Cost regulators in Scotland have approved NHS funding for four new medicines, expanding treatment options for lung cancer, iron excess, hepatitis C and chronic pain, but Janssen's Darzalex was rejected for multiple myeloma.
MSD's Keytruda (pembrolizumab) was accepted for the treatment of late-stage non-small cell lung cancer (NSCLC) after being considered through SMC's Patient and Clinician Engagement (PACE) process, designed for appraising medicines used at the end of life and for very rare conditions.
During the PACE meeting, patient groups and clinicians highlighted the severe symptoms associated with late-stage NSCLC and the need for treatments that are better tolerated than those currently available. Keytruda can offer patients around two months additional survival time which is valuable in the context of limited life expectancy, the SMC noted.
The drugmaker's Zepatier (elbasvir-grazoprevir) was also accepted, for the treatment of chronic Hepatitis C. The regulator noted that in patients with genotype 1a, 1b or 4, the treatment significantly increased sustained virologic suppression compared with a regimen containing a non-structural protein 5B (NS5B) inhibitor, an interferon and ribavirin.
Elsewhere, Novartis' Exjade (deferasirox) was endorsed as a means of removing excess iron in the body because of frequent blood transfusions in some patients with a rare blood disorder known as Myelodysplastic Syndrome (MDS), which can cause heart, liver and kidney problems.
Patient groups and clinicians highlighted that the only other available treatment is given via an infusion pump worn overnight or under clothes for eight hours at a time on five to seven days every week; Exjade offers an oral alternative.
The Committee also accepted a Qdem Pharmaceuticals' skin path Butec (buprenorphine) for use in people over 65 years old with chronic pain that is not associated with cancer. Chronic pain is difficult to manage effectively and patch, which is applied to the skin once a week, offers an alternative to oral treatment options, the SMC noted.
Meanwhile, Allergan's antibiotic Xydalba (dalbavancin) is to be funded for the treatment of acute bacterial skin and skin structure infections in adults. The medicine was actually accepted by the Committee in October 2015, but the decision could not be published until the medicine was made available for use in NHS Scotland.
On the downside, the SMC was unable to accept Janssen's Darzalex (daratumumab) for the treatment of patients at an advanced stage of white blood cell cancer multiple myeloma, despite it having been considered through the PACE process. The Committee said it was concerned that uncertainties in the company's evidence on its benefits meant it would not be a good use of NHS resources.
Janssen said it is disappointed with the decision. "The rare and aggressive nature of multiple myeloma and the lack of effective and well-tolerated treatment options for patients who have been previously treated and become resistant to other therapies means that there is a real and urgent need for new options like daratumumab," noted Jennifer Lee, director of Health Economics, Market Access and Reimbursement (HEMAR) and Advocacy at Janssen UK. "We will continue to work collaboratively with the SMC and remain committed to finding a solution so that patients and doctors in Scotland can access the medicines they need."
In a further blow to patients with the disease, the SMC was also unable to accept Kyprolis (carfilzomib), as it was not satisfied that the evidence about the overall survival benefits offered by the drug was strong enough to justify its cost to the NHS.
Myeloma UK said it made a strong case to the regulator for the approval of both indications and is "very disappointed" by the decision not to reject their routine use.
"Despite the welcome approval of new drugs in recent years, there is still an urgent need for new myeloma treatments; especially for relapsed patients who have exhausted other standard treatment options such as Velcade and Revlimid," noted the charity's Policy and Public Affairs Manager Kate Morgan.
"Kyprolis and Darzalex are effective new treatments which can deliver real benefits in improving survival and quality of life. We will continue to work with the companies, and contribute to future appraisals involving these two drugs, to help deliver access for patients."