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The National Institute for Health and Care Excellence (NICE) has today issued negative draft guidance on new drug ibrutinib (marketed by Janssen as Imbruvica®), which does not recommend its use for the treatment of chronic lymphocytic leukaemia (CLL).
This draft guidance comes via the Appraisal Consultation Document (ACD), which sets out NICE’s preliminary recommendation. At this point, NICE has not recommended ibrutinib within its marketing authorisation for treating “chronic lymphocytic leukaemia (CLL) patients, that is either: for people who have had at least one prior therapy or, for people with 17p deletion or TP53 mutation and for whom chemo-immunotherapy is unsuitable”. Ibrutinib has previously been licensed by the European Medicines Agency (EMA) for use in Europe in this population.
A copy of the draft guidance is available here.
Chronic lymphocytic leukaemia (CLL) is the most common form of leukaemia, with approximately 3,200 people diagnosed in the UK each year. It is a leukaemia that affects B lymphocytes (a type of white blood cell). When a patient has CLL, the bone marrow produces too many B lymphocyte cells that are not fully developed and these cells fill up the bone marrow, preventing it from producing normal, healthy cells. Common symptoms of CLL include severe fatigue, breathlessness, anaemia, headaches, frequent infections, fever, unexplained weight loss, unexplained bleeding and bruising, night sweats and enlarged lymph nodes.
Ibrutinib is an oral inhibitor of a protein known as Bruton’s Tyrosine Kinase (BTK). Ibrutinib works by blocking the transmission of cell signals, which helps kill cancer cells and prevents cells multiplying, slowing down the progression of the cancer. At present ibrutinib is available to patients in England via the Cancer Drugs Fund (CDF) for the treatment of relapsed or refractory chronic lymphocytic leukaemia (where certain criteria apply).
NICE’s draft guidance is based on the available data from clinical trials, which has been submitted by the pharmaceutical company. Ibrutinib was licensed early (based on phase 2 trial data), so the phase 3 clinical trials are still ongoing. Ibrutinib is currently being studied in the phase 3 clinical trial RESONATE against the comparator treatment ofatumumab to determine whether ibrutinib offers patients an improvement in their treatment options. At this stage full survival data is not yet available for the group treated with ibrutinib, which has led NICE to say that it is not certain whether the clinical benefits of the drug justify the cost to the NHS.
The data required is ‘median progression-free survival’ (median PFS), a statistical term referring to the average time taken for half of the patients’ disease to progress. After a follow up period of 16 months, the PFS for ofatumumab was 8.1 months. At this point the PFS value for ibrutinib is not available, because PFS has not yet been reached. The PFS has not been reached because after 16 months of follow up patients on the trial were still being treated with ibrutinib (because they’re still responding to treatment). As such, the available clinical trial data demonstrates an improved PFS benefit (of at least 16 months, compared to 8.1). However, at present the extent of the benefit is ‘uncertain’.
As this is draft guidance, a consultation process involving NICE, Janssen (the manufacturer), professional bodies and patient organisations (including Leukaemia CARE) will now begin. It is hoped that this process will be able to collectively provide the information NICE requires to turn this ‘no’ into a ‘yes’. If the NICE decision (not to recommend ibrutinib) is upheld in the final guidance (expected June 2016) patients receiving the drug at that point would be able to continue to do so until their clinician feels it appropriate to stop.
Zack Pemberton-Whiteley, Head of Campaigns and Advocacy at Leukaemia CARE commented:
“We are extremely disappointed with NICE’s draft decision not to recommend the use of ibrutinib. If this decision is upheld in their final recommendation, it will severely limit access for patients to an extremely innovative medicine.
NICE’s failure to recommend ibrutinib, which is widely recommended throughout Europe, demonstrates the numerous flaws in NICE’s methodology when dealing with uncertainty. With the UK cancer survival outcomes continuing to lag far behind the rest of Europe, decisions like this cannot be allowed to continue.
It is particularly worrying, given the decision of the NHS England board last week to align NICE and the Cancer Drugs Fund into a single process that will assess all cancer drugs. In situations such as the appraisal of ibrutinib, an innovative medicine for a small patient population, NICE will need to resolve this uncertainty to enable patients to access the most clinically effective drugs at the earliest possible stage. If they are unable to do this, long-term access for patients will be extremely limited.
We urge NICE to reconsider their draft recommendation and hope that NICE and Janssen will continue to work together to find a solution to ensure that patients can continue to access ibrutinib in the future.”
For further information please contact our CARE lLine on 08088 010444 or email our Campaigns and Advocacy team on: email@example.com