Conference Day Two

9:25 am Chair’s Opening Remarks

Determining the Role of Real-World Data in EAPs

9:30 am Leveraging AI Tools & a Digital Patient Community to Advance Expanded Access Programs

  • Jennifer Joe Director Global Medical Strategy & Population Health, AstraZeneca


• Using a digital patient community to incorporate a centralized way to identify way patients with the highest need

• Mitigating the legal and data privacy complications

• Setting up an expanded access through value-based organisations to obtain a large community of patients and carry this out with ease as it’s completed through one system

10:00 am Roundtable Discussion: Is Collecting Data Vital for Running a Successful EAP?


• Collecting data to help pricing and reimbursement decisions whilst remaining cautious to not impact regulatory strategy

• What are the necessary ethical considerations and protocols needed to be in place to collect data?

• What are the implications of this for an unlicensed product?

10:30 am Morning Break & Networking

Charting the Path Forward for Clinical Supply Excellence

11:00 am Highlighting Practical Strategies to Successfully Deliver a Project in the Middle East


• Sharing previous experiences on how to navigate the regulations in the Middle East and Africa

• What are the important factors to consider ensuring it runs smoothly?

• Highlighting the lessons learnt from when it hasn’t gone according to plan

11:30 am Panel Discussion: Ensuring Labelling Consistency Across the Supply Chain & Its Accordance with GNP

  • Vilem Guryca Associate Director, Expanded Access, BeiGene
  • Katie Bowen Expanded Access Coordinator, GSK
  • Irfan Mohammed Director, Chemistry, Manufacturing & Controls Team Lead Product Development & Clinical Supply, Alexion AstraZeneca Rare Disease


• Streamlining the labelling process to ensure patients receive the drug in a timely manner

• Establishing clarity and consistency on what’s considered pre-approval access

• Reviewing multinational labelling requirements and seeking guidance for optimal interpretation

12:00 pm Lunch Break & Networking

1:00 pm Licence Withdrawal: Rapidly Pivoting to Ensure Continuity of Care


• Ensuring continued drug supply to several hundred patients

• Effective communication with HCPs and internal stakeholders

• Setting up the internal and external infrastructure to ensure streamlined approach to processing the volume of requests

Overcoming Operational Challenges & Mitigating Data Privacy & Legal Issues

1:30 pm Deep Diving into Processes & Systems for EAP


• Developing a platform for program management

• Partnering with digital teams and vendors

• Reporting metrics and turnarounds times

2:00 pm Afternoon Break & Networking

2:30 pm Navigating & Successfully Setting Up an Expanded Access Program for an Ultrarare Disease

  • David Stein Senior Director, Medical Study Operations, Regeneron Pharmaceuticals Inc
  • Maggie McDermott Senior Specialist, Medical Study Operations, Regeneron Pharmaceuticals Inc


• How do you handle an ultrarare disease?

• How do you get drugs into countries without well-defined regulations

• How do you proceed with continuing a drug within this disease post FDA approval in different countries?

3:00 pm Roundtable Discussion: Informed Consent & Assent: Raising Consciousness About the Importance of Consent


• What are the best practices globally about how you do consent?

• Ensuring patients are fully aware of the risks involved

• Optimizing the circulation of data between patients, physicians, and manufacturers

3:30 pm Chair’s Closing Remarks