Agency, a disease is defined as rare if it affects fewer than 5 in 10,000 people across the European Union.

The World Health Organization defines a rare disease or an orphan disease as one that affects fewer than 65 per 100,000 people. As per Orphanet, orphan designation is a legal procedure that allows for the designation of a medicinal substance with therapeutic potential for a rare disease, before its first administration in humans or during its clinical development.

However, Developing orphan drugs is less financially attractive for pharmaceutical companies due to the limited patient size, and less market potential. To encourage the development of these novel therapies, governments and regulatory bodies across the globe offer incentives like tax credits, market exclusivity, and expedited regulatory approval.

Market Dynamics: Drivers & Restraints

Rising Novel Drug Development Activities and Orphan Drug Designations

As the demand for advanced therapeutics continues to rise among patients with rare diseases, manufacturers are actively involved in developing these novel drugs to cater to the high unmet needs. The regulatory bodies across the world have been continually supporting the manufacturers considering the high unmet needs, and providing orphan drug designations and accelrated approvals to these new drugs.

For instance, in February 2024, the U.S. Food and Drug Administration (FDA) granted orphan drug designation for olezarsen developed by Ionis Pharmaceuticals, Inc. Olezarsen is an investigational drug aimed at the treatment of familial chylomicronemia syndrome (FCS) which is a rare, genetic disease characterized by extremely elevated triglyceride levels and recurrent acute pancreatitis (AP). Ionis states that If approved, olezarsen has the potential to be the standard of care in the U.S. for the treatment of FCS. In January 2023, the drug has already received Fast Track Designation from the U.S. FDA.

Moreover, in October 2024, Kind Pharmaceuticals LLC announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AND017 in the treatment of Sickle Cell Disease (SCD). AND017 is a first-in-class hemoglobin-elevating agent that targets multiple stages of the red blood cell (RBC) life cycle. Kind Pharmaceuticals states that the orphan drug designation to AND017 underscores the urgent medical need for new therapies, particularly oral drugs to safely and effectively treat patients with SCD.

These increasing Product development activities and orphan drug designations are expected to create significant traction in various therapeutic areas with high unmet needs and propel the overall market growth.

The high cost of orphan drugs may restrain the market growth.

Orphan drugs, although revolutionizing the treatment scenario for several rare diseases, their high cost may restrain the adoption rate, especially among low to middle-income patients. For instance, as per Drugs.com the list price of Spinraza is $750,000 for the first year and then $350,000 in later years. Spinraza is an alternative drug to Zolgensma for the treatment of patients with spinal muscular atrophy (SMA). The same drug costs approximately INR 7 million in India, where the majority of the patients lack proper medical insurance.

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Segment Analysis

The global orphan drugs market is segmented based on product type, therapy area, and region.

Oncology is dominating the therapy area segment of the orphan drugs market with the highest market share.

Oncology is the most widely explored indication for orphan drugs, due to high unmet needs for various types of rare cancers. Majority of the current approved orphan drugs are aimed for rare cancer treatments. For instance below is the list of top-selling orphan drugs with different types of cancers as their major indications. The top 5 selling orphan drugs for oncology accounted for nearly US$24.2 billion in 2023.

Geographical Analysis

North America is expected to hold a significant share of the orphan drugs market.

North America dominates the market for orphan drugs and is expected to show a similar trend over the forecast period. The factors contributing to the region's dominance include the advanced healthcare system, government initiatives, and the strong presence of many market players. The FDA Office of Orphan Products Development approves orphan drug designation for novel drugs and biologics that are used for the safe and effective treatment, diagnosis, and prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows key players to qualify for various incentives, including tax credits for qualified clinical trials and, upon regulatory approval, 7 years of market exclusivity.

Moreover, all the manufacturers of orphan drugs majorly operate in the region, generating higher sales, especially from the U.S. Below is the list of top 5 selling orphan drugs and their revenue share from the U.S.

This represents the dominance of North America, especially the U.S. in the global market for orphan drugs.

Competitive Landscape

The major global players in the orphan drugs market are Johnson & Johnson Services, Inc., AbbVie Inc., Vertex Pharmaceuticals Incorporated, AstraZeneca., Incyte., Biogen., Novartis AG, F. Hoffmann-La Roche Ltd, Pfizer Inc., and Bristol-Myers Squibb Company among others.

Emerging Players

The emerging players in the orphan drugs market include Can-Fite, Mustang Bio, Kind Pharmaceuticals LLC, Cabaletta Bio, Inc. and Ionis Pharmaceuticals, Inc. among others.

Key Developments

• In October 2024, Can-Fite BioPharma Ltd. Announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation for Namodenoson which is indicated for the treatment of pancreatic cancer.
• In April 2024, the European Commission granted orphan medicinal product designation for Zatolmilast developed by Shionogi & Co., Ltd. Zatolmilast is the investigational drug for the treatment of Fragile X syndrome (FXS), which is a leading cause of inherited intellectual disability and autism.

Why Purchase the Report?

• Pipeline & Innovations: Reviews ongoing clinical trials, and Product Type pipelines, and forecasts upcoming pharmaceutical advancements.
• Product Type Performance & Market Positioning: Analyzes Product Type performance, market positioning, and growth potential to optimize strategies.
• Real-World Evidence: Integrates patient feedback and data into Product Type development for improved outcomes.
• Physician Preferences & Health System Impact: Examines healthcare provider behaviors and the impact of health system mergers on adoption strategies.
• Market Updates & Industry Changes: Covers recent regulatory changes, new policies, and emerging technologies.
• Competitive Strategies: Analyzes competitor strategies, market share, and emerging players.
• Pricing & Market Access: Reviews pricing models, reimbursement trends, and market access strategies.
• Market Entry & Expansion: Identifies optimal strategies for entering new markets and partnerships.
• Regional Growth & Investment: Highlights high-growth regions and investment opportunities.
• Supply Chain Optimization: Assesses supply chain risks and distribution strategies for efficient Product Type delivery.
• Sustainability & Regulatory Impact: Focuses on eco-friendly practices and evolving regulations in healthcare.
• Post-market Surveillance: Uses post-market data to enhance Product Type safety and access.
• Pharmacoeconomics & Value-Based Pricing: Analyzes the shift to value-based pricing and data-driven decision-making in R&D.

The global Orphan drugs market report would provide approximately 54 tables, 50 figures, and 179 pages.

Target Audience 2023

• Manufacturers: Pharmaceutical, Biotech Companies, Contract Manufacturers, Distributors, Hospitals.
• Regulatory & Policy: Compliance Officers, Government, Health Economists, Market Access Specialists.
• Technology & Innovation: R&D Professionals, Clinical Trial Managers, Pharmacovigilance Experts.
• Investors: Healthcare Investors, Venture Fund Investors, Pharma Marketing & Sales.
• Consulting & Advisory: Healthcare Consultants, Industry Associations, Analysts.
• Supply Chain: Distribution and Supply Chain Managers.
• Consumers & Advocacy: Patients, Advocacy Groups, Insurance Companies.
• Academic & Research: Academic Institutions.

Table of Contents

1. Methodology and Scope

• 1.1. Research Methodology
• 1.2. Research Objective and Scope of the Report

2. Definition and Overview

3. Executive Summary

• 3.1. Snippet by Product Type
• 3.2. Snippet by Therapy Area
• 3.3. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. Rising Novel Drug Development Activities and Orphan Drug Designations
    • 4.1.1.2. Rising Demand from Unmet Population with Rare Diseases
  • 4.1.2. Restraints
    • 4.1.2.1. High Cost of Treatment
  • 4.1.3. Opportunities
  • 4.1.4. Impact Analysis

5. Industry Analysis

• 5.1. Porter's Five Force Analysis
• 5.2. Supply Chain Analysis
• 5.3. Epidemiology
• 5.4. Pricing Analysis
• 5.5. Pipeline Analysis
• 5.6. Patent Analysis
• 5.7. Regulatory Analysis

6. By Product Type

• 6.1. Introduction
  • 6.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Product Type
  • 6.1.2. Market Attractiveness Index, By Product Type
• 6.2. Small Molecules*
  • 6.2.1. Introduction
  • 6.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 6.3. Biologic Drugs
  • 6.3.1. Gene Therapies
  • 6.3.2. Monoclonal Antibodies
  • 6.3.3. Others
• 6.4. Others

7. By Therapy Area

• 7.1. Introduction
  • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Area
  • 7.1.2. Market Attractiveness Index, By Therapy Area
• 7.2. Oncology*
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 7.3. Hematology Diseases
• 7.4. Central Nervous System
• 7.5. Cardiovascular
• 7.6. Endocrine
• 7.7. Respiratory
• 7.8. Immunologic Conditions
• 7.9. Gastro-Intestinal
• 7.10. Musculoskeletal
• 7.11. Dermatology
• 7.12. Others

8. By Region

• 8.1. Introduction
  • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
  • 8.1.2. Market Attractiveness Index, By Region
• 8.2. North America
  • 8.2.1. Introduction
  • 8.2.2. Key Region-Specific Dynamics
  • 8.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Product Type
  • 8.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Area
  • 8.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.2.5.1. U.S.
    • 8.2.5.2. Canada
    • 8.2.5.3. Mexico
• 8.3. Europe
  • 8.3.1. Introduction
  • 8.3.2. Key Region-Specific Dynamics
  • 8.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Product Type
  • 8.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Area
  • 8.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.3.5.1. Germany
    • 8.3.5.2. U.K.
    • 8.3.5.3. France
    • 8.3.5.4. Spain
    • 8.3.5.5. Italy
    • 8.3.5.6. Rest of Europe
• 8.4. South America
  • 8.4.1. Introduction
  • 8.4.2. Key Region-Specific Dynamics
  • 8.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Product Type
  • 8.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Area
  • 8.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.4.5.1. Brazil
    • 8.4.5.2. Argentina
    • 8.4.5.3. Rest of South America
• 8.5. Asia-Pacific
  • 8.5.1. Introduction
  • 8.5.2. Key Region-Specific Dynamics
  • 8.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Product Type
  • 8.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Area
  • 8.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.5.5.1. China
    • 8.5.5.2. India
    • 8.5.5.3. Japan
    • 8.5.5.4. South Korea
    • 8.5.5.5. Rest of Asia-Pacific
• 8.6. Middle East and Africa
  • 8.6.1. Introduction
  • 8.6.2. Key Region-Specific Dynamics
  • 8.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Product Type
  • 8.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Therapy Area

9. Competitive Landscape

• 9.1. Competitive Scenario
• 9.2. Market Positioning/Share Analysis
• 9.3. Mergers and Acquisitions Analysis

10. Company Profiles

• 10.1. Johnson & Johnson Services, Inc.*
  • 10.1.1. Company Overview
  • 10.1.2. Product Type Portfolio and Description
  • 10.1.3. Financial Overview
  • 10.1.4. Key Developments
• 10.2. AbbVie Inc.
• 10.3. Vertex Pharmaceuticals Incorporated
• 10.4. AstraZeneca.
• 10.5. Incyte.
• 10.6. Biogen.
• 10.7. Novartis AG
• 10.8. F. Hoffmann-La Roche Ltd
• 10.9. Pfizer Inc.
• 10.10. Bristol-Myers Squibb Company

LIST NOT EXHAUSTIVE

11. Appendix

• 11.1. About Us and Services
• 11.2. Contact Us