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 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
12:00 pm Fireside Chat: From Plan to Execution: Aligning Stakeholders to Adopt the Right Technology for an Expanded Access Program
Synopsis
- Retire the spreadsheets – gain the audit trail: move from scattered Excel trackers to a validated digital workflow that logs every approval, version, and comment automatically, slashing manual reconciliation time and ensuring compliance
- Unify a global, cross-functional team in one workspace: choose a cloud-based, GxP-ready platform that lets medical, regulatory, supply, and country affiliates collaborate in real time with role-based permissions and e-signatures
- Frame it before you launch it: map stakeholders, define SOPs, and validate the system during planning so your Expanded-Access Program can scale smoothly and handle an FDA audit
12:30 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
1:00 pm Lunch Break & Networking
2:00 pm Presented by myTomorrows: Motivations for Pursuing Expanded Access: Strategic Value and Drivers for Stakeholders
Synopsis
- Explain the strategic benefits of initiating Expanded Access Programs (EAPs) early in drug development, including their role in generating real-world insights, informing clinical and commercial strategies, and supporting global access planning
- Identify key stakeholder engagement opportunities, including with KOLs, patient advocacy groups, regulators, payers, and internal teams, and describe how Expanded Access fosters collaboration, trust, and early alignment across the development lifecycle
- Discuss the operational and logistical considerations for implementing EAPs effectively, including proactive planning for supply, data collection, and regulatory coordination
2:30 pm Roundtable Discussion: Securing Stakeholder Buy-In: Strategies for Driving Successful Program Implementation
Synopsis
- How do you identify and engage the key stakeholders who are critical for securing program buy-in?
- What criteria do you use to determine the right team or resources to effectively operationalize EA programs
- What common concerns or "pain points" do stakeholders typically raise, and how can these be proactively addressed to support successful adoption and implementation?
3:00 pm Afternoon Break & Networking
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?