Bristol-Myers Squibb (BMS) has announced that the Medicines Healthcare Products and Regulatory Agency (MHRA) has granted UK patients with the most common type of kidney cancer,1 renal cell carcinoma (RCC), access to the investigational immunotherapy treatment, nivolumab, through the Early Access to Medicines Scheme (EAMS). Through EAMS, nivolumab will be available as monotherapy for the treatment of adult patients with advanced renal cell carcinoma after prior therapy. The scientific opinion is based on data from a Phase III clinical trial, in which nivolumab demonstrated a significant median overall survival benefit of 5.4 months compared to standard therapy, everolimus in patients who received prior therapy.2 BMS will provide funding for the scheme.
Dr James Larkin, Consultant Medical Oncologist, The Royal Marsden, commented: "In the UK, kidney cancer incidence has more than doubled in the last 30 years and for patients with advanced stages of the disease, the long-term outlook can be very poor and there is a high unmet medical need in this setting. Nivolumab is the first immunotherapy to demonstrate in a clinical trial, significant improvement in kidney cancer survival compared to standard therapy. Earlier access to innovative treatments is of huge importance and I am very pleased that the MHRA has recognised the life-extending benefit nivolumab can offer to patients with advanced kidney cancer."
Kidney cancer is the eighth most common cancer in the UK.3 During 2012, around 10,400 people were diagnosed with kidney cancer and around 4,300 people died from the disease (an average of more than 11 people per day) in the UK.3 Approximately 27% of kidney cancer patients will have stage IV disease when first diagnosed (cancer that has spread/metastasised).4 In these cases, it is estimated that around 15% of men and 10% of women will survive for five years or more, after diagnosis.5
Commenting on the decision, Life Sciences Minister, George Freeman said: "Advanced kidney cancer is a devastating condition, but thanks to our Early Access to Medicines Scheme NHS patients with the most common form of the disease will now be amongst the first in Europe to have access to this medicine. This scheme, launched in 2014, is making a real difference in speeding up access to innovative medicines, but we want to go further, which is why the independent Accelerated Access Review will recommend how we can get new treatments to patients even faster."
Nivolumab is a PD-1 (programmed death-1) immune checkpoint inhibitor that works by harnessing the ability of the immune system to fight cancer.6,7 Of the seven positive scientific opinions under this Scheme, today's decision marks the fourth positive opinion for nivolumab.8
In 2015, nivolumab was licensed in the European Union for the treatment of adult patients with locally advanced or metastatic squamous non-small cell lung cancer (NSCLC) after prior chemotherapy;6 NICE has since issued draft guidance not recommending it for reimbursement in NHS patients in this indication and a final decision from NICE is currently pending. In contrast, in January 2016, NICE issued draft guidance recommending nivolumab as monotherapy for use within its licence, as an option for treating advanced (unresectable or metastatic) melanoma in adults. NICE will also assess nivolumab for use in kidney cancer pending marketing authorisation.
Commenting on the announcement, Johanna Mercier, General Manager, Bristol-Myers Squibb UK & Ireland said: "Bristol-Myers Squibb has worked in collaboration with the MHRA to ensure early access to nivolumab, this time in patients with kidney cancer. Recent draft guidance issued by NICE not recommending nivolumab for squamous lung cancer patients is disappointing, however, we are hopeful that health bodies in the UK will recognise nivolumab for its value, innovation and advantageous survival benefit for patients, and ensure prompt reimbursement as soon as possible across all licensed indications."
Data supporting nivolumab
The data supporting early access to nivolumab today is based on a Phase III study comparing nivolumab to everolimus in previously-treated patients with advanced clear-cell renal cell carcinoma after prior anti-angiogenic treatment.2 In this study, nivolumab demonstrated median overall survival of 25 months (95% CI, 21.8-NE) versus 19.6 months (95% CI, 17.6- 23.1) for everolimus.2
The safety profile of nivolumab in the study was consistent with prior studies and favourable versus everolimus. Fewer grade 3-4 treatment-related adverse events (AEs) occurred with nivolumab (19%) compared to everolimus (37%). Any grade treatment-related AEs occurred in 79% of patients treated with nivolumab and 88% of patients treated with everolimus. The most frequent treatment-related AEs were fatigue (33%), pruritis (14%) and nausea (14%) in the nivolumab arm and fatigue (34%), stomatitis (29%) and anaemia (24%) in the everolimus arm.2
About the Early Access to Medicines Scheme (EAMS)9
Started in 2014 and granted only to therapies which are deemed to offer significant innovation over other medicines, the EAMS aims to give patients with life-threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation when there is a clear unmet medical need.
Under the scheme, the MHRA will give a scientific opinion on the benefit/risk balance of the medicine, based on the data available when the EAMS submission was made. The opinion lasts for a year and can be renewed.