The first step is to file an Investigational New Drug (IND) application which is supported by the data from the Pre-Clinical Phase. Studies performed under an IND are often classified into phases as if they are separate and distinct steps in the process. In reality, the phases overlap and trials in one phase are often conducted simultaneously with trials in other phases. Clinical studies are grouped according to their objective into three types or phases:

 

Phase I Clinical Development (Human Pharmacology) – Thirty days after a biopharmaceutical company has filed its IND, it may begin a small-scale Phase I clinical trial unless the FDA places a hold on the study. Phase I studies are used to evaluate pharmacokinetic parameters and tolerance, generally in healthy volunteers.  These studies include initial single-dose studies, dose escalation, and short-term repeated-dose studies.

Phase II Clinical Development (Therapeutic Exploratory) – Phase II clinical studies are small-scale trials to evaluate a drug’s preliminary efficacy and side-effect profile in 100 to 250 patients.  Additional safety and clinical pharmacology studies are also included in this category.

Phase III Clinical Development (Therapeutic Confirmatory) – Phase III studies are large-scale clinical trials for safety and efficacy in large patient populations. While phase III studies are in progress, preparations are made for submitting the Biologics License Application (BLA) or the New Drug Application (NDA).  BLAs are currently reviewed by the FDA’s Center for Biologics Evaluation and Research (CBER).  NDAs are reviewed by the Center for Drug Evaluation and Research (CDER).

 

The biological activity of nanoparticles can be influenced by the manufacturing process.