scholarly journals Crowd Funding for Orphan Drugs: The Case of Baby Pia

2021 ◽  
Vol 12 ◽  
Author(s):  
Wim Pinxten
Keyword(s):  
Ethical Issues ◽  
Orphan Drugs ◽  
New Strategy

Medical crowdfunding is a relatively new strategy to obtain access to orphan drugs. The case of Baby Pia, a Belgian girl with SMA type 1 for whom in 2018 more than $ 2.1 million was raised to get her treated with Zolgensma®, illustrates well the potential power of medical crowdfunding. But apart from the success in raising money, the case is also of particular importance for the ethical issues it brings to the surface as related to justice, equity, power imbalances, responsibility, accountability, indebtedness and privacy.

scholarly journals Left Of Bang Interventions in Trauma: ethical implications for military medical prophylaxis

2017 ◽  
Vol 44 (7) ◽  
pp. 504-508 ◽  
Author(s):  
Neil Eisenstein ◽  
David Naumann ◽  
Daniel Burns ◽  
Sarah Stapley ◽  
Heather Draper
Keyword(s):  
Side Effect ◽  
Ethical Issues ◽  
Concept Development ◽  
Development Research ◽  
Ethical Implications ◽  
Medical Context ◽  
The Military

Advances in medical capability should be accompanied by discussion of their ethical implications. In the military medical context there is a growing interest in developing prophylactic interventions that will mitigate the effects of trauma and improve survival. The ethics of this novel capability are currently unexplored. This paper describes the concept of trauma prophylaxis (Left Of Bang Interventions in Trauma) and outlines some of the ethical issues that need to be considered, including within concept development, research and implementation. Trauma prophylaxis can be divided into interventions that do not (type 1) and those that do (type 2) have medical enhancement as an unintended side effect of their prophylactic action. We conclude that type 1 interventions have much in common with established military medical prophylaxis, and the potentially enhancing qualities of type 2 interventions raise different issues. We welcome further debate on both interventions.

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 Topical Review: Crowdsourcing as a Novel Approach to Qualitative Research

2020 ◽  
Author(s):  
Erica Sood ◽  
Tim Wysocki ◽  
Melissa A Alderfer ◽  
Karen Aroian ◽  
Jennifer Christofferson ◽  
...  
Keyword(s):  
Qualitative Research ◽  
Ethical Issues ◽  
Emotional Engagement ◽  
Personal Contact ◽  
Novel Approach ◽  
Crowdsourced Data ◽  
Hard To Reach Populations ◽  
Five Phases ◽  
Iterative Feedback

Abstract Objective  To describe a novel, five-phase approach to collecting qualitative data from hard-to-reach populations using crowdsourcing methods. Methods  Drawing from experiences across recent studies with type 1 diabetes and congenital heart disease stakeholders, we describe five phases of crowdsourcing methodology, an innovative approach to conducting qualitative research within an online environment, and discuss relevant practical and ethical issues. Results  Phases of crowdsourcing methodology are: (I) Preparing; (II) Forming Crowds; (III) Collecting Crowdsourced Data; (IV) Coding and Analyzing Crowdsourced Data; and (V) Generating and Disseminating Findings. Iterative feedback from stakeholders is obtained in all five phases. Practical and ethical issues include accessing diverse stakeholders, emotional engagement of crowd participants, responsiveness and transparency of crowdsourcing methodology, and limited personal contact with crowd participants. Conclusions  Crowdsourcing is an innovative, efficient, feasible, and timely approach to engaging hard-to-reach populations in qualitative research.

scholarly journals Personalized medicine: Legal and ethical challenges

2020 ◽  
Keyword(s):  
Type 1 Diabetes ◽  
Personalized Medicine ◽  
Ethical Issues ◽  
Personalised Medicine ◽  
Academic Community ◽  
Final Report ◽  
Ethical Challenges ◽  
Research Activity ◽  
Computational Data

This anthology deals with the legal and ethical challenges regarding personalized (precision) medicine and healthcare. It can also be regarded as the final report of a research project on the legal and ethical aspects of personalized medicine. It complements the reported results of the consortium ‘Personalised medicine to predict and prevent Type 1 Diabetes (P4 Diabetes)’ which were briefly presented in the booklet entitled ‘Better, Smarter, Now: Personalised Health – From Genes to Society (pHealth)’, Academy of Finland, Helsinki 2019. The articles of this anthology are not limited to the aspects of predicting and preventing Type 1 Diabetes only, as the name of the consortium would suggest. The list of participating researchers indicates that many-sided medical expertise was represented in the consortium and, in addition, computational data analysis as well as legal and ethical issues were covered by the participating sites of research. A comprehensive examination of the issues of personalized medicine requires multidisciplinary approaches. In this anthology, the legally and ethically oriented mainstream of writings has been complemented with an article of a computer scientist in order to recognize the possibilities and challenges of machine learning when interpreting the patient’s need for help. It is our hope that this anthology would be useful both for the academic community and for the decision-makers in the fields of healthcare and (personalized) medicine. It is also advisable that the anthology would give an impetus for further research activity in these new spheres of medical law and biolaw.

scholarly journals A new strategy for vascular complications in young people with type 1 diabetes mellitus

2019 ◽  
Vol 15 (7) ◽  
pp. 429-435 ◽  
Author(s):  
M. Loredana Marcovecchio ◽  
◽  
R. Neil Dalton ◽  
Denis Daneman ◽  
John Deanfield ◽  
...  
Keyword(s):  
Diabetes Mellitus ◽  
Type 1 Diabetes ◽  
Young People ◽  
Type 1 Diabetes Mellitus ◽  
Vascular Complications ◽  
New Strategy

Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I

2011 ◽  
Vol 37 (4) ◽  
pp. 233-239 ◽  
Author(s):  
R. Boy ◽  
I. V. D. Schwartz ◽  
B. C. Krug ◽  
L. C. Santana-da-Silva ◽  
C. E. Steiner ◽  
...  
Keyword(s):  
Ethical Issues ◽  
Orphan Drugs ◽  
Type I ◽  
Mucopolysaccharidosis Type ◽  
Mucopolysaccharidosis Type I

scholarly journals Efficacy of program for treatment and training of patients with type 1 diabetes

1994 ◽  
Vol 40 (3) ◽  
pp. 15-19
Author(s):  
Ye. G. Starostina ◽  
M. B. Antsiferov ◽  
G. R. Galstyan ◽  
I. I. Dedov
Keyword(s):  
Diabetes Mellitus ◽  
Age Groups ◽  
Basic Principles ◽  
Results Of Treatment ◽  
Glycemia Level ◽  
New Strategy ◽  
Traditional Approaches ◽  
And Training ◽  
Insulindependent Diabetes

The authors analyze the results of comprehensive, prospective, controlled investigation of the program for intensive care and training of 121 patients with insulindependent diabetes mellitus (IDDM). The basic principles of the program are training on a teams basis, intensive insulin therapy, automonitoring of metabolism, and liberal diet. The results demonstrate that glycemia level approaching the normal may be attained and maintained in the majority of patients without increasing the risk of grave hypoglycemia, cases with severe diabetic ketoacidosis may be eliminated, and periods of temporary invalidity of patients reduced by 7-10 times. The selected training program was equally effective for patients of various age groups and levels of education, as well as for patients with labile diabetes. Results of treatment did not depend on the type of insulin preparations used. The authors conclude on a higher efficacy of new strategy of IDDM treatment in comparison with the traditional approaches.

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