Accounting for Genomic Testing Costs in Cost-Effectiveness Evaluations: Challenges and Lessons Learned from the NICE Single Technology Appraisal of Erdafitinib for Treating Unresectable or Metastatic Urothelial Cancer with FGFR3 Alterations After a PD-1 or PD-L1 Inhibitor

Precision medicine incorporates genomic and molecular profiling into clinical decision-making, enabling treatments to be tailored to the unique characteristics of each patient and their disease [ 1 , 2 ]. For instance, therapies may target epidermal growth factor receptor (EGFR) or anaplastic lymphoma kinase (ALK) mutations, B-Raf proto-oncogene (BRAF) variants, or programmed death-ligand 1 (PD-L1) expression levels [ 3 ]. Over the past decade, the implementation of precision medicine in oncology has resulted in improved survival rates, reduced off-target toxicities, and enhanced quality of life compared with non-targeted therapies [ 3 , 4 , 5 , 6 , 7 ]. However, precision oncology presents new challenges. Alongside the high acquisition costs of precision therapies, influenced by the complex research, development, and smaller treatment populations [ 8 ], patients must undergo additional genomic testing to determine eligibility [ 4 ]. These diagnostic requirements introduce further costs that can influence cost-effectiveness evaluations, especially when genomic testing is not (yet) part of routine clinical practice, and the prevalence of actionable mutations is low [ 9 , 10 ]. Applying the full cost of genomic testing to populations with low prevalence can substantially increase incremental cost-effectiveness ratios (ICERs), which may risk discouraging innovation [ 11 , 12 ]. In contrast, in larger populations, testing costs are distributed across more patients, thereby reducing their impact. Yet, oncology is increasingly moving towards molecularly defined subgroups as more targeted therapies emerge [ 3 , 8 ]. This commentary highlights the challenges and key learnings of integrating genomic testing costs in cost-effectiveness evaluations from an External Assessment Group (EAG) perspective, using the example of the National Institute for Health and Care Excellence (NICE) Single Technology Appraisal (STA) of erdafitinib for treating unresectable or metastatic urothelial cancer (UC) with fibroblast growth factor receptor 3 (FGFR3) alterations after a programmed death receptor-1 (PD-1) or PD-L1 inhibitor in adults.