Series: Pragmatic trials and real world evidence: Paper 4. Informed consent

Kalkman, Shona; Thiel, Ghislaine J. M. W. van; Zuidgeest, Mira G. P.; Goetz, Iris; Pfeiffer, Boris M; Grobbee, Diederick E.; Delden, Johannes J. M. van

doi:10.1016/j.jclinepi.2017.03.019

Cited by 34 publications

(27 citation statements)

References 32 publications

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“…However, both the Declaration of Helsinki and the ICH Guidance on Good Clinical Practice are clear in that the subject must receive detailed explanations on the experimental nature and design of the trial, including its purpose, the tested treatments and the probability for random assignment to each treatment [18, 19]. Also another study mentions the importance of informed consent in pragmatic randomization [20]. Whether a general acceptance of data collection in a RDR for comparative use may overcome such recommendations can be controversial.…”

Section: Resultsmentioning

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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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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Section: Resultsmentioning

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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“…Key challenges for obtaining informed consent from patient‐participants in both genomic medicine research and PCTs have been described. Informed consent for genomic medicine studies are usually more involved as they often entail less rigorously tested interventions and must address specific issues associated with how, when, and in what form potentially sensitive genetic information should be integrated into the EHR or delivered to patients .…”

Section: Discussionmentioning

confidence: 99%

“…Informed consent for genomic medicine studies are usually more involved as they often entail less rigorously tested interventions and must address specific issues associated with how, when, and in what form potentially sensitive genetic information should be integrated into the EHR or delivered to patients . Lengthy informed consent procedures may limit a trial's degree of pragmatism and have the potential to bias the engagement of some groups . Most PCTs outside genomic medicine, on the other hand, use highly vetted interventions, physician oversight, or interventions whose probability of harm is minimal .…”

Section: Discussionmentioning

confidence: 99%

“…40 Lengthy informed consent procedures may limit a trial's degree of pragmatism and have the potential to bias the engagement of some groups. 41 Most PCTs outside genomic medicine, on the other hand, use highly vetted interventions, physician oversight, or interventions whose probability of harm is minimal. 42 The I-PICC Study navigated the sometimes competing demands of informed consent and pragmatism by consenting a pool of eligible patients through letters and telephone calls, offloading the clinical encounter, but ultimately allowing providers to enroll individual patients at the point of care by signing an order for SLCO1B1 testing on an existing clinical blood sample.…”

Section: Pragmatic Trials In Genomic Medicinementioning

confidence: 99%

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Pragmatic Trials in Genomic Medicine: The Integrating Pharmacogenetics In Clinical Care (I‐PICC) Study

Brunette

Miller

Majahalme

et al. 2019

Clinical Translational Sci

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Pragmatic clinical trials (PCTs) have an established presence in clinical research and yet have only recently garnered attention within the landscape of genomic medicine. Using the PRagmatic‐Explanatory Continuum Indicator Summary 2 (PRECIS‐2) as a framework, this paper illustrates the application of PCT principles to The Integrating Pharmacogenetics In Clinical Care (I‐PICC) Study, a trial of pharmacogenetic testing prior to statin initiation for cardiovascular disease prevention in primary care. The trial achieved high engagement with providers (85% enrolled of those approached) and enrolled a representative sample of participants for which statin therapy would be recommended. The I‐PICC Study has a high level of pragmatism, which should enhance the generalizability of its findings. The PRECIS‐2 may be useful in the design and evaluation of PCTs of genomic medicine interventions, contributing to the generation of evidence that can bridge the gap between genomics innovation and clinical adoption.

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“…proposed [34]. The concept of a learning health care system also has emerged in which knowledge generation is ''a natural outgrowth and product of the health care delivery process'' [35].…”

Section: Does Not Change Routine Clinical Practicementioning

confidence: 99%

Series: Pragmatic trials and real world evidence: Paper 8. Data collection and management

Meinecke

Welsing

Kafatos

et al. 2017

Journal of Clinical Epidemiology

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Pragmatic trials can improve our understanding of how treatments will perform in routine practice. In a series of eight papers, the GetReal Consortium has evaluated the challenges in designing and conducting pragmatic trials and their specific methodological, operational, regulatory, and ethical implications. The present final paper of the series discusses the operational and methodological challenges of data collection in pragmatic trials. A more pragmatic data collection needs to balance the delivery of highly accurate and complete data with minimizing the level of interference that data entry and verification induce with clinical practice. Furthermore, it should allow for the involvement of a representative sample of practices, physicians, and patients who prescribe/receive treatment in routine care. This paper discusses challenges that are related to the different methods of data collection and presents potential solutions where possible. No one-size-fits-all recommendation can be given for the collection of data in pragmatic trials, although in general the application of existing routinely used data-collection systems and processes seems to best suit the pragmatic approach. However, data access and privacy, the time points of data collection, the level of detail in the data, and the lack of a clear understanding of the data-collection process were identified as main challenges for the usage of routinely collected data in pragmatic trials. A first step should be to determine to what extent existing health care databases provide the necessary study data and can accommodate data collection and management. When more elaborate or detailed data collection or more structured follow-up is required, data collection in a pragmatic trial will have to be tailor-made, often using a hybrid approach using a dedicated electronic case report form (eCRF). In this case, the eCRF should be kept as simple as possible to reduce the burden for practitioners and minimize influence on routine clinical practice.

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Series: Pragmatic trials and real world evidence: Paper 4. Informed consent

Cited by 34 publications

References 32 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

Pragmatic Trials in Genomic Medicine: The Integrating Pharmacogenetics In Clinical Care (I‐PICC) Study

Series: Pragmatic trials and real world evidence: Paper 8. Data collection and management

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