The 3rd Stat4Onc Annual Symposium

Developing treatments for rare diseases

Speaker Biography and Abstract

Andrea Gross, M.D.

Bio:

Dr. Andrea Gross is a board-certified pediatrician who earned her medical degree at the University of Connecticut and completed pediatric residency and a chief resident year at Cincinnati Children’s Hospital Medical Center. She completed a pediatric hematology/oncology fellowship at Children’s National Medical Center and is currently an Assistant Research Physician working in the Pediatric Oncology Branch at the National Cancer Institute under the mentorship of Dr. Brigitte Widemann. Dr. Gross has been the lead associate investigator on the phase 2 trial of selumetinib for patients with NF1 and inoperable plexiform neurofibromas since 2015.

Title:

Clinical Trials for Rare Diseases: The NCI Pediatric Oncology Branch Experience

Abstract:

The world of pediatric oncology research exemplifies both the challenges in designing and implementing trials for rare diseases as well as the scientific breakthroughs that can be found from the careful study of rare conditions. From the development of Dr. Knudson’s “two hit hypothesis” from the study of hereditary retinoblastoma through to the revolutionary changes that chimeric antigen receptor (CAR) therapies are bringing to the treatment of leukemia, the study of rare pediatric diseases has been changing our understanding of disease pathology and treatment for decades. In recent years, our team, led by Dr. Brigitte Widemann, has studied the natural history of the genetic tumor predisposition syndrome neurofibromatosis type 1 (NF1) to establish novel clinical trial endpoints which have led for the first time to the discovery of effective agents for treating some manifestations of this condition. We are currently developing new projects to utilize this work as a model for learning about other tumor predisposition syndromes, such as germline mutations in the RAS pathway, with the hope of developing new therapies and generating new insights about cancer biology.

Jingjing Ye, Ph.D.

Bio:

Jingjing Ye is a statistics team leader in division of biometric V in CDER/FDA supporting pre-market reviews and approvals in DHP (Division of Hematology Products). She received her PhD in statistics from University of California, Davis. She has more than 13 years working experience in pharmaceutical industry and FDA. She is a statistics representative in OCE (oncology center of excellence) PeRC (Pediatric Review Committee) subcommittee.

Title:

Statistical and regulatory considerations on registrational trials in pediatric cancer

Abstract:

The Pediatric Research Equity Act (PREA; which provides the requirement for studies) and the Best pharmaceuticals for Children Act (BPCA; which provides additional exclusivity for sponsors who conduct requested studies in the pediatric population) were enacted in 1997 to correct the serious deficiencies on lack of pediatric prescribing information for approved drugs. Despite the increasing regulations and submissions based on the pediatric clinical trials in general, there are unique challenges present in cancer since the period also coincides with the explosion of oncology and hematology drug development. Because the pediatric and adult population do not share the same biology, natural history and disease progression, requirement of the registration studies to include pediatric prescribing information can be greatly varied according to the disease indications. In this presentation, we will present the current approvals in pediatric cancers, the statistical and regulatory considerations in the trials in support of approvals, in particular when it is rare cancer indications. We will present case studies when possible Bayesian approaches are considered.

Tiina Urv, Ph.D.

Bio:

Tiina Urv is the program director for the Rare Diseases Clinical Research Network (RDCRN), a multidisciplinary international program in the Office of Rare Diseases Research (ORDR). As the lead for the RDCRN program, Tiina collaborates with 10 NIH Institutes to manage 22 consortia and a central Data Management Coordinating Center(link is external). The RDCRN has more than 200 participating sites in 17 countries and more than 100 Patient Advocacy Groups as research partners and conducts research on about 200 rare diseases. Before joining the ORDR, Urv was a program director in the Division of Clinical Innovation where she provided stewardship for multiple Clinical and Translational Science Awards Program hubs and worked with the Trial Innovation Network as well as NCATS’ ORDR.

Tiina came to the National Institutes of Health (NIH) in October 2006, working as a program director at the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) in the Intellectual and Developmental Disabilities Branch. Prior to joining NIH, she was an assistant professor at the University of Massachusetts Medical School’s Eunice Kennedy Shriver Center and a research scientist at the New York State Institute for Basic Research in Developmental Disabilities. At NICHD, Tiina coordinated the Hunter Kelly Newborn Screening Research Program, chaired the trans-NIH Fragile X research program, and managed a diverse portfolio of basic, behavioral and bio-behavioral research related to developmental disabilities and rare diseases.

Tiina Urv is a developmental disabilities specialist with a Ph.D. from Columbia University. She earned her undergraduate degree from the University of Washington.