Mature versus registration studies of immuno-oncology agents: Does value improve with time?

PURPOSE A unique feature of immuno-oncology agents is the potential for durable survival for a subset of patients; however, this benefit usually cannot not be seen in the early published data used for regulatory approval. Value frameworks developed by ASCO and the European Society for Medical Oncology (ESMO) assess the clinical benefit demonstrated in clinical trials. Proven benefit may change with time as more mature data are available. Our objective was to evaluate the impact of mature data for immuno-oncology agents on ASCO and ESMO scores and to examine the concordance of these frameworks using more mature data. METHODS We reviewed Food and Drug Administration (FDA) approvals for immuno-oncology agents between 2011 and 2017, calculated the ASCO-Net Health Benefit (NHB) score and ESMO-Magnitude of Clinical Benefit Score (MCBS), checked which agents fulfilled the criteria of being rewarded for durable survival, assessed the concordance between models using the Spearman correlation test, and compared the initial results of registration studies with mature follow-up data from the same studies. RESULTS The FDA approved 27 solid tumor indications for immuno-oncology agents between 2011 and 2017. The correlation between ASCO-NHB score and ESMO-MCBS was high (0.88). Mature follow-up data were available for 13 of these indications, in which 6 studies were found to have improved in the grade of ASCO and/or ESMO value frameworks, whereas 2 cases were downgraded in the scale. CONCLUSION Despite different approaches, the high concordance between ASCO and ESMO value frameworks indicates that both models reward treatments as beneficial for the same immuno-oncology agents. Mature data with longer follow-up reaffirmed most of the findings found in the evaluation in the initially published registration studies.