Abatacept, adalimumab, etanercept and tocilizumab for treating juvenile idiopathic arthritis Technology appraisal guidance Published: 16 December 2015 nice.org.uk/guidance/ta373 © NICE 2019. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions#notice-of- rights). Abatacept, adalimumab, etanercept and tocilizumab for treating juvenile idiopathic arthritis (TA373) Your responsibility The recommendations in this guidance represent the view of NICE, arrived at after careful consideration of the evidence available. When exercising their judgement, health professionals are expected to take this guidance fully into account, alongside the individual needs, preferences and values of their patients. The application of the recommendations in this guidance are at the discretion of health professionals and their individual patients and do not override the responsibility of healthcare professionals to make decisions appropriate to the circumstances of the individual patient, in consultation with the patient and/or their carer or guardian. Commissioners and/or providers have a responsibility to provide the funding required to enable the guidance to be applied when individual health professionals and their patients wish to use it, in accordance with the NHS Constitution. They should do so in light of their duties to have due regard to the need to eliminate unlawful discrimination, to advance equality of opportunity and to reduce health inequalities. Commissioners and providers have a responsibility to promote an environmentally sustainable health and care system and should assess and reduce the environmental impact of implementing NICE recommendations wherever possible. © NICE 2019. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- Page 2 of conditions#notice-of-rights). 58 Abatacept, adalimumab, etanercept and tocilizumab for treating juvenile idiopathic arthritis (TA373) Contents 1 Guidance ............................................................................................................................................................................ 4 2 Clinical need and practice ........................................................................................................................................... 6 3 The technologies............................................................................................................................................................. 8 Abatacept............................................................................................................................................................................................ 8 Adalimumab ....................................................................................................................................................................................... 8 Etanercept .......................................................................................................................................................................................... 9 Tocilizumab......................................................................................................................................................................................... 10 4 Evidence and interpretation ...................................................................................................................................... 12 Clinical effectiveness evidence .................................................................................................................................................. 12 Cost-effectiveness evidence ....................................................................................................................................................... 18 Roche assumed that 1% of patients die every 6 months.................................................................................................. 26 Comments from consultees on the assessment report .................................................................................................... 27 Consideration of the evidence.................................................................................................................................................... 29 Clinical effectiveness...................................................................................................................................................................... 32 Cost effectiveness ........................................................................................................................................................................... 34 Summary of Appraisal Committee's key conclusions ........................................................................................................ 42 5 Implementation............................................................................................................................................................... 48 6 Recommendations for further research................................................................................................................ 50 7 Review of guidance........................................................................................................................................................ 51 8 Appraisal Committee members, guideline representatives and NICE project team .......................... 52 Appraisal Committee members ................................................................................................................................................. 52 NICE project team ........................................................................................................................................................................... 53 9 Sources of evidence considered by the Committee ......................................................................................... 55 About this guidance........................................................................................................................................................... 57 © NICE 2019. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- Page 3 of conditions#notice-of-rights). 58 Abatacept, adalimumab, etanercept and tocilizumab for treating juvenile idiopathic arthritis (TA373) This guidance replaces TA35. 1 Guidance This guidance replaces NICE technology appraisal guidance on the use of etanercept for the treatment of juvenile idiopathic arthritis. 1.1 Abatacept, adalimumab, etanercept and tocilizumab are recommended, within their marketing authorisations, as options for treating polyarticular juvenile idiopathic arthritis (JIA), including polyarticular-onset, polyarticular-course and extended oligoarticular JIA. That is: for abatacept, people 6 years and older whose disease has responded inadequately to other disease-modifying anti-rheumatic drugs (DMARDs) including at least 1 tumour necrosis factor (TNF) inhibitor for adalimumab, people 2 years and older whose disease has responded inadequately to 1 or more DMARD for etanercept, people 2 years and older whose disease has responded inadequately to, or who are intolerant of, methotrexate for tocilizumab, people 2 years and older whose disease has responded inadequately to previous therapy with methotrexate. Abatacept and tocilizumab are recommended only if the companies provide them with the discounts agreed in the patient access schemes for these technologies. 1.2 Adalimumab and etanercept are recommended, within their marketing authorisations, as options for treating enthesitis-related JIA, that is, for people 6 years and older (adalimumab) and 12 years and older (etanercept) whose disease has responded inadequately to, or who are intolerant of, conventional therapy. 1.3 Etanercept is recommended, within its marketing authorisation, as an option for treating psoriatic JIA, that is, in people aged 12 years and over whose disease has responded inadequately to, or who are intolerant of, methotrexate. © NICE 2019. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- Page 4 of conditions#notice-of-rights). 58 Abatacept, adalimumab, etanercept and tocilizumab for treating juvenile idiopathic arthritis (TA373) 1.4 When more than 1 technology is suitable (taking into account extra-articular manifestations) treatment should be started with the least expensive technology, taking into account administration costs, the dose needed and the product cost per dose. © NICE 2019. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- Page 5 of conditions#notice-of-rights). 58 Abatacept, adalimumab, etanercept and tocilizumab for treating juvenile idiopathic arthritis (TA373) 2 Clinical need and practice 2.1 Juvenile idiopathic arthritis (JIA) describes all forms of arthritis that have an unknown cause, an onset younger than 16 years, and joint inflammation that lasts more than 6 weeks. JIA is classified yb the number of joints affected. Oligoarticular JIA, also known as oligoarthritis, is diagnosed when 4 or fewer joints are affected over the first 6 months after diagnosis. Polyarticular-onset JIA, also known as polyarthritis, is diagnosed when 5 or more joints are affected over the first 6 months after diagnosis. After 6 months from diagnosis, if 5 or more joints become affected it is then referred to as polyarticular-course JIA. This includes people who are diagnosed with oligoarticular JIA but