Considerations In Clinical Research Research Articles

H - 17 Neuropsychological Assessment in Rare Pediatric Neurogenetic Disorders: Considerations for Clinical Research.

Approximately 80% of rare diseases in childhood have a genetic cause. Neuropsychological assessments offer a greater understanding of the clinical presentation and trajectory of neurogenetic disorders. Given the rarity of the conditions, the limited funding allocated to study psychosocial factors, the diversity within the patient populations (e.g., country of origin, clinical phenotypes), and the limitations of our tools, there are no clear guidelines for evaluations. We aim to present a systematic approach to create assessment protocols that track disease progression and/or efficacy of therapeutic interventions. Our gene therapy program has created nine assessment protocols to evaluate conditions ranging from epileptic encephalopathies, lysosomal storage, neuromuscular, and mitochondrial diseases. Through review of past protocols, we created a neuropsychological assessment selection checklist (NASC) and standard operating procedure (SOP) checklist. The NASC systematizes updated multidisciplinary disease knowledge and relevant desirable outcomes (e.g., cognitive, motor, quality of life). Cultural elements (e.g., beliefs around health, disease, testing) and necessary accommodations (e.g., interpreters, sensory impairments) must be considered. Additional sociocultural and socioeconomic information of the sample is germane not only to contextualize results but also to help narrow possible tools. The SOP aims to increase standardization (e.g., starting points) and maximize use of the data collected (e.g., deferment reasons.). Neuropsychological assessments offer a wealth of cognitive, behavioral, emotional, and adaptive functioning data that is crucial to better understand the progression and treatment of rare neurogenetic diseases. Cultural considerations and in-depth clinical and assessment skills are essential to creating evaluation protocols that are valid, useful, and comprehensive.

Understanding of Scales of Measurements and its Consideration in Clinical Research

In the present digital world, regardless of scale of measurements, often collected data are likely to be inappropriately considered as numeric. It might be mainly because even data on qualitative cum categorical variables are collected using certain numeric codes which look like quantitative instead of qualitative variables. Such inappropriate understanding about scales of measurements often leads to consideration of inappropriate analytical methods providing inaccurate results. Hence, such practice results to not only inaccurate interpretations but sometimes disastrous implications. Therefore, understandings of scales of measurements go in a long way to appropriately plan & execute study; collect accurate data, analyze appropriately, interpret results accurately and derive policy driven implications.

Dupilumab for the treatment of adult atopic dermatitis in special populations

Background Special populations (SPs) involve people who require additional consideration in clinical research. Effectiveness of treatment or occurrence of side effects may be different in SPs with respect to not-SPs. Objectives To retrospectively compare the effectiveness and safety of dupilumab in AD treatment of SPs versus not-SPs. Methods A 52-weeks retrospective study was performed enrolling patients with a diagnosis of moderate-to-severe AD undergoing treatment with dupilumab at labeled dosage. Patients were divided in Group A (SPs patients) and Group B (not-SPs patients). Disease severity was assessed using Eczema Area Severity Index (EASI), Pruritus–Numerical Rating Scale (P-NRS), and Dermatology Life Quality Index (DLQI) score at baseline and after 4 weeks (W4), W16, W24, and W52. Results A total of 263 patients were enrolled and divided in Group A (25) and Group B (238). SPs included history of cancer, severe kidney failure, viral hepatitis, neurological diseases, acquired immunodeficiency syndrome, and transplanted patients. A statistically significant reduction of EASI, DLQI, and P-NRS was assessed in both groups at each follow-up visit (p < .0001), without significant differences between the groups. No differences were recorded for safety. Conclusions There are not significant differences between SPs and not-SPs as regards effectiveness and safety of dupilumab in AD management.

Ethical Considerations in Clinical Research: A Comprehensive Review

Ethical Considerations in Clinical Research: A Comprehensive Review

Emerging treatments for lupus nephritis: health equity considerations in clinical research and coverage.

DISCLOSURES: No funding was received for the writing of this commentary. The authors report no conflicts of interest.

Principles of ethical consideration required for clinical research involving children.

Ethical considerations are more stringent in pediatric clinical research than in research targeting adults. However, in Japan, clear guidelines have yet to be presented on the necessary ethical considerations for clinical research involving children. The "Principles of Ethical Consideration Required for Clinical Research Involving Children" provide guiding principles for ethical considerations and the essential ways of thinking that all involved in clinical research on children need to understand in advance.

The Need to Objectively Measure Physical Activity During Pregnancy: Considerations for Clinical Research and Public Health Impact.

Engaging in recommended levels of physical activity during pregnancy can provide a host of physical and mental health benefits for the expecting mother and her child. However, methodological issues related to physical activity measurement have plagued many studies examining the effects of physical activity during this important life stage. Burgeoning support exists for the more widespread use of objective methods, and accelerometers specifically, for an accurate appraisal of maternal physical activity. In this commentary, we highlight discrepancies between activity estimates obtained via self-report and objective measures and describe the implications of erroneous measurement when making clinical recommendations and in conducting future physical activity and pregnancy research. Most importantly, we aim to foster academic discussion and propose a call to action requiring a paradigm shift where we acknowledge the shortcomings of self-report and move toward an empirically driven approach for physical activity measurement. Results from more high-quality research studies will help support public health messaging and facilitate trust among health care providers, clinical researchers, and expecting mothers regarding the health benefits of physical activity recommendations.

Ethical Considerations for Clinical Research and Off-label Use of Ketamine to Treat Mood Disorders: The Balance Between Risks and Benefits

Previous research conducted in 1999 highlighted ethical concerns behind challenge studies inducing psychosis with ketamine and made recommendations to enhance ethical standards. Recently, a plethora of clinical trials have evaluated the efficacy of ketamine to treat mood disorders, which lead to complex ethical issues. Pharmaceutical companies and researchers hope to profit by developing patentable variations on ketamine for treating depression. Media have labeled ketamine as a “miracle” antidepressant. Some clinics offer expensive off-label use of ketamine to treat mood disorders. This article examines the ecological validity of ketamine trials, measures to protect patients, informed consent procedures, financial inducements to participants and conflict of interest of researchers, therapeutic misconception, concealment, and deception. Further recommendations are purposed to improve ethical standard of clinical research involving ketamine.

Ethical considerations in clinical research on herbal medicine for prevention of cardiovascular disease in the ageing

BackgroundCardiovascular disease (CVD) in the ageing is a major public health problem worldwide. The nature of most CVD is subclinical with pathological processes that can span over years. Use of preventive measures could be an appropriate approach to prevailing over CVD in the ageing, and herbal medicine is one of the promising preventive approaches and is currently of interest among medical societies. In the evidence-based era, herbal medicine is, however, often underestimated and approached with skepticism, mainly due to the paucity of scientific evidence. Properly designed clinical trials on herbal medicine for prevention of CVD in a geriatric population are thus of importance and of clinical value. PurposeTo review ethical issues and discuss considerations when such research is proposed. Chapters/sectionsFour ethical issues, including the scientific validity of research, risk-benefit assessments, subject selection and vulnerability, and informed consent, are structured and extensively discussed in this article. ConclusionsEthical core considerations of prevention research of CVD on herbal medicine involve particular attention on the scientific validity of research, risk-benefit assessments, subject selection and vulnerability, and informed consent. These issues and considerations are keys, although they must be adapted to an individual research setting in which a clinical study is proposed.

English

English

Empathy, sex and fluid intelligence as predictors of theory of mind

Individual differences in theory of mind (ToM) are affected by a variety of factors. We investigated the relationship between empathy, sex and fluid intelligence (FI) as predictors of ToM in a random probabilistic sample of secondary students. First, we explored whether sex, as well as high, average or low levels of empathy and FI affect ToM performance. Furthermore, we assessed the contribution of empathy, sex and FI in predicting ToM by using a path analysis. This method allows testing of causal models of directed dependencies among a set of variables. The causal dependencies of empathy, sex and fluid intelligence were confirmed and identified. In addition, the model confirmed the direct effect of empathy, sex and fluid intelligence on ToM; and the indirect effect of sex mediated by empathy. Thus, individual differences in ToM levels are partially attributable to sex, empathy and fluid intelligence variability, raising important considerations for clinical research as well as ToM’s theoretical models of domain specificity.

Ethical Considerations in Clinical Research

Ethical Considerations in Clinical Research

The screening mammography paradox: better when found, perhaps better not to find

The screening mammography paradox: better when found, perhaps better not to find

Foreword: Envisioning Health Disparities

I. INTRODUCTION No one approves of it. Our laws prohibit it. Yet racism will not go away. We continue to demonstrate that a colorblind society is just a society where color lurks in the blind spot. Disparities in healthcare offer a telling illustration of how durably racism is woven in our social fabric, and how easy it is for subtle, unconscious differences in treatment to add up to significant disparities in outcome. Disparities also give us opportunity to see health differently. Instead of focusing on the outcomes of individual cases, disparities invite us to examine the social structures and processes through which the level and distribution of health are determined. Like racism, health is to a considerable degree a social product, reflecting how we order the world rather than our biological or genetic limits. The articles in this Symposium reflect a growing willingness in health and healthcare research to place individual outcomes in a larger, ecological framework.1 They are part of a process of social learning, a process of changing the focus from the final pathological outcomes that now more or less dramatically define our problems to the durable and flexible eco-social machinery that generates the problems and determines how they will be distributed. Solving problems, we are learning, is on its own insufficient: the problem-generating machinery simply makes new ones. The trick is to study the problem production system, and find structural interventions that begin to disrupt it.2 No single law review article can be expected to provide the solution to the problem of health disparities, but each article in this issue is a contribution to our ability to understand the problem and make that understanding salient to policy-makers and the public. In this Foreword, I suggest that the most important job of the health law or health services scholar tackling health disparities is to consider how law operates in relation to other social determinants as a cause of disparities,3 and how it may be used as a structural intervention to influence the social processes that produce them.4 II. RACISM AS A STIGMA Race as described in the articles in this Symposium is essentially a form of stigma, an enduring condition, status, or attribute that is negatively valued by a society and whose possession consequently discredits and disadvantages individual.5 Both prejudice-negative attitudes-and discrimination-overt negative behavior-may flow from and help enforce a stigma, but the real power of stigma as a form of social control is that the negative valuation of the stigmatized trait is built into people's basic understanding of the world: it is not opinion, but a fact; it is not asserted, but taken for granted. Racism can thus be seen as a social process that continues to operate throughout social life even when specific manifestations are universally disfavored. Jim Crow has been repudiated, and racial equality is a valued norm, yet a country full of people who believe in equality and oppose segregation remains physically divided along racial lines. The question of whether race has sufficient biological reality to require or justify its active consideration in clinical research is still being debated,6 but we cannot advance public health or healthcare without accepting the reality of racism in our society.7 As Ross and Walsh point out in their contribution to this Symposium, racism as a cultural force could be producing disparities even if race as a biologic factor makes no contribution.8 The American concept of race is really quite a formidable achievement. Given how undramatically we Americans ultimately differ in shape, genes, culture, values and so on, a visitor from another planet might well be impressed with how intricately we can sort each other by subtle differences in shading and social position. A great deal of effort has gone and continues to go into making sure that we can recognize, respect and enforce racial differences. …

Ethical considerations in clinical research

Ethical considerations in clinical research