Rare Disease Drug Development Market By Application: Drug Discovery, Clinical Trials, By Application: Drug Discovery, Clinical Trials, By End-Use: Pharmaceutical Companies, Research Institutions, By Region: North America, Europe, Asia Pacific, Segment Forecasts: 2024 - 2030
Rare Disease Drug Development Market Trends
The rare disease drug development market was valued at USD 20.3 billion in 2024 and is expected to grow at a CAGR of 12.8% from 2025 to 2030. The increasing focus on developing therapies for rare diseases is driven by advancements in research and regulatory support.
Product Insights
Drug discovery applications represented the largest market segment in 2024, accounting for 58.9% of total revenue. Clinical trials are also expanding significantly, reflecting the increasing number of orphan drugs entering development.
End-Use Insights
Pharmaceutical companies were the largest end-users in 2024, holding a 65.1% market share, as they prioritize rare disease therapies in their pipelines. Research institutions are also playing a crucial role in advancing drug development efforts.
Regional Insights
North America dominated the rare disease drug development market with a 47.6% share in 2024, supported by a strong regulatory framework and significant investment in research. The Asia Pacific region is expected to witness rapid growth, with a CAGR of 15.3%, as awareness of rare diseases increases.
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