The 3rd Stat4Onc Annual Symposium
Developing treatments for rare diseases
Speaker Biography and Abstract
Andrea Gross, M.D.
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.
Trials for Rare Diseases: The NCI Pediatric Oncology Branch Experience
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.
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.
and regulatory considerations on registrational trials in pediatric cancer
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.
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
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.
is a developmental disabilities specialist with a Ph.D. from Columbia University.
She earned her undergraduate degree from the University of Washington.