Listings

The rapidly evolving field of regenerative medicine, marked by advancements in cell and gene therapies, has prompted regulatory agencies to work diligently to adapt to these innovations. This course offers an overview of regenerative medicine therapies and the necessary FDA’s oversight.

The course delves into the development of a novel treatment for focal cartilage lesions, highlighting the transition of such treatments into clinical trials. Insights into the process clinical trial initiation are transferrable, offering valuable lessons applicable to other novel treatment developments.

This course discusses the manufacturing of Chimeric Antigen Receptor (CAR) - T cells within an academic GMP facility, along with addressing the challenges associated with integrating this cell therapy into clinical practice.

Age-related macular degeneration (AMD) is the leading cause of permanent vision loss in people 65 years and older. This course provides an overview of the disease and highlights the development of stem cell therapy treatments. It discusses the translation of stem cell therapy to restore retinal photoreceptor cells, including early phase 1b trial results of first in human clinical trials.

The steps and requirements for developing gene therapies for Huntington’s Disease (HD) will be discussed. Current HD therapies being studied and those already approved will be covered.

The fundamental principles of statistics, including hypothesis testing, power, multiplicity, mathematical and data adjustments, and statistical confidence will be discussed. These principles applied to study design inform clinical endpoints, sample size, perceptual influences, validity, and missing data.

Basic considerations for designing medical device trials such as device complexity and limitations, resource constraints, availability of a control group, and key stakeholders (i.e., hospital administration, radiologists, and clinicians) will be discussed. Characteristics unique to medical devices will be further explored via case studies using adaptive trial design for device testing.

A general overview of pediatric trials, discussing pediatric subpopulations, laws related to pediatric research, the consent process, and clinical trial design considerations.

This course provides an in-depth exploration of various clinical trial design types (i.e., cohort, case-study, quasi-experimental, etc.) along with the statistical methods often used respective to each type (i.e., Chi-square test, T-test, non-parametric Wilcox logistic regression, etc.).

Policies from the NIH and FDA on participant selection and data reporting in clinical research as well as rationale behind these policies will be discussed. Other topics include subgroup reporting and varied experiences that affect research accuracy and medical advancements.

The fundamentals of genomics testing will be discussed along with the FDA and FDA guidelines on the incorporation of data from genomic tests.

This course discusses the FDA’s Critical Path Initiative (CPI), a national strategy developed to improve the methods used to move therapies from the laboratory to the patient in a more efficient manner.

This course explains how genetic variation influences the response to drug therapy. From discovery of pharmacogenetic associations to the evaluation of utility in clinical practice.

The first human genetic blueprint has just turned 20; the era of exorbitant costs to analyze one genome has now evolved to the use of digital bar codes to sequence DNA. Because of the sizeable quantity of complex data associated with human genomes, genomics is now considered a “big data” field.

This deep dive into HD provides an overview of applying genomics within the drug discovery process and how it relates to diagnosis, target identification, prognosis, and stratification target identification.

The use of technology in the medical device industry facilitates data capture and analysis. An overview of technological systems such as artificial intelligence and machine learning and the way individuals interact with these systems, and the results of those interactions, will be discussed.