FDA Drug Development Fellowship
*Applications will be reviewed on a rolling-basis.
A research opportunity is available in the Office of Translational Sciences / Office of Clinical Pharmacology, Center for Drug Evaluation and Research (CDER), Food and Drug Administration (FDA) in Silver Spring, Maryland.
Growth hormone deficiency (GHD) is a rare disorder characterized by the inadequate secretion of growth hormone (GH) from the anterior pituitary gland, a small gland located at the base of the brain that is responsible for the production of several hormones. The incidence of short stature associated with GHD has been estimated to be about 1:4000 to 1:10000. In children, GHD results in growth retardation and short stature. In adults, GHD results in a number of variable symptoms including reduced energy levels, altered body composition, osteoporosis (reduced bone mineral density), reduced muscle strength, lipid abnormalities such as increased LDL cholesterol, insulin resistance, and impaired cardiac function. The differences in therapeutic goals result in different doses between adult and pediatric patients, therefore makes the pediatric dose selection independent of adult GHD. The pediatric doses are usually found to be supraphysiological in adults and are associated with numerous side effects.
The therapeutic goal of GH replacement therapy for pediatric GHD (pGHD) is normalization of height velocity during childhood and attainment of normal final adult height. Because these changes occur slowly and become evident after at least 6 months of treatment, the primary efficacy endpoint is normally height velocity (HV) at 6 to 12 months in Phase 2 dose ranging studies.
Insulin-like growth factor-1 (IGF-1) is responsible for the great majority of growth hormone effect. Current clinical practice guidelines acknowledge the potential utility of monitoring IGF-1 for safety reasons in children and as a surrogate efficacy endpoint in adults. The outcome of this research is to evaluate IGF-1 – HV relationship to inform clinical trial design, and dose selection, to aid in GH product development in pediatric GHD.
The participant will be trained to collect relevant data, research and review various trial design elements such as study design, primary endpoints, and patient population for different growth hormone product development programs for FDA-approved products. This will provide an opportunity to learn drug development in pediatrics and regulatory science.
Qualifications
The qualified candidate should be currently pursuing or have received a doctoral degree in Bioinformatics, Materials Science Engineering, Mathematics, Pharmacology, Statistics, or a related field. Degree must have been received within five years of the appointment start date.
Background in drug development, clinical trial design, and data analysis are desired.
If you have questions, send an email to ORISE.FDA.CDER@orau.org. Please include the reference code for this opportunity (FDA-CDER-2020-0513) in your email.