NHS Scotland's 'Landmark' Deal for Cystic Fibrosis Drugs

Nicky Broyd

September 12, 2019

NHS Scotland reached an agreement in the long-running discussions over prescribing the cystic fibrosis drug ivacaftor-lumacaftor (Orkambi, Vertex Pharmaceuticals) and ivacaftor-tezacaftor (Symkevi, Vertex Pharmaceuticals). Just last month they had been rejected by the SMC due to uncertainties on the long-term health benefits in relation to costs.

Scotland's health secretary, Jeane Freeman, said in a statement: "This is fantastic news for those living in Scotland with cystic fibrosis and their families. This new pricing agreement with medicines manufacturer Vertex Pharmaceuticals, secured by the Scottish Government, will allow more people with cystic fibrosis to benefit from medicines Orkamb and Symkevi and live fuller lives for longer.

"The agreement has been reached after extensive discussions between the Scottish Government and Vertex Pharmaceuticals and means the medicines will now be made available to patients on the NHS in Scotland, subject to a confidential discount."

The agreement is for 5 years and is the first in the UK.

Reacting to the decision in a statement, David Ramsden, chief executive of the Cystic Fibrosis Trust, said: "This is a landmark moment for the hundreds of people with CF and their families across Scotland who have tirelessly campaigned for years to access these drugs. This breakthrough is a victory for their perseverance and enduring hope. It means 350 eligible people living in Scotland will have access to drugs that stabilise their lung health and reduce the need for hospital admissions.

"We celebrate the news in Scotland today, but our campaign must continue to focus on Vertex and all parties in England, Wales and Northern Ireland. Those in need of the drugs have already waited too long and we must ensure that thousands more people are not subjected to a postcode lottery. Scotland’s success must now be replicated across the UK without further damaging delay."

SMC Decisions

Earlier this week the Scottish Medicines Consortium (SMC) announced that it had accepted five new medicines for lymphoma, non-small cell lung cancer, type 1 diabetes, and vulvovaginal atrophy.

A treatment for insomnia in children, and another NSCLC treatment, were rejected.

Diffuse B Cell Lymphoma

CAR-T cell therapy tisagenlecleucel (Kymriah, Novartis) was approved to treat adults with diffuse B cell lymphoma who have relapsed or not responded after two previous treatments.

The once-only treatment modifies the patient’s own immune cells to destroy cancer cells with a long-term response or cure possible.

The decision involved the Patient and Clinician Engagement (PACE) process for end of life and rare conditions.

Poor prognosis and relatively short survival times were highlighted.

SMC Chairman Dr Alan MacDonald said in a statement: "From the testimonies given by patient groups and clinicians through our PACE process, we know that treatment for diffuse B cell lymphoma at this stage of the condition impacts heavily on both patients and their carers. We hope that our decision on tisagenlecleucel, as a potentially curative medicine, will be welcomed."

Non-small Cell Lung Cancer (NSCLC)

Pembrolizumab (Keytruda, Merck Sharp & Dohme) for metastatic squamous non-small cell lung cancer (NSCLC) was also approved via PACE, citing limited survival with current treatments.

Pembrolizumab, in combination with chemotherapy, can extend lives and keep patients healthier for longer.

Dacomitinib (Vizimpro, Pfizer) was approved for advanced NSCLC whose cancer cells have a defect in the gene that makes a protein called epidermal-growth-factor receptor (EGFR).

Osimertinib (Tagrisso, AstraZeneca) was rejected for this type of NSCLC.

The SMC said this was due to uncertainty over benefits and costs.

Dr MacDonald said: "For those with metastatic NSCLC, pembrolizumab in combination with chemotherapy can give patients the chance of some valuable extra time with their families in the context of this incurable illness.

"Dacomitinib for the treatment of NSCLC in those with a defect in the EGFR gene offers a valuable additional treatment option.

"Unfortunately we were unable to accept osimertinib for the same condition as the company’s evidence around the medicine’s cost effectiveness was not strong enough."

Osimertinib has also been turned down for use within NHS England. AstraZeneca has said it will appeal against the NICE (National Institute for Health and Care Excellence) decision.

Type 1 Diabetes

Dapagliflozin (Forxiga, AstraZeneca) was accepted for patients with type 1 diabetes in combination with insulin for better diabetes control and reduced risk of complications.

Dr MacDonald said: "For those with diabetes type 1, dapagliflozin, used in combination with insulin, may allow better management of their condition."

Vulvovaginal Atrophy

The oral treatment ospemifene (Senshio, Shionogi) was approved to treat vulvovaginal atrophy after menopause in women unable to use topical therapies.

Dr MacDonald said: "Ospemifene provides a treatment option for those unable to use current therapies for vulvovaginal atrophy."

Insomnia in Children

Prolonged-release melatonin (Slenyto, Flynn Pharma) was rejected for insomnia in children with autism spectrum disorder and/or Smith-Magenis syndrome.

Dr MacDonald said: "We were unable to accept prolonged-release melatonin as the evidence around its cost effectiveness was not strong enough when compared to other current preparations."

Editor's Note: This article was updated to include the cystic fibrosis announcements.

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