-
Annals of the New York Academy of... Jan 2022For many years, it was believed that the aging process was inevitable and that age-related diseases could not be prevented or reversed. The geroscience hypothesis,... (Review)
Review
For many years, it was believed that the aging process was inevitable and that age-related diseases could not be prevented or reversed. The geroscience hypothesis, however, posits that aging is, in fact, malleable and, by targeting the hallmarks of biological aging, it is indeed possible to alleviate age-related diseases and dysfunction and extend longevity. This field of geroscience thus aims to prevent the development of multiple disorders with age, thereby extending healthspan, with the reduction of morbidity toward the end of life. Experts in the field have made remarkable advancements in understanding the mechanisms underlying biological aging and identified ways to target aging pathways using both novel agents and repurposed therapies. While geroscience researchers currently face significant barriers in bringing therapies through clinical development, proof-of-concept studies, as well as early-stage clinical trials, are underway to assess the feasibility of drug evaluation and lay a regulatory foundation for future FDA approvals in the future.
Topics: Aging; Autophagy; COVID-19; Cardiovascular Diseases; Congresses as Topic; Geroscience; Humans; Longevity; Metabolomics; Nervous System Diseases; Research Report; Stem Cell Transplantation
PubMed: 34498278
DOI: 10.1111/nyas.14681 -
Yearbook of Medical Informatics Aug 2020To summarize key contributions to current research in the field of Clinical Research Informatics (CRI) and to select best papers published in 2019. (Review)
Review
OBJECTIVES
To summarize key contributions to current research in the field of Clinical Research Informatics (CRI) and to select best papers published in 2019.
METHOD
A bibliographic search using a combination of MeSH descriptors and free-text terms on CRI was performed using PubMed, followed by a double-blind review in order to select a list of candidate best papers to be then peer-reviewed by external reviewers. After peer-review ranking, a consensus meeting between the two section editors and the editorial team was organized to finally conclude on the selected three best papers.
RESULTS
Among the 517 papers, published in 2019, returned by the search, that were in the scope of the various areas of CRI, the full review process selected three best papers. The first best paper describes the use of a homomorphic encryption technique to enable federated analysis of real-world data while complying more easily with data protection requirements. The authors of the second best paper demonstrate the evidence value of federated data networks reporting a large real world data study related to the first line treatment for hypertension. The third best paper reports the migration of the US Food and Drug Administration (FDA) adverse event reporting system database to the OMOP common data model. This work opens the combined analysis of both spontaneous reporting system and electronic health record (EHR) data for pharmacovigilance.
CONCLUSIONS
The most significant research efforts in the CRI field are currently focusing on real world evidence generation and especially the reuse of EHR data. With the progress achieved this year in the areas of phenotyping, data integration, semantic interoperability, and data quality assessment, real world data is becoming more accessible and reusable. High quality data sets are key assets not only for large scale observational studies or for changing the way clinical trials are conducted but also for developing or evaluating artificial intelligence algorithms guiding clinical decision for more personalized care. And lastly, security and confidentiality, ethical and regulatory issues, and more generally speaking data governance are still active research areas this year.
Topics: Adverse Drug Reaction Reporting Systems; Biomedical Research; Computer Security; Data Accuracy; Data Management; Electronic Health Records; Humans; Hypertension; Medical Informatics; Neoplasms; Precision Medicine; Treatment Outcome
PubMed: 32823317
DOI: 10.1055/s-0040-1702007 -
Therapeutic Advances in Drug Safety 2022Comprehensive methods for evaluating safety are needed to objectively assess the full risk profile of a medication. The confidence of the prescribing provider in the... (Review)
Review
UNLABELLED
Comprehensive methods for evaluating safety are needed to objectively assess the full risk profile of a medication. The confidence of the prescribing provider in the safety and effectiveness of pharmaceuticals is extremely important. Pharmacovigilance is a key component of drug safety regulatory processes and is paramount for ensuring the safety profile of medications used to treat patients. All participants in the healthcare system, including healthcare providers and consumers, should understand and meaningfully engage in the pharmacovigilance process; healthcare providers should integrate pharmacovigilance into everyday practice, inviting feedback from patients. This narrative review aims to give an overview of the main topics underlying pharmacovigilance and drug safety in pharmaceutical research phase after the authorization of a drug in the United States. The US Food and Drug Administration guidance and post-approval regulatory actions are considered from an industry perspective.
PLAIN LANGUAGE SUMMARY
Government agencies regulate the safe use of medicinal products. By determining and enforcing pharmacovigilance, the monitoring of drugs for potential risks, they safeguard the welfare of consumers of medicines. Comprehensive, documented methods for evaluating the safety of a drug during its development and its subsequent use allow identification of any risks associated with the drug's use throughout its lifetime. The comprehensive identification of safety issues associated with a drug is improved when all parties involved in the development and use of drugs participate in the pharmacovigilance process. For example, clinicians should regularly ask their patients if they are experiencing any issues with their treatment, and patients should be encouraged to report problems they encounter with a particular medication to their healthcare provider. This narrative review provides an overview of the main topics underlying pharmacovigilance and drug safety after approval of a drug in the United States. Guidelines and actions from the US Food and Drug Administration are considered from an industry perspective.
PubMed: 36187302
DOI: 10.1177/20420986221125006 -
Cancer Medicine Jun 2021In recent years, the field of artificial intelligence (AI) in oncology has grown exponentially. AI solutions have been developed to tackle a variety of cancer-related... (Review)
Review
In recent years, the field of artificial intelligence (AI) in oncology has grown exponentially. AI solutions have been developed to tackle a variety of cancer-related challenges. Medical institutions, hospital systems, and technology companies are developing AI tools aimed at supporting clinical decision making, increasing access to cancer care, and improving clinical efficiency while delivering safe, high-value oncology care. AI in oncology has demonstrated accurate technical performance in image analysis, predictive analytics, and precision oncology delivery. Yet, adoption of AI tools is not widespread, and the impact of AI on patient outcomes remains uncertain. Major barriers for AI implementation in oncology include biased and heterogeneous data, data management and collection burdens, a lack of standardized research reporting, insufficient clinical validation, workflow and user-design challenges, outdated regulatory and legal frameworks, and dynamic knowledge and data. Concrete actions that major stakeholders can take to overcome barriers to AI implementation in oncology include training and educating the oncology workforce in AI; standardizing data, model validation methods, and legal and safety regulations; funding and conducting future research; and developing, studying, and deploying AI tools through multidisciplinary collaboration.
Topics: Artificial Intelligence; Bias; Data Collection; Decision Support Systems, Clinical; Humans; Image Interpretation, Computer-Assisted; Machine Learning; Medical Oncology; Precision Medicine; Research Report
PubMed: 33960708
DOI: 10.1002/cam4.3935 -
Antimicrobial Stewardship & Healthcare... 2022Hospital-onset sepsis accounts for 10%-15% of all sepsis cases and is associated with very high mortality rates, yet to date most hospitals have paid little attention to...
Hospital-onset sepsis accounts for 10%-15% of all sepsis cases and is associated with very high mortality rates, yet to date most hospitals have paid little attention to tracking its incidence and outcomes. This contrasts sharply with the substantial effort that hospitals and regulatory agencies spend tracking and reporting a limited subset of healthcare-associated infections. The recent development of the Center for Disease Control and Prevention's hospital-onset Adult Sepsis Event (ASE) definition, however, provides a validated and standardized mechanism for facilities to identify patients with nosocomial sepsis using routinely available electronic health record data. Recent data have demonstrated that hospital-onset ASE surveillance identifies many infections that are largely missed by current reportable healthcare-associated infections and that are associated with much higher mortality rates. Expanding the breadth of surveillance to include these highly consequential infections could help identify new targets for prevention and quality improvement and ultimately catalyze better outcomes for hospitalized patients. More work is needed, however, to characterize the preventability of hospital-onset ASE, develop and validate robust case-mix adjustment tools, and facilitate widespread uptake in hospitals with limited resources.
PubMed: 36310798
DOI: 10.1017/ash.2022.16 -
BMC Medical Informatics and Decision... Jun 2018A scoping review to characterize the literature on the use of conversations in social media as a potential source of data for detecting adverse events (AEs) related to... (Review)
Review
BACKGROUND
A scoping review to characterize the literature on the use of conversations in social media as a potential source of data for detecting adverse events (AEs) related to health products.
METHODS
Our specific research questions were (1) What social media listening platforms exist to detect adverse events related to health products, and what are their capabilities and characteristics? (2) What is the validity and reliability of data from social media for detecting these adverse events? MEDLINE, EMBASE, Cochrane Library, and relevant websites were searched from inception to May 2016. Any type of document (e.g., manuscripts, reports) that described the use of social media data for detecting health product AEs was included. Two reviewers independently screened citations and full-texts, and one reviewer and one verifier performed data abstraction. Descriptive synthesis was conducted.
RESULTS
After screening 3631 citations and 321 full-texts, 70 unique documents with 7 companion reports available from 2001 to 2016 were included. Forty-six documents (66%) described an automated or semi-automated information extraction system to detect health product AEs from social media conversations (in the developmental phase). Seven pre-existing information extraction systems to mine social media data were identified in eight documents. Nineteen documents compared AEs reported in social media data with validated data and found consistent AE discovery in all except two documents. None of the documents reported the validity and reliability of the overall system, but some reported on the performance of individual steps in processing the data. The validity and reliability results were found for the following steps in the data processing pipeline: data de-identification (n = 1), concept identification (n = 3), concept normalization (n = 2), and relation extraction (n = 8). The methods varied widely, and some approaches yielded better results than others.
CONCLUSIONS
Our results suggest that the use of social media conversations for pharmacovigilance is in its infancy. Although social media data has the potential to supplement data from regulatory agency databases; is able to capture less frequently reported AEs; and can identify AEs earlier than official alerts or regulatory changes, the utility and validity of the data source remains under-studied.
TRIAL REGISTRATION
Open Science Framework ( https://osf.io/kv9hu/ ).
Topics: Adverse Drug Reaction Reporting Systems; Drug-Related Side Effects and Adverse Reactions; Humans; Pharmacovigilance; Social Media
PubMed: 29898743
DOI: 10.1186/s12911-018-0621-y -
Medical Devices (Auckland, N.Z.) 2017Gait disability is a major health care problem worldwide. Powered exoskeletons have recently emerged as devices that can enable users with gait disabilities to ambulate... (Review)
Review
Gait disability is a major health care problem worldwide. Powered exoskeletons have recently emerged as devices that can enable users with gait disabilities to ambulate in an upright posture, and potentially bring other clinical benefits. In 2014, the US Food and Drug Administration approved marketing of the ReWalk™ Personal Exoskeleton as a class II medical device with special controls. Since then, Indego™ and Ekso™ have also received regulatory approval. With similar trends worldwide, this industry is likely to grow rapidly. On the other hand, the regulatory science of powered exoskeletons is still developing. The type and extent of probable risks of these devices are yet to be understood, and industry standards are yet to be developed. To address this gap, Manufacturer and User Facility Device Experience, Clinicaltrials.gov, and PubMed databases were searched for reports of adverse events and inclusion and exclusion criteria involving the use of lower limb powered exoskeletons. Current inclusion and exclusion criteria, which can determine probable risks, were found to be diverse. Reported adverse events and identified risks of current devices are also wide-ranging. In light of these findings, current regulations, standards, and regulatory procedures for medical device applications in the USA, Europe, and Japan were also compared. There is a need to raise awareness of probable risks associated with the use of powered exoskeletons and to develop adequate countermeasures, standards, and regulations for these human-machine systems. With appropriate risk mitigation strategies, adequate standards, comprehensive reporting of adverse events, and regulatory oversight, powered exoskeletons may one day allow individuals with gait disabilities to safely and independently ambulate.
PubMed: 28533700
DOI: 10.2147/MDER.S107134 -
Philosophical Transactions of the Royal... Nov 2016Based on prior research, multiple discriminable dimensions of interoception have been defined: awareness, accuracy and sensibility. Some investigators defined... (Review)
Review
Based on prior research, multiple discriminable dimensions of interoception have been defined: awareness, accuracy and sensibility. Some investigators defined interoceptive awareness as metacognitive awareness of interoceptive accuracy, assessed as correspondence between subjective confidence in and objective accuracy of one's heartbeat detection. However, metacognitive awareness has been understood quite differently: 'a cognitive set in which negative thoughts/feelings are experienced as mental events, rather than as the self' or as 'error awareness'. Interoceptive sensibility, defined as self-reported interoception, distinguishes self-reported interoception from objective interoceptive accuracy, but does not differentiate between anxiety-driven and mindful attention styles towards interoceptive cues, a distinction of key clinical importance: one attention style is associated with somatization and anxiety disorders; the other has been viewed as healthy, adaptive, resilience-enhancing. The self-report Multidimensional Assessment of Interoceptive Awareness was developed to differentiate these attention styles. It has been translated into 16 languages and applied in cross-sectional and longitudinal studies. Findings from these applications suggest that differentiating interoceptive sensibility according to attention style and regulatory aspects (i) provides insights into the psychology of interoceptive awareness, (ii) differentiates between clinically maladaptive and beneficial interoceptive attention, and (iii) helps elucidate therapeutic approaches that claim to provide health benefits by training mindful styles of bodily awareness.This article is part of the themed issue 'Interoception beyond homeostasis: affect, cognition and mental health'.
Topics: Attention; Awareness; Cognition; Humans; Interoception; Self Report
PubMed: 28080970
DOI: 10.1098/rstb.2016.0013 -
Value in Health : the Journal of the... Jun 2018Wearable devices offer huge potential to collect rich sources of data to provide insights into the effects of treatment interventions. Despite this, at the time of... (Review)
Review
BACKGROUND
Wearable devices offer huge potential to collect rich sources of data to provide insights into the effects of treatment interventions. Despite this, at the time of writing this report, limited regulatory guidance on the use of wearables in clinical trial programs has been published.
OBJECTIVES
To present recommendations from the Critical Path Institute's Electronic Patient-Reported Outcome Consortium regarding the selection and evaluation of wearable devices and their measurements for use in regulatory trials and to support labeling claims.
METHODS
The evaluation group was composed of Critical Path Institute's clinical outcome assessment (COA) scientists and COA specialists from pharmaceutical trial eCOA solution providers, including COA development and validation specialists. The resulting recommendations were drawn from a broad range of backgrounds, perspectives, and expertise that enriched the development of this report. Recommendations were developed through analysis of existing regulatory guidance relating to COA development and use in clinical trials, medical device certification/clearance regulations, literature-reported best practice, and practical experience of wearable technology application in clinical trials.
RESULTS
We identify the essential properties of fit-for-purpose wearables and propose evidence needed to support their use. In addition, we overview the activities required to establish clinical endpoints derived from wearables data.
CONCLUSIONS
Using this framework, we believe there is enough current understanding to promote the appropriate use of wearables in study protocols. We hope this will provide a basis for discussion among clinical trial stakeholders and catalyze the development of more robust regulatory guidance.
Topics: Clinical Trials as Topic; Decision Making; Endpoint Determination; Evidence-Based Medicine; Humans; Legislation, Medical; Outcome Assessment, Health Care; Product Labeling; Reproducibility of Results; Research Design; Treatment Outcome; Wearable Electronic Devices
PubMed: 29909867
DOI: 10.1016/j.jval.2017.09.012