Evidence-based health care policy in reimbursement decisions : lessons from a series of six equivocal case-studies

Context: Health care technological evolution through new drugs, implants and other interventions is a key driver of healthcare spending. Policy makers are currently challenged to strengthen the evidence for and cost-effectiveness of reimbursement decisions, while not reducing the capacity for real innovations. This article examines six cases of reimbursement decision making at the national health insurance authority in Belgium, with outcomes that were contested from an evidence-based perspective in scientific or public media. Methods: In depth interviews with key stakeholders based on the adapted framework of Davies allowed us to identify the relative impact of clinical and health economic evidence; experience, expertise & judgment; financial impact & resources; values, ideology & political beliefs; habit & tradition; lobbyists & pressure groups; pragmatics & contingencies; media attention; and adoption from other payers & countries. Findings: Evidence was not the sole criterion on which reimbursement decisions were based. Across six equivocal cases numerous other criteria were perceived to influence reimbursement policy. These included other considerations that stakeholders deemed crucial in this area, such as taking into account the cost to the patient, and managing crisis scenarios. However, negative impacts were also reported, in the form of bypassing regular procedures unnecessarily, dominance of an opinion leader, using information selectively, and influential conflicts of interest. Conclusions: 'Evidence' and 'negotiation' are both essential inputs of reimbursement policy. Yet, purposely selected equivocal cases in Belgium provide a rich source to learn from and to improve the interaction between both. We formulated policy recommendations to reconcile the impact of all factors identified. A more systematic approach to reimburse new care may be one of many instruments to resolve the budgetary crisis in health care in other countries as well, by separating what is truly innovative and value for money from additional 'waste'

How to reform western care payment systems according to physicians, policy makers, healthcare executives and researchers: a discrete choice experiment

Background: Many developed countries are reforming healthcare payment systems in order to limit costs and improve clinical outcomes. Knowledge on how different groups of professional stakeholders trade off the merits and downsides of healthcare payment systems is limited. Methods: Using a discrete choice experiment we asked a sample of physicians, policy makers, healthcare executives and researchers from Canada, Europe, Oceania, and the United States to choose between profiles of hypothetical outcomes on eleven healthcare performance objectives which may arise from a healthcare payment system reform. We used a Bayesian D-optimal design with partial profiles, which enables studying a large number of attributes, i.e. the eleven performance objectives, in the experiment. Results: Our findings suggest that (a) moving from current payment systems to a value-based system is supported by physicians, despite an income trade-off, if effectiveness and long term cost containment improve. (b) Physicians would gain in terms of overall objective fulfillment in Eastern Europe and the US, but not in Canada, Oceania and Western Europe. Finally, (c) such payment reform more closely aligns the overall fulfillment of objectives between stakeholders such as physicians versus healthcare executives. Conclusions: Although the findings should be interpreted with caution due to the potential selection effects of participants, it seems that the value driven nature of newly proposed and/or introduced care payment reforms is more closely aligned with what stakeholders favor in some health systems, but not in others. Future studies, including the use of random samples, should examine the contextual factors that explain such differences in values and buy-in. JEL classification: C90, C99, E61, I11, I18, O5

Healthcare payment reforms across western countries on three continents: Lessons from stakeholder preferences when asked to rate the supportiveness for fulfilling patients’ needs

International audienceTo test the hypothesis that care typology-being complex and highly unpredictable versus being clear-cut and highly predictable-guides healthcare payment preferences of physicians, policy makers, healthcare executives, and researchers. We collected survey data from 942 stakeholders across Canada, Europe, Oceania, and the United States. A total of 48 international societies invited their members to participate in our study. Study design Cross-sectional analysis of stakeholder survey data linked to four scenarios of care typology: primary prevention, trial-and-error care, standard care and network care. Principal findings We identified two “extremes”: (1) dominant preferences of physicians, who embraced fee for service (FFS), even when this precludes the advantages of other payment systems associated with a minimal risk of harm (OR 1.85 for primary prevention; OR 1.89 for standard care, compared to non-physicians); and (2) the dominant preferences of healthcare executives and researchers, who supported quality bonus or adjustment (OR 1.92) and capitation (OR 2.05), respectively, even when these could cause harm. Conclusions Based on exploratory findings, we can cautiously state that payment reform will prove to be difficult as long as physicians, healthcare executives, and researchers misalign payment systems with the nature of care. Replication studies are needed to (dis)confirm our findings within representative subsamples per area and stakeholder grou

Downstream processing from hot-melt extrusion towards tablets : a quality by design approach

Since the concept of continuous processing is gaining momentum in pharmaceutical manufacturing, a thorough understanding on how process and formulation parameters can impact the critical quality attributes (CQA) of the end product is more than ever required. This study was designed to screen the influence of process parameters and drug load during HME on both extrudate properties and tableting behaviour of an amorphous solid dispersion formulation using a quality-by-design (QbD) approach. A full factorial experimental design with 19 experiments was used to evaluate the effect of several process variables (barrel temperature: 160-200 degrees C, screw speed: 50-200 rpm, throughput: 0.2-0.5 kg/h) and drug load (0-20%) as formulation parameter on the hot-melt extrusion (HME) process, extrudate and tablet quality of Soluplus (R)-Celecoxib amorphous solid dispersions. A prominent impact of the formulation parameter on the CQA of the extrudates (i.e. solid state properties, moisture content, particle size distribution) and tablets (ie. tabletability, compactibility, fragmentary behaviour, elastic recovery) was discovered. The resistance of the polymer matrix to thermo-mechanical stress during HME was confirmed throughout the experimental design space. In addition, the suitability of Raman spectroscopy as verification method for the active pharmaceutical ingredient (API) concentration in solid dispersions was evaluated. Incorporation of the Raman spectroscopy data in a PLS model enabled API quantification in the extrudate powders with none of the DOE-experiments resulting in extrudates with a CEL content deviating > 3% of the label claim. This research paper emphasized that HME is a robust process throughout the experimental design space for obtaining amorphous glassy solutions and for tabletting of such formulations since only minimal impact of the process parameters was detected on the extrudate and tablet properties. However, the quality of extrudates and tablets can be optimized by adjusting specific formulations parameters (e.g. drug load)

Mechanisms of stretch-mediated skin expansion at single-cell resolution.

The ability of the skin to grow in response to stretching has been exploited in reconstructive surgery1. Although the response of epidermal cells to stretching has been studied in vitro2,3, it remains unclear how mechanical forces affect their behaviour in vivo. Here we develop a mouse model in which the consequences of stretching on skin epidermis can be studied at single-cell resolution. Using a multidisciplinary approach that combines clonal analysis with quantitative modelling and single-cell RNA sequencing, we show that stretching induces skin expansion by creating a transient bias in the renewal activity of epidermal stem cells, while a second subpopulation of basal progenitors remains committed to differentiation. Transcriptional and chromatin profiling identifies how cell states and gene-regulatory networks are modulated by stretching. Using pharmacological inhibitors and mouse mutants, we define the step-by-step mechanisms that control stretch-mediated tissue expansion at single-cell resolution in vivo.Wellcome Trust Royal Societ