European Union Pediatric Legislation Jeopardizes Worldwide, Timely Future Advances in the Care of Children With Cancer

Rose, Klaus

doi:10.1016/j.clinthera.2014.01.009

Cited by 34 publications

(38 citation statements)

References 18 publications

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“…because drugs were increasingly being studied in children of younger ages and because the formulation proposals had prompted discussion with companies on many issues (72). However, the value of some paediatric trials is disputed by some stakeholders arguing that different companies were obliged to conduct similar studies in small patient populations (62). Moreover, there are uncertainties on the effectiveness of the Regulation to reduce off-label and unlicensed paediatric prescription rates as many essential and common drugs have been licensed before the Regulation entered into force.…”

Section: Paediatric Regulation: Progress and Future Perspectivesmentioning

confidence: 99%

Paediatric Drug Development and Formulation Design—a European Perspective

Riet‐Nales

Kozarewicz²,

Aylward

et al. 2016

AAPS PharmSciTech

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Abstract.The availability of licensed paediatric drugs is lagging behind those for adults, and there is a lack of safe formulations in suitable doses that children are able and willing to take. As a consequence, children are commonly treated with off-label or unlicensed drugs. As off-label and unlicensed drug use are associated with a greater risk for harm than on-label drug use, a range of global initiatives have been developed to realize Bbetter^medicines for children. This review describes the challenges and achievements of the European Union to realize this goal, with a focus on paediatric drug development and formulation design. In 2007, a European Paediatric Regulation was installed enforcing companies to consider children in the early development of drugs with a new drug substance, for a new indication or with a new route of administration. The Regulation, e.g. requires companies to develop a paediatric investigation plan discussing the proposed clinical trials in children of different ages and the formulations for future marketing. Since 2013, the pharmaceutical design of any newly marketed paediatric drug should comply with the BGuideline on the Pharmaceutical Development of Medicines for Paediatric Use.^Companies should, e.g. justify the route of administration, dosage form, formulation characteristics, safety of excipients, dosing frequency, container closure system, administration device, patient acceptability and user information. In this review, the guideline's key aspects are discussed with a focus on novel formulations such as mini-tablets and orodispersible films, excipients with a potential risk for harm such as azo dyes and adequate user instructions.

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Section: Paediatric Regulation: Progress and Future Perspectivesmentioning

confidence: 99%

Paediatric Drug Development and Formulation Design—a European Perspective

Riet‐Nales

Kozarewicz²,

Aylward

et al. 2016

AAPS PharmSciTech

View full text Add to dashboard Cite

show abstract

“…All of these guidance documents suggest that studies in juvenile animals should be conducted infrequently, and a recent review suggests that the percent of pediatric investigation plans (PIP) supported by a juvenile toxicity study has remained fairly stable (Hurtt and Engel, 2015). However, requests for and conduct of these studies is increasing with the increasing number of pediatric programs overall, and, at present, there is no consistent message regarding the value of the data generated in these studies for improving the design, conduct and interpretation of safety data from subsequent clinical studies in children (Rose, 2011, Rose, 2014, Leconte et al , 2011, Anderson et al , 2009, Bailey and Marien, 2009, Bailey and Marien, 2011). In response to this, the ICH has endorsed the development of a new harmonized guideline for nonclinical safety assessment to support pediatric drug development (ICH S11).…”

Section: Pathology Endpoints In Nonclinical Studies Supporting Pedmentioning

confidence: 99%

Scientific and Regulatory Policy Committee Points to Consider Review

Halpern¹,

Ameri

Bowman

et al. 2016

Toxicol Pathol

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Standard components of nonclinical toxicity testing for novel pharmaceuticals include clinical and anatomic pathology, as well as separate evaluation of effects on reproduction and development to inform clinical development and labeling. General study designs in regulatory guidances do not specifically mandate use of pathology or reproductive endpoints across all study types; thus, inclusion and use of these endpoints are variable. The Scientific and Regulatory Policy Committee (SRPC) of the Society of Toxicologic Pathology (STP) formed a Working Group to assess the current guidelines and practices on the use of reproductive, anatomic pathology and clinical pathology endpoints in General, Reproductive, and Developmental toxicology studies. The Working Group constructed a survey sent to pathologists and reproductive toxicologists, and responses from participating organizations were collected through the STP for evaluation by the Working Group. The regulatory context, relevant survey results, and collective experience of the Working Group are discussed and provide the basis of each assessment by study type. Overall, the current practice of including specific endpoints on a case-by-case basis is considered appropriate. Points to consider are summarized for inclusion of reproductive endpoints in general toxicity studies, and for the informed use of pathology endpoints in reproductive and developmental toxicity studies.

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“…Es bedarf einer sorgfältigen Abwägung des Benefit-Risk-Verhältnisses. Mit der Einführung moderner Arzneimittellabels begann der Hinweis, dass das jeweilige Medikament nicht für Kinder erprobt und zu- [7,8]. Detaillierte Analysen wurden zu Melanom [7], Leukämie [6], zystischer Fibrose …”

Section: Zusammenfassungunclassified

Medikamente und ihre Entwicklung für Kinder

2016

Schweiz Ärzteztg

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European Union Pediatric Legislation Jeopardizes Worldwide, Timely Future Advances in the Care of Children With Cancer

Cited by 34 publications

References 18 publications

Paediatric Drug Development and Formulation Design—a European Perspective

Paediatric Drug Development and Formulation Design—a European Perspective

Scientific and Regulatory Policy Committee Points to Consider Review

Medikamente und ihre Entwicklung für Kinder

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