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DIA 2021 Global Annual Meeting


Patient-Focused Drug Development in Rare Diseases: Applying Guidances and Case Studies from the Field

Session Chair(s)

K. Kimberly  McCleary

K. Kimberly McCleary

  • Founder and Chief Executive Officer
  • The Kith Collective, LLC, United States
FDA guidances from the three product centers (CDER, CBER and CDRH) provide direction for how to involve patients and caregivers in medical product development and to integrate their perspectives and preferences into across the lifecycle. A team of subject matter experts from life science companies and patient advocacy organizations convened by the EveryLife Foundation and partners has workshopped the guidances to apply them to stages of product development of innovative therapies for rare diseases. This session features learnings from this activity, as well as case studies from the field.
Learning Objective : Describe FDA guidances that can be applied to patient-focused medical product development; Identify how these guidances can be applied across the development lifecycle for an innovative drug, gene/cell therapy, or medical device intended to treat a rare disease; Discuss using case studies of patient involvement in therapy development for rare diseases.

Speaker(s)

Annie  Kennedy

Project Leader

Annie Kennedy

  • Chief of Policy and Advocacy
  • EveryLife Foundation for Rare Diseases, United States
Pujita  Vaidya, MPH

Steering Committee Member

Pujita Vaidya, MPH

  • Director, Global Regulatory and R&D Policy, Patient Centricity, Pediatrics, ROD
  • Amgen, United States
Kristin  Van Goor, PhD

Panelist

Kristin Van Goor, PhD

  • Senior Director, North America Regulatory Policy and Intelligence
  • Vertex Pharmaceuticals, Inc., United States
Jill  Jarecki, PhD

Patient-Focused Drug Development in Rare Diseases: Applying Guidances and Case Studies from the Field

Jill Jarecki, PhD

  • Chief Scientific Officer
  • Cure SMA, United States