OBJECTIVES: To present the SIDE-L framework (Statistical consideration, Interpretation, Design, External generalizability and Labeling) assessing the quality and suitability of efficacy/effectiveness evidence from “non-traditional” studies (such as real-world evidence) for inclusion in pharmaceutical product labelling.

METHODS: After reviewing the literature, considering, among others, Robins I, the ISPOR Relevance and Credibility Questionnaire for Observational studies, NICE DSU Technical Support Document 17, GRACE checklist, AHRQ and ENCePP checklists, ICH E-documents and bias lists of Sacket, we concluded based on Pharmaceutical labelling practice that a new framework needed to be developed. The framework was built and tested with two large observational studies. Pilots are ongoing for validation and optimization of the SIDE-L framework.

RESULTS: The SIDE component pertains to the quality assessment comprising 14 items in four sections: Statistical considerations, Interpretation, Study Design and External generalizability. The Labelling (L) component contains 7 items pertaining the suitability of a study for inclusion in pharmaceutical product labelling. Each item contains detailed criteria allowing determination whether the item is 1) met, 2) not met, possible to mitigate 3) not met, not possible to mitigate for SIDE and whetherthe criterion is a hurdle implying that it is no longer possible to include the study for labelling for the L part. Initial testing demonstrates that the framework is simple, adaptable and permits a robust and transparent assessment at both the end of a study and at its planning stages.

CONCLUSIONS: The SIDE-L framework is a novel framework developed for the evaluation of study quality and suitability of efficacy/effectiveness results from “non-traditional” studies, which can be used for both labelling and Health Technology Assessment (HTA), especially given the growing interest in Real World Evidence in HTA