Estimands, PICOs and Co. - Are we losing or gaining in translation?

It was fantastic to participate in this webinar on estimands, PICOs and the target trial framework from the HTA European Special Interest Group. Different areas in clinical development, patient access and health technology assessment differ in how they approach their respective research or policy questions. The estimand framework is currently the focus in clinical development, but the PICO framework is prevalent in health technology assessment. Moreover, the target trial emulation framework appears to be preferred for observational comparisons based on real-world data. This webinar aimed to compare and contrast the different frameworks and discuss how they may complement each other, in light of the new European Union regulation for health technology assessment.

I spoke about the PICO framework, which is used in health technology assessment to translate policy questions into research questions. PICOs consist of five components: (1) population; (2) intervention; (3) comparator(s); and (4) outcome. During the reimbursement process, the manufacturer typically submits an evidence dossier that addresses the research questions(s) included in the scope. Best-practice guidelines recommend specifying relevant PICO question(s) in the HTA scoping process. In evidence synthesis, PICO questions are often formulated prior to the analysis in order to guide the data extraction required for systematic literature reviews.

Robert Hemmings (Consilium Salmonson & Hemmings) provided some great insights on the estimand framework and Nicholas Latimer (University of Sheffield) gave a fantastic presentation on decision problems, featuring the perspective of the National Institute for Health and Care Excellence in the United Kingdom. Many thanks to Arthur Allignol (Daiichi Sankyo) for the invitation to present.