Compassionate use of gene therapies in pediatrics: An ethical analysis

Neuhaus, Carolyn P.; Zacharias, Rachel

doi:10.1053/j.semperi.2018.09.010

Cited by 6 publications

(5 citation statements)

References 18 publications

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“…First, the expected knowledge to comprehend the dilemma was determined (condition, diagnosis, treatment, prognosis and ethics) ( Knowledge ), 7,9,11,12,15‐17,24‐28 as well as the different clinical management pathways for mothers with fetuses diagnosed with isolated severe left sided CDH ( Practice ). Based on the literature regarding RCTs and equipoise, and the discussion preceding the TOTAL trial, 15,16,29‐38 statements were formulated to create contrasting opinions ( Attitude ). The agreed draft was pilot‐tested for readability and understandability among a group of fetal‐maternal researchers (Data S1), by means of the “think‐aloud” method: the fetal‐maternal specialists were asked to read the question and repeat in their own words what the question was asking, followed by an explanation of how they would answer the question and why 39 .…”

Section: Methodsmentioning

confidence: 99%

The TOTAL trial dilemma: A survey among professionals on equipoise regarding fetal therapy for severe congenital diaphragmatic hernia

et al. 2020

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Objective Running randomized clinical trials (RCT) in fetal therapy is challenging. This is no different for fetoscopic endoluminal tracheal occlusion (FETO) for severe left‐sided Congenital Diaphragmatic Hernia (CDH). We assessed the knowledge, attitude and practice (KAP) of maternal‐fetal medicine specialists toward the antenatal management of CDH, and the randomized controlled clinical (RCT) “Tracheal Occlusion To Accelerate Lung growth‐trial.” Methods A cross‐sectional KAP‐survey was conducted among 311 registrants of the 18th World Congress in Fetal Medicine. Results The overall knowledge of CDH and FETO was high. Remarkably only 45% considers prenatal prediction of neonatal outcome reliable. Despite, in their clinical practice they perform severity assessment (80%) and refer families for FETO either within the context of an RCT (43%) or on patient request (32%). Seventy percent perceives not offering FETO on patient demand seems as if no treatment is provided to a fetus with predicted poor outcome. Only 20% of respondents considers denying access to FETO on patient demand not as a psychological burden. Conclusion Often the views of individual respondents contradicted with their clinical practice. It seems that, for severe CDH, clinicians face personal and practical dilemmas that undermine equipoise. To us, this indicates the tension between the clinical and scientific obligations physicians experience.

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Section: Methodsmentioning

confidence: 99%

The TOTAL trial dilemma: A survey among professionals on equipoise regarding fetal therapy for severe congenital diaphragmatic hernia

et al. 2020

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“…This uniqueness not only adds complexity but, when combined with inherent long-term clinical uncertainties, makes patient access to GTs highly problematic [30,31]. On one hand, denying access to these pioneering treatments not only poses a risk for patients but also raises ethical concerns [32], emphasizing the moral obligation to ensure fair access, especially in cases where GTs are the only therapeutic option in place [33]. On the other hand, manufacturers grapple with delicately balancing shareholder interests and societal responsibilities.…”

Section: Balancing Cost Access and Ethical Responsibilitiesmentioning

confidence: 99%

Navigating Gene Therapy Access: The Case of Bulgaria in the Context of the EU Regulatory Landscape

Kostadinov,

Marinova,

Dimitrov

et al. 2024

Healthcare

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Gene therapies (GTs) have recently emerged as revolutionary personalized therapeutic options. Despite their promising potential, challenges such as uncertainty regarding long-term health benefits and safety, along with extreme price tags, pose significant obstacles to patient access. Within the EU, the European Medicines Agency plays a pivotal role with regards to GT market authorization. However, national authorities are responsible for pricing and reimbursement, which results in fragment patient access within the EU. This study aimed to provide an overview of the complex landscape of post-market authorization accessibility for GT products in Bulgaria, comparing it with neighboring EU countries. We applied a mixed-methods approach, including desk research, public data requests, and list price comparisons. As of 1 April 2023, 14 GTs had a valid market authorization at the EU level. In Bulgaria, Kymriah® was the only GT included in the Positive Drug List (PDL), with an official list price of EUR 335,636.94. Similar results were found in Romania, whereas five GTs were included in Greece’s PDL. Additionally, Zolgensma® was found accessible in Bulgaria through an alternative individual access scheme at an estimated price of EUR 1,945,000.00. In conclusion, this study emphasized targeted policy interventions to address health inequalities and to ensure timely access to GTs within the EU.

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“…One of the most sensitive issues related to genetic interventions is related to the right of descendants to decide how they live their lives without this right being influenced by the decision of their predecessors. [52][53][54] The counterargument to this position is that children's autonomy is limited, and parents make many decisions that influence future children anyway. In this sense, if parents act to expand their children's life options by eliminating diseases, then this removes any possible restriction imposed by autonomy, and the limitation of unborn children's autonomy would be offset by the beneficial effects of increased autonomy after birth.…”

Section: Individual Autonomymentioning

confidence: 99%

Advances in Genetic Editing of the Human Embryo

Astărăstoae

Ioan

Rogozea

et al. 2023

American Journal of Therapeutics

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Background: Genetic engineering has allowed a major development of research in this field, with specialists attempting to edit the human genome, after the successful editing of the genomes of plants and animals. However, human gene editing technologies are at the center of ethical debates around the world.Areas of Uncertainty: Ethical concerns about genetic editing of the human embryo raise several issues that can be viewed through the prism of optimism and reluctance leading to a number of recommendations regarding the acceptance of what may soon become a reality.Data Sources: A literature search was conducted through PubMed, MEDLINE, Plus, Scopus, and Web of Science (2015-2022) using combinations of keywords, including: human genome or gene editing plus ethics.Ethics and Therapeutic Advances: Gene therapy is seen by researchers as a way to solve congenital diseases, multifactorial diseases in general or specific diseases such as cystic fibrosis, muscular dystrophy, or can increase resistance to HIV infection. Genome editing technologies, germline gene editing, clustered regularly interspaced short palindromic repeats gene editing technology, technologies such as zinc finger nucleases are not only advanced gene therapies that require solving technical problems, but also techniques that require complex and complete analysis of ethical problems. Genetic engineering raises many ethical concerns such as: safety concerns especially the risk of off-target effects; autonomy of the individual-with the limitation of the future generations to consent for an intervention over their genome; social justice-keeping in mind the costs of the procedures and their availability to the general population. Discussions can go further from questions such as "How can we do this?" to questions such as "Should we do this?" or "Is society ready to accept this technology and is it able to manage it rationally?" Conclusions: The ethics of biomedical research should be based on global dialogue, on the involvement of experts and the public, to achieve a broad social consensus. The fundamental review of the ethics of genetics is a desire and an opportunity of the current period.

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Compassionate use of gene therapies in pediatrics: An ethical analysis

Cited by 6 publications

References 18 publications

The TOTAL trial dilemma: A survey among professionals on equipoise regarding fetal therapy for severe congenital diaphragmatic hernia

The TOTAL trial dilemma: A survey among professionals on equipoise regarding fetal therapy for severe congenital diaphragmatic hernia

Navigating Gene Therapy Access: The Case of Bulgaria in the Context of the EU Regulatory Landscape

Advances in Genetic Editing of the Human Embryo

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