Presentation (CTI)

OBJECTIVES: Ex-U.S. drug prices are increasingly cited as value-based benchmarks in U.S. pricing policy, yet the evidentiary standards supporting those prices remain unexamined. The objective is to assess the rigor of comparative effectiveness research (CER) and the use of patient-reported outcomes (PROs) in ex-U.S. health technology assessments (HTA) for top-spending U.S. Medicare Part B biologics.
METHODS: Using the Medicare Part B Drug Spending Dashboard (2023), the top-spending biologics—aflibercept, denosumab, daratumumab, nivolumab, and pembrolizumab—were identified. For each, two FDA-approved indications with the largest U.S. market share were selected. HTA reports from ex-U.S. G7 countries (Canada, France, Germany, Italy, Japan, UK) were retrieved from NAVLIN. Reports were assessed using a framework capturing CER presence and type, design validity, comparator appropriateness, evidence quality, transparency, study outcomes, economic assessments, and HTA and reimbursement decisions. A CER-rigor score categorized reports as Low (0-3), Moderate (4), or High (5-6).
RESULTS: Among 47 reports, CER was used in 80.9%, with 55.3% relying on direct evidence. CER use and rigor varied across agencies. Indirect comparisons appeared in 11 reports, but only one was considered valid by the HTA. Comparator appropriateness (59.6%), CER design quality (55.3%), and transparency (57.4%) were present in roughly half of reports. Overall, 51.1% had Low, 4.3% Moderate, and 44.7% had High CER rigor. Favorable HTA/reimbursement decisions were more common in high-rigor reports. Only 19 (40.4%) reports, mainly from the UK and Canada, incorporated PROs into their final decision rationale. Economic assessments included cost-utility (55.3%) and budget-impact analyses (48.9%).
CONCLUSIONS: Inconsistent CER rigor and sparse PRO use across ex-U.S. G7 countries show the risk of referencing foreign prices without scrutinizing evidence. U.S. pricing policy must depend on benchmarks supported by strong CER and patient-centered outcomes. The framework and CER-rigor score provide a proof-of-concept foundation that future work can refine to assess evidence robustness for U.S. pricing decisions.