NICE offers hope for thousands who until now, potentially faced a life of disability and pain21 January 2021 The National Institute for Health & Care Excellence (NICE) today released the Final Appraisal Document (FAD) on a new JAK inhibitor* filgotinib (Jyseleca) used in the treatment of Rheumatoid Arthritis (RA). NRAS warmly welcomes the fact that the FAD confirms that this drug will be available to not just to those most severely affected by this dreadful disease but also those with active uncontrolled disease previously referred to as having ‘moderate’ disease activity. NRAS has campaigned alongside the British Society for Rheumatology (BSR) for a number of years to gain access to advanced therapies, such as this, for people with ‘moderate’ disease who often fare just as badly as those with ‘severe’ disease. Many people with ‘moderate’ disease still experience the same painful and debilitating symptoms of RA as those with ‘severe’ disease but don’t quite meet the current NICE eligibility criteria of a DAS score of >5.1. We are therefore delighted that NICE has extended the use of this new targeted synthetic disease modifying anti-rheumatic drug to people categorised as having ‘moderate’ RA with a Disease Activity Score falling between 3.2 and 5.1 This FAD from NICE comes in the same week as a paper published by NRAS (P. Kiely et al – Rheumatology Advances in Practice – 5/1/21) entitled “RA disease impact in patients not treated with advanced therapies; survey findings from the National Rheumatoid Arthritis Society”. Our paper describes the results of a survey of over 600 people with RA who are not currently receiving an advanced therapy, and demonstrates very clearly that many of these individuals experience profound difficulties in everyday living with RA, across a broad range of measures. The survey included the RA Impact of Disease (RAID) questionnaire and only 12.4% of respondents were currently in a patient acceptable state, as defined by a total RAID score <2. The high impact of so-called “moderately active” RA on everyday life is further emphasised by the finding that in all seven domains >50% respondents recorded scores in the high range, indicating a significant burden in the last one week. This is supported by impact on work data from the survey, with 70% of respondents reporting a change in working hours due to their RA. Difficulties with daily physical activities and worsened physical and emotional well-being were significantly associated with higher pain, greater number of flares and worsened ability to cope. RA flares were extremely common with 90% experiencing at least one flare and nearly a quarter reporting 6 or more flares in the last year. Thus, across all assessed patient reported outcome measures, many RA patients not currently taking advanced therapies experience a related burden of adverse outcomes from their disease. NRAS has always advocated that access to the most appropriate treatment at the right time to bring RA disease activity under optimal control will greatly reduce the impact and burden of disease on individual lives. This change will also enable many more people living with RA to continue to work and live full, productive lives. Earlier treatment in the disease pathway of those with uncontrolled disease will benefit not only the individual but their families, the economy and the health service leading to potentially less surgical and clinical intervention and ultimately less disability. Most European countries do not have the very high eligibility criteria imposed by NICE in the UK for the last two decades which, by comparison, has disadvantaged people with RA in the UK. This very welcome news regarding filgotinib (Jyseleca) brings us a step closer to alignment with most of the rest of Europe. Our helpline hears on a daily basis just how poor quality of life can be for people with moderate disease who don’t quite reach the currently eligibility criteria to move to a more effective treatment which could bring their disease under better control. It is incredibly reassuring that NICE has now widened access for this new drug and we hope that soon access to other therapies will also be considered as NRAS and the BSR have been calling for. Ailsa Bosworth, MBE, National Patient Champion Living with constant pain, fatigue and the fear of long-term disability with the anxiety and potential mental health issues that this can cause, has been a situation which has troubled us at NRAS for a long time. ‘Moderate’ is not a word we would use to describe RA for many of the people categorised as such. The word conjures up thoughts of perhaps something ‘not too serious’, and while ‘uncomfortable – bearable’. I don’t think I have ever met someone living with RA with disease categorised as ‘moderate’ who would agree that their disease is ‘bearable’. They have to bear with it, but they pay a very heavy price in every aspect of their lives as has been shown by our above paper. This new JAK inhibitor is the start of a much brighter future for many once clinicians have this as an option for treating ‘moderate’ disease. It is a very significant milestone in the treatment of rheumatoid arthritis. Clare Jacklin, NRAS Chief Executive It has been wonderful to see enormous advances in clinical science over the last generation with the development of many highly effective therapies. But for far too long in the UK, access to the most potent treatments has been restricted to those individuals with the highest disease activity. This has meant that many people living with debilitating symptoms related to disease activity have been denied the chance to try potent drugs and, in some cases, to prevent further disease progression. It is therefore wonderful news that filgotinib (Jyseleca) will now be available to help some such individuals. It is important to remember, however, that your physician will need to discuss the potential benefits as well as any potential risks of available therapies in your individual case. We still cannot cure or completely remove all the features of RA. But it is the case that the outlook is better than ever before! Prof Peter Taylor, Chief Medical Advisor to NRAS Article Here for You Here for You is a new service by NRAS that allows you to arrange a call with someone with RA who understands you.