Informing patient and caregiver decisions towards gene therapy

Written on Monday 4th October 2021

Gene therapy offers hope for many people living with a rare disease. These treatments have the potential to transform lives in disease areas where there may be no treatment options, where there is high unmet need, or where there is high treatment burden. The science is fascinating, the innovation remarkable, the logistics mind bending and the reimbursement pathway uncharted. Through all this excitement, challenge, and planning, let’s not forget one fundamental factor that will be a constant as more and more therapies from the frontier of medicine are introduced. We shouldn’t underestimate the magnitude of the decisions facing patients and caregivers as they consider gene therapy.

This webinar explores patient and caregiver experiences and the keys to ensuring truly informed decisions towards gene therapy, with a focus on the psychology of decision-making, the role of healthcare professionals, and the ways in which different groups can work together to aid patients and caregivers in making informed decisions.


  • Ms. Debra Miller – Founder and CEO, Cure Duchenne
  • Mr. Luke Pembroke – Creative Director, Haemnet and Patient Advocate
  • Dr. Stephen A. Kagan – Global Medical Lead, Gene Therapy Platform and Cross Portfolio, Pfizer Rare Disease
  • Dr. Albert Freedman – Founder and Consulting Psychologist, Freedman Counselling Associates
  • Dr. Declan O’Rourke – Consultant Pediatric Neurologist, Associate Clinical Professor UCD, and Associate Director of Research CHI


  • Gavin Jones – Global Advisor, Rare Disease, OPEN Health