Working With Patient Advocacy Organizations to Inform Development

19 Jan 2023
Patient and Clinical Considerations Track

11:00am Chair Introduction

Sharon Hesterlee, Chief Research Officer, Muscular Dystrophy Association USA


11:05am Presentation:

Leveraging Human Factors From Medical Devices to Advanced Therapies

  • History of human factors in medical device, lessons learned and how to get it right the first time for advanced therapy manufacturing
  • Importance of human factors in cell and gene therapy manufacturing: use errors encountered in prior cell therapy manufacturing system
  • Incorporating human factors into the patient, clinician, and caregiver workflows associated with advanced therapies

Tessa Rowe, User Experience and Human Factors Engineering, Tensentric



Incorporating Patient Perspectives at FDA, a Continuing Evolution (Recorded) 

  • Overview of avenues in which FDA gathers patient input
  • Intersection of stakeholder goals for gathering patient perspectives
  • Effective partnerships

Anne Rowzee, Associate Director for Policy, OTAT, FDA (Remote)


11:35am Presentation: Supporting Blood Cancer Patients: LLS’s Holistic 360◦ Approach (Recorded) 

  • The Leukemia & Lymphoma Society (LLS) supports patients and their families through every part of their blood cancer experience with advocacy, research, education and financial support and can be an important connector in helping biopharma understand and target the unique needs of blood cancer patients.
  • Learn ways that LLS and biopharma can work together to advance the next generation of immunotherapy and ensure more patients can access it.

Gwen Nichols, Chief Medical Officer, Leukemia and Lymphoma Society (REMOTE) 


11:50am Presentation:

Supporting Patients & Families On Their Journey to Activate the Cure

  • An update on Emily Whitehead, now ten years cancer free after becoming the first pediatric patient to receive CAR T-cell therapy
  • How the Emily Whitehead Foundation helps connect patients and families to clinical trials and treatment programs
  • How the Emily Whitehead Foundation will grow going into the next decade of CAR-T, and how you can help

Tom Whitehead, Co-Founder, Emily Whitehead Foundation



12:05pm Closing Panel with Q&A

With all session participants

Joined by Wilson Bryan, Director, OTAT, FDA


Anne Rowzee
Associate Director for Policy, Office of Tissues and Advanced Therapies
Gwen Nichols
Chief Medical Officer
The Leukemia & Lymphoma Society
Sharon Hesterlee
Executive Vice President, Chief Research Officer
Muscular Dystrophy Association USA
Tessa Rowe
User Experience and Human Factors Engineering
Wilson Bryan
Director, Office of Tissues and Advanced Therapies