Several treatments recommended by NICE for moderate rheumatoid arthritis
On 10 June, the National Institute for Health and Care Excellence (NICE) published new final guidance that has recommended several new treatment options for around 25 000 people with moderate rheumatoid arthritis who have previously not responded to conventional therapies.
Rheumatoid arthritis is an incurable chronic systemic inflammatory autoimmune disease that affects the synovial joints (such as those in the hands and feet), which become inflamed, causing pain, swelling and stiffness.
As well as these new treatment options adalimumab, etanercept and infliximab, taken with methotrexate have been recommended for use within the NHS. Adalimumab and etanercept can also be used as monotherapy when methotrexate is contraindicated or not tolerated.
Previously, NICE has recommended biological treatments only for severe rheumatoid arthritis. The guidance was reviewed because biosimilars have been available, which means that these treatments are now available to the NHS at a lower price. A biosimilar medicine is a medication that is developed to be similar to an existing biological medicine.
During clinical trial, evidence showed that these treatments provide similar benefits for people with moderate disease as those with severe disease.
The treatments have been recommended for use only if intensive therapy with two or more conventional disease-modifying antirheumatic drugs have not controlled the disease.
Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE said, ‘I am delighted that we are able to recommend additional treatment options for people with moderate rheumatoid arthritis whose disease hasn't responded to conventional treatments. These recommendations come after a pragmatic review of existing guidance in response to the availability of biosimilars in the NHS. We are pleased that the introduction of biosimilars has lowered overall costs of treatment, allowing our independent committee to recommend biological treatment for more people with rheumatoid arthritis so they can enjoy a better quality of life.’
New NICE draft guidance does not recommend risdiplam for treating spinal muscular atrophy
Spinal muscular atrophy (SMA) is a progressive neuromuscular condition affecting the nerves in the spinal cord controlling movement. This leads to muscle weakness, progressive movement loss and difficulty breathing and swallowing.
SMA can be grouped into five main types (type 0 to 4), based on age of onset and the maximum motor function reached. SMA type 1 is one of the most severe forms of SMA and people with it usually die before the age of 2≥years old if untreated. Because SMA is a progressive disorder, everyone will experience more severe symptoms over time irrespective of type.
Risdiplam is currently licensed to treat people with SMA types 1, 2 and 3. Around 1500 people England would have been eligible for treatment with risdiplam, but NICE draft guidance does not recommend it.
The NICE committee decided that the clinical trials demonstrate that risdiplam meaningfully improves motor function for people with types 1, 2 and 3, but the long-term benefits remain uncertain. The committee also agreed that risdiplam is innovative, in that its oral administration means it is an alternative for people who cannot have other treatments for SMA. It also means people can have it at home.
NICE could not recommend risdiplam at this time, because the cost-effectiveness estimates were substantially higher than the range normally considered a cost-effective use of NHS resources. For type 1, this was despite agreeing that risdiplam could be considered a life-extending treatment at the end of life. The committee also acknowledged the rarity and severity of the disease in its decision making.
Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE, said, ‘We are disappointed not to be able to recommend this innovative new treatment as an option for people with this devastating condition, particularly where there remains a high unmet clinical need.
‘The committee considered that there may be some benefits that have not been captured in the company's economic model, such as improvements in respiratory function, fine motor skills, swallowing and ability to communicate. We will continue to work closely with the company to help them address the committee's concerns ahead of its next meeting.’