NICE unable to recommend ovarian cancer drug in final guidance due to lack of appropriate evidence

In final guidance published by NICE today (27 April 2011), trabectedin (Yondelis, PharmaMar) in combination with pegylated liposomal doxorubicin hydrochloride (PLDH) is not recommended as a treatment for ovarian cancer.

The independent Appraisal Committee was not able to recommend this combination treatment in patients with platinum-sensitive ovarian cancer that has returned six months or more after initial treatment with chemotherapy which included platinum, because of concerns over how well it works compared with the most commonly-used treatments.

The Appraisal Committee considered all the available evidence, including clinical trial data submitted by the manufacturer and expert opinion from clinical specialists, as well as a Patient Access Scheme to make the treatment more cost effective for the NHS. No appeals were received against the final draft guidance published in March 2011.

Sir Andrew Dillon, NICE Chief Executive, said: "Unfortunately, NICE is not able to recommend trabectedin as a treatment option for relapsed platinum-sensitive ovarian cancer. Expert clinical opinion states that platinum-based chemotherapy is the preferred option for this type of ovarian cancer that has relapsed. However, because the manufacturer of trabectedin did not submit any evidence comparing the treatment with this type of chemotherapy, comparing it instead with drug regimens that did not include platinum, we cannot be sure that trabectedin extends patients' lives for longer than the most routinely used treatments.

"Clinical trial data suggested that the drug might be more effective in women with ‘partially platinum-sensitive' ovarian cancer, which is when the disease recurs between 6 and 12 months after initial platinum chemotherapy. However, the Committee could not be sure that the effectiveness of trabectedin in this group was genuinely different from that seen in the wider group of patients for which the drug is licensed and concluded that the subgroup data was not robust.

"The Committee felt that the Patient Access Scheme offered by the manufacturer did not improve the cost-effectiveness of trabectedin enough to justify its use for this indication, given the uncertain benefits it may provide patients."

The final guidance is available on the NICE website from Wednesday 27 April at:

Once NICE issues its final guidance on a technology, it replaces local recommendations across the country.


Download PDF version

Notes to Editors

  • The guidance is available on the NICE website from Wednesday 27 April at:
  • Trabectedin is an intravenous drug that works by damaging the DNA in cancer cells, making the cells unable to grow and spread. It is administered via a drip every three weeks with the recommended dose being 1.1mg per m² of body surface area.
  • The manufacturer estimated that the cost of treatment with trabectedin plus PLDH, assuming an average of six cycles and a body surface area of 1.72 m², is £23,917, excluding administration costs.
  • Taking into account the impact of all the uncertainties in the evidence presented to them, the Appraisal Committee considered that, for the entire platinum-sensitive population the incremental cost-effectiveness ratio (ICER) could be higher than £95,000 per QALY gained.
  • For the partially platinum-sensitive population (where the disease relapses between 6 and 12 months after initial platinum treatment), taking into account the Patient Access Scheme (whereby the manufacturer would pay the cost of trabectedin after the fifth cycle of treatment [that is, cycle 6 and beyond] for partially platinum-sensitive patients), the committee considered that the best estimate of the ICER was £68,000 per QALY gained.
  • Ovarian cancer is the fifth most common cancer in women in the UK1 with nearly 6,600 women diagnosed with the disease each year2. It is thought the cancer recurs after initial treatment in around 80% of cases and just over two fifths of these patients would be eligible for trabectedin, if the drug were to be approved3.
  • A range of treatments are already available on the NHS for patients with relapsed ovarian cancer. Published in 2005, 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.
  • In 2010, NICE recommended trabectedin as a treatment for some patients with an advanced form of a rare type of cancer called soft tissue sarcoma. This guidance is available to view at:
  • NICE is also publishing a new clinical guideline on the recognition and initial management of ovarian cancer. This will be available on the NICE website from Wednesday 27 April at:



2. In 2008, the year for which the latest incidence figures are available, 6,597 women were diagnosed with ovarian cancer (sources: Office for National Statistics, 2011 Cancer Statistics registrations: registrations of cancer diagnosed in 2008, England. Welsh Cancer Intelligence and Surveillance Unit, Cancer Incidence in Wales. ISD Online Cancer Incidence, Mortality and Survival data. Accessed 2011. Northern Ireland Cancer Registry, 2011 Cancer Incidence and Mortality.

3. According to the manufacturer's submission, the size of the platinum-sensitive population for ovarian cancer is approximately 2,765 people

About NICE

1. The National Institute for Health and Care Excellence (NICE) is the independent organisation responsible for providing national guidance and standards on the promotion of good health and the prevention and treatment of ill health

2. NICE produces guidance in three areas of health:

  • public health - guidance on the promotion of good health and the prevention of ill health for those working in the NHS, local authorities and the wider public and voluntary sector
  • health technologies - guidance on the use of new and existing medicines, treatments, medical technologies (including devices and diagnostics) and procedures within the NHS
  • clinical practice - guidance on the appropriate treatment and care of people with specific diseases and conditions within the NHS.

3. NICE produces standards for patient care:

  • quality standards - these reflect the very best in high quality patient care, to help healthcare practitioners and commissioners of care deliver excellent services
  • Quality and Outcomes Framework - NICE develops the clinical and health improvement indicators in the QOF, the Department of Health scheme which rewards GPs for how well they care for patients

4. NICE provides advice and support on putting NICE guidance and standards into practice through its implementation programme, and it collates and accredits high quality health guidance, research andinformation to help health professionals deliver the best patient care through NHS Evidence.

This page was last updated: 21 April 2011

Accessibility | Cymraeg | Freedom of information | Vision Impaired | Contact Us | Glossary | Data protection | Copyright | Disclaimer | Terms and conditions

Copyright 2014 National Institute for Health and Care Excellence. All rights reserved.

Selected, reliable information for health and social care in one place

Accessibility | Cymraeg | Freedom of information | Vision Impaired | Contact Us | Glossary | Data protection | Copyright | Disclaimer | Terms and conditions

Copyright 2014 National Institute for Health and Care Excellence. All rights reserved.