4–1: Appendix 4–1 Questions for Assessing Clinical Trials

OVERALL ASSESSMENT

• Was the article published in a reputable, peer-reviewed journal?

• Are the investigator’s training/education/practice sites adequate for the study objective?

• Can the funding source bias the study?

TITLE/ABSTRACT

• Was the title unbiased?

• Did the abstract contain information not found within the study?

• Did the abstract provide a clear over view of the purpose, methods, results, and conclusions of the study?

INTRODUCTION

• Did the authors provide sufficient background information to demonstrate the rationale for the study?

• Were the study objectives clearly identified?

• What were the major null hypothesis and alternate hypothesis?

METHODS

• Was an appropriate study design used to answer the question?

• Were reasonable inclusion/exclusion criteria presented to represent an appropriate patient population?

• Was a selection bias present?

• Was subject recruitment described? If so, how were subjects recruited? Was the method appropriate?

• Was IRB approval obtained?

• Was subject informed consent obtained?

• Were the intervention and control regimens appropriate?

• What type of blinding was used? Was this type appropriate?

• Was randomization included? If so, what type was used? Was this appropriate?

• Who generated the allocation sequence, enrolled participants, and assigned participants to groups? Was this appropriate?

• Which ancillary treatments were permitted? Would they have affected the outcome?

• Was a run-in period included? How does this affect the results?

• Did the investigators measure compliance? How was compliance measured? Was compliance adequate?

• Was the primary endpoint appropriate for the study objective?

• Were secondary endpoints measured? If so, were they adequate for what was being studied?

• Were planned subgroup analyses planned? If so, were they appropriate?

• Was the method used to measure the primary endpoint appropriate?

• What type of data best describes the primary endpoint?

• Were data collected appropriately?

• What number of patients was needed for the primary endpoint to detect a difference between groups (power analysis)? Was the necessary sample size calculated? Were there enough patients enrolled to reach this endpoint?

• What were the alpha (α) and beta (β) values? Were these appropriate?

• Were the statistical tests used appropriate?

RESULTS

• Were the numbers of patients screened, enrolled, administered treatment, completing, and withdrawing from the study reported? Were reasons for subject discontinuations reported? Were withdrawals handled appropriately?

• Was the trial adequately powered?

• Were the subject demographics between groups similar at baseline? If not, were the differ- ences likely to have an affect on the outcome data?

• Were data presented clearly?

• Were the results adjusted to take into account confounding variables?

• Was intention-to-treat analysis used? Was this appropriate?

• Were estimated effect size, p-values, and confidence intervals reported?

• Were the results statistically significant? Clinically different?

• Was the null hypothesis accepted or rejected?

• Can the trial results be extrapolated to the population?

• Based on the results, could a Type I or Type II error have occurred?

• Are subgroup analysis presented? Are these appropriate?

• Was ancillary therapy included? ...