8:00 am
Registration and Morning Networking

8:30 am Chairperson’s Opening Remarks

DISCOVERING THE VALUE OF MEDICAL AFFAIRS IN EARLY STAGE DEVELOPMENT

8:40 am Assess the Value of MSL’s in Early Stage Development to Strengthen KOL Engagement and Reinforce Clinical Programs

Synopsis

  • Identify how MSL’s are engaging with KOLs earlier in development to communicate the science, technology and early phase data
  • Interactions and scientific exchange help gain insights into unmet needs, standard of care treatments and developing landscape to strengthen program development
  • Assess the advantages of building an early stage foundational knowledge to ensure proof of concept and secure advocates by phase III of development

9:10 am A Gene Therapy Platform Approach to Medical Education and Site Readiness

  • Stephen A. Kagan Global Medical Lead, Gene Therapy Platform and Cross Portfolio, Rare Disease, Pfizer

Synopsis

  • Understand the structure and role of the gene therapy platform medical team in delivering non-therapeutic area gene therapy education to HCPs (including the multidisciplinary team), patients, caregivers, payers, authorities and others
  • Understand the need to support site readiness in delivering future gene therapies
  • Understand the need and build a roadmap to upskill internal Medical Affairs colleagues on gene therapy concepts

9:40 am Best Practices to Fostering Sustainable Engagements with KOLs to Secure Early Advocates

  • Alexis Russell Senior Director, US Gene Therapy, Therapeutic Area Lead, PTC Therapeutics

Synopsis

  • In an increasingly competitive market, learn the initiatives Medical Affairs teams are adopting to differentiate themselves and ensure KOL’s prioritize time for initial meetings
  • Understand the key topics that should be covered during an initial meeting to ensure that KOL’s remain engaged and perceive the time as valuable

10:10 am
Structured Networking

Synopsis

Join us for face-to-face networking. We will pair you up with fellow attendees so you can get one on one 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.

11:10 am
Morning Coffee Break

11:40 am Leveraging Medical Affairs Preliminary Feasibility Assessments to Successfully Identify Centers of Excellence That Strengthen Gene Therapy Clinical Programs

Synopsis

  • Uncover how MSL’s are engaging KOLs early to help determine institutional gene therapy trial capabilities and capacity
  • Explore the role of field medical affairs in assisting Clinical Operations in securing potential clinical trial sites
  • Discuss how clinical insights from KOLs are shared internally to support Clinical Development in trial design and execution

LEVERAGING THE ROLE OF MEDICAL AFFAIRS IN INTERNAL CROSS-FUNCTIONAL EDUCATION

12:10 pm Identify The Importance of Medical Affairs as a Strategic Pillar For R&D Teams to Ensure R&D Are Designing Robust Clinical Programs that Meet a Distinct Unmet Need

  • Catherine Parham Vice President, Head of Global Medical Affairs, Astellas Gene Therapies

Synopsis

  • Learn how Medical Affairs teams are sharing insights on the unmet needs and recommended measurements and assessments to show efficacy that would address those unmet needs
  • Identify how Medical Affairs are in turn preparing clinical teams for the scientific and strategic industry questions

12:40 pm
Lunch & Networking

2:00 pm Identify the Innovative Formats being Adopted by Medical Affairs to Upskill Cross-Functional Teams and Ensure Internal Alignment on the Medical Strategy

Synopsis

  • Uncover the novel approach of using a white paper to provide a material for internal education and alignment on the gene therapy value proposition and the internal medical strategy
  • Assess the impact of robust internal education during early stage development to ensure consistency in communications with external stakeholders including investors and opinion leaders

2:30 pm Discover How External KOL Questions are Reinforcing Internal Training Initiatives to Prepare and Upskill Field Medical Teams During Clinical Development

Synopsis

  • Identify how Field Teams are being educated on both the principles and challenges surrounding gene therapies to ensure they are prepared sufficiently for the anticipated external questions
  • Discover the different formats being applied to internal education initiatives
  • Identify the considerations for applying internal training formats externally, and explore some of the flexible content being used in different therapeutics including on-demand bitesize modules

3:00 pm
Afternoon Networking Break

HIGHLIGHTING THE ROLE OF MEDICAL AFFAIRS IN IMPROVING THE COMMUNICATION OF MEDICAL AND SCIENTIFIC GENE THERAPY CONTENT FOR PATIENTS

3:30 pm Explore How to Apply Health Literacy Principles to Optimize Gene Therapy Patient Materials

  • Tara Moro Director, Gene Therapy Platform and Cross Portfolio, Rare Diseases, Global Medical Affairs, Pfizer

Synopsis

  • Appreciate that health literacy is how a patient can understand and act on health information, and that lower health literacy levels negatively impact the access and use of health services
  • Identify the three core principles of health literacy: readability, understandability, and actionability
  • Know how to apply health literacy principles to gene therapy patient materials to improve communication and shared decision-making between patients and health care professionals
  • Find health literacy resources that can be used to improve gene therapy patient materials

4:00 pm How to Create that ‘Aha Moment’: Inclusive Communication Tools and Guidance from a Patient Advocacy Group on Integrating Patients and Caregivers into Accessible, Gene Therapy-Focused Communications

Synopsis

  • Understand how patient advocacy groups perceive current gene therapy presentations and the top tips to improving future speaking engagements to ensure that the messaging is accessible and appropriate
  • Discuss the critical responsibility of Medical Affairs in communicating openly and honestly on the risk:benefit profile of gene therapies to ensure patient trust during early development
  • Discuss the role of Patient Advocacy Groups in Health Technology Appraisal decisions, and evaluate the unmet needs that are hindering patient recruitment

4:30 pm Identify the Education Programs for Referral Sites that Support Accurate Information Sharing with HCPs to Improve Patient Enrolment

  • Ruth Haile-Meskale Medical Science Liaison Lead, Gene Therapy, Hematology, Spark Therapeutics

Synopsis

  • Unique challenges associated with referring patients to gene therapy protocols
  • Targeted HCP education can enable informed patient referrals to gene therapy protocols

5:00 pm Chairperson’s Closing Remarks

5:10 pm End of Conference Day One