희귀질환 의약품 서비스 시장 보고서(2026년)
Rare Disease Pharmaceutical Service Global Market Report 2026
상품코드 : 1957733
리서치사 : The Business Research Company
발행일 : On Demand Report
페이지 정보 : 영문 250 Pages
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한글목차

희귀질환용 의약품 서비스 시장 규모는 최근 급성장하고 있습니다. 2025년 1,334억 달러에서 2026년에는 1,509억 6,000만 달러에 이르고, CAGR 13.2%의 성장이 전망되고 있습니다. 지난 수년간의 성장 요인으로는 희귀질환 치료제 개발 프로그램 확대, 희귀질환 진단율 향상, 임상연구 인프라 확충, 희귀질환에 대한 규제 혜택 증가, 전문 서비스 제공업체 진입 확대 등을 꼽을 수 있습니다.

희귀질환 의약품 서비스 시장 규모는 향후 몇 년간 급성장이 전망됩니다. 2030년에는 2,451억 4,000만 달러에 이르고, CAGR은 12.9%를 나타낼 전망입니다. 예측 기간 동안의 성장에는 희귀질환 개발 활동의 아웃소싱 증가, 디지털 임상시험 플랫폼 도입 확대, 세계 희귀질환 파이프라인 확대, 리얼월드 데이터(RWE) 생성에 대한 관심 증가, 전문적 규제 지식에 대한 수요 증가 등이 주요 요인으로 작용할 것으로 예측됩니다. 예측 기간의 주요 동향으로는 통합형 희귀질환 서비스 수요 증가, 엔드투엔드 의약품 개발 지원에 대한 관심 증가, 환자 중심 치료 모델에 대한 관심 확대, 규제 및 시장 접근 서비스 확대, 데이터 기반 임상 관리 활용 강화 등을 꼽을 수 있습니다.

개인 맞춤형 의료의 도입 확대는 향후 희귀질환 의약품 서비스 시장의 성장을 견인할 것으로 예측됩니다. 개인 맞춤형 의료란 개인의 유전자 프로파일을 기반으로 설계된 치료법을 의미하며, 효능을 높이고 부작용을 최소화하는 치료법을 말합니다. 유전체학, 바이오마커 연구, 데이터 분석의 발전으로 맞춤의료의 성장이 가속화되고 있으며, 기존의 획일적인 접근 방식보다 환자의 유전적 구성에 맞는 맞춤형 치료를 통해 치료 결과를 개선하고, 부작용을 줄이며, 미충족 수요를 보다 정밀하게 충족시킬 수 있습니다. 의약품 서비스는 표적치료제 개발, 유전자 및 바이오마커 정보 활용, 환자 개개인의 특정 니즈에 맞는 맞춤형 치료 솔루션 제공을 통해 개인 맞춤형 의료를 지원하고 있습니다. 예를 들어, 미국 비영리단체인 '맞춤의료연합'에 따르면, 2024년 2월 현재 FDA는 2023년 희귀질환 환자를 위한 16건의 새로운 맞춤치료제를 승인했습니다. 이는 2022년 6건에서 증가한 수치입니다. 이러한 맞춤형 의료의 부상은 희귀질환 의약품 서비스 시장의 성장을 가속하고 있습니다.

희귀질환 의약품 서비스 시장에서 사업을 전개하는 주요 기업들은 치료 효과 향상, 환자 결과 개선, 희귀 신경질환 및 대사성 질환의 미충족 수요에 대응하기 위해 아세틸로이신과 같은 혁신적인 치료 전략 개발에 집중하고 있습니다. 아세틸로이신(변형 아미노산: N-아세틸-L-로이신)은 운동기능, 평형감각, 신경학적 건강상태 유지를 목적으로 하는 치료제로서 해당 환자들에게 도움을 주고 있습니다. 예를 들어, 2024년 9월 미국 소재 바이오 제약사 인트라바이오(IntraBio)는 희귀하고 생명을 위협하는 신경퇴행성 질환인 니만스픽병 C형에 대한 최초의 단독 치료제 'AQNEURSA'에 대해 FDA의 승인을 받았습니다. 이 치료법은 신경증상을 신속하고 유의미하게 개선하고, 희귀질환을 앓고 있는 소아 및 성인 환자 모두에게 획기적인 치료 옵션을 제공했다는 점에서 주목할 만한 성과라고 할 수 있습니다.

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

A rare disease pharmaceutical service involves the specialized research, development, and provision of medicines for conditions impacting a very small group of patients. It concentrates on meeting unmet medical needs by offering innovative treatments, regulatory assistance, and patient care solutions. These services guarantee accessibility, affordability, and consistent therapy for individuals affected by rare diseases.

The primary types of rare disease pharmaceutical services encompass drug discovery, clinical trials, and other related activities. Drug discovery involves the systematic use of these materials to identify, optimize, and validate potential therapeutic candidates. Its various applications include congenital and genetic disorders, tumors and cancers, and endocrine diseases, and these services are employed by pharmaceutical companies, biotechnology firms, academic and research institutions, hospitals and specialty clinics, contract research organizations (CROs) and contract development and manufacturing organizations (CDMOs), as well as diagnostic laboratories and genetic testing centers.

Tariffs are impacting the rare disease pharmaceutical service market by increasing costs of imported laboratory equipment, clinical trial supplies, data management systems, and manufacturing inputs used across research and development services. Pharmaceutical and biotechnology companies in North America and Europe are most affected due to reliance on global service supply chains, while Asia-Pacific faces cost pressures in service exports and manufacturing support. These tariffs are increasing service pricing and project costs. However, they are also encouraging regional service hubs, localized manufacturing support, and investment in domestic pharmaceutical service ecosystems.

The rare disease pharmaceutical service market research report is one of a series of new reports from The Business Research Company that provides rare disease pharmaceutical service market statistics, including rare disease pharmaceutical service industry global market size, regional shares, competitors with a rare disease pharmaceutical service market share, detailed rare disease pharmaceutical service market segments, market trends and opportunities, and any further data you may need to thrive in the rare disease pharmaceutical service industry. This rare disease pharmaceutical 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 rare disease pharmaceutical service market size has grown rapidly in recent years. It will grow from $133.4 billion in 2025 to $150.96 billion in 2026 at a compound annual growth rate (CAGR) of 13.2%. The growth in the historic period can be attributed to growth in orphan drug development programs, increasing rare disease diagnosis rates, expansion of clinical research infrastructure, rising regulatory incentives for rare diseases, growing involvement of specialized service providers.

The rare disease pharmaceutical service market size is expected to see rapid growth in the next few years. It will grow to $245.14 billion in 2030 at a compound annual growth rate (CAGR) of 12.9%. The growth in the forecast period can be attributed to increasing outsourcing of rare disease development activities, rising adoption of digital clinical trial platforms, expansion of global rare disease pipelines, growing focus on real-world evidence generation, increasing demand for specialized regulatory expertise. Major trends in the forecast period include increasing demand for integrated rare disease services, rising focus on end-to-end drug development support, growing emphasis on patient-centric care models, expansion of regulatory and market access services, enhanced use of data-driven clinical management.

The increasing adoption of personalized medicine is expected to drive the growth of the rare disease pharmaceutical service market going forward. Personalized medicine refers to treatments designed according to an individual's genetic profile, enhancing effectiveness and minimizing adverse effects. The growth of personalized medicine is fueled by advancements in genomics, biomarker research, and data analytics, enabling therapies to be customized to a patient's genetic makeup, improving outcomes, reducing side effects, and addressing unmet medical needs more precisely than conventional one-size-fits-all approaches. Pharmaceutical services support personalized medicine by developing targeted therapies, utilizing genetic and biomarker information, and offering customized treatment solutions to address the specific needs of individual patients. For example, in February 2024, according to the Personalized Medicine Coalition, a US-based non-profit organization, the FDA approved 16 new personalized treatments for rare disease patients in 2023, compared to 6 approvals in 2022. Consequently, the rise of personalized medicine is fueling the growth of the rare disease pharmaceutical service market.

Major companies operating in the rare disease pharmaceutical service market are increasingly concentrating on developing innovative treatment strategies, such as acetylleucine, to enhance treatment efficacy, improve patient outcomes, and address unmet needs in rare neurological and metabolic disorders. Acetylleucine, a modified amino acid (N-acetyl-L-leucine), is employed as a therapeutic agent to support motor function, balance, and neurological health in affected patients. For example, in September 2024, IntraBio, a US-based biopharmaceutical company, received FDA approval for AQNEURSA, the first stand-alone therapy for Niemann-Pick Disease Type C, a rare, life-threatening neurodegenerative disorder. This achievement is noteworthy, as the treatment showed rapid and meaningful improvements in neurological symptoms, providing a groundbreaking therapeutic option for both pediatric and adult patients living with this rare disease.

In March 2024, AstraZeneca, a UK-based pharmaceutical company, acquired Amolyt Pharma for an undisclosed amount. Through this acquisition, AstraZeneca aims to enhance its late-stage rare disease pipeline, particularly within its Alexion division, by incorporating innovative therapies for rare endocrine disorders. Amolyt Pharma is a France-based clinical-stage biotechnology company that focuses on developing peptide-based therapies for rare endocrine diseases.

Major companies operating in the rare disease pharmaceutical service market are Johnson & Johnson, Merck & Co. Inc., Pfizer Inc., AbbVie Inc., Sanofi S.A., Novartis AG, AstraZeneca PLC, GlaxoSmithKline PLC (GSK), Takeda Pharmaceutical Company Limited, Amgen Inc., Biogen Inc., Chiesi Farmaceutici S.p.A., Recordati Industria Chimica e Farmaceutica S.p.A., BioMarin Pharmaceutical Inc., Sarepta Therapeutics Inc., Ultragenyx Pharmaceutical Inc., Amicus Therapeutics Inc., Travere Therapeutics Inc., Sciensus Healthcare Ltd., SOM Innovation Biotech S.L.

North America was the largest region in the rare disease pharmaceutical service market in 2025. The regions covered in the rare disease pharmaceutical 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 rare disease pharmaceutical service market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Taiwan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

The rare disease pharmaceutical service market consists of revenues earned by entities by providing services such as clinical trial management, patient recruitment, regulatory compliance, market access, and safety monitoring. 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.

Rare Disease Pharmaceutical 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 rare disease pharmaceutical 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 rare disease pharmaceutical 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 rare disease pharmaceutical 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. Rare Disease Pharmaceutical Service Market Characteristics

3. Rare Disease Pharmaceutical Service Market Supply Chain Analysis

4. Global Rare Disease Pharmaceutical Service Market Trends And Strategies

5. Rare Disease Pharmaceutical Service Market Analysis Of End Use Industries

6. Rare Disease Pharmaceutical 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 Rare Disease Pharmaceutical Service Strategic Analysis Framework, Current Market Size, Market Comparisons And Growth Rate Analysis

8. Global Rare Disease Pharmaceutical Service Total Addressable Market (TAM) Analysis for the Market

9. Rare Disease Pharmaceutical Service Market Segmentation

10. Rare Disease Pharmaceutical Service Market Regional And Country Analysis

11. Asia-Pacific Rare Disease Pharmaceutical Service Market

12. China Rare Disease Pharmaceutical Service Market

13. India Rare Disease Pharmaceutical Service Market

14. Japan Rare Disease Pharmaceutical Service Market

15. Australia Rare Disease Pharmaceutical Service Market

16. Indonesia Rare Disease Pharmaceutical Service Market

17. South Korea Rare Disease Pharmaceutical Service Market

18. Taiwan Rare Disease Pharmaceutical Service Market

19. South East Asia Rare Disease Pharmaceutical Service Market

20. Western Europe Rare Disease Pharmaceutical Service Market

21. UK Rare Disease Pharmaceutical Service Market

22. Germany Rare Disease Pharmaceutical Service Market

23. France Rare Disease Pharmaceutical Service Market

24. Italy Rare Disease Pharmaceutical Service Market

25. Spain Rare Disease Pharmaceutical Service Market

26. Eastern Europe Rare Disease Pharmaceutical Service Market

27. Russia Rare Disease Pharmaceutical Service Market

28. North America Rare Disease Pharmaceutical Service Market

29. USA Rare Disease Pharmaceutical Service Market

30. Canada Rare Disease Pharmaceutical Service Market

31. South America Rare Disease Pharmaceutical Service Market

32. Brazil Rare Disease Pharmaceutical Service Market

33. Middle East Rare Disease Pharmaceutical Service Market

34. Africa Rare Disease Pharmaceutical Service Market

35. Rare Disease Pharmaceutical Service Market Regulatory and Investment Landscape

36. Rare Disease Pharmaceutical Service Market Competitive Landscape And Company Profiles

37. Rare Disease Pharmaceutical Service Market Other Major And Innovative Companies

38. Global Rare Disease Pharmaceutical Service Market Competitive Benchmarking And Dashboard

39. Key Mergers And Acquisitions In The Rare Disease Pharmaceutical Service Market

40. Rare Disease Pharmaceutical Service Market High Potential Countries, Segments and Strategies

41. Appendix

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