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
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
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
2:30 pm - 3:10 pm
Interactive Roundtable: Building a Competitive Mindset Across Launch Teams to Succeed in an Increasingly Crowded Rare Disease Market
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
3:10 pm - 3:40 pm
Case Study Presentation: Learning How to Utilise Healthcare Systems to Accelerate Adoption of Rare Disease Therapies & Influence Launch
A key pillar of rare disease launch excellence is ensuring healthcare systems can successfully adopt and consistently utilise new therapies to treat patients. To achieve this, healthcare systems must be able to identify eligible patients, improve diagnostic pathways, facilitate access to treatment, and leverage real-world evidence to educate patients, healthcare professionals, payers, and health technology assessment bodies on the benefits of their rare disease therapy.
• Assessing whether healthcare professionals are using your therapy effectively in clinical practice across different markets
• Ensuring healthcare professionals are appropriately trained to administer and support your rare disease therapy
• Navigating the unique challenges of healthcare systems adopting your rare disease therapy compared with launches in more established therapeutic areas
