DAY TWO - Friday May 31, 2024

8:00 am Check in & Refreshments

8:50 am Chair’s Opening Remarks

ELEVATING THE PATIENT VOICE IN GENE THERAPY DEVELOPMENT

9:00 am Integrating All Departments into Patient Engagement Initiatives for Gene Therapy Companies

  • Amanda Rohrig Director, Patient Advocacy, BridgeBio Gene Therapy

Synopsis

  • Highlight the challenge of engaging colleagues from non-scientific departments and the importance of recognizing and respecting diverse perspectives within the organization
  • Explore the variety of tools employed to connect colleagues to the patient experience, ranging from in-person events to podcasts and video interviews
  • Discuss the role of incentives in encouraging colleagues to participate in patient advocacy events, such as leadership endorsement
  • Highlight the mutual benefit of patient engagement efforts, not only in sharing the patient voice but also in facilitating colleagues’ ability to directly hear from the community

9:30 am Navigating Business Demands and Upholding Patient-Centric Values in Gene Therapy

  • Natacha Raphael Former Regional Head, Corporate Affairs and Patient Advocacy, Biogen

Synopsis

  • Exploring struggles faced by gene therapy companies in balancing business priorities and clinical development timelines
  • Discussing the role of advocacy individuals in these companies as voices for prioritizing the patient community
  • Investigating case studies and insights from industry leaders, encouraging collaboration and sharing best practices

10:30 am Break & Networking

10:30 am Considerations and Strategies with Early Patient Engagement and Regulatory Integration for Gene Therapy Drug Development

  • Tiara Johnson Global Patient Affairs, BioMarin Pharmaceutical
  • Jewell Martin Director US Regulatory Policy, BioMarin Pharmaceutical

Synopsis

  • Explore the pivotal role of early patient integration in shaping regulatory decisions for gene therapy programs.
  • Understand how elevating the patient voice from the outset enhances program development and addresses unmet needs.
  • Delve into effective strategies for integrating patient perspectives into the regulatory decision-making process.

11:30 am Panel Discussion: Elevating the Patient Voice with Early Engagement in Gene Therapy Development

  • Tara Hastings Patient Engagement Lead, Rare Diseases, Sanofi
  • Kara Fick Director - Patient Advocacy & Medical Affairs, Atsena Therapeutics
  • Nan Doyle Principal, Takeda Pharmaceutical Co. Ltd.
  • Jennifer Helfer Patient Advocacy & Engagement, Viridian Therapeutics
  • Su Smith Head of Patient Strategy, Origins

Synopsis

  • Exploring challenges in gene therapy development where regulatory approval often overshadows patient-centric initiatives
  • Discussing consequences of sidelining patient voices and the impact on the success of therapeutic interventions
  • Analyzing ongoing efforts in advocacy to emphasize the significance of including patient perspectives from the outset of gene therapy development
  • Sharing strategies and best practices for educating clinical and regulatory professionals on the importance of integrating the patient voice

BRIDGING DIVERSITY: A GLOBAL LENS ON GENE THERAPY TRIALS

12:15 pm Gene Therapy Patient Care: Fostering Diversity and Inclusion through Inclusive Care Coordination

Synopsis

  • Exploring challenges in diverse communities, including unique barriers faced by different ethnic and cultural groups
  • Sharing insights on the varied trust dynamics within different communities
  • Highlighting strategies for improved patient access and inclusivity in gene therapy

STRATEGIC APPROACHES TO LONG-TERM ENGAGEMENT: ADDRESSING BARRIERS AND ALIGNING EXPERIENCES

12:45 pm Navigating the Shift to Remote Gene Therapy Trials and Sustaining Success Beyond COVID

  • Amanda Rohrig Director, Patient Advocacy, BridgeBio Gene Therapy
  • Chrissy Burton Senior Director Clinical Operations, BridgeBio Gene Therapy

Synopsis

  • Explore how the changes implemented during the pandemic were sustained and evolved beyond; emphasizing the ongoing commitment to keeping families engaged and minimizing travel-related challenges
  • Delve into the logistical and operational challenges encountered during the transition to remote options, with a focus on providing insights into overcoming the learning curve associated with adopting virtual platforms
  • Discuss the sustainability of the remote approach and its potential impact on the future landscape of gene therapy trials.
  • Share key takeaways, lessons learned, and best practices from the experience of navigating the shift to remote options

1:15 pm Lunch & Networking

2:15 pm Navigating Patient Adherence in Ongoing Gene Therapy: From Trials to Real-Life Experience

Synopsis

  • Explore the unique challenges in maintaining patient adherence in an ongoing gene therapy, considering factors such as travel, patient advocacy, and the perceived benefit of participation
  • Discuss effective communication strategies post-approval to educate patients about the expected results, emphasizing the importance of clear and relatable messaging to ensure continued patient engagement
  • Examine how lessons learned from diverse therapies can be applied to improve patient adherence and satisfaction

2:45 pm Revolutionizing Gene Therapy Trials with Decentralized Manufacturing and Patient-Centric Approaches

Synopsis

  • Exploring the challenges and opportunities associated with decentralizing gene therapy trials
  • Addressing the attachment of physicians from a sponsor’s perspective and the complexities in oversight when patients are assessed at multiple sites
  • Assessing the challenges of implementing home healthcare in gene therapy trials
  • Investigating innovative approaches, such hybrid models, to enhance the patient experience in gene therapy trials

APPROACHES TO EQUIP COMMUNITY ADVOCATES WITH THE KNOWLEDGE AND CONFIDENCE TO COMMUNICATE GENE THERAPY TRIALS EFFECTIVELY

3:15 pm Mastermind Session: Empowering Community Advocates for Effective Patient Education, Recruitment, and Support

Synopsis

  • Leveraging the power of patient advocates in the recruitment process
  • Exploring collaborative efforts to empower advocates with the knowledge needed to drive informed decisions and engagement within their communities
  • Facilitating training programs and resources to equip advocates with the latest information, ensuring they serve as informed and effective ambassadors for gene therapy trials

4:00 pm Chair’s Closing Remarks