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PP-9 Participating in paediatric drug research: identifying the burden
  1. Dewolf,
  2. Amza,
  3. Christiaens,
  4. De Bruyne,
  5. De Cock,
  6. Vande Walle
  1. UZ GENT, GENT, Belgium

Abstract

Background Nowadays, academic researchers, pharma-ceutical companies and regulatory authorities are more aware of the need for paediatric drug research. Conse-quently, more academic and industry-driven paediatric trials are conducted to evaluate the efficacy and safety of new drugs and to a lesser extent of off-patent and off-la-bel drugs. However little information is available on the burden associated with participating in clinical trials for the patients and their family/caregivers. In attempt of be-coming a Centre of Excellence in paediatric drug research it is important for us to fully understand this burden.

Methods This is a retrospective, single centre, observa-tional study. A questionnaire will be designed focusing on the overall costs and time investment for the participants and their caregivers. Topics of interest will be absenteeism at school, at work or in leisure; number of specific study related visits (out of standard of care); financial reward by the sponsor; etc. Additional questions will gauge the per-ception and experience of the patients and their parents. We will contact the parents of patients who participated in either an academic or industry driven trial between 2010 and 2017 at the departments of paediatric nephrol-ogy and gastroenterology of the Elisabeth Children’s Hospital (Ghent University Hospital). We will display the results of this questionnaire by using descriptive statistics.

Discussion By evaluating the results, we will identify what brings most burden to patients and their family/caregivers in participating in clinical trials. This will enable us to better understand this burden and eventually to anticipate by more and better information and support during the participation. This may increase compliance, especially important in drug trials. The data can help us to include these aspects in discussions with both ethical committee and sponsors (industry) during the develop-ment of the study design and during negotiation of the clinical trial agreement (inclusive of some compensation) between research centres and sponsors.

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