OBJECTIVES: Understanding evidence associated with oncology drug approval is critical to creating an integrated clinical development and access plan. Accordingly, we sought to investigate how approval of oncology drugs by the FDA might be associated with data quality (trial size, phase, end-points), the level of unmet need (5-year survival rate) in an indication, and the entry sequence of drugs in that indication (first or later).

METHODS: Submission data were collected for oncology drugs with FDA approval between January 2014 and June 2018. Sampling criteria included: indication; launch period (2014-18); multiple drugs available in the indication. A total of 31 drug/indication combinations (12 indications and 25 molecules) were included. FDA designation, approval date and pivotal trial data were acquired from the Drugs@FDA index.

RESULTS: For 29 of 31 drug/indication combinations investigated, those launched second or later were approved with trial data equal to or more mature (superior endpoint or later trial phase) than first drugs to market. Early trial data (Phase I or II) and immature endpoints (objective response rate) were more likely to be submitted for launches in indications with a higher unmet need. Indications with a 5-year survival rate < 20% were more likely to submit immature data (9/15, 60%) than indications with a 5-year survival rate >20% (6/13, 46%), (one drug was excluded from analysis). Phase I data were only submitted for first-to-market drugs (N=2) with 5-year survival rates of <20%.

CONCLUSIONS: In this sample, 9/15 oncology products launched in indications with low (<20%) 5-year survival were approved with immature data for FDA approval compared to 6/13 in indications with 5-year survival rates >20%. This has implications for strategies by pharmaceutical companies in terms of evidence generation and launch sequencing in the US market and the resulting access to oncology drugs for high unmet needs.