8:50 am
Chair’s Opening Remarks

Optimizing Data Collection & Real World Data Generation

9:00 am Panel Discussion – Navigating Global Data Generation Regulations

  • Jeffrey Waldron Managing Director, Sherborn Consulting Group
  • Richard Klein Director, Expanded Access Programs & Policy, GE2P2 Global Foundation
  • Alaa Hamed Global Head of Medical Affairs Rare Diseases, Sanofi Genzyme
  • Ramona Reichenbach Head of Managed Access Center of Excellence, Novartis


• Incorporating data collection into expanded access programs
• Harnessing data for regulatory filings
• Managing global compliance rules when collecting data in an expanded access program

9:45 am Panel Discussion: Collecting Real World Data in Compliance with European Regulations

10:30 am Understanding the Limitations on Data Collection in Expanded Access Programs


• Addressing the challenges of global regulations for data collection
• Managing the patchy outcomes data from expanded access programs
• Building efficiencies in capturing data in an expanded access program

11:00 am
Networking Break

Discussing the Ethical Considerations When Running an Expanded Access Program

11:30 am Panel Discussion: Examining Patient Engagement Issues

  • Jeffrey Waldron Managing Director, Sherborn Consulting Group
  • Lisa Kearns Researcher, Division of Medical Ethics, NYU Grossman School of Medicine


  • Discussing ideas to increase patient awareness
  • Gaining a better idea of how to educate providers
  • Analysing global regulations around patient involvement

12:15 pm Navigating the Challenges of Establishing Expanded Access Programs for Rare Diseases

  • Alaa Hamed Global Head of Medical Affairs Rare Diseases, Sanofi Genzyme


• Outlining a holistic overview of the challenges of running an expanded access program with a focus on rare disease
• Establishing a global reach for your expanded access program
• Creating a cross-functional team to run a rare disease expanded access program

12:45 pm Ethical Considerations in Expanded Access for Gene Therapy Companies

  • Lisa Kearns Researcher, Division of Medical Ethics, NYU Grossman School of Medicine


• Recognizing the ethical imperative for gene therapy companies to proactively develop expanded access policies
• Understanding obstacles developers face in providing expanded access to gene therapy products
• Recognizing that conditions with limited windows for treatment deserve special consideration in expanded access

1:15 pm

2:15 pm Running a Gene Therapy Expanded Access Program


• Understanding the importance of providing an early access program for acute diseases
• Using an early access program to provide patients treatments between clinical trials and commercial approval
• Considering the additional requirements when running an expanded access program for gene therapies

2:45 pm Equity and Ethical Considerations of Expanded Access Programs

  • Jean Godin Global Head of Humanitarian & Managed Access Programs and Medical Governance, Sanofi Genzyme


• Defining what a serious condition is
• Analyzing the availability of the information from patient to the physician
• Discussing drug allocation (factors supporting country selection and number of patients in each country)
• Understanding patient selection
• Factoring in ethical considerations when ending a program

3:15 pm
Networking Break

Setting Up and Navigating the Logistical Hurdles of an Expanded Access Program

3:45 pm Navigating the Logistical Challenges of Expanded Access Programs in Small Biotech


• Moving from an approved clinical trial to expanded access in small biotech
• Highlighting the logistical challenges of coordinating single patient expanded access
• Managing multiple expanded access trials during a pandemic

4:15 pm Managing Supply Chain Logistics


• Revealing the different requirements for marketed access and investigational access
• Predicting labelling, packaging and shelf-life requirements for a treatment when opening an expanded access program in a new country
• Ensuring submissions are made to relevant health authorities in each country

4:45 pm
Chair’s Closing Remarks