Pharma Launch Excellence Summit Pre-Conference Focus Day: Launch Excellence in Rare Disease: Tuesday 1st December
Pre-Conference Focus Day: Launch Excellence in Rare Disease
Pharma Launch Excellence Summit Workshop Day: Tuesday 1st December
Pharma Launch Excellence Summit Main Conference Day 1: Wednesday 2nd December
Pharma Launch Excellence Summit Main Conference Day 2: Thursday 3rd December
Pharma Launch Excellence Summit Pre-Conference Focus Day: Launch Excellence in Rare Disease: Tuesday 1st December
9:00 am - 10:00 am
Registration
10:00 am - 10:40 am
Case Study Presentation: Launching Rare Disease Products Through Uncertainty: Lessons from Neuromuscular Rare Disease Launches in Partner Markets
Manel Saidani -
Head of Marketing & Analytics & International Partner Markets,
Biogen
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
10:40 am - 11:20 am
Panel Discussion: Partnering with Patient Advocacy Groups in Drug Development to Accelerate Rare Disease Launch Success
Karen Cheng -
Patient Advocate,
International Myositis Society
Eugene Osei-Bonsu - Senior Director - Neuroscience Marketing and Strategy Lead - Global Portfolio D, Takeda
Philipp Gallwitz - Managing Director, Partners4Patients
Patient advocacy groups can play a pivotal role in the successful development and launch of rare disease therapies. Early collaboration with patient advocacy groups can be a critical determinant of launch success. How can launch excellence teams ensure patient perspectives are meaningfully embedded throughout the drug development lifecycle?
• Building strong relationships with patients, caregivers, patient advocacy groups and external experts to maximise the success of your rare disease therapy launch
• Leveraging patient advocacy group partnerships to gain critical insights, engage diverse patient populations and support market access efforts to ensure your therapy successfully reaches patients who need it most
• Discovering how successful rare disease launches benefited from strong collaboration with patient advocacy groups to apply lessons learned to your next launch
Eugene Osei-Bonsu
Senior Director - Neuroscience Marketing and Strategy Lead - Global Portfolio DTakeda
11:20 am - 11:50 am
Networking Break
11:50 am - 12:20 pm
Leveraging Emerging Tools & Technologies to Make Data-Driven Decisions During Launch
12:20 pm - 1:00 pm
Case Study Presentation: Building a Robust Cross-Functional Alignment Strategy to Improve Rare Disease Launch Execution
Dijana Krafcsik -
Director, International Marketing - Rare Nephrology,
CSL
Rare disease launches involve small patient populations, making every decision critical to success. To maximise impact, teams need clear objectives, streamlined operations and strong cross-functional alignment. Yet aligning global, regional, and functional teams remains a significant challenge. How can launch excellence teams improve collaboration, accountability, and visibility while maintaining the agility needed to navigate an unpredictable launch environment?
• Defining team roles and responsibilities to ensure accountability for key launch milestones.
• Increasing transparency across global, regional, and functional teams to improve visibility of progress against shared objectives
• Developing an effective tracking tool to simplify the management of cross-functional launch activities across global teams
• Building a proactive risk mitigation strategy to help teams anticipate and respond to the unique challenges and uncertainties of rare disease launches
1:30 pm - 2:30 pm
Networking Lunch Break
2:30 pm - 3:10 pm
Interactive Roundtable: Building a Competitive Mindset Across Launch Teams to Succeed in an Increasingly Crowded Rare Disease Market
Veidehi Patel -
Associate Director | Customer Strategy and Operational Excellence,
Jazz Pharmaceuticals
As rare disease markets become increasingly competitive, launch teams can no longer rely on traditional approaches. Organisations must foster a stronger competitive mindset across medical, commercial, and market access functions, drawing on proven strategies from rare disease markets to anticipate competitors, sharpen differentiation, and execute with greater confidence.
Join this interactive roundtable discussion to benchmark approaches to foster a winner's mindset within your team to make sure your next rare
disease launch strategy is successful from conception through to execution.
• Building a competitive mindset across cross-functional launch teams to anticipate competitor launch tactics and build a strategy to ensure your launch delivers
• Applying proven competitive strategies from established rare disease launches to maximise the success of your rare disease launch
• Discussing tips and tricks to strengthen launch readiness through proactive competitor planning and differentiation
Veidehi Patel
Associate Director | Customer Strategy and Operational ExcellenceJazz Pharmaceuticals
3:10 pm - 3:40 pm
Going from Zero to Launch: Building a Sustainable Patient Pathway to Accelerate Therapy Adoption & Ensure Commercial Success
Ben Whitehouse -
UK & IE General Manager,
Ascendis Pharma
Rare disease launches depend on finding the right patients fast, navigating diverse healthcare systems, turning limited datasets into actionable launch strategies, and building internal confidence to invest early. Launch leaders who get this right unlock faster therapy uptake and higher chances of launch success. Attend this expert-led session to understand how to:
• Leverage datasets to identify crisis and lost patients to accelerate early therapy uptake
• Apply AI-driven insights to build scalable, high impact patient pathways that enable
commercial launch success
• Secure internal buy-in to confidently navigate uncertain outcomes during launch execution
