Appendix A
NATIONAL INSTITUTE FOR HEALTH AND CLINICAL EXCELLENCE
Single Technology Appraisal
Trabectedin for the treatment of relapsed ovarian cancer
Final scope
Remit/Appraisal objective To appraise the clinical and cost effectiveness of trabectedin within its licensed indication for the treatment of relapsed ovarian cancer.
Background Ovarian cancer is the most common gynaecological cancer, resulting in 3939 deaths in England and Wales in 2005. Ovarian cancer is often asymptomatic in the early stages and over 75% of cases are diagnosed with advanced stage III or stage IV disease, around 3800 cases per year.
Although a significant percentage of women with ovarian cancer respond to initial chemotherapy, most ovarian cancers recur and respond moderately or poorly to subsequent chemotherapy. Between 55% and 75% of women whose tumours respond to first line therapy relapse within 2 years of completing treatment. Ovarian cancer may be categorised according to the duration of response to first-line platinum chemotherapy as follows: platinum- sensitive disease , that is, responds to first-line platinum-based therapy but relapses 12 months or more after completion of initial platinum-based chemotherapy; partially platinum-sensitive, responds to first-line platinum- based therapy but relapses between 6 and 12 months after completion of initial platinum-based chemotherapy; platinum-resistant, relapses within 6 months of completion of initial platinum-based chemotherapy and platinum- refractory, does not respond to initial platinum-based chemotherapy.
NICE Technology Appraisal No. 91 recommends the following as options for second-line (or subsequent) treatment:
• paclitaxel in combination with a platinum compound in platinum-sensitive or partially platinum sensitive disease • pegylated liposomal doxorubicin hydrochloride in partially platinum- resistant disease • single agent paclitaxel or pegylated liposomal doxorubicin hydrochloride in platinum-resistant or platinum refractory disease or for those with an allergy to platinum-based compounds • topotecan only where the other recommended options are considered inappropriate.
Other antineoplastic agents are also used in clinical practice for the treatment of relapsed ovarian cancer.
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The technology Trabectedin (Yondelis; PharmaMar) is an alkylating anticancer agent which acts as a DNA antagonist, affecting transcription factors to block the cell cycle. Trabectedin in combination pegylated liposomal doxorubicin hydrochloride received positive opinion from the European Medicines Agency Committee for Medicinal Products for Human Use for the treatment of patients with relapsed platinum-sensitive ovarian cancer.
Intervention(s) Trabectedin in combination with pegylated liposomal doxorubicin hydrochloride
Population(s) Women with ovarian cancer that has relapsed following first-line platinum-based chemotherapy regimen, and whose cancer is platinum-sensitive.
Standard Women whose cancer has relapsed more than 12 comparators months after completion of initial platinum-based chemotherapy: • platinum based chemotherapy (single agent or combination). Women whose cancer has relapsed between 6 and 12 months after completion of initial platinum-based chemotherapy: • platinum based chemotherapy (single agent or combination) • pegylated liposomal doxorubicin hydrochloride as monotherapy • paclitaxel as monotherapy • topotecan as monotherapy.
Outcomes The outcome measures to be considered include: • overall survival • progression-free survival • response rate • health-related quality of life • adverse effects of treatment.
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Economic analysis The reference case stipulates that the cost effectiveness of treatments should be expressed in terms of incremental cost per quality-adjusted life year. The reference case stipulates that the time horizon for estimating clinical and cost effectiveness should be sufficiently long to reflect any differences in costs or outcomes between the technologies being compared. Costs will be considered from an NHS and Personal Social Services perspective.
Other Guidance will only be issued in accordance with the considerations marketing authorisation.
Related NICE Related Technology Appraisals: recommendations Technology Appraisal No. 91, May 2005. Topotecan, pegylated liposomal doxorubicin hydrochloride and paclitaxel for the treatment of advanced cancer (Review of TA 28, TA 45 and TA 55 [for relapsed disease only]).
Technology Appraisal No. 55, January 2003. Review of the clinical effectiveness and cost effectiveness of paclitaxel for ovarian cancer. Related Guidelines: Clinical Guideline in Preparation. The recognition and initial management of ovarian cancer. Earliest anticipated date of publication April 2011.
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