Bethesda, MD
Description
Investigators seeking to develop and conduct Phase IIB/III clinical trials in heart, lung, blood and sleep diseases are often faced with challenges that arise from issues such as the rarity of the disease, access to the target population, and sufficient clinical trial design support necessary for development of robust and competitive investigator initiated clinical trial applications to NHLBI.
Purpose
The National Heart, Lung, and Blood Institute (NHLBI) convened a workgroup of multidisciplinary researchers on May 2 and 3 2016, in Bethesda, MD. The goal was to identify and provide recommendations to address the barriers to the design and implementation of Phase IIB/III clinical trials for challenging populations and indications facing heart, lung, blood and sleep investigators. Challenges include but are not limited to trials for uncommon diseases, and non-centralized patient management. The main goals of the workgroup were as follows:
- Identify key barriers to investigator and trial success
- Link barriers to gaps in training and clinical trial resources
- Make recommendations for the National Heart, Lung, and Blood Institute (NHLBI) to consider to close gaps and support both trainees and established investigators
Workgroup Co-Chairs:
- Susan S. Ellenberg, Ph.D., University of Pennsylvania Perelman School of Medicine
- Suresh Vedantham, M.D., Washington University School of Medicine
Workgroup Participants:
- Eugene Blackstone, M.D., Case Western Reserve University
- Diane Catellier, Ph.D., RTI International
- Marianne S. Clancy, R.D.H., M.P.A.; HHT Foundation International
- Ed Connor, M.D., M.B.E., Clinical Research Alliance, LLC
- Susan Geyer, Ph.D., University of South Florida
- Michael B. Jordan, M.D., Cincinnati Children's Hospital
- Fedor Lurie, M.D., Ph.D., University of Michigan and President, American Venous Forum
- Sarah O’Brien, M.D., M.Sc., Nationwide Children’s Hospital
- Jeff Sestokas, M.S., Children’s National Health System
- Mark Walters, M.D., Children's Hospital and Research Center
- Deepika Darbari, M.D., Children’s Research Institute
- Alice Kuaban, M.S., American Society of Hematology
- Amanda Kasper, MPH, Children’s National Health System
NHLBI staff:
- Amy Patterson, M.D., NHLBI Director of the Scientific Research Program, Policy, and Strategic Initiatives
- W. Keith Hoots, M.D., Director, Division of Blood Diseases and Resources
- Donna DiMichele, M.D., Deputy Director, Division of Blood Diseases and Resources
- Jacqueline Corrigan-Curay, M.D., Office of the Director
- Pablo Cure, M.D., M.P.H., Division of Blood Diseases and Resources
- Nancy DiFronzo, Ph.D., Division of Blood Diseases and Resources
- Simone Glynn, M.D., Division of Blood Diseases and Resources
- Malgorzata Klauzinska, Ph.D., Division of Blood Diseases and Resources
- Andrei Kindzelski, M.D., Division of Blood Diseases and Resources
- Traci Mondoro, Ph.D., Division of Blood Diseases and Resources
- Lisbeth Welniak, Ph.D., Division of Blood Diseases and Resources