NICE consults on preliminary recommendations for a new kidney cancer drug

NICE consults on preliminary recommendations for a new kidney cancer drug

NICE, the healthcare guidance body, has issued draft guidance not recommending axitinib (Inlyta, Pfizer) for the treatment of advanced kidney cancer.

The preliminary decision by the independent Appraisal Committee is that axitinib should not be recommended for the treatment of advanced renal cell carcinoma after failure of prior treatment with sunitinib or a cytokine.

NICE was asked by the Department of Health to look at the use of axitinib for the two populations specified in the drug's marketing authorisation - those previously treated with sunitinib and those previously treated with a cytokine therapy. However, clinical experts informed the independent Appraisal Committee that the use of cytokines is decreasing in clinical practice, with only a few people currently receiving them, the majority of patients begin treatment with NICE-recommended sunitinib or pazopanib.

Commenting on the draft guidance, Sir Andrew Dillon, NICE Chief Executive, said: "Before we recommend any new treatment we have to be sure the evidence on how well it works is robust and that it is cost effective. We do not want to divert NHS funds to a treatment that costs more but doesn't help people live longer.

"When evaluating a new drug, the Appraisal Committee looks at how effective it is when compared to a treatment previously identified as appropriate in the scope of the appraisal. The trial data provided by the manufacturer of axitinib included a direct comparison of the drug to sofafenib, a drug not recommended by NICE and not identified in the scope. The trial also lacked a comparison to 'best supportive care', which is what the majority of patients receive at the moment; therefore an 'indirect' and 'simulated' comparison was made using separate data from another trial. When the Committee considered this comparison, they noted that limited analysis was undertaken to identify uncertainties within this simulated method of comparison and therefore they were concerned about its validity and reliability.

"Consultees, including the manufacturer, healthcare professionals and members of the public are now able to comment on the preliminary recommendations which are available for public consultation. Pfizer can also provide further comment on the evidence it provided and have the opportunity to submit a further patient access scheme to the Department of Health if they wish. Comments received during this consultation will be fully considered by the Committee and following this meeting the next draft guidance will be issued."

The manufacturer of axitinib has agreed a patient access scheme with the Department of Health. The size of the discount is commercial in confidence.

This draft guidance does not mean that people currently taking axitinib will stop receiving it. Until final guidance is issued to the NHS, NHS bodies should make decisions locally on the funding of specific treatments. Once NICE issues its final guidance on a technology, it replaces local recommendations.

Notes to Editors
About the guidance

1. The draft guidance will be available from 7 December 2012. Embargoed copies of the draft guidance are available from the NICE press office on request.

2. The simulated treatment comparison resulted in an estimated median progression-free survival of 4.6 months with axitinib in the prior treatment with sunitinib group compared with best supportive care and 8.3 months for median overall survival in favour of axitinib.

The Committee considered that the possible over-estimation of the overall survival in the TARGET trial was carried over into the overall survival results in the indirect comparison and ultimately affected the model results for the best supportive care group

3. The Committee noted that the cost per QALY for the prior-cytokine group had been over-estimated based on the unlikely overall survival gains with best supportive care in the prior-cytokine population, but that there were other uncertainties that might push the ICER higher so the plausible figure was likely to still be above £50,000. For the group that had received and failed on sunitinib the Committee considered that the cost per QALY was approximately £62,000, but this figure is uncertain.

4. Axitinib is available in 1-mg and 5-mg film-coated tablets at net prices of £703.40 and £3517 per 56-tablet pack respectively (excluding VAT). The recommended starting dose is 5 mg twice daily. This may be increased to 7 mg and then up to 10 mg, or decreased to 3 mg and then down to 2 mg, depending on individual safety and tolerability.

The manufacturer of axitinib has agreed a patient access scheme with the Department of Health, in which a discount on the list price of axitinib is offered. The size of the discount is commercial-in-confidence at the manufacturer's request.

5. The Committee concluded that the prior-cytokine group was not a large population for treatment with axitinib because of the small patient numbers and because other tyrosine kinase inhibitor treatments are available for use in this population.

6. The Committee was satisfied that axitinib met the criteria for being a life-extending end-of-life treatment. agreed that there was sufficient evidence to indicate that the treatment offers an extension to life of at least an additional 3 months in the case of the prior-sunitinib population, compared with current NHS treatment which is best supportive care. However in the case of the prior-cytokine group the Committee considered that these were people who could receive sunitinib or pazopanib but no comparison had been available.

7. The manufacturer estimated the eligible population for axitinib (the majority being in the prior-sunitinib group) is 1,580 people.

8. There are currently no second-line drugs approved by NICE, although it was noted that the Cancer Drugs Fund funds requests for everolimus. Information on the Cancer Drugs fund is available from the Department of Health.

9. The SMC has not yet published guidance on axitinib for this condition.
About NICE

10. 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

11. 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.

12. 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
    Commissioning Outcomes Framework - NICE develops the potential indicators for the COF, the scheme starting in 2013, which will help measure the health outcomes and quality of care commissioned by Clinical Commissioning Groups.

13. 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.