세계의 희귀의약품 서비스 시장 보고서(2025년)
Orphan Drugs Service Global Market Report 2025
상품코드 : 1855941
리서치사 : The Business Research Company
발행일 : On Demand Report
페이지 정보 : 영문 250 Pages
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한글목차

희귀의약품 서비스 시장 규모는 최근 급속히 확대되고 있습니다. 2024년 370억 5,000만 달러에서 2025년에는 434억 달러에 달하고, CAGR 17.1%를 나타낼 전망입니다. 희귀질환의 유병률 증가, 정부 지원 증가, 환자 지원 이니셔티브 확대, 암멧 의료 요구에 대한 주목 증가, 의료 제공업체와 연구자의 협력 관계 증가 등이 이 기간의 성장을 견인했습니다.

희귀의약품 서비스 시장 규모는 향후 수년간 급성장할 것으로 전망됩니다. 2029년에는 CAGR 16.7%로 806억 2,000만 달러로 성장할 전망입니다. 예측 기간 동안의 성장은 바이오 의약품 연구에 대한 투자 증가, 정밀의료에 대한 수요 증가, 첨단 임상시험 디자인 채택 확대, 벤처 캐피탈의 자금 조달 증가, 세계 규제 조화에 중점화 등이 배경에 있습니다. 예측 기간의 주요 동향은 창약 플랫폼의 진보, 세포 및 유전자 치료 서비스의 혁신, 임상시험에 있어서의 인공지능의 통합, 바이오마커에 근거한 환자 선택의 발전, 환자 모니터링을 위한 디지털 헬스 솔루션의 채용 등을 들 수 있습니다.

희귀질환에 대한 투자 증가는 향후 수년간 희귀의약품 서비스 시장의 성장을 가속할 것으로 예측됩니다. 희귀질환이란 인구의 극히 일부, 일반적으로 2,000명 중 1명 미만이 병에 걸리는 건강 상태입니다. 이러한 질병은 종종 만성적이고 진행성이며 생명을 위협하거나 장애를 일으킬 수 있습니다. 투자 증가는 암메트 메디컬 니즈가 인식되게 되어 희귀질환용 의약품의 지정, 시장 독점권, 개발 비용의 저감 등 규제상의 인센티브에 힘입어 큰 수익을 기대할 수 있게 된 것이 주요 요인입니다. 투자 증가는 연구개발을 위한 추가 자금을 제공하고, 지금까지 간과되었던 질환에 대한 치료법의 창출을 가능하게 함으로써, 희귀질환용 의약품(오펀드래그) 서비스를 향상시킵니다. 또한 임상시험, 규제 당국의 승인, 환자 접근 개선 등을 지원함으로써 의약품 공급을 가속화하고 희귀질환 환자의 치료 옵션을 확대합니다. 예를 들어 미국에 본사를 둔 비영리단체 세계 청바지는 2024년 4월 희귀질환 치료제에 주력하는 기업이 2024년 1분기에 71억 달러를 조달했다고 보고했으며, 이는 2023년 동시기 조달액 18억 달러에서 307%라는 대폭적인 성장을 보였습니다. 그 결과, 희귀질환에 대한 투자 확대가 희귀의약품 서비스 서비스 시장 확대에 박차를 가하고 있습니다.

희귀의약품 서비스 시장의 기업은 희귀질환 치료법의 개발과 이용을 촉진하기 위해 규제 당국의 승인 취득에 점점 주력하고 있습니다. 약사 승인은 의약품 및 치료법의 합법적인 시험, 제조 및 판매에 필수적입니다. 예를 들어, 2023년 2월 미국의 유전자 치료 기업인 Askbio GmbH는 사지대상근 이영양증에 대한 신규 유전자 치료제 AB-1003에 대해 유럽 위원회로부터 희귀질환용 의약품의 지정을 받았습니다. 이 아데노 관련 바이러스(AAV) 기반 치료제는 단일 정맥 주사로 근육 세포에서 FKRP 효소 활성을 회복시키는 것을 목표로합니다. AB-1003은 미국 식품의약국(FDA)에 의해 희귀소아질환 및 패스트트랙 지정과 함께 희귀질환용 의약품(희귀의약품)으로 지정되어 있으며, 미충족 요구의 중요성을 뒷받침하고 있습니다. 본 치료법은 현재 유전적으로 확인된 사지대형 근이영양증 2I/R9형(LGMD2I/R9)의 성인을 대상으로 안전성과 효능을 평가하는 제1/2상 임상시험(LION-CS101)을 실시 중입니다.

목차

제1장 주요 요약

제2장 시장 특징

제3장 시장 동향과 전략

제4장 시장 : 금리, 인플레이션, 지정학, 무역전쟁과 관세, 그리고 코로나 및 회복이 시장에 미치는 영향을 포함한 거시경제 시나리오

제5장 세계의 성장 분석과 전략 분석 프레임워크

제6장 시장 세분화

제7장 지역별/국가별 분석

제8장 아시아태평양 시장

제9장 중국 시장

제10장 인도 시장

제11장 일본 시장

제12장 호주 시장

제13장 인도네시아 시장

제14장 한국 시장

제15장 서유럽 시장

제16장 영국 시장

제17장 독일 시장

제18장 프랑스 시장

제19장 이탈리아 시장

제20장 스페인 시장

제21장 동유럽 시장

제22장 러시아 시장

제23장 북미 시장

제24장 미국 시장

제25장 캐나다 시장

제26장 남미 시장

제27장 브라질 시장

제28장 중동 시장

제29장 아프리카 시장

제30장 경쟁 구도와 기업 프로파일

제31장 기타 주요 기업 및 혁신 기업

제32장 세계 시장 경쟁 벤치마킹과 대시보드

제33장 주요 인수합병(M&A)

제34장 최근 시장 동향

제35장 시장의 잠재력이 높은 국가, 부문, 전략

제36장 부록

KTH
영문 목차

영문목차

Orphan drug services refer to specialized healthcare and pharmaceutical support focused on facilitating the research, development, approval, and accessibility of medications for rare diseases, which affect a small and often underserved portion of the population. These services play a critical role in navigating complex regulatory and development processes, ensuring that essential therapies reach patients who have limited or no alternative treatment options.

The primary types of orphan drug services include congenital disease, genetic disease, tumors and cancer, chronic conditions, and others. Congenital diseases refer to medical conditions present from birth, caused by genetic abnormalities, developmental issues during pregnancy, or environmental factors affecting fetal development. These services are applied across therapeutic areas such as oncology, metabolic disorders, neurological disorders, hematology, infectious diseases, and others, serving both adult and pediatric patient populations.

Note that the outlook for this market is being affected by rapid changes in trade relations and tariffs globally. The report will be updated prior to delivery to reflect the latest status, including revised forecasts and quantified impact analysis. The report's Recommendations and Conclusions sections will be updated to give strategies for entities dealing with the fast-moving international environment.

The sudden escalation of U.S. tariffs and the consequent trade frictions in spring 2025 are severely impacting the pharmaceutical companies contend with tariffs on APIs, glass vials, and lab equipment inputs with few alternative sources. Generic drug makers, operating on razor-thin margins, are especially vulnerable, with some reducing production of low-profit medicines. Biotech firms face delays in clinical trials due to tariff-related shortages of specialized reagents. In response, the industry is expanding API production in India and Europe, increasing inventory stockpiles, and pushing for trade exemptions for essential medicines.

The orphan drugs service market research report is one of a series of new reports from The Business Research Company that provides orphan drugs service market statistics, including orphan drugs service industry global market size, regional shares, competitors with an orphan drugs service market share, detailed orphan drugs service market segments, market trends and opportunities, and any further data you may need to thrive in the orphan drugs service industry. This orphan drugs service market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.

The orphan drugs service market size has grown rapidly in recent years. It will grow from $37.05 billion in 2024 to $43.40 billion in 2025 at a compound annual growth rate (CAGR) of 17.1%. The growth during the historic period was driven by increasing prevalence of rare diseases, rising government support, expanding patient advocacy initiatives, growing focus on unmet medical needs, and increasing collaboration between healthcare providers and researchers.

The orphan drugs service market size is expected to see rapid growth in the next few years. It will grow to $80.62 billion in 2029 at a compound annual growth rate (CAGR) of 16.7%. The growth during the forecast period is fueled by rising investment in biopharmaceutical research, growing demand for precision medicine, expanding adoption of advanced clinical trial designs, increasing venture capital funding, and greater emphasis on global regulatory harmonization. The primary trends in the forecast period include advancements in drug discovery platforms, innovation in cell and gene therapy services, integration of artificial intelligence in clinical trials, developments in biomarker-based patient selection, and adoption of digital health solutions for patient monitoring.

The increasing investment in rare diseases is expected to drive the growth of the orphan drugs service market in the coming years. Rare diseases are health conditions that affect a small portion of the population, typically fewer than 1 in 2,000 people. These conditions are often chronic, progressive, and can be life-threatening or disabling. The rise in investment is primarily driven by the growing recognition of unmet medical needs and the potential for substantial returns, supported by regulatory incentives such as orphan drug designation, market exclusivity, and lower development costs. Increased investment enhances orphan drug services by providing additional funding for research and development, enabling the creation of therapies for previously overlooked conditions. It accelerates the availability of drugs by supporting clinical trials, regulatory approvals, and improving patient access, thereby expanding treatment options for those with rare diseases. For example, in April 2024, Global Genes, a US-based nonprofit organization, reported that companies focused on rare disease drugs raised $7.1 billion in the first quarter of 2024, a significant 307% increase from the $1.8 billion raised during the same period in 2023. As a result, the growing investment in rare diseases is fueling the expansion of the orphan drugs service market.

Companies in the orphan drugs service market are increasingly focused on obtaining regulatory approvals to expedite the development and availability of treatments for rare diseases. Regulatory approvals are essential for legal testing, manufacturing, and marketing of drugs and treatments. For example, in February 2023, Askbio GmbH, a US-based gene therapy company, received orphan drug designation from the European Commission for AB-1003, a novel gene therapy for limb-girdle muscular dystrophy. This adeno-associated virus (AAV)-based therapy aims to restore FKRP enzyme activity in muscle cells through a single intravenous infusion. AB-1003 has also been granted orphan drug status by the U.S. Food and Drug Administration (FDA), along with rare pediatric disease and fast track designations, underscoring the critical unmet need. The therapy is currently undergoing a Phase 1/2 clinical trial (LION-CS101) to evaluate its safety and efficacy in adults with genetically confirmed limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).

In April 2025, Norgine B.V., a Netherlands-based pharmaceutical company, acquired Theravia Pharma for an undisclosed amount. This acquisition allows Norgine to strengthen its rare disease portfolio and accelerate its growth in the rare and specialty pharmaceuticals sector. By expanding its range of life-changing therapies for patients with high unmet medical needs in Europe and the Asia-Pacific region (ANZ), Norgine aims to become a leading partner in rare diseases. Theravia Pharma, based in France, specializes in rare diseases and orphan drug services.

Major players in the orphan drugs service market are Johnson & Johnson Services Inc., F. Hoffmann-La Roche Ltd., Pfizer Inc., AbbVie Inc., Bayer AG, Sanofi S.A., Novartis AG, AstraZeneca PLC, The Bristol-Myers Squibb Company, GSK plc., Eli Lilly and Company, Takeda Pharmaceutical Company Limited, Amgen Inc., IQVIA Inc., CSL Behring GmbH, Lonza Group AG, BioMarin Pharmaceutical Inc., Fortrea Holdings Inc., Medpace Inc., Sarepta Therapeutics, Caidya Inc., and Cevidra Inc.

North America was the largest region in the orphan drugs service market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in orphan drugs service report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.

The countries covered in the orphan drugs service market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

The orphan drugs service market includes revenues earned by entities through clinical trial management, pharmacovigilance and safety monitoring, distribution and supply chain services, patient support and access programs, and data management and biostatistics services. The market value includes the value of related goods sold by the service provider or included within the service offering. Only goods and services traded between entities or sold to end consumers are included.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD unless otherwise specified).

The revenues for a specified geography are consumption values and are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

Orphan Drugs Service Global Market Report 2025 from The Business Research Company provides strategists, marketers and senior management with the critical information they need to assess the market.

This report focuses on orphan drugs service market which is experiencing strong growth. The report gives a guide to the trends which will be shaping the market over the next ten years and beyond.

Reasons to Purchase

Where is the largest and fastest growing market for orphan drugs service ? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward, including technological disruption, regulatory shifts, and changing consumer preferences? The orphan drugs service market global report from the Business Research Company answers all these questions and many more.

The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market's historic and forecast market growth by geography.

The forecasts are made after considering the major factors currently impacting the market. These include the technological advancements such as AI and automation, Russia-Ukraine war, trade tariffs (government-imposed import/export duties), elevated inflation and interest rates.

Scope

Table of Contents

1. Executive Summary

2. Orphan Drugs Service Market Characteristics

3. Orphan Drugs Service Market Trends And Strategies

4. Orphan Drugs Service Market - Macro Economic Scenario Including The Impact Of Interest Rates, Inflation, Geopolitics, Trade Wars and Tariffs, And Covid And Recovery On The Market

5. Global Orphan Drugs Service Growth Analysis And Strategic Analysis Framework

6. Orphan Drugs Service Market Segmentation

7. Orphan Drugs Service Market Regional And Country Analysis

8. Asia-Pacific Orphan Drugs Service Market

9. China Orphan Drugs Service Market

10. India Orphan Drugs Service Market

11. Japan Orphan Drugs Service Market

12. Australia Orphan Drugs Service Market

13. Indonesia Orphan Drugs Service Market

14. South Korea Orphan Drugs Service Market

15. Western Europe Orphan Drugs Service Market

16. UK Orphan Drugs Service Market

17. Germany Orphan Drugs Service Market

18. France Orphan Drugs Service Market

19. Italy Orphan Drugs Service Market

20. Spain Orphan Drugs Service Market

21. Eastern Europe Orphan Drugs Service Market

22. Russia Orphan Drugs Service Market

23. North America Orphan Drugs Service Market

24. USA Orphan Drugs Service Market

25. Canada Orphan Drugs Service Market

26. South America Orphan Drugs Service Market

27. Brazil Orphan Drugs Service Market

28. Middle East Orphan Drugs Service Market

29. Africa Orphan Drugs Service Market

30. Orphan Drugs Service Market Competitive Landscape And Company Profiles

31. Orphan Drugs Service Market Other Major And Innovative Companies

32. Global Orphan Drugs Service Market Competitive Benchmarking And Dashboard

33. Key Mergers And Acquisitions In The Orphan Drugs Service Market

34. Recent Developments In The Orphan Drugs Service Market

35. Orphan Drugs Service Market High Potential Countries, Segments and Strategies

36. Appendix

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