WELCOME TO GENE THERAPY PATIENT ENGAGEMENT
7:45 am Online Registration
8:20 am Chair’s Opening Remarks
EVALUATING THE CURRENT PATIENT-CENTRIC VALUES IN THE GENE THERAPY COMMUNITY
8:30 am Setting the Scene: Progress of Gene Therapy so Far in the Context of Patient Centricity
Synopsis
- Patients are central to any drug development process – what makes gene therapy unique in terms of incorporating the patient voice effectively?
- Understanding the increased industry presence particularly within rare disease, and how this has altered stakeholder perspectives and priorities
- Achieving success (or failure) of gene therapy programs is impacted by how effectively patient values are both heard and actioned
9:00 am Patient Advocacy and Gene Therapy: What’s Different?
Synopsis
- Understanding how to ensure patient advocacy and patient engagement reflect the unique challenges of gene therapy
- Identifying how to generate actionable insights along the development and commercialization continuum
- Spotlighting internal and external partnerships that tap patients and caregivers’ knowledge, experience and preferences
- Examining how companies have met the challenge of gene therapy-related patient advocacy and patient engagement
9:30 am Case Study: What Happens When the Wrong Functional Endpoint is Chosen
Synopsis
- In Duchenne there have been 29 failed trials costing the industry over $250 million and leaving families searching for hope
- Could some of the failed trials have met standards for approval had the different, patient-informed functional endpoint been selected?
- Casimir partners with stakeholders of disease communities while applying uncompromising scientific rigor to data that can inform how a patient feels, functions, or survives
- The heterogeneity of symptoms imposed by a disease determine the method(s) used for outcome measure development. Casimir relies on data from Individual Activity Assessments (IAA) to video capture
10:00 am PANEL DISCUSSION: Bringing all Stakeholders in Alignment to Work Towards a Common Goal
Synopsis
- Understanding who the stakeholders in gene therapy development are in the context of incorporating patient views
- What does ‘good’ collaboration look like between sponsors, patient advocacy groups, and patients themselves?
- Strategies to understand the needs and concerns of stakeholders, and ways these can realistically be addressed and
solved
10:30 am Virtual Speed Networking
Synopsis
Grab a cup of coffee from the comfort of your own kitchen and jump straight into this exclusive virtual speed networking! This session is the ideal opportunity to meet face-to-face (albeit 2D faces) with many of the brightest minds in the industry to establish meaningful business relationships.
EXPLORING EFFECTIVE STRATEGIES IN PATIENT IDENTIFICATION & ENROLMENT
11:30 am Collaborating with Patient Advocacy Groups on Resources to Identify Patients
Synopsis
- Making the most of existing natural history studies & patient registries
- Building out resources to find patients
- Strengthening patient and industry collaboration is key, particularly where the
space is ‘crowded’
12:00 pm Exploring Several Avenues to Maximize Patient Outreach
Synopsis
- Identifying where social media can play a potential role in raising awareness
- Does presence at patient meetings make a positive impact on the patient
community? - Understanding the importance of sponsor/clinician rapport to provide
patients with the most relevant information
12:30 pm Lunch & Networking
1:30 pm Developing a Newborn Screening Toolkit for Adrenoleukodystrophy: Key Learnings
Synopsis
- Understanding the importance of newborn screening in the context of gene therapy
- Collaborating with patient communities on development of an advocacy toolkit specifically focused on newborn screening
- Exploring what went well, but also what improvements could be made to the process for future projects given what is known now
2:00 pm Improving Diversity in Clinical Trials to More Accurately Reflect the Patient Community
Synopsis
- Building trust more effectively between sponsors and patient communities
- Investigating the reasoning behind eligibility criteria and whether those limitations are necessary
- Engaging with community partners to better understand the patient community
2:30 pm Afternoon Break & Networking
DISCUSSING APPROACHES TO MANAGE EXPECTATIONS ACROSS STAKEHOLDERS
3:00 pm Communicating Across Functional Departments on Patient Centricity Beyond the Patient Advocacy Team
Synopsis
- Exploring ways to ensure departments who are not necessarily patient-facing are able to maintain a patient-centric approach
- Collaborating with different teams to ensure the patient voice is heard for every function, including manufacturing, clinical development, medical affairs
& market access - Highlighting examples of internal alignment on patient engagement, and identifying where there is room for improvement
3:30 pm PANEL DISCUSSION: Hearing the Needs of the Patient Community: What do Patients Want to See from Industry?
Synopsis
- The problem with positioning gene therapy as a cure
- How can we hear the perspectives of the everyday patient?
- Listening to patients and making decisions based on their input are two different things. How can we close that gap?