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.

We are rare disease specialists.

CRA’s experience stems from hundreds of rare disease engagements across many therapeutic areas.

  • 50+
    Rare disease areas covered
  • 450+
    Rare disease engagements since 2017

Our rare disease offerings span across the product lifecycle.

  • Early development
    • Early PMA assessment
    • Pipeline opportunity assessment
    • Diagnostics & screening
    • Incentives review
    • 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
    • Regulatory and P&R policies
    • 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
    Issue Our client sought to understand the current state of healthcare across borders (also known as medical tourism) and the opportunities and risks this...
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  • 02
    Global pricing and access strategic issues assessment for rare diseases gene therapy portfolio
    Issue Our client is developing gene therapies for different rare disease indications, some with effective treatments available and others with only symptom...
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  • 03
    Understanding and segmenting a rare disease market
    Client Issue Our client sought to develop a better understanding of the market for a rare disease, including patient diversity and treatment, in anticipation...
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