The Challenge.

A mid-sized pharmaceutical company with a solid track record in specialty care was preparing to enter unfamiliar territory with its first rare disease asset. Although the organisation had launched multiple medicines before, this new therapy area required a different pace, mindset, and playbook. Evidence expectations were more demanding, the stakeholder environment more complex, and internal assumptions about roles, readiness, and reach no longer applied.

What We Did.

We partnered with the client to conduct a rare disease-specific gap analysis and rebuild their launch roadmap around the realities of this new landscape. The engagement included:

• Conducting a cross-launch audit to identify which previous practices would carry forward — and which could limit success in rare disease
• Redesigning the launch roadmap to reflect rare disease requirements: earlier medical engagement, more nuanced access planning, and tighter sequencing between regulatory, evidence, and commercial milestones
• Advising on a revised evidence generation strategy, tailored to new endpoints, smaller patient populations, and heightened payer scrutiny
• Using Perceptor Vision to assess team mindset and behavioural readiness — particularly in areas like ambiguity tolerance, risk ownership, and cross-functional communication
• Supporting senior leaders to lead the internal shift, clarify priorities, and realign expectations across functions