21st Century Patient Registries, Ebook Addendum to Registries for Evaluating Patient Outcomes: A User's Guide, 3rd Edition

BackgroundLow prevalence, lack of knowledge about the disease course, and phenotype heterogeneity hamper the development of drugs for rare diseases. Rare disease registries (RDRs) can be helpful by playing a role in understanding the course of the disease, and providing information necessary for clinical trial design, if designed and maintained properly. We describe the potential applications of a RDR and what type of information should be incorporated to support the design of clinical trials in the process of drug development, based on a broad inventory of registry experience. We evaluated two existing RDRs in more detail to check the completeness of these RDRs for trial design.ResultsBefore and during the application for regulatory approval a RDR can improve the efficiency and quality in clinical trial design by informing the sample size calculation and expected disease course. In exceptional circumstances information from RDRs has been used as historical controls for a one-armed clinical trial, and high quality RDRs may be used for registry-based randomized controlled trials. In the post marketing phase of (conditional) drug approval a disease-specific RDR is likely to provide more relevant information than a product-specific registry.ConclusionsA RDR can be very helpful to improve the efficiency and quality of clinical trial design in several ways. To enable the applicability and optimal use of a RDR longitudinal data collection is indispensable, and specific data collection, prepared for repeated measurement, is needed. The developed checklist can help to define the appropriate variables to include. Attention should be paid to the inclusion of patient-relevant outcome measures in the RDR from the start. More research and experience is needed on the possibilities and limitations of combining RDR information with clinical trial data to maximize the availability of relevant evidence for regulatory decisions in rare diseases.

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“…Several initiatives have been launched for standardization of outcome measurement [11]. One of these is the COMET initiative [42].…”

Section: Discussionmentioning

confidence: 99%

Rare disease registries: potential applications towards impact on development of new drug treatments

Weide

Gaasterland

Roes

et al. 2018

Orphanet J Rare Dis

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“…The Agency for Healthcare Research and Quality [21] reported that hospitals are most likely to participate in a quality improvement registry because of interest in the research question or because it helps them fulfill reimbursement or regulatory requirements. This report also cited staff time and costs for data collection as a key barrier to participation.…”

Section: Discussionmentioning

confidence: 99%

Hospital recruitment for a pragmatic cluster-randomized clinical trial: Lessons learned from the COMPASS study

Johnson

Jones

Duncan

et al. 2018

Trials

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BackgroundPragmatic randomized clinical trials are essential to determine the effectiveness of interventions in “real-world” clinical practice. These trials frequently use a cluster-randomized methodology, with randomization at the site level. Despite policymakers’ increased interest in supporting pragmatic randomized clinical trials, no studies to date have reported on the unique recruitment challenges faced by cluster-randomized pragmatic trials. We investigated key challenges and successful strategies for hospital recruitment in the Comprehensive Post-Acute Stroke Services (COMPASS) study.MethodsThe COMPASS study is designed to compare the effectiveness of the COMPASS model versus usual care in improving functional outcomes, reducing the numbers of hospital readmissions, and reducing caregiver strain for patients discharged home after stroke or transient ischemic attack. This model integrates early supported discharge planning with transitional care management, including nurse-led follow-up phone calls after 2, 30, and 60 days and an in-person clinic visit at 7–14 days involving a functional assessment and neurological examination. We present descriptive statistics of the characteristics of successfully recruited hospitals compared with all eligible hospitals, reasons for non-participation, and effective recruitment strategies.ResultsWe successfully recruited 41 (43%) of 95 eligible North Carolina hospitals. Leading, non-exclusive reasons for non-participation included: insufficient staff or financial resources (n = 33, 61%), lack of health system support (n = 16, 30%), and lack of support of individual decision-makers (n = 11, 20%). Successful recruitment strategies included: building and nurturing relationships, engaging team members and community partners with a diverse skill mix, identifying gatekeepers, finding mutually beneficial solutions, having a central institutional review board, sharing published pilot data, and integrating contracts and review board administrators.ConclusionsAlthough we incorporated strategies based on the best available evidence at the outset of the study, hospital recruitment required three times as much time and considerably more staff than anticipated. To reach our goal, we tailored strategies to individuals, hospitals, and health systems. Successful recruitment of a sufficient number and representative mix of hospitals requires considerable preparation, planning, and flexibility. Strategies presented here may assist future trial organizers in implementing cluster-randomized pragmatic trials.Trial registrationClinicaltrials.gov, NCT02588664. Registered on 23 October 2015.Electronic supplementary materialThe online version of this article (doi:10.1186/s13063-017-2434-1) contains supplementary material, which is available to authorized users.

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“…In this quality improvement framework, the focus of outcomes is on the impact of care on the person receiving it. Ideally the assessment of outcome of care should always reflect what patients actually experience and consider important [26]. Thus HROM are measures or scales or instruments that are designed to capture outcomes (domains and constructs) in a health care context.…”

Section: Introductionmentioning

confidence: 99%

Protocol for a Delphi consensus exercise to identify a core set of criteria for selecting health related outcome measures (HROM) to be used in primary health care

et al. 2018

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BackgroundPromoting the collection and use of health related outcome measures (HROM) in daily practice has long been a goal for improving and assessing the effectiveness of care provided to patients. However, there has been a lack of consensus on what criteria to use to select outcomes or instruments, particularly in the context of primary health care settings where patients present with multiple concurrent health conditions and interventions are whole-health and person-focused. The purpose of this proposed study is to undertake a formal consensus exercise to establish criteria for selecting HROM (including patient-reported (PRO or PROM), observer-reported (ObsR)), clinician-reported (ClinRO) and performance related outcomes (PerfO) for use in shared decision-making, or in assessing, screening or monitoring health status in primary health care settings.MethodsA Delphi consensus online survey will be developed. Criteria for the Delphi panel participants to consider were selected from a targeted literature search. These initial criteria (n = 35) were grouped into four categories within which items will be presented in the Delphi survey, with the option to suggest additional items. Panel members invited to participate will include primary health care practitioners and administrators, policy-makers, researchers, and experts in HROM development; patients will be excluded. Standard Delphi methodology will be employed with an expectation of at least 3 rounds to achieve consensus (75% agreement). As the final list of criteria for selecting HROM emerges, panel members will be asked to provide opinions about potential weighting of items. The Delphi survey was approved by the Ethics Committee in the Faculty of Health Sciences at McMaster University.DiscussionPrevious literature establishing criteria for selecting HROM were developed with a focus on patient reported outcomes, psychological/ behavioural outcomes or outcomes for minimum core outcome sets in clinical trials. Although helpful, these criteria may not be applicable and feasible for application in a primary health care context where patients with multi-morbidity and complex interventions are typical and the constraints of providing health services differ from those in research studies. The findings from this Delphi consensus study will address a gap for establishing consensus on criteria for selecting HROM for use across primary health care settings.

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21st Century Patient Registries, Ebook Addendum to Registries for Evaluating Patient Outcomes: A User's Guide, 3rd Edition

Cited by 269 publications

References 9 publications

Rare disease registries: potential applications towards impact on development of new drug treatments

Rare disease registries: potential applications towards impact on development of new drug treatments

Hospital recruitment for a pragmatic cluster-randomized clinical trial: Lessons learned from the COMPASS study

Protocol for a Delphi consensus exercise to identify a core set of criteria for selecting health related outcome measures (HROM) to be used in primary health care

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