Two of these medicines, sorafenib (Nexavar) for liver cancer and tolvaptan (Jinarc) for Autosomal Dominant Polycystic Kidney Disease (ADPKD) were accepted after consideration under SMC’s PACE (Patient and Clinician Engagement) process. PACE aims to improve patient access to new medicines for the treatment of end of life and very rare conditions.

 

Sorafenib is used to treat a type of liver cancer called hepatocellular carcinoma, which affects a small number of patients. During the PACE meeting, it was highlighted by patient groups and clinicians that this cancer is often diagnosed at an advanced stage and patients have a poor prognosis. Sorafenib, which is taken orally, is the only treatment with evidence of benefit in patients who are not suitable for surgical treatment and SMC accepted it for use in these patients. 

 

Tolvaptan is used to treat the inherited kidney disease ADPKD, where fluid filled cysts grow in the kidneys causing a loss of kidney function. In the PACE meeting, it was noted that symptoms generally first appear in a relatively young patient group who are often working or raising children. Tolvaptan is the first medicine to address the underlying causes of ADPKD. It can help to slow the rate of decline in kidney function thus delaying the need for kidney dialysis. There are currently no other licensed treatment options for ADPKD. 

 

Also accepted was netupitant/palonosetron (Akynzeo), a combination of two medicines which can be used to prevent and treat nausea brought on by cancer chemotherapy. Netupitant/palonosetron offers another option for symptom control in this patient group. 

 

The committee also accepted two injectable medicines for the treatment of type 2 diabetes, dulaglutide (Trulicity) and albiglutide (Eperzan). Patient groups highlighted that both medicines are once weekly treatments which may be beneficial for some patients.  

 

Professor Jonathan Fox, chairman of SMC, said:

 

“I am pleased the committee has been able to accept these five medicines for routine use in NHSScotland. We know from the testimony given by patient groups and clinicians at the PACE meetings that sorafenib for liver cancer and tolvaptan for ADKPD will be welcomed. The patient group contribution played an important part in helping the committee reach its decisions on these medicines.”