Best supportive care in clinical trials: review of the inconsistency in control arm design

R D Nipp, D C Currow, N I Cherny, F Strasser, A P Abernethy, S Y Zafar, R D Nipp, D C Currow, N I Cherny, F Strasser, A P Abernethy, S Y Zafar

Abstract

Background: Best supportive care (BSC) as a control arm in clinical trials is poorly defined. We conducted a review to evaluate clinical trials' concordance with published, consensus-based framework for BSC delivery in trials.

Methods: A consensus-based Delphi panel previously identified four key domains of BSC delivery in trials: multidisciplinary care; supportive care documentation; symptom assessment; and symptom management. We reviewed trials including BSC control arms from 2002 to 2014 to assess concordance to BSC standards and to selected items from the CONSORT 2010 guidelines.

Results: Of 408 articles retrieved, we retained 18 after applying exclusion criteria. Overall, trials conformed to the CONSORT guidelines better than the BSC standards (28% vs 16%). One-third of articles offered a detailed description of BSC, 61% reported regular symptom assessment, and 44% reported using validated symptom assessment measures. One-third reported symptom assessment at identical intervals in both arms. None documented evidence-based symptom management. No studies reported educating patients about symptom management or goals of therapy. No studies reported offering access to palliative care specialists.

Conclusions: Reporting of BSC in trials is incomplete, resulting in uncertain internal and external validity. Such studies risk systematically over-estimating the net clinical effect of the comparator arms.

Figures

Figure 1
Figure 1
Poorly designed BSC can produce trial results that are internally and externally invalid.
Figure 2
Figure 2
Literature review exclusion tree.

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