Seminar Series : Seminars Series II

James Wilkinson, MD, MPH

Issues to Consider in the Design of Randomized Controlled Trials

Issues to Consider in the Design of Randomized Controlled Trials

This seminar will discuss the approach to the design of randomized controlled trials. Potential topics will include: observational vs. experimental studies, the rationale for randomization, simple vs. adaptive randomization, explanatory vs. intention to treat analysis, hypothesis classification (superiority vs. non-inferiority vs. equivalence trials), allocation concealment, blinding, the definitions and purposes of blocking, bias and confounding, attention controls, stopping rules, CONSORT, and the importance of statistical expertise in designing, monitoring, and interpreting the study.

Richard A Preston MD, MSPH, MBA

Getting a Project Started: The One-Page Protocol Synopsis

Getting a Project Started: The One-Page Protocol Synopsis

This lecture will describe a simple and relatively painless technique that can be used as an initial step in the development of a clinical research protocol. This lecture is especially intended for those who are new to clinical research.  Attention will be given to selection of study design, determination of endpoints, sample size estimation, and feasibility.

Erin Kobetz, PhD

Community-Based Research: From Bench to Trench

Community-Based Research: From Bench to Trench

This lecture will define Community-Based Participatory Research (CBPR), and will describe how CBPR can be used to address health disparities, how CBPR informs the scope and direction of future research and social change, and how CBPR advances the translation of scientific discoveries.

Steven Lipshultz, MD

Biomarkers, Course, and Surrogate Endpoints in Pediatric Cardiomyopathy

Biomarkers, Course, and Surrogate Endpoints in Pediatric Cardiomyopathy

This lecture will provide an overview of the importance of understanding the course in pediatric cardiomyopathy and the utility of biomarkers as surrogate endpoints for clinically important cardiovascular disease.

Jochen Reiser, MD, PhD

Kidney Service: Maintaining Filter Function Kidney Service: Maintaining Filter Function Kidney Se

Kidney Service: Maintaining Filter Function

This lecture will review novel pathomechanisms for proteinuric renal disease, novel principles of kidney filter function, and how to improve kidney health through targeting of podocytes.

Yellowlees Douglas, PhD

Benefiting from a K30 Program: Opportunities for Sponsored Research and Career Advancement

Benefiting from a K30 Program: Opportunities for Sponsored Research and Career Advancement

Dr. Douglas will address the opportunities for the University of Miami’s fellows and faculty stemming from their participation in UM’s new K30 program. She will discuss her experience as a faculty member in UF’s K30 Advanced Program in Post-Clinical Investigation (APPCI) and also focus on data gathered from the program’s twelve years of participants. Douglas will explore how the program can impact the participants’ amount of publications, intra- and extramural grants funded, and opportunities for career advancement. She will also consider how K30 participants can maximize the opportunities offered by the program, based on both surveys and her work with an array of APPCI participants.

Marc Lippman, MD, MACP

New Insights Into Metastatic Breast Cancer

Dr. Lippman will review the current status of clinical management of systemic breast cancer, with its obvious achievements and challenges. He will describe a new model for understanding the metastatic progression of breast cancer and the identification of both site specific and global genes likely involved in the metastatic process. Dr. Lippman will also discuss changes induced by the primary breast cancer in target tissues which make them receptive of metastases which are therapeutically exploitable.

Jeffery M. Vance, MD, PhD

Whole Exome Sequencing: The New Diagnostic Method for Genetic Disorders

Whole Exome Sequencing: The New Diagnostic Method for Genetic Disorders

Currently, over 4,000 monogenic syndromes have been described, but for nearly two-thirds of these, the molecular basis has not been identified due to the limitations of appropriate families or technology (www.omim.org). Recently, whole exome sequencing (sequencing just the genes that code for the actual proteins) has been very successful in identifying the gene defect in these patients. Increasingly, these newly identified gene defects have led to successful therapeutic intervention for the patient or family members. More than half of these syndromes are characterized by one or more birth defects. In addition, by identifying specific mutations in specific birth defects, we can return to the medical genetics clinics with the information that can form the basis for genetic testing for these previously un-examined genetic disorders. This will lead to better reproductive counseling and prenatal or pre-implantation diagnosis, which in turn leads to preventing the disease from affecting the child and family. By identifying the genetic mutations in these disorders, this information can be incorporated into the databases for clinical sequencing in the future. It provides the opportunity to greatly reduce the occurrence of these syndromes. This is analogous to the 90% drop in the incidence of Tay-Sachs disease over the past decade due to carrier testing in the Ashkenazi Jewish population.

Philip Chen, MD, PhD

Merging Clinical And Financial Informatics – The New Frontier Of Translational Medicine

Merging Clinical And Financial Informatics – The New Frontier Of Translational Medicine

As we transition from “Managing Individual Illness” in the 20th century to “Managing Population Health” in the 21st, we must deliver evidence-based and financially sustainable healthcare. Dr. Chen will discuss the development of informatics tools to decipher clinical and financial data, the approaches to identify and design interventional targets and how to integrate these initiatives in Value-based Purchasing (VBP) and Value-based benefit design (VBBD) in population health management and healthcare financial reform.

. R. Rodney Howell, MD, FAAP, FACMG

Newborn Screening: From the Public Health Laboratory, Into Federal Policy And Into Translational Res

Newborn Screening: From the Public Health Laboratory, Into Federal Policy And Into Translational Research

Dr. Howell will discuss the history of newborn screening, and how the work that began in the public health laboratory has led to the development of many new public policies, and increasingly is providing the impetus for important NIH-funded research developments.

Neil Schneiderman, PhD

Hispanic Community Health Study: Clinical and Translational Aspects and Implications

Hispanic Community Health Study: Clinical and Translational Aspects and Implications

The Hispanic Community Health Study/Study of Latinos (HCHS/SOL) is the largest NIH funded study of Hispanic/Latino health ever conducted. The HCHS/SOL is important because it is (a) examining likely causal factors of disease in a diverse population; and (b) identifying important variables that are likely to influence health care as health maintenance moves from the clinic to the community. The study is assessing risk factors for and prevalence of heart, lung, blood, and sleep disorders, kidney and liver dysfunction, diabetes, cognitive impairment, dental problems, and hearing disorders. Data from the study will be presented. The lecture will also address current ancillary studies and opportunities for UM personnel to propose new ancillary studies or publications based on the HCHS/SOL.

Ram H. Datar, M.Phil., Ph.D.

Nanotechnology in Clinical and Translational Research

Nanotechnology in Clinical and Translational Research

The presentation will address the role that microscale and nanoscale technologies play in clinical translational research, with some specific examples from Dr. Datar’s own research, and introduce the nascent Biomedical Nanotechnology Institute at University of Miami (BioNIUM) to the audience.