2020 PCORI Virtual Annual Meeting Breakout Session

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Conducting stakeholder-driven, investigator-initiated research in rare diseases is challenging. Information about treatments and natural disease progression is often lacking. Identifying rare disease study cohorts and designing clinical trials are difficult, given the small number of patients affected. Because patients’ families often receive variable care, they feel disempowered, resulting in additional evidence gaps about what outcomes matter most. This session showcases collaboration with rare disease communities, data sharing across institutions, and efforts to bridge the evidence gap with respect to knowledge sharing, clinical care, treatment, and comparative effectiveness research.

Introduction

Denese Neu, PhD
Engagement Officer
Patient-Centered Outcomes Research Institute

Nora McGhee, PhD
Senior Program Officer, Science
Patient-Centered Outcomes Research Institute

Moderator

Libby Hoy
Founder and Chief Executive Officer
PFCCpartners

Speaker Panelists

Catherine Anderson
Founder and Administrator
Living with Idiopathic Subglottic Stenosis Support Group
Comparing Results of Three Treatments for Idiopathic Subglottic Stenosis

Richard Aplenc, MD, PhD, MSCE
Professor of Pediatrics and Epidemiology 
Assistant Vice President & Chief Clinical Research Officer
Children’s Hospital of Philadelphia
Perelman School of Medicine 
University of Pennsylvania
Home or Away from Home: Comparing Clinician and Patient/Family-Centered Outcomes Relevant to the Care of Pediatric Acute Myeloid Leukemia during Periods of Neutropenia

Jennifer Canvasser, MSW
Founder and Director
Necrotizing Enterocolitis (NEC) Society
Building Capacity for PCOR/CER in the Neonatal NEC Community

Farah Contractor
Clinical Research Coordinator
Children’s Hospital of Philadelphia
Home or Away from Home: Comparing Clinician and Patient/Family-Centered Outcomes Relevant to the Care of Pediatric Acute Myeloid Leukemia during Periods of Neutropenia

Alexander Gelbard, MD
Co-Director 
Vanderbilt Center for Complex Airway Reconstruction
Associate Professor 
Vanderbilt University Medical School
Comparing Results of Three Treatments for Idiopathic Subglottic Stenosis

Seema Lalani, MD
Clinical Geneticist
Baylor College of Medicine
Engaging Patients in Outcomes Research in TANGO2-related disorder

Kasha Morris, MA
Cofounder
TANGO2 Research Foundation
Engaging Patients in Outcomes Research in TANGO2-related disorder

Ravi Patel, MD, MSc
Neonatologist and Associate Professor
Emory University
Building Capacity for PCOR/CER in the Neonatal NEC Community

Rohini Mohanraj, MHA
Program Associate
Patient-Centered Outcomes Research Institute

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