Conference Day Two

Thursday, July 31

8:00 am Check-In & Welcome Breakfast

8:45 am Chairperson’s Welcome Address

  • Vanessa Mora Senior Case Manager, Ultragenyx Pharmaceuticals

When Should an Expanded Access Program Start to Sunset: Re-Thinking Exit Strategies, Reimbursement Opportunities & Transitions to Commercial Products or Termination

9:00 am Fireside Chat: Developing Thoughtful & Transparent Exit Plans to Effectively Manage Expectations & Minimize Potential HCP Burden

  • Annie Drelles Senior Director & Head, Office of Medical Access, Global Oncology Medical Affairs, Daiichi Sankyo
  • Vanessa Mora Senior Case Manager, Ultragenyx Pharmaceuticals

Synopsis

  • Developing a clear exit strategy through successful forward-planning that enables seamless transition of patients from expanded access, to commercial availability or other milestone-based activities
  • Mitigating the impact of terminating expanded access through effective, transparent communication with external stakeholders
  • Developing a closure process with internal stakeholders to ensure a compliant transition for expanded access patients in each participating country

10:00 am Roundtable Discussion: Breaking Down Siloes Through Effective, Proactive Collaboration for a Successful Commercial Launch

Synopsis

  • Discussing how to effectively leverage the expertise of Market Access teams while preserving the core values of early access initiatives
  • Understanding the value of gaining input from commercial teams to define the most appropriate strategy for your program
  • Defining the value of leading early discussions with local health ministries and patient advocates prior to launching your expanded access program to highlight future commercial paths within different countries

11:00 am Morning Break

11:30 am Strategic Considerations for a Patient-Centric Approach in the Route to Commercialization

  • Debra Litwak Senior Director, Global Medical Affairs - Investigator Initiated Trials & Expanded Access Programs, BeOne Medicines
  • Anita Cruz Manager, IIT & Expanded Access Programs, Global Medical Affairs, BeOne Medicines

Synopsis

  • Examining the ethical considerations on when and where is it appropriate to charge for access
  • Balancing the pre-commercialization price with providing equitable access and the impact on future commercial pricing levels
  • Unpacking the impacts of charging for unlicensed drug in regions without commercialization intention

Developing Proactive Strategies for Post Trial Access & Equitable Programs to Ensure “No Patient Left Behind”

12:00 pm Looking to the Future: Planning Ahead for Post-Trial Access to Create Equitable Solutions for All Stakeholders Involved

  • Ben Rotz Associate Vice President - Global Medical Policy, Strategy, & Operations, Eli Lilly & Co.

Synopsis

  • Ethical considerations on the decisions to provide post-trial access to investigational medicine
  • Defining an effective transition strategy with minimal disruption to patient care, regulatory compliance, and overall program efficiency through careful coordination and early engagement with clinical trial teams
  • Understanding the different post-trial access regulations and policies from country-to-country and factors to consider when determining where to provide post-trial access

12:30 pm Expanding Patient Reach in EAPs: Overcoming Operational & Regulatory Challenges to Address Global Health Inequity related to Treatment Access

Synopsis

  • Sustainability Challenge: Importance of sustainable medical access in low-to-medium income countries (LMICs) and emerging markets
  • Stakeholders Engagement: The role of governments, NGOs, private sector, and international organizations
  • Case Studies: Brief overview of successful models and key gaps that remain unaddressed

1:00 pm Lunch Break & Networking

Overcoming Supply & Labelling Hurdles to Drive Practical Supply Management in Expanded Access

2:00 pm Balancing Clinical Trial Requirements & EAPs Through Effective Inventory Management to Ensure Appropriate Resource Allocation

Synopsis

  • Enabling appropriate forecasting of supply through effective technology integration to satisfy clinical trial needs alongside, post-trial access requirements, and anticipated expanded access demand of these drugs for patients in need
  • Lessons learned from the delivery of a pre-approval programme alongside clinical pipeline progression

Leveraging Real-World Data from Expanded Access to Support Regulatory Approvals & Reimbursement Strategies for Better Patient Outcomes

2:30 pm Differentiated Approach to EAPs Using a Reimagined Model for Advancing Therapeutics

Synopsis

  • Analyzing a novel funding model that is solely focused on delivering “hope for life” to patients
  • Exploring lessons learned about gathering actionable clinical insights from the program
  • Compliantly tracking longitudinal patient data to enable actionable insights, supplement clinical trial data, inform regulatory decision-making and advance cancer treatment decisions

3:00 pm Unpacking the Ethical Considerations for Informed Consent within Expanded Access to Effectively Manage Patient and Sponsor Expectations

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

Synopsis

  • Exploring the ethical importance of informed consent and assent in Expanded Access to ensure patients understand the potential risks and benefits of these investigational drugs
  • Highlighting the sponsor responsibility of creating a minimal programmatic standard for consent and ensuring that effective informed consent/assent has been successfully secured from the patient
  • Considering the additional ethical obligations to patients in the context of collecting RWD including how it will be protected and used, both now and in the future

3:30 pm Chair’s Closing Remarks

  • Vanessa Mora Senior Case Manager, Ultragenyx Pharmaceuticals

3:45 pm End of Operationalize: Expanded Access Programs Summit West