OBJECTIVES

: To understand the impact of overall survival (OS) maturity on FDA vs EMA regulatory approval timeline and decision-making.

METHODS

: The research reviewed publicly available FDA label statements and EMA decision documents for a set of oncology drugs approved in 85 indications by both the FDA and the EMA over a 5-year period (2013-2017).

RESULTS

: Compared to the inconsistent mention of OS in the FDA labels, all EMA decision documents mentioned OS in some form. More than half (55%) of all EMA-approved indications provided median OS data at launch while less than a third (30%) of all FDA-approved indications provided median OS data at launch. Further, 50 indications presented a different OS dataset to the EMA in comparison to the FDA, and 16 of the 50 indications presented statistically significant median OS data at EMA launch. Availability of statistically significant median OS data in this case was associated with a longer timeframe between FDA and EMA approvals (212 days) compared to the indications with other OS types (i.e., immature, non-estimable, not reached, or not statistically significant OS data). The evidence supports the hypothesis that, if median OS evidence availability is expected in a reasonable timeframe, a pharmaceutical manufacturer may postpone EMA regulatory submission to ensure local European HTA requirements are satisfied, as these HTA requirements are more demanding relative to most USA managed care organizations’ assessment criteria.

CONCLUSIONS

: The analysis emphasizes the importance of OS data for regulatory approval of oncology indications in Europe compared to the USA, where secondary endpoints (such as progression-free survival and response rate) are more frequently used as a basis for regulatory approval and market access. The analysis suggests that manufacturers recognize the EU HTA’s preference for OS data and may wait to submit to the EMA until some form of OS data is obtained.