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Thursday, July 1 • 10:00am - 11:00am
#410 SL: Patient-Focused Drug Development in Rare Diseases: Applying Guidances and Case Studies from the Field

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Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-21-639-L04-P; CME 1.00; IACET 1.00; RN 1.00

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 Objectives

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.

Chair

K. Kimberly McCleary

Speaker

Project Leader
Annie Kennedy

Steering Committee Member
Pujita Vaidya, MPH

Panelist
Kristin Van Goor, PhD

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



Speakers
avatar for K. Kimberly McCleary

K. Kimberly McCleary

Founder and Chief Executive Officer, The Kith Collective, LLC, United States
Kim McCleary has been at the forefront of patient engagement for nearly 30 years. She is a nationally regarded subject matter expert on patient-focused medical product development, patient-centered benefit-risk assessment, and organizational change. Kim has authored scores of publications... Read More →
avatar for Annie Kennedy

Annie Kennedy

Chief of Policy and Advocacy, EveryLife Foundation for Rare Diseases, United States
Annie has served within the community for nearly three decades through her roles with Parent Project Muscular Dystrophy (PPMD) and the Muscular Dystrophy Association (MDA). In that time she helped lead legislative efforts around passage and implementation of the MD-CARE Act (2001... Read More →
avatar for Kristin Van Goor

Kristin Van Goor

Regulatory Policy and Intelligence, Global Regulatory Affairs, Vertex Pharmaceuticals, Inc., United States
Kristin Van Goor received her Ph.D. in Genetics from Yale University and continued her research as a post-doctoral fellow in the Cancer Genetics Branch of the National Human Genome Research Institute at the NIH. She then spent a number of years at PhRMA as the Assistant Vice President... Read More →
avatar for Jill Jarecki

Jill Jarecki

Chief Scientific Officer, Cure SMA, United States
Jill Jarecki, PhD, is currently the Chief Scientific Officer at Cure SMA and has been with the organization since 2005. In this capacity, she has managed a broad portfolio of SMA research investments totally of almost $60 million in basic research, preclinical drug development, and... Read More →
avatar for Pujita Vaidya

Pujita Vaidya

Director, Global Regulatory and R&D Policy, Patient Centricity, Pediatrics, ROD, Amgen, United States
Pujita Vaidya serves as a Global Regulatory and R&D Policy Director at Amgen, working to develop, advocate for, and implement Amgen positions on regulatory policies, specifically Patient Centricity, Pediatrics, and Rare Diseases. Her work includes partnering with cross-functional... Read More →
avatar for Pujita Vaidya

Pujita Vaidya

Global Regulatory and R&D Policy Director, Amgen, United States
Pujita Vaidya serves as a Global Regulatory and R&D Policy Director at Amgen, working to develop, advocate for, and implement Amgen positions on regulatory policies, specifically Patient Centricity, Pediatrics, and Rare Diseases. Her work includes partnering with cross-functional... Read More →


Thursday July 1, 2021 10:00am - 11:00am EDT
TBD Virtual Event Horsham, PA 19044
  05: Patient Engagement, Session