Report

Statistics and Statistical Careers in Clinical Trials Joseph Massaro Department of Biostatistics Boston University School of Public Health [email protected] 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