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Bayesian Adaptive Designs in Drug Development
Published in Emmanuel Lesaffre, Gianluca Baio, Bruno Boulanger, Bayesian Methods in Pharmaceutical Research, 2020
This chapter presents an overview of Bayesian adaptive designs in clinical studies. Clinical studies across the drug development spectrum have included various forms of pre-specified adaptation for a long time, sometimes in an ad hoc manner (e.g. the so-called 3+3 dose escalation design — see Chapter 7) and often based on frequentist considerations (e.g. the O’Brien-Fleming group sequential design, O’Brien and Fleming, 1979). Interest in applying Bayesian considerations when designing clinical studies began to grow during the last quarter of the twentieth century. Part of the reason interest has grown is the greater flexibility afforded by the Bayesian approach to statistical inference. The Bayesian paradigm is one of learning. The paradigm provides the inferential tools to use information, be it data-derived or opinion-based, to increase certainty about treatment effects, associations, or whatever the focus of the analysis is. Much of the recent interest stems from interest in adaptive randomization, in which data from earlier enrollees influence treatment assignment probabilities for later study participants.
Mutuality
Published in Nigel Starey, Health and Social Care in the Digital World, 2020
The end of the previous chapter outlined features that have made the “medical model” the predominant paradigm of the primary care sector during the last seventy years. In reality this paradigm is really a professional rather than simply a medical model. The whole physiology of the sector is based on the relationship between professionals and citizens, and driven by a culture and set of values which describe a view of professionalism. All the clinical professions involved in providing care in the sector have been part of this medical model or “professional paradigm”. The roots of this model of “professionalism” stretch back into history (see Chapter 3) but need to be refreshed in this digital world of universal access to data and information; the more mature, educated and empowered citizen; and the requirement on all professionals (from clergy to solicitors, teachers and bankers) to be more open and transparent about their performance and more accountable to the people they serve and to the general public.
Orthomolecular Approaches for the Use of Intravenous Vitamin C
Published in Qi Chen, Margreet C.M. Vissers, Cancer and Vitamin C, 2020
What may be lost in current scientific reports are nuances of in-office administration that may be appreciated when patients are treated outside of protocols. Those of us who trained with the pioneers learned by sitting with patients and observing their reactions during IV administration, documenting progress or lack thereof through laboratory testing and imaging, and learning to make decisions about dosing. After all, this is about patients, not about paradigms.
Utilization of experimental design in the formulation and optimization of hyaluronic acid–based nanoemulgel loaded with a turmeric–curry leaf oil nanoemulsion for gingivitis
Published in Drug Delivery, 2023
Amal M. Sindi, Khaled M. Hosny, Waleed Y. Rizg, Fahad Y. Sabei, Osama A. Madkhali, Mohammed Ali Bakkari, Eman Alfayez, Hanaa Alkharobi, Samar A Alghamdi, Arwa A. Banjar, Mohammed Majrashi, Mohammed Alissa
Ever since the World Health Organization broadened the definition of health in 1948, there has been an acknowledgement of the quality-of-life factors associated with health (Baiju et al., 2017). As a result, the medical model of health and disease has given way to a biopsychosocial paradigm (Pinheiro et al., 2017). In addition to the absence of disease, oral health also refers to a person’s general health, which allows the person to engage in daily activities such as eating, talking, and smiling, as well as to make creative contributions to society (Nguyen et al., 2017). The health-related quality of life focuses on how illness and the treatment of illness affect the quality of life. Different hypothetical models have been put forth to explain the concept, with Wilson and Cleary’s conceptual model from 1995 being the most thorough (WHOQoL Group, 1995). The definition of the oral health-related quality of life is still nebulous despite extensive study and thousands of articles written about it. In clinical dentistry practice, dental education, and dental research, the patient’s perception of his or her oral health and associated life quality is important (Schwartzmann, 2003). It has been amply demonstrated that oral health issues can affect daily life in a variety of ways. Numerous scales or measurements are available to evaluate this. They vary depending on the type of answer, the number of items, the application setting, and the target population (Gift & Atchinson, 1995).
ECMO as a Palliative Bridge to Death
Published in The American Journal of Bioethics, 2023
Rachel Rutz Voumard, Zied Ltaief, Lucas Liaudet, Ralf J. Jox
We invite clinicians to consider a paradigm shift. In such situations as presented above, the goal of care is no longer to extend life until recovery or organ transplantation. Instead, the goal of ECMO in these situations can be to attain a sufficient quality of life, even if this is only for a limited period before death. ECMO is a life-sustaining technology that, more than any other form of assistance, allows clinicians to go beyond the limits of organ viability and survival. Yet, ECMO therapy is also associated with unavoidable severe complications (thrombo-embolic, hemorrhagic, infectious), jeopardizing the patient’s consciousness and survival (Combes 2018). As any other life-sustaining treatment, it can increase quantity and quality of life but ultimately does not exempt from death. We face similar challenges regarding artificial nutrition or hydration for terminally ill patients. In such challenging cases, it may be beneficial and respectful to offer a delay, value its temporality, and support patients and families in their highly personal end-of-life process. While maintaining ECMO treatment, withholding treatment of future complications may be seen as more proportionate for awake patients on ECMO that withdrawing ECMO.
The next phase in the implementation of value-based healthcare: Adding patient-relevant cost drivers to existing outcome measure sets
Published in International Journal of Healthcare Management, 2023
Gijs J. van Steenbergen, Paul Cremers, Lukas Dekker, Dennis van Veghel
Healthcare expenditure is increasing globally and in many developed countries, healthcare costs are outgrowing economic growth and even account for more than 10% of the gross domestic product (GDP) [1]. In the following years, a further increase in demand and expenditure of healthcare is expected due to more therapeutic options, increasing amount of patients with chronic conditions, increasing prosperity, life style deterioration, general ageing of the population and demographic growth [1]. In addition, most healthcare systems in developed countries are volume-driven and predominantly use fee-for-service or case-based (diagnosis-related group) reimbursement models [2]. These healthcare models are focussed on the volume of care delivered and not the quality of care provided [3]. As a result, significant disparity in outcomes exists among centres worldwide or even in the same geographical areas [4]. Given the mentioned challenges, there is increased attention for a paradigm shift from a volume-driven to a value-driven system to keep healthcare systems of good quality and financially sustainable [1].