NICE Recommends MSD's Welireg for Rare von Hippel-Lindau Disease

MSD's Welireg (belzutifan) has received a managed access recommendation from the National Institute for Health and Care Excellence (NICE) for treating von Hippel-Lindau (VHL) disease, a rare genetic condition that leads to tumour formation in major organs. Welireg will now be available on the National Health Service (NHS) in England and Wales for adult patients with VHL-associated renal cell carcinomas, central nervous system haemangioblastomas, or pancreatic neuroendocrine tumours, especially in cases where localized procedures are not viable or preferred.

VHL disease affects roughly one in every 70,000 individuals and is caused by a mutation in the VHL gene, leading to abnormal cell growth. This abnormal growth can result in cysts or tumours in various body parts, including the kidneys, brain, and pancreas, potentially impairing function or becoming malignant. Previously, the primary treatment options were invasive surgeries or radiotherapy, often requiring multiple procedures throughout a patient's life.

Benson Fayehun, the head of oncology at MSD UK, highlighted the severe impact of VHL disease, noting that patients often serve as caregivers for affected family members. He pointed out that the new decision to provide Welireg offers a less invasive treatment alternative, potentially reducing the need for multiple surgeries. "This decision provides a welcome option for patients, reducing the need for and impact of numerous surgeries," Fayehun stated.

NHS England estimates that approximately 100 people could benefit from Welireg in its first year of availability, with around 50 people per year thereafter. The final draft guidance from NICE, which was facilitated through a collaboration between NHS England and MSD and enabled access via the Cancer Drugs Fund, was bolstered by clinical trial results. These trials indicated that 95% of patients did not experience tumour growth over two years of taking Welireg, and 56% of patients saw their tumours shrink.

Despite these promising results, NICE noted certain uncertainties and emphasized the need for additional evidence before Welireg can be considered for routine NHS use. Thus, the drug has been recommended for use with ongoing data collection to gather more evidence. 

Helen Knight, director of medicines evaluation at NICE, expressed optimism about the treatment's potential benefits. "This is a new treatment that has the potential to prevent loss of organ function from repeated tumours and surgeries and improve people’s quality of life at a price that is good value for the taxpayer. We’re therefore pleased to be able to recommend it today while more evidence is collected," Knight said.

In summary, the introduction of Welireg represents a significant advancement for patients with VHL disease, offering a less invasive treatment alternative that could improve quality of life and reduce the need for repeated surgeries. The drug's approval for managed access reflects a cautious yet optimistic approach, balancing immediate patient needs with the requirement for further evidence to confirm its long-term efficacy and safety.

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