All times shown in EST

8:30 am Virtual Coffee Morning

8:50 am
Chair’s Opening Remarks

Reviewing the Global Regulatory Environment

9:00 am Overview of Project Facilitate and Expanded Access in Oncology

  • Tamy Kim Director for Regulatory Affairs and Policy Oncology Center for Excellence (OCE) , FDA

Synopsis

  • Detailing requests received and where they come from
  • EAP data and use in product approvals
  • EAP in specific disease areas

9:30 am UK Approaches to Early Access: Lessons Learned and Future Directions

Synopsis

• Evolving the Early Access to Medicines Scheme (EAMS)
• Public consultation; creating a legal framework for Early Access
• Balancing EAMS approach versus other early access flexibilities

10:00 am
Networking Break

10:30 am Examining the Regulatory Policies in Emerging Markets to Run a Global Expanded Access Program

Synopsis

• Addressing the challenges of operationalizing an expanded access program in LATAM counties
• Navigating the patchwork of expanded access program regulations in Asia Pacific
• Discussing the unique operational challenges posed in emerging markets

Improving Internal Collaboration to Maximize Expanded Access Program Success

11:00 am Unpacking Expanded Access Regulations in the United States

  • Richard Klein Director, Expanded Access Programs and Policy, GE2P2 Global Foundation

Synopsis

  • Exploring the different kinds of expanded access for the treatment use of investigational agents
  • Discussing what kinds of access are possible under FDA regulations
  • Assessing who is eligible for expanded access and who makes the decisions

 

11:30 am Proactively Preparing for Operational Success

  • Paula Singer Senior Manager, Global Medical Affairs, Early Access, Alnylam Pharmaceuticals

Synopsis

• Factoring operational challenges into the planning stage
• Aligning internal teams for operational success

12:00 pm
Lunch

1:00 pm Panel Discussion: Working Collaboratively to Develop In-House Expanded Access Programs Expertise

  • Marjorie Zettler Director of Clinical Science, Regor Pharmaceuticals
  • Christian Valencia Medical Director, Global Medical Affairs Oncology, EMD Serono
  • Helen Kellar-Wood Head, Pre-Approval Access Operations, Global Development Operations, Bristol Myers Squibb
  • Rachel Harrison Associate Director, Early Access Programs, Apellis Pharmaceuticals

Synopsis

• Securing buy-in across departments, including medical, regulatory, and development
• Outlining the benefits of creating an in-house team for expanded access programs
• Leveraging existing knowledge across teams to build cross functional expertise quickly

1:45 pm Expanded Access Data Uses and Collection Strategies – Research and Insights

Synopsis

• Use cases of FDA/EMA and NICE/ICER decisions that include Expanded Access data
• Expanded Access RWD collection strategies and additional potential uses
• Lessons learned to support decision making when considering RWD collection

2:15 pm
Networking Break

2:45 pm Harmonizing Expanded Access Programs Across your Business

  • Kathleen Long Director, Medical Affairs Operations, Alkermes, inc.

Synopsis

• Building standard operating procedures across programs to build consistency
• Educating key stakeholders on the strengths and limitations of expanded access programs
• Designing frameworks to assess establishing an expanded access program quickly

3:15 pm Software to unlock efficient collaboration and decision-making regarding Expanded Access

Synopsis

• Real-time visibility and control over programs and treatment requests for life
sciences companies and CROs/clinical service providers
• Complying with regulatory frameworks including those pertaining to patient
data
• Features including case-based management of teams, roles, documents, and
communications
• Flexible, open software allows integration with shipment, supply, single sign-on,
and other 3rd-party systems

3:30 pm Interactive Round Table Discussions

  • Karen Frascello Director, Global Medical Affairs, Early Access , Alnylam Pharmaceuticals
  • Donna Cowan Associate Director Expanded Access Programs and Registry, Stealth BioTherapeutics

Synopsis

  • Real World Data
  • Ethical Considerations
  • Regulatory Challenges
  • Preparation for an Expanded Access Program

This interactive session will give you the opportunity to dive into the conversation with your fellow colleagues to discuss, debate and get inspiration on how to approach specific challenges. Choose the table most relevant to you, and get ready to be part of this conversation!

4:15 pm
Chair’s Closing Remarks