Conference Day 2 |
Thursday, August 28, 2025

10:00 am
Registration & Light Breakfast

10:25 am
Chair’s Opening Remarks

Empowering the Patient & Developing the Value for Patient Groups in an Orphan or Competitive Drug Market to Increase Uptake

10:30 am Understanding The Different Types of Engagements That Can Be Developed with Patient Advocacy Groups to Best Inform Launch Strategy

  • Betty Tran Executive Director, Head of Patient Services, BridgeBio Pharma, Inc.

Synopsis

  • How to leverage patient advocacy groups without overstepping internal stakeholders
  • Understanding the mechanisms on how to engage with patient advocacy groups
  • Balancing patient support programs with patient advocacy education and initiatives

11:00 am Understanding The Importance of Patient Liaison and How We Can Support Patients When Embarking on Uptake of a New Therapeutic

  • Kathleen Gilluly Patient Education & Rare Disease Liaison, Amicus Therapeutics, Inc.

Synopsis

  • How to assure patients whilst not being an extension of health care provision
  • How best to support patients with the uptake of treatment and maintain regular monitoring without burdening the patient
  • How to best collect information from patient and how to comply with policy
  • How to best assess you patient, create individualised patient centric plans & encourage them to be their own self-advocate

11:30 am
Morning Break & Speed Networking

Innovating Internal Education of Commercialization to Allow for a Smoother Commercialization & Regulation Strategy

12:00 pm Preparing for a Shift in the Company Strategy from Early-Stage Commercialization to Late-Stage Commercialization

Synopsis

  • How to develop understanding and educate the internal business on the shift between early to late-stage commercialization
  • Understand the shift in prorates from early to late-stage commercialisation and how individual functions may change
  • Addressing new regulatory hurdles, post-approval obligations, and real-world evidence requirements

12:30 pm Mastermind: Demystifying the Adaptations to Policy when Regarding Rare Disease to Ensure a Smoother Approvable Commercial Strategy

Synopsis

  • Understanding how orphan drug designations, accelerated pathways, and regulatory incentives impact commercialization strategy
  • Keeping pace with shifting global regulations to ensure compliance and optimize market access for rare disease treatments
  • Effectively aligning regulatory strategy with pricing, reimbursement, and patient access considerations

1:00 pm
Lunch & Networking

Fuelling A Successful Go-to-Market Strategy by Redefining a Standard of Care Versus Creating an Orphan Drug Treatment & Progressing External Disease Education to Enable Maximum Commercial Success

2:00 pm Evaluating The Market Shaping That Needs to Be Carried Out in a Disease Area with Therapeutics Already Available

  • Vikram Patel Executive Director, HEOR field, ACADIA Pharmaceuticals Inc.

Synopsis

  • Assessing unmet needs & differentiation strategies identifying gaps in current treatments to position a new therapy effectively
  • Engaging payers & HTAs early addressing pricing, reimbursement, and value demonstration challenges in an already competitive market
  • Overcoming physician prescribing habits developing strategies to shift physician behaviour when an established standard of care exists

2:30 pm Comparing The Education Needs of the Market in a Commercial Launch of an Orphan Drug with a Launch in a Competitive Space

  • Liz Pfau Neurology Global Marketing Lead, Rare Disease, Amgen Inc.

Synopsis

  • Engaging KOLs & patient advocacy groups leveraging external voices to shape perceptions and drive adoption in both launch scenarios
  • Tailoring messaging for competitive markets vs first-to-market therapies differentiating education strategies based on existing treatment landscapes

3:00 pm How Best to Profile an Asset to Make Informed Decisions in the Beginning of a Commercial Strategy

  • Marsha Pelletier Global Pipeline & Business Development Commercial Strategy, BioMarin Pharmaceutical

Synopsis

  • Defining the target patient population utilizing epidemiology data and real-world evidence to refine market size and potential
  • Aligning clinical development with commercial objectives ensuring trial endpoints support regulatory approval and payer negotiations
  • Prioritizing key geographies and markets identifying regions with the highest unmet need, fastest regulatory pathways, and most favourable reimbursement environments to maximize commercial success

3:30 pm
Afternoon Break & Networking

Engaging All Stakeholders & Unleashing Better Co-Creation When Working Across Multifunctional Commercial Teams

4:30 pm Ensuring Patient Voice Is the Influential Driver in Commercial Strategy

Synopsis

  • Integrating patient-reported outcomes into value proposition demonstrating the realworld impact of treatment on quality of life
  • Delving into the integration of patient report outcomes from the clinic and how to integrate this into this to benefit the asset long term
  • Methods of collecting patient data and how this can influence are asset development
  • Uncovering best practice on complying with FDA guidance on patient engagement

5:00 pm Analysing The Commercial Team & And How to Build Out Strategy That Combines Multiple Cross Functional Roles

Synopsis

  • Defining key roles & responsibilities in rare disease commercialization structuring teams for maximum efficiency in a niche market
  • Breaking down silos between clinical, medical, and commercial teams encouraging data-sharing and collaboration from development to launch
  • Leveraging competitive intelligence & market insights ensuring all functions are aligned with market trends and emerging competitor strategies

5:30 pm
Chair’s Closing Remarks

5:40 pm
End of Conference Day Two