Ethical and Social Values for Paediatric Health Technology Assessment and Drug Policy.

BACKGROUND: Public policy approaches to funding paediatric medicines in advanced health systems remain understudied. In particular, the ethical and social values dimensions of health technology assessment (HTA) and drug coverage decisions for children have received almost no attention in research or policy. METHODS: To elicit and understand the social values that influence decision-making for public funding of paediatric drugs, we undertook a series of in-depth, semi-structured interviews with a stratified purposive sample (n = 22) of stakeholders involved with or affected by drug funding decisions for children at the provincial (Ontario) and national levels in Canada. Constructivist grounded theory methodology guided data collection and thematic analysis. RESULTS: Our study provides empirical evidence about the unique ethical and social values dimensions of HTA for children, and describes a novel social values typology for paediatric drug policy decision-making. Three principal categories of values emerged from stakeholder reflections on HTA and drug policy-making for children: procedural values, structural values, and sociocultural values. Key findings include the importance of attention to the procedural legitimacy of HTA for children, with emphasis on the inclusion of child health voices in processes of technology appraisal and policy uptake; a role for HTA institutions to consider the equity impacts of technologies, both in setting review priorities and in assessing the value of technologies for public coverage; and the potential benefits of a distinct national framework to guide drug policy for children. CONCLUSION: Current approaches to HTA are not well designed for the realities of child health and illness, nor the societal priorities regarding children that our study identified. This research generates new knowledge to inform decision-making on paediatric drugs by HTA institutions and government payers in Canada and other publicly-funded health systems, through insights into the relevant social values for child drug funding decisions from varied stakeholder groups.

"The Hopkins Mongol Case": The Dawn of the Bioethics Movement.

One of the earliest controversies in the modern history of bioethics was known at the time as "the Hopkins Mongol case," involving an infant with Trisomy 21 and duodenal atresia whose parents declined to consent to surgery. Fluids and feeding were withheld, and the infant died of dehydration after 15 days. The child's short life had a profound impact on the author's career and that of several others and ultimately led to changes in the care of children and adults with disabilities and the way difficult end-of-life decisions are made in US hospitals today. It also contributed to the growth of the modern bioethics movement and scholarship focused on pediatric bioethics issues.

The Ethics of Creating a Resource Allocation Strategy During the COVID-19 Pandemic.

The coronavirus disease 2019 pandemic has affected nearly every aspect of medicine and raises numerous moral dilemmas for clinicians. Foremost of these quandaries is how to delineate and implement crisis standards of care and, specifically, how to consider how health care resources should be distributed in times of shortage. We review basic principles of disaster planning and resource stewardship with ethical relevance for this and future public health crises, explore the role of illness severity scoring systems and their limitations and potential contribution to health disparities, and consider the role for exceptionally resource-intensive interventions. We also review the philosophical and practical underpinnings of crisis standards of care and describe historical approaches to scarce resource allocation to offer analysis and guidance for pediatric clinicians. Particular attention is given to the impact on children of this endeavor. Although few children have required hospitalization for symptomatic infection, children nonetheless have the potential to be profoundly affected by the strain on the health care system imposed by the pandemic and should be considered prospectively in resource allocation frameworks.

Innovative approaches to investigator-initiated, multicentre paediatric clinical trials in Canada.

Data from clinical trials are needed to guide the safe and effective use of medicines in children. Clinical trials are challenging to design and implement in all populations, and children present additional considerations. Several regions including the UK, USA and Europe have established clinical trial infrastructure to capitalise on expertise and promote clinical trials enrolling children. Our objective is to describe the partnerships and operational considerations for the development of paediatric clinical trials infrastructure in Canada. We describe the design and conduct of four emergency room paediatric trials, with four separate sponsors, across four provinces in parallel. Operations discussed include multisite contract development, centralised risk-based data monitoring, ethical review and patient engagement. We conclude with lessons learnt, additional challenges and potential solutions to facilitate drug development for children in Canada.

A Case Study in Cross-cultural Health Care and Ethics: Who Decides What Is in the Child's "Best Interest"?

Health care in the United States is increasingly delivered in cross-cultural contexts. Empathy, mutual regard, respect, and compassionate communication are necessary to achieve the highest standard of care for each individual. Moral and ethical perspectives on life and death, health, and health care are not universal but rather have their origins within culture and societal norms. In a cross-cultural context, "the right decision" may be seen differently depending on an individual's cultural background, discipline, and type of education. This pediatric case study is intended to stimulate conversation on the need for culturally sensitive health care decision making and the shortcomings of a "one-size-fits-all" approach to bioethics in our increasingly interconnected world.

The ethics of concurrent care for children: A social justice perspective.

Recent estimates indicate that over 40,000 children die annually in the United States and a majority have life-limiting conditions. Children at end of life require extensive healthcare resources, including multiple hospital readmissions and emergency room visits. Yet, many children still suffer from symptoms at end of life-including fatigue, pain, dyspnea, and anxiety-with less than 10% of these children utilizing hospice care services. A critical barrier to pediatric hospice use was the original federal regulations associated with the hospice care that required a diagnosis of 6 months to live and the discontinuation of all curative treatments. The Concurrent Care Provision of the United States' Affordable Care Act eliminated the need to forgo curative therapies in order to enroll in hospice for children in Medicaid or Children's Health Insurance Program. Concurrent care for children can help mitigate the tension families experience in choosing between essential forms of care, as well as contribute to improved end-of-life outcomes for the child and possibly bereavement outcomes for the family. Understanding concurrent care for children from a social justice perspective has important advocacy and research implications for hospice and palliative care clinicians providing care for children and their families. We apply Powers and Faden's theory of social justice "as the moral foundation of public health and health policy" to the provision of concurrent care to children near end of life and families in the United States. The goals of applying this theory are to explore additional insights and perspectives into concurrent care policy may provide and to assess the usefulness of this theory when applied to end-of-life health policy. We argue that concurrent care policy is socially just since it has potential to promote well-being in vulnerable children and families and can limit the inequity children at end-of-life experience in access to high-quality hospice care.

Two's company, is three a crowd? Ethical cognition in decision making and the role of industry third parties in pediatric diabetes care.

Families of children with diabetes increasingly obtain health information from a variety of sources. Doctor-patient relationships have accordingly become more fluid and dynamic with input from other parties. These outside parties include representatives from the diabetes health care industry-industry third parties (ITPs). This review is an exploration of the ethical principles and cognitive processes involved when doctors and patients negotiate around health care practices and the role of ITPs in that dialogue. Ethical principles of conflicts of interest, beneficence (act in the best interests of the patient), non-maleficence (act so as to do no harm) and justice (act so as to allocate resources fairly or justly) are relevant considerations. Reflexive and analytic thinking and various cognitive biases also play a significant part in clinical decision making. A complex case example is analyzed to highlight a process of ethical cognition in decision making to ensure high-value care and optimal patient outcomes.

Personalised medicine in paediatric oncology: Ethical practice outside the clinical trial framework?

Modern paediatric oncology practice has rapidly evolved from a fatal condition at diagnosis in the 1940s to modern survival rates of over 80%. With the advent of the 'omics' era and modern diagnostics platforms, we can now determine many of the molecular driving mechanisms of malignancy. Current molecular diagnostics trials PRISM and AIM/MNP, open in Australia, allow accurate diagnosis and determination of the molecular drivers of many cancers leading to new targeted opportunities for treatment. Unfortunately, clinical trial support, development and drug access for children has lagged behind. This is leaving clinicians and their institutions with increasingly difficult medical and ethical decisions, further complicating an already demanding profession.

Targeted Dosing as a Precision Health Approach to Pharmacotherapy in Children with Inflammatory Bowel Disease.

As clinicians have begun to provide targeted pharmacotherapy for children with inflammatory bowel disease (IBD), several ethical challenges have arisen. In this paper, we review 3 challenges related to applying a precision health approach to pediatric IBD populations: selection of a disease monitoring method, pharmacotherapy optimization, and economic considerations in clinical decision making.

Assessment of a shortened informed consent form for pediatric research: a pilot study.

BACKGROUND: Inherent to clinical research is the informed consent process, with the informed consent form (ICF), a key component of human participant protections. We wished to examine whether a shortened and simplified ICF, accompanied by an appendix, improved participant understanding of a study compared with a conventional ICF. METHODS: A shortened ICF was developed from an existing conventional ICF for a neonatal study. Either the shortened or conventional ICF was randomly distributed to members of two parental advocacy groups. Participants answered survey questions about the form they received. RESULTS: Thirty-one out of forty-one (76%) parents in the shortened ICF and 28/41 (68%) in the conventional ICF group responded. Significantly more parents in the shortened ICF group found their form "short and to the point". Although they also stated that the shortened ICF did not provide enough information, there were no significant differences between groups measuring the understanding of key study components. CONCLUSION: A shortened ICF did not impact the understanding of the clinical trial. It will be important to compare the shortened and conventional forms in actual clinical trials.

Developing child autonomy in pediatric healthcare: towards an ethical model. / Desarrollo de la autonomía del niño en la atención pediátrica: hacia un modelo ético.

The changes initiated by the new National Civil and Commercial Code in Argentina underline the pediatric task to empower children's and adolescents' developing autonomy. In this paper, we have framed a model describing autonomy in child healthcare. We carried out a literature review focusing on i) the concept of autonomy referring to the absolute value of the autonomous individual, and ii) the age-driven process of competent decisionmaking development. We summarized our findings developing a conceptual model that includes the child, the pediatrician and the parents. The pediatricianchild relationship is based on different forms of guidance and cooperation, resulting in varying levels of activity and passivity. Parental authority influences the extent of autonomy, based on the level of respect of the child's moral equality. Contextual, existential, conceptual, and socialethical conditions shall be considered when applying the model to facilitate dialogue between pediatricians, children, parents and other actors.

Los cambios del nuevo Código Civil y Comercial de la Argentina impulsa el desarrollo de la autonomía en niños y adolescentes. En este trabajo, estructuramos un modelo que describe la autonomía en la atención de la salud infantil. Hicimos un análisis bibliográfico enfocado en I) concepto de autonomía relacionado con el valor absoluto del individuo autónomo y II) el proceso de desarrollo de toma idónea de decisiones en función de la edad. Resumimos nuestros hallazgos mediante el desarrollo de un modelo conceptual en el niño, el pediatra y los padres. La relación pediatraniño se fundamenta en formas de orientación y cooperación con diversos niveles de actividad y pasividad. La autoridad de los padres influye sobre el grado de autonomía en función del nivel de respeto e igualdad moral del niño. Al aplicar el modelo para facilitar el diálogo entre pediatras, niños, padres y otros, se considerarán las circunstancias contextuales, existenciales, conceptuales y socio-éticas.

Ethics in Pediatric Endocrinology: Curriculum for Fellows and Faculty.

Introduction: In clinical practice, ethical dilemmas are frequently faced by pediatric endocrinologists. This initiative's objectives were to (a) determine if endocrine fellows and faculty perceived that an effective ethics curriculum existed and (b) evaluate whether case-based modules would be an effective tool for ethics education. Methods: Participation was sought from eight large pediatric endocrine programs (home programs and affiliates of the Pediatric Endocrine Society's Ethics Committee members) after the distribution of eight case-based modules (geared mainly to fellows) and pre- and postsurveys. Questions examining self-reported knowledge (K) of the ethical pillars (beneficence, nonmaleficence, autonomy, and justice), attitudes (A) towards these, and the individual's likelihood of utilizing them in clinical practice (P), in addition to the need for/benefit of this curriculum, were assessed using a 5-point Likert scale. Results: Six out of eight programs participated, with surveys completed by fellows (n = 29), faculty (n = 7), and advanced practitioners (n = 3). Of the respondents, only 20.3% believed an effective ethics curriculum was already in place. After module completion, KAP scores improved, with the greatest improvement seen in knowledge scores. Additionally, 94.9% of respondents strongly agreed (n = 26) or agreed (n = 11) that the curriculum would be a valuable addition to fellowship training. All faculty believed that the curriculum was helpful in imparting ethical principles of clinical practice. Discussion: The findings suggest that this curriculum would be useful in knowledge advancement of ethical principles and could fulfill a long-standing need to provide clinical ethics education for faculty and fellows.

What Do Clinicians Caring for Children Need to Know about Pediatric Medical Traumatic Stress and the Ethics of Trauma-Informed Approaches?

Medical experiences can be frightening and traumatic for children. Ill and injured children can experience pediatric medical traumatic stress-psychological and physiological distress responses related to their medical event and subsequent medical treatment experiences-which can lead to symptoms of posttraumatic stress disorder (PTSD) and suboptimal health outcomes. Trauma-informed care provides a framework for acknowledging, addressing, and mitigating the risks of psychological trauma associated with medical treatment experiences and is congruent with the ethical principles of respect for autonomy, beneficence, nonmaleficence, and justice. Health care systems and professionals are encouraged to apply the principles of trauma-informed care to address the effects of pediatric medical traumatic stress.