life sciences

Rare Disease

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.

Follow us on LinkedIn: CRA on Rare Disease 

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 development
    • Pipeline opportunity assessment
    • Diagnostics & screening
    • Early Pricing Marketing Access (PMA)
    • Licensing / commercially reasonable efforts disputes
  • Late development
    • Market access & evidence generation strategy
    • Payer value proposition
    • Patient finding
    • Landscape assessment & policy shaping
  • Launch
    • Launch pricing strategy
    • Payer negotiation
    • Launch excellence
    • Stakeholder engagement
    • Competitive simulations
    • Uptake and diffusion
  • Life cycle & LOE
    • Indication & geographic expansion
    • Competitor response strategies
    • Price/access/uptake benchmarking and assessments
    • LOE strategies


  • 01
    Evaluating the potential impact of cross-border healthcare for gene therapies
    CRA's Rare Disease team evaluated the dynamics of cross-border healthcare using secondary research to explore current precedence and/or previous product...
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  • 02
    Global pricing and access strategic issues assessment for rare diseases gene therapy portfolio
    CRA delivered recommendations on optimization and risk mitigation for gene therapy pricing strategy implementation...
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  • 03
    Understanding and segmenting a rare disease market
    Our client sought to develop a better understanding of the market for a rare disease, including patient diversity and treatment, in anticipation of...
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