El horizonte de la atención médica en pediatría: ¿qué hacer en el caso de niños que se encuentran en situación de abandono, conflicto, daño o peligro, aunado a una enfermedad grave? / The horizon of medical attention in pediatrics: what to do in the case of children who are in abandonment, conflict, harm or danger situations in combination with a severe disease?

Resumen: Introducción: Las leyes refieren que los menores no tienen la capacidad para dar su consentimiento informado para su propia atención médica; sin embargo, hay condiciones especiales en las que se les permite determinar lo referente a su salud. Cuanto mayores sean las limitaciones de juicio y experiencia en los menores, menos peso se otorga a los valores y objetivos que expresan; cuanto más adversas sean las consecuencias, se deberá exigir un nivel más alto de autoridad para decidir en nombre del menor, dejando al Estado la capacidad de garantizar el bienestar del menor. Caso clínico: Niña de 12 años con diagnóstico de leucemia linfoblástica aguda LI, con antecedentes familiares y sociales precarios; evolución entorpecida por el desapego al tratamiento y sus condiciones insalubres y pobreza extrema. Ambos padres fallecieron al poco tiempo de iniciar su tratamiento, quedando ella al cuidado de su medio hermana mayor de edad. Se exponen la labor y el dilema ético del oncólogo tratante y del personal del Hospital Infantil de México Federico Gómez en la creación de redes de apoyo con el objetivo de priorizar el bienestar de la menor, sin dar lugar al quebrantamiento y la desintegración familiar, consiguiendo exitosamente su recuperación. Conclusiones: El caso fue sometido al Comité de Bioética Hospitalaria. Se formaron redes de apoyo interinstitucionales para intervenir en la dinámica familiar, resolviendo los requerimientos de la menor, y se consiguió con éxito superar la enfermedad.

Abstract: Background: Laws refer that minors do not have the capability to give informed consent for their own medical attention. However, there are special conditions in which they are allowed to decide about their health. The greater the judgement and experience limitations in minors, the less weight is given to the values and objectives they express. Also, the more adverse consequences might be, the higher the level of authority that is demanded to decide on behalf of the minor, thus granting the State the capability to guarantee the well-being of the minor. Case report: 12-year-old female patient with a diagnosis of acute lymphoblastic leukemia, with precarious social and family background; evolution of the disease obstructed by the disregard of the treatment due to her unsanitary and extreme poverty conditions. Both of her parents died soon after the start of the treatment and she was kept under the care of her half-sister of legal age. The work and the ethical dilemma of the pediatrician and the staff of Hospital Infantil de México Federico Gómez are exposed within the building of support networks with the objective of prioritizing the minor's well-being, without allowing family break-up or disintegration, thus succeeding in her recovery. Conclusions: The case was submitted to the Hospital Bioethics Committee. Inter-institutional support networks were built in order to improve dynamics of the family, thus solving the needs of the minor. Despite the misfortune of the situation, the disease was successfully overcome.

[The horizon of medical attention in pediatrics: what to do in the case of children who are in abandonment, conflict, harm or danger situations in combination with a severe disease?] / El horizonte de la atención médica en pediatría: ¿qué hacer en el caso de niños que se encuentran en situación de abandono, conflicto, daño o peligro, aunado a una enfermedad grave?

Background: Laws refer that minors do not have the capability to give informed consent for their own medical attention. However, there are special conditions in which they are allowed to decide about their health. The greater the judgement and experience limitations in minors, the less weight is given to the values and objectives they express. Also, the more adverse consequences might be, the higher the level of authority that is demanded to decide on behalf of the minor, thus granting the State the capability to guarantee the well-being of the minor. Case report: 12-year-old female patient with a diagnosis of acute lymphoblastic leukemia, with precarious social and family background; evolution of the disease obstructed by the disregard of the treatment due to her unsanitary and extreme poverty conditions. Both of her parents died soon after the start of the treatment and she was kept under the care of her half-sister of legal age. The work and the ethical dilemma of the pediatrician and the staff of Hospital Infantil de México Federico Gómez are exposed within the building of support -networks with the objective of prioritizing the minor's well-being, without allowing family break-up or disintegration, thus succeeding in her recovery. Conclusions: The case was submitted to the Hospital Bioethics Committee. Inter-institutional support networks were built in order to improve dynamics of the family, thus solving the needs of the minor. Despite the misfortune of the situation, the disease was successfully overcome.

Introducción: Las leyes refieren que los menores no tienen la capacidad para dar su consentimiento informado para su propia atención médica; sin embargo, hay condiciones especiales en las que se les permite determinar lo referente a su salud. Cuanto mayores sean las limitaciones de juicio y experiencia en los menores, menos peso se otorga a los valores y objetivos que expresan; cuanto más adversas sean las consecuencias, se deberá exigir un nivel más alto de autoridad para decidir en nombre del menor, dejando al Estado la capacidad de garantizar el bienestar del menor. Caso clínico: Niña de 12 años con diagnóstico de leucemia linfoblástica aguda LI, con antecedentes familiares y sociales precarios; evolución entorpecida por el desapego al tratamiento y sus condiciones insalubres y pobreza extrema. Ambos padres fallecieron al poco tiempo de iniciar su tratamiento, quedando ella al cuidado de su medio hermana mayor de edad. Se exponen la labor y el dilema ético del oncólogo tratante y del personal del Hospital Infantil de México Federico Gómez en la creación de redes de apoyo con el objetivo de priorizar el bienestar de la menor, sin dar lugar al quebrantamiento y la desintegración familiar, consiguiendo exitosamente su recuperación. Conclusiones: El caso fue sometido al Comité de Bioética Hospitalaria. Se formaron redes de apoyo interinstitucionales para intervenir en la dinámica familiar, resolviendo los requerimientos de la menor, y se consiguió con éxito superar la enfermedad.

[Criteria for good prescribing practice in children]. / Inhaltliche Kriterien für eine gute Verordnung bei Kindern.

Paediatric prescribing is complex. A whole range of aspects needs to be considered to achieve an efficacious and safe drug therapy for children. Legal requirements for prescribing are clearly insufficient for this purpose. Children are immature individuals under constant growth and development. Consequently, based on age and cognitive abilities of the child individual drugs and dosing regimens have to be chosen. Frequent off-label use and a lack of age-appropriate formulation worsen the situation. Additionally, not all dosage forms are similarly adequate in different age groups. Taste significantly influences patient adherence. Dose calculations based on body weight are prone to errors, putting a point on the wrong place or mixing up measuring units easily result in ten-fold dosing errors. Computer-based tools to enhance prescribing are promising but, however, not yet widely implemented in paediatrics because of missing evidence-based data sources and the hugely complex process. Communication between clinicians and pharmacists as well as with the patient remains very important.

Who are the PDCO?

In January 2007, the Paediatric Regulation entered into force and established the Paediatric Committee (PDCO) within the European Medicines Agency. The goal of the PDCO is to improve the health of the children of Europe by increasing high-quality research for medicinal products and promoting the development and authorization of such medicines at the EU level. A major function of the PDCO is to formulate and authorize Paediatric Investigation Plans and Paediatric Use Marketing Authorisations. The EU's Seventh Framework Programme for Research has facilitated the establishment of consortia whose ultimate goal is to answer important clinical questions involving medicines commonly used "off-label", in children. The benefits of these consortia include enhanced collaboration amongst paediatricians, scientists and small to medium enterprises whose ultimate goal is to obtain an authorization for a new indication or formulation for use in the paediatric population. It will be interesting in a number of years time to measure the success of this very important European initiative.

A pediatric diabetes toolbox for creating centres of reference.

INTRODUCTION: ISPAD guidelines recommend age appropriate diabetes education concepts for young patients and their families as well as tools for nutritional management, psychosocial assessment, and psychological advice but their implementation in Europe is presently unknown. METHODS: On the basis of a structured survey among the European SWEET members information on established tools and programs in national languages were analyzed using an extensive literature and desk search. These were differentiated according to five age-groups and five target groups (young people with diabetes, parents, and other close relations, carers in school and nursery, and healthcare professionals). RESULTS: Responses and original tools were received from 11 SWEET countries reflecting the European status in 2011. More or less structured information for parents, close relations, and carers in school or nursery are available in all 11 participating countries. However, only two countries followed the recommendations of having published a structured, curriculum lead, and evaluated program for different age-groups and carers. One of these was evaluated nationwide and funded by the respective National Health Care System after accreditation. In addition a huge variety of creative tools, e.g., booklets, leaflets, games, videos, and material for educating children of different age-groups and their parents are available - but most of them are not linked to a structured education program. CONCLUSIONS: Harmonizing and integrating these materials into quality assured structured holistic national education programs will be an important future task for the ongoing SWEET project. A comprehensive European diabetes educational toolbox is aimed to be published and continuously updated on the SWEET website.

The health law partnership: adding a lawyer to the health care team reduces system costs and improves provider satisfaction.

Addressing the legal issues of patients of low socioeconomic status can be useful in increasing organizational reimbursements, reducing costs and improving access to care. Medical-legal partnership is an addition to the health care armamentarium that directly addresses this goal. A medical-legal partnership is an interdisciplinary collaboration between a medical entity such as a hospital or clinic and a legal entity such as a law school or legal aid society that addresses barriers to access to care and limitations to well-being experienced by patients of low socioeconomic status. The Health Law Partnership is one such medical legal partnership that provides a holistic, interdisciplinary approach to health care. An evaluation of the legal and educational services provided by Health Law Partnership showed that Health Law Partnership secured otherwise unreimbursed Medicaid payments for services over a 4-year period from 2006 to 2010, increased physician satisfaction, and saved hospital employers approximately $10 000 in continuing education costs annually.

Pediatric use of complementary and alternative medicine: legal, ethical, and clinical issues in decision-making.

In this article we introduce a series of 8 case scenarios and commentaries and explore the complex legal, ethical, and clinical concerns that arise when pediatric patients and their parents or health care providers use or are interested in using complementary and alternative medicine (CAM). People around the world rely on CAM, so similar issues face clinicians in many countries. In law, few cases have dealt with CAM use. The few that have apply the same general legal principles used in cases that involved conventional care while taking into account considerations unique to CAM. In ethics, as with conventional care, the issues surrounding pediatric CAM use usually involve questions about who the appropriate decision-makers are, on what ethical principles should clinical decision-making rely, and what obligations arise on the part of physicians and other health care providers. Clinical decision-making is made more complex by the relatively limited research on the efficacy and safety of CAM compared with conventional medicine, especially in children, which requires clinicians to make decisions under conditions of uncertainty. The clinical scenarios presented focus on patients who represent a range of ages, clinical conditions, and settings. They act as anchors to explore particular CAM policy issues and illustrate the application of and shortcomings in existing guidance and intervention principles. Although the focus on a pediatric population adds another layer of complexity to the analysis, many of the concepts, issues, principles, and recommendations also apply to adults.

Concluding comments: maximizing good patient care and minimizing potential liability when considering complementary and alternative medicine.

Our goal for this supplemental issue of Pediatrics was to consider what practitioners, parents, patients, institutions, and policy-makers need to take into account to make good decisions about using complementary and alternative medicine (CAM) to treat children and to develop guidelines for appropriate use. We began by explaining underlying concepts and principles in ethical, legal, and clinical reasoning and then used case scenarios to explore how they apply and identify gaps that remain in practice and policy. In this concluding article, we review our major findings, summarize our recommendations, and suggest further research. We focus on several key areas: practitioner and patient/parent relationships; decision-making; dispute resolution; standards of practice; hospital/health facility policies; patient safety; education; and research. Ethical principles, standards, and rules applicable when making decisions about conventional care for children apply to decision-making about CAM as well. The same is true of legal reasoning. Although CAM use has seldom led to litigation, general legal principles relied on in cases involving conventional medical care provide the starting point for analysis. Similarly, with respect to clinical decision-making, clinicians are guided by clinical judgment and the best interests of their patient. Whether a therapy is CAM or conventional, clinicians must weigh the relative risks and benefits of therapeutic options and take into account their patient's values, beliefs, and preferences. Consequently, many of our observations apply to conventional and CAM care and to both adult and pediatric patients.

Preventive medicines: vaccination, prophylaxis of infectious diseases, disinfectants.

Immunizations belong to the most successful interventions in medicine. Like other drugs, vaccines undergo long periods of pre-clinical development, followed by careful clinical testing through study Phases I, II, and III before they receive licensure. A successful candidate vaccine will move on to be an investigational vaccine to undergo three phases of pre-licensure clinical trials in a stepwise fashion before it can be considered for approval, followed by an optional fourth phase of post-marketing assessment. The overall risk-benefit assessment of a candidate vaccine is very critical in making the licensure decision for regulatory authorities, supported by their scientific committees. It includes analyses of immunogenicity, efficacy, reactogenicity or tolerability, and safety of the vaccine. Public trust in vaccines is a key to the success of immunization programs worldwide. Maintaining this trust requires knowledge of the benefits and scientific understanding of real or perceived risks of immunizations. Under certain circumstances, pre- or post-exposure passive immunization can be achieved by administration of immunoglobulines. In terms of prevention of infectious diseases, disinfection can be applied to reduce the risk of transmission of pathogens from patient to patient, health-care workers to patients, patients to health-care workers, and objects or medical devices to patients.

Preclinical safety evaluations supporting pediatric drug development with biopharmaceuticals: strategy, challenges, current practices.

Evaluation of pharmaceutical agents in children is now conducted earlier in the drug development process. An important consideration for this pediatric use is how to assess and support its safety. This article is a collaborative effort of industry toxicologists to review strategies, challenges, and current practice regarding preclinical safety evaluations supporting pediatric drug development with biopharmaceuticals. Biopharmaceuticals include a diverse group of molecular, cell-based or gene therapeutics derived from biological sources or complex biotechnological processes. The principles of preclinical support of pediatric drug development for biopharmaceuticals are similar to those for small molecule pharmaceuticals and in general follow the same regulatory guidances outlined by the Food and Drug Administration and European Medicines Agency. However, many biopharmaceuticals are also inherently different, with limited species specificity or immunogenic potential which may impact the approach taken. This article discusses several key areas to aid in the support of pediatric clinical use, study design considerations for juvenile toxicity studies when they are needed, and current practices to support pediatric drug development based on surveys specifically targeting biopharmaceutical development.

The value of juvenile animal studies: a Japanese industry perspective.

Pharmaceuticals have been used on adults and children; however, they were previously investigated only by adult human clinical studies and adult animal nonclinical studies. The US FDA finalized the guidance of juvenile animal toxicity studies in 2006, and EMEA was finalized in 2008. At that point, juvenile animal toxicity studies were encouraged to investigate the safety of the pediatric population. In Japan, the awareness of the development of pediatric drugs is increasing, and many scientific meetings about juvenile animal studies are being held. A Japanese guideline for juvenile animal toxicity studies has been long awaited by many Japanese pharmaceutical companies because concrete directionality has not been available in Japan thus far. The Ministry of Health, Labour, and Welfare started to prepare the guideline for nonclinical safety studies in juvenile animals since October 2010. After completion of the Japanese guideline, guidelines would exist in the three regions: Japan, US, and Europe. Then, global development of pediatric pharmaceuticals would be accelerated effectively.

Nonclinical support of pediatric drug development in a global context: an industry perspective.

The earlier inclusion of children into clinical trials has challenged toxicologists to develop nonclinical strategies to support these trials early in the drug development process, and the routine practise of global development strategies (i.e., concomitant development and filing in multiple geographical regions) adds another complication. Ideally, one would like to develop a stagey that would meet regulatory requirements from all regions. This presentation illustrated the challenges faced in developing a strategy regarding the need to perform a toxicity study in juvenile animals and the design of any necessary study that will receive global regulatory agreement.

Juvenile animal studies and pediatric drug development retrospective review: use in regulatory decisions and labeling.

Juvenile animal toxicity studies are conducted to support applications for drugs intended for use in children. They are designed to address specific questions of potential toxicity in the growing animal or provide data about long-term safety effects of drugs that cannot be obtained from clinical trials. Decisions to conduct a juvenile animal study are based on existing data, such as a safety signal already identified in adult studies, or previous knowledge of the drug or chemical class for its potential to impair growth or developmental milestones. In 2006, the FDA issued an industry guidance in which considerations for determining when a juvenile animal study is warranted were outlined. A retrospective study was conducted covering years both before and after the issued guideline to examine the contribution of juvenile animal toxicity studies to the risk/benefit assessment of pediatric drugs at the FDA. The initial findings were presented as part of the May 2010 HESI workshop on the value of juvenile animal studies. The objective of the review was to better understand the value that the juvenile animal study contributes to regulatory decision making for pediatric drug development by looking at when the studies have been included in the product assessment; what, if any, impact the studies had on the regulatory decisions made; and whether the data were incorporated into the label. The data described below represent a first look at impact of the juvenile animal study since the pediatric legislation and the juvenile animal guidance were issued in the US.

Frontline ethical issues in pediatric clinical research: ethical and regulatory aspects of seven current bottlenecks in pediatric clinical research.

In the course of the past decades, considerable effort has been expended on the ethical guidance and legal regulation of pediatric clinical trials in Europe. Nonetheless, the conduct of clinical research in the population of minors continues to generate myriad ethical and regulatory issues. This paper explores seven bottlenecks in the ethical guidance and legal regulation that currently govern pediatric clinical research: (1) the integration of research in therapy, (2) the education of clinicians, (3) the empowerment of families, (4) the harmonization of protocol review, (5) the assessment non-clinical research objectives, (6) the control of placebo use, and (7) the provision of fair incentives for pediatric research conduct. For all of these issues, a clear view on the way forward is largely lacking, either because these issues have not been discussed in depth to date or because the existing debates have failed to generate a generally supported consensus.

Child consent and the law: an insight and discussion into the law relating to consent and competence.

BACKGROUND: The law governing consent for children is not very clear. A child can consent to treatment but usually in practice is unable to refuse it. Even if both the child and parents refuse treatment, courts are reluctant to accept this, particularly if it is in the best interest of the child. DISCUSSION: In order to consent to treatment, a child must be competent enough to do so, and this competence is judged usually by a doctor. Children can even consent to contraceptives and abortion if 'competent' to do so. This concept perfectly lacks moral, ethical and emotional competence, and judgement of competence is carried out usually purely scientifically by pure science-orientated objective professionals like doctors. A broad discussion about the issues of children refusing treatment is conducted from the legal, ethical and philosophical point of view. Life-saving treatment and various other cases are also discussed. CONCLUSION: There is no right answer to the question. A more holistic approach is needed, and not only doctors but also sociologists, care specialists and even clergymen should be asked to judge competence in a multidisciplinary environment, particularly for contraceptives and abortion. This multidisciplinary working can be extended to other areas in medical law as well particularly in light of changes in medicine. Experience in life should be valued in a decision-making environment for judging competence. The law in relation to child consent is unclear and requires changes in order to clarify what is perceived as the child's best interest.

Legal and ethical issues relating to use of complementary therapies in pediatric hematology/oncology.

Pediatricians increasingly are asked to advise pediatric patients and their families concerning integration into conventional care (including hematology and oncology) of complementary and alternative medical (CAM) therapies such as chiropractic, massage therapy, and herbal medicine. Inclusion of CAM therapies in pediatric oncology and hematology--as in any medical subspecialty--is not itself "unethical," clinically inadvisable, or legally risky; the danger comes from over-reliance on one or more CAM therapies (particularly those with evidence of danger and/or paltry evidence of success) to the exclusion of conventional care that is curative and imminently necessary. Pediatricians can help address potential malpractice liability issues by evaluating the level of clinical risk, engaging the patient in shared decision making and documenting this in the medical record, continuing to monitor conventionally, and being prepared to intervene conventionally when medically required.

Complementary therapies in pediatrics: a legal perspective.

Increasing use of complementary and alternative medicine (CAM) therapies such as chiropractic, massage therapy, and herbal medicine, raises questions about the clinically appropriate use of CAM in pediatrics. Nonjudicious use of CAM therapies may cause either direct harm or, by creating an unwarranted financial and emotional burden, indirect harm. When advising patients concerning CAM therapies, pediatricians face 2 major legal risks: medical malpractice and professional discipline. Pediatricians can incorporate these considerations into advising and clinical decision-making about CAM therapies to address the best interest of the pediatric patient while helping to manage potential liability risk. This article provides a suggested framework, including asking the following questions: (1) Do parents elect to abandon effective care when the child's condition is serious or life-threatening? (2) Will use of the CAM therapy otherwise divert the child from imminently necessary conventional treatment? (3) Are the CAM therapies selected known to be unsafe and/or ineffective? (4) Have the proper parties consented to the use of the CAM therapy? (5) Is the risk-benefit ratio of the proposed CAM therapy acceptable to a reasonable, similarly situated clinician, and does the therapy have at least minority acceptance or support in the medical literature? Such an approach ideally can help guide the pediatrician toward clinical conduct that is clinically responsible, ethically appropriate, and legally defensible.