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Journal of Clinical Epidemiology Jan 2021There is considerable actual and potential waste in research. Evidence-based research ensures worthwhile and valuable research. The aim of this series, which this...
OBJECTIVES
There is considerable actual and potential waste in research. Evidence-based research ensures worthwhile and valuable research. The aim of this series, which this article introduces, is to describe the evidence-based research approach.
STUDY DESIGN AND SETTING
In this first article of a three-article series, we introduce the evidence-based research approach. Evidence-based research is the use of prior research in a systematic and transparent way to inform a new study so that it is answering questions that matter in a valid, efficient, and accessible manner.
RESULTS
We describe evidence-based research and provide an overview of the approach of systematically and transparently using previous research before starting a new study to justify and design the new study (article #2 in series) and-on study completion-place its results in the context with what is already known (article #3 in series).
CONCLUSION
This series introduces evidence-based research as an approach to minimize unnecessary and irrelevant clinical health research that is unscientific, wasteful, and unethical.
Topics: Biomedical Research; Clinical Trials as Topic; Ethics, Research; Evidence-Based Medicine; Humans; Needs Assessment; Reproducibility of Results; Research Design; Systematic Reviews as Topic; Treatment Outcome
PubMed: 32979491
DOI: 10.1016/j.jclinepi.2020.07.020 -
Frontiers in Public Health 2022Decentralized clinical trials (DCTs) are studies in which the need for patients to physically access hospital-based trial sites is reduced or eliminated. The CoViD-19... (Review)
Review
Decentralized clinical trials (DCTs) are studies in which the need for patients to physically access hospital-based trial sites is reduced or eliminated. The CoViD-19 pandemic has caused a significant increase in DCT: a survey shows that 76% of pharmaceutical companies, device manufacturers, and Contract Research Organizations adopted decentralized techniques during the early phase of the pandemic. The implementation of DCTs relies on the use of digital tools such as e-consent, apps, wearable devices, Electronic Patient-Reported Outcomes (ePRO), telemedicine, as well as on moving trial activities to the patient's home (e.g., drug delivery) or to local healthcare settings (i.e., community-based diagnosis and care facilities). DCTs adapt to patients' routines, allow patients to participate regardless of where they live by removing logistical barriers, offer better access to the study and the investigational product, and permit the inclusion of more diverse and more representative populations. The feasibility and quality of DCTs depends on several requirements including dedicated infrastructures and staff, an adequate regulatory framework, and partnerships between research sites, patients and sponsors. The evaluation of Ethics Committees (ECs) is crucial to the process of innovating and digitalizing clinical trials: adequate assessment tools and a suitable regulatory framework are needed for evaluation by ECs. DCTs also raise issues, many of which are of considerable ethical significance. These include the implications for the relationship between patients and healthcare staff, for the social dimension of the patient, for data integrity (at the source, during transmission, in the analysis phase), for personal data protection, and for the possible risks to health and safety. Despite their considerable growth, DCTs have only received little attention from bioethicists. This paper offers a review on some ethical implications and requirements of DCTs in order to encourage further ethical reflection on this rapidly emerging field.
Topics: Humans; COVID-19; Delivery of Health Care; Pandemics; Telemedicine; Clinical Trials as Topic
PubMed: 36590004
DOI: 10.3389/fpubh.2022.1081150 -
Psychiatry Research Oct 2019The traditional research pipeline that encourages a staged approach to moving an intervention from efficacy trials to the real world can take a long time. To address...
The traditional research pipeline that encourages a staged approach to moving an intervention from efficacy trials to the real world can take a long time. To address this issue, hybrid effectiveness-implementation designs were codified to promote examination of both effectiveness and implementation outcomes within a study. There are three types of hybrid designs and they vary based on their primary focus and the amount of emphasis on effectiveness versus implementation outcomes. A type 1 hybrid focuses primarily on the effectiveness outcomes of an intervention while exploring the "implementability" of the intervention. A type 2 hybrid has a dual focus on effectiveness and implementation outcomes; these designs allow for the simultaneous testing or piloting of implementation strategies during an effectiveness trial. A type 3 hybrid focuses primarily on implementation outcomes while also collecting effectiveness outcomes as they relate to uptake or fidelity of the intervention. This paper provides an introduction to these designs and describes each of the three types, design considerations, and examples for each.
Topics: Biomedical Research; Clinical Trials as Topic; Humans; Research Design; Treatment Outcome
PubMed: 31434011
DOI: 10.1016/j.psychres.2019.112513 -
Current Treatment Options in Oncology Jul 2021Clinical trials play a critical role in discovering new treatments, but the path to regulatory approval can be cumbersome and time consuming. Efforts to increase the... (Review)
Review
Clinical trials play a critical role in discovering new treatments, but the path to regulatory approval can be cumbersome and time consuming. Efforts to increase the efficiency and interpretability of clinical trials within the neuro-oncology community have focused on standardization of response assessment, development of consensus guidelines for clinical trial conduct, decentralization of clinical trials, removal of barriers to clinical trial accrual, and re-examination of patient eligibility criteria.
Topics: Clinical Trials as Topic; Humans; Medical Oncology; Nervous System Neoplasms; Outcome Assessment, Health Care; Practice Guidelines as Topic; Research Design
PubMed: 34213625
DOI: 10.1007/s11864-021-00875-8 -
The Quarterly Journal of Nuclear... Dec 2019In radiomics, quantitative features that describe phenotypic tumor characteristics are derived from radiographic images. Because radiomics generates information from... (Review)
Review
In radiomics, quantitative features that describe phenotypic tumor characteristics are derived from radiographic images. Because radiomics generates information from routine medical images, it is a powerful way to non-invasively examine the spatial and temporal heterogeneity of disease, and thus has potential to significantly impact clinical trial design, execution, and ultimately patient care. The aim of this review article is to discuss how radiomics may address some of the current challenges in clinical randomized control trials, and the difficulties of integrating robust and repeatable radiomics analysis into trial design. Each step of the radiomics process, including image acquisition and reconstruction, image segmentation, feature extraction, and computational analysis, requires extensive standardization in order to be successfully incorporated into clinical trials and inform clinical decision making. By addressing these challenges, the potential of radiomics may be realized.
Topics: Clinical Trials as Topic; Diagnostic Imaging; Humans; Image Processing, Computer-Assisted; Neoplasms
PubMed: 31527581
DOI: 10.23736/S1824-4785.19.03217-5 -
Neurotherapeutics : the Journal of the... Jul 2021
Topics: Anticonvulsants; Clinical Trials as Topic; Epilepsy; Genetic Testing; Humans; Precision Medicine
PubMed: 34704188
DOI: 10.1007/s13311-021-01147-x -
Journal of Surgical Oncology Jan 2022Treatment of regional lymph nodes in melanoma has been controversial for more than a century. A series of clinical trials evaluating elective lymph node dissection and... (Review)
Review
Treatment of regional lymph nodes in melanoma has been controversial for more than a century. A series of clinical trials evaluating elective lymph node dissection and then sentinel lymph node biopsy have helped define the current standard of care. These trials resulted in increasingly selective application of surgical intervention for regional lymph nodes in melanoma. First by focusing on optimal candidates for elective lymph node dissection and then by identifying patients through sentinel lymph node biopsy. The current standard of sentinel lymph node biopsy for appropriately selected patients and nodal observation for many patients, even with involved sentinel nodes is both more accurate in staging and much less morbid than what came before.
Topics: Clinical Trials as Topic; Clinical Trials, Phase III as Topic; History, 19th Century; History, 20th Century; History, 21st Century; Humans; Lymph Node Excision; Lymph Nodes; Lymphatic Metastasis; Melanoma; Multicenter Studies as Topic; Randomized Controlled Trials as Topic; Sentinel Lymph Node Biopsy
PubMed: 34897707
DOI: 10.1002/jso.26744 -
World Neurosurgery Oct 2019Traumatic brain injury (TBI) represents a major health care problem and a significant social and economic issue worldwide. Considering the generalized failure in... (Review)
Review
Traumatic brain injury (TBI) represents a major health care problem and a significant social and economic issue worldwide. Considering the generalized failure in introducing effective drugs and clinical protocols, there is an urgent need for efficient treatment modalities, able to improve devastating posttraumatic morbidity and mortality. In this work, the status of brain trauma research is analyzed in all its aspects, including basic and translational science and clinical trials. Implicit and explicit challenges to different lines of research are discussed and clinical trial structures and outcomes are scrutinized, along with possible explanations for systematic therapeutic failures and their implications for future development of drug and clinical trials. Despite significant advances in basic and clinical research in recent years, no specific therapeutic protocols for TBI have been shown to be effective. New potential therapeutic targets have been identified, following a better understanding of pathophysiologic mechanisms underlying TBI, although with disappointing results. Several reasons can be pinpointed at different levels, from inaccurate animal models of disease to faulty preclinical and clinical trials, with poor design and subjective outcome measures. Distinct strategies can be delineated to overcome specific shortcomings of research studies. Identifying and contextualizing the failures that have dominated TBI research is mandatory. This review analyzes current approaches and discusses possible strategies for improving outcomes.
Topics: Animals; Biomedical Research; Brain Injuries, Traumatic; Clinical Trials as Topic; Disease Models, Animal; Humans
PubMed: 31284053
DOI: 10.1016/j.wneu.2019.06.212 -
Progress in Cardiovascular Diseases 2019Tricuspid regurgitation (TR), particularly functional or secondary TR, is increasingly recognized in clinical practice and when at least moderate in severity is... (Review)
Review
Tricuspid regurgitation (TR), particularly functional or secondary TR, is increasingly recognized in clinical practice and when at least moderate in severity is associated with significant increase in mortality. In recent years multiple new trans-catheter devices have been developed to treat tricuspid regurgitation and are now undergoing clinical trial evaluations. The choice of appropriate endpoints in TR trials is particularly challenging as the disease is complex, often co-exists with left heart disease and pulmonary hypertension, and has not been extensively studied. Endpoints utilized in left heart disease trials have been applied with success to TR trials, and innovative trial designs will allow the initiation of pivotal randomized trials. Ultimately the development of TR specific endpoints may provide for more specific and robust assessment of these novel therapies.
Topics: Cardiac Catheterization; Clinical Trials as Topic; Endpoint Determination; Heart Valve Prosthesis Implantation; Humans; Severity of Illness Index; Tricuspid Valve Insufficiency
PubMed: 31805295
DOI: 10.1016/j.pcad.2019.11.019 -
The Lancet. Oncology May 2020There is a large variability regarding the definition and choice of primary endpoints in phase 2 and phase 3 multimodal rectal cancer trials, resulting in inconsistency... (Review)
Review
There is a large variability regarding the definition and choice of primary endpoints in phase 2 and phase 3 multimodal rectal cancer trials, resulting in inconsistency and difficulty of data interpretation. Also, surrogate properties of early and intermediate endpoints have not been systematically assessed. We provide a comprehensive review of clinical and surrogate endpoints used in trials for non-metastatic rectal cancer. The applicability, advantages, and disadvantages of these endpoints are summarised, with recommendations on clinical endpoints for the different phase trials, including limited surgery or non-operative management for organ preservation. We discuss how early and intermediate endpoints, including patient-reported outcomes and involvement of patients in decision making, can be used to guide trial design and facilitate consistency in reporting trial results in rectal cancer.
Topics: Clinical Trials as Topic; Combined Modality Therapy; Endpoint Determination; Humans; Patient Reported Outcome Measures; Rectal Neoplasms; Research Design; Time Factors; Treatment Outcome
PubMed: 32359501
DOI: 10.1016/S1470-2045(20)30024-3