NCPE Recommends Belzutifan (Welireg) Not Reimbursed for VHL Disease on Cost-Effectiveness Grounds (ncpe.ie)

• NCPE recommends belzutifan not reimbursed for VHL disease unless cost-effectiveness improved.
• Single-arm LITESPARK-004 trial showed ORR 70.5% (RCC), 50% (CNS-Hbs), 90.9% (pNETs).
• All 61 patients experienced AEs; most common anaemia (93.4%), fatigue (77%).
• NCPE cited lack of comparative data, immature survival, missing QoL data, and concerns with indirect comparisons.

"The National Centre for Pharmacoeconomics (NCPE) has recommended that belzutifan (Welireg) not be reimbursed by the HSE for treating von Hippel-Lindau (VHL) disease unless its cost-effectiveness improves. The recommendation follows an HTA submission from MSD assessing the drug against existing standard of care (surgery, radiation, ablation). Clinical data from the single-arm LITESPARK-004 trial showed objective response rates of 70.5% for renal cell carcinoma, 50% for CNS haemangioblastomas, and 90.9% for pancreatic neuroendocrine tumours. However, all patients experienced adverse events (most commonly anaemia and fatigue). The NCPE found limitations in indirect comparisons, immature survival data, and lack of quality-of-life data, and concluded the price did not justify the clinical benefits."