New Chemotherapy Treatments in Advanced Cancer Patients An Easily Applicable Evaluation of Clinical Efficacy and Cost–effectiveness

Abstract

Novel cytostatic drugs have recently been introduced to treat advanced cancer patients. Although of only modest efficacy, their use is widespread, considerably increasing treatment costs. An easily applicable method to assess their efficacy and cost-effectiveness is needed. We have documented new cytostatic drugs whose consumption increased by over 60% from January 1998 to December 2000 in patients with advanced or metastatic cancer of the colorectum, lung (non-small cell), breast, ovary or brain. A review of the literature yielded 17 treatments that included these agents. For each regimen, we recorded six efficacy variables [median survival time (MS), survival rate at 1 year, absolute risk reduction, time to progression, quality of life (QoL), and patients needed to treat (NNT)]. A four-point (A–D) efficacy (E) scale and a five-point (1–5) strength of evidence (SE) scale were applied. We obtained the cost differential of each regimen for a 4-week treatment, cost per extra month of MS, and cost per NNT. One combination was rated with A efficacy (MS>9 months+improved QoL) and nine with D (no MS or QoL improvement); 12 studies presented good quality (grade 1–2) evidence. The QoL of patients was significantly improved in only two regimens. The average cost differential was 1 311 € (all new regimens except one showed higher cost); the average cost per extra month of MS was 6 415 €; and treatment cost per NNT was 87 767 €. Our method proved to be easy to apply, enabled comparisons with other treatments to be made and revealed that these very costly changes in clinical practice are not justified by available studies.