1. Biostatistics, Epidemiology, and Research Design (BERD) PAUL NIETERT, PHD SCTR LUNCH-N-LEARN DECEMBER 9, 2015

2. The BERD TEAM  Madison Hyer, MS  Beth Wolf, PhD  Jeff Korte, PhD  Paul Nietert, PhD  Yuko Palesch, PhD  Ramesh Ramakrishnan, PhD  Amy Wahlquist, MS  We are all faculty member in the Department of Public Health Sciences.

3. BERD: Aims for the Next 5 years  AIM 1: Provide methodological guidance to translational researchers.  AIM 2: Provide an innovative, specialized consultative service within the Clinical Trials Design Center, aimed at assisting investigators with the planning and design of multicenter trials.  AIM 3: Conduct research into novel biostatistical methodologies.

4. BERD: Aims for the Next 5 years  AIM 4: Participate in the biostatistical educational development of trainees and junior investigators.  AIM 5: Review applications for internal SCTR funding for scientific merit, with emphasis on study design, statistical analysis plans and sample size justification.

5. AIM 1: Provide methodological guidance to translational researchers.  Investigators can make requests through SPARC.  We provide assistance with:  Study design  Sample size estimation / power analyses  Statistical analysis plans  Randomization

6. AIM 1: Provide methodological guidance to translational researchers.  We also provide assistance with:  Data analyses *  Manuscript preparation * * Priority given to SCTR trainees/scholars and pilot project awardees

7. AIM 2: Multisite Clinical Trials Design Consultative service  Investigators can make requests through SPARC.  Consultations will be with M. Chimowitz and Y. Palesch  We provide assistance with:  study design  statistics and data management  interaction with the government agencies involved in clinical trials (NIH, CMS, and FDA)  industry partners  preparation of the trial budget  implementation and coordination of funded trials

8. AIM 3: Conduct research into novel biostatistical methodologies.  Identifying relevant gene x gene and gene x environment interactions  Improving adoption/dissemination of novel biostatistical methods

9. AIM 4: B iostatistical educational development of trainees and junior investigators.  Lectures within the MSCR and MD/PhD programs  Lectures for clinicians covering basic concepts of biostatistics  Ad hoc lectures upon request  One-on-one training upon request within our biostatistics “clinics”  Advice about on-line resources

10. AIM 5: Review applications for internal SCTR funding  Assist with the reviews of SCTR pilot projects  Does the project have an adequate analysis plan?  Has the number of subjects been thoroughly justified?  Does the research team have the expertise needed to conduct the analyses?  Does the study design seem appropriate to address the research question(s)?

11. SCTR Pilot Project Proposals – General Comments  Biostatisticians are, by nature, analytical. We like correct grammar.  When presenting error bars on graphs, make sure to provide a footnote that says what your error bars reflect (e.g. standard errors, standard deviations, confidence intervals).  Make sure to indicate how this project will help you plan the next project.

12. SCTR Pilot Project Proposals – Common Mistakes  Typical Errors in Analysis Plans:  No analysis plan provided  Analysis plan is not appropriate for the proposed study  Too generic (e.g. “Groups will be compared using ANOVA.”)  The required analyses are very complicated, but the investigators don’t have the training to conduct the analyses.

13. SCTR Pilot Project Proposals – Common Mistakes  Typical Errors in Sample Size Justifications:  No sample sizes are provided.  No justification is provided.  Statements like “We will use data from n=15 subjects, because that is what is typically done with these types of studies.”  A power analysis is provided, but it is extremely vague and not reproducible.  A power analysis is provided, but it doesn’t match the study design.

14. Sample Size Justification  Keep in mind that generally speaking, there is more variability in humans than in mice.  Incorporate prior data into the power analysis if possible.  Consider whether your analyses and sample sizes need to account for multiple comparisons.

15. Key Take-Away Messages  We are here to help investigators get pilot projects and other grants funded.  We are happy to meet with investigators to help ensure their proposals are the best they can be.

16. Contact Information  Paul Nietert, PhD  Director, SCTR Biostatistics, Epidemiology, and Research Design Program  nieterpj@musc.edu  843-876-1204

17. Questions?