Launching a rare disease therapy requires navigating significant uncertainty, from evolving regulatory pathways and limited disease awareness to fragmented patient populations and changing operational realities. Success depends not only on launch planning but on the ability to continuously adapt while maintaining focus on patient impact.
Drawing on launch experiences across ALS, Friedreich's Ataxia (FA), and Spinal Muscular Atrophy (SMA), this session will share practical lessons on building resilient launch strategies, strengthening internal alignment, and addressing market-specific challenges to maximise patient access and commercial success.
• Conducting effective opportunity assessment and outlining the unique risks associated with rare disease launches and the corresponding agile strategy
• Building a compelling business cases to advocate for different opportunities and addressing the unmet needs of rare disease patients while minimising business complexity
• Developing an agile GTM to adapt to local uncertainties and navigate complex ecosystems and patient journeys
• Overcoming internal operational challenges while adapting global processes to meet diverse local market needs during launch
• Leveraging various access mechanisms to accelerate treatment availability and address unmet needs as early as possible: Drawing conclusions from HCP and Authorities engagement in very different territories (Turkey, Algeria, Greece, Balkans)
• Accelerating speed of referral and diagnosis while adapting to local ecosystems from the most centralised to the most decentralised ones