We are delighted to announce that NICE (the National Institute for Health and Care Excellence) has recently released guidance recommending fedratinib as an option within the NHS for treating disease-related splenomegaly (enlarged spleen) or symptoms of myelofibrosis in adult patients who have previously been treated with ruxolitinib and for whom momelotinib is unsuitable.
This positive news follows a detailed appraisal process by NICE, during which MPN Voice, in conjunction with Leukaemia Care, made written submissions on behalf of all MF patients and participated in the NICE committee meeting at which the suitability of fedratinib as an additional treatment option for MF patients was fully evaluated.
Although this guidance initially applies only to patients in England, we hope that it will soon also be adopted in Wales. Fedratinib is already available to eligible MF patients in Scotland and Northern Ireland.
We are very grateful to the clinicians who worked with us throughout the appraisal process and in particular to a number of MF patients already being treated with fedratinib on a trial basis, who spoke to us about their use of this drug and about living with MF – their first-hand experience added invaluable real-life testimony to the submissions that we made to NICE.
Myelofibrosis can be an extremely debilitating illness that has a significant impact on patients’ lives in terms of day-to-day symptoms and affects not only patients but also their families and carers. Existing therapies for MF can have limitations and side effects and may not adequately reduce some patients’ main symptoms, including an enlarged spleen, fatigue, bone pain and anaemia. Even in cases where patients initially respond well to the existing therapies, those can lose their effectiveness over time and we therefore welcome the recommendation of fedratinib as an additional targeted treatment option for certain groups of MF patients.
This guidance from NICE, meaning that there are now three recommended targeted treatment options for MF patients, underlines how important it is that we continue to support research into the development of new treatments for MPNs. The work that the volunteers on our Advocacy Team do is a key part of this but this work also relies on very valuable input from the whole MPN community, through responses to surveys and questionnaires and above all through fundraising to help us continue our work.
If you’d like to know more about what we do and want to help us achieve our mission through volunteering for MPN Voice or helping to raise funds we’d love to hear from you at info@mpnvoice.org.uk.
