SMC accepts treatments for breast cancer, myeloma and ADHD

One of these medicines, fulvestrant (Faslodex) for advanced breast cancer, was accepted following consideration under the SMC’s Patient and Clinician Engagement Process (PACE), 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 accept eculizumab (Soliris) for atypical Haemolytic Uraemic Syndrome (aHUS). This is a rare, life threatening disease that causes abnormal blood clotting in small blood vessels and the breakdown of red blood cells resulting in kidney failure. It can also affect the heart, lungs, brain and digestive system. Eculizumab is an ultra-orphan medicine that was considered under the PACE process.

Fulvestrant is used to treat advanced breast cancer. During the PACE meeting, it was highlighted that fulvestrant can delay the time to chemotherapy for some patients. It provides an alternative treatment option for patients where chemotherapy is not appropriate or for patients unable to take oral medication due to swallowing difficulties.

Panobinostat (Farydak) is used to treat multiple myeloma, a rare cancer of the white blood cells. It offers a further treatment option to patients who have received at least two prior treatments.
Guanfacine hydrocholoride (Intuniv) was accepted for the treatment of attention deficit hyperactivity disorder (ADHD) in children and adolescents aged 6 – 17 years. Guanfacine provides an alternative for those patients who do not respond to or are unable to tolerate the side effects of currently available treatments.

Ulipristral acetate (Esmya) can be used to treat uterine fibroids (non-cancerous muscle tumours that develop in the wall of the womb). Fibroids affect women of reproductive age and ulipristal acetate can help some patients avoid or delay the need for surgery.

The Committee also accepted golimumab (Simponi) for axial spondyloarthritis, an inflammatory arthritis that affects the spine and the joints that lie at the junction of the spine and pelvis. Golimumab offers another treatment option for those patients who have not responded to or are unable to take other currently available treatments.

The Committee did not recommend eculizumab (Soliris) for routine use in aHUS. At the PACE meeting, patient groups and clinicians highlighted the potential benefits of eculizumab but, despite applying the considerably increased flexibility that this process allows, the committee was unable to accept eculizumab as it was not considered to represent an effective use of NHSScotland’s resources.

Professor Jonathan Fox, chairman of SMC, said:

"We are pleased to be able to accept five new medicines and hope that patients and their families will benefit from these decisions.

"We were disappointed that we were unable to accept eculizumab for routine use in aHUS. The Committee recognised the benefits it provides for patients with this condition.

"While the PACE process gives the Committee additional flexibility in order to increase access to medicines used at the end of life and for very rare conditions, that access cannot come at any price – we have to consider value for money and take account of the needs of all patients who need treatment, not only those affected by the condition under consideration.

"We understand that this decision will be very disappointing for patients. We would welcome a resubmission for eculizumab that takes into account the issues raised by the SMC appraisal."