Drugs to treat a rare form of cancer and prevent infection following a stem cell transplant have been recommended for routine use on the NHS in Scotland.
The two drugs were accepted by the Scottish Medicines Consortium (SMC), which provides advice to NHS Scotland about the value of newly licensed medicines.
Liposomal daunorubicin/cytarabine, also known as Vyxeos, was accepted for treating a rare and aggressive blood cancer in adults, known as high risk types of acute myeloid leukaemia (AML).
It is the first new chemotherapy in four decades for these types of cancer and the SMC found it can improve remission rates and survival – giving an increased chance of a potential cure through stem cell transplant.
The treatment involves fewer doses and shorter hospital stays than current chemotherapy options for patients.
Dr Mark Drummond, consultant haematologist at the Beatson Cancer Centre, Glasgow, said the decision is an “important milestone”.
He said: “It is the first new chemotherapy in four decades for these types of high-risk AML that has demonstrated an improved overall survival when compared to a standard of care regimen.
“There has been a real need for new, effective treatment options as response rates to current therapies are substantially lower than those for other types of AML.”
The consortium also accepted Letermovir, also known as Prevymis, which is used to prevent stem cell transplant patients from developing a potentially serious infection.
Cytomegalovirus (CMV) infection can lead to serious illness and delay recovery following transplant but the drug can guard against this.
This is great news for stem cell transplant patients in Scotland
Scotland is the first part of the UK to recommend accepting the drug for routine use on the NHS.
Chloe Anthias, medical director of Anthony Nolan – which operates a potential stem cell donor register, said: “This is great news for stem cell transplant patients in Scotland.
“The current treatments for CMV reactivation can have a very negative effect on quality of life, but the SMC decision represents a significant step forward.”
SMC chairman Dr Alan MacDonald said: “We are pleased to be able to accept these two new medicines for use by NHS Scotland.”
“Patients with high-risk acute myeloid leukaemia have a very poor prognosis and high unmet need.
“Liposomal daunorubicin/cytarabine offers an improvement in overall survival and may lead to a potentially curative stem cell transplant for some patients.
“For patients who have undergone a stem cell transplant, letermovir can aid their recovery by reducing the risk of CMV infection.”
He said the committee did not accept a third drug tisagenlecleucel, also known as Kymriah, used for adult patients with a type of lymph node cancer who have relapsed or not responded to previous treatments, as the “company’s evidence around its long-term benefits was not clear”.
Dr Alasdair Rankin, director of research and patient experience at Bloodwise, said it is a “disappointment” for patients as the treatment can offer a “last-chance lifeline”.
He said: “It is another setback for patients who are unable to access the same therapies as their counterparts in England.”