Dispensing behaviour of pharmacies in prescription drug markets.

OBJECTIVE: We aim to investigate pharmacies' dispensing behaviour under the existing dispensing regulations in Germany. METHODS: Using administrative data, we performed a cross-sectional retrospective study to analyse whether the competitive environment and pharmacy characteristics, i.e., organisation, lead to dispensing choices aimed at by third-party payers. We specified generalised linear models with the share of imported pharmaceuticals, generic share, and share of preferred brands as dependent variables. RESULTS: The final dataset contained 49,260,902 prescriptions from 16,797 pharmacies. The average share of imported pharmaceuticals across the pharmacies was 18.4% (standard deviation (SD) 8.8), the average generic share was 92.8% (SD 2.1), and compliance with preferred brands was 81.3% (SD 5.9). Pharmacies with little competition used fewer imported pharmaceuticals (p<0.001), generics (p<0.001) and preferred brands (p<0.001); less organised pharmacies yielded similar results. The difference in outcomes between pharmacies in the first and 4th quartiles of the pharmacy organisation variable is 17.4% vs. 17.0% for share of imported pharmaceuticals, 92.8% vs. 92.7% for generic share and 81.9% vs. 81.1% for compliance with preferred brands. CONCLUSION: We show that pharmacies' dispensing choices meet the aims of payers at high levels. However, dispensing behaviour varies between pharmacies. Increasing competition among pharmacies and targeting pharmacies with high shares of bill auditing seem viable options to improving dispensing behaviour as defined by payers.

Early benefit assessment of pharmaceuticals in Germany: manufacturers' expectations versus the Federal Joint Committee's decisions.

BACKGROUND: Since 2011, when the German Pharmaceutical Market Restructuring Act (AMNOG) came into effect, newly licensed pharmaceuticals must demonstrate an added benefit over a comparator treatment to be reimbursed at a value greater than the reference price. Evidence submitted by manufacturers is assessed by the Institute for Quality and Efficiency in Health Care (IQWiG) and subsequently appraised by the German Federal Joint Committee (FJC) as part of so-called early benefit assessments (EBA). This study aims to explain the decisions made, clarify the roles of the parties (manufacturers, IQWiG, FJC) involved, and guide manufacturers in developing future submissions by analyzing 42 EBAs concluded since January 2011. METHODS: We developed a variable list representing the essential components of the EBA: the rating decisions of manufacturers, IQWiG, and the FJC regarding each pharmaceutical's added benefit; the characteristics of the pharmaceutical; the characteristics of the EBA process; the types of evidence submitted; the methods used to generate evidence; and the pharmaceutical's maximum possible budget impact. We used Cohen's kappa to analyze agreement between the rating decisions of the different parties. The chi-square test and bivariate regression were used to identify associations between components of the EBA process and the rating decisions of the FJC. RESULTS: We observed a low level of agreement between manufacturers and the FJC (kappa = 0.21; 95% CI 0.107-0.31) and a substantial level of agreement between IQWiG and the FJC (kappa = 0.64; 95% CI 0.451-0.827) in their rating decisions. The characteristics of the EBA process--for example, duration of the process (P = 0.357), participation in the official hearing (P = 0.227), and the pharmaceutical's budget impact (P = 0.725)--did not have a significant effect on the rating decisions of the FJC. There was, however, an association between the type of evidence submitted and the FJC's rating decision when the manufacturer's dossier reported outcomes related to morbidity (P = 0.009) or adverse events (P < 0.001) but not mortality (P = 0.718) or quality of life (P = 0.783). CONCLUSIONS: While the FJC tends to disagree with the rating of benefit by manufacturers, it softens IQWiG's decisions, potentially to make the final outcome more acceptable. Concerns voiced that the FJC might be exceeding its statutory authority by taking cost or procedural considerations into account appear to be unfounded. Choosing appropriate evidence to submit for each endpoint remains a challenge, as submission of health outcomes evidently influences decisions.

Link between process and appraisal in coverage decisions: an analysis with structural equation modeling.

BACKGROUND: To achieve fair-coverage decision making, both material criteria and criteria of procedural justice have been proposed. The relationship between these is still unclear. OBJECTIVE: To analyze hypotheses underlying the assumption that more assessment, transparency, and participation have a positive impact on the reasonableness of coverage decisions. METHODS: We developed a structural equation model in which the process components were considered latent constructs and operationalized by a set of observable indicators. The dependent variable "reasonableness" was defined by the relevance of clinical, economic, and other ethical criteria in technology appraisal (as opposed to appraisal based on stakeholder lobbying). We conducted an Internet survey among conference participants familiar with coverage decisions of third-party payers in industrialized countries between 2006 and 2011. Partial least squares path modeling (PLS-PM) was used, which allows analyzing small sample sizes without distributional assumptions. Data on 97 coverage decisions from 15 countries and 40 experts were used for model estimation. RESULTS: Stakeholder participation (regression coefficient [RC] =0.289; P = 0.005) and scientific rigor of assessment (RC = 0.485; P < 0.001) had a significant influence on the construct of reasonableness. The path from transparency to reasonableness was not significant (RC = 0.289; P = 0.358). For the reasonableness construct, a considerable share of the variance was explained (R (2) = 0.44). Biases from missing data and nesting effects were assessed through sensitivity analyses. Limitations. The results are limited by a small sample size and the overrepresentation of some decision makers. CONCLUSIONS: Rigorous assessment and intense stakeholder participation appeared effective in promoting reasonable decision making, whereas the influence of transparency was not significant. A sound evidence base seems most important as the degree of scientific rigor of assessment had the strongest effect.

Transparency vs. closed-door policy: do process characteristics have an impact on the outcomes of coverage decisions? A statistical analysis.

The aim of this study was to analyze influences of process- and technology-related characteristics on the outcomes of coverage decisions. Using survey data on 77 decisions from 13 countries, we examined whether outcomes differ by 14 variables that describe components of decision-making processes and the technology. We analyzed the likelihood of committees covering a technology, i.e. positive (including partial coverage) vs. negative coverage decisions. We performed non-parametric univariate tests and binomial logistic regression with a stepwise variable selection procedure. We identified a negative association between a positive decision and whether the technology is a prescribed medicine (p=0.0097). Other significant influences on a positive decision outcome included one disease area (p=0.0311) and whether a technology was judged to be (cost-)effective (p<0.0001). The first estimation of the logistic regression yielded a quasi-complete separation for technologies that were clearly judged (cost-)effective. In uncertain decisions, a higher number of stakeholders involved in voting (odds ratio=2.52; p=0.03) increased the likelihood of a positive outcome. The results suggest that decisions followed the lines of evidence-based decision-making. Despite claims for transparent and participative decision-making, the phase of evidence generation seemed most critical as decision-makers usually adopted the assessment recommendations. We identified little impact of process configurations.

Decision-making in healthcare: a practical application of partial least square path modelling to coverage of newborn screening programmes.

BACKGROUND: Decision-making in healthcare is complex. Research on coverage decision-making has focused on comparative studies for several countries, statistical analyses for single decision-makers, the decision outcome and appraisal criteria. Accounting for decision processes extends the complexity, as they are multidimensional and process elements need to be regarded as latent constructs (composites) that are not observed directly. The objective of this study was to present a practical application of partial least square path modelling (PLS-PM) to evaluate how it offers a method for empirical analysis of decision-making in healthcare. METHODS: Empirical approaches that applied PLS-PM to decision-making in healthcare were identified through a systematic literature search. PLS-PM was used as an estimation technique for a structural equation model that specified hypotheses between the components of decision processes and the reasonableness of decision-making in terms of medical, economic and other ethical criteria. The model was estimated for a sample of 55 coverage decisions on the extension of newborn screening programmes in Europe. Results were evaluated by standard reliability and validity measures for PLS-PM. RESULTS: After modification by dropping two indicators that showed poor measures in the measurement models' quality assessment and were not meaningful for newborn screening, the structural equation model estimation produced plausible results. The presence of three influences was supported: the links between both stakeholder participation or transparency and the reasonableness of decision-making; and the effect of transparency on the degree of scientific rigour of assessment. Reliable and valid measurement models were obtained to describe the composites of 'transparency', 'participation', 'scientific rigour' and 'reasonableness'. CONCLUSIONS: The structural equation model was among the first applications of PLS-PM to coverage decision-making. It allowed testing of hypotheses in situations where there are links between several non-observable constructs. PLS-PM was compatible in accounting for the complexity of coverage decisions to obtain a more realistic perspective for empirical analysis. The model specification can be used for hypothesis testing by using larger sample sizes and for data in the full domain of health technologies.

The role of health technology assessment in coverage decisions on newborn screening.

OBJECTIVES: The role and impact of health technology assessment (HTA) in health policy has been widely discussed. Researchers have started to analyze how decisions on coverage of new technologies are made. Although the involvement of HTA may be an indicator of a well established decision process, this hypothesis requires validation. Also, it is not known whether HTA involvement is associated with other characteristics of decision making like participation or transparency. The primary objective of this study was to develop and test statements on the association between the publication of an HTA and coverage decision making for newborn screening tests in European Union countries. METHODS: Five statements were defined on the relative role of HTA during the steps of decision processes: trigger, participation, publication, assessment, and appraisal. For this purpose, data on twenty-two decision processes in the area of newborn screening across Europe were analyzed, defined as a coverage decision for a given disorder in a specific country. Decision processes were compared by whether the decision was accompanied by the publication of an HTA report. To test differences, nonparametric statistical tests were used. RESULTS: The decision steps of trigger, participation and publication differed between the HTA and the non-HTA groups. No clear association between HTA and assessment methods in coverage decision making was identified. CONCLUSIONS: It appeared that there is an association between HTA and coverage decision processes that are more explicit, inclusive, and transparent. It is unclear whether HTA is associated with formal evidence reviews and economic evaluations.

A structured tool to analyse coverage decisions: development and feasibility test in the field of cancer screening and prevention.

OBJECTIVES: The comparison of fourth hurdle processes is challenging because they are heterogeneous and decision practice may deviate from formal process rules. This study applies a published framework consisting of key steps of coverage decision processes to the area of cancer prevention. METHODS: A research design was developed for analysis of case studies on past decision processes. Decisions were identified and information on the process steps was elicited by semi-structured telephone interviews with decision-makers and experts. The scheme was validated with experts from the areas of screening and prevention and fourth hurdle decision making. RESULTS: Indicators for a structured empirical comparison of coverage decisions were derived. Corresponding ordinal rankings were proposed. Details on six decisions about cancer screening (colorectal and prostate cancer) and vaccination against human papillomavirus in Sweden, Austria and Lithuania are presented. CONCLUSIONS: The development of the structured scheme for analysis of coverage decisions allows validation of official statements on decision processes and collection of larger data sets for empirical analysis. However, the semi-structured phone interviews were time-consuming for collecting information on a larger number of decisions. Further validation of the structured scheme and development of a research tool for large-scale empirical studies is still needed.