CHAPTER 2
Strategy and Statistics in Clinical Trials
©
2011 Elsevier Inc. All rights reserved. 17
n Trial design: multidisciplinary considerations
n Developing a diagnostic kit
n Intended use and trial design
n Essential questions for planning a study
INTRODUCTION
In Chapter 1, I described a situation in which management asks you to justify
a molecule's further development. You then prepare a presentation on the
scientific merits of the product. Having heard the presentation, management
decides to allocate resources for a first trial in humans with the disease. It is
only then that you begin to fret about the upcoming study's design. Or is it?
Questions For Planning Trials
CONTENTS
I said before that “everything is connected to everything
else”—that most every issue in clinical trials cannot be con-
sidered in isolation from others. Well, I will now belabor the
point. It is that important. One can only hope that having
made it twice, I shall henceforth desist (doubtful).
To the point belabored: Scientific justification for a medical
product is crucial. Yet, this sort of evidence by itself cannot
and should not determine whether to develop a product. To
take an obvious example, a company's resources must be
considered as well. If the money is not there, the trial will go
begging irrespective of the scientific merit for it. Moreover,
potential costs and benefits must be weighed in the context
of an organization's overall pipeline—the other products it is
currently developing and their needs and potential returns.
Management has now heard that CTC-11 is a scientifically
promising molecule and thinks favorably of it. But it also
knows that Allerton's Palsy is relatively rare, so the mole-
cule's potential market is small. What should a company do
when choosing between a product with, say, more scientific
promise than another but with less market potential? In
addition, market potential itself is difficult to estimate in
that it depends on numerous factors in addition to disease
MORAL OF THE SAME
Continued

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