Tag: Scottish Medicines Consortium

Charity welcomes Scotland’s approval of treatment on the NHS for children with rare brain tumours

Children in Scotland diagnosed with a brain tumour that has a specific genetic mutation can now be treated on the NHS with a kinder, more effective targeted therapy. 

The Scottish Medicines Consortium has today approved prescribing dabrafenib to treat children aged one and above who have been diagnosed with a low- or high-grade glioma that has the BRAF V600E mutation: https://scottishmedicines.org.uk/medicines-advice/dabrafenib-finlee-full-smc2667/

It’s used in combination with another medicine, trametinib, and both can be administered at home as they are taken orally. This means fewer hospital visits. 

The news follows the approval given in April this year by the National Institute for Health and Care Excellence (NICE). 

Current standard treatment for the condition typically involves surgery – where that’s possible – chemotherapy and/or radiotherapy. But less than 30% of children diagnosed with high-grade gliomas survive for five years or more and the treatment often has brutal side effects.  

Outcomes for low grade gliomas – the most common childhood brain tumour – are better at a 90% survival rate following surgery. But chemotherapy may also be prescribed whereas this treatment means improved quality of life for patients as clinical trials showed its side effects were rare.  

The combination medication is the first targeted treatment to be made available on the NHS for paediatric gliomas. It is already licensed for use in the USA, and for use in the UK in adults with the BRAF mutation who have some types of lung and skin cancers. 

The two drugs work together to block the growth signal coming from the mutant BRAF protein, slowing or even stopping the tumour from growing.  

Brain tumours affect around 500 children a year in the UK, of whom between 150 to 175 may have a glioma and fewer still will have the BRAF mutation. But despite the small numbers involved, this is the first progress made in treating the disease in years. 

In 2008, research carried out by Dr David Jones which was funded by The Brain Tumour Charity (then known as the Samantha Dickson Brain Tumour Trust), identified a BRAF mutation that was common in pilocytic astrocytoma brain tumours. 

This was the first time that a specific genetic change had been detected in these tumour types and it paved the way for drug development as it could then become a target for diagnostic tests, changing the research landscape for low grade brain tumours in children. 

Dr Jones now leads the EVEREST Centre in Germany, which carries out research into paediatric low grade brain tumours. 

Dr Michele Afif, Chief Executive at The Brain Tumour Charity, said: “We are delighted that the Scottish Medicines Consortium has joined NICE in approving the first new treatment for paediatric brain tumours in decades.

“We hope that this will be the first of many new treatments that will ensure our community can live longer and better lives.” 

To find out more about The Brain Tumour Charity, visit:

www.thebraintumourcharity.org/]www.thebraintumourcharity.org/ 

Scotland approves two new blood cancer drugs

Two new drugs for diffuse large B-cell lymphoma (DLBCL) have been made available by the Scottish Medicines Consortium (SMC) for use on the NHS in Scotland as a third-line treatment.

Tepkinly (epcoritamab), and Columvi (glofitamab) will be available for use in patients with relapsed or refractory DLBCL after two or more lines of systemic therapy.

Previously, NICE approved Tepkinly and Columvi for use on the NHS in England and Wales for people with DLBCL who’s disease had not responded to at least two previous treatments.

DLBCL is a type of non-Hodgkin lymphoma, which develops in the lymphatic system. Around 5,500 people a year are diagnosed with this form of blood cancer in the UK. And for around 700 of those people, their disease won’t respond to two previous treatments.

Tepkinly is a drug, manufactured by Abbvie, while Columvi is manufactured by Roche. They are both bispecific antibodies designed to recognise and attach to the cancer cells and immune cells, so that the body’s immune system can destroy them.

Josh Hill, Blood Cancer UK’s Scottish policy officer said: “Diffuse large B-cell lymphoma (DLBCL) is a type of blood cancer and while most people go into remission after standard treatment, some people can see their cancer return or stop responding to treatment.

“Today’s decision by the SMC to approve epcoritamab and glofitmab for use on the NHS in Scotland is a welcome step for many.

“Like many that experience blood cancer, people with relapsed or refractory DLBCL experience anxiety around the prospect of not responding to treatment or the cancer once again returning. Advances in bispecifics is set to be an active area of research and drug development in coming years.”

Dr Andrejs Ivanovs, consultant haematologist at the Edinburgh Cancer Centre, said: “The clinical evidence from drug trials using bispecific antibodies including epcoritamab and glofitimab is positive.

“These drugs are incredibly useful as they’re available ‘off-the-shelf’ meaning drugs can be used without delay for lymphoma patients who did not have a lot of success with other treatments. 

“Bispecific antibody treatments usually do not require a hospital admission and can be administered in day units and can achieve response in about 50-60% of patients. This is likely to prolong survival rates for people with blood cancer here in Scotland, and that’s exactly what people with blood cancer deserve.”

Aileen Lamb, from Edinburgh whose now in remission from blood cancer said:
“As someone who’s been through blood cancer treatment, I am thrilled that the SMC have given full approval for use of epcoritamab and glofitmab to treat diffuse large B-cell lymphoma in Scotland.

“This is important for the whole blood cancer community and gives us hope for our future.”

Treatment for Duchenne Muscular Dystrophy accepted by Scottish Medicines Consortium

·       A drug called Translarna can help treat an underlying genetic cause of Duchenne muscular dystrophy

·       Muscular Dystrophy UK, Action Duchenne, Duchenne Family Support Group, and Duchenne UK appeared before the Scottish Medicines Consortium in March

·       The Scottish Medicines Consortium has accepted the drug for use on NHSScotland over the next three years

·       The drug company must now submit a plan to Scottish Government before it can be given on NHS Scotland

A DRUG that can treat an underlying genetic cause of a muscle-wasting condition has been accepted for use on NHSScotland, it has been announced.

This will pave the way for the drug to be made available across the next three years, through a system called the ‘ultra-orphan pathway’.

The news comes after Muscular Dystrophy UK, Action Duchenne, Duchenne Family Support Group and Duchenne UK appeared before the Scottish Medicines Consortium (SMC) last month, following an earlier joint submission to accept Translarna for use on NHSScotland.

The drug can help treat patients with an underlying genetic cause of Duchenne muscular dystrophy (DMD), called nonsense mutations. This condition primarily affects boys and causes muscles to weaken and waste over time, with most of those affected needing to use a wheelchair by the age of 12. The life-limiting disease also causes other severe health problems to the heart and breathing muscles.

In yesterday’s announcement, the SMC said it accepts that Translarna meets the definition of what is known an ultra-orphan medicine, used to treat extremely rare conditions.

It means that eligible DMD patients aged two or over and who are able to walk should be able to have the treatment on NHSScotland under the ultra-orphan pathway for at least the next three years. This is provided that the company manufacturing Translarna submits a data collection plan to the Scottish Government.

The plan should outline how further data, including evidence outlining the experiences of patients and carers, will be collected over the next three years. At the end of this period, the drug company should provide the SMC with an updated submission for reassessment. The SMC will then review the evidence before deciding if Translarna can be routinely used on NHSScotland.

In the announcement, the SMC acknowledged that Translarna “may allow affected children to retain the ability to walk for longer, potentially leading to increased independence”.

More information can be read here.

Provided that the drug company has submitted its plans to the Scottish Government, Translarna will be given in addition to the current standard treatments on NHSScotland.

Families and individuals should speak with their clinician to find out more about eligibility. More information will be shared with relevant patient groups accordingly.

A spokesperson on behalf of the four charities said: “Muscular Dystrophy UK, Action Duchenne, Duchenne Family Support Group and Duchenne UK are delighted the SMC has accepted Translarna for use on the ultra-orphan pathway for the next three years.

“Today’s announcement is excellent progress in our bid to help those with Duchenne muscular dystrophy access appropriate treatment in Scotland, especially because every day is a race against time to slow down the condition.

“We believe that the physical and mental health benefits of Translarna have the potential to improve lives for so many families living with Duchenne. The four charities are therefore very much looking forward to hearing the drug company’s plans going forwards, so that patients can start to access treatment as quickly as possible.”

Charities urge SMC to approve treatment for life-limiting condition

A group of charities is urging for the approval of a drug that can treat an underlying genetic cause of a life-limiting muscle-wasting condition.

Duchenne muscular dystrophy (DMD) primarily affects boys and causes muscles to weaken and waste over time, with most of those affected needing to use a wheelchair by the age of 12. The life-limiting condition also causes other severe health problems to the heart and breathing muscles.

The drug Translarna, however, can help treat cases of a version of Duchenne that is caused by nonsense mutations.

Today, Muscular Dystrophy UK, Action Duchenne, Duchenne Family Support Group and Duchenne UK are appearing before the Scottish Medicines Consortium (SMC) to represent patients who could benefit from Translarna and to seek its approval for use on the NHS in Scotland.

In its joint submission to the SMC ahead of today’s meeting, the charities outlined evidence from a survey conducted earlier this year.

They provided evidence that highlights clear improvements to quality of life reported by those receiving the drug. Significantly, many families of children receiving Translarna say they retained the ability to walk for longer than expected, and that this gave their child greater independence to complete daily tasks and take part in activities with friends.

Families also reported huge behaviour improvements at school and home, as well as positive mental health outcomes.

If SMC approves the submission, Translarna would be the first treatment routinely available through the NHS in Scotland that addresses an underlying genetic cause of Duchenne muscular dystrophy.

One parent who took part in the survey, and whose thoughts were included as evidence in the submission, explained: “For our son knowing that he has a medication to help his muscles is a massive boost to him.

“We believe that every time we receive our Translarna it lifts his spirits in this fight. We never for a moment take for granted how lucky we are to be receiving this drug and feel it’s something in our corner as we face this battle head on.”

If approved, Translarna would be given in addition to the current standard treatments on the NHS in Scotland.

A spokesperson for the four charities said: “Every day is a race against time to slow down Duchenne muscular dystrophy, a progressive condition that robs children of their ability to walk and their independence.

“Muscular Dystrophy UK, Action Duchenne, Duchenne Family Support Group and Duchenne UK are pleased to have represented the voices of patients in our joint submission to the SMC.

“We believe our submission highlights both the physical and mental health benefits of Translarna, a drug that has the potential to improve lives for so many families living with Duchenne. We very much look forward to hearing the SMC committee’s thoughts on our submission.”