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The Need to Accelerate Therapeutic Development — Must Randomized Controlled Trials Give Way?

Available via

LIVESTREAM

The Need to Accelerate Therapeutic Development — Must Randomized Controlled Trials Give Way?

Wednesday, June 21, 2017 - Thursday, June 22, 2017

The New York Academy of Sciences, 7 World Trade Center, 250 Greenwich St Fl 40, New York, USA

Presented By

NYU School of Medicine

The New York Academy of Sciences

 

Randomized controlled trials (RCT) are frequently used as part of the clinical trial process, and are generally considered the gold standard for determining the efficacy and safety of an investigational product. As such, they have become a crucial part of the regulatory pathway whereby a new treatment can gain access to the market. While randomized controlled trials are widely considered valuable for their ability to present unbiased and consistent results, ethical concerns have been raised in recent years. For example, questions have surfaced regarding the ethics of placebo usage, especially if there are viable standards of care available; the length of time to complete development programs using traditional RCT paradigms, specifically when the patient population is small or the patient need for a treatment is immediate (e.g. the Ebola outbreak); or the potential of providing a treatment to the experimental group that may prove to be inferior than the standard treatment. In many situations, a case could be made for the need for alternatives to RCTs to speed development and bring treatments to patients in need more rapidly without compromising establishment of appropriate levels of efficacy and safety for registration.

To address these important issues, the NYU School of Medicine and the New York Academy of Sciences will present the two-day colloquium, The Need to Accelerate Therapeutic Development — Must Randomized Controlled Trials Give Way?, which will convene thought leaders who — in a public-facing dialogue — will discuss the ethical, legal, medical, scientific, safety, and economic issues pertaining to the design of clinical trials. Session topics will include the history and future of randomized controlled trials, the role of alternative trials, the ethical and scientific issues associated the randomized controlled trials and alternatives, a case study of a modified randomized controlled trial, and risk and liability associated with accelerated trials. Pitting randomized controlled trials against alternatives will inevitably result in a highly nuanced, ethically ambiguous discussion, and it could have profound effects on public health and the regulation of pharmaceuticals.

Livestream

All panel sessions and keynote lectures will be livestreamed during the two days of the colloquium. Registration is not required to view the livestream, watch it here.

Registration Pricing

This event is free but registration is required.

Registration

Member
$0
Nonmember
$0
Nonmember Academia, Faculty, etc.
$0
Nonmember Corporate, Other
$0
Nonmember Not for Profit
$0
Nonmember Student, Undergrad, Grad, Fellow
$0
Member Student, Post-Doc, Fellow
$0

Sponsors

This symposium was made possible with support from


Presented by

NYU Langone Medical Center

Promotional Partners

Contemporary Clinical Trials

LUNGevity Foundation

Wednesday

June 21, 2017

8:00 AM

Registration and Breakfast

8:40 AM

Welcoming Remarks

Speakers

Melanie Brickman Borchard, PhD, MSc
The New York Academy of Sciences
Arthur Caplan, PhD
NYU School of Medicine
Joanne Waldstreicher, MD
Johnson & Johnson
9:00 AM

Setting the Stage

Speaker

Arthur Caplan, PhD
NYU School of Medicine

Session I: The History and Contribution of Randomized Controlled Trials to Public Health

9:30 AM

Panel Discussion

Speakers

Arthur Caplan, PhD (Moderator)
NYU School of Medicine
Susan S. Ellenberg, PhD
Perelman School of Medicine, University of Pennsylvania
Howard Fingert, MD, FACP
Takeda Pharmaceuticals
Susan E. Lederer, PhD
University of Wisconsin School of Medicine and Public Health
Jane Perlmutter, PhD
Gemini Group
Note: Included in all panels will be approximately 10–15 minutes for Q&A with attendees of the Colloquium.
10:45 AM

Networking Coffee Break

Session II: Beyond RCTs — Assessing the Need for Alternatives

11:15 AM

Panel Discussion

Speakers

Charles Weijer, MD, PhD (Moderator)
Western University
Luciana Lopes Borio, MD
U.S. Food and Drug Administration
Barry J. Gertz, MD, PhD
Clarus Ventures
Andrea Troxel, ScD
NYU School of Medicine
Note: Included in all panels will be approximately 10–15 minutes for Q&A with attendees of the Colloquium.
12:30 PM

Networking Luncheon

1:45 PM

Keynote Lecture: Finding the Right Balance in Learning about Therapies

Speaker

Robert Califf, MD
Duke University

Session III: Weighing the Risks of Randomized Controlled Trials and Alternatives

2:25 PM

Panel Discussion

Speakers

Steve Usdin (Moderator)
BioCentury
Holly Fernandez Lynch, JD, BE
The Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School
Amrit Ray, MD, MBA
Johnson & Johnson
Matthew Rotelli, PhD
Eli Lilly and Company
Robert Walker, MD
U.S. Department of Health and Human Services
Note: Included in all panels will be approximately 10–15 minutes for Q&A with attendees of the Colloquium.
3:40 PM

Networking Coffee Break

Session IV: Ethics and Patient Advocacy in Clinical Trial Design

4:10 PM

Panel Discussion

Speakers

Alison Bateman-House, PhD, MPH, MA (Moderator)
NYU School of Medicine
Rebecca Susan Dresser, JD
Washington University in St. Louis
Andrew McFayden
The Isaac Foundation
Jane Reese-Coulbourne, MS, ChE
MK&A
J. Russell Teagarden
NYU School of Medicine Working Group on Compassionate Use and Pre-Approval Access
Note: Included in all panels will be approximately 10–15 minutes for Q&A with attendees of the Colloquium.
5:25 PM

Closing Remarks

5:30 PM

Networking Reception

7:00 PM

Day 1 Adjourns

Thursday

June 22, 2017

8:00 AM

Registration and Breakfast

8:45 AM

Keynote Lecture: Modern Trends in Clinical Drug Development

Speaker

Janet Woodcock, MD
U.S. Food and Drug Administration

Session V: Lessons from the Eteplirsen Drug Trial for Duchenne Muscular Dystrophy

9:30 AM

Panel Discussion

Speakers

Meg Tirrell (Moderator)
CNBC
Pat Furlong
Parent Project Muscular Dystrophy
Edward M. Kaye, MD
Sarepta Therapeutics
David Scheer
Scheer & Company, Inc.
Ellis Unger, MD
U.S. Food and Drug Administration
Note: Included in all panels will be approximately 10–15 minutes for Q&A with attendees of the Colloquium.
10:45 AM

Networking Coffee Break

Session VI: A Way Forward: Shaping Clinical Trial Innovation

11:15 AM

Panel Discussion

Speakers

Donald Berry, PhD (Moderator)
MD Anderson Cancer Center
Anne Cropp, PharmD
Early Access Care, LLC
George Demetri, MD
Dana-Farber Cancer Institute
Clifton Leaf
Fortune
Christopher Robertson, PhD, JD
University of Arizona
John (L.P.) Thompson, PhD
Columbia University
Note: Included in all panels will be approximately 10–15 minutes for Q&A with attendees of the Colloquium.
12:30 PM

Networking Luncheon

Session VII: Ethics Panel Wrap-up: What is the Future of Accelerated Development and the Randomized Controlled Trial Standards?

1:45 PM

Panel Discussion

Speakers

Timothy Caulfield, LLM, FRSC, FCAHS (Moderator)
University of Alberta
Nancy M.P. King, JD
Wake Forest School of Medicine
Vinay Prasad, MD, MPH
Oregon Health and Science University
Eric H. Rubin, MD
Merck & Co., Inc.
Jeffrey S. Weber, MD, PhD
NYU Langone Medical Center
Note: Included in all panels will be approximately 10–15 minutes for Q&A with attendees of the Colloquium.
3:00 PM

Closing Remarks

3:20 PM

Colloquium Concludes

To view full attendee list, you must first register for the event, then log in to the Academy website. This list is provided for the personal, noncommercial and informational use only of event attendees, in a manner that is consistent with the New York Academy of Sciences’ mission, goals and activities.
  • AARP

  • Abeona Therapeutics

  • Abigail Alliance for Better Access to Developmental Drugs

  • Achillion

  • Adelphi University

  • Alexion

  • Alexion Pharmaceutical

  • Amicus Therapeutics

  • Applied Biological Laboratories

  • AVAC

  • BioAegis Therapeutics Inc.

  • BioCentury Publications Inc.

  • Bioethics International

  • BioMarin Pharmaceutical, Inc.

  • Biopharmininsight

  • Boehringer Ingelheim Pharmaceuticals

  • Boehringer Ingelheim Pharmaceuticals, Inc.

  • Breast Cancer Action

  • Bristol-Myers Squibb

  • Bronx-Lebanon Hospital Center

  • Business Insider

  • Canadian Organization for Rare Disorders

  • Celgene

  • Celgene/Rutgers University

  • Center for Drug Evaluation and Research, U.S. Food and Drug Administration

  • Chugai

  • CitizenRacecar, LLC

  • Clarus Ventures

  • Clinilabs, Inc.

  • CNBC

  • Coalition Against Childhood Cancer (CAC2)

  • Cohen Veterans Bioscience

  • Cold Spring Harbor Laboratory

  • Collins Consulting

  • Columbia University

  • Columbia University Medical Center

  • Columbia University School of Public Health

  • Consultant

  • Covance

  • Crossover Search

  • CurePSP

  • Damon Runyon Cancer Research Foundation

  • Dana Farber Cancer Institute DFCI

  • Deerfield Institute; Contemporary Clinical Trials; Contemporary Clinical Trials Communications

  • Deerfield Management

  • Depression and Bipolar Support Alliance

  • Doctors for America

  • Duke University

  • Early Access Care, LLC

  • Edelman

  • Eli Lilly and Company

  • Esai Inc

  • EURORDIS

  • FCBVIO/Mount Sinai

  • Forbes

  • Forest Research Institute

  • Fortune

  • Freelance

  • G Stone Connections, L.L.C.

  • GE2P2 Global - CVEP

  • Gemini Group

  • GlobalData

  • Green Corsair Tech Labs Global TM

  • GSAS, Columbia University

  • Harvard Medical School

  • Health Research Alliance

  • HOSPITAL DE ESPECIALIDADES EUGENIO ESPEJO

  • Hunter College

  • IAVI

  • IBM

  • Icahn School of Medicine at Mount Sinai

  • Icahn School of Medicine at Mt. Sinai

  • Image Reading Center, Inc.

  • Institute for Clinical and Economic Review (ICER)

  • Internation AIDS Vaccine Initiative

  • Intra-Cellular Therapies

  • Intra-Cellular Therapies, Inc.

  • Iron Disorders Institute

  • J&J

  • Janssen

  • Janssen Research & Development

  • Janssen Research and Development

  • JDRF

  • Johnson & Johnson

  • Johnson and Johnson

  • Johnson&Johnson

  • Ketchum

  • Kidney Cancer Research Alliance (KCCure)

  • Lehigh University

  • Lenox Hill Hospital - Northwell Health

  • LSU Health Sciences Center in New Orleans, 1542 Tulane Ave.,

  • LUNGevity Foundation

  • MARKETWATCH

  • MD Anderson Cancer Center

  • Memorial Sloan Kettering Cancer Center

  • Merck

  • Merck & Co.

  • Merck & Co., Inc

  • Merck & Co., Inc.

  • Merck and Co Inc

  • Merck Sharp & Dohme Corp.

  • microbiology

  • MK&A

  • Molekule Consulting

  • Montefiore Medical Center

  • Mount Sinai School of Medicine

  • MPM Capital

  • MSKCC

  • Myeloma Patient referred by SmartPatients

  • National Academies of Sciences, Engineering, and Medicine

  • Neurotez, Inc.

  • New York Institute of Technology College of Osteopathic Medicine

  • New York University

  • New York University College of Dentistry

  • NINDS

  • NINDS/NIH

  • Nonviolent Medicine

  • North Highland

  • Northwell Health at Staten Island University Hospital

  • NYC DEPARTMENT OF HEALTH AND MENTAL HYGIENE

  • NYU

  • NYU Division of Medical Ethics

  • NYU Langone Medical Center

  • NYU Medical Center

  • NYU School of Medicine

  • NYU School of Medicine & The Hastings Center

  • NYU School of Medicine Working Group on Compassionate Use and Pre-Approval Access

  • NYU The HUMAN Project

  • NYU@Woodhull

  • One-World Inc.

  • Oregon Health and Science University

  • Our Her2Cancer Struggle

  • Ozeion Biologics

  • Parent Project Muscular Dystrophy

  • Patient advocate

  • Pfizer Inc.

  • Pleasant Hill Consultants, Inc

  • PNAS

  • Post University

  • Princeton University

  • PWR

  • Renal Research Institute

  • Roche

  • Rockefeller Univeristy

  • Rudder Serendip LLC

  • Saint Ann's School

  • Samani BioStrategist.com

  • Sanofi

  • Sanofi Pasteur

  • Sarepta Therapeutics

  • Sawmill Editorial Services

  • Scheer & Company, Inc.

  • Scioderm, An Amicus Therapeutics Company

  • Silverback Therapeutics, Inc.

  • Slate Magazine

  • St John's University

  • Stand Up To Cancer

  • Takeda Pharmaceuticals

  • Terrapinn Inc

  • Texas A&M University

  • The Albert Einstein College of Medicine

  • The Hastings Center

  • The Icahn School of Medicine at Mount Sinai

  • The Isaac Foundation

  • The Michael J Fox Foundation for Parkinson's Research

  • The New York Academy of Sciences

  • The New York Academy of Sciences NYAS

  • The OCI Group/SJU

  • The Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School

  • The Telmarc Group

  • The University of Pennsylvania

  • Treatment Action Group

  • Tufts Medical Center

  • U.S. Food and Drug Administration

  • University of Alberta

  • University of Arizona

  • University of Pennsylvania

  • University of Wisconsin School of Medicine & Public Health

  • Vels university

  • Wake Forest School of Medicine

  • Washington University in St. Louis

  • Weill Cornell Medical College

  • Weill Cornell Medicine

  • Western University of Health Sciences

  • Yale University, Collaboration on Research Integrity and Transparency