The Scottish Medicines Consortium (SMC), which advises on newly licensed medicines for use by NHSScotland, has published advice accepting four new medicines.
Ruxolitinib (Jakavi) was accepted for the treatment of polycythaemia vera (PV). PV is a rare and incurable condition that causes too many red blood cells to be produced, reducing blood flow to the organs due to ‘thickening’ of the blood and occasionally the formation of blood clots. 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. PACE participants emphasised that patients may suffer from debilitating fatigue and intense skin itching, which can be severe and relentless. Patients also have an increased risk of thrombotic events which can lead to hospitalisation due to blood clots or stroke. Current options are limited for patients who are unable to tolerate standard treatments. Ruxolitinib can help reduce the symptom burden of PV, allowing patients to resume work and social activities.
Lanadelumab (Takhzyro) was accepted for the routine prevention of frequent severe attacks of hereditary angioedema. Patients with this rare condition can have rapid swelling under the skin in areas such as the face, throat, arms and legs. In the PACE meeting, patient groups and clinicians highlighted that hereditary angioedema is a life-long condition and can cause significant anxiety. Attacks are unpredictable and can be life threatening when the swelling around the throat presses against the airway. Current preventative treatment is given intravenously which can be difficult for some patients and may not always be feasible. Lanadelumab can help maintain an acceptable level of attack control and is well tolerated, which can help reduce anxiety around the threat of further attacks. As the treatment is given by subcutaneous injection, with the opportunity for patients to self-administer, fewer hospital visits are required.
Olaparib (Lynparza) for ovarian cancer was also accepted following consideration through PACE. PACE participants highlighted that this diagnosis is often made at an advanced stage and patients tend to be younger and have significant family and work commitments. Olaparib maintenance treatment, after initial chemotherapy, increases the time that patients are free of recurrence, delaying the time to next chemotherapy and potentially offering increased survival benefits. As an oral treatment, it also reduces the number of hospital visits.
Also accepted was lusutrombopag (Mulpleo), which can be used to prevent excessive bleeding in adults with thrombocytopenia caused by long-standing liver disease. Patients with thrombocytopenia have a reduced number of platelets (components in the blood that help it to clot). Due to an increased risk of bleeding, patients may require platelet infusions when undergoing invasive procedures (for example endoscopy, liver biopsy, treatment for cancers etc). Lusutrombopag is an oral medicine that is taken prior to the procedure and can reduce the need for platelet transfusions.
The committee was unable to accept trabectedin (Yondelis) for the treatment of advanced soft-tissue sarcoma, a type of cancer that develops from the soft, supporting tissues of the body. Despite consideration through PACE, the committee was unable to accept the medicine because the company’s evidence around the clinical and cost effectiveness of treatment was not clear.
SMC Chairman Dr Alan MacDonald said:
“I’m pleased we were able to accept these four new medicines for use by NHSScotland. As we heard through our PACE process, patients with polycythaemia vera can suffer significant symptoms and treatment options are limited. We know that our decision on ruxolitinib will be welcomed.”
“For patients with hereditary angioedema, the way current preventative treatments must be given can be cumbersome. Our decision on lanadelumab provides a treatment which helps to prevent attacks, can help reduce anxiety and gives patients the option to self-administer rather than go to hospital to receive treatment.
“Olaparib can delay the time before patients require further chemotherapy and may offer increased survival benefits, giving patients valuable additional time with family and friends.
“For those with thrombocytopenia caused by long-standing liver disease, lusutrombopag provides a more convenient option for reducing the risk of bleeding during invasive procedures.
“Although the PACE process gives our committee members additional flexibility in their decision making, they were unable to accept trabectedin (Yondelis) for the treatment of advanced soft-tissue sarcoma as the company’s evidence around the clinical and economic benefits of using the medicine were not clear.”