How to Improve the Implementation of Academic Clinical Pediatric Trials Involving Drug Therapy? A Qualitative Study of Multiple Stakeholders

Delphine Girard,Françoise Brion,Annick Tibi,Corinne Alberti,Olivier Bourdon,Hendy Abdoul,Sonia Prot-Labarthe,Imti Choonara

doi:10.1371/journal.pone.0064516

Delphine Girard, Françoise Brion + Show 6 more

Open Access

https://doi.org/10.1371/journal.pone.0064516

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Abstract

ObjectiveThe need for encouraging pediatric drug research is widely recognized. However, hospital-based clinical trials of drug treatments are extremely time-consuming, and delays in trial implementation are common. The objective of this qualitative study was to collect information on the perceptions and experience of health professionals involved in hospital-based pediatric drug trials.MethodsTwo independent researchers conducted in-depth semi-structured interviews with principal investigators (n = 17), pharmacists (n = 7), sponsor representatives (n = 4), and drug regulatory agency representatives (n = 3) who participated in institutionally sponsored clinical trials of experimental drugs in pediatric patients between 2002 and 2008.ResultsDissatisfaction was reported by 67% (16/24) of principal investigators and pharmacists: all 7 pharmacists felt they were involved too late in the trial implementation process, whereas 11 (65%) principal investigators complained of an excessive regulatory burden and felt they were insufficiently involved in the basic research questions. Both groups perceived clinical trial implementation as burdensome and time-consuming. The sponsor and regulatory agency representatives reported a number of difficulties but were not dissatisfied.ConclusionsThe heavy burden related to regulatory requirements, and suboptimal communication across disciplines involved, seem to be the main reasons for the major delays in pediatric drug trial implementation. The pharmaceutical aspects are intrinsically tied to trial methodology and implementation and must therefore be examined, in particular by involving Clinical Research Pharmacists at early stages of study conception.

Highlights

In everyday practice, drug therapy in infants and children is often guided by personal experience and good intentions rather than by evidence from clinical trials
For older medicines not covered by a patent, a pediatric investigation plan can be devised in order to obtain a pediatric-use marketing authorization, which is associated with a patent that is protected for 10 years [19]
The study was done in 17 principal investigators, 7 pharmacists, 4 sponsor representatives, and 3 ANSM representatives

Summary

Introduction

Drug therapy in infants and children is often guided by personal experience and good intentions rather than by evidence from clinical trials. To address the paucity of pediatric research and to encourage investment by pharmaceutical companies, the United States and Europe have enacted new legislation about efficacy and safety drug trials in children [11,12,13,14,15,16,17,18]. For older medicines not covered by a patent, a pediatric investigation plan can be devised in order to obtain a pediatric-use marketing authorization, which is associated with a patent that is protected for 10 years [19]. This regulation appears to have addressed the availability of medicines with ageappropriate information [20]. Studies done in the USA showed that drugs frequently used in pediatric patients were underrepresented among drugs qualifying for pediatric exclusivity [20,21,22,23]

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