SMC approves funding for six new therapies on the NHS

Pfizer’s Besponsa (inotuzumab ozogamicin) was endorsed for the treatment of acute lymphoblastic leukaemia (ALL), a rare, very aggressive and rapidly progressing form of the disease with a particularly poor prognosis.

According to participants of the SMC’s Patient and Clinician Engagement (PACE) process, chemotherapy is often ineffective and the majority of patients are unable to go on to receive a transplant.

Besponsa can improve rates of remission and increase a patient’s chance of receiving a transplant, can be delivered without requiring a stay in hospital, and is better tolerated than other treatments, they said.

"We are delighted that patients in Scotland will now have routine access to inotuzumab ozogamicin, an important medicine which has been shown to help some of the most vulnerable leukaemia patients achieve complete remission," said Craig Eagle, head of Oncology, Pfizer UK.

"Following this good news in Scotland, we remain focused on continuing discussions with NICE with the aim to ensure this medicine can be made available to all eligible patients across the UK."

The Institute rejected the drug last year but following an appeal by Pfizer and subsequent re-assessment of the evidence, asked for more information on use of the drug in this setting in May.

Elsewhere, the SMC also accepted for use on NHS Scotland Novartis’ Votubia (everolimus) as a treatment for epilepsy in patients with tuberous sclerosis complex (TSC), a rare genetic disorder affecting both children and adults that causes mainly non-cancerous (benign) tumours to develop in different parts of the body, including the brain.

Through PACE, patient groups and clinicians said existing epilepsy medication is often unable to control these seizures caused by the condition, and that Votubia can reduce the frequency of these seizures, offering patients a better quality of life.

The Committee also backed use of Novartis’ Rydapt (midostaurin) for adults with newly diagnosed acute myeloid leukaemia (AML), after hearing from PACE participants that when added to current treatment the drug can improve survival and reduce the risk of disease relapse without substantial side-effects.

Pfizer’s Xalkori (crizotinib) was accepted for the treatment of with ROS1-positive advanced non-small cell lung cancer (NSCLC), a rare subtype of the disease.

The drug is the first targeted therapy for this group of patients and is associated with a high response rate, and evidence on use of Xalkori in a similar subtype of NSCLC indicates it is likely to result in an extended period of progression free and overall survival compared to chemotherapy treatment.

Ipsen Pharma’s Xermelo (telotristat ethyl) will be available on NHS Scotland to treat severe diarrhea associated with a rare condition called carcinoid syndrome, which can occur with a certain type of tumour.

PACE participants emphasized that the condition can be extremely debilitating and distressing, resulting in a poor quality of life, and that Xermelo is an oral therapy that offers increased symptom control.

The Committee also accepted Janssen’s Tremfya (guselkumab) for the treatment of severe plaque psoriasis in adults, expanding treatment options for those patients who have failed to respond to standard therapies.

Tremfya is the first biologic to selectively target interleukin (IL)-23, a key protein involved in the immune inflammatory response in psoriasis.

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