Cost regulators for NHS treatments in Scotland have backed six medicines offering new options to some patients with cancer, chronic migraine, high cholesterol and iron deficiency.
First up, AstraZeneca's Tagrisso (osimertinib) was endorsed as a treatment for non-small cell lung cancer (NSCLC) after having been considered through the Scottish Medicines Consortium's Patient and Clinician Engagement (PACE) process.
The PACE meeting heard that the drug is better tolerated than other current treatments and is taken orally, factors which can allow patients to lead more normal lives and possibly return to work. AZ also offered a patient access scheme (PAS) that improves the drug's cost-effectiveness, allowing its acceptance for use to treat adult patients with locally advanced or metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive forms of NSCLC.
Novartis' Afinitor (everolimus) was backed for the treatment of unresectable or metastatic, well-differentiated non-functional neuroendocrine tumours of gastrointestinal or lung origin in adults with progressive disease.
During the PACE process patient groups stressed that current treatment options are limited and there is no other proven effective therapy for use in the later stages of the disease. Afinitor can offer patients a valuable delay in time to disease progression with the potential to extend survival, and also has the advantage of being an oral therapy that can be taken at home, the regulator noted.
Acceptance by the SMC is also dependent on the continued provision of a PAS that improves the cost-effectiveness of the drug.
Servier Laboratories' Lonsurf - a combination of the nucleoside analogue anti-viral trifluridine and enzyme-inhibitor tipiracil - was accepted for the treatment of advanced colorectal cancer in those patients who have already undergone several other treatments.
Through PACE, patient groups and clinicians described how other treatment options are extremely limited for this group. Lonsurf is an oral treatment with manageable side effects, and may offer patients an additional two months survival, which is important in the context of limited remaining time, the SMC highlighted.
Allergan's Botox (botulinum toxin type A) was endorsed as an option for the prevention of chronic migraine in the group of patients who experience headaches on at least 15 days a month and who have been unsuccessfully treated with the other therapy options available.
There are currently limited treatment options for patients with this condition, which can have a debilitating impact on daily life because of related symptoms such severe headache, nausea and/or vomiting, diarrhoea, hypersensitivity to light, and Botox can significantly reduce the number of 'headache days'.
The SMC has, however, restricted its use to adults whose condition has failed to respond to at least three prior oral prophylactic treatments, where medication overuse has been appropriately managed.
The Committee also accepted Amgen's Repatha (evolocumab) for routine use to lower cholesterol levels in patients at high cardiovascular risk in whom standard drug therapy has not lowered cholesterol levels adequately, as well as Pharmacosmos UK's Diafer (iron (III) isomaltoside 1000) for the treatment of iron deficiency in patients with chronic kidney disease who are on dialysis.
On the downside, it was unable to recommend use of Ferring's Noqdima (desmospressin) for the treatment of idiopathic nocturnal polyuria, a condition where people need to pass urine frequently during the nigh, as it was not satisfied that the company's evidence about the benefits of the medicine was strong enough to justify its cost to the NHS.
The drug was launched in the UK in November last year on trial data which showed that it nearly doubled the probability of patients achieving the primary endpoint - a reduction of night-time bladder voids by 33 percent - and also reduced nocturnal urine volume in men and women by more than 200 ml and increased time to first void in patients, enabling an average undisturbed sleep period of around 4.5 hours.