Evolving PICO Characteristics in the Early Oncology Treatment Lifecycle: Differences from Trials to Treatment Guidelines

OBJECTIVES: Under the new European health technology assessment (HTA) legislation, European countries will largely need to agree on the PICO (population, intervention, comparator, outcome) elements in HTA assessments. Additionally, cross-stakeholder alignment grows in importance due to our ever-evolving healthcare landscape, such as smaller populations and increasingly advanced products. The PICO therefore becomes more subject to variation among decision-makers. This study assessed how the PICO elements evolve over the early treatment lifecycle of oncology drugs and align between stakeholders on a global level.

METHODS: A systematic content analysis of clinical trial publications, regulatory and HTA/payer reports, clinical guidelines was performed. The countries assessed were Australia, Canada, France, the Netherlands, the United Kingdom, and the United States. The assessed and included/approved patient populations, comparators, intervention features and clinical outcomes were collected and quantitatively compared across stakeholder assessments in order to identify patterns.

RESULTS: The results will focus on patterns in population definitions. For example, whether these show discrepancies or grow larger or smaller over time, indicating differences across stakeholders in considering uncertainty in decision-making. Interventions may change over time in dosage, frequency, start- stop criteria, of co-medication or diagnostic testing to support appropriate use. Discrepancies in comparators may be visualized across decision-makers or countries, addressing complexity in choosing an appropriate comparator when designing clinical trials. Differences in outcomes may reflect on the decision criteria and evidence requirements across stakeholders, that are ideally included in the evidence package available for decision-making.

CONCLUSIONS: Lacking alignment among clinical trials, regulatory and HTA decisions and subsequent clinical guideline development can lead to delayed access, additional uncertainty caused by conflicting decisions or advice, or generally, reduced trust by undermining the integrity of the decision-making processes. Aligning on the expectations for the PICO would improve transparency and consistency, creating a shared foundation for decisions-making, while safeguarding the different and independent stakeholder remits.