PATIENT ENGAGEMENT IN THE CONTEXT OF GENE THERAPY: WHAT’S UNIQUE?

8:55 am Chair’s Opening Remarks

9:00 am Unique Characteristics of Gene Therapy & Their Ethical Implications For Clinical Trial Participation & Post-Approval Access

Synopsis

  • Exploring the ethical considerations relevant to gene therapy in research and clinical practice
  • Specific ethical issues relevant to conducting gene therapy research in children
  • Navigating the balance between gene therapy progress and meeting needs
    of patients, caregivers, and patient communities

9:30 am Panel Discussion: What Do Patients Want & Need from Industry?

  • Amy Fisher Head of Patient Advocacy, Spark Therapeutics
  • Pat Furlong Founding President & CEO Parent Project Muscular Dystrophy, Parent Project Muscular Dystrophy
  • Tom Croce VP, Global Patient Advocacy, Jazz Pharmaceuticals

Synopsis

For any relationship between patients and industry to work, it’s well established that the patient voice needs to be heard. However, implementing this and incorporating that voice into gene therapy development is not always actioned effectively.
This panel will discuss:
• Fostering collaboration between patient groups and industry
• Understanding what patients are looking for and how industry can provide for those needs effectively
• Sharing examples of effective and less effective collaboration to streamline future efforts
• What’s different about interactions on gene therapy compared to other modalities?

10:15 am Morning Refreshments & Structured Networking

Synopsis

Our structured networking is the ideal opportunity to get face-to-face time with colleagues and peers who are passionate about incorporating the patient voice into genetic therapy development. Introduce yourself to the attendees that you would like to have more in-depth conversations with, explore common challenges, and establish meaningful relationships to pursue for the rest of the conference and beyond.

MANAGING EXPECTATIONS OF GENE THERAPY ACROSS STAKEHOLDERS

11:00 am Setting & Managing Expectations of Gene Therapy Within The Patient Community

  • Jennifer Helfer Head, Patient Advocacy & Engagement, Encoded Therapeutics

Synopsis

• Educate along the entire research, development, and commercialization continuum
• Create a two-way dialog
• Transparency and the words we use matter
• Don’t go it alone, work with Patient Advocacy Organizations and others

11:30 am Explaining the Nuances of Gene Editing & Preparing For First-in-Human Trials

  • Jordanna Mora Senior Director, Patient Advocacy & Centricity, Beam Therapeutics

Synopsis

• Several transformative medicines are currently in development so understanding the differences between them is key
• Working with patient communities to develop information that is accessible, understandable, and relevant
• Optimizing the patient experience for first-in-human trial using base editing

12:00 pm Roundtables: Managing Expectations at Different Stages of Gene Therapy Development

Synopsis

One of the biggest challenges for genetic medicines in particular is effectively managing expectations of the therapy, from the perspectives of different stakeholders, functions, and at each stage of gene therapy development. This roundtable session will delve into some of those key milestones in development to be able to share best practice and examples of effective managing expectations, including:

 

12:30 pm Lunch & Networking

NAVIGATING INFORMED CONSENT IN GENE THERAPY TRIAL PARTICIPATION

1:30 pm Helping Patients Understand Gene Therapy: Process & Decision Points

  • Tom Croce VP, Global Patient Advocacy, Jazz Pharmaceuticals

Synopsis

• How patients consider gene therapy
• Strategies for compliant, patient-focused education on gene therapy process and support services
• Resources available that facilitate patients’ informed decision making

2:00 pm Building a Patient-Driven Informed Consent Process for a Gene Therapy

  • Pat Furlong Founding President & CEO Parent Project Muscular Dystrophy, Parent Project Muscular Dystrophy

Synopsis

• Ensuring that the informed consent process has been built using patient input
• Understanding the power of language and how we use it in the context of gene therapy, including expectations and assumptions
• How can we be confident that consent is fully informed?

2:30 pm Aligning Internally to Establish Early Patient Engagement Processes

  • Wendy Borsari Senior Manager, Patient Advocacy, Tenaya Therapeutics

Synopsis

• Building out a patient advocacy function early on in gene therapy development
• Sharing examples of strategies to align on this internally, including patient visits to the lab
• Working with R&D functions & manufacturing on effective patient input & understanding of the unique aspects of gene therapy drug development

3:00 pm Afternoon Refreshments & Networking

GENE THERAPY FIELD CHANGING DYNAMICS & THE ROLE OF PATIENT ENGAGEMENT

3:30 pm Exploring Opportunities to Establish a Pan-Therapeutic Strategy For Gene Therapy

Synopsis

• Examine the need to develop a global, pan-therapeutic strategy to support the unique aspects of gene therapies
• Discuss opportunities and best practices to glean patient advocate and community member insights on gene therapy
• Share engagement and collaboration models for collaborating with rare disease communities to build consensus and prioritize and address unmet needs

4:00 pm Building Trust With Patient Groups: Case Study in Sickle Cell Disease

  • Sara Davis Senior Director, Patient Advocacy & Government Affairs, CRISPR Therapeutics

Synopsis

• Working with different patient groups on a state-by-state basis, and exploring different outreach methods
• Bringing patients to speak to the wider company and understand their perspectives
• Bringing these perspectives through to clinical trial design and implementation

5:00 pm Chair’s Closing Remarks