Investigational New Drug
Investigational New Drug - Related Videos
10:22
Basophil Activation Test for Investigation of IgE-Mediated Mechanisms in Drug Hypersensitivity
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Cited by 14 •
2011
Basophil activation test is a potent tool for the detection of IgE-dependent allergies in vitro. Here, an optimized protocol for basophil activation test is used to investigate drug hypersensitivity. A method for the efficient production of covalent drug-protein conjugates and their physicochemical characterization is described.
08:34
A Rapid and Quantitative Fluorimetric Method for Protein-Targeting Small Molecule Drug Screening
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Cited by 6 •
2015
A protocol for small molecular drug screening based on in-situ synthesis of ultrasmall fluorescent gold nanoclusters (Au NCs) using drug-loaded protein as template is presented. This method is simple to determine the binding affinity of drugs to a target protein by a visible fluorescent signal emitted from the protein-templated Au NCs.
05:10
Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
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Cited by 4 •
2016
Here we describe a protocol using the web-based drug repurposing hypothesis generation tool: "RE:fine Drugs." This protocol can be modified to a user's preferences at the level of the query type (gene, drug or disease) and/or the range of available advanced options.
07:02
A Computerized Test Battery to Study Pharmacodynamic Effects on the Central Nervous System of Cholinergic Drugs in Early Phase Drug Development
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Cited by 5 •
2019
A validated computerized battery of neuropsychological and neurophysiological tests is used to study pharmacodynamic effects on the central nervous system of newly developed drugs in early phase development. To demonstrate the test battery, the acute effects of mecamylamine and the reversal of these effects by two agonist drugs are described.
07:43
An Enzyme- and Serum-free Neural Stem Cell Culture Model for EMT Investigation Suited for Drug Discovery
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Cited by 8 •
2016
Epithelial to mesenchymal transition (EMT) allows cancers to become invasive. To investigate EMT, a neural stem cell (NSC)-based in vitro model devoid of serum and enzymes is described. This standardized system allows quantitative and qualitative assessment of cell migration, gene and protein expression. The model is suited for drug discovery.
FAQs
The submission organizes laboratory pharmacology and toxicology findings into evidence for evaluating whether human testing can begin. These data help the U.S. Food and Drug Administration assess whether anticipated participant risks are reasonable in relation to the proposed study. In this way, the process provides a regulatory transition from experimental findings to controlled clinical investigation.
Pharmacology data describe how the candidate drug may act, while toxicology findings address potential harmful effects identified before clinical exposure. Together, they give regulators a scientific basis for judging whether the proposed human study presents reasonable risks. Their role is not merely descriptive; these findings support the decision to permit or withhold progression into clinical research.
The review considers three connected areas: pharmacology and toxicology evidence, drug manufacturing and quality, and the proposed clinical protocol. Examining these elements together allows the FDA to assess both the investigational product and the conditions under which participants would receive it. This integrated evaluation supports decisions about whether the study can proceed safely and appropriately.
In pharmacology, first-in-human studies use the authorized clinical framework to evaluate dose, safety, pharmacokinetics, and pharmacodynamic effects. Pharmacokinetics concerns the drug’s behavior in the body, whereas pharmacodynamic effects concern its biological effects. Considering these outcomes together helps investigators connect administered doses with exposure, tolerability, and measurable drug activity during early clinical research.
A sponsor assembles evidence in three principal categories before submission: preclinical pharmacology and toxicology, manufacturing and product quality, and the proposed clinical protocol. The package therefore addresses scientific evidence, the identity and quality of the drug, and the planned human study. This structure gives the FDA the information needed to evaluate participant risk and study readiness.
Authorization does not end regulatory responsibilities once a study begins. The sponsor and investigators must maintain protocol compliance and provide ongoing safety reporting. These obligations allow emerging safety information to remain part of study oversight and help ensure that the research continues under the conditions reviewed by the FDA. They are essential for responsible translation into therapeutic development.
The process is especially important when a candidate moves from laboratory investigation into first-in-human evaluation. At that point, researchers need structured evidence about dose, safety, pharmacokinetics, and pharmacodynamic effects, while regulators need information for assessing participant risk. In pharmacology, this oversight helps determine whether preclinical findings can support further clinical research and eventual therapeutic development.