The Scottish Medicines Consortium (SMC) the organisation that advises which medicines should be made available via the NHS in Scotland has given their approval to a new drug – tafamidis (Vyndaqel) – to treat transthyretin amyloidosis cardiomyopathy (ATTR-CM). This is the first ever treatment for this type of cardiomyopathy. It has been approved for adults with hereditary or wild-type (non-hereditary) ATTR-CM.
Tafamidis had already been assessed by the in 2021, but was rejected for value for money reasons. This reassessment has therefore granted access on the basis of a ‘Patient Access Scheme’ arrangement. This is a confidential pricing agreement to improve the cost effectiveness of the medicine.
Tafamidis should now be made available by NHS boards in Scotland, which have responsibility for deciding which medicines are available in their area. Once it is on Boards’ formularies (list of approved medicines), doctors will be able to prescribe the drug. While they can use their clinical judgement when prescribing, making their decision on an individual patient's situation, they usually follow the SMC's advice.
In response to the news, Joel Rose, Cardiomyopathy UK’s Chief Executive, said:
“Amyloidosis cardiomyopathy is a serious, progressive, disabling and lethal condition, with a heavy toll on patients, their relatives and carers. So we’re extremely pleased that Tafamidis will become available in Scotland for people affected by this rare form of cardiomyopathy. We sincerely hope that NICE follows suit and the treatment also becomes available in England and Wales.”
How does the treatment work?
Amyloidosis is a group of rare conditions caused by a build-up of an abnormal protein (called amyloid) in the body. This build-up stops the tissues and organs working properly.
In ATTR-CM, a type of amyloid protein called transthyretin that normally circulates in the bloodstream becomes misshapen and builds up in the heart, nerves and other organs. It can affect how well the heart functions, causing cardiomyopathy.
Tafamidis works by inhibiting the formation of amyloid. This delays the onset of cardiac muscle damage.
Our role
We worked closely with the UK Amyloidosis Patient Association to put in a joint submission to the SMC. This submission provided evidence on the impact of the disease on patients, and how the drug could make a difference in improving the quality of life of patients, their family and carers.
We also spoke Pfizer – the pharmaceutical company which owns the drug - about the importance of making the drug affordable to the NHS.
We were then invited to attend the meeting of the SMC’s committee responsible for making decisions on new drugs, to provide more information as needed.
Next steps
We intend to support the assessment of tafamidis by NICE, providing evidence to show how badly needed this drug is in England and Wales.
More broadly, as new treatments are being developed the charity will continue to work with NICE, the SMC and pharmaceutical companies to help them understand the experience of living with cardiomyopathy and to promote the development and use of effective and affordable treatment options.
You can read the full report from the SMC here