Justifying the Study

Your justification should be that originally presented to the executive committee, comprehensive yet summarized in no more than a page or two. Begin by stating the prevalence of the disease condition you propose to treat along with its effects. Here is an example: "Colorectal cancer is the second most common visceral malignancy in the United States. An estimated 156,000 new cases of the disease will occur "

The balance of your justification should briefly summarize previous work you and other investigators have done that would lead one to believe that the intervention you propose will be both safe and efficacious. Include data from any or all of the following sources:

• Pharmacology and biochemical theory—describe the mechanism of action if known

• Animal experiments—include toxicology findings

• Anecdotal studies

• Case-control studies

• Short-term clinical studies including Phase I determinations of the maximum tolerated dose and Phase II studies of the minimum effective dose.

You should also reference any previous full-scale clinical studies when you are proposing extensions of the subject population, modifications to the treatment regimen, or new indications for use of an already marketed intervention.

Brief descriptions of each study should be provided along with journal and text references where available.

Again, note that what distinguishes the full-scale clinical study from prior clinical trials is that it entails measures of both safety and efficacy, involves a predetermined dose or treatment regimen, is long-term, and includes sufficiently many patients that accurate estimates of the incidence of all but the rarest of side effects can be made.

Take pains to differentiate your proposal from prior work. For example, "The proposed study will include both men and women. It will involve more than 10 times the number of patients observed in any single previous study. The study period of one year is eight months in excess of any previous study period."

Remember, the object of your proposal is to convince even the most conservative and cautious reader that the investigation you propose is both prudent (because it is solidly grounded in prior efforts) and desirable (because the study's depth and breadth ensures the public will be fully protected once the study is complete).

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