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Patient-important activity and participation outcomes in clinical trials involving children with chronic conditions

  • Pediatric QOL Special Section
  • Published:
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Abstract

Purpose

Children with chronic conditions experience medical issues over long-term periods of time which can have lasting emotional and social consequences impacting daily life and functioning. Activities and participation outcomes are needed in order to comprehensively assess child-important health in clinical trials. Our objective was to review the extent to which activity and participation outcomes are included in clinical trials of childhood chronic disease and to determine what trial characteristics are associated with their use.

Methods

A review of a large clinical trial registration database (clinicaltrials.gov) was conducted over the 2010 calendar year. The measures used to assess primary and secondary endpoints were coded according to the ICF classification system. Trial characteristics that might be associated with activity and participation outcome use such as sponsorship type, intervention type, health condition, whether the trial was focused on pediatric patients, phase of trial and sample size were also extracted and explored with univariable and multivariable regressions.

Results

Four hundred and ninety-nine trials met inclusion criteria, 495 of which had complete information about hypothesized predictors. Only 36 out of 495 trials included an activity and participation outcome as part of the trial evaluation process. Both univariable and multivariable regression models showed that non-drug trials and late phase of trial (phase IV) showed the strongest likelihood with whether a trial would include an activity and participation outcome.

Discussion

Most registered clinical trials for children with chronic or ongoing medical conditions do not include a comprehensive approach to health outcomes assessment, especially drug trials and early phase trials. Outcome measures in pediatric clinical trials are lagging relative to World Health Organization standards for comprehensive health evaluation.

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Acknowledgments

Nora Fayed is a recipient of the Canadian Institute of Health Research postdoctoral fellowship and thanks postdoctoral mentor Aileen Davis for comments and editing support.

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Authors and Affiliations

  1. Health and Outcomes Research, Toronto Western Hospital, University Health Network, Toronto, ON, Canada

    Nora Fayed

  2. Department of Pediatrics, McMaster University, Hamilton, ON, Canada

    Nora Fayed & Olaf Kraus de Camargo

  3. CanChild Centre for Childhood Disability Research, McMaster University, 1400 Main Street West, Room 408, Hamilton, ON, L8S 1C7, Canada

    Nora Fayed, Olaf Kraus de Camargo & Eyal Cohen

  4. Division of Pediatric Medicine, Department of Pediatrics, The Hospital for Sick Children, University of Toronto, 555 University Avenue, Toronto, ON, M5G 1X8, Canada

    Inam Elahi, Ankita Dubey & Eyal Cohen

  5. Department of Pediatrics, Santa Maria Hospital, Lisbon, Portugal

    Ricardo M. Fernandes

  6. Laboratory of Clinical Pharmacology and Therapeutics, Faculty of Medicine, Instituto de Medicina Molecular, Lisbon, Portugal

    Ricardo M. Fernandes

  7. Physical Medicine and Rehabilitation, University of Pittsburgh, 4401 Penn Ave, Pittsburgh, PA, 15232, USA

    Amy Houtrow

  8. Institute of Health Policy, Management and Evaluation, University of Toronto, 155 College Street, Suite 425, Toronto, ON, M5T 3M6, Canada

    Eyal Cohen

Authors
  1. Nora Fayed
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  2. Olaf Kraus de Camargo
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  3. Inam Elahi
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  4. Ankita Dubey
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  5. Ricardo M. Fernandes
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  6. Amy Houtrow
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  7. Eyal Cohen
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Corresponding author

Correspondence to Nora Fayed.

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Fayed, N., de Camargo, O.K., Elahi, I. et al. Patient-important activity and participation outcomes in clinical trials involving children with chronic conditions. Qual Life Res 23, 751–757 (2014). https://doi.org/10.1007/s11136-013-0483-9

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  • DOI: https://doi.org/10.1007/s11136-013-0483-9

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