July 2015 decisions news release

The Scottish Medicines Consortium (SMC), which reviews newly licensed medicines, has today published advice that includes five new medicines accepted for use in NHSScotland.

Two of these medicines, sorafenib (Nexavar) for thyroid cancer and riociguat (Adempas) for pulmonary arterial hypertension were accepted after consideration under the SMC’s 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.

The committee was unable to recommend two other medicines considered under the PACE process, olaparib (Lynparza) for ovarian cancer and vinflunine (Javlor) for cancer of the bladder and the urinary tract.

Sorafenib is used to treat an extremely rare type of cancer called differentiated thyroid carcinoma (DTC). DTC symptoms can include breathing difficulties and problems swallowing, which can be profoundly debilitating and frightening for patients and their families. Sorafenib was considered under both PACE and the ultra orphan decision making process, which 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. Through PACE, patient groups and clinicians highlighted that, as no alternative treatment options are currently available, sorafenib can be life changing and possibly even life saving for patients as it offers the potential to relieve the most frightening symptoms of DTC, improve pain management and enhance both the quality and quantity of life.

Also accepted after a PACE meeting was riociguat, the first of a new class of medicines used to treat pulmonary arterial hypertension (PAH). PAH is a disease where narrowing of the blood vessels leads to increased pressure in the main artery to the lungs, making physical activity of any kind difficult. In the PACE meeting, patient groups and clinicians stated that the onset of PAH often occurs in people in the 40 – 50 age group who play key roles as carers of children and/or breadwinners. PAH can be extremely debilitating and has a major impact on quality of life. There are limited treatment options for this condition and surgical intervention is not always possible or successful. Riociguat offers patients another treatment option which may help to improve their quality of life. The committee accepted riociguat for initiation and prescribing by specialists in the Scottish Pulmonary Vascular Unit or similar specialists.

Vedolizumab (Entyvio) is indicated for use in moderate to severe Crohn’s disease, a condition that causes inflammation of the gut wall. Patients with Crohn’s disease may experience a range of symptoms including pain due to ulceration and inflammation in the gut, and diarrhoea which can affect their self esteem and quality of life. Vedolizumab suppresses the gut immune system. It works differently to other treatments and potentially may have fewer side effects than other currently available treatments called TNF alpha inhibitors, which can have an effect on the whole immune system. The committee accepted vedolizumab for the treatment of adult patients who have not responded to treatment with TNF alpha inhibitors.

Tinzaparin (Innohep) is used to treat venous thrombo-embolism (VTE) a term which includes two conditions, deep vein thrombosis (affecting lower limbs) and pulmonary embolism (affecting the lungs). VTE develops when a blood clot forms in the vein. The risk of developing VTE is increased in cancer patients with solid tumours, which can be devastating for patients and their families who are already dealing with a cancer diagnosis and consequent treatment. Tinzaparin provides a cost effective alternative option to current treatment which may allow clinicians to better tailor treatment to individual patients.

The committee also accepted the antibiotic medicine ceftobiprole (Zevtera), for the treatment of hospital-acquired pneumonia caused by certain bacteria (such as MRSA) when other appropriate antibiotics cannot be tolerated by the patient.

Olaparib (Lynparza) is used in the treatment of advanced ovarian cancer in some patients who have a BRCA gene mutation. It was considered under the PACE and ultra orphan decision making processes. Patient groups and clinicians involved in PACE highlighted that this is the first medicine to target a population of patients with ovarian cancer according to their genetics. The committee felt there were a number of uncertainties around the overall survival benefits of olaparib and that the health benefits may not justify the cost to NHSScotland. Despite applying the added flexibility of PACE, the ultra-orphan framework, the modifier relating to the lack of treatment alternatives, and a Patient Access Scheme that had been accepted as suitable for implementation in NHSScotland, the committee was unable to accept olaparib for use.

The committee was also unable to recommend vinflunine (Javlor) for the treatment of advanced or metastatic transitional cell carcinoma of the urothelial tract (TCCU). TCCU is a type of cancer that affects the lining of the bladder and the urinary tract. During the PACE meeting participants highlighted that there have been no new treatments for bladder cancer in 30 years and that the potential for increased overall survival offered by vinflunine would be welcomed by patients. Vinflunine also met one of the SMC’s modifying criteria as there are no other treatments of proven benefit for TCCU. However, despite this added flexibility, the committee felt there were weaknesses around the overall costs in relation to health benefits.

The other medicine the committee was unable to recommend was rivaroxaban (Xarelto), a blood thinning medicine used to prevent blood clots in patients with acute coronary syndrome (ACS), a condition caused by a shortage of blood supply to the heart. SMC did not recommend rivaroxaban as it did not offer value for money when compared to a commonly used comparator treatment in NHSScotland.

Professor Jonathan Fox, chairman of SMC, said:

“SMC is pleased to be able to accept five medicines for a variety of conditions that will benefit patients in Scotland. Two of these medicines were considered through our PACE process, and patient groups and clinicians gave powerful testimony on the benefits of sorafenib for thyroid cancer and riociguat for pulmonary arterial hypertension.”

“We are disappointed not to be able to recommend olaparib for use in ovarian cancer and vinflunine for bladder cancer. Despite the powerful additional input from patient groups and clinicians through PACE, we were unable to accept these two medicines due to uncertainty about their clinical benefits in the context of their costs. We know these decisions will disappoint patients and clinicians alike as we understand the devastating impact of these conditions."

“While the PACE process is a determining factor and was designed to increase access to such medicines, that access cannot come at any price – we have to consider value for money and take account of the needs of all patients, not only those affected by the medicine under consideration. NHSScotland does not have infinite resources.”