Health Board Chief Executives in Wales have approved advice for three medicines, infliximab, ustekinumab (Stelera®) and vedolizumab (Entyvio®), after assessing them through the One Wales Medicines process.
Infliximab can be used for the treatment of refractory pulmonary sarcoidosis that has not responded to corticosteroids and other immunosuppressants. Pulmonary sarcoidosis is a rare condition that causes small patches of swollen tissue (called granulomas) to develop in the lungs which can cause a range of symptoms including shortness of breath and a persistent dry cough.
After reviewing the evidence summary report (ESR) compiled by AWTTC, the One Wales Medicines Advisory Group (OWMAG) considered that the evidence provided was sufficient to recommend its off-label use for this group of patients.
Two medicines, ustekinumab (Stelera®) and vedolizumab (Entyvio®) can be used for the treatment of inflammatory bowel disease (IBD), which includes ulcerative colitis and Crohn’s disease, in children and young people aged 6-17 years.
Ustekinumab (Stelera®) can be made available for children aged from 6 years with ulcerative colitis following loss of response, non-response or intolerance to anti-TNF therapies and vedolizumab. It is also available for children aged from 6 years with Crohn’s disease following loss of response, non-response or intolerance to anti-TNF therapies.
Vedolizumab (Entyvio®) can be made available for children aged from 6 years either with ulcerative colitis following loss of response, non-response or intolerance to anti-TNF therapies or with Crohn’s disease following loss of response or non-response to anti-TNF treatment and ustekinumab.
For both medicines, OWMAG considered that the evidence provided in the ESRs was sufficient to recommend their use in children and young people. Vedolizumab and ustekinumab are successfully used to treat IBD in adults and OWMAG were of the opinion that it was reasonable to assume similar efficacy and safety would be seen in people aged from 6 to 17 years. Children and young people with IBD which is poorly controlled are at risk of complications and repeated surgical interventions placing a significant burden on both physical and mental health and overall quality of life at this formative time. Access to these medicines may offer an effective treatment option for these patients and potentially avoid reliance on corticosteroids and/or prevent or delay surgery to control the disease.
The use of infliximab, vedolizumab and ustekinumab in the patient groups considered is unlicensed. Prescribers are reminded that the risks and benefits for each medicine should be clearly stated and discussed with patients or their carers to allow informed consent, and prescribers should consult the guidelines on prescribing unlicensed medicines.
AWTTC will review the advice for each medicine after 12 months or earlier if new evidence becomes available.
More about the One Wales Medicines process, including a new video explaining how the process works is available at https://awttc.nhs.wales/one-wales