OBJECTIVES

To determine 1) to what extent patient-reported outcomes (PROs) are included in initial FDA oncology drug labeling, and 2) when PRO data become available in US and European oncology drug labeling.

METHODS

Reviewed and synthesized; 1) outcomes for 2018 FDA oncology approvals (NDA and BLA) in initial FDA labels, and 2) outcomes four years post-approval for 2014 FDA oncology approvals in current FDA labels, published literature, and EMA SmPCs.

RESULTS

Most new drugs are approved first in US, often with accelerated approval; oncology has a large share of recent approvals.

FDA approved 59 novel drugs in 2018, 18 (30%) in oncology. Among 2018 oncology approvals, 5 (28%) had breakthrough status, 14 (78%) received priority review, 11 (61%) were orphan drugs, and 8 (44%) were approved with only Phase 1 or Phase 2 data. Most frequently reported clinical outcomes in initial FDA labels were response (ORR, CR, PR; 89%), progression-free survival (44%) and overall survival (17%) rates. No PROs were reported.

FDA approved 41 novel drugs in 2014, 9 (21%) in oncology. Among 2014 oncology approvals, all were orphan drugs and 5 (56%) were biologics. Four years post-approval, none of the FDA labels included PRO data. By the end of 2018, 8 of these 9 oncology drugs were approved in Europe and had PRO label data (primarily HRQoL, with EORTC QLQ-C30 and EQ-5D data reported most frequently). PRO data were published for 7 of the 8 drugs approved in Europe.

CONCLUSIONS

Among recently approved oncology drugs, PRO data were not included in the initial FDA label. Four years post-approval, PRO data were published for most oncology drugs, were included in all European labels, but were still not included in the US label.