What Is Driving Global Orphan Drugs Service Market Growth in 2025: The Role of Growing Investment In Rare Diseases Fueling The Growth Of The Market Due To Increasing Funding For Research, Development, And Patient Access

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Orphan Drugs Service Market Size Growth Forecast: What to Expect by 2025?
In recent times, the market size for orphan drugs services has seen a swift expansion. The market, which is predicted to grow from $37.05 billion in 2024 to $43.40 billion in 2025, features a compound annual growth rate (CAGR) of 17.1%. The surge observed in the historical period can be linked to factors such as a higher incidence of rare ailments, escalating government backing, increasing initiatives by patient advocacy groups, a growing emphasis on unresolved medical needs, and stronger collaboration between healthcare providers and researchers.

How Will the Orphan Drugs Service Market Size Evolve and Grow by 2029?
The market size of orphan drugs services is anticipated to experience a significant surge in the upcoming years, forecasted to expand to $80.62 billion by 2029, with a compound annual growth rate (CAGR) of 16.7%. This market growth during the forecast period can be linked to various factors such as increased investment in biopharmaceutical research, growing need for precision medicine, rising incorporation of advanced clinical trial designs, increased funding from venture capital companies, and a heightened focus on regulatory harmonization globally. Key trends in the forecast period encompass advances in drug discovery technology, innovations in cell and gene therapy services, investment in artificial intelligence for clinical trials, progress in patient selection based on biomarkers, and cutting-edge developments in digital health for patient monitoring.

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What Drivers Are Propelling the Growth of Orphan Drugs Service Market Forward?
The surge in investment in rare diseases stands to drive the expansion of the orphan drugs service market. Rare diseases, which affect a tiny proportion of the population, typically less than 1 out of 2,000 individuals, are usually chronic, progressive, and could be lethal or disabling. The primary motivation for investment in rare diseases is the ever-increasing awareness of untapped medical needs and the promise of high yields through regulatory incentives like orphan drug designation, market monopoly, and the reduction of development costs. By investing more into rare diseases, orphan drug services are improved, as there will be more funding available for research and development, potentially leading to the discovery of new treatments for previously overlooked conditions. Moreover, it expedicates the availability of drugs by backing clinical trials, regulatory approvals, and patient access, thus augmenting treatment alternatives for those with rare diseases. For example, as stated by Global Genes, an American nonprofit organization, in April 2024, firms that devise drugs for rare diseases amassed $7.1 billion in the first quarter of 2024, which is a 307% surge compared to the $1.8 billion gathered during the same timeframe in 2023. Therefore, the escalating investment in rare diseases is fueling the growth of the orphan drugs service market.

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Which Emerging Trends Are Transforming the Orphan Drugs Service Market in 2025?
Major corporations active in the orphan drugs industry are focused on securing regulatory approvals as a means to quicken the development and distribution of rare disease treatments. Such approvals are formal authorisations granted by government bodies, permitting legal testing, manufacturing, and marketing of a drug or treatment. For example, Askbio GmbH, an American gene therapy firm, was awarded the European Commission orphan drug designation for AB-1003, an innovative gene therapy under investigation for limb-girdle muscular dystrophy, in February 2023. The aim of this adeno-associated virus (AAV)-based therapy is to revitalize FKRP enzyme activity in muscle cells through a single intravenous infusion. The European Commission and the U.S. Food and Drug Administration (FDA) have both granted AB-1003 orphan drug status, with the FDA also granting rare pediatric disease and fast track designations, reflecting the pressing unmet medical need. AB-1003 is currently undergoing a Phase 1/2 clinical study (LION-CS101), where its safety and effectiveness in adults diagnosed with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) is being evaluated.

What Are the Key Segments in the Orphan Drugs Service Market?
The orphan drugs service market covered in this report is segmented as

1) By Type: Congenital Disease, Genetic Disease, Tumors And Cancer, Chronic, Other Types
2) By Therapeutic Area: Oncology, Metabolic Disorders, Neurological Disorders, Hematology, Infectious Diseases, Other Rare Diseases
3) By Application: Aldult, Children

Subsegments:
1) By Congenital Disease: Heart Defects, Cleft Lip And Palate, Spina Bifida, Down Syndrome
2) By Genetic Disease: Cystic Fibrosis, Huntington Disease, Sickle Cell Anemia, Muscular Dystrophy
3) By Tumors And Cancer: Leukemia, Lymphoma, Neuroblastoma, Sarcoma
4) By Chronic: Rare Metabolic Disorders, Rare Endocrine Disorders, Rare Cardiovascular Disorders, Rare Neurological Disorders
5) By Other Types: Rare Infectious Diseases, Rare Immunological Disorders, Rare Dermatological Conditions, Rare Ophthalmological Disorders

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Who Are the Key Players Shaping the Orphan Drugs Service Market's Competitive Landscape?
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., and Cevidra Inc.

What Geographic Markets Are Powering Growth in the Orphan Drugs Service Market?
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.

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