Balancing the Optimal and the Feasible: A Practical Guide for Setting Up Patient Registries for the Collection of Real-World Data for Health Care Decision Making Based on Dutch Experiences

Groot, Saskia de; Linden, Naomi van der; Franken, M; Blommestein, Hedwig M.; Leeneman, B; Rooijen, Ellen van; Hoeven, J.J.M. van der; Wouters, Michel W. J. M.; Westgeest, Hans M.; Groot, Carin A. Uyl–de

doi:10.1016/j.jval.2016.02.007

Cited by 32 publications

(33 citation statements)

References 27 publications

(27 reference statements)

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“…Moreover, registries need important economical investment to build the structure of the database and the software, disseminate the tool among centers, and maintain and monitor its appropriate use. Registries are receiving increased attention for their potential role in policy-making or decision-making processes for the development of appropriate model of care [3]. In recent years, the worldwide availability of several large multiple sclerosis (MS) databases [4]—combined with a growing ability to collect, share, and analyze large amounts of data—are enabling the conduction of real-world observational studies aimed to identify MS prediction models for poor outcome and treatment response/failure, and to evaluate comparative and long-term effectiveness and safety of disease-modifying treatments in current use 2 , issues that cannot be addressed by RCTs.…”

Section: Introductionmentioning

confidence: 99%

The Italian multiple sclerosis register

Trojano

Bergamaschi²,

Amato

et al. 2018

Neurol Sci

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The past decade has seen extraordinary increase in worldwide availability of and access to several large multiple sclerosis (MS) databases and registries. MS registries represent powerful tools to provide meaningful information on the burden, natural history, and long-term safety and effectiveness of treatments. Moreover, patients, physicians, industry, and policy makers have an active interest in real-world observational studies based on register data, as they have the potential to answer the questions that are most relevant to daily treatment decision-making. In 2014, the Italian MS Foundation, in collaboration with the Italian MS clinical centers, promoted and funded the creation of the Italian MS Register, a project in continuity with the existing Italian MS Database Network set up from 2001. Main objective of the Italian MS Register is to create an organized multicenter structure to collect data of all MS patients for better defining the disease epidemiology, improving quality of care, and promoting research projects in high-priority areas. The aim of this article is to present the current framework and network of the Italian MS register, including the methodology used to improve the quality of data collection and to facilitate the exchange of data and the collaboration among national and international groups.Electronic supplementary materialThe online version of this article (10.1007/s10072-018-3610-0) contains supplementary material, which is available to authorized users.

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Section: Introductionmentioning

confidence: 99%

The Italian multiple sclerosis register

Trojano

Bergamaschi²,

Amato

et al. 2018

Neurol Sci

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“…Due to the complexity and underdiagnosis of PsA, it is important to understand the typical profile of patients with PsA by considering demographics, disease characteristics, medical and family history, lifestyle risk factors, and prior treatment use. Observational studies allow for collection of data in a real-world setting [13, 14]. The Psoriasis Longitudinal Assessment and Registry (PSOLAR) is a large, multicenter, international, longitudinal, disease-based registry with prospective enrollment of approximately 12,000 patients with psoriasis who are receiving, or are candidates for treatment with, systemic therapies for psoriasis [15, 16].…”

Section: Introductionmentioning

confidence: 99%

Demography, baseline disease characteristics, and treatment history of psoriasis patients with self-reported psoriatic arthritis enrolled in the PSOLAR registry

et al. 2018

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Background To evaluate demographics, family history, and previous medication use at enrollment in a subset of psoriasis patients with self-reported psoriatic arthritis (PsA) enrolled in Psoriasis Longitudinal Assessment and Registry (PSOLAR). Methods PSOLAR is an international, prospective, longitudinal, disease-based registry that collects data in patients receiving, or are eligible to receive, systemic or biologic treatments for psoriasis. Baseline demographic, disease characteristics, medical history, and prior medication use at enrollment were evaluated in PSOLAR psoriasis patients self-reporting PsA ( n = 4315); a subset of which had their diagnosis of PsA established by a healthcare provider (HCP; n = 1719); patients with psoriasis only ( n = 7775); and the overall PSOLAR population ( n = 12,090). Results At enrollment, demographic characteristics were distinct between psoriasis patients self-reporting PsA and psoriasis only patients. Of the patients with psoriasis self-reporting PsA, 44.4% had cardiovascular disease (CVD), 26.3% had psychiatric illness, and 3.2% had inflammatory bowel disease (IBD), with each more prevalent than among patients with psoriasis only ( p < 0.001). Overall, 17.5% of psoriasis patients self-reporting PsA had a family history of PsA, 29.8% had used systemic steroids, 39.5% had used nonsteroidal anti-inflammatory drugs, and 83.5% had used biologics. Conclusions Demographics, family history, and previous medication use were generally comparable between “PsA established by a HCP” patients and psoriasis patients self-reporting PsA in the PSOLAR registry, but there were statistical differences compared with the psoriasis only group regarding the prevalence of certain comorbidities (CVD, psychiatric illness, and IBD). These analyses provide important data regarding characteristics of psoriasis patients with self-reported PsA in PSOLAR. Trial registration NCT00508547 . Electronic supplementary material The online version of this article (10.1186/s41927-018-0034-7) contains supplementary material, which is available to authorized users.

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“…Possible explanations for these differences are inclusion of other cost components or sources, other methods of extrapolation of survival or utilities beyond the follow-up time of the trial, variations in time horizon, different model structures, included complications, etc. [ 25 , 26 , 38 , 39 ]. The baseline characteristics of populations differed between studies, especially operable risk.…”

Section: Discussionmentioning

confidence: 99%

Systematic review of model-based economic evaluations of heart valve implantations

2017

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ObjectiveTo review the evidence on the cost-effectiveness of heart valve implantations generated by decision analytic models and to assess their methodological quality.MethodsA systematic review was performed including model-based cost-effectiveness analyses of heart valve implantations. Study and model characteristics and cost-effectiveness results were extracted and the methodological quality was assessed using the Philips checklist.ResultsFourteen decision-analytic models regarding the cost-effectiveness of heart valve implantations were identified. In most studies transcatheter aortic valve implantation (TAVI) was cost-effective compared to standard treatment (ST) in inoperable or high-risk operable patients (ICER range 18,421–120,779 €) and in all studies surgical aortic valve replacement (SAVR) was cost-effective compared to ST in operable patients (ICER range 14,108–40,944 €), but the results were not consistent on the cost-effectiveness of TAVI versus SAVR in high-risk operable patients (ICER range: dominant to dominated by SAVR). Mechanical mitral valve replacement (MVR) had the lowest costs per success compared to mitral valve repair and biological MVR. The methodological quality of the studies was moderate to good.ConclusionThis review showed that improvements can be made in the description and justification of methods and data sources, sensitivity analysis on extrapolation of results, subgroup analyses, consideration of methodological and structural uncertainty, and consistency (i.e. validity) of the models. There are several opportunities for future decision-analytic models of the cost-effectiveness of heart valve implantations: considering heart valve implantations in other valve positions besides the aortic valve, using a societal perspective, and developing patient-simulation models to investigate the impact of patient characteristics on outcomes.Electronic supplementary materialThe online version of this article (doi:10.1007/s10198-017-0880-z) contains supplementary material, which is available to authorized users.

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Balancing the Optimal and the Feasible: A Practical Guide for Setting Up Patient Registries for the Collection of Real-World Data for Health Care Decision Making Based on Dutch Experiences

Abstract: It is crucial to set up an efficient patient registry that serves its aims by collecting the right data of the right patient in the right way. It can be expected that patient registries will become the new standard alongside randomized controlled trials due to their unique value.

Cited by 32 publications

References 27 publications

The Italian multiple sclerosis register

The Italian multiple sclerosis register

Demography, baseline disease characteristics, and treatment history of psoriasis patients with self-reported psoriatic arthritis enrolled in the PSOLAR registry

Systematic review of model-based economic evaluations of heart valve implantations

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