희귀의약품 서비스 시장 보고서(2026년)
Orphan Drugs Service Global Market Report 2026
상품코드 : 1957554
리서치사 : The Business Research Company
발행일 : On Demand Report
페이지 정보 : 영문 250 Pages
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한글목차

희귀의약품 서비스 시장 규모는 최근 급성장하고 있습니다. 2025년 434억 달러에서 2026년에는 507억 5,000만 달러에 이르고, CAGR 16.9%의 성장이 전망되고 있습니다. 지난 수년간의 성장 요인으로는 희귀질환의 지정 증가, 희귀질환 치료제 법제화 확대, 희귀질환 연구에 대한 투자 증가, 규제적 특혜, 환자 지원 단체의 역할 확대 등을 꼽을 수 있습니다.

향후 몇 년간 희귀의약품 서비스 시장 규모는 급성장이 전망됩니다. 2030년에는 939억 3,000만 달러에 이르고, CAGR은 16.6%를 나타낼 전망입니다. 예측 기간 동안 성장 요인으로는 개인 맞춤형 치료법에 대한 수요 증가, 첨단 임상시험 기술 도입 확대, 세계 희귀질환 치료제 파이프라인 확대, 실제 데이터 활용 확대, 희귀질환 치료제 개발에 대한 지속적인 규제 지원 등을 꼽을 수 있습니다. 예측 기간의 주요 동향으로는 전문 규제 및 개발 서비스에 대한 수요 증가, 신속 승인 프로세스 중시, 데이터 기반 희귀질환 연구 플랫폼 활용 확대, 환자 중심 임상 개발 모델 확대, 이해관계자간 협력 강화 등을 꼽을 수 있습니다.

희귀질환에 대한 투자 증가는 향후 몇 년간 희귀의약품 서비스 시장의 성장을 견인할 것으로 예측됩니다. 희귀질환은 인구의 극히 일부(일반적으로 2,000명 중 1명 미만)에게만 영향을 미치는 질환을 말합니다. 이러한 질환은 만성화 및 진행성 질환인 경우가 많으며, 생명을 위협하거나 신체적 장애를 유발할 수 있습니다. 희귀질환에 대한 투자는 주로 미충족 수요에 대한 인식 증가와 희귀질환 치료제 지정, 시장 독점권, 개발 비용 절감과 같은 규제적 특혜를 통한 큰 수익 가능성에 의해 주도되고 있습니다. 희귀질환에 대한 투자 확대는 연구개발에 대한 자금 지원을 확대하고, 그동안 간과되어 왔던 질환에 대한 치료제 개발을 가능하게 함으로써 희귀질환 치료제 서비스를 강화할 수 있습니다. 임상시험, 규제 당국의 승인, 환자 접근을 지원하여 약물의 보급을 가속화하고 희귀질환 환자들의 치료 옵션을 개선합니다. 예를 들어, 미국 비영리단체 Global Genes에 따르면 2024년 4월 기준 희귀질환 치료제를 개발하는 기업들은 2024년 1분기에 71억 달러의 자금을 조달했습니다. 이는 2023년 같은 기간의 18억 달러에 비해 307% 증가한 수치입니다. 그 결과, 희귀질환에 대한 투자 증가가 희귀의약품 서비스 시장의 성장을 가속하고 있습니다.

희귀질환 치료제 서비스 시장에서 사업을 전개하는 주요 기업들은 희귀질환 치료제 개발 및 보급을 가속화하기 위해 규제 당국의 승인 획득을 최우선 과제로 삼고 있습니다. 규제 당국의 승인은 정부 기관의 공식적인 승인으로, 의약품이나 치료법을 환자에게 합법적으로 시험, 제조, 판매할 수 있는 권한을 부여하는 것입니다. 예를 들어, 2023년 2월 미국 유전자치료제 기업 Askbio GmbH는 사지변형근이영양증(LGMD)에 대한 신규 임상시험 단계의 유전자치료제 'AB-1003'에 대해 유럽위원회로부터 희귀질환치료제 지정을 획득했습니다. 이 아데노부수체 바이러스(AAV) 기반 치료법은 단회 정맥 투여를 통해 근육 세포 내 FKRP 효소 활성을 회복시키는 것을 목표로 합니다. AB-1003은 유럽연합 집행위원회와 미국 식품의약국(FDA)으로부터 희귀질환 치료제 지정을 받았으며, FDA로부터 희귀 소아질환 지정 및 패스트트랙 지정을 받아 미충족 의료 수요의 시급성을 강조하고 있습니다. 현재 유전자 검사로 확진된 성인 환자를 대상으로 제2형 I/R9 사지 띠형 근이영양증(LGMD2I/R9)에 대한 안전성 및 유효성을 평가하기 위한 임상 I/II상(LION-CS101)이 진행 중입니다.

목차

제1장 주요 요약

제2장 시장 특징

제3장 시장 공급망 분석

제4장 세계 시장 동향과 전략

제5장 최종 이용 산업 시장 분석

제6장 시장 : 금리, 인플레이션, 지정학, 무역 전쟁과 관세의 영향, 관세 전쟁과 무역 보호주의의 공급망에 대한 영향, 코로나 팬데믹이 시장에 미치는 영향을 포함한 거시경제 시나리오

제7장 세계 전략 분석 프레임워크, 현재 시장 규모, 시장 비교 및 성장률 분석

제8장 TAM(Total Addressable Market) 규모

제9장 시장 세분화

제10장 지역별/국가별 분석

제11장 아시아태평양 시장

제12장 중국 시장

제13장 인도 시장

제14장 일본 시장

제15장 호주 시장

제16장 인도네시아 시장

제17장 한국 시장

제18장 대만 시장

제19장 동남아시아 시장

제20장 서유럽 시장

제21장 영국 시장

제22장 독일 시장

제23장 프랑스 시장

제24장 이탈리아 시장

제25장 스페인 시장

제26장 동유럽 시장

제27장 러시아 시장

제28장 북미 시장

제29장 미국 시장

제30장 캐나다 시장

제31장 남미 시장

제32장 브라질 시장

제33장 중동 시장

제34장 아프리카 시장

제35장 시장 규제 상황과 투자환경

제36장 경쟁 구도와 기업 개요

제37장 기타 주요 기업 및 혁신 기업

제38장 세계 시장 경쟁 벤치마킹과 대시보드

제39장 주요 인수합병(M&A)

제40장 시장 잠재력이 높은 국가, 부문, 전략

제41장 부록

LSH
영문 목차

영문목차

Orphan drugs service is a specialized area of healthcare and pharmaceutical support that concentrates on supporting the research, development, approval, and accessibility of medications designed to treat rare diseases, which impact only a small and often underserved segment of the population. This service is vital in ensuring that these crucial medications can successfully navigate the complex regulatory and development procedures, ultimately providing access to patients who have few or no alternative treatment options.

The primary categories of orphan drug services include congenital disease, genetic disease, tumors and cancer, chronic conditions, and others. Congenital diseases are medical conditions present from birth, resulting from genetic abnormalities, developmental issues during pregnancy, or environmental factors affecting fetal development. These drugs are utilized across various therapeutic areas, such as oncology, metabolic disorders, neurological disorders, hematology, infectious diseases, and more, and are applicable for both adults and children.

Tariffs are impacting the orphan drugs service market by increasing costs of imported laboratory reagents, clinical trial equipment, data management systems, and specialized pharmaceutical inputs required for rare disease drug development. Service providers in North America and Europe are most affected due to reliance on globally sourced research tools, while Asia-Pacific faces higher costs for export-oriented development services. These tariffs are increasing service delivery costs and extending development timelines. However, they are also encouraging regional service localization, domestic research infrastructure investment, and stronger local regulatory support ecosystems.

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 a 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 $43.4 billion in 2025 to $50.75 billion in 2026 at a compound annual growth rate (CAGR) of 16.9%. The growth in the historic period can be attributed to growth in rare disease identification, expansion of orphan drug legislation, increasing investment in rare disease research, availability of regulatory incentives, rising role of patient advocacy groups.

The orphan drugs service market size is expected to see rapid growth in the next few years. It will grow to $93.93 billion in 2030 at a compound annual growth rate (CAGR) of 16.6%. The growth in the forecast period can be attributed to increasing demand for personalized rare disease therapies, rising adoption of advanced clinical trial technologies, expansion of global orphan drug pipelines, growing use of real-world evidence, continued regulatory support for orphan drug development. Major trends in the forecast period include increasing demand for specialized regulatory and development services, growing focus on accelerated approval pathways, rising use of data-driven rare disease research platforms, expansion of patient-centric clinical development models, enhanced collaboration between stakeholders.

The increasing investment in rare diseases is expected to drive the growth of the orphan drugs service market in the coming years. A rare disease is a medical condition that affects a small portion of the population, generally fewer than 1 in 2,000 individuals. These conditions are often chronic, progressive, and can be life-threatening or disabling. Investment in rare diseases is largely driven by the rising recognition of unmet medical needs and the potential for significant returns through regulatory incentives such as orphan drug designation, market exclusivity, and lower development costs. Rising investment in rare diseases strengthens orphan drug services by providing greater funding for research and development, enabling the creation of therapies for previously overlooked conditions. It speeds up drug availability by supporting clinical trials, regulatory approvals, and patient access, thereby improving treatment options for individuals with rare diseases. For example, in April 2024, according to Global Genes, a US-based nonprofit organization, companies working on drugs for rare diseases raised $7.1 billion in the first quarter of 2024, representing a 307% increase compared to the $1.8 billion raised in the same period in 2023. Consequently, the increasing investment in rare diseases is fueling the growth of the orphan drugs service market.

Major companies operating in the orphan drugs service market are prioritizing obtaining regulatory approvals to accelerate the development and accessibility of treatments for rare diseases. Regulatory approvals are official authorizations from government agencies that permit a drug or therapy to be legally tested, manufactured, and marketed for patient use. For example, in February 2023, Askbio GmbH, a US-based gene therapy company, was granted the European Commission orphan drug designation for AB-1003, a novel investigational gene therapy for limb-girdle muscular dystrophy. This adeno-associated virus (AAV)-based therapy is designed to restore FKRP enzyme activity in muscle cells through a single intravenous infusion. AB-1003 has received orphan drug designation from both the European Commission and the U.S. Food and Drug Administration (FDA), as well as rare pediatric disease and fast track designations from the FDA, emphasizing the urgent unmet medical need. It is currently being evaluated in a Phase 1/2 clinical trial (LION-CS101) to assess safety and efficacy in adults with genetically confirmed limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).

In April 2025, Norgine B.V., a pharmaceutical company based in the Netherlands, acquired Theravia Pharma for an undisclosed sum. Through this acquisition, Norgine aims to strengthen its growth trajectory and rare disease portfolio by broadening its range of transformative therapies for patients with significant unmet medical needs in Europe and ANZ, positioning Norgine as a key partner in rare and specialty pharmaceuticals. Theravia Pharma is a pharmaceutical company based in France that specializes in rare diseases and orphan drug services.

Major companies operating 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., Cevidra Inc.

North America was the largest region in the orphan drugs service market in 2025. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in the orphan drugs service market report are Asia-Pacific, South East Asia, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.

The countries covered in the orphan drugs service market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Taiwan, 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 that 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 Market Global Report 2026 from The Business Research Company provides strategists, marketers and senior management with the critical information they need to assess the market.

This report focuses 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, total addressable market (TAM), market attractiveness score (MAS), competitive landscape, market shares, company scoring matrix, trends and strategies for this market. It traces the market's historic and forecast market growth by geography.

Scope

Added Benefits available all on all list-price licence purchases, to be claimed at time of purchase. Customisations within report scope and limited to 20% of content and consultant support time limited to 8 hours.

Table of Contents

1. Executive Summary

2. Orphan Drugs Service Market Characteristics

3. Orphan Drugs Service Market Supply Chain Analysis

4. Global Orphan Drugs Service Market Trends And Strategies

5. Orphan Drugs Service Market Analysis Of End Use Industries

6. Orphan Drugs Service Market - Macro Economic Scenario Including The Impact Of Interest Rates, Inflation, Geopolitics, Trade Wars and Tariffs, Supply Chain Impact from Tariff War & Trade Protectionism, And Covid And Recovery On The Market

7. Global Orphan Drugs Service Strategic Analysis Framework, Current Market Size, Market Comparisons And Growth Rate Analysis

8. Global Orphan Drugs Service Total Addressable Market (TAM) Analysis for the Market

9. Orphan Drugs Service Market Segmentation

10. Orphan Drugs Service Market Regional And Country Analysis

11. Asia-Pacific Orphan Drugs Service Market

12. China Orphan Drugs Service Market

13. India Orphan Drugs Service Market

14. Japan Orphan Drugs Service Market

15. Australia Orphan Drugs Service Market

16. Indonesia Orphan Drugs Service Market

17. South Korea Orphan Drugs Service Market

18. Taiwan Orphan Drugs Service Market

19. South East Asia Orphan Drugs Service Market

20. Western Europe Orphan Drugs Service Market

21. UK Orphan Drugs Service Market

22. Germany Orphan Drugs Service Market

23. France Orphan Drugs Service Market

24. Italy Orphan Drugs Service Market

25. Spain Orphan Drugs Service Market

26. Eastern Europe Orphan Drugs Service Market

27. Russia Orphan Drugs Service Market

28. North America Orphan Drugs Service Market

29. USA Orphan Drugs Service Market

30. Canada Orphan Drugs Service Market

31. South America Orphan Drugs Service Market

32. Brazil Orphan Drugs Service Market

33. Middle East Orphan Drugs Service Market

34. Africa Orphan Drugs Service Market

35. Orphan Drugs Service Market Regulatory and Investment Landscape

36. Orphan Drugs Service Market Competitive Landscape And Company Profiles

37. Orphan Drugs Service Market Other Major And Innovative Companies

38. Global Orphan Drugs Service Market Competitive Benchmarking And Dashboard

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

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

41. Appendix

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