The Scottish Medicines Consortium (SMC) has published advice accepting three medicines.
Eliglustat (Cerdelga) was accepted for the treatment of Gaucher disease. This is a rare inherited disorder which is debilitating, and if left untreated has a significant impact on quality of life due to a range of symptoms including excessive fatigue, spontaneous bleeding and bone pain.
Through SMC’s Patient and Clinician Engagement (PACE) process for medicines used to treat very rare and end of life conditions, it was highlighted that current treatment involves patients or their carers administering fortnightly enzyme replacement therapy via intravenous infusion at home, which is both time consuming and stressful. Eliglustat provides a well-tolerated alternative daily oral treatment which may improve symptom control and quality of life for patients and carers.
Also accepted through PACE was palbociclib (Ibrance) for advanced breast cancer. Participants at the PACE meeting spoke of the limited treatment options and the impact of their condition on their family. Palbociclib can increase the time patients have until the condition progresses and as an oral treatment it is easier to administer.
Also accepted was aviptadil/phenotolamine (Invicorp) for erectile dysfunction. Many physical conditions such as prostate cancer, multiple sclerosis or diabetes are associated with erectile dysfunction and it can impact negatively on psychological health. When oral treatments have failed, aviptadil/phenotolamine injection offers an alternative with fewer side effects than other current options.
The committee was unable to accept brodalumab (Kyntheum) for the treatment of moderate to severe plaque psoriasis, a condition which causes red scaly patches on the skin, because the company did not present a sufficiently robust economic analysis to gain acceptance by SMC.
SMC chairman Dr Alan MacDonald said: “I am pleased the committee has been able to accept these three medicines for use by NHS Scotland.
“From the testimonies we heard from patient groups and clinicians through our PACE meeting, we know eliglustat will be welcomed by patients and their carers, particularly as it reduces the burden of current treatment and may improve quality of life.
“Palbociclib offers patients with advanced breast cancer the potential for valuable additional time with their families and may delay the need for chemotherapy treatment for some patients.
“Aviptadil/phenotolamine is a useful addition for the treatment of erectile dysfunction, particularly as it may have fewer side effects than some other therapies.”