Ethical Preparedness and Performance of Gene Therapy Study Co-Ordinators

2008 ◽  
Vol 15 (2) ◽  
pp. 208-221 ◽  
Author(s):  
Gwen Anderson
Keyword(s):  
Gene Therapy ◽  
Clinical Trials ◽  
Ethical Issues ◽  
Research Integrity ◽  
Convenience Sample ◽  
Role Preparation ◽  
Perceived Preparedness ◽  
The Usa ◽  
And Performance

Little is known about study co-ordinators of gene therapy clinical trials. The purposes of this study were to: (1) describe characteristics of co-ordinators of gene therapy (transfer) clinical trials; (2) assess differences between nurse and non-nurse study co-ordinators; and (3) identify factors indicative of study co-ordinators' role preparation that could affect their role performance. This exploratory correlational study employed a convenience sample of 118 co-ordinators in the USA (55 participants; 47% response rate). The researcher created the Study Coordinator Role Preparedness and Performance Survey to assess factors or correlates of study co-ordinator performance. Analysis of variance was used to compare nurses and non-nurses, and men versus women on their perceived preparedness, perceived quality of orientation, and satisfaction with educational opportunities. The findings contribute to knowledge by identifying present inadequacies in the training of study co-ordinators and in recognizing the need for more effective provision of orientation and continuing education with respect to ethical issues, knowledge of genetic science, and potential research integrity challenges.

Reflections on FDA and WHO recommendations concerning clinical trials

1989 ◽  
Vol 4 (2) ◽  
pp. 117-122 ◽  
Author(s):  
J.-F. Dreyfus ◽  
D. Cremniter ◽  
J.D. Guelfi
Keyword(s):  
Clinical Trials ◽  
Drug Users ◽  
Objective Evaluation ◽  
World Health ◽  
Measuring Instruments ◽  
New Techniques ◽  
The Usa ◽  
The One ◽  
Health Organization

SummaryWe are still confronted by numerous different nosographic models and problems concerning the objective evaluation of patients progress during treatment. It is interesting to consider the consequences of this situation in psychiatry which still involves a relative diversity of practical methods used in clinical trials. The recommendations of the USA Food and Drug Administration, on the one hand, constitute a highly structured and precise reference. The World Health Organization, on the other hand, promulgates general recommendations resulting from a compromise designed to satisfy the greatest number of clinicians.Despite the apparently diverse principles and the different practical methods they propose, both those sets of recommendations have been useful in inspiring clinicians to reflect upon these different methodological approaches. The qualities of the inclusion criteria used in the study of patients and the sensitivity of the different measuring instruments have allowed psychotropic drug users as well as producers to recognize the need for a certain rigour in clinical trials.The FDA and WHO guidelines have certainly improved the quality of clinical trials in psychopharmacology. However, they also represent a source of resistance to innovation.A series of consensus meetings to first reconcile US and European points of view and later to include new techniques in the recognized sets of methods would therefore be helpful.

Ethics of First-in-Human Transplantation Trials of Bioartificial Organs

2021 ◽  
Vol 66 (Special Issue) ◽  
pp. 62-63
Author(s):  
Dide de Jongh ◽  
◽  
Eline Bunnik ◽  
Emma Massey ◽  
◽  
...  
Keyword(s):  
Gene Therapy ◽  
Clinical Trials ◽  
Regenerative Medicine ◽  
Ethical Issues ◽  
Artificial Organs ◽  
Diabetic Patients ◽  
Bioartificial Organs ◽  
Deceased Donors ◽  
Cell And Gene Therapy

"The most effective treatment for type 1 diabetes is transplantation of either a whole pancreas from a deceased donor or islet cells derived from multiple deceased donors. However, transplantation has several limitations, including shortage of post-mortem donors and the need for post-transplant patients to use life-long immunosuppressive medication. In the last decade, the field of regenerative medicine has combined engineering and biological technologies in the attempt to regenerate organs. The European VANGUARD project aims to develop immune-protected bioartificial pancreases for transplantation into non-immunosuppressed type 1 diabetic patients. This project is creating a ‘combination product’ using cells and tissue from a variety of sources, including placentas and deceased donors. The clinical development of this complex product raises ethical questions for first-in-human (FIH) clinical trials. Under what conditions can bio-artificial organs safely are transplanted in humans for the first time? How can patients be selected, recruited and informed responsibly? In this presentation, we investigate the ethical conditions for clinical trials of bio-engineered organs, focusing inter alia on study design, subject selection, risk-benefit assessment, and informed consent. We present the results of a review of the literature on the ethics of clinical trials in regenerative medicine, cell and gene therapy and transplantation, and specify existing ethical guidance in the context of FIH transplantation trials of bioartificial organs. We conclude that this new and innovative area at the intersection of regenerative medicine, cell and gene therapy and transplantation requires adequate consideration of the ethical issues in order to guide responsible research and clinical implementation. "

scholarly journals Research Integrity and Hidden Value Conflicts

2021 ◽  
Author(s):  
Gert Helgesson ◽  
William Bülow
Keyword(s):  
Ethical Issues ◽  
Research Integrity ◽  
Research Process ◽  
Process Data ◽  
Value Conflicts ◽  
Central Values ◽  
Open Discussion ◽  
Group 2 ◽  
Quality Of Research

AbstractResearch integrity is a well-established term used to talk and write about ethical issues in research. Part of its success might be its broad applicability. In this paper, we suggest that this might also be its Achilles heel, since it has the potential to conceal important value conflicts. We identify three broad domains upon which research integrity is applied in the literature: (1) the researcher (or research group), (2) research, and (3) research-related institutions and systems. Integrity in relation to researchers concerns character, although it remains to specify precisely what character traits are the desirable ones in this context and what values researchers should endorse. Integrity in relation to research concerns correct and sufficient description of the research process, data, results, and overall ‘research record’. Hence, it concerns the quality of research. However, whether or not this notion of research integrity covers all ethical aspects of research depends on whether one endorses a wider or a narrower interpretation of the ‘research process’. Integrity in relation to research-related institutions and systems leaves open whether they should be understood as agents in their own right or merely as means to research integrity. Besides the potential lack of clarity that our analysis reveals, we point to how this variety in uses might lead to concealment of value conflicts and propose an open discussion of central values.

scholarly journals Perspectives of people with inherited retinal diseases on ocular gene therapy in Australia: protocol for a national survey

BMJ Open ◽  
2021 ◽  
Vol 11 (6) ◽  
pp. e048361
Author(s):  
Heather G Mack ◽  
Fred K Chen ◽  
John Grigg ◽  
Robyn Jamieson ◽  
John De Roach ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Gene Therapy ◽  
Clinical Trials ◽  
Support Groups ◽  
Retinal Dystrophy ◽  
Retinal Disease ◽  
Related Quality ◽  
Biallelic Mutations ◽  
The University

IntroductionVoretigene neparvovec-rzyl (Luxturna) was approved by the Australian Therapeutic Goods Administration on 4 August 2020 for the treatment of biallelic mutations in the RPE65 gene, a rare cause of congenital and adult-onset retinal dystrophy (predominantly Leber congenital amaurosis). Previous studies have shown that individuals who might participate in gene therapy trials overestimate clinical effect and underestimate risks. However, little is known about the perspectives of patients who may be offered approved gene therapy treatment for ocular conditions (as distinct from participating in clinical trials of gene therapy). The main objective of this study is to develop a tool to assess knowledge, attitudes and perceptions of approved and future genetic therapies among potential recipients of ocular gene therapy. In addition, we aim to assess the quality of life, attitudes towards clinical trials and vision-related quality of life among this cohort.Methods and analysisA new ‘Attitudes to Gene Therapy for the Eye’ tool will be developed following consultation with people with inherited retinal disease (IRD) and content matter experts. Australians with IRD or their guardians will be asked to complete an internet-based survey comprising existing quality of life and visual function instruments and items for the newly proposed tool. We expect to recruit 500 survey participants from patient support groups, the practices of Australian ophthalmologists who are specialists in IRD and Australian ophthalmic research institutions. Launch is anticipated early 2021. Responses will be analysed using item response theory methodology.Ethics and disseminationThis study has received ethics approval from the University of Melbourne (#2057534). The results of the study will be published in a peer-reviewed journal and will be presented at relevant conferences. Organisations involved in recruitment, and the Patient Engagement Advisory committee will assist the research team with dissemination of the study outcomes.

scholarly journals SAFETY PROGRAM TO ADDRESS ETHICAL ISSUES IN CLINICAL TRIALS

2021 ◽  
Vol 9 (01) ◽  
pp. 1087-1089
Author(s):  
Maria Aziz ◽  
◽  
Azma J. Khan ◽  
Sefia Khan ◽  
◽  
...  
Keyword(s):  
Clinical Trials ◽  
Patient Safety ◽  
Staff Training ◽  
Ethical Issues ◽  
Educational Programs ◽  
Evidence Based ◽  
Safety Program ◽  
Site Staff ◽  
Training Opportunities

Ethical issues in clinical trials are of worth concern, hence for we propose a safety program that will help in achieving excellence in the conduct of the trials. Our paper presents a clear outline of the safety program under headings as: organizational structure remodeling, site staff training and educational programs, a plan for incident reporting and data collection.The proposed Safety Program has been designed on evidence based practice and is dedicated to achieve patient safety and quality assurance in clinical trials. The proposed program will help institutions to develop guidelines for better research outcomes, improve quality of projects, help in data management, provide training opportunities to site staff, ensure professional excellence and will help stakeholders to collaborate for patient safety.

scholarly journals Beyond transfusion therapy: new therapies in thalassemia including drugs, alternate donor transplant, and gene therapy

Hematology ◽  
2018 ◽  
Vol 2018 (1) ◽  
pp. 361-370 ◽  
Author(s):  
John Porter
Keyword(s):  
Quality Of Life ◽  
Health Care ◽  
Gene Therapy ◽  
Clinical Trials ◽  
Transforming Growth Factor ◽  
Transfusion Therapy ◽  
Hematopoietic Stem ◽  
Transfusion Requirements ◽  
Long Term Treatment

Abstract Transfusion combined with chelation therapy for severe β thalassemia syndromes (transfusion-dependent thalassemia [TDT]) has been successful in extending life expectancy, decreasing comorbidities and improving quality of life. However, this puts lifelong demands not only on the patients but also on the health care systems that are tasked with delivering long-term treatment and comprehensive support. Prevention programs and curative approaches are therefore an important part of overall strategy. Curative treatments alter the dynamic of a patient's health care costs, from financial commitment over 50 years, into a potential “one-off” investment. Since the 1980s, this has usually been available only to the 30% or so of young children with matched sibling donors. By improving the safety of matched related donors and haploidentical hematopoietic stem cell transplants, the potential size of the donor pool for curative therapies may be increased. Recent advances in gene therapy demonstrate that even patients lacking a matched donor can be rendered transfusion independent with an autograft of genetically modified autologous stem cells, with a low short-term risk. Noncurative treatments are also of potential value by decreasing use of blood and chelators and decreasing hospital visits. An example is luspatercept, an activin-receptor trap that modifies transforming growth factor-β signaling, thereby increasing the efficiency of erythropoiesis. This has entered phase 3 clinical trials for TDT and non-TDT and, usefully increases in both Hb and quality of life in non-TDT as well as decreasing transfusion requirements in TDT. Other novel noncurative treatments are entering clinical trials such improvement of erythropoiesis through pharmacological manipulation of hepcidin and iron metabolism.

Peer review audit of non-specialist occupational physician reports

2020 ◽  
Vol 70 (7) ◽  
pp. 503-506
Author(s):  
D Lalloo ◽  
J Gallagher ◽  
E B Macdonald ◽  
C McDonnell ◽  
S Vargas-Prada Figueroa
Keyword(s):  
Peer Review ◽  
Ethical Issues ◽  
High Standard ◽  
Occupational Physician ◽  
Assessment Instrument ◽  
Convenience Sample ◽  
Physician Group ◽  
Report Quality ◽  
Potential Development

Abstract Background With declining specialist occupational physician (OP) numbers, there is increasing recognition of the importance of non-specialist physicians in occupational health (OH) service delivery, yet to date, this physician group remains understudied and their competency requirements poorly understood. Aims To evaluate the quality of a sample of non-specialist OH reports and compare these with specialist reports. Methods A retrospective peer review audit of a convenience sample of 200 consecutive non-specialist and specialist OH reports from an Irish OH service using an assessment form based on the modified Sheffield Assessment Instrument for Letters SAIL(OH)1. Results Of the 200 peer reviewed OH reports, 159 (80%) were from non-specialists. For all questions, 87% and above of non-specialist reports were ‘satisfactory’ or ‘above expected’. On the overall assessment, out of 10, the mean non-specialist report score was 6.8 (standard deviation (SD) 3–10) and the specialist score was 7.3 (SD 3–10). Comparatively, non-specialist reports highlighted legal/ethical issues marginally more and adhered slightly better to contractual/ethical/legal boundaries, while specialist reports fared better in addressing manager’s questions, in their structure and clarity and in covering all significant aspects of the case, particularly if the case was complex. Conclusions Our findings demonstrate a high standard of OH report quality in this sample of non-specialist OPs that is consistent across all key OH report components. Potential development areas are also identified that can inform education/training tailored to this physician group and assist in competency standard-setting.

scholarly journals EFFECTS OF QUALITY OF BANK PRODUCTS AND SERVICES ON THE PERFORMANCE OF COMMERCIAL BANKS IN MAKUENI COUNTY

2020 ◽  
Vol 5 (1) ◽  
pp. 32
Author(s):  
Michael Nzuma Mutunga ◽  
Susan Njeri Wamitu
Keyword(s):  
Commercial Banks ◽  
High Performance ◽  
Ethical Issues ◽  
Target Population ◽  
Social Scientists ◽  
Data Collection Process ◽  
And Performance ◽  
Quality Of Products ◽  
Study Participants

Purpose: The main purpose of this study was to determine the effect of enhanced quality of bank products and services on the performance of commercial banks in Makueni County. Methodology: This study adopted a descriptive research design. The target population was all the 219 employees from the commercial bank branches in Makueni County from which 130 study participants were sampled; 65 bank managers and 65 assistant managers. A questionnaire was designed, piloted and used to collect data from the study participants. All necessary ethical issues were considered during and after data collection process. Statistical Package for Social Scientists Version 21 was used in data analysis to generate both descriptive and inferential statistics. Findings: There was a positive relationship between the quality of services and products and performance of commercial banks in Makueni County. The regression results also revealed that quality of products and services had a significant influence on the performance of commercial banks at p = 0.015.  Unique contributions to theory, practice and policy: The quality of bank services and products is a major determinant of the performance of commercial banks in the area of study. The findings were similar to those of another study carried out by Odeny (2016).  Since contingency theory argues that there is no single best approach in the management of institutions, the study recommends that continuous research is necessary in commercial banks to establish areas of service and product improvement for their continued high performance.

From Chimera to Reality: Lucy Kirkwood’s Chimerica or ‘What State Are We in?’

2018 ◽  
Vol 6 (1) ◽  
pp. 191-205
Author(s):  
Christine Kiehl
Keyword(s):  
21St Century ◽  
Ethical Issues ◽  
Western Democracy ◽  
Dramatic Form ◽  
Communist China ◽  
The Usa ◽  
Personal Level ◽  
Anglo American ◽  
Nationalism And Identity ◽  
And Performance

AbstractMark Ravenhill’s 2007 Shoot/Get Treasure/Repeat is a relevant state of the nation(s) play drawing an acid portrait of Anglo-American nations and their self-congratulatory ‘freedom and democracy’ propaganda in Irak. Other committed voices have made themselves heard on the British stage in addressing worldwide political and ethical issues. This paper focuses on Lucy Kirkwood, a young British dramatist whose pungent style was revealed in Tinderbox (2008), a dystopian farce set in 21st century England. Today, Lucy Kirkwood (33) who sees herself as ‘a radical young dramatist,’ continues to explore the confused landscape of western democracy: she sketched the relations between the USA and China in Chimerica (2013), an epic drama which won her an Olivier Award for Best New Play. In the wake of Brexit and the Trump election, Lucy Kirkwood has recently announced that she would pursue her investigation of the leading nations’ policies: “The whole of democracy looks fragile and farcical. After writing about communist China in Chimerica, you suddenly look at western democracy and think: is this necessarily better? Maybe this is the endgame” (Lawson, “Chimerica”).This paper explores Kirkwood’s vitriolic portrait of today’s leading nations, and her questioning of universal concerns experienced on a personal level such as power and privacy, nationalism and identity, profit and subservience. I will examine her peculiar ability to reformulate a ‘state-of-the-nation’ format and associate innovation and convention in her treatment of subject matter, language, dramatic form and performance.

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