Presentation (CTI)

OBJECTIVES: Precision oncology (PO) technologies, including biomarker-guided therapies and companion diagnostics, present distinctive challenges for health technology assessment (HTA), including complex treatment pathways, small evidence bases, immature survival data, and uncertainty around test-treatment interdependencies. This study aimed to identify key critiques raised by NICE in PO appraisals.
METHODS: A structured review of NICE oncology appraisals (Jan’2020-Dec’2025) was conducted. Eligible appraisals included biomarker-defined, molecularly stratified, companion diagnostic-linked, or tumour-agnostic technologies where PO considerations shaped deliberations. Data was extracted to identify patterns in evidence generation, diagnostics, comparators, sequencing, implementation, and factors influencing reimbursement decisions and reassessments.
RESULTS: The review identified 23 appraisals to ensure representation across tumour types, precision oncology archetypes, evidence maturity, and NICE decision outcomes: 11 NSCLC, 7 breast cancer, and one each for ovarian/fallopian/peritoneal, tumour-agnostic solid tumour, endometrial, multi-tumour, and prostate cancer. Biomarker testing was required in all appraisals, with diagnostic pathways detailed in 21/23. Majority of challenges included immature OS, followed by lack of direct evidence and hence, use of indirect comparisons or external controls informing comparison (17/23). NICE issued positive recommendations in 21/23, including 4 CDF recommendations. Majority of positive recommendations were restricted to narrower population and 2/23 were not recommended. NSCLC critiques centred on immature OS and comparator uncertainty, while breast cancer critiques focused on comparator uncertainty and cost-effectiveness in narrower biomarker-defined populations. Reassessment over time showed divergent re-review trajectories: lorlatinib (TA909→TA1103) from non-recommendation to recommendation after additional PFS maturity data despite continued OS immaturity, and adjuvant osimertinib (TA761→TA1043) from managed access to routine commissioning after review of long-term trial and real-world evidence.
CONCLUSIONS: PO technologies pose recurring HTA challenges from immature evidence, limited comparative data, and diagnostic-linked patient selection. Residual uncertainty often shaped NICE decisions, resulting in restricted recommendations, while re-appraisals suggest that accumulating clinical and real-world evidence can help resolve key uncertainties over the technology lifecycle.