Presentation (CTI)

OBJECTIVES: To overcome postal-code dependent care and ensure uniformity, clinical relevance criteria (PASKWIL) were set for clinical decision-making on expensive oncology drugs in the late 1990's. Later, the Dutch Healthcare Institute formally added cost-effectiveness assessment with an explicit willingness-to-pay threshold (WTP), which suggests that PASKWIL-criteria became redundant for the initial aim. However, PASKWIL-criteria remain central to reimbursement. The aim of this study is to quantify health gains and net monetary value (NMV) foregone when treatments are cost-effective but fail in line with current PASKWIL-criteria.
METHODS: The focus of this study is on the palliative setting with comparator median Overall Survival (OS) below 12 months, where PASKWIL requires an OS gain >12 weeks and hazard ratio (HR) <0.70, or a two-year OS difference >10% with over 20% survivors. A partitioned survival model for chemotherapy in recurrent/metastatic cervical cancer was developed for a hypothetical PASKWIL-negative intervention (HR_OS 0.70-1.00). NMV equals QALY gain multiplied by a WTP of €80,000 minus incremental non-drug costs, excluding the intervention price, i.e. the maximum spendable amount while remaining cost-effective. Scenario-analyses varied OS distribution, utilities, costs, age, and administration frequency.
RESULTS: At the PASKWIL threshold, a cost-effective intervention generates a discounted QALY gain of 0.46 and an NMV of €18,564 per patient, both declining monotonically as the HR increases. Results were robust across all scenarios, with QALY gain ranging from 0.34 to 0.51 and NMV from €9,000 to €23,000, driven primarily by utility differences between progression-free and progressed health states.
CONCLUSIONS: Therapies not fulfilling PASKWIL-criteria that meet the WTP-threshold, represent substantial foregone health gain and monetary value. Therapies with median OS below 12 months, a QALY gain of 0.46 and NMV of €18,564 per patient, are clinically and economically meaningful. The function of PASKWIL-criteria as an independent threshold excluding cost-effective treatments from reimbursement, warrants reconsideration.