The Scottish Medicines Consortium (SMC), which advises on newly licensed medicines for use by NHSScotland, has published advice accepting 14 new medicines.
Cannabidiol (Epidyolex) was accepted for the treatment of Lennox-Gastaut syndrome, a rare type of epilepsy that presents in early childhood. The medicine was considered through SMC’s Patient and Clinician Engagement (PACE) process, which is used for medicines to treat end of life and very rare conditions. Through PACE, patient groups and clinicians spoke of the devastating impact Lennox-Gastaut syndrome has on the lives of patients, their family and carers. Seizures can be frequent, debilitating, severe and prolonged, often resulting in injury due to sudden rapid drops to the floor and hospital admissions. Cannabidiol in combination with another medicine, clobazam, can reduce seizure frequency for some patients, improving quality of life for patients and their families.
Cannabidiol (Epidyolex) in combination with clobazam was also accepted for the treatment of Dravet syndrome, another rare type of epilepsy, following consideration through PACE. Dravet syndrome is characterised by frequent seizures of various types, many of which are debilitating and severe. Cannabidiol can reduce the frequency and severity of seizures for some patients, enabling them to have a better quality of life.
Esketamine (Spravato) was accepted for treatment-resistant major depression in adults. Esketamine is used in combination with other antidepressants when at least two other treatments have failed. It is given as a nasal spray, supervised by a health professional in a clinic.
Pembrolizumab (Keytruda) was accepted for the treatment of head and neck squamous cell carcinoma (HNSCC) where the cancer has spread or cannot be removed. In the PACE meeting, patient groups and clinicians emphasised that HNSCC is a devastating disease with severe symptoms including pain, disfigurement and problems with breathing, speech and eating which can significantly impact patients’ quality of life. Prognosis is poor, with a life expectancy of 6-12 months. Pembrolizumab offers the potential for improved disease control which could improve quality of life and offer the possibility of improved survival compared with existing treatment.
The committee also accepted pembrolizumab (Keytruda) when given together with axitinib for the treatment of advanced renal cell carcinoma (kidney cancer) following consideration through PACE. PACE participants highlighted that advanced renal cell carcinoma is a devastating disease which is currently incurable. A substantial proportion of patients are diagnosed at the advanced stage of the disease, and can experience symptoms including pain, fatigue and lethargy. Pembrolizumab may improve disease control allowing patients to have more time without these symptoms. It may also enable some patients to return to more normal daily living.
Also accepted through PACE was gilteritinb (Xospata) for the treatment of relapsed or refractory acute myeloid leukaemia (AML) in patients who have a particular change (mutation) in the gene for a protein called FLT3. Through PACE, patient groups and clinicians highlighted how the FLT3 mutation is associated with a higher rate of relapse. The only option currently available is salvage chemotherapy which is associated with considerable toxicity. Gilteritinib is an oral tablet. It has been shown to increase overall survival and may also increase the chance of a patient being suitable for a subsequent stem cell transplant which may cure their disease.
Fluocinolone acetonide intravitreal implant (Iluvien) was accepted to prevent relapse in recurrent non-infectious uveitis (inflammation and swelling) occurring in a specific area of the eye. Patient groups highlighted how this condition affects vision and how patients fear the possibility of blindness. Current options to treat episodes of uveitis involve frequent hospital visits. The Iluvien implant, which is injected into the eye, lasts three years and reduces the chance of uveitis coming back over this period.
In addition to these medicines, SMC has also accepted the following medicines through an expedited approach to minimise delay in patient access following the early phase of the COVID-19 pandemic. More details can be found on its remobilisation webpage.
- Pertuzumab (Perjeta) for the adjuvant treatment of early stage breast cancer.
- Caplacizumab (Cablivi) for a blood clotting disorder known as acquired thrombotic thrombocytopenic purpura (aTTP).
- Sodium zirconium cyclosilicate (Lokelma) for the treatment of hyperkalaemia (high levels of potassium in the blood).
- Brolucizumab (Beovu) for the treatment of the ‘wet’ form of age-related macular degeneration (AMD).
The following medicines have been accepted for interim use subject to ongoing evaluation and future reassessment by SMC. Further information can be found on its interim acceptance webpage.
- Andexanet alfa (Ondexxya) to stop life-threatening or uncontrolled bleeding in adults taking certain medicines used to thin the blood.
- Polatuzumab (Polivy) to treat a type of lymphoma in patients who are unsuitable for a stem cell transplant.
- Holoclar, a stem-cell treatment used to replace damaged cells in the eye after chemical or physical burns.
SMC is now in the process of resuming meetings.