Back To Schedule
Tuesday, June 25 • 8:00am - 9:15am
#213: Facilitating Access: Patient Perspectives on a Streamlined Development Approach for Treatments for Severely-Debilitating or Life-Threatening Diseases

Sign up or log in to save this to your schedule, view media, leave feedback and see who's attending!

Component Type: Forum
Level: Basic
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-581-L04-P; CME 1.25; IACET 1.25; RN 1.25

This forum will introduce a streamlined approach to the development of therapies to address severely-debilitating or life threatening diseases and consider the potential value of global regulatory guidance.

Learning Objectives

Discuss the use of streamlined development programs to expeditiously deliver promising new treatments to patients with severely debilitating or life-threatening diseases from the patient perspective.


Judith Prescott, DVM, PhD


Proposal for Development of Pharmaceuticals for Severly-Debilitating or Life Threatening Indications
Judith Prescott, DVM, PhD

Severely Debilitating or Life-Threatening Hematologic Disorders: Nonclinical Considerations
Haleh Saber, PhD

Perspective from The Patients' Academy For Research Advocacy
Susan Schaeffer

Catherine Kopil, PhD

Isabelle Lousada, MA

avatar for Catherine Kopil

Catherine Kopil

Director, Research Partnerships, The Michael J. Fox Foundation for Parkinson's Research
As Director of Research Partnerships at The Michael J. Fox Foundation (MJFF), Katie focuses on building the Foundation’s capacity as an unprecedented stakeholder in Parkinson’s drug development — a nimble, patient-focused problem-solver whose efforts are demonstrably accelerating... Read More →
avatar for Isabelle Lousada

Isabelle Lousada

Chief Executive Officer and President, Amyloidosis Research Consortium, United States
As a former patient, Lousada has spent 20 years dedicated to patient advocacy, accelerating drug development, and promoting awareness of rare diseases. Lousada founded the Amyloidosis Research Consortium (ARC) in 2015 to address the critical needs in clinical trials and related research... Read More →
avatar for Judith Prescott

Judith Prescott

Executive Director, Safety Assessment, Merck & Co., Inc.
Judith Prescott, DVM, PhD, DACVP is an Executive Director in Safety Assessment, Merck & Co, Inc. She has responsibility for all nonclinical drug development programs supporting FIH through post marketing across the Merck portfolio including small molecules, biologics, and vaccines... Read More →

Haleh Saber

Deputy Director, DHOT/ OHOP/ CDER, FDA
Dr. Saber is the Deputy Director in the Division of Hematology Oncology Toxicology (DHOT). In this role, she provides leadership for day-to-day activities, leads and coordinates scientific research, and participates in guidance development. Dr. Saber has extensive industry and regulatory... Read More →

Susan Schaeffer

President and Chief Executive Officer, The Patients' Academy For Research Advocacy
Susan founded The Patients’ Academy for Research Advocacy in 2018 to equip patients and care partners to apply their unique expertise to inform the development of new and better medicines. She spent 15 years at biopharmaceutical industry journal BioCentury informing and educating... Read More →

Tuesday June 25, 2019 8:00am - 9:15am PDT
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  09: Regulatory, Forum
  • Level Intermediate
  • Featured Topics Patient Focused,Advanced Therapies,Rare Disease
  • Credit Type ACPE, CME, IACET, RN
  • Tags Forum