Randomized controlled trials (RCT) provide data on clinically measurable outcomes (safety and efficacy) in the short-term. The need for longer-term data requires extended follow-up. The biologic era particularly has witnessed an increasing number of reports on post-RCT extension clinical data; their reporting however is fraught with challenges with no guidelines on optimal reporting. At this meeting a scientific abstract is presented on the recommendations recently formulated by a EULAR task force (Buch M et al). My presentation focuses on the above design and reporting challenges.
Advantages of long-term extension studies (LTE) include the well-described study groups and information on the retention of therapy beyond the trial. Under the right circumstances, more evidence on efficacy and safety can accrue. And finally, LTE can enable ongoing access to an effective therapy that would otherwise be unavailable to subjects that participated in the prior RCT. Disadvantages include the increased potential for bias mainly through selection of patients to include, method of analysis (e.g. the choice of the denominator to calculate rates), and lack of transparency in reporting. There are also ethical issues such as the assumption that the experimental agent is effective and treatment should be continued where proof is not yet fully available.
The key recommendations of the Task Force are:
– All patients from the original trial should be accounted for;
– Requirement of a minimum set of data, including time of last observation; reason for: TES exclusion if drug discontinued, cessation of follow-up and cessation for other reasons; progress at each stage from RCT start to TES completion; duration of active treatment;
– Requirement of a flow chart (see figure example for a placebo/background MTX trial);
– Report consistent with existing guidelines (CONSORT, STROBE), ACR/EULAR recommendations;
– Minimum set of outcomes, including sustainability and new achievement of high hurdle outcomes (remission/LDA); drop-outs detailed;
– Efficacy & safety of a TES reported together.
We hope that adoptions of these recommendations will lead to better and more informative LTE reports.
Disclosure of Interest None Declared
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