Focus Area Rare Disease


A global pharma company looking to launch a new gene therapy in a rare disease. The team wanted to pressure test their long-term strategy by looking at a number of market scenarios that could eventuate based on varying FDA labels and competitor launch timeframes.


  • Developed a scenario framework that identified all plausible permutations of competitor launch and asset labelling and consolidated into four key planning scenarios
  • Characterized each scenario identifying HCP, patient, payer and policy maker and key competitor behavior in each case
  • Validated scenario assumptions in interactive workshops with the EU and US cross-functional teams
  • Identified 6 key issues distilled from over 150 comments and observations in the workshops
  • Addressed each issue in a further set of EU and US workshops which looked at mitigation strategies in each case
  • Summarized findings and recommendations for evolution of strategy and tactics in a comprehensive management report


Pressure test strategy
Identification and characterization of a set of plausible scenarios enabled the pressure testing of core strategy
Initiative identification
The depth of thinking encouraged throughout the cross-functional teams allowed countries and functions to identify initiatives that needed to be strengthened or accelerated
Planning confidence
In summary, the project delivered planning confidence in an uncertain market


Comprehensive report containing:

  • A scenario framework distilling multiple future market outcomes into a finite and manageable number of planning scenarios
  • Detailed descriptions of each scenario, including likelihood on impact on HCPs, patients and payers in EU and US
  • Hypothesized competitor strategy, positioning and brand story
  • Priority company issues emerging from scenario planning
  • Strategy and key activities to address each issue