CRA Insights

Rare Disease: A year in review

December 19, 2022
DNA, life science, genetics

As more investments have been made in developing and commercializing treatments for rare diseases, the experience and expertise of our CRA teams have grown. The number of client projects undertaken by CRA consultants in rare diseases now exceeds 600 in the last five years and constitutes over 20% of our total client projects. In the past year alone, we have completed over 120 projects on rare diseases, covering everything from neurology to rare oncology. In addition, our consultants have presented at events, including World Orphan Drug Congresses (US and Europe), ISPOR, and PMSA, and written over 60 published thought leadership articles covering rare disease issues.

We are delighted to present this review to showcase our activities, capabilities, and experience in rare diseases and look forward to working with clients in this expanding and exciting space in the years ahead.

Dr. Greg Bell, Group Vice President and Global Practice Leader, Life Sciences, CRA

It has been an exciting and fruitful year for the CRA Rare Disease Initiative team. In celebration of Rare Disease Day 2022, we launched #RDSpotlight, a series that provides updates on recent trends and developments, insights and thought leadership, and highlights of our key engagements across strategy, policy, and litigation in rare disease. We continued to strengthen our presence and position as a strategic thought partner in rare disease by innovating and enhancing our offerings and capabilities, disseminating pertinent content, and building relationships. Of note, our team has been gathering opinions on the future of rare disease through Project LEOPARD (Long-term Evolution & Outlook for Patient Access in Rare Disease) and developing RADAR (Rare Disease Assessment Reviews), a central source of knowledge for orphan drug assessments across key geographies. With rare disease being a key area of focus for CRA, the RD Initiative team will continue to build upon the depth and breadth of our experience to partner with organizations connecting patients to the care they need.

Dr. Bhavesh Patel, Principal, Lead for CRA Rare Disease Initiative

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