CRA Insights

Rare Disease: 2025 in review

January 27, 2026
Genetic research, conceptual image

2025 has been a year of progress in the rare disease (RD) space. At CRA, we continue to leverage our cross-functional expertise in strategy, policy, analytics, and litigation to deliver holistic solutions and uncover new opportunities for our clients.

Over the past five years, CRA has supported more than 700 RD projects – nearly 150 of them in 2025 alone – covering a wide range of therapeutic areas and modalities.

Our Rare Disease Initiative Team was more active than ever this year in sharing insights at leading global events such as the World Orphan Drug Congress (WODC) and ISPOR, and publishing thought leadership to spark dialogue on the future of RD innovation.

This review captures the scope of our contributions and the impact of our partnerships. We look forward to continued collaboration as we navigate and shape the future of the RD space.

Read our 2025 year in review here.

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