The National Institute for Health and Care Excellence is backing the use of Janssen’s biologic Stelara to treat adult patients with moderately to severely active Crohn’s disease.
The cost watchdog has published a Final Appraisal Determination recommending the drug in line with its licensed indication, that is for patients who have failed to respond to conventional therapy or an anti-TNF-alpha therapy.
Crohn's disease is a chronic inflammatory bowel disease that affects the lining of the digestive tract at any point, affecting around 115,000 people in the UK. The exact cause of the condition is unknown, but it is associated with abnormalities of the immune system that could be triggered by a genetic predisposition or other environmental factor.
There is no known cure for the disease, so current treatment aims focus on stopping the inflammatory process, relieving symptoms and the avoidance of surgery wherever possible. However, there remains significant unmet need for patients with the condition, as just over half maintain relief from their symptoms for more than a year with currently available therapies or no appropriate treatment.
Stelara (ustekinumab) offers a different mode of action from other licensed therapies, being the first human monoclonal antibody for Crohn’s disease to target the interleukin (IL)-12 and IL-23 cytokines, which are known to play a key role in inflammatory and immune responses.
The drug was approved in Europe in November 2016 on the back of data from clinical studies showing that between 34 percent (UNITI-1 study) and 56 percent (UNITI-2 study) of patients experienced relief from their Crohn's disease symptoms in just six weeks after receiving the one-time intravenous infusion of Stelara.
The firm has also recently presented data showing that treatment with the drug maintains clinical response and remission in some patients for up to two years.
According to NICE’s preliminary guidelines, “the choice of treatment between ustekinumab or another biological therapy should be made on an individual basis after discussion between the patient and their clinician about the advantages and disadvantages of the treatments available. If more than one treatment is suitable, the least expensive should be chosen (taking into account administration costs, dosage and price per dose)”.
Dr Peter Irving, consultant gastroenterologist, Guy's and St Thomas' NHS Foundation Trust, said the decision will be welcomed both by patients and health care providers. “The promising results seen in the clinical trials suggest that the possibility of treating patients with a new drug employing a novel mode of action will reap real benefits for patients with a disease that can have enormous impact on their lives.”
Sarah Berry, Health Policy & Public Affairs Officer from Crohn’s and Colitis UK, said the impact of uncontrolled symptoms of Crohn’s disease “can be profoundly debilitating and current treatment options simply do not work for everyone.
“This ruling gives access to a desperately needed additional treatment option to those who may otherwise have been condemned to living with unmanageable disease progression with devastating impact on their quality of life.”