A randomized pragmatic care trial on endovascular acute stroke interventions (EASI): criticisms, responses, and ethics of integrating research and clinical care

2021

Trials

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Background The current research-care separation was introduced to protect patients from explanatory studies designed to gain knowledge for future patients. Care trials are all-inclusive pragmatic trials integrated into medical practice, with no extra tests, risks, or cost, and have been designed to guide practice under uncertainty in the best medical interest of the patient. Proposed revision Patients need a distinction between validated care, previously verified to provide better outcomes, and promising but unvalidated care, which may include unnecessary or even harmful interventions. While validated care can be practiced normally, unvalidated care should only be offered within declared pragmatic care research, designed to protect patients from harm. The validated/unvalidated care distinction is normative, necessary to the ethics of medical practice. Care trials, which mark the distinction and allow the tentative use of promising interventions necessarily involve patients, and thus the design and conduct of pragmatic care research must respect the overarching rule of care ethics “to always act in the best medical interest of the patient.” Yet, unvalidated interventions offered in contexts of medical uncertainty cannot be prescribed or practiced as if they were validated care. The medical interests of current patients are best protected when unvalidated practices are restricted to a care trial protocol, with 1:1 random allocation (or “hemi-prescription”) versus previously validated care, to optimize potential benefits and minimize risks for each patient. Conclusion Pragmatic trials can regulate medical practice by providing (i) a transparent demarcation between unvalidated and validated care; (ii) norms of medical conduct when using tests and interventions of yet unknown benefits in practice; and eventually (iii) a verdict regarding optimal care.

Section: Care Research Is a Work In Progressmentioning

confidence: 99%

Section: Care Research Is a Work In Progressmentioning

confidence: 99%

Ethical care requires pragmatic care research to guide medical practice under uncertainty

Darsaut

2021

Trials

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“…Integrating a pragmatic trial into medical practice requires adjustments on both the research and care fronts: on the one hand, the trial design must be adapted to offer care in the best interest of each patient, while clinical practice must also be disciplined to transparently acknowledge the current uncertainty and to modify the medical or surgical action accordingly. Unsurprisingly, care trials have been met with resistance from both sides: the pragmatic design adapted to care has been criticized by conventional trialists and the randomized allocation that protects patients from the preventable harm related to prescribing unvalidated interventions remains poorly accepted by the community of clinicians and patients [ 47 , 50 – 53 ]. Many physicians declined participation or did not enroll patients because they preferred to use case-by-case reasoning rather than submit patients to randomized allocation of treatment options, and many patients refuse participation because they wanted their doctor to “rise above the uncertainty” and choose the best option for their particular circumstances.…”

Section: Difficultiesmentioning

confidence: 99%

“…Manuscripts reporting our accumulating experience with care trials have repeatedly been turned down before eventual acceptance for publication [ 35 , 41 , 43 – 45 ], in spite of being rigorously reported using the CONSORT statement [ 45 , 56 ]. A detailed example of criticisms from reviewers has been published [ 47 ]. Many reviewers were concerned by the participating centers’ and physicians’ expertise, claiming that poor selection of clinicians could explain trial results that were not as good as results obtained in previously published (but very likely biased) case series.…”

Section: Publicationsmentioning

confidence: 99%

Practicing outcome-based medical care using pragmatic care trials

Darsaut

2020

Trials

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The current separation between medical research and care is an obstacle to essential aspects of good medical practice: the verification that care interventions actually deliver the good outcomes they promise, and the use of scientific methods to optimize care under uncertainty. Pragmatic care trials have been designed to address these problems. Care trials are all-inclusive randomized trials integrated into care. Every item of trial design is selected in the best medical interest of participating patients. Care trials can eventually show what constitutes good medical practice based on patient outcomes. In the meantime, care trials give clinicians and patients the scientific methods necessary for optimization of medical care when no one really knows what to do. We report the progress of 9 randomized care trials that were used to guide the endovascular or surgical management of 1212 patients with acute stroke, intracranial aneurysms, and arteriovenous malformations in a single center in an elective or acute care context. Care trials were used to address long-standing dilemmas regarding rival medical, surgical, or endovascular management options or to offer innovative instead of standard treatments. The trial methodology, by replacing unrepeatable treatment decisions by 1:1 randomized allocation whenever reliable knowledge was not available, had an immediate impact, transforming unverifiable dogmatic medical practice into verifiable outcome-based medical care. We believe the approach is applicable to all medical or surgical domains, but widespread adoption may require the revision of many currently prevalent views regarding the role of research in clinical practice.

“…This is why clinicians should, instead of looking for erroneous reasons to choose treatment T1 or T2, based on the haphazard allocation of the patient to various "classes of patients," or on various "wrong axis comparisons," use the care trial methodology to offer what is in fact optimal care in the presence of uncertainty. 8,10,31…”

Section: The Wrong Axis Comparisonmentioning

confidence: 99%

The 2018 ter Brugge Lecture: Problems with the Introduction of Innovations in Neurovascular Care

Fahed

Roy

et al. 2019

Can. J. Neurol. Sci.

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ABSTRACT:Most endovascular innovations have been introduced into clinical care by showing good outcomes in small enthusiastic case series of selected patients. Randomized clinical trials (RCTs) have rarely been performed, except for acute ischemic stroke, but even then most trial designs were too explanatory to inform clinical decisions. In this article, we review 2 × 2 tables and forest plots that summarize RCT results to examine methodological issues in the design and interpretation of clinical studies. Research results can apply in practice when RCTs are all-inclusive, pragmatic trials. Common problems include the following: (i) using restrictive eligibility criteria in explanatory trials, instead of including the diversity of patients in need of care, which hampers future generalizability of results; (ii) ignoring an entire line of the 2 × 2 table and excluding patients who do not meet the proposed criteria of a diagnostic test in its evaluation (perfusion studies) which renders clinical inferences misleading; (iii) ignoring an entire column of the 2 × 2 table and comparing different patients treated using the same treatment instead of different treatments in the same patients (the “wrong axis” comparisons of prognostic studies and clinical experience) which leads to unjustified treatment decisions and actions; or (iv) combining all aforementioned problems (case series and epidemiological studies). The most efficient and reliable way to improve patient outcomes, after as well as long before research results are available, is to change the way we practice: to use care trials to guide care in the presence of uncertainty.