Table 2.

Major factors to consider in determining the clinical importance of group differences (adapted from Dworkin et al. [23])

Statistical significance of the primary efficacy analysis (typically necessary but not sufficient to determine that the group difference is clinically meaningful)

Availability of alternative therapies and their benefit-risk profiles

Treatment effect size for the primary outcome variable compared to that of available treatments

Safety and tolerability

Rapidity of onset of treatment effect

Durability of treatment effect

Results for secondary efficacy endpoints (e.g., improvements in physical or emotional functioning)

Limitations of available treatments

Different mechanism of action vs. existing treatments

Cost, convenience, and patient adherence

Other benefits (e.g., few or no drug interactions, availability of a test that predicts a good therapeutic response)