Treatment cost regulators for the NHS in Scotland have approved routine funding for six medicines, offering patients new treatment options for blood cancer, skin cancer, high cholesterol, diabetes, psoriatic arthritis, and infections in people with cystic fibrosis.
First up, the Scottish Medicines Consortium has accepted Janssen's Imbruvica (ibrutinib) for the treatment of mantle cell lymphoma (MCL), an extremely rare, incurable, aggressive form of non-Hodgkin's lymphoma.
The drug was considered through the SMC's Patient and Clinician Engagement process (PACE), for medicines that treat end of life and very rare conditions, during which patient groups and clinicians highlighted the limited effective treatment options for patients.
Imbruvica is targeted treatment for MCL, with the advantage of being an oral capsule which is easy to administer, and is also considered effective and well tolerated.
The Committee also accepted a submission for the drug for a sub-group of patients with chronic lymphocytic leukaemia (CLL) and rare genetic abnormalities (17p deletion or TP53 mutation). Again, current treatments for this condition are limited, and Imbruvica has been shown to delay disease progression and potentially improve quality of life.
Bristol-Myers Squibb's Opdivo (nivolumab) has been cleared by the SMC for use to treat advanced melanoma in patients who have not been previously treated with Yervoy (ipilimumab). The drug has been shown to delay the progression of skin cancer and also has different side-effects to some existing medicines, increasing the range of potential options for patients.
Raptor Pharmaceuticals' antibiotic Quinsair (levofloxacin) was backed for the treatment of long-term infections caused by the bacterium Pseudomonas aeruginosa in adults who have cystic fibrosis, an inherited disease in which there is an accumulation of thick mucus in the lungs that allows bacteria to grow more easily, causing infections. Quinsair is an inhaled treatment that can be used easily, enabling patients to have a better day to day quality of life.
Elsewhere, Sanofi's Praluent (alirocumab) can be used by NHS Scotland for the treatment of high cholesterol in patients at high cardiovascular risk in whom standard drug therapy has not lowered cholesterol levels adequately. The drug is to be prescribed by specialists and will be administered by fortnightly injections.
Novartis' Cosentyx (secukinumab) was accepted for the treatment of psoriatic arthritis, an inflammatory joint disorder associated with psoriasis. The SMC noted that current treatments may improve symptoms in the short term but they do not tackle the underlying cause of the condition; Cosentyx offers another option for those patients who have not responded to previous therapies.
Also in is Novo Nordisk's long-acting insulin Tresiba for the treatment of diabetes in adults, offering patients another treatment option. Clinical studies showed the drug to be non-inferior to other long-acting insulin analogues, assessed by the mean change in glycosylated haemoglobin (HbA1c), the SMC said.
On the down side, the Committee was unable to recommend CSL Behring's Respreeza (human alpha-1 proteinase inhibitor) for the treatment of emphysema due to severe alpha-1 proteinase inhibitor deficiency, as it was not satisfied that the company's evidence on the long term benefits of the medicine was strong enough to justify its cost to the NHS.
"We know this decision is difficult for patients and carers, but when making decisions the Committee has to consider not only the patients that will be treated with that medicine, but every patient who needs treatment by NHS Scotland," said Professor Jonathan Fox, chairman of the SMC Committee. "If SMC were to accept for routine use a medicine whose benefits are not clear, it might mean that patients with other conditions would lose out."