Conference Day One

Wednesday 22nd March 2023

7:00 am
Conference Registration & Welcome Coffee

8:15 am
Chair’s Opening Remarks

Navigating Global Regulations to Implement EAPs at a Global Scale

8:30 am A Small Company Approach to Early Access Programs in Canada, Europe & the United States: Key Similarities & Differences

Synopsis

  • Underscoring importance of strategic cross functional and cross regional alignment to ensure successful early access programs 
  • Understanding regulatory and medical community perspectives across regions as it relates to early access programs 
  • Outlining strategic and tactical considerations for early access programs across regions in a small company 

9:00 am Panel Discussion: Navigating Global Regulations to Effectively Run a Global EAP

  • Mario Aguiar Director - Global Medical Affairs & Rare Diseases, Sanofi Genzyme
  • Richard Klein Director - Expanded Access Programs & Policy, GE2P2 Global Foundation

Synopsis

  • Addressing differences between EAPs globally and how to implement them 
  • Effectively conducting programs in countries with regulatory gaps 
  • Outlining methods to effectively engage with regulatory bodies  

9:45 am Reserved for myTomorrows

  • Anne Cropp Chief Scientific Officer, Early Access Care

10:15 am
Speed Networking

Synopsis

Bump elbows with your fellow EAP enthusiasts! This is your opportunity to network and forge new contacts with peers in the industry. Join speed networking to meet a broad spectrum of attendees at the summit, and exchange details to catch up later in the event. 

10:45 am
Morning Break

11:15 am Getting an EAP Off the Ground

  • Rachel Harrison Associate Director - Early Access Program, Apellis Pharmaceuticals

Synopsis

  • Aligning internal teams on goals, challenges and solutions 
  • Understanding supply, scope and equitability  
  • Establishing the scope of an EAP within budgets 

11:30 am
Explaining the Value Proposition of EAPs to Each Stakeholder

11:45 am Devising a Strategy to Convince Internal Stakeholders to Run an EAP

Synopsis

  • Attaining the appropriate resources from internal stakeholders for a viable EAP  
  • Conveying the value proposition of EAPs  
  • Outlining the benefits of EAPs to the companies that run them  

12:15 pm Effectively Educating & Upskilling Internal Teams Who Are Involved With EAPs

  • Bethany Bearden Senior Manager - Medical Affairs Operations & Project, Blueprint Medicines

Synopsis

  • Creating a robust, thorough training program that covers the background, planning and execution of an EAP  
  • Distinguishing the key differences between EAPs and clinical trials  
  • Building on clinical trial experience to understand timelines, use of data and patient experience 

12:45 pm
Lunch Break & Networking

Explaining the Value Proposition of EAPs to Each Stakeholder

1:45 pm Panel Discussion: Closing the Gap of How we Reach Patients

  • Jeff Waldron Executive Director, Sherborn Consulting
  • Machelle Manuel VP Medical Affairs, Amylyx Pharmaceuticals Corp.
  • Merideth Rodgers Global Director - Medical Affairs, Hematology & Rare Disease, Regeneron Pharmaceuticals Inc

Synopsis

  • Engaging with patient advocacy groups 
  • Building a patient advisory panel to understand patient expectations  
  • Compliantly promoting EAPs to increase patient awareness   

2:30 pm Expanded Access following Clinical Trial or Open Label Extension

  • Anne Cropp Chief Scientific Officer, Early Access Care

Synopsis

  • Characterizing phase 2/3 trial for post-trial EAP 
  • Effectively planning for transition to EAP with your team 
  • Transitioning to EAP with a post-trial placebo-arm 
  • Debating options for data collection  

2:30 pm Reserved for myTomorrows

3:00 pm
Afternoon Break & Networking

3:30 pm Panel Discussion: Building a Mutually Beneficial Relationship with External Stakeholders

  • Christine Istanboulian Operations & Efficiency Lead - Managed Access & Humanitarian Programs, Sanofi Genzyme
  • Allison Webb Medical Affairs Operations Project Manager, Blueprint Medicines
  • Omar Rahman Associate Director - Expanded Access Program, Argenx

Synopsis

  • Establishing KPIs, transparency into metrics and visibility into systems 
  • Aligning sponsors, CROs, clinical trial sites and physicians to execute an effective EAP  
  • Ensuring there's a relationship and confidence between both the sponsor and the external stakeholder  

Opening an EAP Swiftly & Effectively

4:15 pm Assessing the Cost & Benefits of an EAP

Synopsis

  • Balancing the cost of planning, designing and implementing a successful EAP 
  • Considering reimbursement options  
  • Strategically planning an EAP to be cost-neutral or revenue generating  

4:45 pm Global Medical Affairs Leadership in Driving EAPs

  • Liliana Alfaya Medical Affairs and Strategic Collaborations Executive,

Synopsis

  • Key principles of developing the medical strategy for an EAP   
  • Deciding whether to open an access program only in countries where clinical trials are established  
  • Establishing the scope of an EAP within budgets 
  • Fairly and equitably deciding where to run an EAP 

5:15 pm
Chair’s Closing Remarks

All times shown in EST