Fair processes for priority setting: Putting theory into practice: Comment on “expanded HTA: Enhancing fairness and legitimacy”

Embedding health technology assessment (HTA) in a fair process has great potential to capture societal values relevant to public reimbursement decisions on health technologies. However, the development of such processes for priority setting has largely been theoretical. In this paper, we provide further practical lead ways on how these processes can be implemented. We first present the misconception about the relation between facts and values that is since long misleading the conduct of HTA and underlies the current assessment-appraisal split. We then argue that HTA should instead be explicitly organized as an ongoing evidence-informed deliberative process, that facilitates learning among stakeholders. This has important consequences for whose values to consider, how to deal with vested interests, how to consider all values in the decision-making process, and how to communicate decisions. This is in stark contrast to how HTA processes are implemented now. It is time to set the stage for HTA as learning

Priority setting for universal health coverage: We need evidence-informed deliberative processes, not just more evidence on cost-effectiveness

Priority setting of health interventions is generally considered as a valuable approach to support low- and middle-income countries (LMICs) in their strive for universal health coverage (UHC). However, present initiatives on priority setting are mainly geared towards the development of more cost-effectiveness information, and this evidence does not sufficiently support countries to make optimal choices. The reason is that priority setting is in reality a value-laden political process in which multiple criteria beyond cost-effectiveness are important, and stakeholders often justifiably disagree about the relative importance of these criteria. Here, we propose the use of ‘evidence-informed deliberative processes’ as an approach that does explicitly recognise priority setting as a political process and an intrinsically complex task. In these processes, deliberation between stakeholders is crucial to identify, reflect and learn about the meaning and importance of values, informed by evidence on these values. Such processes then result in the use of a broader range of explicit criteria that can be seen as the product of both international learning (‘core’ criteria, which include eg, cost-effectiveness, priority to the worse off, and financial protection) and learning among local stakeholders (‘contextual’ criteria). We believe that, with these evidence-informed deliberative processes in place, priority setting can provide a more meaningful contribution to achieving UHC

The Prospective Dutch Colorectal Cancer (PLCRC) cohort: real-world data facilitating research and clinical care

Real-world data (RWD) sources are important to advance clinical oncology research and evaluate treatments in daily practice. Since 2013, the Prospective Dutch Colorectal Cancer (PLCRC) cohort, linked to the Netherlands Cancer Registry, serves as an infrastructure for scientific research collecting additional patient-reported outcomes (PRO) and biospecimens. Here we report on cohort developments and investigate to what extent PLCRC reflects the “real-world”. Clinical and demographic characteristics of PLCRC participants were compared with the general Dutch CRC population (n = 74,692, Dutch-ref). To study representativeness, standardized differences between PLCRC and Dutch-ref were calculated, and logistic regression models were evaluated on their ability to distinguish cohort participants from the Dutch-ref (AU-ROC 0.5 = preferred, implying participation independent of patient characteristics). Stratified analyses by stage and time-period (2013–2016 and 2017–Aug 2019) were performed to study the evolution towards RWD. In August 2019, 5744 patients were enrolled. Enrollment increased steeply, from 129 participants (1 hospital) in 2013 to 2136 (50 of 75 Dutch hospitals) in 2018. Low AU-ROC (0.65, 95% CI: 0.64–0.65) indicates limited ability to distinguish cohort participants from the Dutch-ref. Characteristics that remained imbalanced in the period 2017–Aug’19 compared with the Dutch-ref were age (65.0 years in PLCRC, 69.3 in the Dutch-ref) and tumor stage (40% stage-III in PLCRC, 30% in the Dutch-ref). PLCRC approaches to represent the Dutch CRC population and will ultimately meet the current demand for high-quality RWD. Efforts are ongoing to improve multidisciplinary recruitment which will further enhance PLCRC’s representativeness and its contribution to a learning healthcare system