“Proof of Concept” (POC) studies can provide “first in man” evidence that a therapeutic strategy has medical merit and therefore commercial value. Often potential investors and development partners wish to see critical information which provides evidence that the proposed therapeutic strategy will work “as advertised” in man. Traditional drug development strategies require moving a molecule along a proscribed drug development path, often taking several years and multiple investments of millions of dollars to obtain a “first in man” experience. These studies often do not address questions which are necessary to determine that the proposed therapeutic approach will work. These questions often deal with issues such as pharmacokinetics, drug distribution, mechanism of action, specificity of a molecule, drug metabolism, and other issues not necessarily associated with efficacy. To help address this need, the FDA permits time/dose-limited, small scale clinical studies to provide data to help guide the decision-making process in drug development, and support investment and partnering decisions. For potential drugs that can be manufactured in the state where the study will be done, an IND from the FDA is not required but state health department sanction is. Ordinarily state requirements are much less costly, quicker and easier to obtain.
An Exploratory IND can help:
- Determine if a mechanism of action can be observed in humans
- Provide information on Pharmacokinetics
- Explore a product’s biodistribution using imaging technologies
- Select the most promising lead product from a group of candidates
The desired outcome is to obtain results which provide POC data to address key questions on the merits of the molecular entity and reduce the risk to potential investors and development partners, while eliminating the normally large resource/dollar commitment and time allocation associated with a traditional IND submission.
ClinSmart has strategies for developing and executing exploratory INDs for the purpose of obtaining initial clinical experience with new proposed therapeutics. ClinSmart can assist in writing protocols for POC studies and have successfully managed many POC studies from start to finish.