Conference Day One
Wednesday, July 30
8:00 am Check-In & Breakfast
8:45 am Chairperson’s Opening Remarks
Turning Regulatory Uncertainty into Opportunities: Navigating Diverse Frameworks to Enhance Global Patient Access
9:00 am Fireside Chat: Adhering to Appropriate Regulatory Guidelines to Optimize Expanded Access Programs in the US & Europe
Synopsis
- Delving into the constantly changing regulatory landscape to ensure you stay ahead of the curve and are able to design compliant strategies for successful global expansion
- Understanding FDA guidelines for expanded access to ensure effective, streamlined operations
- Navigating country-level regulatory standards within Europe, addressing the opportunity for harmonization amongst distinct governing bodies
10:00 am Clarifying Regulatory & Operational Differences Between Cohort & Individual Patient Programs to Align Program Design with Target Populations
Synopsis
- Unpacking important distinctions between cohort and individual patient regulatory pathways and impact on program design
- Exploring cost and resource considerations, including the need for a CRO, which are required to execute cohort and individual patient programs
- Implementing the obligatory commitments of running a cohort program into the preexisting company infrastructure
10:30 am Morning Break & Speed Networking
Synopsis
Join our speed networking session tailored for expanded access professionals, like yourselves, to connect with fellow industry peers to facilitate rapid yet meaningful exchanges of insights and expertise. Elevate your networking experience during this session designed for impactful connections within the space.
Optimizing Stakeholder Alignment: Improving Knowledge-Sharing & Transparency to Drive More Effective Pre-Approval Access Delivery
11:30 am Fireside Chat: Facilitating More Effective Stakeholder Engagement, From Patients & Physicians to Biopharma & Regulators, to Ensure Program Optimization, Regulatory Compliance & Sustainability
Synopsis
- Identifying relevant stakeholders, both internal and external, to engage for expanded access planning and management
- Understanding what the differing pain-points faced by each stakeholder are, and the overall impact
- Considering how strategic expanded access planning can maximize impact for different stakeholders
12:30 pm Securing Corporate Buy-In: Engaging Internal Stakeholders with Timely, Targeted Information to Ensure Seamless & Effective EAP Implementation
Synopsis
- Identifying the appropriate cross-functional teams and their varying roles and remits to ensure understanding, alignment and harmony across the organization
- Mitigating variable expectations for expanded access operations compared to clinical trials, enabling efficient yet compliant execution
- Considering appropriate timing for expanded access planning along the development path, and variable considerations at key milestones ensuring streamlined decision-making through early and targeted discussions
1:00 pm Lunch Break & Networking
2:00 pm Presented by myTomorrows: Motivations for Pursuing EA: Strategic Value & Incentives for Stakeholders
Synopsis
- What are the benefits and advantages of thinking about EA early on?
- How EA can supplement drug development activities and inform commercial launch
- Leveraging multi-stakeholder engagement opportunities
2:30 pm Fostering Collaborative Partnerships: Enhancing Patient Advocacy Relationships Through Open & Productive Communication to Work in Concert
Synopsis
- Recognizing patient advocacy groups as the critical voice of the patient and a vital stakeholder when managing expectations amongst patients on when and if expanded access may be considered as part of a development program
- Establishing meaningful relationships through early engagement, exploration of common pain points and utilization of available resources to boost alignment between biopharma and advocates
- Understanding the needs of patients and establishing proactive solutions for the benefit of all stakeholders
3:00 pm Afternoon Break & Poster Presentation
Synopsis
Take this opportunity to showcase your latest research and innovations with your peers and understand the strategies of your fellow expanded access experts. Visit the website for the full T&Cs of submitting a poster.
Cell & Gene Therapies: The Ideal Candidate for Expanded Access, or an Unfeasible Proposition?
3:30 pm Fireside Chat: Exploring the Potential Opportunities & Risks of Expanded Access for Cell & Gene Therapies
Synopsis
- Is it appropriate to consider cell and gene therapies in the same or similar to drugs and biologics?
- When is the right time to provide expanded access for cell and gene therapies?
- What are the specific considerations related to expanded access for these modalities, including cost, supply chain logistics and drug availability?
- What are the potential risks of providing expanded access for single dose treatments, such as impact on addressable commercial market and reimbursement challenges for these costly therapies?
- How do issues of immunogenicity factor into patient decisions to pursue pre-approval access for cell and gene therapies?