VHL UK/Ireland is pleased to announce that on the 5th September 2024, the National Institute of Health & Care Excellence (NICE) published its Final Draft Guidance for NHS England with a positive recommendation for the use of belzutifan (Welireg) under a Managed Access. Agreement (MAA).
Link: Final Draft Guidance for NHS England
Link: See All Project Documents
Von Hippel-Lindau disease (VHL) is a devastating, rare, life-long, incurable disease, that can cause multiple, malignant, and benign tumours/cysts throughout the body. People with VHL can suffer from tumours on multiple organs, simultaneously, or repetitively, and these can be life-threatening. VHL does not go into remission, tumours do not shrink naturally and there is no cure.
Until now, often high risk, invasive surgery has been the only way to remove these tumours, and it is not uncommon for patients to their lose sight, part or whole organs and in a number of cases, patients suffer neurological issues such as paralysis after spine or brain surgery.
This condition affects all aspects of the patient and their carers lives including family planning, education, careers, leisure and travel, relationships, mental wellbeing and their ability to plan for the future.
Belzutifan (Welireg) is already available to patients in other countries, including the USA, Canada, Australia, and was approved by the Scottish Medicines Consortium (SMC) for use via NHS Scotland in September 2023. Results internationally have been astonishing, demonstrating stability, reduction in growth and in some cases the eradication of tumours.
VHL UK/Ireland believes that belzutifan (Welireg) has the game changing potential to completely transform the lives of qualifying VHL patients. The charity has been in direct contact with NICE as a key stakeholder in this appraisal since 2021, providing numerous written submissions and attending a considerable number of meetings with NICE, clinicians and patient representatives.
Chair of VHL UK/Ireland, Graham Lovitt said:
“We are delighted by NICE’s recommendation of belzutifan (Welireg) under the MAA, a significant milestone in the fight against this rare disease. This decision brings hope to qualifying patients and their families, offering them an alternative treatment option, when others become too high risk. VHL UK/Ireland remains committed to supporting those affected by the condition and helping everyone who can benefit from this breakthrough medicine to access it”.
To discover more about VHL and belzutifan (Welireg), please visit:
.
Frequently Asked Questions (FAQs)
.
.
We want to extend our deepest thanks to all VHL warriors, their loved ones and the dedicated clinicians who have supported the charity through this process. Your support – whether through fundraising, raising awareness, sharing your experiences, or showing remarkable patience during this time – has been truly invaluable.
