NICE plans to say yes to two breakthrough treatments for skin cancer
NICE, the healthcare guidance body, has issued new final draft guidance recommending two new treatments for advanced malignant melanoma:
Ipilimumab (Yervoy, Bristol-Myers Squibb) is recommended for the treatment of advanced malignant melanoma in people who have received prior chemotherapy.
Vemurafenib (Zelboraf, Roche) is recommended for the treatment of unresectable locally advanced or metastatic BRAF V600 mutation-positive melanoma.
Both treatments are recommended on the basis that the manufacturers provide them with a discount, as agreed in separate patient access schemes for each appraisal.
Commenting on the final draft guidance, Professor Carole Longson, Health Technology Evaluation Centre Director at NICE said: "Advanced melanoma can significantly affect patients' quality of life and without effective new therapies, the prognosis for advanced disease is very poor. For many years the treatments available for this condition have been very limited and in some cases restricted to palliative care. However, there are now a number of new treatments being developed and vemurafenib and ipilimumab are the first two that NICE has been asked to review.
"These new draft recommendations represent really good news for skin cancer patients. Vemurafenib and ipilimumab are breakthrough treatments that can potentially significantly affect prognosis for these patients and we are very pleased that the manufacturers have worked with us so that we are now able to recommend both ipilimumab and vemurafenib."
Since the publication of the first draft guidance for consultation last year which did not recommend the drug, the manufacturer of ipilimumab, Bristol-Myers Squibb, provided the Committee with additional data and analysis regarding the cost effectiveness of the drug. They also submitted a patient access scheme. This enabled the Committee to recommend the drug as a cost effective use of NHS resources.
The manufacturer of vemurafenib, Roche, also submitted additional analyses on the effectiveness of the drug in relation to its clinical and cost effectiveness following the publication of draft guidance on which the Committee requested further clarification from the manufacturer.
The draft guidance documents are now with consultees, who have the opportunity to appeal against them. Until NICE issues final guidance, NHS bodies should make decisions locally on the funding of specific treatments.
Notes to Editors
Vemurafenib is a targeted treatment with a biomarker (BRAF V600 mutation) to help identify the group of people most likely to gain long-term benefit from receiving it. Approximately half of patents are not eligible for this targeted treatment. For ipilimumab, no patient characteristics or biomarkers have been identified.
1. The final draft guidance will be available on the NICE website from 2 November 2012. Embargoed copies of the draft guidance are available from the NICE press office on request.
2. Appraisal history:
1st Committee meeting
ACD issued: vemurafenib not recommended (June 2012)
2nd Committee meeting
Discussion of responses to consultation on the ACD including additional analysis submitted by manufacturer which presented results based on a later data cut-off than had been presented previously. This included data on 34% of patients who switched over from dacarbazine to vemurafenib and other drugs of the same type. To account for the impact of this switching manufacturer used a statistical method of adjusting the survival estimate for the dacarbazine arm of the BRIM3.
The committee concluded that they required further clarification on the analysis conducted and its underlying assumptions to be able to make the decision
ACD2 issued requesting further clarification (August 2012)
3rd Committee meeting
Discussion of responses to consultation on the ACD2, which included the manufacturer's response to the clarification request
FAD issued: vemurafenib recommended (October 2012)
3. Evidence from the BRIM3 study indicates that treatment offers an extension to life of at least an additional 3 months, compared with current NHS treatment.
4. Patients with advanced or metastatic malignant melanoma are usually treated with carboplatin-based chemotherapy or dacarbazine, or given best supportive care.
5. Vemurafenib costs £1750 for one pack of 56 x 240-mg tablets (1 week's supply). Treatment for an average duration of 7 months (30 weeks) would cost £52,500
The manufacturer of vemurafenib has agreed a patient access scheme with the Department of Health, in which a discount on the list price of vemurafenib is offered. The size of the discount is commercial-in-confidence.
6. The Committee concluded that the most plausible ICER was in the range of £44,000 to £51,800 per QALY gained.
7. The Committee heard from the manufacturer and clinical specialists that the total number of people who would be eligible for treatment with vemurafenib was less than 1,000 each year in England and Wales.
8. The Committee was satisfied that vemurafenib met the criteria for being a life-extending end-of-life treatment
9. The final draft guidance will be available on the NICE website from 2 November 2012. Embargoed copies of the draft guidance are available from the NICE press office on request.
10. Appraisal history:
1st Committee meeting
ACD issued: ipilimumab not recommended (October 2011)
2nd Committee meeting
Discussion of responses to consultation on the ACD
FAD not published as the manufacturer submitted a patient access scheme after the meeting.
3rd Committee meeting
Discussion about impact of patient access scheme. However, the FAD was not published as manufacturer submitted revised patient access scheme and requested that FAD be delayed in order to present additional data and analyses for discussion at 4th Committee meeting
4th Committee meeting
Discussion of a new analysis submitted by the manufacturer that included data an additional 72 patients a dose-ranging trial pooled with data from the main clinical study as well as an updated patient access scheme.
FAD issued: ipilimumab recommended (October 2012)
11. Assuming an average body weight of 70 kg, each dose of ipilimumab would cost £18,750. A four-dose course would cost £75,000, not including administration costs.
The manufacturer of ipilimumab has agreed a patient access scheme with the Department of Health, in which a discount on the list price of ipilimumab is offered. The size of the discount is commercial-in-confidence.
12. The Committee concluded that the manufacturer's ICER of £42,200 per QALY gained was a plausible estimate, but recognised that the ICER could be higher using other approaches to overall survival modelling.
13. There are approximately 400 to 500 people with advanced melanoma whose disease has progressed after chemotherapy each year in the UK. The number of eligible patients for each drug is different due to variances in their licences.
14. The Committee was satisfied that ipilimumab met the criteria for being a life-extending end-of-life treatment
15. The National Institute for Health and Clinical 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
16. 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.
17. 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
18. 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 and information to help health professionals deliver the best patient care through NHS Evidence.