More medicines for children: impact of the EU paediatric regulation

Nordenmalm, Sofia; Tomasi, Paolo; Pallidis, Chrissi

doi:10.1136/archdischild-2017-313309

Cited by 30 publications

(28 citation statements)

References 4 publications

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“…Table 1 summarises the main differences in definition, regulatory approach and (post)marketing. Particularly for children, this differs significantly from paediatric medicine development 8–11…”

Section: Introductionmentioning

confidence: 99%

Medical devices that look like medicines: safety and regulatory concerns for children in Europe

Huijghebaert

Bruyne²,

Allegaert³

et al. 2019

Arch Dis Child

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IntroductionMedical devices (MedDevs) and medicines are assessed (and monitored) differently before and after launch. There are products for repeated oral ingestion that are marketed in the European Union as MedDevs.Objectives and methodsTo illustrate the consequences of these differences in assessment, we compared the leaflet information of three MedDevs with the standards for medicines and with published evidence at launch. As examples, gelatin tannate (GT), its combination with tyndalised probiotics (TP) (GTTP) for diarrhoea and a gel containing hyaluronic acid (HA)/chondroitin sulfate (CS)/poloxamer (Pol407) (HACSPol) for gastro-oesophageal reflux disease were examined.ResultsApplying standards for medicines, product composition is insufficiently defined in the MedDev leaflet (eg, plant origin, polymerisation grade, dose and ratio of the relevant constituents). As no age limit is mentioned in the leaflets, all 3 products allow use in children from birth onwards, although published clinical documentation in children was poor (GT) or lacking (GTTP and HACSPol). MedDev leaflets do not mention adverse events (AEs), while literature search suggests safety concerns such as tannic acid (TA) cytotoxicity, potentially more diarrhoea/AEs with TP, use of doses higher than established safe (TA and HA) and lack of chronic toxicity studies for oral Pol407. None refers to interactions with medicines, although some ingredients may affect medicine absorption.ConclusionAlthough these MedDevs require repeated oral intake as do medicines, their assessment and monitoring differ significantly from the standards for medicines. Compared with medicines, MedDevs for repeated oral use are poorly labelled and rely on very limited clinical information at market release.

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Section: Introductionmentioning

confidence: 99%

Medical devices that look like medicines: safety and regulatory concerns for children in Europe

Huijghebaert

Bruyne²,

Allegaert³

et al. 2019

Arch Dis Child

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“…Lack of clinical studies are not the only reason for a medicine being off‐label. It is a quite common situation that pharmaceutical companies research on medicines for children are not completed, or fail to put the product on the market, maybe due to lack of profit, leaving paediatricians to have no other choice but use these in off‐label manner . The current use of unlicensed and insufficiently studied medicines in neonates, children and adolescents, increases the risk of facing unexpected adverse drug reactions related to administration and dosage, but also for the administration of too low exposures with insufficient efficacy .…”

Section: Nordic Network For Drug Development In Childrenmentioning

confidence: 99%

Tardy development of safe medicines for children: a Nordic network offers new platform to reduce this inequity

et al. 2019

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“…Informed consent Parents and/or legal guardians of children and adult patients participating in these clinical trials gave their informed consent prior to their inclusion in the studies reported in this manuscript. Hospital Clínico Universitario, Valencia, Spain 10 Hospital Universitario Marqués de Valdecilla, Santander, Spain 11 Hospital Universitario Torrecárdenas, Almeria, Spain 12 Hospital Universitario Miguel Servet, Saragossa, Spain 13 Hospital Universitario Nuestra Señora de la Candelaria, Santa Cruz de Tenerife, Spain 14 Hospital Alvaro Cunqueiro, Vigo, Spain 15 Hospital Universitario Carlos Haya, Malaga, Spain 16 Hospital Universitario Virgen de la Macarena, Seville, Spain…”

Section: Compliance With Ethical Standardsmentioning

confidence: 99%

ECLIM-SEHOP, a new platform to set up and develop international academic clinical trials for childhood cancer and blood disorders in Spain

et al. 2019

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Introduction Cancer and blood disorders in children are rare. The progressive improvement in survival over the last decades largely relies on the development of international academic clinical trials that gather the sufficient number of patients globally to elaborate solid conclusions and drive changes in clinical practice. The participation of Spain into large international academic trials has traditionally lagged behind of other European countries, mainly due to the burden of administrative tasks to open new studies, lack of financial support and limited research infrastructure in our hospitals. Methods The objective of ECLIM-SEHOP platform (Ensayos Clínicos Internacionales Multicéntricos-SEHOP) is to overcome these difficulties and position Spain among the European countries leading the advances in cancer and blood disorders, facilitate the access of our patients to novel diagnostic and therapeutic approaches and, most importantly, continue to improve survival and reducing long-term sequelae. ECLIM-SEHOP provides to the Spanish clinical investigators with the necessary infrastructural support to open and implement academic clinical trials and registries. Results In less than 3 years from its inception, the platform has provided support to 20 clinical trials and 8 observational studies, including 8 trials and 4 observational studies where the platform performs all trial-related tasks (integral support: trial setup, monitoring, etc.) with more than 150 patients recruited since 2017 to these studies. In this manuscript, we provide baseline metrics for academic clinical trial performance that permit future comparisons. Conclusions ECLIM-SEHOP facilitates Spanish children and adolescents diagnosed with cancer and blood disorders to access state-of-the-art diagnostic and therapeutic strategies.

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More medicines for children: impact of the EU paediatric regulation

Abstract: The entry into force of the Paediatric Regulation has had a positive impact on paediatric drug development with more medicines available for children in the EU and substantially more information available for clinicians on paediatric use in the product information.

Cited by 30 publications

References 4 publications

Medical devices that look like medicines: safety and regulatory concerns for children in Europe

Medical devices that look like medicines: safety and regulatory concerns for children in Europe

Tardy development of safe medicines for children: a Nordic network offers new platform to reduce this inequity

ECLIM-SEHOP, a new platform to set up and develop international academic clinical trials for childhood cancer and blood disorders in Spain

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