Understanding global P&MA implications for indication expansion of orphan drug

Drug production and inspection

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

CRA reviewed available data to establish the rare disease market landscape in the context of an expected competitor launch in the expansion indication. We conducted payer and KOL interviews in the US and four EU markets to determine value perception of the orphan drug in the new indication and understand likely P&MA outcomes. Finally, CRA analysed the research findings to develop insights and deliver recommendations to address the strategic business questions.

CRA provided global and geography-specific assessment of the marketplace for the expansion, assessment of anticipated competitor positioning, evidence requirements, unmet needs assessment, and issues framework. We provided consolidated insights regarding payer receptivity of the potential expanded indication, including cross-market and local perspectives and delivered recommendations on value perception, P&MA strategy, and evidence requirements.