Following consideration through PACE, the committee was unable to accept two medicines for the treatment of cystic fibrosis, an inherited disease that has severe effects on the lungs and the digestive system. Lumacaftor-ivacaftor (Orkambi) is used to treat the condition in some patients aged two and older. Tezacaftor-ivacaftor (Symkevi) is used to treat some patients aged 12 and older. In the PACE meetings, patient groups and clinicians emphasised the potential benefits of both medicines on patients’ symptoms, reducing the need for hospital admissions and the overall burden of care. Despite strong evidence from patient groups and the additional flexibility PACE gives committee members in the decision making process, the committee was unable to accept either lumacaftor-ivacaftor or tezacaftor-ivacaftor. This was due to uncertainties in the company’s evidence about the long term health benefits of both medicines in relation to their costs.
Inotersen (Tegsedi) was accepted for the treatment of polyneuropathy (a form of nerve damage) in adults with hereditary transthyretin amyloidosis (hATTR). hATTR amyloidosis is a very rare progressive genetic disease in which abnormal proteins called amyloid build up in tissues around the body including the nerves. The medicine was accepted following consideration 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 spoke of multiple symptoms associated with the disease, including loss of sensation, weakness and muscle wasting. The severe and worsening disability experienced by patients affects all areas of life for them, their families and carers. As the condition is hereditary it is possible more than one family member is affected, adding to the burden of the patient and carer. Inotersen provides an opportunity to halt or slow the progression of the disease, potentially leading to a better quality of life.
Venetoclax (Venclyxto) was accepted for use in combination with another cancer medicine called rituximab, in patients who had received at least one previous treatment for chronic lymphocytic leukaemia (CLL, a type of blood cancer). In the PACE meeting, patient groups and clinicians highlighted that patients experience periods of remission where the leukaemia cannot be detected in the body and there are no symptoms and periods of relapse when the cancer returns and treatment is required. There is an unmet need for this patient group as current treatment options on repeated relapse cause significant side effects. Living with CLL can carry a considerable psychological burden, particularly as patients often have to “watch and wait” for the cancer to return. Venetoclax can offer increased time to progression of CLL.
Buprenorphine (Buvidal) was accepted for the treatment of dependence on opioid (narcotic) drugs such as heroin or morphine in those patients not suitable for treatment with methadone. Treatment with buprenorphine as a subcutaneous injection provides a longer acting treatment than current available treatments, being given monthly or weekly compared to daily doses of oral forms of buprenorphine. When given within a framework of medical, social and psychological treatment, buprenorphine subcutaneous injections may enable patients to focus on recovery and returning to normal daily routines without the daily visits to a pharmacy to receive treatment.
Also accepted was tildrakizumab (Ilumetri) for the treatment of moderate to severe plaque psoriasis (a disease causing red, scaly patches on the skin) in adults. Tildrakizumab offers another treatment option for patients who have failed to respond to standard therapies.
When a medicine is not accepted by SMC, all health boards have procedures in place to consider requests when a healthcare professional feels the medicine would be right for a particular person.
SMC Chairman Dr Alan MacDonald said:
“I am pleased the committee was able to accept these four new medicines for use by NHSScotland.”
“Hereditary transthyretin amyloidosis comes with multiple symptoms that affect every aspect of the lives of patients and their carers. From the evidence given through our PACE meeting we know our decision on inotersen will be welcomed, particularly as it can slow progression of the disease.”
“For patients with chronic lymphocytic lymphoma, venetoclax offers the chance of more good quality time with family and friends before the cancer returns, helping to ease the psychological burden patients can feel due to the relapsing and remitting nature of the disease.”
“Buprenorphine may help those undergoing treatment for opioid dependence with their recovery as part of a wider programme of support.”
“Our decision on tildrakizumab for plaque psoriasis means another treatment option is available for patients who have failed to respond to previous therapies.”
“We recognise that the decisions on lumacaftor-ivacaftor and tezacaftor-ivacaftor will be disappointing. We understand the profound impact that cystic fibrosis has on patients and their families and we are aware of the need for effective treatments that target the underlying cause. Patient groups and clinicians gave powerful testimonies about the impact of the condition and the potential benefits of these medicines. As these are orphan medicines, our committee members were able to apply additional flexibility in their decision making, but there remained significant uncertainty around their overall health benefits in the long term in relation to their costs. In order to be able to accept these medicines the committee will need to be satisfied of their cost-effectiveness and we continue to work with the company to achieve that.”