Janssen statement in response to the NICE Appraisal Consultation Document (ACD) on the use of Imbruvica® (Ibrutinib) for relapsed or refractory Chronic Lymphocytic Leukaemia (CLL)

High Wycombe, 2 March 2016:  Janssen is extremely disappointed with the NICE Appraisal Consultation Document (ACD)1 published today which states that NICE does not intend to recommend Imbruvica® (Ibrutinib) for the treatment of adult patients with relapsed or refractory Chronic Lymphocytic Leukaemia (CLL) who are not suitable for chemo-immunotherapy.
This draft recommendation from NICE for ibrutinib sits in stark contrast to the recommendations of 48 countries globally which have opted to fund or reimburse the medicine including 27 European countries, most recently in Greece.2 In England, ibrutinib is currently available on the Cancer Drugs Fund (CDF) for use in adult patients with relapsed or refractory chronic lymphocytic leukaemia (CLL) and is the most requested drug for CLL on the fund3 - demonstrating the huge clinical demand.
This is a draft decision from NICE and an ongoing consultative process will now begin. Janssen will be actively participating in this process to ensure a solution can be found so that eligible patients can continue to access ibrutinib.
In February 2014, the U.S. Food and Drug Administration (FDA) granted ibrutinib accelerated approval for the treatment of CLL based on the results of a clinical study of previously treated patients.4 It has received three oncology breakthrough therapy designations from the FDA.5
Ibrutinib was granted a UK license on the basis of phase II clinical trial data.6,7 Further phase III data demonstrated a significantly improved survival benefit.8 Encouragingly for people living with CLL, the ibrutinib trials are still ongoing because patients continue to survive on treatment. Unfortunately, however, the current NICE process views ongoing studies as "uncertain" and thus disadvantages innovative medicines that keep people alive longer. 
Mark Hicken, Managing Director of Janssen UK, said: "At Janssen, our first priority is to patients and so we are extremely disappointed with the preliminary recommendation from NICE not to recommend ibrutinib for adult patients with CLL, who have limited treatment options.
"There is very high clinical demand for this innovative medicine, so this decision is yet another example of how the current NICE appraisal process is not fit-for-purpose in responding to the needs of cancer patients and the healthcare professionals who treat them. This is a worrying sign for people living in England and is, unfortunately, likely to be much more common under the new Cancer Drugs Fund process approved by the NHS England Board last week. 
"If this decision is allowed to stand, patients in England are likely to have access to even fewer new cancer medicines in future, leading us to fall even further behind the rest of Western Europe.
"Our submission to NICE demonstrates cost-effectiveness and meets their end of life criteria and we intend to challenge this initial recommendation. We hope NICE will reconsider its position so that this important medicine can be routinely available to the patients that need it."
Approximately one in every twenty-five people will be diagnosed with blood cancer during their lifetime.9 That equates to around 30,000 people each year being diagnosed with blood cancer in the UK;10 of these around 3,400 will have CLL11.
The public consultation has now started and interested parties may submit a response through the NICE website. The deadline for response is 23rd March, 2016.1
- ENDS -
For further information please contact:
Janssen                                                Munro & Forster
Miriam Cox: 01494 567 609             Rita Martins: 0207 089 6104
Notes to Editors:
About ibrutinib
Ibrutinib is a first-in-class Bruton's tyrosine kinase (BTK) inhibitor, which works by forming a strong covalent bond with BTK to block the transmission of cell survival signals within the malignant B cells.12 By blocking this BTK protein, ibrutinib helps kill and reduce the number of cancer cells. It also slows down the worsening of the cancer.13
Ibrutinib is approved in Europe for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL), or adult patients with chronic lymphocytic leukaemia (CLL) who have received at least one prior therapy, or in first line patients with CLL in the presence of 17p deletion or TP53 mutation in patients unsuitable for chemo-immunotherapy;14 it is also approved for adult patients with Waldenström's macroglobulinemia (WM) who have received at least one prior therapy, or in first line treatment for patients unsuitable for chemo-immunotherapy;14 regulatory approval for additional uses has not yet been granted.
The most commonly occurring adverse drug reactions (≥ 20%) reported in clinical studies were neutropenia, anaemia, diarrhoea, musculoskeletal pain, upper respiratory tract infection, bruising, rash, nausea and pyrexia. The most common grade 3/4 adverse drug reactions (≥ 5%) were anaemia, neutropenia, pneumonia and thrombocytopenia.14 For more information, visit https://www.medicines.org.uk/emc/medicine/29383.
Ibrutinib is co-developed by Cilag GmbH International, a member of the Janssen Pharmaceutical Companies, and Pharmacyclics LLC, an AbbVie company. Janssen affiliates market ibrutinib in EMEA (Europe, Middle East and Africa) as well as the rest of the world, except for the United States, where Janssen Biotech, Inc. and Pharmacyclics LLC co-market it. Janssen and Pharmacyclics are continuing an extensive clinical development programme for ibrutinib, including Phase 3 study commitments in multiple patient populations.
Janssen in Oncology
In oncology, our goal is to fundamentally alter the way cancer is understood, diagnosed, and managed, reinforcing our commitment to the patients who inspire us. In looking to find innovative ways to address the cancer challenge, our primary efforts focus on several treatment and prevention solutions. These include a focus on haematologic malignancies, prostate cancer and lung cancer; cancer interception with the goal of developing products that interrupt the carcinogenic process; biomarkers that may help guide targeted, individualised use of our therapies; as well as safe and effective identification and treatment of early changes in the tumour microenvironment.
About Janssen
Janssen Pharmaceutical Companies of Johnson & Johnson are dedicated to addressing and solving the most important unmet medical needs of our time, including oncology (e.g., multiple myeloma and prostate cancer), immunology (e.g., psoriasis), neuroscience (e.g., schizophrenia, dementia and pain), infectious disease (e.g., HIV/AIDS, hepatitis C and tuberculosis), and cardiovascular and metabolic diseases (e.g., diabetes). Driven by our commitment to patients, we develop sustainable, integrated healthcare solutions by working side-by-side with healthcare stakeholders, based on partnerships of trust and transparency. More information can be found on www.janssen.co.uk. Follow us on www.twitter.com/janssenUK for our latest news.
About Johnson & Johnson
Caring for the world, one person at a time, inspires and unites the people of Johnson & Johnson. We embrace research and science – bringing innovative ideas, products and services to advance the health and well-being of people. Our approximately 126,500 employees at more than 265 Johnson & Johnson operating companies work with partners in health care to touch the lives of over a billion people every day, throughout the world.
1.      NICE ACD. Leukaemia (chronic lymphocytic) - ibrutinib [ID749]. Available at www.nice.org.uk
2.      Janssen Data on File, Ibrutinib_DoF_01March2016_FH_001
3.      NHS England. Cancer Drugs Fund Quarterly Figures Reporting Template. Reporting Period: April – September 2015. Available at https://www.england.nhs.uk/ourwork/cancer/cdf/. Last accessed February 2016
4.      U.S. Food and Drug Administration. FDA approves Imbruvica to treat chronic lymphocytic leukemia. Press Release. February 2014. Available at http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm385764.htm. Last accessed February 2016
5.      U.S. Food and Drug Administration. Drugs and Biologic Approval Reports. December 2015. Available at http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/DrugandBiologicApprovalReports/NDAandBLAApprovalReports/ucm373418.htm. Last accessed February 2016
6.      Byrd JC et al. Targeting BTK with Ibrutinib in Relapsed Chronic Lymphocytic Leukemia N Engl J Med 2013; 369(1):32–42
7.      Wang ML, Rule S, Martin P, et al. Targeting BTK with ibrutinib in relapsed or refractory mantle-cell lymphoma. N Engl J Med 2013;369:507-16
8.      Byrd JC, Brown JR, O'Brien S, et al. Ibrutinib versus ofatumumab in previously treated chronic lymphoid leukemia. N Engl J Med 2014;371(3):213-23
9.      Leukaemia and Lymphoma Research. Newly diagnosed with blood cancer. Available at http://leukaemialymphomaresearch.org.uk/sites/default/files/newly_diagnosed_pdf_online_glossary.pdf Last accessed February 2016
10.   Delete Blood Cancer. About blood cancer. Available at http://www.deletebloodcancer.org.uk/en/about-blood-cancer. Last accessed February 2016
11.   Cancer Research UK. Chronic lymphocytic leukaemia (CLL) statistics. Available at http://www.cancerresearchuk.org/health-professional/chronic-lymphocytic-leukaemia-cll-statistics. Last accessed February 2016
12.   O'Brien S, Furman RR, Coutre SE, et al. Ibrutinib as initial therapy for elderly patients with chronic lymphocytic leukaemia or small lymphocytic lymphoma: an open-label, multicentre, phase 1b/2 trial. Lancet Oncol 2014;15:48-58
13.   European Medicines Agency. How is the medicine expected to work? http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/orphans/2012/06/human_orphan_001058.jsp&mid=WC0b01ac058001d12b. Last accessed February 2016
14.   Imbruvica Summary of Product Characteristics Last Updated on eMC 18-Nov-2015. Available at: https://www.medicines.org.uk/emc/medicine/29383. Last accessed February 2016