“Include me if you can”—reasons for low enrollment of pediatric patients in a psychopharmacological trial

Abstract

Background Low recruitment in clinical trials is a common and costly problem which undermines medical research. This study aimed to investigate the challenges faced in recruiting children and adolescents with obsessive-compulsive disorder and autism spectrum disorder for a randomized, double-blind, placebo-controlled clinical trial and to analyze reasons for non-participation. The trial was part of the EU FP7 project TACTICS (Translational Adolescent and Childhood Therapeutic Interventions in Compulsive Syndromes). Methods Demographic data on pre-screening patients were collected systematically, including documented reasons for non-participation. Findings were grouped according to content, and descriptive statistical analyses of the data were performed. Results In total, n = 173 patients were pre-screened for potential participation in the clinical trial. Of these, only five (2.9%) were eventually enrolled. The main reasons for non-inclusion were as follows: failure to meet all inclusion criteria/meeting one or more of the exclusion criteria (n = 73; 42.2%), no interest in the trial or trials in general (n = 40; 23.1%), and not wanting changes to current therapy/medication (n = 14; 8.1%). Conclusions The findings from this study add valuable information to the existing knowledge on reasons for low clinical trial recruitment rates in pediatric psychiatric populations. Low enrollment and high exclusion rates raise the question of whether such selective study populations are representative of clinical patient cohorts. Consequently, the generalizability of the results of such trials may be limited. The present findings will be useful in the development of improved recruitment strategies and may guide future research in establishing the measurement of representativeness to ensure enhanced external validity in psychopharmacological clinical trials in pediatric populations. Trial registration EudraCT 2014-003080-38. Registered on 14 July 2014.

Memantine as treatment for compulsivity in child and adolescent psychiatry: Descriptive findings from an incompleted randomized, double-blind, placebo-controlled trial

“…Due to this very small sample size, we were not able to perform statistical analyses and comparisons between the verum and placebo group. As is the case in many (pediatric) clinical psychopharmacology trials [ 49 ], low enrollment and high exclusion rates during the recruitment period raise the question of participants’ representativeness of real-world patients and therefore the generalizability of the respective trial results [ 50 ]. Out of a total of N = 173 pre-screened patients, only n = 5 (2.9%) were eventually enrolled in the study.…”

“…Out of the n = 168 not included patients, n = 73 (43.5%) failed to meet all of the trial inclusion criteria or met one or more of the exclusion criteria, n = 75 (44.6%) declined participation due to personal reasons, and n = 20 (11.9%) did not participate due to general or other reasons (e.g., not yet being stable on medication). Reasons for low enrollment in this study have been comprehensively analyzed and discussed separately [ 50 ].…”

“…Further applications for ethical approvals in the UK (National Research Ethics Service Committee London – Camberwell St Giles) and in the Netherlands (Commissie Mensgebonden Onderzoek, Regio Arnhem-Nijmegen) were granted in June 2016 (Nijmegen), March 2017 (Utrecht) and June 2018 (London), respectively. Due to a small sample size, only descriptive statistical analyses of the data were performed (see Niemeyer et al [ 50 ] for details on recruitment difficulties). This study was conducted in accordance with the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) Guideline.…”

Challenges in Recruitment and Retention

“…In other words, the demand for participants in cancer research trials exceeds the number of patients motivated or able to participate. When research enrollment lags to this degree, a key component of health care is lost, innovation suffers, diagnoses continue to occur when it is too late, and investigators are left with a costly and sometimes trial ending problem 11 …”

“…When research enrollment lags to this degree, a key component of health care is lost, innovation suffers, diagnoses continue to occur when it is too late, and investigators are left with a costly and sometimes trial ending problem. 11 There are many factors that impact access to, and participation in, clinical trials. Different types of studies require different outreach accommodations and modifications.…”