Question

TESU has discovered a new fictitious compound called Edistatin, a Low-Chol, they believe has promising pharmacologic benefit. A patent was granted for the compound, and all of the required animal testing was submitted to the FDA with the IND application. The FDA gave approval to proceed with human testing. Construct a one-page summary of what you think should be the required studies Phase I, II, and III to submit in the NDA. For each phase, include the objective of the study (studies), type of subjects selected, outcomes measures (i.e. cholesterol reduction and safety), and results. Be creative!! There are no right or wrong answers. We are interested in your approach based upon your readings.

Background: Assume that the effective dose is 10 and 20 mg. The safety profile is that of other statin products such as Lipitor, etc.

Answer 1

Phase I, II, III and IV studies include the following steps;

Phase I

Study Participants: 20 to 100 healthy volunteers or people with the disease/condition.

Length of Study: Several months

Purpose: Safety and dosage

Approximately 70% of drugs move to the next phase

Phase II

Study Participants: Up to several hundred people with the disease/condition.

Length of Study: Several months to 2 years

Purpose: Efficacy and side effects

Approximately 33% of drugs move to the next phase

Phase III

Study Participants: 300 to 3,000 volunteers who have the disease or condition

Length of Study: 1 to 4 years

Purpose: Efficacy and monitoring of adverse reactions

Approximately 25-30% of drugs move to the next phase

Phase IV

Study Participants: Several thousand volunteers who have the disease/condition

Purpose: Safety and efficacy

In the IND application, the drug developers must include:

·         Animal study data and toxicity including the side effects that cause great harm to the data

·         Manufacturing information

·         Clinical protocols including the study plans for studies to be conducted

·         Data from any prior human research

·         Information about the investigator