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Global Orphan Drugs Market Analysis & Forecast 2024-2034: Market By Drug Type; By Therapy; By Distribution Channel; and By Region

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    Report

  • 145 Pages
  • October 2023
  • Region: Global
  • Erevna Healthcare
  • ID: 5890365
The global orphan drugs market size was estimated to be USD 177.01 billion in 2023 and is expected to reach at USD 638.47 billion by 2034 with a CAGR of 12.37% during the forecast period 2024-2034. Surge in research & development initiatives, rising investment for the development of innovative products, increasing awareness regarding rare diseases, surge in number of clinical trial procedures, and growing approvals of innovative orphan drugs by regulatory bodies are some of the key factors boosting the market growth.

Growing approvals of innovative orphan drugs by regulatory bodies is predicted to boost the market growth during the forecast period. The occurrence of rare diseases in the global population has been on the rise. To address this challenge, both developed and developing nations have established policies that support the advancement of medications for rare diseases and ensure their accessibility to patients. For instance, in March 2022, Ztalmy (ganaxolone) from Marinus Pharmaceuticals has been given U.S. FDA approval to treat seizures in individuals two years of age and older who have a rare cyclin-dependent kinase-like 5 (CDKL5) deficient condition (CDD).

By drug type, biologics was the highest revenue-grossing segment in the global orphan drugs market in 2023 owing to the existence of offerings labelled as biologics, rising mergers & acquisitions within market players, and increasing regulatory approvals. For instance, in June 2022, The Food and Drug Administration (FDA) has awarded orphan drug designation to evorpacept, a cutting-edge CD47 blocker developed by ALX Oncology Holdings Inc., for its potential use in treating individuals diagnosed with acute myeloid leukemia (AML). Additionally, non-biologics is predicted to grow at fastest CAGR during the forecast period owing to the growing research & development activities and increasing number of clinical trial procedures.

By therapy, oncology was the highest revenue-grossing segment in the global orphan drugs market in 2023 owing to growing research & development activities, presence of various oncology drugs, and surge in approvals of novel drugs. For instance, in February 2022, The U.S. FDA has approved CTI BioPharma's medication Pacritinib for the treatment of adult patients with myelofibrosis, a rare bone cancer that affects over 21,000 individuals in the country. Additionally, hematology is predicted to grow at fastest CAGR during the forecast period owing to the increasing number of product launches and growing approvals by regulatory bodies.

By distribution channel, hospital pharmacies was the highest revenue-grossing segment in the global orphan drugs market in 2023 owing to a large number of medications must be given intravenously by qualified medical personnel in hospitals and surge in approvals by regulatory bodies. For instance, in March 2022, The Food and Drug Administration (FDA) awarded orphan drug classification to Intellia Therapeutics' CRISPR-edited T cell receptor (TCR) T-cell therapy, which is being tested for the treatment of acute myeloid leukemia (AML). Additionally, online pharmacies is predicted to grow at fastest CAGR during the forecast period owing to the increasing numbers of individuals are purchasing these medications from internet pharmacies.

North America region is anticipated for the highest revenue share during the forecast period owing to the rising expenditures on orphan drugs, increasing focus on development of innovative products, and surge in mergers & acquisitions within market players. For instance, in November 2022, AstraZeneca subsidiary Alexion, which focuses on rare illnesses, has acquired LogicBio Therapeutics, Inc., a pioneer in the field of genomic medicine. This purchase was undertaken to hasten Alexion's advancement in genomic medicine and the treatment of uncommon illnesses. Additionally, Asia Pacific region is predicted to grow at fastest CAGR during the forecast period owing to the growing healthcare expenditures, rising awareness regarding rare diseases, and increasing partnerships within market players. For instance, in December 2022, Amgen Inc. made the announcement that it will buy Horizon Therapeutics Plc and its orphan medication portfolio for USD 27.8 billion.

Segmentation: Orphan Drugs Market Report 2022 - 2033

Orphan Drugs Market Analysis & Forecast by Drug Type 2023 - 2034 (Revenue USD Bn)

  • Non-Biologics
  • Biologics

Orphan Drugs Market Analysis & Forecast by Therapy 2023 - 2034 (Revenue USD Bn)

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

Orphan Drugs Market Analysis & Forecast by Distribution Channel 2023 - 2034 (Revenue USD Bn)

  • Online Pharmacies
  • Hospital Pharmacies
  • Retail Pharmacies

Orphan Drugs Market Analysis & Forecast by Region 2023 - 2034 (Revenue USD Bn)

North America

  • U.S.
  • Canada

Europe

  • Germany
  • France
  • UK
  • Spain
  • Italy
  • Rest of Europe

Asia Pacific

  • China
  • Japan
  • India
  • Australia
  • South Korea
  • Rest of APAC

Latin America

  • Brazil
  • Mexico
  • Argentina
  • Rest of LATAM

Middle East & Africa

  • South Africa
  • GCC
  • Rest of MEA

Table of Contents

1. Research Methodology
1.1. Study Objectives
1.2. Study Scope
1.3. Research Assumptions
1.4. Research Framework
2. Introduction
2.1. Market Definition
2.2. Global Orphan Drugs Market Overview
3. Executive Summary
4. Market Environment Analysis
4.1. Porter’s 5 Forces Analysis
4.2. PESTEL Analysis
4.3. SWOT Analysis
5. Market Dynamics
5.1. Drivers Analysis
5.2. Restraints Analysis
5.3. Opportunities Analysis
5.4. Threats Analysis
5.5. Trend Analysis
6. COVID-19 Impact Analysis
7. Orphan Drugs Market: Drug Type Estimates & Trend Analysis
7.1. Drug Type Segment Opportunity Analysis
7.2. Non-Biologics
7.2.1. Non-Biologics Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
7.3. Biologics
7.3.1. Biologics Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
8. Orphan Drugs Market: Therapy Estimates & Trend Analysis
8.1. Therapy Segment Opportunity Analysis
8.2. Neurology
8.2.1. Neurology Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
8.3. Cardiovascular
8.3.1. Cardiovascular Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
8.4. Oncology
8.4.1. Oncology Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
8.5. Hematology
8.5.1. Hematology Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
8.6. Endocrinology
8.6.1. Endocrinology Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
8.7. Immunotherapy
8.7.1. Immunotherapy Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
8.8. Infectious Diseases
8.8.1. Infectious Diseases Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
8.9. Respiratory
8.9.1. Respiratory Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
8.10. Others
8.10.1. Others Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
9. Orphan Drugs Market: Distribution Channel Estimates & Trend Analysis
9.1. Distribution Channel Segment Opportunity Analysis
9.2. Online Pharmacies
9.2.1. Online Pharmacies Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
9.3. Hospital Pharmacies
9.3.1. Hospital Pharmacies Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
9.4. Retail Pharmacies
9.4.1. Retail Pharmacies Market Analysis & Forecast, 2023-2034 (Revenue, USD Bn)
10. Regional Market Analysis
10.1. Regional Market Opportunity Analysis
11. North America Orphan Drugs Market
11.1. North America Orphan Drugs Market
11.1.1. North America Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
11.1.2. North America Orphan Drugs Market Size and Forecast, By Country, 2023-2034 (Revenue USD Bn)
11.1.3. North America Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
11.1.4. North America Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
11.1.5. North America Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
11.2. U.S. Global Orphan Drugs Market
11.2.1. U.S. Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
11.2.2. U.S. Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
11.2.3. U.S. Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
11.2.4. U.S. Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
11.3. Canada Global Orphan Drugs Market
11.3.1. Canada Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
11.3.2. Canada Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
11.3.3. Canada Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
11.3.4. Canada Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
12. Europe Global Orphan Drugs Market
12.1. Europe Global Orphan Drugs Market
12.1.1. Europe Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
12.1.2. Europe Orphan Drugs Market Size and Forecast, By Country, 2023-2034 (Revenue USD Bn)
12.1.3. Europe Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
12.1.4. Europe Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
12.1.5. Europe Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
12.2. Germany Global Orphan Drugs Market
12.2.1. Germany Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
12.2.2. Germany Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
12.2.3. Germany Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
12.2.4. Germany Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
12.3. UK Global Orphan Drugs Market
12.3.1. UK Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
12.3.2. UK Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
12.3.3. UK Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
12.3.4. UK Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
12.4. France Global Orphan Drugs Market
12.4.1. France Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
12.4.2. France Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
12.4.3. France Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
12.4.4. France Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
12.5. Spain Global Orphan Drugs Market
12.5.1. Spain Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
12.5.2. Spain Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
12.5.3. Spain Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
12.5.4. Spain Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
12.6. Italy Global Orphan Drugs Market
12.6.1. Italy Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
12.6.2. Italy Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
12.6.3. Italy Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
12.6.4. Italy Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
12.7. Rest of Europe Global Orphan Drugs Market
12.7.1. Rest of Europe Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
12.7.2. Rest of Europe Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
12.7.3. Rest of Europe Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
12.7.4. Rest of Europe Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
13. Asia Pacific Global Orphan Drugs Market
13.1. Asia Pacific Global Orphan Drugs Market
13.1.1. Asia Pacific Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
13.1.2. Asia Pacific Orphan Drugs Market Size and Forecast, By Country, 2023-2034 (Revenue USD Bn)
13.1.3. Asia Pacific Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
13.1.4. Asia Pacific Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
13.1.5. Asia Pacific Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
13.2. Japan Global Orphan Drugs Market
13.2.1. Japan Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
13.2.2. Japan Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
13.2.3. Japan Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
13.2.4. Japan Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
13.3. China Global Orphan Drugs Market
13.3.1. China Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
13.3.2. China Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
13.3.3. China Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
13.3.4. China Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
13.4. India Global Orphan Drugs Market
13.4.1. India Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
13.4.2. India Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
13.4.3. India Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
13.4.4. India Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
13.5. South Korea Global Orphan Drugs Market
13.5.1. South Korea Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
13.5.2. South Korea Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
13.5.3. South Korea Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
13.5.4. South Korea Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
13.6. Australia Global Orphan Drugs Market
13.6.1. Australia Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
13.6.2. Australia Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
13.6.3. Australia Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
13.6.4. Australia Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
13.7. Rest of Asia Pacific Global Orphan Drugs Market
13.7.1. Rest of Asia Pacific Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
13.7.2. Rest of Asia Pacific Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
13.7.3. Rest of Asia Pacific Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
13.7.4. Rest of Asia Pacific Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
14. Latin America Global Orphan Drugs Market
14.1. Latin America Global Orphan Drugs Market
14.1.1. Latin America Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
14.1.2. Latin America Orphan Drugs Market Size and Forecast, By Country, 2023-2034 (Revenue USD Bn)
14.1.3. Latin America Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
14.1.4. Latin America Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
14.1.5. Latin America Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
14.2. Brazil Global Orphan Drugs Market
14.2.1. Brazil Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
14.2.2. Brazil Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
14.2.3. Brazil Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
14.2.4. Brazil Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
14.3. Mexico Global Orphan Drugs Market
14.3.1. Mexico Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
14.3.2. Mexico Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
14.3.3. Mexico Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
14.3.4. Mexico Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
14.4. Argentina Global Orphan Drugs Market
14.4.1. Argentina Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
14.4.2. Argentina Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
14.4.3. Argentina Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
14.4.4. Argentina Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
14.5. Rest of Latin America Global Orphan Drugs Market
14.5.1. Rest of Latin America Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
14.5.2. Rest of Latin America Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
14.5.3. Rest of Latin America Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
14.5.4. Rest of Latin America Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
15. MEA Global Orphan Drugs Market
15.1. MEA Global Orphan Drugs Market
15.1.1. MEA Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
15.1.2. MEA Orphan Drugs Market Size and Forecast, By Country, 2023-2034 (Revenue USD Bn)
15.1.3. MEA Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
15.1.4. MEA Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
15.1.5. MEA Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
15.2. GCC Global Orphan Drugs Market
15.2.1. GCC Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
15.2.2. GCC Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
15.2.3. GCC Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
15.2.4. GCC Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
15.3. South Africa Global Orphan Drugs Market
15.3.1. South Africa Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
15.3.2. South Africa Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
15.3.3. South Africa Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
15.3.4. South Africa Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
15.4. Rest of MEA Global Orphan Drugs Market
15.4.1. Rest of MEA Orphan Drugs Market Size and Forecast, 2023-2034 (Revenue USD Bn)
15.4.2. Rest of MEA Orphan Drugs Market Size and Forecast, By Drug Type, 2023-2034 (Revenue USD Bn)
15.4.3. Rest of MEA Orphan Drugs Market Size and Forecast, By Therapy, 2023-2034 (Revenue USD Bn)
15.4.4. Rest of MEA Orphan Drugs Market Size and Forecast, By Distribution Channel, 2023-2034 (Revenue USD Bn)
16. Competitor Analysis
16.1. Company Market Share Analysis, 2023
16.2. Major Recent Developments
17. Company Profiles
17.1. Bristol-Myers Squibb Company
17.2. Amryt Pharma Plc.
17.3. Glaxosmithkline Plc (Gsk)
17.4. Johnson & Johnson (Janssen Global Services, Llc)
17.5. Sanofi S.A
17.6. Novartis International Ag (Sandoz)
17.7. Pfizer Inc.
17.8. Abbvie Inc.
17.9. F. Hoffmann-La Roche Ltd.
17.10. Amgen Inc.
17.11. Other Prominent Players
18. Conclusion19. Recommendations

Companies Mentioned

  • Bristol-Myers Squibb Company
  • Amryt Pharma Plc.
  • Glaxosmithkline Plc (Gsk)
  • Johnson & Johnson (Janssen Global Services LLC)
  • Sanofi S.A
  • Novartis International Ag (Sandoz)
  • Pfizer Inc.
  • Abbvie Inc.
  • F. Hoffmann-La Roche Ltd.
  • Amgen Inc.

Table Information