Potential Cost Implications for All US Food and Drug Administration Oncology Drug Approvals in 2018

This economic evaluation study estimates the number of patients with cancer who are eligible for the newly approved drug-indication pairs and projects potential spending and use of the approvals in the United States. Importance The growth of cancer drug spending in the US has outpaced spending in nearly all other sectors, and an increasing proportion of the drug development pipeline is devoted to oncology. In 2018, there was a record number of drugs entering the US market. Objective To estimate the number of patients with cancer who are eligible for the newly approved drug-indication pairs, and project potential spending and use of the approvals in the US. Design, Setting, Participants This is a retrospective review of 2018 US Food and Drug Administration (FDA) oncology drug approvals with estimation of the eligible population. The cost of new therapy was estimated, and savings from displaced therapies were subtracted. Two-way sensitivity analysis explored uncertainty in pricing and market diffusion. Data were collected between March 1, 2019, and September 30, 2019. Exposures Data related to the cancer drug approval (ie, indications, approval pathway, basis for approval), cancer incidence, and drug price were extracted from publicly available sources, including the FDA, National Cancer Institute, and American Cancer Society websites, as well as the RED BOOK database. Main Outcomes and Measures The primary outcome was the projected net expenditure in the US associated with the new therapies. The secondary outcome described how variable market diffusion and pricing permit expected levels of spending. Results A total of 46 oncology approvals were included in the analysis, with 17 novel drugs and 29 new indications. The average price per patient per treatment course was $150384. From a national perspective and with 100% market diffusion, the projected net expenditure for newly approved drugs was $39.5 billion per year. To maintain the recent trend of cancer drug spending, the 2018 cancer drug approvals need to be used in fewer than 20% of eligible patients. Conclusions and Relevance New cancer drugs approved by the FDA in 2018 would drastically increase cancer drug spending in the US if used widely. Alternatively, only low-level use of the new drugs is consistent with market forecasting. Question For cancer drugs approved by the US Food and Drug Administration in 2018, what is the potential influence on cancer drug spending, and how many patients may receive the new approvals? Finding This economic evaluation study determined that if used in all eligible patients, the 2018 oncology drug approvals would add $39.5 billion to US cancer drug spending (>75% increase from total 2017 cancer drug spending). Using the approved drugs in fewer than 20% of eligible patients would be consistent with industry forecasting for drug spending. Meaning The US would face an unprecedented increase in cancer drug spending if 2018 oncology drug approvals are used widely, while only limited uptake of the drugs maintains the current spending trajectories.