Analysis of the global rare disease landscape

Robotic pipette with test tubes

CRA experts applied policy expertise spanning 40 global markets to help our client plan their approach to rare disease therapy development and patient access in different areas per market type.

For each market, CRA provided insights on the organisation of care (including information on screening and diagnosis of rare disease), access (including early access programs, value assessments, funding mechanisms), policy (including frameworks and rare disease plans), regulatory frameworks, and research/innovation financial incentives or funding. We created an overview of the rare disease landscape across markets to provide a snapshot of where provisions and policies are in place or require development.

CRA delivered a toolkit for global and local teams to use to better understand the rare disease environment, best practices for R&D and access, and to develop their rare disease product and portfolio strategy.