OBJECTIVES:

In France, hospital drugs are reimbursed only for indications of the marketing authorization approved by the French National Authority for Health (HAS). Drugs are financed by the Diagnosis-Related-Groups (DRG)-based payment system or in addition to the DRGs for innovative and costly medicines. Some off-label indications are also reimbursed, particularly if they are scientifically justified. Use of Anti-PD(L)1 drugs is closely monitored by Authorities because of the high cost of treatments (up to €90K annually per patient) and the growing number of indications. The objectives of this study are to confirm the prescriptions’ compliance with reimbursed indications in Paris University Hospitals (AP-HP, the largest hospital center in Europe, 7 million patients annually), and to evaluate the impact on spending.

METHODS:

Anti-PD(L)1 drugs consumption and spending data were extracted from AP-HP hospitals discharge database and crossed with indications filled in by prescribers, over the January-December 2021 period. Transparency committee summaries from HAS website and Official Journal of the French Republic publications, were used to check which indications are approved.

RESULTS:

In 2021, five Anti-PD(L)1 drugs were used at AP-HP for a total cost of €215M. Pembrolizumab and nivolumab were the most prescribed drugs (44% and 41% respectively). Approximately 92% (€198M) of spending were related to reimbursed indications, or 4,125/4,651 patients, 7% (€16M) to justified off-label indications, or 507/4,651 patients, and 0.4% (€912K) to non-reimbursed indications, or 19/4,651 patients. Moreover, prescriptions of avelumab in merkel cell carcinoma (3/19 patients), pembrolizumab in head and neck squamous carcinoma (2/19) and non-small cell lung cancer (2/19), and nivolumab in melanoma (2/19) were the most frequent non-compliant uses.

CONCLUSIONS:

Anti-PD(L)1 drugs seem to be used appropriately by prescribers, considering that more than 90% of prescriptions conform to indications both authorized and reimbursed.