The most severely ill advanced renal cell cancer patients in England & Wales (numbering around 390 people) have been dealt a blow by the decision of the National Institute for Health and Clinical Excellence (NICE) to reject an appeal by Wyeth against NICEs earlier decision not to recommend Torisel (temsirolimus) for use by the NHS in England & Wales.

As the only treatment evaluated in NICEs recent Health Technology Appraisal of kidney cancer drugs to have demonstrated a significant extension to life in this severely ill group of patients1, the rejection of Torisel will be seen as further limiting the options available to patients fighting advanced kidney cancer.

“The rejection of this appeal and the failure to recommend Torisel the only treatment proven to extend life in these patients is a devastating blow to patients and their families” said Dr Vignesh Rajah, Medical Director, Wyeth. “It further accentuates the disparity in cancer care between the UK and other advanced countries, where Torisel and other innovative renal cell cancer drugs are routinely used. NICEs rejection also conflicts with professional treatment guidelines on renal cell cancer published earlier this year, recommending Torisel as first line treatment for poor prognosis patients2,3.”

Torisel is licensed to treat a very small group of advanced renal cell cancer patients with the poorest prognosis. Given the rarity of this severe form of renal cancer, it is termed an “ultraorphan” condition. NICE has previously acknowledged that appraising drugs for very rare conditions, such as this, “presents special difficulties”.4

Wyeth believes that NICE failed to fully take into account both the very small number of patients eligible for treatment with Torisel and their degree of clinical need. Consequently, Wyeth believes that this has resulted in a decision that discriminates against the most seriously ill renal cell cancer patients.

NICE has already taken an important step forward in recommending an innovative lifeextending treatment for some other groups of renal cancer patients. Nevertheless, the hardest hit patients those with advanced renal cell cancer with the poorest prognosis have been left without access to the only drug proven to extend their lives.

Wyeth appealed against the proposed Final Appraisal Determination with respect to the fact that NICE had failed to act fairly and in accordance with its published procedures and with regard to the fact that it had prepared a Final Appraisal Determination which was perverse in the light of evidence submitted.

Dr Rajah commented “Wyeth is very disappointed by the rejection of this appeal as we believe the Appraisal Committee failed to correctly apply the Institutes own guidance (known as the End of Life Supplementary Advice) for appraising lifeextending, endoflife treatments. This requires due consideration to be given to the impact of giving greater weight to the quality of life of patients in the later stage of terminal diseases in order to fully capture the treatment benefits of life extending drugs, such as Torisel.

He continued “In addition, we consider that the Appraisal Committee failed to adequately use its discretion in applying directions from the Secretary of State for Health that allow particular features of a condition and the patient population it affects to be taken into account when determining the cost effectiveness of a treatment.”

Following the rejection of its appeal, Wyeth is considering its position and the available options to secure patient access to Torisel.

About Torisel

Torisel (temsirolimus) is the first and only renal cell cancer therapy proven to extend overall survival in the treatment of advanced renal cell carcinoma (RCC) in patients with poor prognosis compared with interferonalpha.1 An openlabel, randomised, phase 3 study compared Torisel alone, a combination of Torisel plus interferonalpha, and interferonalpha alone as firstline therapy in 626 patients with advanced RCC who had at least three of six prognostic risk factors.1

In this study, Torisel alone significantly increased median overall survival by 49 % compared with interferonalpha alone (10.9 months for Torisel vs. 7.3 months for interferonalpha, P=0.0078).1Torisel was also associated with statistically significant improvement over interferonalpha in the secondary end point of progressionfree survival (PFS), when the disease does not get worse (3.8 months of PFS vs. 1.9 months of PFS, P