Association between FDA label restriction and immunotherapy and chemotherapy use in bladder cancer

September 25, 2019

Our summary

In 2017, the FDA granted accelerated approval to two PD-1/PD-L1 immunotherapies for the first-line treatment of cisplatin-ineligible bladder cancer patients, regardless of PD-L1 status, based on Phase II studies. However, data from ongoing Phase III trials subsequently demonstrated that patients with PD-L1 negative tumors who received first-line immunotherapy had decreased survival relative to non-cisplatin chemotherapy. As a result, in 2018, the FDA restricted the indication for these novel drugs to cisplatin-ineligible patients with PD-L1 positive tumors.

In collaboration with researchers from the FDA and the University of Pennsylvania, Flatiron tested the impact of an FDA alert and label change on clinical practice using obtained from electronic health records. Findings suggest that the FDA label changes were associated with changes in practice, even when the decision was based on emerging trial data.

Why this matters

Often, registrational trials support initial regulatory approvals but do not represent the last word in a given body of clinical evidence. Emerging data may warrant the revision of the terms of an approval. The realization of those changes in clinical care requires a whole ecosystem (including payers, diagnostic companies and clinicians) to process those shifts, and RWD can help us understand that dynamic in practice. In this case, once the FDA narrowed the indication for immunotherapy in bladder cancer, the system responded swiftly, resulting in a quick reversion to non-immunotherapy treatments for patients affected by the label revision.

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