Faculty Bibliography

Objectives: Family-centered rounds (FCR) can improve communication and patient/family engagement. While use of informational resources (e.g., tablets, computers on wheels, paper notes) can guide FCR, there are limited data concerning parental perspectives on how use of these resources during FCR, or other factors, affect their engagement. Our objectives were to examine parental perspectives on factors that affect their participation during FCR and preferences for informational resources used. Methods: We performed a cross-sectional study with English-speaking parents (n = 200), of pediatric inpatients at an academic medical center, present during FCR. We surveyed parents to ascertain factors they believed affect their engagement during FCR. We asked about their preferences regarding informational resources used by the medical team. Responses were analyzed using descriptive statistics. Parents described their reasoning behind resource preferences, and we categorized these responses. Results: Parents reported that participation was affected by: clarity of the medical team's explanations (78.5%), understanding the information (75.5%), the child's health (74.5%), and being asked for their input (71%). Few (25%) parents believed the informational resource affects participation. Tablets were the preferred resource (24%) due to portability and ease of use, although 56% of parents had no preference. Conclusions: Parents of hospitalized children placed importance on delivery of clear information and an "invitation" to participate during FCR. The resource used by the team was less important, although tablets were most preferred. Next steps are to examine factors associated with objective measures of participation and further study FCR in families with limited English proficiency.

Longitudinally extensive spinal cord lesions (LECL) restricted to gray matter are poorly understood as are their neurodevelopmental repercussions in children. We herein report the critical case of a 13-year-old male presenting with progressive quadriparesis found to have cervical LECL restricted to the anterior horns. Challenged with a rare diagnostic dilemma, the clinical team systematically worked through potential vascular, genetic, infectious, rheumatologic, and paraneoplastic diagnoses before assigning a working diagnosis of acute inflammatory myelopathy. Nuanced consideration of and workup for both potential ischemic causes (arterial dissection, fibrocartilaginous embolism, vascular malformation) and specific inflammatory conditions including Transverse Myelitis, Neuromyelitis Optica Spectrum Disorders (NMOSD), Multiple Sclerosis (MS), Acute Disseminated Encephalomyelitis (ADEM), and Acute Flaccid Myelitis (AFM) is explained in the context of a comprehensive systematic review of the literature on previous reports of gray matter-restricted longitudinally extensive cord lesions in children. Treatment strategy was ultimately based on additional literature review of treatment-refractory acute inflammatory neurological syndromes in children. A combination of high-dose steroids and plasmapheresis was employed with significant improvement in functional outcome, suggesting a potential benefit of combination immune-modulatory treatment in these patients. This case furthermore highlights quality clinical reasoning with respect to the elusive nature of diagnosis, nuances in neuroimaging, and multifocal treatment strategies in pediatric LECL.

Drug discovery and development (DDD) is an interdisciplinary enterprise that spans the translational continuum. Despite DDD's importance, formal training within medical and biomedical schools is lacking. In this tutorial, we outline the current educational landscape in DDD and the growing educational need in this area. Lastly, we describe the Health Innovations and Therapeutics concentration as an example of how to design and implement an educational program in DDD.

PhDs and post-doctoral biomedical graduates, in greater numbers, are choosing industry based careers. However, most scientists do not have formal training in business strategies and venture creation and may find senior management positions untenable. To fill this training gap, "Biotechnology Industry: Structure and Strategy" was offered at New York University School of Medicine (NYUSOM). The course focuses on the business aspects of translational medicine and research translation and incorporates the practice of business case discussions, mock negotiation, and direct interactions into the didactic. The goal is to teach scientists at an early career stage how to create solutions, whether at the molecular level or via the creation of devices or software, to benefit those with disease. In doing so, young, talented scientists can develop a congruent mindset with biotechnology/industry executives. Our data demonstrates that the course enhances students' knowledge of the biotechnology industry. In turn, these learned skills may further encourage scientists to seek leadership positions in the field. Implementation of similar courses and educational programs will enhance scientists' training and inspire them to become innovative leaders in the discovery and development of therapeutics.

Drug discovery and development (DDD) is a collaborative, dynamic process of great interest to researchers, but an area where there is a lack of formal training. The Drug Development Educational Program (DDEP) at New York University was created in 2012 to stimulate an improved, multidisciplinary DDD workforce by educating early stage scientists as well as a variety of other like-minded students. The first course of the program emphasizes post-compounding aspects of DDD; the second course focuses on molecular signaling pathways. In five years, 196 students (candidates for PhD, MD, Master's degree, and post-doctoral MD/PhD) from different schools (Medicine, Biomedical Sciences, Dentistry, Engineering, Business, and Education) completed the course(s). Pre/post surveys demonstrate knowledge gain across all course topics. 26 students were granted career development awards (73% women, 23% underrepresented minorities). Some graduates of their respective degree-granting/post-doctoral programs embarked on DDD related careers. This program serves as a framework for other academic institutions to develop compatible programs designed to train a more informed DDD workforce.

Drug development (DD) is a multidisciplinary process that spans the translational continuum, yet remains an understudied entity in medical schools and biomedical science institutes. In response to a growing interest and unmet need, we implemented a DD course series that details identification of viable molecular targets, clinical trial design, intellectual property, and marketing. Enrollment is open to faculty, postdoctoral trainees, and MD, PhD, and MS students. After 2 years, 37 students and 23 students completed the fall and spring courses, respectively. Pre/post-surveys demonstrated gained knowledge across course topics, with mean survey scores increased by 66% (p < 0.001) after each course. Lectures for each course were consistently rated highly, with a mean course rating of 4.1/5. Through this program, trainees will have a more innovative approach toward identification of therapeutic targets and modalities. Furthermore, they will learn to integrate technology and biomedical informatics to find creative solutions in the DD process.

To develop the next generation of translational investigators, New York University School of Medicine (NYUSOM) and the NYU-NYC Health and Hospitals Corporation Clinical and Translational Science Institute (NYU-HHC CTSI) developed the Master's of Science in Clinical Investigation dual-degree (MD/MSCI) program. This 5-year program dedicates 1 year to coursework and biomedical research, followed by a medical school/research overlap year, to prepare students for academic research careers. This paper details the MD/MSCI program's curriculum and approach to mentorship, describes the research/professional interests of students, and reports student productivity. In the first 4 years of the program (2010-2014) 20 students were matriculated; 7 (35%) were women, and 12 (60%) research projects were in surgical specialties. To date, 14 students have applied to residency, and half pursued surgical residency programs. Our students have produced 68 accepted abstracts, 15 abstracts in submission, 38 accepted papers, and 24 papers in submission. Despite the time-limited nature of this program, additional training in research design and implementation has promoted a high level of productivity. We conclude that dual-degree training in medicine and translational research is feasible for medical students and allows for meaningful participation in valuable projects. Follow-up is warranted to evaluate the academic trajectory of these students. Clin Trans Sci 2015; Volume #: 1-6.

Cost-containment in healthcare spending has become a central issue in public policy and healthcare reform, especially as the affordable care act adds millions of people to public and private insurance rolls. In this climate, longstanding criticism of pharmaceutical pricing has grown sharper, and many in both policy and medicine have characterized the costs of newly developed drugs as both exorbitant and wasteful of scarce healthcare resources. At the same time, pharmaceutical research and development pipeline costs are increasing exponentially.Price resistance poses a significant threat to the development of drugs to treat rare pediatric diseases, where exceptionally high prices are a sine qua non of commercial viability. This article examines the trends in public discussion of high cost drugs and the potential consequences for orphan drug development. We conclude that despite growing public hostility towards high unit costs, drugs that treat rare diseases in children are likely to remain well-compensated and commercially viable.