In situations of limited treatment options, off-label use (OLU) may be the most promising therapy for cancer patients. However, OLU is regulated by reimbursement restrictions. Little is known about the prevalence of OLU and factors that drive reimbursement decisions. We investigate the reimbursement reality of OLU in patients with solid or hematological malignancies in the Swiss healthcare system, which is characterized by a high diversity of jurisdictions and statutory health insurers.
We conduct an ongoing cross-sectional study using routinely collected health data. All patients with cancer who received drug treatment at three major hospitals in Switzerland between 01/2015 and 07/2018 are screened for OLU. For patients with at least one reimbursement request, we extract demographics, disease and treatment characteristics, and correspondence with the health insurer. We define OLU as intentional drug use outside of the Swissmedic (Swiss Agency for Therapeutic Products) approval label at the time of request. We use descriptive statistics to describe the frequency and characteristics of OLU requests and multivariable logistic regression to assess the association of pre-specified patient characteristics and the reimbursement decisions.
So far, we screened medical records of 1561 eligible patients. For 276/1561 patients (18%), at least one reimbursement request for OLU was issued (319 requests in total, 1 - 3 per patient). The most frequent indications were adjuvant zoledronic acid in breast cancer (27; 8%), adjuvant nivolumab in melanoma (11; 3%) and atezolizumab in advanced urothelial cancer (10; 3%). Health insurers rejected the request in 93/319 cases (29%). Preliminary analyses showed no association of patient characteristics and reimbursement decisions (e.g. odds for disapproval for solid vs hematological malignancies, OR 1.43, 95% CI 0.70 to 2.91, p = 0.32).
Preliminary results indicate that access to cancer care with OLU in Switzerland is characterized by substantial inequity and lack of transparency of the underlying decision-making process. Further results and details on the relationship of reimbursement decisions and the underlying clinical evidence for OLU will be presented at the meeting.
The authors.
Swiss Cancer League.
All authors have declared no conflicts of interest.