Exploring the opportunities for alignment of regulatory postauthorization requirements and data required for performance-based managed entry agreements.

Performance-based managed entry agreements (PB-MEAs) might allow patient access to new medicines, but practical hurdles make competent authorities for pricing and reimbursement (CAPR) reluctant to implement PB-MEAs. We explored if the feasibility of PB-MEAs might improve by better aligning regulatory postauthorization requirements with the data generation of PB-MEAs and by active collaboration and data sharing. Reviewers from seven CAPRs provided structured assessments of the information available at the European Medicines Agency (EMA) Web site on regulatory postauthorization requirements for fifteen recently authorized products. The reviewers judged to what extent regulatory postauthorization studies could help implement PB-MEAs by addressing uncertainty gaps. Study domains assessed were: patient population, intervention, comparators, outcomes, time horizon, anticipated data quality, and anticipated robustness of analysis. Reviewers shared general comments about PB-MEAs for each product and on cooperation with other CAPRs. Reviewers rated regulatory postauthorization requirements at least partly helpful for most products and across domains except the comparator domain. One quarter of responses indicated that public information provided by the EMA was insufficient to support the implementation of PB-MEAs. Few PB-MEAs were in place for these products, but the potential for implementation of PB-MEAs or collaboration across CAPRs was seen as more favorable. Responses helped delineate a set of conditions where PB-MEAs may help reduce uncertainty. In conclusion, PB-MEAs are not a preferred option for CAPRs, but we identified conditions where PB-MEAs might be worth considering. The complexities of implementing PB-MEAs remain a hurdle, but collaboration across silos and more transparency on postauthorization studies could help overcome some barriers.

Impact of Complications and Comorbidities on the Intensive Care Length of Stay after Aneurysmal Subarachnoid Haemorrhage.

In this observational study, we analysed a cohort of 164 subarachnoid haemorrhage survivors (until discharge from intensive care) with the aim to detect factors that influence the length of stay (LOS) in intensive care with multiple linear regression methods. Moreover, binary logistic regression methods were used to examine whether the time in intensive care is a predictor of outcome after 1 year. The clinical 1-year outcome was measured prospectively in a 12-month follow-up by telephone interview and categorised by the modified Rankin Scale (mRS). Patients who died during their stay in intensive care were excluded. Complications like pneumonia (ß = 5.11; 95% CI = 1.75-8.46; p = 0.0031), sepsis (ß = 9.54; 95% CI = 3.27-15.82; p = 0.0031), hydrocephalus (ß = 4.63; 95% CI = 1.82-7.45; p = 0.0014), and delayed cerebral ischemia (DCI) (ß = 3.38; 95% CI = 0.19-6.56; p = 0.038) were critical factors depending the LOS in intensive care as well as decompressive craniectomy (ß = 5.02; 95% CI = 1.35-8.70; p = 0.0077). All analysed comorbidities such as hypertension, diabetes, hypothyroidism, cholesterinemia, and smoking history had no significant impact on the LOS in intensive care. LOS in intensive care (OR = 1.09; 95% CI = 1.03-1.15; p = 0.0023) as well as WFNS grade (OR = 3.72; 95% CI = 2.23-6.21; p < 0.0001) and age (OR = 1.06; 95% CI = 1.02-1.10; p = 0.0061) were significant factors that had an impact on the outcome after 1 year. Complications in intensive care but not comorbidities are associated with higher LOS in intensive care. LOS in intensive care is a modest but significant predictor of outcomes after subarachnoid haemorrhage.

Data Rich, Information Poor: Can We Use Electronic Health Records to Create a Learning Healthcare System for Pharmaceuticals?

Judicious use of real-world data (RWD) is expected to make all steps in the development and use of pharmaceuticals more effective and efficient, including research and development, regulatory decision making, health technology assessment, pricing, and reimbursement decisions and treatment. A "learning healthcare system" based on electronic health records and other routinely collected data will be required to harness the full potential of RWD to complement evidence based on randomized controlled trials. We describe and illustrate with examples the growing demand for a learning healthcare system; we contrast the exigencies of an efficient pharmaceutical ecosystem in the future with current deficiencies highlighted in recently published Organisation for Economic Co-operation and Development (OECD) reports; and we reflect on the steps necessary to enable the transition from healthcare data to actionable information. A coordinated effort from all stakeholders and international cooperation will be required to increase the speed of implementation of the learning healthcare system, to everybody's benefit.

Prices For Cardiac Implant Devices May Be Up To Six Times Higher In The US Than In Some European Countries.

Medical devices are estimated to account for 6 percent of health expenditures in the US and 7 percent in European Union (EU) countries. Cardiac implants are a large segment of the market, but little is known about their prices. Using 2006-14 data from a large hospital panel survey, this article provides a systematic comparison of prices of cardiac implants between the US and four EU countries. The data reveal that prices were two to six times higher in the US than in Germany, where cardiac implants were generally the cheapest. Prices also varied between EU countries. Prices of coronary stents and pacemakers were higher in France and Italy than in the UK, where prices were similar to those in Germany. The data also confirmed findings of prior studies that found significant price variation between hospitals within a given country. This study found that within- and between-country variations were similar in magnitude. While these findings can help assess whether technology-related policies effectively control the prices of cardiac devices, future research is necessary to establish a causal relationship between prices and several possible explanatory factors and help policy makers understand which mechanisms are available to control device prices.

Health policy in times of austerity-A conceptual framework for evaluating effects of policy on efficiency and equity illustrated with examples from Europe since 2008.

The objective of this paper is to provide a framework for evaluation of changes in health policy against overarching health system goals. We propose a categorisation of policies into seven distinct health system domains. We then develop existing analytical concepts of insurance coverage and cost-effectiveness further to evaluate the effects of policies in each domain on equity and efficiency. The framework is illustrated with likely effects of policy changes implemented in a sample of European countries since 2008. Our illustrative analysis suggests that cost containment has been the main focus and that countries have implemented a mix of measures that are efficient or efficiency neutral. Similarly, policies are likely to have mixed effects on equity. Additional user charges were a common theme but these were frequently accompanied by additional exemptions, making their likely effects on equity difficult to evaluate. We provide a framework for future, and more detailed, evaluations of changes in health policy.

Prone Positioning in Cardiac Surgery: For Many, But Not for Everyone.

Prone positioning is a therapeutic maneuver to improve arterial oxygenation in patients with acute lung injury that is not implemented in most centers performing adult cardiac surgery. The aim of this study was to review our experience with prone positioning to assess the effects of this maneuver in patients with postoperative acute respiratory failure. From 2010-2014, 127 adult patients with postoperative acute respiratory failure were treated with prone positioning in addition to specific therapy. Univariate and multivariate logistic regression analyses were performed to identify independent risk factors associated with in-hospital mortality. In-hospital mortality was 22.8% (n = 29). No significant differences were observed in preoperative risk factors between patients who survived (S) and those who died (D), except for age (62.7 ± 11.2 vs 70.2 ± 11.3; P = 0.007-at multivariate analysis P = 0.03, odds ratio [OR] = 1.1/year). Preproning values of PaO2/FiO2 were significantly different between groups (D vs S: 115 ± 46 vs 150 ± 56; P = 0.006), but only preproning FiO2 remained highly significant at multivariate analysis (D vs S: 0.82 ± 0.18 vs 0.67 ± 0.16; P = 0.001, OR = 1.07; with FiO2 > 0.75 vs < 75, OR = 19.6). D showed a higher improvement of PaO2/FiO2 immediately after prone positioning (207 ± 100 vs 219 ± 90, P = 0.56; within-group analysis between preproning and 1 hour after proning: S-P = 0.49, D-P = 0.019; at 12 hours: 286 ± 123 vs 240 ± 120, P = 0.06; within-group analysis between 1 hour and 12 hours after proning: S-P = 0.15; D-P = 0.17; between groups-P = 0.05). D had higher peak WBC count (26 ± 9.8 vs 17.7 ± 5.9×103/mL; P = 0.0001) and a higher rate of low output syndrome (15 vs 9 patients-51.7% vs 9.2%; P = 0.0001). At multivariate analysis, white blood cell count: P = 0.005, OR = 1.11/103 white blood cell; low output syndrome: P = 0.0002, OR = 20.5. In conclusion, our results show that prone positioning, if performed early, is a safe and effective adjunct measure for patients with postoperative acute hypoxemic respiratory failure of noncardiogenic origin.

Commissioning for equity in the NHS: rhetoric and practice.

BACKGROUND: This paper reviews evidence on equity as a policy goal of resource allocation in the English NHS, focussing on the role of clinical commissioning groups (CCGs) as purchasers of health services since 2013 and their capacity to achieve equity through the process of commissioning. SOURCES OF DATA: A systematic search of literature published since 1990 and review of grey literature, including policy documents published by CCGs and other organizations in the healthcare system. AREAS OF AGREEMENT: Despite a stated policy commitment to equity of access in the NHS, the 2012 reforms have created a structure that allows and encourages great variation between devolved purchasers of care. AREAS OF CONTROVERSY: Evidence suggests that CCGs, which are structurally separated from public health, have limited capacity and incentives to commission for equity. Concepts of equity of access and health inequalities lack consistent definitions and may not be implemented. However, it remains unclear whether variation between CCGs implies inequity. GROWING POINTS: The 2012 reforms have sought to contain costs and improve quality, thus achieving efficiency gains, while equity has remained an afterthought. The NHS should be expected to work towards equity of access to healthcare and can contribute to reducing health inequalities; however to achieve a more equitable distribution of health, wider social policies are also needed. AREAS TIMELY FOR DEVELOPING RESEARCH: Additional assessments of structural capacity should be complemented by further development of indicators of equity of access and studies that quantify inequities. Research should also explore how an equity principle can be embedded in commissioning, which currently revolves around cost containment and efficiency.