For Whom the Price Escalates: High Price and Uncertain Value of Cancer Drugs

The price of cancer drugs has skyrocketed, yet it is not clear whether their value is commensurate with their price. More cancer drugs are approved under expedited review, which considers less rigorous clinical evidence, yet only 20% of them show an overall survival gain in the confirmatory trial. Moreover, clinical data are often generated based on small, single-arm studies with surrogate outcomes, challenging economic evaluation. With their high price and uncertain (marginal) clinical value, cancer drugs are frequently rejected by health technology assessment (HTA) bodies. Therefore, agencies, including the UK's National Institute for Health and Care Excellence (NICE), have adopted cancer drug funds (CDF) or risk-sharing schemes to provide extra access for expensive cancer drugs which fail to meet NICE's cost effectiveness threshold. With rising pricing and fewer new cancer medications with novel mechanisms of action, it is unclear if newly marketed cancer therapies address unmet clinical needs or whether we are paying too much. Transparency, equity, innovativeness, and sustainability are all harmed by a special approach for cancer medications. If early access is allowed, confirmatory trials within a certain time frame and economic evaluation should be conducted, and label changes or disinvestment should be carried out based on those evaluations.

Automated summary

The skyrocketing prices of cancer drugs raise concerns about their commensurate value. Despite expedited reviews leading to approvals, only 20% of cancer drugs show overall survival gains in confirmatory trials. Small-scale studies with surrogate outcomes challenge economic evaluation. Health technology assessment bodies frequently reject expensive cancer drugs due to their high price and uncertain clinical value. To address this issue, cancer drug funds or risk-sharing schemes have been adopted. However, the lack of novel mechanisms of action and rising prices raise questions about whether newly marketed cancer therapies meet clinical needs and whether they are overpriced. A special approach for cancer medications harms transparency, equity, innovativeness, and sustainability. If early access is allowed, confirmatory trials and economic evaluations should be conducted within a certain time frame, and label changes or disinvestment should be based on these evaluations.