Enabling Development of Paediatric Medicines in Europe: 10 Years of the EU Paediatric Regulation

Tomasi, Paolo; Egger, Gunter F.; Pallidis, Chrissi; Saint‐Raymond, Agnès

doi:10.1007/s40272-017-0261-1

Cited by 68 publications

(64 citation statements)

References 13 publications

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“…Despite the great efforts made in the last decade to authorize medicines for pediatric use [ 11 ], the treatment of inflammatory disorders in children largely relies on off-label use of drugs developed for adults [ 12 ]. This is especially true when dealing with severe rare diseases [ 13 ].…”

Section: Discussionmentioning

confidence: 99%

Biological and Clinical Changes in a Pediatric Series Treated with Off-Label JAK Inhibitors

Tesser

Pastore

et al. 2020

IJMS

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Off-label use of medications is still a common practice in pediatric rheumatology. JAK inhibitors are authorized in adults in the treatment of rheumatoid arthritis, psoriatic arthritis and ulcerative colitis. Although their use is not authorized yet in children, JAK inhibitors, based on their mechanism of action and on clinical experiences in small series, have been suggested to be useful in the treatment of pediatric interferon-mediated inflammation. Accordingly, an increased interferon score may help to identify those patients who might benefit of JAK inhibitors. We describe the clinical experience with JAK inhibitors in seven children affected with severe inflammatory conditions and we discuss the correlation between clinical features and transcriptomic data. Clinical improvements were recorded in all cases. A reduction of interferon signaling was recorded in three out of seven subjects at last follow-up, irrespectively from clinical improvements. Other signal pathways with significant differences between patients and controls included upregulation of DNA repair pathway and downregulation of extracellular collagen homeostasis. Two patients developed drug-related adverse events, which were considered serious in one case. In conclusion, JAK inhibitors may offer a valuable option for children with severe interferon-mediated inflammatory disorders reducing the interferon score as well as influencing other signal pathways that deserve future studies.

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Section: Discussionmentioning

confidence: 99%

Biological and Clinical Changes in a Pediatric Series Treated with Off-Label JAK Inhibitors

Tesser

Pastore

et al. 2020

IJMS

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“…However, the focus in this brochure is the authorization of drugs, not clinical care. The EMA 10-year report on EU pediatric regulation [ 46 ] in comparison to its recent publication by EMA employees [ 47 ] demonstrates their true motivation.…”

Section: Discussionmentioning

confidence: 99%

Pediatric Melanoma and Drug Development

Rose¹,

Grant‐Kels

2018

Children

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Importance—Pediatric melanoma occurs, albeit rarely. Should patients be treated by today’s medical standards, or be subjected to medically unnecessary clinical studies? Observations—We identified international, industry-sponsored pediatric melanoma studies triggered by regulatory demands in and further pediatric melanoma studies demanded by European Union pediatric investigation plans. We retrieved related regulatory documents from the internet. We analyzed these studies for rationale and medical beneficence on the basis of physiology, pediatric clinical pharmacology and rationale. Regulatory authorities define children by chronological age, not physiologically. Newborns’ organs are immature but they develop and mature rapidly. Separate proof of efficacy in underage patients is justified formally/regulatorily but lacks medical sense. Children—especially post-puberty—and adults vis-a-vis medications are physiologically very similar. Two adolescent melanoma studies were terminated in 2016 because of waning recruitment, while five studies in pediatric melanoma and other solid tumors, triggered by European Union pediatric investigation plans, continue recruiting worldwide. Conclusions and Relevance—Regulatory-demanded pediatric melanoma studies are medically superfluous. Melanoma patients of all ages should be treated with effective combination treatment. Babies need special attention. Children need dose-finding and pharmacokinetic studies but adolescents metabolize and respond to drugs similarly to adults. Institutional Review Boards/ethics committees should suspend ongoing questionable pediatric melanoma studies and reject newly submitted questionable studies.

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“…Nowadays, formulation research and development in the pediatric area remains essential and is required [1,2]. Governments allocate considerable efforts to promote the availability of age-appropriate, safe, and effective pediatric medicines by regulatory incentives [3,4]. Most drugs are often not appropriate for pediatrics due to the drug dosage form (tablets or capsules) and strengths.…”

Section: Introductionmentioning

confidence: 99%

Availability of Authorizations from EMA and FDA for Age-Appropriate Medicines Contained in the WHO Essential Medicines List for Children 2019

delMoral-Sanchez,

Gonzalez-Alvarez,

Gonzalez-Alvarez

et al. 2020

Pharmaceutics

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Lack of age-appropriate commercially drug products availability is a common problem in pediatric therapeutics; this population needs improved and safer drug delivery. In addition, biopharmaceutic aspects, dosage requirements, and swallowing abilities demand pediatric forms different to adult formulations. The objective of this study was to evaluate the authorization availability from United States Food and Drug Administration (FDA) and European Medicines Agency (EMA) of oral essential medicines for children and analyze its age-appropriateness for oral administration in children. All oral drugs from 7th List of Essential Medicines for Children by World Health Organization (WHO) were selected. Availability of commercial drug products was collected from OrangeBook, Spanish drug product catalogue, British electronic Medicines Compendium, and the International Vademecum. Tablets, effervescent tablets, and capsules were considered as not age-appropriate forms. Liquid forms, powder for oral suspension, mini tablets, granules, and soluble films were considered as age-appropriate forms due to their flexibility. More than 80% of the studied drugs possess a commercial authorization in oral forms in both EMA and FDA. Nevertheless, around 50% of these formulations are not age-appropriate for most pediatric groups. This study shows the lack of age-appropriate medicines for children. More efforts are needed to improve development and approval of pediatric medicines.

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Enabling Development of Paediatric Medicines in Europe: 10 Years of the EU Paediatric Regulation

Cited by 68 publications

References 13 publications

Biological and Clinical Changes in a Pediatric Series Treated with Off-Label JAK Inhibitors

Biological and Clinical Changes in a Pediatric Series Treated with Off-Label JAK Inhibitors

Pediatric Melanoma and Drug Development

Availability of Authorizations from EMA and FDA for Age-Appropriate Medicines Contained in the WHO Essential Medicines List for Children 2019

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