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

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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.

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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
Matthew Rotelli, PhD
Eli Lilly and Company
Amrit Ray, MD, MBA
Johnson & Johnson
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
Jane Reese-Coulbourne, MS, ChE
MK&A
J. Russell Teagarden
NYU School of Medicine Working Group on Compassionate Use and Pre-Approval Access
Andrew McFayden
The Isaac Foundation
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