Targeted Therapies for NSCLC May Be Underused in Medicaid Programs, Study Suggests


A new study estimates that prescription of standard-of-care non–small-cell lung cancer (NSCLC) therapies is lower than expected in Medicaid programs, with significant variation between states.

Targeted therapies for non–small-cell lung cancer (NSCLC) may be underused in many state Medicaid programs, according to a study published in JAMA Network Open.

Drugs targeting specific mutations can significantly improve outcomes for certain patients, including subsets of patients with NSCLC. EGFR variants and ALK rearrangements are the most common targetable variations in NSCLC, and there are currently 8 approved drugs indicated for tumors with these genomic alternations.

Despite the potential benefits of targeted therapy for cancers with certain mutations, prior research suggests that they are underused in patients insured by Medicaid. The high cost of these drugs is a significant barrier to uptake within Medicaid programs, which have fixed overall budgets and are required to cover drugs for almost every FDA-approved indication, the study authors wrote.

Considering prior research has shown that prescription of expensive drugs varies significantly across state Medicaid programs, the new study specifically explored patterns of use of novel oncology therapies among state Medicaid programs, where data are historically lacking. The authors also aimed to describe factors that may be associated with state-level variation in targeted therapy use for Medicaid patients.

In 2020 and 2021, a total of 2281 person-years of therapies targeting EGFR and ALK were dispensed in state Medicaid programs overall. However, there were 3461 expected person-years of first-line targeted therapy during the same time frame based on real-world treatment durations of osimertinib and alectinib, which are standard-of-care first line treatments targeting EGFR and ALK, respectively.

This suggests that Medicaid beneficiaries with EGFR or ALK alterations received targeted therapy an estimated 66% of the person-time that was expected. Osimertinib and alectinib accounted for 83% of targeted therapy prescriptions.

States with at least 20 estimated person-years of EGFR-altered NSCLC in the study time frame were included in the analysis, and a total of 33 states met this threshold. There were 3258 expected person-years of targeted therapy use in these states overall, but only 1991 person-years of osimertinib and alectinib prescription.

In these states, Medicaid beneficiaries received osimertinib or alectinib an estimated 61% of the expected person-time. The proportions varied substantially by state, with the lowest being 18% in Arkansas and the highest 113% in Massachusetts. A total of 3 states showed dispensing rates similar to those that were expected. These variations were associated with factors including Medicaid policies, state gross domestic product per capita, and the per-capita number of oncologists in each state.

Overall, the findings suggest targeted therapies are underused in Medicaid beneficiaries and that there is significant variation in targeted therapy use in this patient population across states.

“Just 66% of person-years in whom targeted therapies were indicated in 2020 and 2021 were associated with use of those medications, suggesting that at least 500 Medicaid patients with a diagnosis of EGFR- or ALK-altered metastatic NSCLC during these years did not receive targeted therapy when indicated,” the authors wrote. “Given the efficacy of targeted therapies, this underuse could have led to an estimated 855 preventable years of life lost during the period of analysis.”

Several factors could affect these rates, according to the study authors. Ensuring that patients and clinicians have access to genomic testing to identify alterations and that prior authorization policies do not deter oncologists from using targeted therapies are both important aspects of more widespread access. Especially in states where underuse is confirmed, the onus should be on policy makers to examine these programs and ensure patient access to these standard of care therapies, the authors concluded.


Roberts TJ, Kesselheim AS, Avorn J. Variation in use of lung cancer targeted therapies across state Medicaid programs, 2020-2021. JAMA Netw Open. Published online January 25, 2023. doi:10.1001/jamanetworkopen.2022.52562

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