ContextThe severity of Sjögren’s syndrome has been evaluated using a wide variety of clinical measures and patient-reported outcomes (PROs). This may contribute to the lack of clarity concerning the burden of Sjögren’s from the patient perspective.ObjectiveTo perform a comprehensive peer-reviewed literature analysis of the patient aspects of Sjögren’s, focusing on PROs, to investigate the complexity underlying the evaluation of the syndrome and to elucidate the discordance between the different measures.MethodsWe searched Embase for articles published between January 2005 and September 2015. Research articles, clinical and diagnostic reviews, and validation studies with a focus on patient aspects of Sjögren’s were selected as the primary information source.Results157 articles met the eligibility criteria. A wide variety of assessment measures used to evaluate glandular, extraglandular and functional domains were observed. Many different, non-validated Visual Analogue Scales, with a wide range of anchor words, were used in the quantification of Sjögren’s disease burden, impeding comparisons between studies. Relatively few clinical trials of drug therapies used validated scales: European League Against Rheumatism Sjögren’s Syndrome Patient Reported Index was used most often for symptom assessment and 36 Item Short Form Survey for quality of life (QoL).ConclusionA wide range and diversity of measures are used to evaluate the patient burden of Sjögren’s; most are not validated for use in this disease. PRO endpoints, validated specifically in Sjögren’s, that demonstrate improvement are needed. These measures should focus on QoL aspects important to patients and will most likely involve gauging change in function rather than patient-reported symptoms.
Objectives: Value and health technology assessment (V/HTA) is often used in clinical, access, and reimbursement decisions. V/ HTA data-source selection may not be transparent, which is a necessary element for stakeholder understanding and trust and for fostering accountability among decision makers. Peer review is considered one mechanism for judging data trustworthiness. Our objective was (1) to use publicly available documentation of V/HTA methods to identify requirements for inclusion of peer-reviewed evidence sources, (2) to compare and contrast US and non-US approaches, and (3) to assess evidence sources used in published V/HTA reports.Methods: Publicly available methods documentation from 11 V/HTA organizations in North America and Europe were manually searched and abstracted for descriptions of requirements and recommendations regarding search strategy and evidence-source selection. The bibliographies of a subset of V/HTA reports published in 2018 were manually abstracted for evidence-source types used in each.Results: Heterogeneity in evidence-source retrieval and selection was observed across all V/HTA organizations, with more pronounced differences between US and non-US organizations. Not all documentation of organizations' methods address the evidence-source selection processes (7 of 11), and few explicitly reference peer-reviewed sources (3 of 11). Documentation of the evidence-source selection strategy was inconsistent across reports (6 of 13), and the level of detail provided varied across organizations. Some information on evidence-source selection was often included in confidential documentation and was not publicly available.Conclusions: Disparities exist among V/HTA organizations in requirements and guidance regarding evidence-source selection. Standardization of evidence-source selection strategies and documentation could help improve V/HTA transparency and has implications for decision making based on report findings.
There is currently a heightened need for transparency in pharmaceutical sectors. The inclusion of real-world (RW) evidence, in addition to clinical trial evidence, in decision-making processes, was an important step forward toward a more inclusive established value proposition. This advance has introduced new transparency challenges. Increasing transparency is a critical step toward accelerating improvement in type, quality, and access to data, regardless of whether these originate from clinical trials or from RW studies. However, so far, advances in transparency have been relatively restricted to clinical trials, and there remains a lack of similar expectations or standards of transparency concerning the generation and reporting of RW data. This perspective paper aims to highlight the need for transparency concerning RW studies, data, and evidence across health care sectors, to identify areas for improvement, and provide concrete recommendations and practices for the future. Specific issues are discussed from different stakeholder perspectives, culminating in recommended actions, from individual stakeholder perspectives, for improved RW study, data, and evidence transparency. Furthermore, a list of potential guidelines for consideration by stakeholders is proposed. While recommendations from different stakeholder perspectives are made, true transparency in the processes involved in the generation, reporting, and use of RW evidence will require a concerted effort from all stakeholders across health care sectors.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.
hi@scite.ai
10624 S. Eastern Ave., Ste. A-614
Henderson, NV 89052, USA
Copyright © 2024 scite LLC. All rights reserved.
Made with 💙 for researchers
Part of the Research Solutions Family.