Wednesday, September 29, 2021

Sarcoma UK is disappointed that avapritinib (Ayvakyt) will no longer be considered for routine use on the NHS in England.

Earlier this year, the National Institute for Health and Social Care (NICE) were looking to appraise avapritinib which, if successful, would mean all eligible patients in England, Wales, and Northern Ireland would have access to this new drug. However, on Wednesday 29 September 2021, NICE announced that this appraisal had been terminated, meaning that the decision will no longer go ahead.

Avapritinib is a new drug for Gastro-Intestinal Stromal Tumour (GIST) patients whose disease is unresectable or metastatic, who have a particular genetic mutation in the make-up of their tumour (PDGFRA D842-V). GISTs are the most common type of sarcoma. It develops in the gastrointestinal (GI) tract, a long tube running through the body from the oesophagus (gullet) to the anus (back passage) and includes the stomach and intestines.

It is very rare for a GIST to have this mutation, with only an estimated 5-10 patients eligible each year across the whole of the UK. This is around 1% of the 943 GISTs diagnosed per year in the UK, according to the most recent data. There are currently three approved treatments for GIST in England – imatinib, sunitinib, and regorafenib. These treatments may not be effective for everyone, and tumours may become resistant to these over time.

Sarcoma UK submitted written evidence on patient experience of GIST (using data from the National Sarcoma Survey 2020), and the lack of effectiveness of the other drugs. We also gave evidence in person at the committee stage of the appraisal.

Fortunately, Blueprint Medicines, who make avapritinib, have said that all patients currently receiving avapritinib through their patient access program will continue to receive treatment free of charge. If a new patient is identified with this specific mutation and they may be eligible for avapritinib, please get in touch with Sarcoma UK, who will be able to advise on access.

'It's disappointing to hear that these patients with this particularly devastating type of GIST cancer will not have access to avapritinib,' said Bradley Price, Policy and Public Affairs Manager at Sarcoma UK. 'For cancers like sarcoma, where the number of drugs and treatment options are already limited, access to new and effective treatments is vital. A diagnosis of sarcoma can be devastating for patients and their loved ones, and patients have the right to access the most appropriate evidence-based care for them.'

Sarcoma UK is committed to helping people with sarcoma access the most effective treatments for their cancer and represented the views of sarcoma patients throughout the review process, and gave evidence to NICE on this appraisal. We will continue to work with other charities, the manufacturer, and health bodies in England to find a way for patients to have routine access to this drug.

Avapritinib for treating unresectable or metastatic gastrointestinal stromal tumours (terminated appraisal) NICE, 29 Sept 2021