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. Author manuscript; available in PMC: 2014 Aug 1.
Published in final edited form as: Epilepsia. 2013 Aug;54(0 4):13–23. doi: 10.1111/epi.12295

Table 1.

Standards for study design and reporting of early preclinical studies

Aspect of study Preferred study design and reporting practices
Experimental design Describe the species, strains, and genetic background, if known, for both experimental groups and controls
Justification of selected animal models or experimental setting
Statement of adherence to ethical and animal care guidelines
Justification for route of treatment delivery/dosing
Report the timing of treatment delivery in regards to time of the day and endpoint assessment
Justification of the timing of treatment based on expected PK-PDs or anticipated time of action of the treatment in the studied strain
Use consistent housing, breeding and handling conditions across groups
Randomized, placebo/vehicle-controlled, blinded design
State criteria for dose selection; dose-response studies may inform on therapeutic window
Age, sex, stage of the disease, length of treatment and observation for outcomes, and other covariants that may affect outcomes (handling, litter effect etc) to be randomly distributed across groups
Apply uniform handling procedures across groups
Inclusion of methods to assess target relevance and target engagement
Select outcomes that are appropriate for the symptom/disease target or target population
Select measurable and quantifiable endpoints that allow rigorous objective comparisons
The purity, stability, manufacturing reproducibility of selected biologicals (viruses, cell lines, etc) should be described
Data collection and analysis Describe the power analyses that set the sample size requirements
Pre-define and report the inclusion and exclusion criteria and criteria for outliers
Pre-define and report rules for sampling data or stopping data collection
Describe causes and extent of lost data
Report interim analyses
Report number of replicates per group and statistics for each experiment; clarify if replicates are biological or technical and from how many experiments or animals they are derived from
Report the reproducibility of results across the experiments in the study and the distribution of values (scattergrams), if possible
Description and justification of the selected statistical methods
Minimizing bias Data collection and analyses to be done blinded to group allocation
Description of blinding methods and randomization of groups; report issues that hinder blinding
Strategies for randomization and/or stratification to be described
Inclusions and exclusions to be applied blindly to group allocation
Report missing data
Report both positive and negative data
Report conflicts of interests
Reporting of results Experimental methods need to be described in sufficient detail to permit replication
Describe housing, breeding and handling conditions: light/dark cycle, number of animals per cage, time of day of the experiment, type and sequence of experimental procedures per rat or group
Information on materials, tests, and assays used should be included to support their validation for their specificity and appropriateness for the studied population and study aims
Describe vehicle composition and any properties that might influence the demonstrated efficacy and tolerability of the AET
Report number of replicates per group and statistics for each experiment
Interpretation Discuss clinical relevance of findings: target population, clinical applicability of treatment protocol (therapeutic window, tolerability of recommended doses, prior positive or adverse clinical experience with similar treatments)
Discuss evidence for target relevance and target engagement
Discuss evidence for reproducibility, robustness or limitations of current study
Discuss effect size in regards to potential clinical impact
Discuss evidence in favor or against replication/validation of results
Discuss alternative interpretations of the findings
Discuss available concurring or discordant studies from the literature