When you look at the agenda, you will see 3 different session types:

Gene Therapy Medical Affairs Discover
Gene Therapy Medical Affairs Develop
Gene Therapy Medical Affairs Action

Discover Sessions

Offering you the chance to hear the most admired medical affairs professionals from the gene therapy world share the stories and case studies of what they’ve done to solve their more pressing challenges.

Develop Sessions

Providing you with the unique opportunity to have your burning questions answered in a virtual room filled with your industry peers, and move beyond the “what” and the “why” to get to the “how”.

Action Sessions

Designed to help you work with your team to set a clear, bespoke action plan of what you are going to change when you get back to your organization to ensure you see a fast ROI.

8:15 am Online Registration & Virtual Coffee Networking

8:50 am Chair’s Opening Remarks

GENE THERAPY LAUNCH READINESS – MAKING OR BREAKING A SUCCESSFUL LAUNCH

9:00 am Discover: Building the Plane as You Fly it – Learnings from Gene Therapy Launches

  • Sue Lacey Scientific & Medical Affairs Consultant, Astraea Medical Consulting

Synopsis

• Ripping up the rule book – why and how gene therapy launches should deviate from the ‘norm’
• The importance of early stakeholder engagement: who, when and what to discuss
• Why (in my completely biased opinion) medical affairs has the most critical role to play

9:20 am Discover: Launch Excellence in Gene Therapy: Is it Achievable?

  • Ramin Farhood Vice President & Head of Global Medical Affairs, Novartis Gene Therapy

Synopsis

• What does launch excellence look like?

9:40 am Discover: Launch Readiness

  • Laura Campbell Global Director Medical Affairs, Orchard Therapeutics

Synopsis

• From academia to commercial product
• Site qualification
• Gene therapy – a unique launch?

10:00 am Develop: What Are the Keys to Success When it Comes to the Role of Medical Affairs in the Successful Launch of a Gene Therapy Product?

  • Sue Lacey Scientific & Medical Affairs Consultant, Astraea Medical Consulting
  • Ramin Farhood Vice President & Head of Global Medical Affairs, Novartis Gene Therapy
  • Laura Campbell Global Director Medical Affairs, Orchard Therapeutics

Synopsis

This is your opportunity to have the speakers answer your questions!

10:30 am Action: What Are You Now Going to Change or Implement to Your Current or Future Launch Strategy in Order To Maximize Its Effectiveness?

10:50 am Virtual Speed Networking & Morning Break

Synopsis

Recreating face-to-face networking in the virtual world. We will pair you up with fellow attendees
so you can get face-to-face time with the brightest minds working in the gene therapy field in
order to establish meaningful business relationships to pursue for the rest of the conference.

OPTIMIZING YOUR MARKET ACCESS STRATEGY

11:50 am Discover: How can Market Access and Medical Affairs collaborate Effectively to Maximize Access to Gene Therapies?

Synopsis

• Key challenges facing access to gene therapy and how it’s different from other “standard” treatments
• How can Medical Affairs optimize market access strategy and support reimbursement discussions
• The importance of early insights on the clinical development plan and early external stakeholder engagement

12:10 pm Discover: Explore Delivering Gene Therapy to Patients

  • Nick Li Senior Director HEOR, UniQure

Synopsis

• How gene therapy changes the treatment paradigm
• New value and access challenges with gene therapy
• Patient-centric approach to demonstrate value and achieve optimal access for gene therapies

12:30 pm Develop: What Are the Keys to Success to Ensure You Are Able to Communicate Effectively with Key Stakeholders, So They Understand the Value Your Drug Brings to Patients?

Synopsis

This is your opportunity to have the speakers answer your questions!

12:50 pm Action: What Are You Now Going to Change About Your Approach to Market Access to Ensure That Stakeholders Are Fully Aware of the Value Your Drug Brings to the Market?

1:10 pm Lunch & Virtual Networking

THE ROLE OF MEDICAL AFFAIRS IN CLINICAL DEVELOPMENT

2:10 pm Discover: Leveraging the Value of Medial Affairs to Optimize Gene Therapy Clinical Development in Rare Disease

  • Jodie Gillon Vice President Patient Advocacy & Clinical Affairs, Abeona Therapeutics
  • Kent Christopherson Senior Director US Medical Affairs, Orchard Therapeutics

Synopsis

• The role of medical affairs in establishing kol relationships early in the clinical development product life cycle
• Optimizing gene therapy clinical trial design in rare disease though value driven medical affairs field engagement
• Importance of medical affairs in clinical development trial site selection, qualification, and management

2:30 pm Discover: Removing the Barriers to Effectively Communicate & Collaborate with Your Clinical Trial Operations Team

  • Thomas Board Senior Liaison Medical Science, Neurocrine Biosciences

Synopsis

• What do these barriers look like?
• How more effective communication and collaboration can lead to better site selection
• A case study on optimizing your medical affairs clinical trials strategy

2:50 pm Discover: Strategies for KOL Engagement and Gene Therapy Clinical Trials

3:10 pm Develop: What are the Keys to Success for Medical Affairs Professionals to Effectively Manage the Clinical Development of Their Gene Therapy Product?

Synopsis

This is your opportunity to have the speakers answer your questions!

3:40 pm Action: What Are You Now Going to Change in Your Approach to Clinical Development Planning to Ensure You Are Better Prepared to Meet Your Clinical Goals?

4:00 pm Afternoon Break & Virtual Networking

EVIDENCE GENERATION & LONG TERM FOLLOW UP

4:30 pm Discover: Review Aligning Medical Affairs and (Real-World) Evidence Generation Strategies – Better Together”

Synopsis

• Explore unique gene therapy real-world evidence generation needs
• Optimize medical affairs resources in support of RWE generation
• Discuss best practice examples and what can go wrong

4:50 pm Discover: Long term follow up of Gene Therapy Patients – Medical Affairs and Beyond

Synopsis

• Overview of the role registries may play in delivering LTFU data
• Case studies on the role medical affairs plays in collaborative registry development
• Future perspectives on passive data collection to deliver long term data collection

5:10 pm Develop: What Are the Keys to Success in Developing an Effective Post-Launch Strategy for Gene Therapy Products?

Synopsis

This is your opportunity to have the speakers answer your questions!

5:30 pm Action: What Will You Now Change About Your Evidence Generation & Long Term Follow Strategy to Maximise its Effectiveness?

5:50 pm Chair’s Closing Remarks