Draft NICE guidance recommends tocilizumab for systemic juvenile idiopathic arthritis
In final draft guidance published today, NICE is recommending tocilizumab (RoActemra, Roche Products) for treating systemic juvenile idiopathic arthritis (JIA). The draft recommendation is for the use of tocilizumab in children and young people aged 2 years and older, where specific previous treatments have not produced an adequate response, if the manufacturer makes tocilizumab available with the discount agreed as part of the patient access scheme.
At consultation stage, NICE's independent appraisal committee asked the manufacturer of tocilizumab for more information on its use in treating systemic JIA. The manufacturer has now provided this information and has also proposed a patient access scheme which it has agreed with the Department of Health. Taking this new information into account, the committee has now been able to recommend tocilizumab in this draft guidance. In cases where children and young people aged 2 years and older whose disease continues to respond to methotrexate or who have not been treated with methotrexate, tocilizumab isn't recommended. NICE has not yet issued final guidance to the NHS.
Although arthritis is commonly associated with older people, children and young people can also be affected. JIA has no known cause, and covers various forms of the condition. Systemic JIA may start with symptoms such as a fever or rash, with joints eventually becoming swollen and inflamed. It can affect children of any age causing severe pain and difficulties in their everyday life.
Professor Carole Longson, Health Technology Evaluation Centre Director at NICE said: “The discount agreed as part of the patient access scheme put forward by the manufacturer, in addition to the further information they provided at the Committee's request, has enabled the Committee to recommend tocilizumab for systemic juvenile idiopathic arthritis in today's draft guidance. The draft guidance proposes that tocilizumab should be the treatment for children aged over 2 years and young people where their systemic JIA hasn't responded well to other treatments. However if the disease is still responding to methotrexate, or it hasn't been tried yet, then tocilizumab isn't recommended. Systemic JIA has a huge impact on the lives of the children and young people who have the disease - it causes severe pain, fatigue and disability. This affects the child's family and school life, as well as their physical and emotional well-being. Tocilizumab as a potential treament would be good news for children with systemic JIA and those caring for them.”
Until final guidance is issued NHS bodies should make decisions locally on the funding of specific treatments.
Notes to Editors
About the guidance ‘tocilizumab for the treatment of systemic juvenile idiopathic arthritis'
1. More information on this appraisal can be found here: http://guidance.nice.org.uk/TA/Wave14/86
2. The full draft recommendations are:
i. Tocilizumab is recommended for the treatment of systemic juvenile idiopathic arthritis in children and young people aged 2 years and older whose disease has responded inadequately to non-steroidal anti-inflammatory drugs (NSAIDs), systemic corticosteroids and methotrexate, if the manufacturer makes tocilizumab available with the discount agreed as part of the patient access scheme.
ii. Tocilizumab is not recommended for the treatment of systemic juvenile idiopathic arthritis in children and young people aged 2 years and older whose disease continues to respond to methotrexate or who have not been treated with methotrexate.
iii. Children and young people currently receiving tocilizumab for the treatment of systemic juvenile idiopathic arthritis who do not meet the criteria in (i) should have the option to continue treatment until it is considered appropriate to stop. This decision should be made jointly by the clinicians, and the child or young person and/or their parents or carers.
3. The manufacturer of tocilizumab (RoActemra) is Roche Products.
4. Tocilizumab is a humanised monoclonal antibody that helps reduce inflammation in the joints, which can help prevent long-term damage, and improve quality of life and function.
5. The manufacturer submitted a patient access scheme (PAS) to the Department of Health, which is commercial in confidence. Despite the considerable uncertainty around the ICERs (incremental cost-effectiveness ratio, which is the extra cost per unit of health gained that the NHS is asked to pay), the most plausible ICERs for the strategy tocilizumab followed by infliximab compared with infliximab alone were within a range that would be considered an acceptable use of NHS resources. Given the other factors that had not been taken account of in the manufacturer's model (such as steroid sparing, a decrease in health-related quality of life of the parents or carers, reduction in future orthopaedic surgical operations, bone marrow transplantation and stem cell procedures), on balance, the Committee concluded that the resulting cost-effectiveness estimate would be at the lower end of this range.
6. The average cost of treatment is £7987.20 per year for a 30 kg patient and £9984 per year for a 25 kg patient assuming no wastage. Costs may vary in different settings because of negotiated procurement discounts. More of the drug is used in younger/smaller children - this is to do with the development of their organs. As their liver is still small the drug passes through their bodies faster, and so more of the drug is needed for the same benefit.
7. At public consultation stage in August 2011, NICE's independent appraisal committee requested that the manufacturer provide a range of detailed information on tocilizumab when it's used in treating systemic JIA where the individual's condition has responded inadequately to non-steroidal anti-inflammatory drugs (NSAIDs), systemic corticosteroids and methotrexate. The data requested included analysis of tocilizumab compared with other drugs commonly used to treat systemic JIA, data about joint damage for patients receiving tocilizumab including long-term follow-up, and a revised model. At that point, without this information, the Committee was minded to not recommend tocilizumab for treating systemic JIA.
8. The National Institute for Health and Care Excellence (NICE) is the independent organisation responsible for providing national guidance and standards on the promotion of good health and the prevention and treatment of ill health
9. NICE produces guidance in three areas of health:
- public health - guidance on the promotion of good health and the prevention of ill health for those working in the NHS, local authorities and the wider public and voluntary sector
- health technologies - guidance on the use of new and existing medicines, treatments, medical technologies (including devices and diagnostics) and procedures within the NHS
- clinical practice - guidance on the appropriate treatment and care of people with specific diseases and conditions within the NHS.
10. NICE produces standards for patient care:
- quality standards - these reflect the very best in high quality patient care, to help healthcare practitioners and commissioners of care deliver excellent services
- Quality and Outcomes Framework - NICE develops the clinical and health improvement indicators in the QOF, the Department of Health scheme which rewards GPs for how well they care for patients
11. NICE provides advice and support on putting NICE guidance and standards into practice through its implementation programme, and it collates and accredits high quality health guidance, research and information to help health professionals deliver the best patient care through NHS Evidence.
This page was last updated: 26 October 2011