title: Limited capacity in US pediatric drug trials: qualitative analysis of expert interviews.
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.
  
  
  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.
authors.affiliation: Pediatric Research in Office Settings (PROS), Department of Research, American Academy of Pediatrics, Elk Grove Village, Illinois, USA. richard.wasserman@uvm.edu
authors.forename: Richard
authors.initials: R
authors.lastname: Wasserman
authors.forename: Alison
authors.initials: A
authors.lastname: Bocian
authors.forename: Donna
authors.initials: D
authors.lastname: Harris
authors.forename: Eric
authors.initials: E
authors.lastname: Slora
mesh_terms: D002648:Child; D002986:Clinical Trials as Topic / Q000706:statistics & numerical data*; D006801:Humans; D007407:Interviews as Topic; D010372:Pediatrics / Q000706:statistics & numerical data*; D012108:Research Personnel; D014481:United States