TA1011 · single_technology_appraisal

Cancer Drugs Fund data collection arrangement for the single technology appraisal of belzutifan for treating tumours associated with von Hippel-Lindau disease

Recommended for Cancer Drugs FundAugust 2024

Managed Access Agreement with Cancer Drugs Fund - data collection arrangement. Treatment to be continued until disease progression, unacceptable toxicity, patient choice to stop, or occurrence of intervention with localised procedure. Monotherapy only for VHL-associated RCC, CNS haemangioblastoma, and/or pNET where localised procedures are unsuitable or undesirable.

Source documents

Intervention

belzutifan (Not stated)
· HIF-2α inhibitor · Not stated

Conditions

tumours associated with von hippel-lindau diseaseoncology · locally_advanced
von hippel-lindau disease-associated tumoursoncology · metastatic

Clinical trials

TrialDesignPhasePivotal
LITESPARK-004single_armPhase II

Evidence gaps

short follow upLong-term efficacy of belzutifan
single arm evidence onlyLITESPARK-004 is a single-arm trial with no comparator
immature overall survivalTreatment waning and long-term safety profile

Commercial arrangement

managed access agreement · critical for recommendation

Special considerations

Cancer Drugs Fund eligible