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Journal of Surgical Oncology Jan 2022Surgeon-led clinical trials have defined the standard of care for locoregional pancreatic cancer to date. The infrastructure and collaborative nature of cooperative...
Surgeon-led clinical trials have defined the standard of care for locoregional pancreatic cancer to date. The infrastructure and collaborative nature of cooperative oncology groups offer many advantages, such as providing an ideal mechanism through which multidisciplinary pancreatic cancer trials are performed. As key members of the treatment team, surgeons bring experience and expertise to the design of surgical and multidisciplinary trials and are uniquely poised to be leaders of future pancreatic cancer trials.
Topics: Clinical Trials as Topic; Humans; Multicenter Studies as Topic; Neoplasm Staging; Pancreatic Neoplasms; Patient Care Team; Randomized Controlled Trials as Topic; Surgical Oncology
PubMed: 34586635
DOI: 10.1002/jso.26701 -
Pediatrics Jun 2014Adolescents (aged 15-19 years) have not experienced the same survival gains as children and older adults diagnosed with cancer. Poor clinical trial enrollment and... (Review)
Review
Adolescents (aged 15-19 years) have not experienced the same survival gains as children and older adults diagnosed with cancer. Poor clinical trial enrollment and adherence rates among adolescents may account for some of this disparity. Although biological, regulatory, systemic, and practice-related challenges to clinical trial enrollment and adherence have been examined, studies of psychosocial factors, which can serve as barriers or facilitators to enrollment and adherence, are limited. To bring attention to these psychological factors, we reviewed existing literature on psychosocial barriers and facilitators that can affect an adolescent's decision to enroll and adhere to a clinical trial. We also provide potential strategies to address psychosocial factors affecting clinical trial accrual and adherence.
Topics: Adolescent; Clinical Trials as Topic; Health Knowledge, Attitudes, Practice; Humans; Neoplasms; Patient Compliance; Patient Selection; Psychology
PubMed: 24918211
DOI: 10.1542/peds.2014-0122I -
Alzheimer's & Dementia : the Journal of... May 2011Developing new therapies for Alzheimer's disease (AD) is critically important to avoid the impending public health disaster imposed by this common disorder. Means must... (Review)
Review
Developing new therapies for Alzheimer's disease (AD) is critically important to avoid the impending public health disaster imposed by this common disorder. Means must be found to prevent, delay the onset, or slow the progression of AD. These goals will be achieved by identifying disease-modifying therapies and testing them in clinical trials. Biomarkers play an increasingly important role in AD drug development. In preclinical testing, they assist in decisions to develop an agent. Biomarkers in phase I provide insights into toxic responses and drug metabolism and in Phase II proof-of-concept trials they facilitate go/no-go decisions and dose finding. Biomarkers can play a role in identifying presymptomatic patients or specific patient subgroups. They can provide evidence of target engagement before clinical changes can be expected. Brain imaging can serve as a primary outcome in Phase II trials and as a key secondary outcome in Phase III trials. Magnetic resonance imaging is currently best positioned for use in large multicenter clinical trials. Cerebrospinal fluid (CSF) measures of amyloid beta protein (Aβ), tau protein, and hyperphosphorylated tau (p-tau) protein are sensitive and specific to the diagnosis of AD and may serve as inclusion criteria and possibly as outcomes in clinical trials targeting relevant pathways. Plasma measures of Aβ are of limited diagnostic value but may provide important information as a measure of treatment response. A wide variety of measures of detectable products of cellular processes are being developed as possible biomarkers accessible in the cerebrospinal fluid and plasma or serum. Surrogate markers that can function as outcomes in pivotal trials and reliably predict clinical outcomes are needed to facilitate primary prevention trials of asymptomatic persons where clinical measures may be of limited value. Fit-for-purpose biomarkers are increasingly available to guide AD drug development decisions.
Topics: Alzheimer Disease; Biomarkers, Pharmacological; Clinical Trials as Topic; Diagnostic Imaging; Drug Delivery Systems; Drug Discovery; Humans
PubMed: 21550318
DOI: 10.1016/j.jalz.2010.06.004 -
Journal of Clinical Oncology : Official... Apr 2015
Topics: Clinical Trials as Topic; Contrast Media; Humans; Image Enhancement; Lymphoma; Positron-Emission Tomography; Radiation Dosage; Radiation Monitoring; Research Design; Tomography, X-Ray Computed
PubMed: 25691672
DOI: 10.1200/JCO.2014.60.0486 -
Current Problems in Cancer Oct 2021Evaluation of novel treatments through clinical trials remains the backbone of oncological clinical research, but only a minor portion have been tested in Phase III... (Review)
Review
Evaluation of novel treatments through clinical trials remains the backbone of oncological clinical research, but only a minor portion have been tested in Phase III trials. The continued publication of underpowered trials provides an ongoing need for meta-analyses to detect clinically significant outcomes. Although tumor relapse and survival are important issues and easily measured outcomes in trials, they are often not the most relevant indicators for treatment success. As diagnostic technologies and treatments continue to advance, methodologies defining high quality studies have been established, but still enthusiasm to adopt novel technologies that leads to studies holding well-described bias that do not aid the rational use of the studied test. Global awareness of such bias and standard research methodology is the clue toward iconic studies giving rational supporting novel cancer treatments and patients' support.
Topics: Clinical Trials as Topic; Humans; Neoplasms; Prognosis; Randomized Controlled Trials as Topic; Treatment Outcome
PubMed: 33715867
DOI: 10.1016/j.currproblcancer.2021.100725 -
Expert Review of Pharmacoeconomics &... Dec 2011Many authors and guidelines have proposed to exclude protocol-driven costs from cost-effectiveness analyses alongside clinical trials because they do not occur in... (Review)
Review
Many authors and guidelines have proposed to exclude protocol-driven costs from cost-effectiveness analyses alongside clinical trials because they do not occur in clinical practice. This article, however, argues that only costs to improve patient adherence can be excluded, as the underlying protocol-driven activities have a clearly distinguishable cost and utility impact (most of the time). All other protocol-driven costs need to be included because the cost and utility impact of the underlying protocol-driven activities cannot be easily separated.
Topics: Clinical Protocols; Clinical Trials as Topic; Cost-Benefit Analysis; Data Collection; Diagnostic Tests, Routine; Economics, Pharmaceutical; Health Care Costs; Humans; Medication Adherence; Models, Economic; Quality Assurance, Health Care; Research Design
PubMed: 22098282
DOI: 10.1586/erp.11.75 -
JCO Clinical Cancer Informatics Jun 2019In this work, we present a conceptual framework to support clinical trial optimization and enrollment workflows and review the current state, limitations, and future...
In this work, we present a conceptual framework to support clinical trial optimization and enrollment workflows and review the current state, limitations, and future trends in this space. This framework includes knowledge representation of clinical trials, clinical trial optimization, clinical trial design, enrollment workflows for prospective clinical trial matching, waitlist management, and, finally, evaluation strategies for assessing improvement.
Topics: Algorithms; Clinical Trials as Topic; Databases, Factual; Decision Support Systems, Clinical; Humans; Medical Informatics; Natural Language Processing; Research Design; Software; Workflow
PubMed: 31225983
DOI: 10.1200/CCI.19.00033 -
Journal of Clinical Neuroscience :... May 2012After the introduction of Guglielmi Detachable Coils (GDC), endovascular management of ruptured and unruptured aneurysms became a viable alternative to surgical clipping... (Review)
Review
After the introduction of Guglielmi Detachable Coils (GDC), endovascular management of ruptured and unruptured aneurysms became a viable alternative to surgical clipping as a "minimally invasive" option. Endovascular management of aneurysms became even more common after the International Subarachnoid Aneurysm Trial, which was one of the first prospective, randomized trials comparing clipping and coiling, showed reduced dependency and death in patients undergoing coiling after two months and one year. As the numbers of patients treated by endovascular therapy grow neurosurgeons are facing increasing challenges of clipping difficult aneurysms not suitable for coiling, including those that are wide-necked, thrombosed or involving many perforators. In addition, treatment failures (recurrent and residual aneurysms after coiling) pose difficult treatment scenarios fraught with complications due to surrounding adhesions, coil migration and involvement of adjacent neurovascular structures. Thus, we analyzed the recent literature dealing with the nuances of clipping after coiling and reviewed the current management principles involved in treating these difficult aneurysms.
Topics: Clinical Trials as Topic; Humans; Intracranial Aneurysm; Radiography; Subarachnoid Space; Surgical Instruments; Vascular Surgical Procedures
PubMed: 22417455
DOI: 10.1016/j.jocn.2011.08.022 -
Journal of Visceral Surgery Feb 2014Endpoints are measurable clinical and biological findings that are used for the development and assessment of treatment options. In the treatment of cancer, endpoints... (Review)
Review
Endpoints are measurable clinical and biological findings that are used for the development and assessment of treatment options. In the treatment of cancer, endpoints can be classified into two categories: "patient-centered clinical endpoints" including overall survival (OS) and health-related quality of life (QoL), and "tumor-centered clinical endpoints" such as progression-free survival. Surrogate endpoints are tumor-centered clinical endpoints that can be used as substitutes for patient-centered clinical endpoints, particularly OS. The choice of endpoints in oncology trials is a major problem. The published Consolidated Standards of Reporting Trials (CONSORT) best-practice guidelines encourage the reporting of clearly defined primary and secondary outcome measures. OS is the gold standard of endpoints but as increasing numbers of effective salvage treatments become available for many types of cancer, much larger numbers of patients are included; this requires a longer follow-up period and increases the cost of clinical trials. Thus, tumor-centered clinical endpoints that can be assessed earlier and used as surrogates for overall survival are increasingly studied, but most of them currently lack standardized definitions to enable cross comparison of results among different clinical trials and they have not been validated as surrogate endpoints. In addition, the variability of their definition can strongly impact the trial's conclusions by affecting both statistical power and estimation. In this context, QoL constitutes an available and useful surrogate endpoint for trials to ensure treatment benefit from both the patient and public health points of view. Methodological research should be pursued to develop standard outcome definitions for use in cancer clinical trials and to define a standardized longitudinal analysis of QoL data.
Topics: Clinical Trials as Topic; Disease-Free Survival; Humans; Neoplasms; Outcome Assessment, Health Care; Quality of Life; Research Design; Survival Rate; Treatment Outcome
PubMed: 24440056
DOI: 10.1016/j.jviscsurg.2013.10.001 -
Nature Oct 2015An enhanced understanding of the molecular pathology of disease gained from genomic studies is facilitating the development of treatments that target discrete molecular... (Review)
Review
An enhanced understanding of the molecular pathology of disease gained from genomic studies is facilitating the development of treatments that target discrete molecular subclasses of tumours. Considerable associated challenges include how to advance and implement targeted drug-development strategies. Precision medicine centres on delivering the most appropriate therapy to a patient on the basis of clinical and molecular features of their disease. The development of therapeutic agents that target molecular mechanisms is driving innovation in clinical-trial strategies. Although progress has been made, modifications to existing core paradigms in oncology drug development will be required to realize fully the promise of precision medicine.
Topics: Clinical Trials as Topic; Drug Discovery; Genetic Testing; Humans; Medical Oncology; Neoplasms; Patient Selection; Precision Medicine
PubMed: 26469047
DOI: 10.1038/nature15819