Innovation in Rare Diseases in the Real World

Date: April 2, 2015, 4:00–5:00 PM
Location: 105 Annenberg
Speaker:James E. Lock, Department of Cardiology, Children’s Hospital Boston

Pediatric diseases are uncommon, and thus all who try to develop new treatments have to develop strategies that do not rely on the power of very large data bases to inform the innovation process. The cardiac program at Boston Childrens Hospital has successfully developed nearly 30 new procedures in the past several decades to treat cardiac diseases in children and young adults. This rich experience provides important insights into the innovation process.

First, I will describe of the development of a method to open obstructed aortic valves in the 26 week fetus, whose total heart is the size of a grape. This procedure has now been shown to prevent involution of the heart, allowing the development of a 4 chambered heart at birth. I will discuss the reasons why we picked this condition to start with, how we decided which patients to try it on, the trial and error process that produced our initial success, and the way we modified those methods, tools and patient selection to become by far the largest fatal treatment center in the world.

Next, I will generalize the process of performing a "first-in-man" procedure into 8 discrete steps, and draw examples for each of these steps from diverse procedures and conditions, illustrating the importance of a disciplined approach to successful innovation.

Finally, I will describe a new approach to develop the data and analyses necessary to accelerate the improvements in innovation. I will also describe how this new approach has begun to transform all of medicine by capturing the wisdom of the group, the insights that are provided by deviant practices, and the ability of Bayesian concepts to reduce data burden and provide pervasive data at an accelerated timeline. This approach not only improves innovation. It has been shown to rather remarkably reduce medical expenses by identifying and reducing unnecessary utilization.

In summary, this talk will use many real world examples of first-in-man therapies to provide a blueprint for successful innovation for the future.

James E. Lock, M.D.

James Lock Dr. James Lock is Cardiologist-in-Chief and Chairman of the Department of Cardiology at Children's Hospital Boston, and the Alexander S. Nadas Professor of Pediatrics at Harvard Medical School. Dr. Lock attended medical school at Stanford University, pursued pediatric residency and cardiology fellowship at the University of Minnesota, and trained in cardiovascular physiology for two years at the University of Toronto, Hospital for Sick Children. Scientific work on pulmonary vascular control was supported by an AHA Established Investigator Award, the March of Dimes (Basil O'Connor Award) and National Institutes of Health (RO1 HL). Dr. Lock shifted his research focus to experimental interventional cardiology, providing most of the basic research in that field. In 1984, he joined Children's Hospital as Director of the Cardiac Catheterization Laboratory, and became Department Chair in 1993.

Dr. Lock has invented several cardiac catheterization devices and has developed several dozen new non-surgical procedures to improve care for children with complex heart disease. In September 1999, the CardioSEAL device developed by Dr. Lock and others became the first septal occlusion device to receive FDA approval for use inside the human heart. He has authored or co-authored 250 peer-reviewed scientific papers. He is the president of Boston Children's Heart Foundation, the president of the Aldo Castaneda Foundation, and serves on the Finance Committee Board of Directors of the Children's Hospital.

During his research career, Dr. Lock has trained nearly a hundred academic physicians in cardiopulmonary physiology, experimental interventional cardiology and clinical interventional cardiology. Beginning five years ago, he and his colleagues started a highly innovative program to standardize clinical care in a fashion that improves outcomes, reduces unnecessary utilization and supports innovation. That program, termed SCAMPs, is now rapidly gaining acceptance throughout the medical community.