A medicine used to treat a rare blood cancer in children and young adults has today been accepted by the Scottish Medicines Consortium (SMC) for use in NHSScotland.
Tisagenlecleucel (Kymriah) is used to treat acute lymphoblastic leukaemia in children and young adults who have not responded to previous treatment or in whom the condition has relapsed. Tisagenlecleucel was accepted following consideration through the SMC’s Patient and Clinician Engagement (PACE) process, which is used for medicines to treat end of life and very rare conditions. In the PACE meeting, patient groups and clinicians highlighted that treatment options for this rare and rapidly progressing form of leukaemia are limited. Tisagenlecleucel is a new type of medicine called CAR-T cell therapy. It uses the patient’s own immune cells (T-cells) to destroy the cancer cells. The patient’s T-cells are collected and modified in the laboratory to create CAR-T cells. These cells are then given back to the patient through a drip into the vein. The CAR-T cells bind to and kill the cancer cells. Tisagenlecleucel offers the possibility of long-term remission and is potentially a lifesaving treatment option.
Dabrafenib (Tafinlar) was accepted for use in combination with another medicine, trametinib, for the treatment of stage III melanoma (skin cancer) in patients following surgical removal of the tumour. Dabrafenib with trametinib has been shown to extend the length of time before the recurrence of disease.
Tofacitinib citrate (Xeljanz) was accepted for the treatment of moderately to severely active ulcerative colitis, a disease which causes inflammation of the gut. Patients with this condition can experience frequent diarrhoea, abdominal pain and fatigue, which can affect their ability to work and socialise. Tofacitinib is the first oral treatment for this condition and offers another treatment option for those patients who have not responded to or been intolerant of other currently available treatments.
Rivaroxaban (Xarelto) was accepted for use in combination with aspirin in some patients with coronary heart disease, a condition that may lead to heart attacks or strokes. Rivaroxaban reduces the risk of blood clots by blocking a blood clotting component called Factor Xa.
The committee was unable to accept pembrolizumab (Keytruda) in combination with chemotherapy as a first line treatment of advanced lung cancer following consideration through the PACE process. Despite the additional flexibility this process allows, the committee did not recommend pembrolizumab as there was too much uncertainty around the company’s evidence about the treatment’s cost in relation to its health benefits and in addition, the committee did not consider it to be a good use of NHS resources.
Cabozantinib (Cabometyx) as a first line treatment advanced renal cell carcinoma (kidney cancer) was also not recommended following consideration through PACE. The committee was unable to accept cabozantinib as there was too much uncertainty in the company’s evidence around the cost effectiveness of the medicine compared to treatment options already available.
Axicabtagene ciloleucel (Yescarta) for lymphoma (a type of blood cancer) was not recommended following consideration through PACE. PACE participants spoke of how axicabtagene ciloleucel could potentially offer long-term disease control and may be life extending for some patients. The committee was unable to accept the medicine because of uncertainty in the company’s evidence around its long term benefits.
SMC Chairman Dr Alan MacDonald said:
“The committee is pleased to be able to accept these four new medicines for use by NHSScotland.
“Acute lymphoblastic lymphoma is a very rare form of leukaemia which can progress quickly and there are currently very few treatment options for those whose condition has relapsed or not responded. Tisagenlecleucel offers innovative and potentially lifesaving treatment for children and young adults with this condition, and we hope our decision will benefit them, their families and carers.
“For those with stage III melanoma, following surgery dabrafenib in combination with trametinib may help reduce the risk of the melanoma returning, and as an oral therapy it is easier for patients to take.
“Tofacitinib offers another important treatment option for those who have not responded to previous treatments for ulcerative colitis.
“For some patients at risk of heart attacks due to coronary artery disease, rivaroxaban provides a helpful new treatment option.
“We were unable to accept pembrolizumab (Keytruda) for the treatment of advanced lung cancer as there was too much uncertainty in the company’s evidence about its cost-effectiveness.
“Despite the additional flexibility provided by our PACE process, the committee was unable to accept axicabtagene ciloleucel (Yescarta) for lymphoma as there was uncertainty in the company’s evidence around its long-term benefits and its cost-effectiveness.
“We were also unable to accept cabozantinib for advanced renal cell carcinoma due to uncertainties in the company’s evidence around the cost-effectiveness of the medicine.”