March 2015 decisions news release
The Scottish Medicines Consortium (SMC) has announced this month that seven new medicines have been accepted for use in NHSScotland.
Two of these medicines, dabrafenib (Tafinlar) for melanoma (skin cancer) and ruxolitinib (Jakavi) for disease-related splenomegaly (enlarged spleen) or symptoms in adults with primary myeolfibrosis (a condition where bone marrow tissue is gradually replaced with tough scar-like tissue) were accepted after consideration under the PACE (Patient and Clinician Engagement) process, which aims to improve patient access to new medicines for the treatment of end of life and very rare conditions.
An ultra-orphan medicine (medicine for a very rare condition) considered under the new processes, cabozantinib (Cometriq) for metastatic medullary thyroid carcinoma (MTC), a rare form of thyroid cancer, was not recommended for use.
Dabrafenib is for certain types of melanoma (skin cancer) which cannot be removed surgically or have spread to other parts of the body. Patient groups involved in the PACE process highlighted that the condition is particularly distressing for patients and carers as it often affects young adults. Dabrafenib offers patients and clinicians an alternative treatment which is as effective as other currently available treatments but which has a different side effect profile, so may be used in patients who are unable to tolerate these other treatments.
Ruxolitinib is used to treat disease-related splenomegaly (enlarged spleen) or symptoms in adults with primary myeolfibrosis. Because the abnormal bone marrow can no longer produce enough normal blood cells, the spleen often becomes enlarged as it takes over the production of blood cells. Patient groups and clinicians taking part in PACE spoke about the debilitating symptoms patients suffer, including extreme fatigue, night sweats and severe itching. There are no current medical treatments that effectively address these symptoms and ruxolitinib can greatly improve patients’ quality of life.
Also accepted for restricted use was idelalisib (Zydelig), which is used to treat chronic lymphocytic leukaemia (CLL), the most common form of leukaemia. While CLL can be treated by surgery, chemotherapy, radiation or a combination of these treatments, some patients with certain genetic changes in their cancer cells have a poor response to these treatments. SMC accepted idelalisib for use in patients with relapsed CLL who are unsuitable for chemotherapy and as a first line treatment for patients who have specific genetic changes which mean they are unsuitable for chemo-immunotherapy.
Ledipasvir/sofosbuvir (Harvoni) was accepted for restricted use in the treatment of certain types of chronic hepatitis C (CHC) in adults. Ledipasvir/sofosbuvir is a combination of two medicines that inhibit the replication of the hepatitis C virus (HCV) and may offer the prospect of cure for hepatitis C infection.
The committee also accepted two new biosimilar preparations of infliximab (Inflectra and Remsima) for restricted use in line with the current SMC and Healthcare Improvement Scotland advice for the reference product infliximab (Remicade®). Infliximab is used to treat joint conditions (such rheumatoid arthritis, psoriatic arthritis and ankylosing spondylitis), gastrointestinal conditions (Crohn’s diease and ulcerative colitis) and psoriasis. The managed introduction of these biosimilar preparations is expected to yield savings for the NHS as they are generally cheaper than the originator product.
Apixaban (Eliquis) is a blood thinning medicine (or anticoagulant). It has previously been accepted by SMC for the prevention of harmful blood clots (known as deep vein thrombosis) in adults undergoing hip and knee surgery and also for the prevention of stroke and systemic embolism in adults with non-valvular atrial fibrillation (heart rhythm disorder) . It has now also been accepted by the committee for the treatment of deep vein thrombosis (DVT) and pulmonary embolism (PE) and prevention of recurrent DVT and PE in adults.
SMC was unable to recommend cabozantinib (Cometriq), a treatment for a rare type of thyroid cancer. Cabozantinib was considered under the SMC’s decision making process for very rare (ultra-orphan) conditions. This process allows the committee to consider the wider impact a medicine may have for patients and their carers beyond direct health benefits and also its impact on specialist services and costs to the NHS and Personal Social Services. A PACE meeting was also held, where patient groups and clinicians gave powerful testimony about the benefits cabozantinib can offer to patients with no other treatment options. Cabozantinib also met one of the criteria that SMC can take into consideration in reaching a decision (known as modifiers); this was the absence of other treatments of proven benefit. However, the committee felt there were uncertainties around the clinical benefits and tolerability of cabozantinib. Despite applying the added flexibility of PACE and modifiers, SMC was unable to recommend cabozantinib due to weaknesses in the clinical and economic data.
Professor Jonathan Fox, chairman of the SMC, said:
“SMC is pleased to have accepted seven medicines that will benefit patients in Scotland with a variety of conditions. Two of these medicines were considered through our PACE process, bringing the number of medicines approved under this process to ten.
“Although the committee applied as much flexibility as possible, and despite the powerful additional input from patient groups and clinicians through PACE, we were unable to accept cabozantinib for medullary thyroid carcinoma. We know this decision will disappoint patients and clinicians alike as we understand the devastating impact of this condition.”