The Scottish Medicines Consortium (SMC), which advises on newly licensed medicines for use by NHSScotland, has published advice accepting four new medicines, including ovarian and lung cancer treatments.

Niraparib (Zejula) was accepted for use for advanced ovarian cancer. It was considered through the SMC’s Patient and Clinician Engagement (PACE) process, which is used for medicines that treat end of life and very rare conditions. Niraparib is the first maintenance treatment for use in patients with ovarian cancer who do not have the BRCA mutation (an alteration in a particular gene that increases the chance of developing ovarian cancer). There are currently fewer satisfactory alternative treatments for this group of patients.

Niraparib has been shown to extend the period of relapse-free time until the next chemotherapy treatment. In addition, as an oral treatment, it can easily be taken at home.
Alectinib (Alecensa) was accepted for the treatment of a specific rare type of advanced non-small cell lung cancer (NSCLC). Patients are often diagnosed when the disease is at a late stage and treatment options are limited. Alectinib offers the benefit of an increase in the time to disease progression compared with existing therapy.

Glycerol phenylbutyrate (Ravicti) was accepted for the long term treatment of urea cycle disorders (UCDs) in adults and children from the age of two months. A UCD is a rare, inherited genetic condition where patients are unable to get rid of waste nitrogen from their bodies. The nitrogen accumulates in the form of ammonia and may lead to irreversible brain damage, coma and/or death if untreated. Glycerol phenylbutyrate helps to reduce levels of ammonia in the body. It offers an alternative to current treatment, which is often difficult for patients to adhere to, particularly for children, due to an unpleasant taste and the high number of tablets required.

Also accepted was conestat alfa (Ruconest) for the treatment of patients with the rare condition of acute hereditary angioedema. Patients with angioedema have attacks of swelling that can occur anywhere in the body, such as in the face or limbs, or around the gut, causing discomfort and pain. PACE participants described how patients can experience more than two attacks a week, which may each last several days. The unpredictability of an attack can cause significant disruption to the lives of patients and their families. Conestat alfa provides another treatment option for this condition.

The committee was unable to accept sapropterin (Kuvan), a medicine which treats phenylketonuria (PKU), a rare genetic condition where patients are unable to process the amino acid phenylalanine from dietary protein and therefore must adhere to a phenylalanine restricted diet. Following consideration through PACE, the committee did not recommend sapropterin due to uncertainties in the company’s evidence about both the long-term benefits of the medicine and its cost effectiveness. A meeting has been arranged between SMC and the submitting company to discuss next steps.

The committee was also unable to accept patiromer (Veltassa) for the treatment of hyperkalaemia, a condition in which potassium levels in the blood are raised. This was because the evidence provided by the company about the cost effectiveness of the medicine was not strong enough to justify accepting it for use by NHSScotland.

SMC Chairman Dr Alan MacDonald said: “The committee is pleased to be able to accept these medicines for use by NHSScotland.

“Through the evidence given by PACE participants, we know that our decision on niraparib for ovarian cancer will be welcomed, particularly as it provides the first maintenance treatment for patients without the BRCA mutation.

“For those with a specific type of NSCLC, alectinib can help delay the time to the progression of the disease, giving patients more opportunity to continue leading a normal life.

“As a more palatable alternative to current treatments for urea cycle disorders, glycerol phenylbutyrate may make it easier for patients to adhere to treatment, particularly children.

“Conestat alfa offers a helpful additional option for those with the painful and unpredictable condition of acute hereditary angioedema.

“Unfortunately, we were unable to accept sapropterin for PKU, as the evidence provided by the company on the clinical and economic benefits of using this medicine was not clear enough to justify accepting it for use.

“We were also unable to accept patiromer for the treatment of hyperkalaemia as the evidence provided by the company about the cost effectiveness of the medicine was not sufficiently strong.”