Premature infants with bronchopulmonary dysplasia (BPD) often die and survivors have life-long morbidities. BPD is the most common morbidity of prematurity, affecting >18,000 infants per year. The NIH defines BPD as the receipt of exogenous oxygen or positive airway pressure at 36 weeks adjusted age. The costs of BPD are both social and economic and are measured in impaired childhood health and quality of life, family stress and economic hardship, and increased healthcare costs. Because the consequences of BPD are catastrophic, neonatologists frequently use drugs without proven efficacy in an attempt to prevent and treat BPD. Unfortunately, there are no FDA approved drugs for the prevention or treatment of BPD. Developing safe and effective drugs for BPD is an ideal opportunity to improve public health and provides a platform to develop trainees? expertise in trial design, regulatory requirements, and rigorous quantitative methods that will be applied to their own independent research career. The investigator, Dr. Matthew M. Laughon, MD, MPH is a practicing neonatologist with an MPH in epidemiology and training in clinical pharmacology. Dr. Laughon has secured access for trainee development to The University of North Carolina at Chapel Hill programs including the Division of Neonatal-Perinatal Medicine, the Gillings School of Public Health, and the Eshelman School of Pharmacy. Dr. Laughon also has a longstanding academic partnership with the world?s largest academic research organization, the Duke Clinical Research Institute. Through these programs, Dr. Laughon will mentor trainees at UNC and Duke and provide a pathway for trainees to secure advanced training in epidemiology, clinical pharmacology, or pharmacoepidemiology. Beginning clinician investigators will follow a logical progression including designing an original hypothesis driven research project, completing didactic studies, participation in one on one and group mentoring, and regular evaluation and feedback. Trainees will focus on the preparation and conduct of early-phase drug trials in neonatal lung disease, primarily BPD. Proof of the feasibility of these expectations is provided by current and former trainees who have achieved success in quantitative, patient-oriented research. Determining the PK and safety of drugs for BPD in premature infants is an urgent, unmet public health need. Investigating drugs for BPD represents an ideal opportunity to approach the problem of the research gap of drugs in premature infants because: 1) Premature infants with BPD have life-long morbidities; 2) There are no drugs that have been proven to prevent or treat BPD; 3) Neonatologists are commonly using drugs in premature infants without appropriate dosing and safety studies; and 4) Preventing mortality and morbidities associated with premature birth has life-long benefits. The proposed trainee-led research project will use an opportunistic study design of 10 commonly used drugs that will provide a platform for trainee to develop all aspects of clinical research skills and a rational approach to drug development in premature infants.
The candidate will use the protected time of the K24 to help develop trainees? skills so they can develop into an independent researcher, to expand the current premature infant lung disease research program through trainee-led cohort studies, and increase mentoring skills. These efforts will be synergistic, increasing the program productivity and the pool of well-trained clinical researchers for the future. Successful completion of the proposed opportunistic PK and safety study of BPD drugs will provide a platform for rational drug dosing to prevent and treat BPD in premature infants while simultaneously allow trainees to develop their clinical research skills.
