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Limited Capacity in US Pediatric Drug Trials

Qualitative Analysis of Expert Interviews

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Abstract

Background

The recently renewed Best Pharmaceuticals for Children and Pediatric Research Equity Acts (BPCA/PREA) have continued industry incentives and opportunities for pediatric drug trials (PDTs). However, there is no current assessment of the capacity to perform PDTs.

Objective

The aim of this study was to deepen understanding of the capacity for US PDTs by assessing PDT infrastructure, present barriers to PDTs, and potential approaches and solutions to identified issues.

Design/Methods

Pediatric clinical research experts participated in semi-structured interviews on current US pediatric research capacity (February–July 2007). An initial informant list was developed using purposive sampling, and supplemented and refined to generate a group of respondents to explore emerging themes. Each phone interview included a physician researcher and two health researchers who took notes and recorded the calls. Health researchers produced detailed summaries, which were verified by the physician researcher and informants. We then undertook qualitative analysis of the summaries, employing multiple coding, with the two health researchers and the physician researcher independently coding each summary for themes and subthemes. Coding variations were resolved by physician researcher/health researcher discussion and consensus achieved on themes and subthemes.

Results

The 33 informants’ primary or secondary roles included academia (n = 21), federal official (5), industry medical officer (8), pediatric research network leader (10), pediatric specialist leader (8), pediatric clinical pharmacologist (5), and practitioner/research site director (9). While most experts noted an increase in PDTs since the initial passage of BPCA/PREA, a dominant theme of insufficient US PDT capacity emerged. Subthemes included (i) lack of systems for finding, incentivizing, and/or maintaining trial sites; (ii) complexity/demands of conducting PDTs in clinical settings; (iii) inadequate numbers of qualified pediatric pharmacologists and clinician investigators trained in FDA Good Clinical Practice; and (iv) poor PDT protocol design resulting in operational and enrollment difficulties in the pediatric population. Suggested potential solutions for insufficient PDT capacity included (i) consensus-building among stakeholders to create PDT systems; (ii) initiatives to train more pediatric pharmacologists and educate clinicians in Good Clinical Practice; (iii) advocacy for PDT protocols designed by individuals sensitive to pediatric issues; and (iv) physician and public education on the importance of PDTs.

Conclusions

Insufficient US PDT capacity may hinder the development of new drugs for children and limit studies on the safety and efficacy of drugs presently used to treat pediatric conditions. Further public policy initiatives may be needed to achieve the full promise of BPCA/PREA.

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Table I

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Acknowledgements

Support for this study was provided by the AAP. The authors have no conflicts of interest to disclose. The authors thank the 33 individuals who agreed to be interviewed and share their thoughts for the study. The authors also thank Harry Pellman, MD, for serving as a subject in the pilot phase of interview development, and Richard Gorman, MD, for his thoughtful critique of an early draft of the manuscript.

Author information

Correspondence to Dr Richard Wasserman MD, MPH.

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Wasserman, R., Bocian, A., Harris, D. et al. Limited Capacity in US Pediatric Drug Trials. Pediatr-Drugs 13, 119–124 (2011). https://doi.org/10.2165/11584240-000000000-00000

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Keywords

  • Health Researcher
  • Dominant Theme
  • Pediatric Drug
  • Contract Research Organization
  • Pediatric Pharmacologist