An estimated 30 million people in the United States are afflicted with rare (orphan) diseases or conditions; however, the availability of novel treatments for these diseases and conditions has been…
As Physiologically Based Pharmacokinetic (PBPK) modeling becomes more widely used in regulatory submissions, there are some common misconceptions that have been brought to our attention. We would like to dispel or…
The 505(b)(2) New Drug Application (NDA) is a streamlined NDA process in which the applicant relies upon one or more investigations conducted by someone other than the applicant and for…
In recent weeks, the world has welcomed very positive and much anticipated news on the COVID-19 vaccine front. At least two high-profile vaccines (mRNA vaccines from Pfizer/BioNTech and Moderna) have…
Inaccessibility, inadequate engagement, and lack of trust—these are just some of the issues that limit inclusion and lead to a lack of diversity in clinical trials. So, why is diversity…
What is Cellular & Gene Therapy? Genes are defined by the NIH as the “basic physical and functional unit of heredity.” We receive these valuable sequences of DNA from our…
What is PBPK Modeling & Simulation? Physiological based pharmacokinetic modeling and simulation (PBPK) is a computer modeling approach that incorporates blood flow and tissue composition of organs to define the…