GCRC Biostatisticians

Susan M. Perkins, PhD

274-2626, sperkin1@iupui.edu

Chandan Saha, PhD

278-4283, cksaha@iupui.edu
Our Role in the GCRC
♦ Project Support
♦ Protocol Review
♦ Follow-Up Review
♦ Teaching of Residents
Project Support
♦ Study Design
♦ Statistical Methods
♦ Sample Size and Power Calculations
♦ Data Management Advice
♦ Data Analysis
Protocol Review
♦ Hypothesis
♦ Study Design
♦ Data Collection
♦ Statistical Methods
♦ Sample Size and Power Calculations
A Research Project: Comparison of the Antihypertensive
Efficacy of Torasemide and Furosemide in Patients with
Chronic Renal Insufficiency
♦ Patients with chronic renal insufficiency show decreased
salt and water excretion which leads to systemic arterial
hypertension.
♦ Torasemide and Furosemide have been found to be
effective in increased salt and water excretion for these
patients.
♦ Preliminary studies suggest that Torasemide has a
bioavailability of 90-100%, but Furosemide has a
bioavailability of 60%.
♦ This study aims at comparing these two drugs in Na
excretion and antihypertensive effects.
GCRC Biostatistics Section
♦ Hypothesis: Primary and Secondary
● A statistical hypothesis is an assertion
about a population.
● Example: Torasemide is superior to
Furosemide in Na excretion and
antihypertensive effects.
GCRC Biostatistics Section

♦ Study Design
● Design is the process or structure that produces
valid data to answer the research question.

● Example: (Randomized, double blinded,

crossover design) Study participants received
both drugs either Torasemide first and
Furosemide second or Furosemide first and
Torasemide second with one week washout
period.
GCRC Biostatistics Section
♦ Data Collection
● Description of how and what data will be
collected for answering the research question.

● Example: For each treatment (Torasemide and

Furosemide), both baseline and 24-hour post
treatment Na excretion and BP data were
collected at each period.
GCRC Biostatistics Section
♦ Statistical Methods
● Statistical tools to test the hypothesis.

● Example: If there is no carry-over effect,

treatment effects can be compared using a paired
t-test on the response variable,
D = (YT2 – YT1) – (YF2 – YF1), where T and F
indicate Torasemide and Furosemide, and 1
and 2 indicate time before and after treatment.
GCRC Biostatistics Section
♦ Sample Size and Power Calculations
● How many subjects are required to detect an
effect of certain magnitude with power x%
(usually 80% or 90%).

● Power (probability of detecting an effect if it

exists).

● Example: Using a paired t-test at the 5% level of

significance, 15 subjects would provide 80% power to
detect an effect that is at least 0.81 times the standard
deviation.
Writing Biostatistics Section
♦ Hypothesis
● Clearly state the primary and secondary (if any)
hypotheses.

♦ Study Design
● Describe how the study will be conducted.
● Will it be a single/double blinded design?
● How the randomization will be performed.
● How the study subjects will be enrolled.
● When will subjects be seen?
● Include a timeline.
Writing Biostatistics Section
♦ Statistical Methods
● Mention what descriptive statistics will be used.
● Choose an appropriate statistical tool to test the
hypothesis.
● Justify the choice of the statistical tool to test the
hypothesis

● Include if any confidence interval for an estimate will

be reported.
● State how the assumption(s) (if any) of the statistical
method will be tested.
Writing Biostatistics Section
♦ Data Collection
● Describe when, how and what data will
be collected.
● Do you have all relevant data to test the
hypothesis and for other secondary analysis?
Writing Biostatistics Section
♦ Sample Size and Power Calculation
● Pilot study? If so, describe.
● Minimum detectable difference.
● Variability assumed or from previous similar study.
● Power (often 80%)
● Significance level (often 5%)
● 1 or 2 sided
● Test used (usually the same as in statistical methods)
● What sample size do you need?
Writing Biostatistics Section
♦ Drug Study or NIH Grant?
● Need elements described earlier
● Should be in the protocol or grant
Follow-Up Review
♦ Provide our feed back to the investigators.
♦ Ask for a meeting with the investigators if
further clarifications are needed.
♦ Suggest the modifications if required in
study design, statistical methods and sample
size calculation.
♦ Review the second time submitted
protocols.
Teaching of GCRC Residents
♦ Provide only to the GCRC Residents
♦ Understanding and Application of Statistical
Concepts to Clinical Research
♦ Textbook: Norma and Streiner, Biostatistics,
The Bare Essentials
♦ One to One Session, 6-8 Sessions, 30 – 60
Minutes Per Session
♦ Meet Twice a Week
♦ Provide Reading Assignments and Discuss the
Materials