Clinical trials in children

Abstract

Safety and efficacy data on many medicines used in children are surprisingly scarce. As a result children are sometimes given ineffective medicines or medicines with unknown harmful side effects. Better and more relevant clinical trials in children are needed to increase our knowledge of the effects of medicines and to prevent the delayed or non-use of beneficial therapies. Clinical trials provide reliable evidence of treatment effects by rigorous controlled testing of interventions on human subjects. Paediatric trials are more challenging to conduct than trials in adults because of the paucity of funding, uniqueness of children and particular ethical concerns. Although current regulations and initiatives are improving the scope, quantity and quality of trials in children, there are still deficiencies that need to be addressed to accelerate radically equitable access to evidence-based therapies in children.

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“…Consenting in children remains the biggest dilemma faced by all stakeholders of clinical research (11). Joseph et al emphasize the urgent need to promote awareness regarding CT in children for the strengthening of evidence based therapies in children (19). Poor recruitment has been associated to the inadequate knowledge of participants on CT.…”

Stakeholder Views of Clinical Trials in Low- and Middle-Income Countries: A Systematic Review

“…3,51 The parents' implicit trust in physicians' recommendations to enroll children in trials was apparent, which may contrast to parental reluctance in high-income countries. 51 Although involving parents and families in the protocol design is encouraged universally, this involvement is more difficult in LMICs in which there are specific misconceptions regarding trials.…”

“…51 Although involving parents and families in the protocol design is encouraged universally, this involvement is more difficult in LMICs in which there are specific misconceptions regarding trials. 3 Rumors such as "blood and organ stealing," as well as belief in witchcraft as a cause of illness and traditional medicines as a cure, were often regarded as more important and relevant than biomedical risks. 22 These rumors and fears of stigmatization for participation may be constructively addressed by contextualization, community education to improve health literacy, and engaging community representatives to help build trust by fostering mutual understanding, respect, and equity.…”

“…1,2 Although research has contributed positively by promoting therapeutic advances in health care of children, inequities are still evident across different income settings. 3 The "10/90 gap" suggests that only 10% of global health research funds are invested into medical conditions that account for 90% of the global disease burden, which resides mainly in the low-and middle-income countries (LMICs). 4,5 Although 89% of children live in LMICs and contribute 98% to the global burden of disease, only one-quarter of trials are conducted in this setting.…”

Extrapolation of efficacy and other data to support the development of new medicines for children: A systematic review of methods

“…Adult data are often available at the time development of a new medicine begins in children. Moreover, trials in children can be more challenging to conduct due to practical constraints on available sample sizes and pharmacokinetic sampling [11]. There is also a common perception that recruitment into paediatric trials will be challenging, although this has been contradicted by recent research finding that parents and practitioners are willing to enter children into trials 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20 21 22 23 24 25 26 27 28 29 30 31 32 33 34 35 36 37 38 39 40 41 42 43 44 45 46 47 48 49 50 51 52 53 54 55 56 57 58 59 60 F o r P e e r R e v i e w [12].…”