Characteristics of Cost-effectiveness Studies for Oncology Drugs Approved in the United States From 2015-2020


Abstract

Importance: Increasingly, cost-effectiveness analyses are being done to determine the value of rapidly increasing oncology drugs; however, this assumes that these analyses are unbiased.

Objective: To analyze the characteristics of cost-effectiveness studies and to determine characteristics associated with whether an oncology drug is found to be cost-effective.

Design, setting, and participants: This retrospective cross-sectional study included 254 cost-effectiveness analyses for 116 oncology drugs that were approved by the US Food and Drug Administration from 2015 to 2020.

Exposures: Each drug was analyzed for the incremental cost-effectiveness ratio per quality-adjusted life year, the funding of the study, the authors'' conflict of interest, the threshold of willingness-to-pay, from what country''s perspective the analysis was done, and whether a National Institute for Health and Care Excellence cost-effectiveness analysis had been done.

Main outcomes and measures: The main outcome was the odds of a study concluding that a drug was cost-effective.

Results: There were 116 drug approvals with 254 studies and country perspectives. Of the country perspectives, 132 (52%) were from the US. Forty-seven of 78 drugs with cost-effective studies had been shown to improve overall survival, whereas 15 of 38 of drugs without a cost-effectiveness study had been shown to improve overall survival. Having a study funded by a pharmaceutical company was associated with higher odds of a study concluding that a drug was cost-effective than studies without funding (odds ratio, 41.36; 95% CI, 11.86-262.23).

Conclusions and relevance: In this cross-sectional study, pharmaceutical funding was associated with greater odds that an oncology drug would be found to be cost-effective. These findings suggest that simply disclosing potential conflict of interest is inadequate. We encourage cost-effectiveness analyses by independent groups.

Conflict of interest statement

Conflict of Interest Disclosures: Dr Prasad reported receiving fees from Arnold Ventures during the conduct of the study and receiving personal fees from Johns Hopkins Press, Medscape, MedPage, Evicore, New Century Health, UnitedHealthcare Consulting, and Patreon outside of the submitted work. No other disclosures were reported.

Figures

Figure.
Figure.. Difference Between the QALY ICER and the Threshold by Study Funding Source in Studies From a US Perspective That Declared Funding
ALL indicates acute lymphoblastic leukemia; CRC, colorectal cancer; DLBCL, diffuse large B-cell lymphoma; HCC, hepatocellular carcinoma; HNSCC, head and neck squamous cell carcinoma; ICER, incremental cost-effectiveness ratio; NSCLC, non–small cell lung cancer; QALY, quality-adjusted life year; RCC, renal cell carcinoma; SCLC, small cell lung cancer.

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