Conference Day Two

Thursday 23rd March 2023

8:00 am
Conference Registration & Welcome Coffee

8:45 am
Chair’s Opening Remarks

Opening an EAP Swiftly & Effectively

9:00 am Aligning all Stakeholders During the Planning Stages of an EAP in Big Pharma vs a Start-Up

  • Ana Paula Tediosi International Expanded Access Program operation Lead, Stemline Therapeutics

Synopsis

  • Setting priorities for the internal Medical Affairs, Clinical, Regulatory, Quality, Pharmacovigilance, Supply, and Legal  
  • Aligning internal teams on goals, challenges and solutions 
  • Establishing an efficient communication structure to ensure teams remain united throughout an EAP 

9:30 am Reserved for Caligor Coghlan

10:00 am
Morning Break & Networking

Anticipating Potential Bottlenecks to the Running of an EAP & How to Overcome Them

11:00 am Roundtable Discussion:

Synopsis

This interactive session gives you the opportunity to be part of the discussion, share ideas and learn from your peers.  

 

Choose the table most relevant to you, and get ready to be part of this conversation: 

 

  • Attaining a suitable level of supply to move quickly after a request 
  • Protecting patient’s data 
  • Connecting with physicians and pharmacies 

12:00 pm Conducting an Equitable EAP for an Ultra Rare Disease in Europe: A Small Biotech Company’s Experience

  • Eva Gallagher Vice President & Head of Medical Affairs, Senior Director, Medical Science Liaison Field Team Medical Affairs, Agios

Synopsis

  • Effectively utilizing internal resources in the absence of a large internal framework  
  • Providing drugs in countries where there is approval but no means to commercialize  
  • Balancing costs while reaching as many patients as possible  

12:30 pm
Lunch Break & Networking

1:30 pm Panel Discussion: Navigating the Challenges of Operationalizing an Early Access Program for Rare Diseases

  • Tiffany Kawai Director, Program Management and Operational Excellence, Global Oncology Medical Affairs, Daiichi Sankyo, Inc.
  • Jeff Waldron Executive Director, Sherborn Consulting
  • Eva Gallagher Vice President & Head of Medical Affairs, Senior Director, Medical Science Liaison Field Team Medical Affairs, Agios

Synopsis

  • Guaranteeing that people with rare diseases get early access to treatments 
  • Managing marketing, commercial, logistic and supply chain costs  
  • Generating endpoints data in EAPs to provide evidence for regulatory filings 

2:15 pm Distinguishing When to End an Early Access Program & Move to Commercialization

  • Kjersten Teeter Director & Head Of Medical Affairs Operations, Blueprint Medicines

Synopsis

  • Outlining when the drug is no longer considered investigational and is commercially viable  
  • Navigating the ethics of ending an EAP 
  • Establishing post-trial access to treatment to continue benefits for the patient

2:15 pm Access Program across the Product’s Regulatory Path

  • Michelle Clausen Senior Director - Expanded Access & Enterprise Medical Community, Pfizer

Synopsis

  • Review Expanded Access request volume across countries over time
  • Considerations such as supply availability, internal stakeholder engagement,
    program eligibility criteria, request management
  • Country challenges including opening / closing EA program

2:45 pm
Afternoon Break & Networking

Anticipating Potential Bottlenecks to the Running of an EAP & How to Overcome Them

3:15 pm Interactive Group Discussion – Comparing & Contrasting Strategies for Planning & Running a Single Patient Program vs an EAP

Synopsis

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: 

 

Understanding submission requirements for regulatory agencies  

Attaining the appropriate quantity of drugs  

Handling unexpected requests in an ethical manner 

3:45 pm Harnessing Innovation to Create a Patient Centric EAP

  • Dorian Readnour Executive Director, Global Patient Access, Sarepta Therapeutics

Synopsis

  • Developing a unique model to support complex medicines in small populations 
  • Integrating and managing home health solutions into your EAP 
  • Identifying unlikely champions within your own organization  

4:15 pm
Chair’s Closing Remarks & End of Conference

All times shown in EST