Webinars

More Information Coming Soon.

Webinar Session #1: Introductory NERDS Session

• Time: Nov 2, 1-3 PM EST.
• Confirmed Speakers:
  • Sandeep Menon (Pfizer)
  • L. J. Wei (Harvard)
    How to Select Endpoints and Quantify Treatment Effect in a Comparative Clinical Study on COVID-19 Diseases (slides)
  • Ying Yuan (MD Anderson)
    Borrowing Information from Historical Data: A Double-edged Sword (slides)
• Recording: https://pfizerevents.webex.com/pfizerevents/lsr.php?RCID=13985dd6a939e0e4f8998620b4a428b1 (Password: hWFURkY5)

Webinar Session #2: CID Program Update

• Time: Nov 13, 1-3 PM EST.
• Confirmed Speakers:
  • Steve Lake (Wave Life Sciences)
    A Bayesian Adaptive Trial in Duchenne Muscular Dystrophy (slides)
    
  • Dionne Price (FDA)
    Innovative Designs in the Rare Disease Setting (slides)
• Recording: https://pfizerevents.webex.com/pfizerevents/lsr.php?RCID=1bc6e1d02b1002d62c170a17473e3d54 (Password: pMbpsJT7)

Webinar Session #3: Gene Therapy in Rare Disease Development

• Time: Nov 20, 1-3 PM EST.
• Confirmed Speakers:
  • Michelle Casey (Pfizer)
    Gene Therapy: Opportunities and Challenges for the Treatment of Hemophilia (slides)
  • Marie-Laure Névoret / John Zhong (REGENXBIO)
• Recording: https://pfizerevents.webex.com/pfizerevents/lsr.php?RCID=ccaaeefdd89e63e72892e9650397f117 (Password: NERDS456)

Webinar Session #4: Case Studies in Rare Disease Drug Development

• Time: Dec 11, 1:30-3:30 PM EST.
• Confirmed Speakers:
  • Margaret (Meg) Gamalo (Pfizer)
    Lessons from Notable New Drug Application involving External Controls (slides)
  • Glen Laird (Vertex)
    Design and Analysis of Drop the Losers Studies in the Rare Disease Setting (slides)
  • Cindy Lu (Biogen)
    A Case Study Using Nature History Data to Understand Long Term Disease Progression (slides)
• Recording: https://pfizerevents.webex.com/pfizerevents/lsr.php?RCID=e227bbfd8699459593dffc5ce07cd795 (Password: Pq8pBWgU)