May 2018 decisions news release

The Scottish Medicines Consortium (SMC), which advises on newly licensed medicines for use by NHSScotland, has today (Monday 7 May) published advice accepting five medicines. Four of these medicines were accepted through SMC’s Patient and Clinician Engagement (PACE) process, which is used to consider medicines used to treat end of life and very rare conditions.

Nusinersen (Spinraza) was accepted for the treatment of infantile onset 5q spinal muscular atrophy (type 1 SMA). SMA is a very rare genetic disease that results in devastating muscle wasting that can cause patients to lose the ability to move, breathe and swallow. In type 1 SMA, symptoms usually start shortly after birth, and affected children rarely survive beyond two years of age. The committee was unable to recommend nusinersen for the treatment of type 2 and 3 SMA.

Avelumab (Bavencio) was accepted for the treatment of Merkel cell carcinoma (MCC), an aggressive, rare form of skin cancer. The PACE meeting heard that MCC can cause disfiguring tumours which can be distressing for both patients and their families. The current treatment is chemotherapy, which is not suitable for many patients and is often poorly tolerated. Additionally, patients often relapse quickly with chemotherapy. Avelumab may shrink visible tumours and offers the potential to significantly improve outcomes for patients through longer responses and reduced toxicity compared to chemotherapy.

Regorafenib (Stivarga) was accepted for the treatment of advanced liver cancer in patients who have already undergone previous treatment. PACE participants spoke of how regorafenib can offer increased life expectancy in the context of a limited overall survival time, something which can be very important for both patients and their families. As an oral medicine, it also reduces the burden of hospital visits.

Also accepted through PACE was selexipag (Uptravi). Selexipag can be used to treat pulmonary arterial hypertension (PAH, abnormally high blood pressure in the arteries of the lungs). Patient groups and clinicians told the PACE meeting that patients are often disabled by breathlessness and fatigue, unable to work and require significant care from their families. Current treatments often involve an inhaled medication (using a nebuliser), which is administered frequently throughout the day and can be difficult to use, carry and maintain. Selexipag is an oral treatment taken twice daily, giving patients greater freedom to lead a regular daily routine.

The SMC Committee also accepted brodalumab (Kyntheum) for the treatment of severe plaque psoriasis, an inflammatory skin condition which causes red scaly patches (known as plaques) on the skin. Brodalumab extends the range of disease modifying treatment options for patients who have failed to respond to standard therapies.

SMC Chairman Dr Alan MacDonald said:

“I am pleased the committee was able to accept these five medicines for use by NHSScotland.”

“The committee heard compelling evidence on the benefits of nusinersen, supported by the views of experts and patient groups and was able to apply a very high degree of flexibility in accepting this medicine for type 1 SMA. Unfortunately, the evidence presented suggested that nusinersen was substantially less cost effective when used in types 2 and 3 and therefore could not be accepted by the committee. These are incredibly difficult decisions. The medicine is extremely expensive and there is a need to consider all those who need treatment by NHS Scotland. We would welcome a resubmission from the company which addresses the issues raised.”

“For those with MCC, avelumab can help improve overall outcomes without the need for chemotherapy, and also offers the chance to shrink visible tumours which are distressing for patients.”

“Regorafenib for liver cancer offers patients important additional survival time. As an oral treatment it also reduces the need for hospital visits at what can be a difficult time for both them and their families.”

“Selexipag can help reduce the burden of treatment for those with PAH, while brodalumab offers an important additional treatment option for those with plaque psoriasis.”

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