Novartis Oncology concerned by initial NICE decision to deny advanced kidney cancer patients effective treatment with Afinitor® (everolimus)1

·          Without everolimus, advanced kidney cancer patients who have failed on the only NICE approved first-line therapy will have no effective treatment option available to them2

Frimley, UK, 09 February 2010 — Novartis Oncology is seriously concerned that the National Institute for Health and Clinical Excellence (NICE) has decided against recommending Afinitor® (everolimus), the only licensed treatment proven effective for advanced kidney cancer patients who have failed on the only NICE approved first-line therapy (vascular endothelial growth factor (VEGF)-targeted therapy), because it says it is not cost-effective.1,2

Today‘s initial recommendation has been made despite the fact that NICE agrees everolimus meets its ‗end of life criteria‘ with both clinical and patient experts confirming the value of everolimus for advanced kidney cancer patients.1

―We simply do not understand how NICE can justify denying advanced kidney cancer patients access to an effective licensed treatment that will give them a chance for a longer and better quality of life,‖ said Panos Alexakos, Oncology Business Unit Head for Novartis UK. ―Once again, many advanced kidney cancer patients will have to spend the precious time they have left fighting for access to a treatment that is available in, and reimbursed by, many countries in Europe and in the US.‖

Every year there are 7,000 newly diagnosed cases of kidney cancer which causes around 3,700 deaths a year.3 While many kidney cancer patients will be diagnosed early and undergo surgery to cure them, 40% of patients will be diagnosed in the advanced stages when prognosis is extremely poor and the cancer is notoriously difficult to treat.4

Novartis has worked closely with the Department of Health (DH) to establish a patient access scheme (PAS) aimed at discounting the cost of everolimus to the NHS. The PAS was approved by the DH prior to Novartis submitting the application to NICE. Novartis acknowledges that the DH is very clear that a PAS does not guarantee a positive NICE decision but hoped that this upfront scheme would help ensure access to everolimus for those critically ill patients who have no other treatment options available to them.

The European Commission (EC) approved everolimus for the treatment of advanced kidney cancer after failure of treatments which prevent the growth of the tumour‘s blood vessels on 3rd August 2009.5 Prior to this, there were no licensed treatment options for advanced kidney cancer patients whose cancer progressed while on or after treatment with these targeted VEGF therapies.2 UK expert consensus opinion, published in a review journal in May 2009, recommends everolimus as a treatment option for advanced kidney cancer therapy after progression on targeted therapy.6

The Appraisal Consultation Document (ACD) from NICE provides a preliminary recommendation on whether a medicine should be funded by the NHS. This ACD states that although NICE acknowledges the clinical effectiveness of everolimus and recognises that patients have no other effective treatment options, the medicine is not considered to be ―cost effective‖ and would not represent an appropriate use of NHS funds.1 However, the decision is preliminary and should not prevent patient access to everolimus as the appraisal process continues. Novartis strongly believes that every cancer patient, regardless of their prognosis, should be offered the opportunity to gain access to effective licensed treatments.

NICE has invited Novartis Oncology and other formal consultees in the appraisal process, including patient/carer groups, to comment on the ACD by 5pm on 2 March. It is also possible for members of the public to comment via the NICE website.

Novartis Oncology intends to challenge this initial decision in the hope that patients will not have to fight for access to a treatment that they believe is both clinically and cost effective.

-          Ends –

Notes to Editors

NICE timings

The Appraisal Committee will meet again on 9 March to consider the original evidence and ACD in the light of the views of the consultees and members of the public. The Final Appraisal Document (FAD) is due to be published in mid-April.

About kidney cancer

Kidney cancer accounts for approximately 2% of all new cancers.7 In the UK, the incidence of kidney cancer is increasing3, due in part to obesity and smoking.8

In kidney cancer, cancer cells develop in the lining of the kidney‘s tubes and grow into a tumour. If left untreated, the tumour can spread to neighbouring lymph nodes and eventually to other organs.

About everolimus

In the UK, everolimus is indicated for patients with advanced kidney cancer whose disease progressed on or after treatment with VEGF-targeted therapy.6

In cancer cells, everolimus continuously targets the mammalian target of rapamycin (mTOR), a protein that acts as a central regulator of tumour cell division, blood vessel growth and cell metabolism.9

Data that led to the European approval shows that everolimus, when compared with placebo, more than doubled the median time without tumour growth or death in patients with advanced kidney cancer whose disease progressed following prior VEGF-targeted therapy (4.9 vs. 1.9 months).10,2 The data showed the reduction of the risk of disease progression or death by 67% based on the primary endpoint of progression-free survival (PFS) (hazard ratio=0.33 with 95% confidence interval 0.25 to 0.43; P<0.001).10

Important safety information

In the phase III trial, the most common adverse reactions, irrespective of causality, (incidence ≥30%) were stomatitis, infections, asthenia, fatigue, cough and diarrhoea. The most common grade 3/4 adverse reactions, irrespective of causality, (incidence ≥3%) were infections, dyspnoea, fatigue, stomatitis, dehydration, pneumonitis, abdominal pain and asthenia. The most common laboratory abnormalities (incidence ≥50%) were anaemia, hypercholesterolaemia, hypertriglyceridaemia, hyperglycaemia, lymphopaenia and increased creatinine. The most common grade 3/4 laboratory abnormalities (incidence ≥3%) were lymphopaenia, hyperglycaemia, anaemia, hypophosphataemia and hypercholesterolaemia. Deaths due to acute respiratory failure (0.7%), infection (0.7%) and acute renal failure (0.7%) were observed in patients receiving Afinitor. Afinitor may cause foetal harm in pregnant women.2

About Novartis

Novartis AG provides healthcare solutions that address the evolving needs of patients and societies. Focused solely on healthcare, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, cost-saving generic pharmaceuticals, preventive vaccines, diagnostic tools and consumer health products. Novartis is the only company with leading positions in these areas. In 2008, the Group‘s continuing operations achieved net sales of USD 41.5 billion and net income of USD 8.2 billion. Approximately USD 7.2 billion was invested in R&D activities throughout the Group. Headquartered in Basel, Switzerland, Novartis Group companies employ approximately 98,000 full-time-equivalent associates and operate in more than 140 countries around the world. For more information, please visit www.novartis.co.uk.

References

1. National Institute for Health and Clinical Excellence Appraisal consultation document – Everolimus for the second-line treatment of advanced renal cell carcinoma Issue date: February 2010

2. Motzer RJ, Escudier B, Oudard S et al for the RECORD-1 study group. Efficacy of everolimus in advanced renal cell carcinoma: a double-blind, randomised, placebo-controlled phase III trial. The Lancet. August 2008; 372;9637:449-456

3. Cancer Research UK: http://info.cancerresearchuk.org/cancerstats/types/kidney/incidence/. Accessed February 2010

4. The University of Texas MD Anderson Cancer Center. Kidney Cancer. Available at http://www.mdanderson.org/patient-and-cancer-information/cancer-information/cancer-types/kidney-cancer/index.html. Accessed September 2009

5. EPARs for Authorised Medicinal Products for Human Use http://www.emea.europa.eu/humandocs/Humans/EPAR/afinitor/afinitor.htm. Accessed February 2010

6. Nathan, P. et al. UK Guidelines for the Systemic Treatment of Renal Cell Carcinoma. British Journal of Hospital Medicine. Vol. 70, Iss. 5, May 13, 2009

7. McLaughlin JK, Lipworth L, Tarone RE. Epidemiological aspects of renal cell carcinoma. Semin Oncol. 2006 Oct;33(5):527-33. [Abstract]

8. Eisen, et al. Sorafenib for Older Patients With Renal Cell Carcinoma: Subset Analysis From a Randomized Trial. Journal of the National Cancer Institute. 2008; 100(20):1454-1463

9. Highlights of the NCCN 13th Annual Conference: Clinical Practice Guidelines & Quality Cancer Care, published as a supplement to The Oncology Report http://www.nccn.org/professionals/meetings/13thannual/highlights/1316.html. Accessed February 2010

10. Escudier, B et al. 72O – Phase III Randomised Trial of Everolimus (RAD001) vs Placebo in Metastatic Renal Cell Carcinoma. Presented at the European Society for Medical Oncology (ESMO) 33rd Congress, Stockholm, Sweden on 16 September 2008

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