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Wednesday, June 30 • 4:00pm - 5:00pm
#351 SL: The Rare Disease Clinical Outcome Assessment Consortium: Collaboration Aimed at Accelerating Rare Disease Drug Development

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Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-21-626-L04-P; CME 1.00; IACET 1.00; RN 1.00

A key aim of the Rare Disease Clinical Outcome Assessment (COA) Consortium is the creation of a resource of publicly available COAs suitable as trial endpoint measures. Methodological challenges in rare disease trial settings will also be addressed.

Learning Objectives

Describe the unmet need that the Rare Disease Clinical Outcome Assessment Consortium seeks to address; Describe the Rare Disease COA Resource, including the purpose, process for development, domains included, and progress to-date; Describe a literature review to identify approaches to evaluate clinical benefit in populations with heterogeneity; Describe COVID-19 mitigation strategies in pediatric rare disease clinical trials.


Lindsey Murray, PhD, MPH


Academic Update
Tori Brooks, MPH

Academic Update
Kiera Berggren, MA, MS

Industry Update
Timothy Andrew Howell, MA

FDA Update
Naomi Knoble, PhD

Patient Update
Allison Seebald

avatar for Lindsey Murray

Lindsey Murray

Associate Director, PRO Consortium, Critical Path Institute, United States
Lindsey Murray, PhD, is the Associate Director for Rare Diseases at the Critical Path Institute’s (C-Path) Patient-Reported Outcome (PRO) Consortium. Dr. Murray has more than 14 years of experience in clinical and health outcomes research, specializing in quantifying the patient’s... Read More →

Tori Brooks

Research Associate II, Mapi Research Trust, United States
Tori is a scientific research associate at Mapi Research Trust (MRT), involved in the management of literature review projects in the field of Clinical Outcome Assessments. Her work has spanned a variety of therapeutic areas including musculoskeletal diseases, oncology, and cardiovascular... Read More →

Kiera Berggren

Research Speech-Language Pathologist, Department of Neurology, Virginia Commonwealth University, United States
Kiera Berggren is a research speech-language pathologist in the department of Neurology at VCU. She currently has dual roles in research and in the clinical setting, specializing in working with patients and study participants of all ages with rare neuromuscular diseases. She has... Read More →

Timothy Howell

Research Associate III, Evidera, United States
Timothy Howell, MA, is a Research Associate III with Evidera’s Patient-Centered Research Group in Bethesda, Maryland. In his role at Evidera, Mr. Howell’s responsibilities include managing logistic and financial aspects of projects, conducting concept elicitation and cognitive... Read More →

Naomi Knoble

Reviewer, Division of Clinical Outcome Assessment, OND, CDER, FDA, United States
Naomi Knoble, PhD, is a reviewer in the Division of Clinical Outcome Assessment (DCOA), Office of New Drugs (OND), Center for Drug Evaluation Research (CDER), with the US Food and Drug Administration (FDA). Prior to FDA, Dr. Knoble was a research scientist and global healthcare consultant... Read More →

Allison Seebald

Senior Research Program Manager, National Organization for Rare Disorders (NORD), United States

Wednesday June 30, 2021 4:00pm - 5:00pm EDT
TBD Virtual Event Horsham, PA 19044
  03: Data-Data Standards, Session |   02: ClinTrials -ClinOps, Session