The clinical and cost effectiveness of topotecan for ovarian cancer


The aim of this review was to examine the clinical effectiveness and cost-effectiveness of oral and intravenous topotecan (Hycamtin) for the treatment of all stages of ovarian cancer.


This review indicates that there is little evidence in the form of RCTs on which to base an assessment of the effectiveness of topotecan as second-line therapy for advanced ovarian cancer. The evidence found suggests there were no statistically significant differences overall between topotecan and paclitaxel, or topotecan and PLDH in clinical outcomes. However, statistically significant differences were observed in the incidence of adverse effects. The clinical significance of the findings is not discussed. Overall, the effects of topotecan could at best be described as modest, but the alternative agents offer no real advantages except fewer side-effects and possibly improved cost-effectiveness. Both of the clinical effectiveness studies on which this evidence is based had methodological flaws, the most serious being the lack of a blinded assessor in the topotecan versus PLDH trial, which is important for unbiased assessment of response outcomes. The economic evaluations also suffered from a number of potential problems.

Conducted by: C Forbes1, L Shirran1, A-M Bagnall1, S Duffy1, G ter Riet1

1. NHS Centre for Reviews and Dissemination

Further details

Project page on HTA Programme website

Related guidance

Commissioned to inform NICE Technology Appraisal 28: Ovarian cancer - topotecan. London: National Institute for Clinical Excellence; 2001. NB: This guidance is replaced by TA91 Ovarian cancer (advanced) - paclitaxel, pegylated liposomal doxorubicin hydrochloride and topotecan (review)


Forbes CA, Shirran L, Bagnall AM, Duffy S, ter Riet G. A rapid and systematic review of the clinical effectiveness and cost-effectiveness of topotecan for ovarian cancer. Health Technol Assess. 2001;5(28):1-109


Commissioned by the HTA Programme on behalf of NICE's Technology Assessment Report (TAR) process