(181) Do More Rigorously Tested Drugs Garner Higher Prices Upon Market Launch?

Publication Number: 855

Background: The US Food and Drug Administration (FDA) has, on occasion, approved new molecular entities based on results of a single-arm, Phase I open-label clinical trial while other new oncology drugs have demonstrated gain in overall survival in blinded, randomized trials with active comparators.

Objectives: To measure whether new oncology drugs with more extensive testing, and thus more certainty about clinical benefit, command higher prices upon product launch.

Methods: We used publicly available FDA review documents to assess clinical testing of 57 new oncology drugs approved from 2015 to 2019. We obtained the US 30-day treatment cost at launch from a commercial global drug pricing database. Wholesale acquisition costs were adjusted for cycles, loading and maintenance doses and estimated for one month of treatment. Extent of testing at approval was assessed with 2 primary endpoints: (A) Were benefits established in at least 1 randomized clinical trial (RCT) with a placebo or active drug comparator-or was the drug approved on the basis of an uncontrolled, open-label pivotal trial? (B) Did the pivotal trial primary endpoint demonstrate clinical benefit through increased overall survival or rely on surrogate endpoints such as objective response rate or progression free survival? To compare groups we log-transformed 30-day prices to approximate a normal distribution, and tested differences with 1-way analysis of variance.

Results: Wholesale acquisition costs at launch for 30 days of treatment with new oncology drugs varied more than 10-fold with median (range) of U.S. $12 935 ($5 124 - $83 760). The FDA approved 33/57 new oncology drugs (57.9%) on the basis of RCTs, but accepted the remaining 24 without blinding or a comparator. Only 6/57 new drugs (10.5%) were approved with RCTs that established a survival benefit. There was no statistically significant difference in the launch price between new drugs with and without RCTs (p > 0.384), or for those demonstrating clinical benefit rather than using surrogate endpoints (p > 0.236). However, the trends were unfavorable for the drugs with more scientific certainty about benefits, with a geometric mean 30-day cost of $14 263 vs $16 329 for drugs with and without RCTs, and $11 589 vs $15 577 for new drugs with and without established survival benefits. Among the 10 highest priced new drugs, 5 were tested in RCTs; none documented a clinical benefit.

Conclusions: In this cohort of more than four dozen oncology drugs recently approved for marketing in the US we found no evidence that drugs with greater certainty about treatment benefits commanded higher prices upon product launch. The 30-day wholesale acquisition costs did not provide a reliable indication of either extent of testing or degree of benefit of newly approved oncology drugs.