The Scottish Medicines Consortium (SMC) has published advice accepting four new medicines; Akcea’s Tegsedi (inotersen), Abbvie’s Venclyxto (venetoclax), Camurus’ Buvidal (buprenorphine) and Almirall’s Ilumetri (tildrakizumab).

The committee, which advises on newly licensed medicines for use by NHSScotland, firstly accepted Akcea’s Tegsedi for the treatment of polyneuropathy (a form of nerve damage) in adults with hereditary transthyretin amyloidosis (hATTR).

The medicine was accepted for the disorder, a very rare progressive genetic disease in which abnormal proteins build up in tissues around the body including the nerves, 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.

SMC chairman, Dr Alan MacDonald explained that “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.”

Secondly, the consortium accepted Abbvie and Roche’s Venclyxto, a drug predicted to become a blockbuster by 2020, for use in combination with rituximab, in patients who had received at least one previous treatment for chronic lymphocytic leukaemia (CLL).

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.

The acceptance is good news for those affected, because there is an unmet need for this patient group as current treatment options on repeated relapse cause significant side effects.

Camurus’ Buvidal (buprenorphine) was also accepted by the organisation for the treatment of dependence on opioid (narcotic) drugs such as heroin or morphine in those patients not suitable for treatment with methadone.

The group made the decision citing that ‘treatment with Buvidal 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 Buvidal.’

Also accepted was Illumetri (tildrakizumab), an Almirall drug for moderate to severe plaque psoriasis in adults. The treatment offers another treatment option for patients who have failed to respond to standard therapies.

Dr MacDonald said that he is “pleased the committee was able to accept these four new medicines for use by NHSScotland,” going on to explain that “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.”

However, the committee was unable to accept two medicines for the treatment of cystic fibrosis following consideration through PACE; Orkambi (lumacaftor-ivacaftor) and Symkevi (tezacaftor-ivacaftor), both drugs by Vertex.

The rise of Orkambi to the market has been a controversial process, as the company has refused to drop the staggering price of £104,000 per patient per year to make it affordable to the NHS. The pricing means that The National Institute for Health and Care Excellence (NICE) has already deemed it not cost effective, and the SMC cited its rejection reasons as ‘uncertainties in the company’s evidence about the long term health benefits of both medicines in relation to their costs’.

Vertex responded to the ‘disappointing’ news, saying that since learning of the decision the company has been “working with the Scottish Government and other interested parties to develop a solution to allow broad access for all eligible patients in Scotland with CF.”

The spokesman continued, “We are all deeply committed to reaching such a solution as a matter of urgency and we are working closely with the Government to finalise it over the coming weeks.

“While this work continues, Vertex will continue to provide access to our medicines via the PACS Tier 2 process, until a broad access agreement is in place.”

The decisions were announced in the committee’s monthly meeting on August 12th.