March 2020 decisions news release
The Scottish Medicines Consortium, (SMC), which advises on newly licensed medicines for use by NHSScotland, has today (Monday 9 March) published advice on four new medicines. All of the medicines were considered through the SMC’s Patient and Clinician Engagement (PACE) process, which is used for medicines to treat end of life and very rare conditions.
Lorlatinib (Lorviqua) was accepted for interim use subject to ongoing evaluation and future reassessment by SMC. Lorlatinib can be used to treat a rare, advanced form of non-small cell lung cancer (NSCLC) in patients who have not responded to previous treatments. It is only used if the NSCLC is ‘ALK-positive’, which means the cancer cells have tested positive for a specific gene mutation. In the PACE meeting, patient groups and clinicians explained that this type of lung cancer often affects younger, fitter, non-smoking patients. It usually presents at a late stage when it has already spread to the brain, so can have a profound impact on quality of life and symptoms can be particularly difficult to manage. There are limited treatment options for this patient group. Lorlatinib may extend the time before the cancer returns and improve overall survival. The committee accepted lorlatinib on an interim basis and will consider further evidence on its effectiveness once this is available. Further information on interim acceptance can be found on the SMC website.
Rucaparib (Rubraca) was accepted for use as a maintenance treatment in patients whose recurring ovarian cancer has responded (partially or completely) to treatment with platinum-based cancer medicines. PACE participants highlighted how patients currently receive gruelling rounds of chemotherapy with increasingly shorter time between relapses. Rucaparib has been accepted for use only in patients who do not have the BRCA mutation (an alteration in a particular gene that increases the chance of developing ovarian cancer). Rucaparib has been shown to extend the period of relapse-free time until the next chemotherapy treatment and provides an alternative maintenance therapy option in this patient group. In addition, as an oral treatment, it can easily be taken at home.
Blinatumomab (Blincyto) was accepted for the treatment of Philadelphia-chromosome-negative CD19‑positive B‑precursor acute lymphoblastic leukaemia (ALL), a rare and aggressive form of leukaemia in patients who have gone into complete remission but retain some minimal residual disease. During the PACE meeting, patient groups and clinicians spoke of how patients are often diagnosed in their mid to late thirties, when many have significant work and family commitments. There is currently a lack of treatment options for this condition and blinatumomab may provide a bridge to curative bone marrow transplant for some patients. This advice extends the recommendation made by SMC for blinatumomab in June 2016, allowing it to be used earlier in the treatment pathway.
The committee was unable to accept mexiletine (Namuscla) for the treatment of symptoms of myotonia (muscle stiffness) in patients with non-dystrophic myotonic disorders, a group of inherited muscle disorders in which muscles are slow to relax after movement. In the PACE meeting, patient groups and clinicians highlighted that the main symptoms of this rare condition are muscle stiffness, pain, weakness and fatigue. The committee was unable to accept mexiletine as the company’s health economics evidence was not strong enough.
SMC co-vice chair Dr Mark MacGregor said:
“I am pleased our committee members were able to accept these three new medicines for use by NHSScotland.”
“Our decision to accept lorlatinib on an interim basis means that those with ALK-positive NSCLC will be able to access treatment while further data are gathered on the benefits of this medicine.”
“For those with recurring ovarian cancer who do not have the BRCA mutation, rucaparib provides a maintenance treatment that can increase the time until they need further chemotherapy. From the evidence given by patients and clinicians in our PACE meeting for this medicine, we know our decision will be welcomed.”
“The decision for blinatumomab offers patients with ALL not only an earlier treatment for their condition but for some it may provide a bridge to bone marrow transplant.”
“Although our PACE process allows committee members additional flexibility in their decision making, they were unable to accept mexiletine for the treatment of treatment of symptoms of myotonia (muscle stiffness) as the company’s evidence on the benefits of the medicine were not clear.”