Patients in England and Wales living with advanced kidney cancer (renal cell carcinoma - RCC) are one step closer to being able to access Votrient® (pazopanib), a targeted oral treatment, on the National Health Service (NHS). The National Institute for Health and Clinical Excellence (NICE) has issued the Final Appraisal Determination (FAD) recommending Votrient as a first-line treatment option for people with advanced RCC who have not previously received cytokine therapy and who are of Eastern Cooperative Oncology Group (ECOG) performance status 0-1, on the basis that GSK provides the agreed patient access scheme. The access scheme offers a straight discount and makes provision for a possible partial rebate to the NHS in the future, conditional upon the outcome of a head-to-head trial with current standard of care treatment.1

The introduction of targeted cancer therapies has transformed the management of advanced RCC.2-4 However, despite improvements in efficacy, side effects observed with available treatments to date can affect patients' quality of life and their ability to carry out normal daily activities.5-10 This was acknowledged by the NICE Appraisal Committee in their evaluation of evidence from the patient experts and clinicians. With only one targeted treatment option (sunitinib) recommended by NICE as a first-line treatment until now,11 the NICE recommendation of Votrient will offer patients and clinicians a choice of effective treatment options with different side-effect profiles.

Votrient has been shown to effectively slow down the progression of advanced RCC whilst maintaining QoL compared with placebo; a significant consideration for patients at an advanced stage of disease.12,13 Votrient has an acceptable and manageable toxicity profile. The most frequent adverse events related to Votrient treatment (all grade incidence ?‰?20%) are diarrhoea, hair colour change, hypertension, nausea, fatigue, anorexia and increased liver enzymes.12,14 There was a low incidence of grade 3/4 hand-foot syndrome, mucositis/stomatitis and fatigue.12

Professor Robert Hawkins, Consultant Medical Oncologist at The Christie, Manchester commented on the NICE decision. "We have had a major role in the trials of pazopanib and I welcome its approval by NICE for the treatment of patients with kidney cancer. This is a significant development for advanced kidney cancer patients living in England and Wales as it gives access to a further oral drug with a different side effect profile from that of other licensed agents. With pazopanib, patients have a chance to control their kidney cancer whilst maintaining quality-of-life and avoiding some of the side effects, which can be severe, that can occur with current treatments. The importance of today's decision, both from the point of view of patients and those treating them, should not be under-estimated."

Advanced RCC is an aggressive form of kidney cancer with a poor prognosis, due in part to its resistance to chemotherapy, radiotherapy and hormone therapy.15,16 Over 8,000 people in the UK are diagnosed with kidney cancer each year17 and around one third show signs of advanced RCC at the time of diagnosis.2,18,19

Simon Jose, General Manager, GlaxoSmithKline (GSK) UK commented: "NICE has acted quickly in recognising Votrient as a clinical and cost-effective treatment option for advanced kidney cancer. It is often difficult to demonstrate the full value of innovative cancer medicines initially, as the evidence driving that value typically evolves over time as further studies are completed. We recognise this challenge and that is why we have offered an innovative potential future value rebate scheme. We are confident that the ongoing head-to-head study will confirm the full value of Votrient in this setting, but, if not, we will pay a partial rebate to the NHS. This is great news for patients and physicians who can have rapid and equitable access to this treatment, good for the NHS as it delivers value for money and also allows GSK to achieve a fair return for its innovation."

Notes

About the NICE Final Appraisal Determination

The Final Appraisal Determination (FAD) recommends Votrient as a first-line treatment option for people with advanced RCC who have not previously received cytokine therapy and have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.1

The ECOG is a scale used by doctors to assess how a patient's disease is progressing, how the disease affects the daily living abilities of the patient and used by doctors to determine appropriate treatment and prognosis. An ECOG performance status score of 0 or 1 is an indicator of good prognosis, which means that the patient is relatively healthy and well given the status of their disease. This is the same patient population as those enrolled in the Votrient and sunitinib phase III clinical trials.

About Votrient

Votrient is a new, selective20,21 oral treatment which effectively slows down the progression of advanced RCC12 whilst maintaining patients' quality of life (QoL) compared with placebo, as shown by the mean change from baseline in QoL scores.12,13 Votrient was granted a conditional marketing authorisation by the EMA in June 2010 and is indicated for the first-line treatment of advanced RCC and for patients who have received prior cytokine therapy for advanced disease. The licence is conditional14 upon the outcome of the ongoing head-to-head study versus sunitinib.

Votrient is a type of medicine called a tyrosine kinase inhibitor (TKI). It works by inhibiting angiogenesis (the process of developing new blood vessels), thereby slowing tumour growth and the spread of cancer to another part of the body.20-24 It also appears to have a different side effect profile from the other licensed protein TKIs in this setting.20,21,22,24

The most common adverse reactions (experienced by at least 10% of the patients of any grade) include: diarrhoea, hair colour change, hypertension, nausea, fatigue, anorexia, vomiting, dysgeusia, and elevated transaminases.12,14 There was a low incidence of grade 3/4 hand-foot syndrome, mucositis/stomatitis and fatigue.12 Serious adverse events with an incidence of