A Delphi Process to Optimize Quality and Performance of Drug Evaluation in Neonates

Abstract

BackgroundNeonatal trials remain difficult to conduct for several reasons: in particular the need for study sites to have an existing infrastructure in place, with trained investigators and validated quality procedures to ensure good clinical, laboratory practices and a respect for high ethical standards. The objective of this work was to identify the major criteria considered necessary for selecting neonatal intensive care units that are able to perform drug evaluations competently.Methodology and Main FindingsThis Delphi process was conducted with an international multidisciplinary panel of 25 experts from 13 countries, selected to be part of two committees (a scientific committee and an expert committee), in order to validate criteria required to perform drug evaluation in neonates. Eighty six items were initially selected and classified under 7 headings: “NICUs description - Level of care” (21), “Ability to perform drug trials: NICU organization and processes (15), “Research Experience” (12), “Scientific competencies and area of expertise” (8), “Quality Management” (16), “Training and educational capacity” (8) and “Public involvement” (6). Sixty-one items were retained and headings were rearranged after the first round, 34 were selected after the second round. A third round was required to validate 13 additional items. The final set includes 47 items divided under 5 headings.ConclusionA set of 47 relevant criteria will help to NICUs that want to implement, conduct or participate in drug trials within a neonatal network identify important issues to be aware of.Summary Points1) Neonatal trials remain difficult to conduct for several reasons: in particular the need for study sites to have an existing infrastructure in place, with trained investigators and validated quality procedures to ensure good clinical, laboratory practices and a respect for high ethical standards. 2) The present Delphi study was conducted with an international multidisciplinary panel of 25 experts from 13 countries and aims to identify the major criteria considered necessary for selecting neonatal intensive care units (NICUs) that are able to perform drug evaluations competently. 3) Of the 86 items initially selected and classified under 7 headings - “NICUs description - Level of care” (21), “Ability to perform drug trials: NICU organization and processes (15), “Research Experience” (12), “Scientific competencies and area of expertise” (8), “Quality Management” (16), “Training and educational capacity” (8) and “Public involvement” (6) - 47 items were selected following a three rounds Delphi process. 4) The present consensus will help NICUs to implement, conduct or participate in drug trials within a neonatal network.

Clinical trials in neonates: How to optimise informed consent and decision making? A European Delphi survey of parent representatives and clinicians

“…However, the methods of Delphi studies do vary as noted in a systematic review conducted by Boulkedid and colleagues [ 10 ] and the “The RAND/UCLA Appropriateness Method User's Manual” [ 13 ]. Based on currently used cut-off values, we used a scoring of 1–9 for the importance of the different items with median scores of 7–9 being considered high enough to reach consensus [ 10 , 14 , 15 ].…”

Paediatric clinical research in Europe: an insight on experts’ needs and perspectives

“…To draft the questionnaire the following sources were considered: the CORBEL (Coordinated Research Infrastructures Building Enduring Life-science services) project [ 11 ]; the FP7 TINN (Treat Infection in Neonates) project questionnaire [ 12 ]; and the survey previously run by the TEDDY European Network of Excellence for Paediatric Research [ 13 ].…”

Capacities and Competences for Drug Evaluation in European Neonatal Intensive Care Units: A Survey and Key Issues for Improvement

“…This study (TINNTER) was based on a questionnaire developed after our previous Delphi study 7 conducted with an international multidisciplinary panel of 25 experts from 13 countries and selected to be part of two committees (a scientific committee and an expert committee). These DELPHI questions were initially grouped into five parts: (1) NICUs description, (2) NICU organization and processes, (3) scientific competences and expertise, (4) quality management, and (5) public involvement that agreed on 47 out of 86 items.…”

“…Most studies have to be multicenter, national, or international to recruit patients in the different neonatal subgroups, according to guidelines for drug evaluation in neonates (European Medicines Agency [EMA]). 6 As European neonatal research would benefit from a network for conducting high-quality studies, we previously conducted a Delphi study 7 and used the validated items to build a questionnaire named TINNTER, developed under two European Seventh Framework Program 7 (FP7) projects, namely, TINN (Treat Infections in Neonates) and GRiP (Global Research in Pediatrics), to survey research experience and capacities of European Union (EU) NICUs and to identify areas for improvement.…”