8:50 am Chair’s Opening Remarks

9:00 am Hearing the Regulatory Perspective on Patient Input for Gene Therapies

Synopsis

  • Understanding FDA guidance around patient input and how this applies to gene therapies
  • Aligning regulatory expectations, patient needs, and sponsor responsibilities
  • Highlighting examples of initiatives the FDA are putting in place to learn from patients

RETAINING PATIENTS ON GENE THERAPY CLINICAL TRIALS WITH SIGNIFICANT FOLLOW-UP TIMES

9:30 am Developing Remote Clinical Assessments to Reduce Patient Burden & Support Long-Term Patient Commitment

  • Linda Lowes Principal Investigator, Center for Gene Therapy, Nationwide Children’s Hospital

Synopsis

  • Reducing functional assessments could reduce the patient burden – can this realistically be achieved?
  • What are the potential draw-backs of using caregivers as clinical evaluators?
  • In the context of the current pandemic, developing a remote assessment method is more important than ever – can we rely on patient reported outcomes (PRO)

10:00 am PANEL DISCUSSION: What Does Committing to a Gene Therapy Clinical Trial Mean?

Synopsis

  • Aligning expectations across stakeholders is vital before any agreement to participate is made
  • What are patients willing to do, and what are they not? At what stage should this be determined?
  • Exploring the implementation of plain language summaries

10:30 am Morning Break & Networking

INCORPORATING PATIENT INPUT INTO CLINICAL TRIAL DESIGN & DEVELOPMENT

11:00 am Designing Bespoke Clinical Endpoints with Meaning to Patients and Regulators

Synopsis

  • Creating tools that enable effective assessment of an endpoint that is both measurable and meaningful
  • Bringing in the patient perspective– through what medium is this most effectively achieved?
  • Developing a suite of secondary endpoints that will support the primary endpoint mirroring what the patient cares about

11:30 am Creating a Natural History Study Through Comprehensive Stakeholder Engagement

Synopsis

  • Designing methods for building natural history studies to serve as external controls, reduce the burden for patient participation in clinical trials, and provide innovative methods for the use of natural history study data to help accelerate therapeutic drug development and regulatory decision-making
  • Establishing and engaging with multi-stakeholder advisory boards in every step of the research process.
  • Highlighting ways the study was improved via patient input, and where to learn from for the future

12:00 pm Lunch & Networking

DEVELOPING EDUCATIONAL MATERIALS TO EFFECTIVELY DISTRIBUTE THE MOST RELEVANT INFORMATION

1:30 pm Developing and Distributing Educational Materials on Gene Therapy in a Digital World

  • Edgar Vega Senior Manager, Patient Advocacy, uniQure

Synopsis

  • Understanding the challenges of distributing educational materials to raise awareness
  • Exploring the role of digital patient conferences and virtual booths as a way to disseminate information
  • How have outreach methods evolved now that meeting in person is not a viable option?

2:00 pm Developing Materials on Gene Therapy Education & the Complexities of Gene Therapy Manufacturing

  • Nita Patel Senior Director, Patient & Professional Advocacy, Amicus Therapeutics

Synopsis

  • Developing comprehensive educational resources on understanding what gene therapy is
  • Including manufacturing as part of the educational materials and working with patient communities to ensure they are hitting the right points

2:30 pm PANEL DISCUSSION: What Role and Responsibility do Gene Therapy Companies Have as a Source of Relevant Information for their Communities?

  • Edgar Vega Senior Manager, Patient Advocacy, uniQure
  • Nita Patel Senior Director, Patient & Professional Advocacy, Amicus Therapeutics
  • Brian Robinson Senior Vice President & Head of Global Medical Affairs, Freeline Therapeutics

Synopsis

  • Understanding the role sponsors could and should play in educating communities more widely
  • Exploring the areas where patient advocacy groups are best utilized in the context of gene therapy education
  • Highlighting examples of successful strategies, and room for improvement, around gene therapy education from an industry perspective

3:00 pm Afternoon Break & Networking

COLLABORATING WITH PATIENT ADVOCACY GROUPS: NICE TO KNOW VS. NEED TO KNOW

3:30 pm Strengthening a Partnership to Last throughout Gene Therapy Development and into Commercialization

  • Erin Frey Senior Director of Advocacy, CureDuchenne

Synopsis

  • Gathering and sharing patient insights before, during and after the drug development process
  • Program development that matches the patient journey
  • Values and principals in listening to patient communities

4:00 pm Leveraging Experience from the Foundation for Angelman Syndrome Therapeutics (FAST) on Patient Engagement in Drug Development

Synopsis

  • Outlining the work FAST has done around engaging with patients to support novel outcome measures
  • Understanding how to leverage relationships with patient communities on development of therapies

4:30 pm Chair’s Closing Remarks

4:40 pm Close of Day Two & End of Gene Therapy Patient Engagement 2021