Four new therapies to be funded by NHS Scotland

Pfizer’s Mylotarg (gemtuzumab ozogamicin) was accepted for the treatment of acute myeloid leukaemia (AML), having been considered through the SMC’s Patient and Clinician Engagement (PACE) process.

Current initial treatment for the condition is an intensive chemotherapy regimen, but for those who achieve remission, there is a high risk of relapse.

Mylotarg 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," the cost watchdog noted.

Sobi’s Kineret (anakinra) was endorsed as a treatment for Still’s disease, a rare type of inflammatory arthritis that can lead to joint contractures, eye problems, destructive joint disease requiring joint replacements, permanent disability and life-threatening sudden hyperactivity of the immune system.

Kineret, which is administered daily via subcutaneous injection, is a biologic immunosuppressive which blocks the activity of interleukin 1 (IL-1), a chemical messenger linked with typical symptoms such as arthritis in multiple joints, spiking fever, skin rash, hepatosplenomegaly, and serositis.

Elsewhere, Diurnal’s Alkindi (hydrocortisone) was accepted for the treatment of adrenal insufficiency in infants, children and adolescents.

Patients with this condition cannot produce enough cortisol in their adrenal glands, which can cause weight loss, muscle weakness, tiredness and low blood pressure.

Children are currently treated by crushing tablets of an adult formulation of hydrocortisone prepared by a pharmacist, leading to a risk of under- or over-dosing when parents try to administer the bitter-tasting crushed tablet as best as they can.

"This new formulation offers more accurate dosing and is tasteless, meaning it is likely to be easier for parents and carers to administer," the SMC said.

Also accepted was Eli Lilly’s biologic Taltz (ixekizumab) for psoriatic arthritis, an inflammatory joint disorder associated with the skin disease psoriasis.

The drug, which is administered as an injection, either alone or in combination with a conventional disease-modifying antirheumatic drug such as methotrexate, offers an additional option for those patients who have not responded to previous conventional and biologic therapies.

The SMC turned down Ipsen’s Cabometyx (cabozantinib) 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," it said.

Kidney Cancer Scotland said it is "very disappointed" with the decision.

"We know first-hand from patients that where this medicine is currently used as a second-line treatment, the side effects are more tolerable than other treatments available and, the progression of the cancer in many has slowed.

"We would like for all treatment options to be available to newly diagnosed kidney cancer patients and for clinicians to be the decider in when the patient has access to a medicine on their treatment pathway. Each kidney cancer patient is unique and responds differently to the treatments, there is no ‘one size fits all’ in the treatment of this disease."

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