The Lived Experience of Pediatric Gene Therapy: A Scoping Review.

Little is known about patients' and families' lived experiences of participating in pediatric gene therapy (GT) clinical trials. Currently, pediatric GT research targets a broad range of indications--including rare and ultra-rare diseases--which vary in severity and in the availability of alternative therapies. Pediatric GT differs meaningfully from adult GT because the decision to participate involves a dyad of both the child and parent or caregiver/s. It is critical to understand patients' and caregivers' perceptions and experiences of social, emotional, physical, and logistical burdens or benefits of participating in such trials, and how they weigh and prioritize these factors when deciding whether to participate. We conducted a scoping review of the current literature in this subject area with objectives to (1) provide an overview of existing literature, (2) identify gaps and areas for further research, and (3) better understand the lived impact of pediatric GT research on patients and their parents/caregivers. Four themes emerged, including (1) weighing risks and benefits (2) timing of GT trial participation, (3) value of clear communication, and (4) potential impact on quality of life. Notably, our sample surfaced articles about how patients/parents/caregivers were thinking about GT-their understanding of its safety, efficacy, and risks-rather than accounts of their experiences, which was our initial intention. Nevertheless, our findings offer useful insights to improve the informed consent process and promote a more patient- and family-centered approach. Moreover, our findings can contribute to patient advocacy organizations' efforts to develop educational materials tailored to patients' and families' expressed informational needs and perspectives, and can inform more patient- and family-centered policies from GT clinical trial sponsors.

Off-Label Prescription of COVID-19 Vaccines in Children: Clinical, Ethical, and Legal Issues.

The US Food and Drug Administration (FDA) approval of the biologics license application for the Pfizer-BioNTech coronavirus disease 2019 vaccine (Comirnaty) on August 23, 2021, opened the door to the off-label vaccination of children younger than the age range currently covered by either the biologics license application (16 years old and older) or the emergency use authorization (12 to 15 years old). Although prescribing medications at doses, for conditions, or in populations other than those approved by the FDA is generally legal and is common in pediatrics, the FDA, the Centers for Disease Control and Prevention, and the American Academy of Pediatrics have recommended against off-label prescription of the coronavirus disease 2019 vaccine. Several commentaries consider a case in which parents ask their child's pediatrician to prescribe the vaccine for their 11-year-old with special health care needs before approval or authorization in her age group. The first commentary considers the potential benefits and risks to the patient, as well as to the family, the provider, and society, emphasizing the unknown risks in younger patients and the need for adequate informed consent. The second commentary describes an algorithm and principles for evaluating off-label prescribing and argues that the current benefits of prescribing Comirnaty off label to children <12 do not outweigh the risks. The third commentary addresses ethical and legal issues, ultimately calling on federal agencies to remove legal barriers to making the vaccine available to children in age groups that currently lack authorization.

Genomic Testing, Unexpected Consanguinity, and Adolescent Parents.

This case study about an infant with epidermolysis bullosa and his fourteen-year-old mother involves pediatric genomic testing and concerns about the adolescent parent's decision-making capacity. Four commentaries explore ethics issues in the case, including the following challenges: The current ethics guidelines regarding informed decision-making for genomic testing and return of results have been relatively silent on how to involve parents who are minors in decision-making on behalf of their children. This lack of guidance can be particularly difficult for physicians when genetic test results revealing consanguinity raise concerns about sexual abuse of a minor, provoking questions about mandatory reporting requirements. Finally, medical teams may find themselves having to evaluate an adolescent parent's capacity in making medical decisions for her child while questioning the role and relationship of her support person.

Ethics of Pediatric and Young Adult Medical Decision-Making: Case-Based Discussions Exploring Consent, Capacity, and Surrogate Decision-Making.

Introduction: Most medical decisions in pediatrics involve surrogate decision-makers. Because of this, pediatricians are even more likely to encounter ethical conflicts and dilemmas surrounding medical decision-making. Pediatricians continue to report a lack of preparedness to manage situations when conflicts and dilemmas arise, suggesting a gap in education. In response to this gap, we developed a module on the ethics of medical decision-making focused on pediatrics. Methods: The Ethics of Pediatric and Young Adult Medical Decision-Making module included three case-based, small-group sessions on decision-making capacity and advance directives, parental decision-making, and informed consent and adolescent assent. Session materials were developed based on expert opinion and previously published content. Sessions were developed for pediatric residents; however, medical students rotating on pediatrics also participated in most sessions. Trainees completed pre- and postsession assessments of comfort and understanding. Results: An average of 19 learners completed each session. Understanding of ethical principles increased after each session. Seventy-nine percent of trainees reported increased understanding of ethical principles related to decision-making capacity, and 88% reported increased understanding of standards of surrogate decision-making. Following the session on obtaining consent and assent, 71% of trainees reported comfort obtaining consent compared to 57% reporting comfort obtaining assent. Discussion: This module successfully increased trainee comfort with many ethical issues related to pediatric medical decision-making. Areas where trainee comfort was still low postsession-specifically, obtaining consent or assent-are content areas where actual practice of these psychomotor skills is likely necessary.

Teaching Ethics and Professionalism: A National Survey of Ophthalmology Residency Program Directors.

Background While ethics and professionalism are important components of graduate medical education, there is limited data about how ethics and professionalism curricula are taught or assessed in ophthalmology residency programs. Objective This study aimed to determine how U.S. ophthalmology residency programs teach and assess ethics and professionalism and explore trainee preparedness in these areas. Methods Directors from accredited U.S. ophthalmology residency programs completed an online survey about components of programs' ethics and professionalism teaching curricula, strategies for assessing competence, and trainee preparedness in these areas. Results Directors from 55 of 116 programs (46%) responded. The most common ethics and professionalism topics taught were informed consent (38/49, 78%) and risk management and litigation (38/49, 78%), respectively; most programs assessed trainee competence via 360-degree global evaluation (36/48, 75%). While most (46/48, 95%) respondents reported that their trainees were well or very well prepared at the time of graduation, 15 of 48 (31%) had prohibited a trainee from graduating or required remediation prior to graduation due to unethical or unprofessional conduct. Nearly every program (37/48, 98%) thought that it was very important to dedicate curricular time to teaching ethics and professionalism. Overall, 16 of 48 respondents (33%) felt that the time spent teaching these topics was too little. Conclusion Ophthalmology residency program directors recognized the importance of an ethics and professionalism curriculum. However, there was marked variation in teaching and assessment methods. Additional work is necessary to identify optimal strategies for teaching and assessing competence in these areas. In addition, a substantial number of trainees were prohibited from graduating or required remediation due to ethics and professionalism issues, suggesting an impact of unethical and unprofessional behavior on resident attrition.

Ethical Dilemmas at the Beginning and End of Life: A Needs-Based, Experience-Informed, Small-Group, Case-Based Curriculum for Pediatric Residents.

Introduction: Pediatric residents are faced with ethical dilemmas in beginning- and end-of-life situations throughout their training. These situations are innately challenging, yet despite recommendations that residents receive training in ethics and end-of-life domains, they continue to report the need for additional training. To address these concerns, we developed an interactive and reflective palliative care and medical ethics curriculum including sessions focusing on ethical dilemmas at the beginning and end of life. Methods: This module includes a trio of case-based, small-group discussions on artificial nutrition and hydration, futility, and ethical considerations in neonatology. Content was developed based on a needs assessment, input from local experts, and previously published material. Trainees completed assessments of comfort and understanding before and after each session. Results: The module was attended and assessed by an average of 27 trainees per session, including residents and medical students. Knowledge of ethical considerations improved after individual sessions, with 86% of trainees reporting understanding ethical considerations involved in the decision to withdraw or withhold medically provided nutrition and hydration and 67% of trainees reporting understanding the use of the term futility. Trainee comfort in providing counseling or recommendations regarding specific ethical issues demonstrated a trend toward improvement but did not reach statistical significance. Discussion: We successfully implemented this innovative module, which increased trainees' comfort with end-of-life care and ethical conflicts. Future studies should focus on the trainees' ability to implement these skills in clinical practice.

When Specialty Care Is Unavailable to Rural Families.

We present the case of a 2-year-old boy with epidermolysis bullosa and supraglottic stenosis whose parents refuse an elective tracheostomy because of the significant care the tracheostomy would require. The patient's family lives in a rural area with few health care resources and his parents are already handling hours of daily skin care for his epidermolysis bullosa. In an attempt to convince the parents to pursue the intervention, the medical team recommends that the family move to an area with additional resources to assist in the child's care. The parents refuse to move, citing the many benefits their home environment provides for their son. The medical team calls an ethics consultation, questioning whether this decision constitutes medical neglect. This case raises important questions about medical decision-making in pediatrics. First, is a parent's refusal of a recommended medical intervention because it would require moving their family to a new environment a reasonable decision? Second, how broadly can parents define their child's best interest? Should only physical interests be included when making medical decisions? Is there a limit to what can be considered a relevant interest? Third, can parents only consider the interests of the individual child, or can they consider the interests of other members of the family? Finally, what is the threshold for overruling a parental decision? Is it whenever the parent's definition of a patient's best interest is different from the medical team's, or do other criteria have to be met?

Systematic Review of Typologies Used to Characterize Clinical Ethics Consultations.

INTRODUCTION: Classifying the ethical issues in clinical ethics consultations is important to clinical practice and scholarship. We conducted a systematic review to characterize the typologies used to analyze clinical ethics consultations. METHODS: We identified empirical studies of clinical ethics consultation that report types of ethical issues using PubMed. We screened these articles based on their titles and abstracts, and then by a review of their full text. We extracted study characteristics and typologies and coded the typologies. RESULTS: We reviewed 438 articles; 30 of the articles fulfilled our inclusion criteria. We identified 27 unique typologies. Each typology contained five to 47 categories (mean was 18). The most common categories were DNAR (do-not-attempt-resuscitation) orders (19 of the 27 typologies, or 70 percent), capacity (18 of the typologies, or 67 percent), withholding (18 of the typologies, or 67 percent), withdrawing (17 of the typologies, or 63 percent), and surrogate or proxy (16 of the typologies, or 59 percent). Only seven (26 percent) of the typologies contained all five of the most common categories. The typologies we used to characterize clinical ethics consultation exhibit significant heterogeneity and several conceptual limitations. A common typology is needed whose development may require multi-institutional collaboration and could be facilitated by professional organizations.