On June 10, 2021, the UK National Institute for Health and Care Excellence (NICE) released draft guidance recommending several tumour necrosis factor (TNF) inhibitors for patients in the UK with moderately active rheumatoid arthritis, potentially ending decades of needless suffering for some 25 000 patients.
Until now, TNF inhibitors were approved only for patients in the UK with severe rheumatoid arthritis—a disease activity score (DAS28) higher than 5·1—leaving those with moderate disease (scores between 3·2 and 5·1) bereft of advanced treatment options, even if they respond inadequately to conventional therapies. Many argued that the UK's previous cost-driven prescribing rules flew in the face of scientific evidence. TNF inhibitors are known to work equally well in patients with moderate disease as they do in those with severe disease, and without these drugs, patients with moderate disease have increased risk of functional deterioration and need for major surgery. The rule also seemed at odds with NICE's own treatment recommendations, which promote a treat-to-target approach aiming to achieve low disease activity (DAS28 less than 3·2) for all patients. Clinical experts advising the earlier (2016) NICE decision also argued that the DAS28 cutoff value was arbitrary, with the potential for clinically similar patients to fall on opposing sides of the cutoff.
The unmet need among patients with moderately active disease who are not treated with advanced therapies is borne out by the clinical data. In a recent survey by the UK National Rheumatoid Arthritis Society (NRAS) that included 612 such patients, 90% said they had had a flare in the past year, with 1 in 4 reporting more than six flares. Nearly three-quarters said that they had experienced sleep problems and severe fatigue; 70% had changed their working hours as a result of their disease.
This barrier in access to proven, effective treatment options for patients with moderate disease spurred a 3 year campaign by the NRAS and the British Society of Rheumatology to challenge the NICE eligibility criteria. But despite such powerful advocacy efforts, the new NICE guidance was primarily driven by the availability of biosimilars of originator TNF inhibitors, which has driven down the cost of these drugs, making them more affordable for the UK National Health Service (NHS) and allowing for relaxation of prescribing rules.
While cost considerations are inevitable and appropriate, whether the accessibility of proven, licensed medications for patients in the UK should be less stringent and allow for more physician discretion is worthy of consideration. In many European countries, the choice of the most appropriate medication is less prescriptive and is more often made as a shared decision between the treating physician and the patient. Indeed, a 2017 study placed the UK among the countries with the lowest access to biological disease-modifying antirheumatic drugs (DMARDs) in Europe, with an estimated 13% of patients eligible for biological DMARD treatment (based on 2013 EULAR criteria) able to access these therapies, compared with 59% in high-access countries (including Germany, Ireland, and Spain, among others). Not surprisingly, the mean DAS28 score of patients first prescribed a TNF inhibitor was the higher in the UK (mean DAS28 of 6·6) than in 11 other European countries studied in a 2011 report. The 2016 UK criteria also lacked the flexibility to expedite treatment in cases of rapid disease progression.
Time is also of the essence for patients with rheumatoid arthritis, with studies clearly showing that delayed treatment results in worse disease, more joint damage, and increased disability. However, in the UK, 1 in 4 patients with rheumatoid or inflammatory arthritis wait longer than 3 months for referral to a rheumatology specialist. The need to strengthen prevention of disease and disability was among the central priorities outlined by the recent Commission on the future of the NHS from the London School of Economics and The Lancet, which noted that the NHS continues to prioritise treatment over prevention, with funding for public health consistently decreasing relative to overall NHS funding.
The updated NICE guidance is a positive step in improving access to advanced treatment options for patients in the UK and will help to stem the burden of disability associated with rheumatoid arthritis. Going forward, however, a sharper focus on prevention is needed to further reduce the burden of disease and disability and to ensure that patients with rheumatoid arthritis have timely access to the best possible treatment.
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