Rare Disease: 2024 in review
As we reflect on another year in the rare disease space, we are proud of the continued growth and evolution of CRA’s rare disease capabilities and offerings....
Strategy in rare disease requires specialist expertise and understanding. CRA understands the unique challenges that rare disease companies face, and we implement a distinctive approach to help drive their success.
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We are rare disease specialists.
CRA’s experience stems from hundreds of rare disease engagements across many therapeutic areas.
- 50+Rare disease areas covered
- 500+Rare disease engagements since 2019
Our rare disease offerings span across the product lifecycle.
- Early developmentDiscover
- Pipeline opportunity assessment
- Diagnostics & screening
- Early Pricing Marketing Access (PMA)
- Licensing / commercially reasonable efforts disputes
- Late developmentDevelop
- Market access & evidence generation strategy
- Payer value proposition
- Patient finding
- Landscape assessment & policy shaping
- LaunchDeliver
- Launch pricing strategy
- Payer negotiation
- Launch excellence
- Stakeholder engagement
- Competitive simulations
- Uptake and diffusion
- Life cycle & LOEDefend
- Indication & geographic expansion
- Competitor response strategies
- Price/access/uptake benchmarking and assessments
- LOE strategies
Engagements
- 01Developed dynamic GTN model to guide pricing and contracting strategy for a rare disease assetA leading pharmaceutical company engaged CRA’s Life Sciences consultants for support in developing a pricing and contracting strategy for a rare disease assetView engagement
- 02Evaluating the potential impact of cross-border healthcare for gene therapiesCRA's Rare Disease team evaluated the dynamics of cross-border healthcare using secondary research to explore current precedence and/or previous product...View engagement
- 03Global pricing and access strategic issues assessment for rare diseases gene therapy portfolioCRA delivered recommendations on optimization and risk mitigation for gene therapy pricing strategy implementation...View engagement
Contact a Rare Disease expert.
See all Rare Disease expertsRecent and Upcoming Events
ISPOR Europe 2024
CRA had four posters accepted at the conference. Kirsten Axelsen and Charlotte Poon present their poster titled “Assessing the economic burden of rare disease...
World Orphan Drug Congress EU 2024
CRA is a proud sponsor of the conference. Come visit us at booth #41! Bhavesh Patel and Owen Male present the session titled “Maximizing Value & Positioning in...