October 2018 decisions news release

The Scottish Medicines Consortium (SMC), which advises on newly licensed medicines for use by NHSScotland, has today (Monday 8 October) published advice accepting four new medicines.

Gemtuzumab ozogamicin (Mylotarg) was accepted for the treatment of acute myeloid leukaemia (AML) following consideration through the SMC’s Patient and Clinician Engagement (PACE) process, which is used for medicines that treat end of life and very rare conditions. In the PACE meeting, patient groups and clinicians spoke of the devastating nature of AML, with patients often being diagnosed following an emergency admission. The condition carries a significant symptom burden, with fatigue, bleeding and frequent infections. Current initial treatment is an intensive chemotherapy regimen. For those who achieve remission, there is a high risk of relapse. Gemtuzumab ozogamicin is added to standard chemotherapy and can prolong the period before relapse and the time to further chemotherapy, allowing patients significant additional time with a better quality of life.

Anakinra (Kineret) was accepted for the treatment of Still’s disease, a rare type of inflammatory arthritis that is similar to rheumatoid arthritis. Still’s disease may begin during childhood as Systemic Juvenile Idiopathic Arthritis (SJJA) or in adulthood as Adult Onset Still’s Disease (AOSD). SJIA can lead to joint contractures, eye problems, destructive joint disease requiring joint replacements, permanent disability and life-threatening sudden hyperactivity of the immune system. Those with AOSD experience similar symptoms. For both patient groups, the condition can have a considerable impact on their personal lives and those of their families. Anakinra provides a useful additional treatment option for patients with Still’s disease, potentially improving their quality of life.

Hydrocortisone (Alkindi) was accepted for the treatment of adrenal insufficiency in infants, children and adolescents. Patients with this condition cannot produce enough of a hormone called cortisol in their adrenal glands. Cortisol is required to control many processes in the body, and a lack of it can cause weight loss, muscle weakness, tiredness and low blood pressure. Current treatment for children can be difficult to administer and adhere to, as it requires tablets to be crushed and mixed with water. Doses can often be difficult to measure and the bitter taste can make them difficult for children to take. This new formulation offers more accurate dosing and is tasteless, meaning it is likely to be easier for parents and carers to administer.

Also accepted was ixekizumab (Taltz), a bioengineered protein (biologic) therapy for psoriatic arthritis, an inflammatory joint disorder associated with the skin disease psoriasis. Psoriatic arthritis usually develops between the ages of 30 and 50, causing stiffness, pain and lack of movement in affected joints which can be irreversible and lead to disability. This can be particularly difficult for patients to deal with as they may have work and family commitments. Ixekizumab offers another option for those patients who have not responded to previous conventional and biologic therapies.

The committee were unable to accept cabozantinib (Cabometyx) for advanced renal cell carcinoma (kidney cancer). Despite the additional flexibility the PACE process allows, the committee was unable to recommend cabozantinib as there was too much uncertainty in the company’s evidence around the cost effectiveness of the medicine compared to the treatment options already available in NHSScotland.

SMC Chairman Dr Alan MacDonald said:

“The committee is pleased to be able to accept these four new medicines for use by NHSScotland.”

“Through the PACE meeting, we heard clearly how patients with AML have to endure significant symptoms from a condition for which there have been no new treatments for almost 30 years. We hope our decision on gemtuzumab ozogamicin will be of benefit to patients by allowing the opportunity for a period of significant additional time in remission, when quality of life is not reduced by disease symptoms or side-effects of chemotherapy.”

“For those with Still’s disease, anakinra is a helpful additional treatment option which may improve their quality of life.”

“The new formulation of hydrocortisone intended specifically for infants, children and adolescents with adrenal insufficiency will hopefully make treatment easier to administer and adhere to for both patients and their carers.”

“New therapies for psoriatic arthritis are always welcome and ixekizumab offers another option for those patients who have not responded to previous treatments.”

“Unfortunately we were unable to accept cabozantinib (Cabometyx) for advanced renal cell carcinoma as the company’s evidence about the cost effectiveness of the medicine when compared to other treatments currently in use was not strong enough.”

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