gms | German Medical Science

Background/research question: Off-label drug use (OLU) may be a growing treatment option for cancer patients in situations of limited approved treatment regimens and is common practice in oncology. OLU is partly regulated by reimbursement restrictions, as anti-cancer drugs are often highly expensive and their reimbursement is a challenge for health-care systems. In Switzerland, a reimbursement request needs to be issued by the treating oncologist before OLU treatment can be initiated. For evidence-based health care, reimbursement of OLU would require sufficient evidence for a treatment benefit. However, little is known about factors that drive reimbursement decisions. We aim to investigate the relationship of the reimbursement decision with underlying evidence on treatment benefits.

Methods: In this ongoing project, we use routinely collected health data from patients with cancer who received drug treatment at three major hospitals in Switzerland between 2015 and July 2018. For patients with at least one reimbursement request, we extract demographics, disease and treatment characteristics, and correspondence with the health insurer. In a second step, all reimbursement requests were classified as on label use or OLU, based on the Swissmedic label information at the specific time of the request. For the most frequent OLU indications, we determine the randomized trial evidence for treatment benefits at the date of the request using rapid review techniques and standard approaches for evidence synthesis.

Results: So far, we screened medical records of 2434 eligible patients. For 478 patients (20%), at least one reimbursement request for OLU was issued. Health insurers rejected the request in 30% of cases. Preliminary results based on data from the 280 most frequently requested OLU indications indicate that about 30% of intended OLU treatment is supported by trial evidence for survival benefits and about 15% by trial evidence only indicating better progression-free-survival. Thus far we don’t clearly see that reimbursement is more likely associated with clinical trial evidence for treatment benefits.

Conclusion: Preliminary results do not clearly indicate that reimbursement decisions of OLU are based on randomized clinical evidence for potential OS or PFS benefit. It remains unclear which factors drive reimbursement decisions. More mature results will be presented at the meeting.

Competing interests: All authors have declared no conflicts of interest. The project is supported by a competitive grant from Swiss Cancer League (Krebsliga Schweiz, KFS-4262-08.2017).