30

SECTION I

PHARMACEUTICAL PRACTICE

3. Number needed to treat (NNT)

a) Number of subjects needed to treat over a
defined period of time to experience one
benefit of therapy
b) NNT 1/ARR
IV. Evaluating Clinical Trials
A. Questions to consider
1. Why was this study conducted?
2. Were previous trials conducted?
B. Consider the power/significance of the study
1. Statistically
2. Clinically
C. Critique the trial
1. Population
2. Intervention
3. Endpoints: were they appropriate?
4. Statistics
a) Consider the appropriateness of each
statistical test and result
D. Can the findings from this study be extrapolated to
patient/consumer?
V. Study Types in Clinical Research
A. Randomized controlled trial
1. An experiment in which investigators assign, by
random allocation, eligible subjects into
intervention groups to receive or not to receive
one or more interventions that are being
compared
2. Gold standard: Randomized controlled trials are
considered to have the highest validity and
reliability of various research designs, as they
eliminate causes of bias and provide a high
level of experimental control.
3. Necessary for Food and Drug Administration
(FDA) approval
B. Cohort studies
1. Group of subjects who have not yet
experienced the outcome of interest
2. Subjects exposed to a factor of interest are
compared to a group not exposed and followed
prospectively over time
C. Case-control studies
1. Subjects with a particular characteristic are
compared to a similar group without the
characteristic to determine the cause
2. Retrospective
D. Case reports
1. No control
2. No designed intervention
3. Descriptive account of a subject
E. Meta-analysis
1. A method of combining results of previous and
similar research to determine a single estimate
of treatment
F. Cross-sectional studies
1. Measurements taken at a single point in time
G. Survey
1. Research used to study the incidence,
distribution, and relationship
H. N-of-1 trials
1. Randomized controlled study involving a single
subject
2. Crossover design
3. Lack of generalizability to a population

VI. Research involved in the FDA Drug Approval Process

A. Preclinical research
1. Goal: assess potential therapeutic effects
2. Does not predict human response
B. Phase I
1. Initial study, usually in healthy human volunteers
2. Small number of subjects (fewer than 100
subjects); brief length of study (less than 1 year
3. Determines toxicology, metabolism, and
pharmacologic activities; early evidence of
effectiveness
C. Phase II
1. Expanded drug study to obtain preliminary
efficacy data and safety in humans
2. Small and highly homogeneous population
of patients for whom the drug is intended
(N several hundred participants)
D. Phase III
1. Pivotal trials
2. Larger study (N hundreds to thousands of
participants)
3. Long-term (up to several years)
4. Semidiverse population (representing target
population)
5. Establishes final formulation, marketing claims,
product stability, packaging, and storage concerns
6. Successful completion may mean ready to
submit compound to FDA for approval
E. Phase IV (postmarketing surveillance)
F. New Drug Application (NDA) form
G. Abbreviated NDA (aNDA) form: generic drug
approval
H. Supplemental NDA (sNDA): approval for new
indication

Reference
Haney MS, Meek PD: Essential clinical concepts of
biostatistics, Kansas City, 1999, ACCP.

REVIEW QUESTIONS
(Answers and Rationales on page 320.)
1. A customer requests a recommendation for a
reliable brand for ginseng. To ensure that she gets a
ginseng product that has been tested for quality,
what website(s) should a pharmacist consult?
I. ConsumerLab.com
II. ConsumerReports.org
III. American Society of Health-System Pharmacists
(ASHP) Essentials
a.
b.
c.
d.
e.

I only
III only
I and II only
II and III only
I, II, and III

2. A customer requests a recommendation for a

reliable brand for honeysuckle. To ensure that she
gets a honeysuckle product that has been tested for
quality, a pharmacist should NOT consult which of
the following websites?
a. ConsumerLab.com
b. ConsumerReports.org