Search

Purpose: Trabecular bone score (TBS) is a grey-level textural measurement acquired from dual-energy X-ray absorptiometry lumbar spine images and is a validated index of bone microarchitecture. In 2015, a Working Group of the European Society on Clinical and Economic Aspects of Osteoporosis, Osteoarthritis and Musculoskeletal Diseases (ESCEO) published a review of the TBS literature, concluding that TBS predicts hip and major osteoporotic fracture, at least partly independent of bone mineral density (BMD) and clinical risk factors. It was also concluded that TBS is potentially amenable to change as a result of pharmacological therapy. Further evidence on the utility of TBS has since accumulated in both primary and secondary osteoporosis, and the introduction of FRAX and BMD T-score adjustment for TBS has accelerated adoption. This position paper therefore presents a review of the updated scientific literature and provides expert consensus statements and corresponding operational guidelines for the use of TBS. Methods: An Expert Working Group was convened by the ESCEO and a systematic review of the evidence undertaken, with defined search strategies for four key topics with respect to the potential use of TBS: (1) fracture prediction in men and women; (2) initiating and monitoring treatment in postmenopausal osteoporosis; (3) fracture prediction in secondary osteoporosis; and (4) treatment monitoring in secondary osteoporosis. Statements to guide the clinical use of TBS were derived from the review and graded by consensus using the Grades of Recommendation, Assessment, Development and Evaluation (GRADE) approach. Results: A total of 96 articles were reviewed and included data on the use of TBS for fracture prediction in men and women, from over 20 countries. The updated evidence shows that TBS enhances fracture risk prediction in both primary and secondary osteoporosis, and can, when taken with BMD and clinical risk factors, inform treatment initiation and the choice of antiosteoporosis treatment. Evidence also indicates that TBS provides useful adjunctive information in monitoring treatment with long-term denosumab and anabolic agents. All expert consensus statements were voted as strongly recommended. Conclusion: The addition of TBS assessment to FRAX and/or BMD enhances fracture risk prediction in primary and secondary osteoporosis, adding useful information for treatment decision-making and monitoring. The expert consensus statements provided in this paper can be used to guide the integration of TBS in clinical practice for the assessment and management of osteoporosis. An example of an operational approach is provided in the appendix. Summary: This position paper presents an up-to-date review of the evidence base, synthesised through expert consensus statements, which informs the implementation of Trabecular Bone Score in clinical practice. [ABSTRACT FROM AUTHOR]

Purpose: Over the last few years, the share of public spending for orphan drugs (ODs) has increased in several western countries, raising concern on the exemptions granted to this sector with respect to the implementation of health technology assessment (HTA) principles. The aim of this paper is to shed light on both the HTA criteria adopted and the international agreements implemented in the OD regulation, given the new challenges imposed on western countries by a growing number of therapies for rare diseases. Methods: We carried out a literature review to analyse the development of the international debate on the adaptability of HTA criteria for the OD assessment and regulation. The time span lies between January 1990 and May 2018, and the policies considered relate to both market authorization and reimbursement decisions within western countries. We focus specifically on HTA criteria in some of the dimensions included in the Core Model of the European net for HTA (EUnetHTA). Results: OD high prices, the absence of clarity on the possible high revenues realized by the distribution of a new OD outside the national borders, the risk that – once marketed – a new OD can be used to treat common diseases, are all issues that raise concern on OD regulation and have to be carefully monitored by policymakers in the next future. Conclusions: Across western countries, the preferential track granted to ODs in the implementation of HTA principles is not homogeneous, but fragmented and differentiated. The need for common rules at an international level is underlined, with a view to assessing the sustainability of a sector which, due to this regulatory void, can lend itself to producers' strategic and opportunistic behaviours. [ABSTRACT FROM AUTHOR]

Background: Although health technology assessment (HTA) systems base their decision making process either on economic evaluations or comparative clinical benefit assessment, a central aim of recent approaches to value measurement, including value based assessment and pricing, points towards the incorporation of supplementary evidence and criteria that capture additional dimensions of value.Objective: To study the practices, processes and policies of value-assessment for new medicines across eight European countries and the role of HTA beyond economic evaluation and clinical benefit assessment.Methods: A systematic (peer review and grey) literature review was conducted using an analytical framework examining: (1) 'Responsibilities and structure of HTA agencies'; (2) 'Evidence and evaluation criteria considered in HTAs'; (3) 'Methods and techniques applied in HTAs'; and (4) 'Outcomes and implementation of HTAs'. Study countries were France, Germany, England, Sweden, Italy, Netherlands, Poland and Spain. Evidence from the literature was validated and updated through two rounds of feedback involving primary data collection from national experts.Results: All countries assess similar types of evidence; however, the specific criteria/endpoints used, their level of provision and requirement, and the way they are incorporated (e.g. explicitly vs. implicitly) varies across countries, with their relative importance remaining generally unknown. Incorporation of additional 'social value judgements' (beyond clinical benefit assessment) and economic evaluation could help explain heterogeneity in coverage recommendations and decision-making.Conclusion: More comprehensive and systematic assessment procedures characterised by increased transparency, in terms of selection of evaluation criteria, their importance and intensity of use, could lead to more rational evidence-based decision-making, possibly improving efficiency in resource allocation, while also raising public confidence and fairness. [ABSTRACT FROM AUTHOR]