TRIAL CONDUCT
AND
DRUG DEVELOPMENT
AN OVERVIEW OF
INVESTIGATOR RESPONSIBILITIES
NEW DRUG APPLICATION (NDA)
The NDA is the formal request from the sponsor to the FDA to provide approval for the marketing of a new drug.
BINDING / MASKING
A procedure in which one or more parties to the trial are kept unaware of the treatment assignment(s). Human behavior is influenced by what we know or believe. In research there is a particular risk of expectation influencing findings, leading to biased results. Binding (sometimes called Masking) is used to try and eliminate such bias.
COMPOUND IDENTIFICATION
PHASE I - Ref. CFR 312.21
The principal concern of Phase 1 is that the study be conducted safely. Protocols are established.
PHASE II - Ref. CFR 312.21
Trials are performed on larger groups (100-300) and are designed to assess clinical
efficacy of the therapy. The trials become more disease specific.
PHASE III - Ref. CFR 312.21
Studies are large double-blind randomized controlled trials performed on larger groups
(several hundred to several thousand), and are aimed at being the definitive assessment
of efficacy of the new therapy, especially in comparison to other standard therapies.
PHASE IV - Ref. CFR 312.85
FSA Approved Drug - Trials involve post launch safety surveillance and ongoing technical
support of a drug. Safety surveillance is designed to detect any rare or long term adverse effects over a much larger patient population and timescale than possible during clinical trials.
Special Studies - ICH 3.1.4 - may be required to assess the drug on special populations such as pregnant or nursing women, children, or the elderly. They may also address issues like drug to drug interactions, or studies of drug metabolites.
FDA DRUG APPROVAL PROCESS
Investigational New Drug (IND) Application - CFR-312
The IND application usually contains evidence of safety and tolerability in animals, a controlled method of manufacture that assures consistency of the final product, specific test for significant toxins, and a well developed research plan than minimizes the risks for human subjects.
After pre-clinical data is compiled, and the application for an investigational new drug (IND) is reviewed and approved by the FDA, the sponsor of the drug is ready to enter human trials.
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