Symposium Examines the Role of Randomized Controlled Trials

NEW YORK, June 7, 2017 — From Wednesday, June 21 to Thursday, June 22 the New York Academy of Sciences and NYU School of Medicine, with support from Johnson & Johnson, will present a colloquium titled, "The Need to Accelerate Therapeutic Development – Must Randomized Controlled Trials Give Way?" at the Academy's headquarters in downtown Manhattan.

Keynote lectures will be delivered by Janet Woodcock, MD, Director of the Center for Drug Evaluation and Research at the U.S. Food and Drug Administration, on the topic of "Modern Trends in Clinical Drug Development," and Robert Califf, MD, former Commissioner of the U.S. Food and Drug Administration, who will discuss "Finding the Right Balance in Learning About Therapies."

Randomized controlled trials (RCTs) are considered the gold standard in determining the efficacy and safety of a potential therapy before it is brought to market. However, in recent years critics have raised a number of ethical issues regarding some aspects of RCT design, including its lack of speed in providing treatments to patients. They argue that there is a need for alternatives to RCTs. As Dr. Woodcock noted, "Randomization is an important tool, but traditional clinical trials are changing as the demands of precision medicine, the need for rapid responses to disease outbreaks, and the emergence of platform trials, to name a few forces, are changing the way drug evaluation is done."

The colloquium will convene thought leaders from government, academia, and industry in a public dialogue to discuss the ethical, legal, medical, scientific, safety, and economic issues surrounding the design of clinical trials. The event will specifically focus on the role of RCTs in the development of new treatments, and the benefits and drawbacks that could be associated with alternative trial designs.

"We are entering a period in which digital technology will enable rapid and powerful analysis of data. As ‘real world data' advances, we need to understand the methods of design and analysis that will yield the most valid and useful advances. This conference will provide a check on the current status of this field," said Dr. Califf.

Ultimately, the comparison between RCTs and their alternatives is a highly nuanced and ethically ambiguous discussion with profound implications for public health and the regulation of pharmaceuticals.

As conference organizer Arthur Caplan, PhD from NYU School of Medicine explained, "The upcoming conference could not be timelier. Debates are now raging about the role of the FDA in governing clinical trials, what EU policies will look like post-Brexit, and what to do about patient demands for more input into clinical trial design and what is accepted as evidence. The speakers and topics go right at these issues in a way that will not only be engaging, but also should help national and international discussion around the future of the randomized clinical trial."

"I am grateful to the NYU School of Medicine and the NYAS for hosting this colloquium to address the history and future of randomized controlled trials, the role of alternative trial designs, and the ethical and scientific considerations. It demonstrates how all stakeholders can work together to enhance our collective ability to serve patients and humanity," shared Joanne Waldstreicher, MD, Chief Medical Officer, Johnson & Johnson. "Now more than ever, we need to find new solutions for getting medicines to patients faster without compromising the scientific rigor and data required to demonstrate efficacy and safety – and the best way for us to do this is by working together and sharing our best thinking."

About NYU School of Medicine

NYU School of Medicine is one of the nation's preeminent academic institutions dedicated to achieving world-class medical educational excellence. For 170 years, NYU School of Medicine has trained thousands of physicians and scientists who have helped to shape the course of medical history and enrich the lives of countless people. An integral part of NYU Langone Medical Center, the School of Medicine at its core is committed to improving the human condition through medical education, scientific research and direct patient care. The School also maintains academic affiliations with area hospitals, including Bellevue Hospital, one of the nation's finest municipal hospitals where its students, residents and faculty provide the clinical and emergency care to New York City's diverse population, which enhances the scope and quality of their medical education and training. Additional information about the NYU School of Medicine is available at http://school.med.nyu.edu/.

About The New York Academy of Sciences

The New York Academy of Sciences is an independent, not-for-profit organization that since 1817 has been driving innovative solutions to society's challenges by advancing scientific research, education, and policy. With more than 20,000 Members in 100 countries, the Academy is creating a global community of science for the benefit of humanity. Please visit us online at www.nyas.org and follow us on Twitter at @NYASciences.

About Johnson & Johnson

Caring for the world, one person at a time... inspires and unites the people of Johnson & Johnson. We embrace research and science — bringing innovative ideas, products and services to advance the health and well-being of people. Our approximately 130,800 employees at more than 250 Johnson & Johnson operating companies work with partners in health care to touch the lives of over a billion people every day, throughout the world.