Statement In Relation To The NICE Appraisal Consultation Document That Does Not Recommend Torisel (R) (temsirolimus) For Advanced Renal Cell Carcinoma

February 9, 2009 at 11:00 am Leave a comment

NICE’s decision not to recommend Torisel (temsirolimus) in its latest Appraisal Consultation Document (ACD)1 is a huge set back for patients with poor prognosis advanced renal cell (kidney) cancer (RCC) and for those in the NHS who treat them.

Whilst Sutent® (sunitinib) has been recommended (subject to appeal), for first line treatment of advanced and/or metastatic RCC in the Final Appraisal Determination (FAD), the Appraisal Committee also concluded that sunitinib could not be considered a clinically effective first-line treatment for people with poor prognosis.2 Torisel was recognised as being clinically effective in this group.1 This means that, if the proposed recommendations in the ACD stand and Torisel is ultimately not recommended, patients with advanced RCC of poor prognosis, who are among the sickest, would be denied access to the only effective treatment that has been shown to significantly extend survival.3

Wyeth believes that denying patients access to the real and measurable benefits of this drug in extending life-expectancy compared to standard existing therapy3 is a devastating and cruel blow to patients and their families. There are about 450 patients in the UK eligible for treatment with Torisel.

Torisel, despite being found to be clinically effective, has been deemed not to be cost-effective in the current assessment. NICE itself has previously acknowledged that appraising drugs for very rare conditions “presents special difficulties”.4 Torisel is a drug for a very rare condition or an “ultra-orphan” drug and has not been appropriately assessed as such.

The proposed recommendations made by NICE accentuate the disparity in cancer care between the UK and other advanced countries, where Torisel and other kidney cancer drugs are routinely used.

Wyeth believes that a way must be found to ensure that life-enhancing, life-extending and life-saving medicines are available to patients who need them, whether they are for very rare diseases or more common conditions.

Wyeth remains ready to work constructively with NICE to develop appropriate methodologies to appraise “ultra-orphan” drugs on a fair and equitable basis.


TORISEL is an mTOR inhibitor indicated in the European Union for the first line treatment of patients with advanced renal cell carcinoma who have at least three of six prognostic risk factors. These risk factors are less than one year from time of initial RCC diagnosis to randomisation, Karnofsky performance status of 60 -70, haemoglobin less than the lower limit of normal, corrected calcium of greater than 10mg/dL, lactate dehydrogenase>1.5 times the upper limit of normal and more than one metastatic organ site.

TORISEL is an inhibitor of mTOR leading to a disruption of the mTOR signalling which results in suppression of proteins that regulate progression through cell cycle and angiogenesis.3

In March 2007, The European Association of Urology published guidelines reviewing TORISEL as a treatment to be considered as first-line treatment in patients with advanced RCC with poor-risk prognosis.5

“Orphan” and “Ultra- Orphan” medicines

Medicines used to treat rare conditions are referred to as “orphan”. In the EU, an orphan disease” is one with a prevalence of one case or less per 2,000 .4

“Ultra-orphan” drugs are medicines used to treat conditions with a prevalence of less than 1 in 50,000.4 Potentially 450 patients in the UK are eligible for treatment with Torisel which qualifies it to fall within the group of “ultra-orphan” drugs.

Wyeth Pharmaceuticals

Wyeth Pharmaceuticals, a division of Wyeth, has leading products in the areas of women’s health care, infectious disease, gastrointestinal health, central nervous system, inflammation, transplantation, haemophilia, oncology, vaccines and nutritional products.

Wyeth is one of the world’s largest research driven pharmaceutical and health care products companies. It is a leader in the discovery, development, manufacturing and marketing of pharmaceutical, vaccines, biotechnology products and non prescription medicines that improve the quality of life for people worldwide. The Company’s major divisions include Wyeth Pharmaceuticals, Wyeth Consumer Healthcare and Fort Dodge Animal Health.


1. NICE. Appraisal consultation document. Bevacizumab,sorafenib,sunitinib (second line) and temsirolimus for the treatment of advanced and/or
metastatic renal cell carcinoma. 4 February Available here.
Accessed February 2009.

2. NICE. Final appraisal determination. Sunitinib for the first-line treatment of advanced and/or metastatic renal cell carcinoma. 4 February 2009. Available here. Accessed February 2009

3. Hudes G,Carducci M, Tomczak P, et al. Temsirolimus, interferon alfa, or both for advanced renal-cell carcinoma. N Engl J Med. 2007;356;2271-2281

4. NICE. Appraising Orphan Drugs 16 March 2006. Available here. Accessed February 2009.

5. Ljungberg B, Hanbury DC, Kuczyk MA, et al. Guidelines on Renal Cell Carcinoma. European Association of Urology March 2007.

Wyeth Pharmaceuticals



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