1707P - Cancer drug prices in the US: Efficacy, innovation, clinical trial evidence, and epidemiology

Background

Rising cancer drug prices pose a challenge for patients and healthcare systems in the US. Whilst prices are routinely assigned to a drug’s original indication receiving US Food and Drug Administration (FDA) approval, the influence of supplemental indication approvals on prices remains uncertain. This study identifies and quantifies factors associated with cancer drug prices, distinctly analyzing original and supplemental indications.

Methods

Clinical trial evidence supporting each indication’s FDA approval was collected from the Drugs@FDA database (2003-2022). Disease incidence and disability-adjusted life years (DALYs) were obtained from the Global Burden of Disease study. Indication-specific monthly treatment costs were calculated for patients covered under Medicare Part B and D. The association between log-prices and collected variables was assessed in univariate regression analyses.

Results

We identified 145 drugs approved across 373 cancer indications. Drugs were priced at $24,444 per month on average (median $16,013; IQR 14,648 to 22,348). For original indications, prices were only weakly correlated to improvements in overall survival (β=0.28, 95%CI 0.02 to 0.54, p=0.037) and progression-free survival (β=0.16, 95%CI 0.07 to 0.25, p=0.001). Original indications’ prices were: (1) negatively associated with disease incidence (β=-0.21, 95%CI -0.28 to -0.13, p<0.001) and prevalence; (2) positively correlated for first-in-class drugs (26%, 95%CI -1 to 60, p=0.057), gene & cell therapies (176%, 95%CI 79 to 324, p<0.001), hematologic cancers (52%, 95%CI 28 to 106, p<0.001), and severe diseases with substantial unmet needs (6% per DALY, 95%CI 3 to 8, p<0.001); and (3) negatively correlated to indications with randomized controlled phase III trials. Prices were only poorly associated with supplemental indications’ efficacy, clinical evidence, and epidemiology.

Conclusions

Cancer drug prices are set based on the original indication’s characteristics, thereby omitting the value of supplemental indications. Indication-specific pricing, coverage, and reimbursement policies considering each indication’s safety, efficacy, innovativeness, and unmet needs are necessary to align a drug’s value and price.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

The authors.

Funding

Has not received any funding.

Disclosure

All authors have declared no conflicts of interest.