Statistics and Statistical Careers
in Clinical Trials
Joseph Massaro
Department of Biostatistics
Boston University School of Public Health
jmm@bu.edu
Clinical Trial
• Clinical trials: Trials to evaluate the
effectiveness and safety of medications or
medical devices by monitoring their effects on
large groups of people (www.medterms.com).
Lipitor (Atorvastatin) Effect on MI/Death
(from www.drugs.com/pro/lipitor.html)
Example of a Clinical Trial Question on
Advanced Placement Exam
• FDA will recommend a new cholesterol drug if there is
sufficient evidence it reduces cholesterol >10 mg/dl
more than placebo, on average, over 6 weeks. Let μE,
μP = true mean 6-week cholesterol reduction (mg/dl)
for the experimental and placebo, respectively.
Example of a Clinical Trial Question on
Advanced Placement Exam
• FDA will recommend a new cholesterol drug if there is
sufficient evidence it reduces cholesterol >10 mg/dl
more than placebo, on average, over 6 weeks. Let μE,
μP = true mean 6-week cholesterol reduction (mg/dl)
for the experimental and placebo, respectively.
– A. What is the null and alternative hypothesis of interest?
Example of a Clinical Trial Question on
Advanced Placement Exam
• FDA will recommend a new cholesterol drug if there is
sufficient evidence it reduces cholesterol >10 mg/dl
more than placebo, on average, over 6 weeks. Let μE,
μP = true mean 6-week cholesterol reduction (mg/dl)
for the experimental and placebo, respectively.
– A. What is the null and alternative hypothesis of interest?
H0: μE - μP ≤ 10 vs. H1: μE - μP > 10
Example of a Clinical Trial Question on
Advanced Placement Exam
• FDA will recommend a new cholesterol drug if there is
sufficient evidence it reduces cholesterol >10 mg/dl
more than placebo, on average, over 6 weeks. Let μE,
μP = true mean 6-week cholesterol reduction (mg/dl)
for the experimental and placebo, respectively.
– B. 200 people were in the clinical trial: 100 received
experimental and 100 received control. The sponsor
performed a paired t-test to test H0. Is this appropriate?
Example of a Clinical Trial Question on
Advanced Placement Exam
• FDA will recommend a new cholesterol drug if there is
sufficient evidence it reduces cholesterol >10 mg/dl
more than placebo, on average, over 6 weeks. Let μE,
μP = true mean 6-week cholesterol reduction (mg/dl)
for the experimental and placebo, respectively.
– B. 200 people were in the clinical trial: 100 received
experimental and 100 received control. The sponsor
performed a paired t-test to test H0. Is this appropriate?
No: the two groups are independent.
Degrees and Careers
• To pursue a career in clinical trials, stat/biostat
degrees to first consider are:
– Bachelors (undergraduate)
– Masters of Public Health (“concentration” in
biostatistics)
– Masters in Statistics/Biostatistics
– Ph.D. in Statistics/Biostatistics
Degrees and Careers
• Bachelors
– Gets your “foot in door” – entry level jobs
– To advance career as a clinical trials biostatistician,
strongly consider graduate degree
– Bachelors in stats/biostats not necessarily required
to pursue graduate degree in stats/biostats
• Mathematics
• Biology
• Need at least 2 semesters of Calculus and Linear Algebra
Degrees and Careers
• Masters of Public Health (MPH)
– MPH usually very applied curriculum (little statistical theory)
– Again, gets your “foot in door” as clinical trial biostatistician
• Probably can handle 50-75% of clinical trial analyses
– To advance career as a clinical trial biostatistician, consider
Masters or Ph.D. in statistics/biostatistics
– Other clinical trial opportunities with MPH:
• Require knowledge of statistics but not necessarily performing
statistical analysis
– Statistical Programmer
– Data Manager
– Trial Manager
Degrees and Careers
• Masters in Statistics/Biostatistics
– Applications and underlying theory
– Often more than adequate to have a successful
biostatistical career in clinical trials
• Design clinical trials
• Develop statistical plan for clinical trials
• Carry out analysis
– If “upper management” is a goal, consider Ph.D.
Degrees and Careers
• Ph.D. in Statistics/Biostatistics
– More applications/more advanced theory
– Similar biostatistical opportunities as Master’s, but
perhaps for more complex studies.
• Also, present (and “defend”) designs and analysis
results to regulatory agencies
– Often required for upper
management/supervisory positions
Intangibles
• Be a good “team” member – ability to work with
others
• Ability/willingness to investigate and learn
• Starting out: willingness to do “entry level” tasks
– EVERY task has an opportunity to learn
– While in graduate school I performed data entry for
two years
• I learned in that time about how data is collected and
managed for a clinical trial
– Proofread clinical trial reports/manuscripts
– Double-check computer output (quality control)
Don’t Ignore Other Disciplines in
Undergraduate Career
• E.g., Clinical trial statisticians need to be good
writers (grammar, conciseness)
– Manuscript writing for journals
– Reports for FDA