Objectives: Real-world evidence RWE is essential for the development of pharmaceutical and medical technologies and informs treatment-related decisions by regulatory agencies, payers, healthcare providers, and patients. Given that planning RWE studies present diverse challenges, we developed the RWE Framework, a concise, visual, interactive tool designed to align multidisciplinary stakeholders toward common goals and encourage a methodical approach to RWE study planning.
A conceptual framework for a study design tool was developed based on best practices for RWE studies, enhanced with an infographic design, and refined by multidisciplinary input from RWE researchers.
Pilot testing using case studies of pharmaceutical assets demonstrated the utility of RWE Framework and applicability for use in team environments.
It addresses a broad range of real-world study types and research objectives and was found to enhance RWE decision-making by multidisciplinary teams. Further validation is warranted. Abstract Objectives: Real-world evidence RWE is essential for the development of pharmaceutical and medical technologies and informs treatment-related decisions by regulatory agencies, payers, healthcare providers, and patients.In this paper, we present a modular system capable of catching the attention of a new user, to detect in real-time events and emotions related to them in a stream of microblog posts.
The system is capable of making social sensing and exploiting the information arising on the Internet through user-generated contents, and it is equipped with a conversational engine that manages the interaction with the human user. The whole approach can be applied either by a human user or a robot, which remains a future application to be further improved in the context of our proposed system. This is a preview of subscription content, log in to check access.
Rent this article via DeepDyve. Anaheim, California, USA, pp — Lect Notes Artif Intell 2 — Google Scholar. Amati G, Van Rijsbergen CJ Probabilistic models of information retrieval based on measuring the divergence from randomness.
Barbosa L, Feng J Robust sentiment detection on twitter from biased and noisy data. Bifet A, Frank E Sentiment knowledge discovery in twitter streaming data. In: Discovery science, pp 1— J Mach Learn Res — Proc Comput Sci 35 1 — In: IEEE international conference on robotics and automation, vol 2. IEEE, pp — Artif Intell Med 44 2 — In: ACII, pp 37— Colbaugh R, Glass K Estimating sentiment orientation in social media for intelligence monitoring and analysis. Cordeiro M Twitter event detection: combining wavelet analysis and topic inference summarization.
In: Toward robotic socially believable behaving systems - volume I. Intelligent Systems Reference LibraryppSpringer. Cuzzocrea A, Pilato G Taxonomy-based detection of user emotions for advanced artificial intelligent applications. In: International conference on hybrid artificial intelligence systems.
Springer, Cham, pp — Cuzzocrea A, Fortino G, Rana O Managing data and processes in cloud-enabled large-scale sensor networks: state-of-the-art and future research directions.
Key Takeaways from FDA’s Framework for Real-World Evidence for Pharmaceuticals
Darling WM A theoretical and practical implementation tutorial on topic modeling and gibbs sampling. In: Proceedings of the 49th annual meeting of the association for computational linguistics: human language technologies, pp — Comput Hum Behav — Program 51 3 — Delaherche E, Dumas G, Nadel J, Chetouani M Automatic measure of imitation during social interaction: a behavioral and hyperscanning-eeg benchmark.
Pattern Recognit Lett —The Framework focuses in particular on the use of RWE to support regulatory decisions about effectiveness. The Cures Act also mandated that the agency publish a framework for implementing the RWE Program that describes the sources of RWE; the gaps in data collection activities; the standards and methodologies for collecting and analyzing RWE; and the priority areas, remaining challenges, and potential pilot opportunities that the RWE Program will address.
The new Framework covers drugs and biological products; FDA addressed the use of RWE in the context of medical devices separately in As a threshold matter, FDA underscores the distinction between real-world data and real-world evidence. FDA stresses the importance of continued engagement with all stakeholders in building out its framework.
Within this Framework, FDA sets forth an overarching plan to develop guidance in a number of specific areas, including: the reliability and relevance of RWD from medical claims and electronic health records used to generate RWE regarding drug product effectiveness; potential gaps in sources of RWD and strategies for addressing them one strategy identified in the Framework is exploring the use of mobile technologies, electronic patient reported outcome tools, wearables, and biosensors ; considerations for designing clinical trials that include pragmatic design elements and generate evidence of effectiveness for regulatory decisions; observational study designs using RWD, including whether and how these studies might provide RWE to support product effectiveness in regulatory decision-making; and as discussed in greater detail below, whether additional guidance is needed to address regulatory considerations when utilizing electronic source data.
Of note, the agency says that it will not address HIPAA in future guidance, but it leaves open the possibility that it might provide additional guidance on other aspects of data privacy and cybersecurity of electronic source data. Effectiveness Data. The RWE Program will focus on the potential use of RWE to support changes to labeling about drug product effectiveness, including adding or modifying an indication, adding a new population, or adding comparative effectiveness or safety information.
Although the Cures Act also calls for FDA to establish a program to evaluate the potential use of RWE for fulfillment of postapproval study requirements, the Framework contains little discussion of this potential use of RWE. FDA published the Framework shortly after issuing a proposal for regulating prescription drug-use-related software see our earlier blog post here.
That said, these documents does not discuss whether and how they relate to each other, even though both implicate software disseminated by or on behalf of a drug sponsor. Data Standards for Submissions. FDA recognizes the importance of developing data standards for submissions, to help ensure efficient review of RWD by the agency.
FDA touches on some of the regulatory considerations that arise from use of electronic source data, such as electronic health records and electronic data from clinical studies. The agency points out that it already has published some relevant information on these topics, including regulations that focus on the quality, authenticity, and reliability of electronic records 21 CFR Part 11 and a related guidance published in The agency highlights several key regulatory compliance issues, including informed consent, validation of electronic systems, audit trails for electronic records, and agency inspections.
FDA is considering whether additional guidance on the use of electronic source data is needed. All Rights Reserved.Smart environments, enabled by the Internet of Thing IoT paradigm, advocate for more intelligent and interconnected systems, electronic devices, tools, and appliances. While most efforts are nowadays addressed to provide connectivity or smartness to IoT devices, unfortunately, few have realised the importance of supporting automatic service composition and service reconfiguration capabilities at middleware level.
Due to the openness that characterise such environments, the range of possible interactions and available services and devices cannot be totally defined nor prescribed in advanced.
IT risk assessment frameworks: real-world experience
This uncertainty demands mechanisms to dynamically adapt existing systems, and their functionality, to address unforeseen needs. In order to do so, a general understanding of contexts, services, and device capabilities is needed. From that understanding, new responses can be automatically devised on run-time. This paper presents a semantic middleware specifically devised to support automatic and autonomous service reconfiguration and composition. The novelty consists in moving the semantics, traditionally held at the programming-interface level, to a common-sense knowledge-base system, with higher expressive power and reasoning capabilities.
A semantic middleware architecture for supporting real smartness. A semantic middleware architecture for supporting real smartness A semantic middleware architecture for supporting real smartness. Authors About Citation.
Maria J. David Villa. Felix Villanueva. Soledad Escolar. Juan Carlos Lopez. Santofimia, M. BibTex Citation. Robotics vs. Game-console based Platforms to Learn Computer Architecture.In crisis? This third edition retained the integrity of the model and is now positioned within the current context of population health, health promotion, and recovery.Why the Myers-Briggs test is totally meaningless
A Framework for Support, Third Edition, offers the best of something old and something new; and while the Framework has grown and changed over time, its original core principles continue to be as vibrant and relevant as ever. People with mental illness, their families, and the mental health professionals who try to support them are currently in a state of dynamic tension.
At no other time in history have there been in place the knowledge and understanding, the range of techniques, and the human resources to create the kind of revolutionary change in the lives of consumers that is now possible.
New therapies, the emergence of evidence-based programs, a new awareness of population health factors, and consumer and family empowerment all contribute to this powerful mixture. The source of the tension is clear. It can be found in the gap between what we know we can do and what we are actually doing. We can intervene early in psychotic illness and dramatically improve its course, but in most cases we do not.
We can house people effectively in ways that support independence and dignity, but in many cases we do not. We can support people in regular work and school settings, but in most cases we do not. We know that consumers can help each other if they have the resources, but in most cases they do not.
The list could be longer. Balancing these problems are successes. There are dozens of examples of effective and successful programs, treatments, and other models of support that are now in place and operating.
There is also a rapidly increasing number of consumer and family stories that speak volumes about the possibility of recovery and a dignified life in the community. These stories show that the future is to some degree already at hand. If the ingredients of change are in place, what is holding us back?
There are many factors, with perhaps political will, imagination, and money being the top three. Political will is needed at a time when stigma and discrimination against consumers remain stubbornly entrenched.
The ability to imagine, to look at the situation now and see in it the seeds of a different future, is also vital. This applies to the imagination of the public about their fellow citizens, of families about their relatives, of consumers about themselves, and of professionals about their clients. Money is needed to shift to new programs and ways of providing support, but it is money well invested in the long term because the individual and social costs of mental illness are lowered.
The Framework for Support focuses on three core areas to mark the way forward. The three areas are community, knowledge, and the personal resources needed to cope with mental illness.
It balances the service-focused bias of older policies by calling for full partnerships with consumers and families, and by recognizing the complex range of factors that shape the lives of consumers in the community. The focus on knowledge offers a model that fully engages the wide range of knowledge that we now possess. This includes, but goes beyond, best practices and evidence-based concepts to outline a rich convergence of kinds of understanding that range from the scientific to the experiential.
The focus on personal resources redefines the inner landscape of consumers from a repository of illness and symptoms to a dynamic mixture of skills and capacities that can successfully confront illness. Taken together, these focal points and other elements of the Framework model describe a process for moving ahead.
The major goal of the Framework model — people with serious mental illness living fulfilling lives in the community — requires a clear vision of the future if it is to become a reality.Interpersonal Skills:.
Subscribe to our FREE newsletter and start improving your life in just 5 minutes a day. Our page on Making Decisions discusses some of the issues around decision-making. This page describes one possible framework for making effective decisions. It is a seven-stage model, and was originally designed for use in groups and organisations. However, there is no reason why you cannot use the same method, or a simplified form, for decisions at home.
The important aspect is to go through all the stages in turn, even if only to decide that they are not relevant for the current situation. This process could include brainstorming or some other 'idea-generating' process. This stage is important to the overall decision making processes as a decision will be made from a selection of fixed choices. Always remember to consider the possibility of not making a decision or doing nothing and be aware that both options are actually potential solutions in themselves.
In deciding how much time to make available for the decision-making process, it helps to consider the following:. Remember that it is not always those making the decision who have to assume responsibility for it. Is it an individual, a group or an organisation? This is a key question because the degree to which responsibility for a decision is shared can greatly influence how much risk people are willing to take.
If the decision-making is for work, then it is helpful to consider the structure of the organisation. Finally, you need to know who can actually make the decision. When helping a friend, colleague or client to reach a decision, in most circumstances the final decision and responsibility will be taken by them.
Whenever possible, and if it is not obvious, it is better to agree formally who is responsible for a decision. This idea of responsibility also highlights the need to keep a record of how any decision was made, what information it was based on and who was involved.
Enough information needs to be kept to justify that decision in the future so that, if something does go wrong, it is possible to show that your decision was reasonable in the circumstance and given the knowledge you held at the time.
If there is inadequate or out-dated information then it is more likely that a wrong decision might be made. If there is a lot of irrelevant information, the decision will be difficult to make, and it will be easier to become distracted by unnecessary factors.With the signing of the 21 st Century Cures Actthe US Congress tasked the FDA with developing a framework to evaluate how the use of data from sources other than traditional clinical trials may be used to support drug approvals.
Effective Decision Making – A Framework
This framework will apply to post-market commitments and to new indications for approved drugs. This brings a further advantage to sponsors utilizing the b 2 pathway, on top of the already recognized benefit of streamlined development programs. So how and when will this option be available to sponsors, and has anything really changed? The 21 st Century Cures Act that was enacted into law in December describes the amendment of the Federal Food, Drug, and Cosmetic Act by inserting, among other things, a provision to assess the potential for use of real-world evidence RWE in drug approval programs.
We have previously blogged about earlier versions of this legislation here. The Act states that the framework that FDA must develop will cover gaps in data collection activities, standards and methodologies for collecting the data, and priority areas that the program will address.
The framework must be developed in consultation with industry, academia, medical professional organizations, patient advocacy organizations, consumer organizations, and disease research foundations, and must be implemented within 2 years of the enactment date. A draft guidance for industry must be issued within 5 years followed by a final document 18 months after public comment on the draft document.
The section further notes that real-world evidence may be used for other purposes when there is a sufficient basis for doing so, and that the Act does not alter standards of evidence under which studies are evaluated.
However, the datasets are often not designed to provide the level of evidence required for approval of a product, or for addressing regulatory questions. Instead, the data may be collected with the primary goal of determining treatment for an individual patient, insurance reimbursement, or performing financial analyses.
Not surprisingly, RWD is frequently not of the quality required to meet current regulatory standards. Problems may include missing data and its retrospective interpretation or potential remedy at the data collection stage, accuracy, lack of adverse event data, and unintended bias.
This is in contrast to clinical trials in which extensive measures are taken to reduce variability, to ensure the quality of the data collected, and to obtain detailed data on every adverse event that occurs. After the RWD has been generated, retrospective determination of appropriate methods of analysis can introduce further bias. This list is not exhaustive but gives an idea of how incorrect inferences can be drawn from analyzing RWD. The use of RWE to support regulatory approvals challenges the traditional paradigm in which the only authoritative evidence is generated through prospective randomized clinical trials RCTs.
The enactment of the 21 st Century Cures Act puts the FDA in a difficult situation in which it must establish the framework for evaluating real-world evidence that often does not contain the controls and methodology of the standards of a well-conducted clinical trial.
Yet the standard of evidence, of course, cannot be lowered. The FDA has begun to address the challenges by publishing scholarly articles defining RWE and the factors which improve its utility for regulatory purposes.
The FDA and the National Institutes of Health NIH are working on ways to harmonize data collected from EHRs, claims data, and registries, and to facilitate the provision of actual data that does not require external review or interpretation.
To its credit, the FDA has embraced the positive uses for RWE, particularly its potential for informing hypotheses and study design, and for increasing our knowledge of the effects of a product on more diverse populations than those studied in clinical trials. In fact, the FDA notes that for interventional studies, prospectively planned interventions and randomization can improve the validity of RWE.
The FDA has also raised the issue of the increasing costs of conducting clinical trials, without a corresponding increase in the quantity of evidence produced to support decisions about healthcare.