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
  • 300+
    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 strateg
    • 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
    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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  • 02
    Identification strategy planning support for future disruptive policy trends for orphan drugs
    Our client wanted to identify key disruptive trends impacting the HTA, pricing and market access environment for orphan drugs across a range of EU and emerging...
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  • 03
    Long-term partnership for pricing and market access launch strategy support
    Our client was preparing for the launch of a new orphan drug in Europe and needed support with tactical planning and negotiation skills since most of the...
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