Conference Day One

Day One

Wednesday March 20, 2024

Day Two

Thursday March 21, 2024

7:25 am Check In & Morning Coffee

8:25 am Chair’s Opening Remarks

Spotlighting the Latest Case Studies Detailing the Successes of US & Global Programs

8:30 am Panel Discussion: Global Trends Within Expanded Access & Its Evolvement For All Stakeholders

Synopsis

  • Role of US Expanded Access vs. outside-US
  • Recent regulatory trends ex-US
  • How emerging markets are increasingly coming into play?
  • Addressing the downstream impact on companies' reimbursement status in particular countries

9:00 am Facilitating Treatment Access for Patients Across the Globe

Synopsis

• Delving into the geographic reach of the program

• Long-term commitment to patients

• Creation of a sustainable treatment environment, including market access and patient advocacy

9:30 am Taking it Global: Strategically Designing Expanded Access Programs to Balance the Benefits Vs. Risks

Synopsis

  • Epanded Access Program: The evolving regulatory landscape 
  • Quality Risks: Safety reporting and other GxP considerations 
  • Navigating Local/regional regulatory requirements

10:00 am Cracking the Code to Post-Trial Access

Synopsis

  • Key ways in which post-trial access differs from an extension study
  • Regulatory considerations for post-trial access
  • Opportunities for data collection

10:30 am Speed Networking Break

Synopsis

This session is a great opportunity to introduce yourself to the attendees that you would like to have more in depth conversations with. This session is the ideal opportunity to get face-to-face time with many of the brightest minds working in the EAP field and establish meaningful business relationships

11:30 am Operationalizing an EAP for Pediatric Cancer Patients

  • Joanie Shea Director Medical Affairs, Day One Biopharmaceuticals Inc.

Synopsis

• Sharing successes from a small company taking a program global for an ultra-rare disease

• Maximizing vendor partnership

• Strengthening cross-functional relationships

12:00 pm A Small Pharma Approach to Global Access: Case Study in Cohort Programs

  • Mallory Fredericks Associate Director, Medical Research & Value Demonstration, SpringWorks Therapeutics

Synopsis

• Developing an OUS strategy as a company without a global presence

• Aligning internal stakeholders on submissions for different countries

• Highlighting the importance of vendor resources involved in cohort programs and the regulatory intel they provide

12:30 pm Roundtable Discussion: Mitigating Timelines in LATAM Regions & Improve Relationships with Physicians in Countries with Less Established Regulations

  • Annie Drelles Director & Head, Office of Medical Access, Daiichi Sankyo, Inc.

Synopsis

• Clearly defining what needs to be submitted to the regulatory authorities

• Understanding how to navigate both group and individual patient programs

• What are the necessary labelling and regulatory requirements?

1:00 pm Lunch Break & Networking

2:00 pm What to Consider When Moving Beyond US-Only Expanded Access Programs

Synopsis

  • Understanding all strategic and operational considerations (Policy, supply-chain, PV etc.)
  • Navigating various regulatory archetypes, including country-specific examples. 
  • Effective allocation of company resources for a phased-approach towards commercialization.

2:30 pm Roundtable Discussion: Dare to Care. Early Access Excellence Amidst Sanctions

  • Mark Bainbridge Senior Director, Business Development & Early Access, Early Access Inceptua Group
  • Paul Stanton Senior Director, Global Strategy, Early Access Inceptua Group

Synopsis

  • Delving into factors which drive your company’s approach to running an EAP in Sanctioned Countries
  • What kind of due diligence activities will you undertake to compliantly supply medicine into a sanctioned country?
  • What are the biggest challenges to running an EAP in these countries? 

A Physician’s Perspective: Evaluating the Steps Forward Towards Advancing EAPs by Grasping the Experiences of Patients & Physicians

3:00 pm Bringing Life Saving Therapeutics to Patients & Their Execution

  • Ildiko Csiki Chief Medical & Strategy Officer, Riboscience

Synopsis

• Overcoming the huge unmet need globally

• Highlighting key successful case studies

• Challenges with engaging physicians and patients and unique solutions to overcome this

3:30 pm Expanded Access Drugs in Care: Bridges Built Upon Compassion, Perseverance & Innovation

  • Elif Oral Professor - Department of Internal Medicine, Division of Metabolism Endocrinology and Diabetes (MEND), University of Michigan Health System

Synopsis

• How do physicians become aware of expended access programs?

• How can physicians balance the unmet need with the need to move the field forward?

• Recognition of important regulatory hurdles and ways to overcome them

4:00 pm Afternoon Break & Networking

4:30 pm Panel Discussion: Maximizing the Benefits of an Access Program for All Stakeholders

Synopsis

  • What are the challenges for physicians, patients, and pharma and how can we best address these challenges
  • Do physicians support data collection without compensation
  • How to design a program that meets the needs of all stakeholders

Deep Diving into Effective Methods to Maximize Vendor Selection & Qualification

5:00 pm Ethical Decision-Making for Early Access to Investigational Medicines in Rare Disease

Synopsis

  • Defining guiding principles
  • Determining the key stakeholders
  • Considerations for a decision-making framework

5:30 pm Roundtable Discussion: Bridging the Gap Between Industry & Vendors

Synopsis

• Working together to build strong collaborative relationships between vendors and pharma

• What are key regulatory considerations that vendors must address to be selected?

• How does a vendor’s commitment to a patient-centric approach influence its chances of being selected?

6:00 pm Chair’s Closing Remarks

6:10 pm Drinks Reception

Pre-Conference Day
Day Two