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Wednesday, June 28 • 8:30am - 9:30am
#306: Writing Summary Documents for Rare Disease Submissions: Unique Challenges

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Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-23-610-L04-P; CME 1.00; RN 1.00

Rare diseases are heterogenous and most involve very few patients. Data to support these NDAs comes from multiple sources posing unique challenges in preparing clinical summaries. This session aims to explore and brainstorm potential solutions.

Learning Objectives

Recognize challenges in preparing clinical summary documents for New Drug Applications (NDAs); Analyze the differences and gaps relative to NDAs based on adequately powered controlled studies; Initiate plan of action including contingency planning; Identify stakeholders (internal and external) to engage.

Chair

Anjana Bose, PhD

Speaker

Industry Update
Rosa M Real, MD

Industry Update
Sara Kowalczyk, MA, MPH

Industry Update
Raymond A Huml, DVM, MS, RAC


Speakers
avatar for Anjana Bose

Anjana Bose

Director, Global Submissions, Certara, United States
With a career spanning over 30 years in the pharmaceutical industry, Dr. Bose has extensive experience across clinical development, data analytics, regulatory writing, and leadership role in regulatory submissions. During last 15 years, she has led New Drug Applications and Biologic... Read More →
avatar for Raymond Huml

Raymond Huml

Vice President, Medical and Scientific Management, Head, Rare Disease Consortium, Syneos Health, United States
Raymond A. Huml, MS, DVM, RAC is Vice President of Medical and Scientific Strategy and Head of Syneos Health’s Rare Disease Consortium. Dr. Huml has over 35 years of experience in the clinical and biopharmaceutical industries and almost 30 years in the Contract Research Organization... Read More →
avatar for Sara Kowalczyk

Sara Kowalczyk

Associate Director, Medical Writing, Ultragenyx Pharmaceutical Inc., United States
Sara Kowalczyk, MA, MPH is an Associate Director of Medical Writing at Ultragenyx Pharmaceuticals focusing on clinical regulatory document writing to thoughtfully bring new treatments to market for rare diseases. She has previously held medical writing roles at Alnylam Pharmaceuticals... Read More →
avatar for Rosa Real

Rosa Real

Senior Director, Clinical Development, Homology Medicines, Inc, United States
Rosa M Real MD is a trained pediatrician with over 17 years of experience across multiple disciplines of clinical development, medical affairs, medical communications, and clinical operations in the pharmaceutical industry. She has worked across multiple therapeutic areas in her previous... Read More →

Wednesday June 28, 2023 8:30am - 9:30am EDT
Room 253 C Boston Convention and Exhibition Center 415 Summer Street, Boston, MA 02210 USA
  04: MedAffairs-SciComm, Session |   09: Regulatory, Session