NHS Scotland's August 2019 Funding Decisions

Nicky Broyd

August 12, 2019

The Scottish Medicines Consortium (SMC) has this month turned down two medicines for treating cystic fibrosis and approved new drugs for hereditary transthyretin amyloidosis, chronic lymphocytic leukaemia, plaque psoriasis, and opioid dependence.

Cystic Fibrosis

Despite consideration using the PACE process (Patient and Clinician Engagement for rare and end of life care), two medicines for cystic fibrosis were rejected.

  • Lumacaftor-ivacaftor (Orkambi, Vertex Pharmaceuticals) is used for some patients aged 2 and older.

  • Tezacaftor-ivacaftor (Symkevi, Vertex Pharmaceuticals) is used for some patients aged 12 and older.

The assessment teams heard how the treatments had benefits on symptoms and reduced hospital admissions. However, the SMC said the rejection was due to uncertainties on the long-term health benefits in relation to their costs.

In England, the National Institute for Health and Care Excellence (NICE) has also been unable to recommend Orkambi on cost grounds since 2016. The Department of Health and Social Care began new talks with the manufacturer in March. The head of the company told the Commons Health and Social Care Committee that England had already been offered "the best price in the world" for the drug.

SMC Chairman Dr Alan MacDonald said: "We recognise that the decisions on lumacaftor-ivacaftor and tezacaftor-ivacaftor will be disappointing. We understand the profound impact that cystic fibrosis has on patients and their families and we are aware of the need for effective treatments that target the underlying cause. Patient groups and clinicians gave powerful testimonies about the impact of the condition and the potential benefits of these medicines.

"As these are orphan medicines, our committee members were able to apply additional flexibility in their decision making, but there remained significant uncertainty around their overall health benefits in the long-term in relation to their costs. In order to be able to accept these medicines the committee will need to be satisfied of their cost-effectiveness and we continue to work with the company to achieve that."

The Cystic Fibrosis Trust said the conclusion was disappointing not just for people in Scotland, but across the UK, where it had been hoped a positive decision by the SMC would demonstrate to the other three nations that a deal could be done.

David Ramsden, chief executive at the Cystic Fibrosis Trust, said: "The SMC’s advice will come as a shock to people with cystic fibrosis and their families in Scotland. While talk of further negotiation between Vertex Pharmaceuticals and the Scottish Government is positive, people living in Scotland need these life-saving drugs now. The tragic wait has to end and now is the time for the Scottish Government to remain committed and Vertex to do all they can to ensure that this chance does not slip away again."

Hereditary Transthyretin Amyloidosis (hATTR)

Inotersen (Tegsedi, Akcea Therapeutics) was accepted for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis.

The decision was made under the PACE process which heard about symptoms including loss of sensation, weakness, muscle wasting, and worsening disability affecting quality of life.

In a statement, SMC chair Dr MacDonald, said: "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."

Chronic Lymphocytic Leukaemia

Venetoclax (Venclyxto, AbbVie) was accepted for use in combination with rituximab (MabThera, Roche, and others) in patients who had received at least one previous treatment for chronic lymphocytic leukaemia (CLL).

The PACE process highlighted the need for treatments with fewer side effects during periods of symptom remission.

Venetoclax can offer increased time to progression of CLL, the SMC said.

Dr MacDonald said: "For patients with chronic lymphocytic lymphoma, venetoclax offers the chance of more good quality time with family and friends before the cancer returns, helping to ease the psychological burden patients can feel due to the relapsing and remitting nature of the disease."

Opioid Dependence

Buprenorphine (Buvidal, Camurus) was accepted for the treatment of opioid dependence in patients unsuitable for methadone treatment.

Buprenorphine is given by subcutaneous injection and lasts months or weeks compared with current daily treatments.

Along with other medical, social, and psychological treatment, the SMC said buprenorphine may enable patients to focus on recovery and returning to normal routines without the need for daily visits to a pharmacy.

Dr MacDonald said: "Buprenorphine may help those undergoing treatment for opioid dependence with their recovery as part of a wider programme of support."

Plaque Psoriasis

Tildrakizumab (Ilumetri, Almirall) was accepted for the treatment of moderate to severe plaque psoriasis in adults who have failed to respond to standard therapies.

Dr MacDonald said: "Our decision on tildrakizumab for plaque psoriasis means another treatment option is available for patients who have failed to respond to previous therapies."


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