Understanding global PMA implications for gene therapy

A leading biotech company sought to evaluate market receptivity for its breakthrough Phase III drug in a rare hematology disorder.

CRA approach

  • Review available data to establish market landscape in the context of an expected competitor launch in expanded indication
  • Develop research materials and screening criteria for execution of payer and KOL interviews
  • Conduct interviews to determine value perception of drug in the new indication and understand likely pricing and market access (PMA) outcomes
  • Analyze research findings to develop insights and deliver recommendations to address the strategic business questions

Client impact

  • Provided global and geographic-specific assessment of the marketplace for the expansion, assessment of anticipated competitor positioning, evidence requirements, unmet needs assessment, and issues framework
  • Provided consolidated insights regarding payer receptivity of the potential expanded indication, including cross-market and local perspectives
  • Delivered recommendations on value perception, PMA strategy, and evidence requirements
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