ABSTRACT
This article expands on a session, titled "Patient Centricity: Design and Conduct of Clinical Trials in Orphan Diseases," that was presented as part of a two-day meeting on Pediatric Drug Development at the International Society for Central Nervous System (CNS) Clinical Trials and Methodology (ISCTM) Autumn Conference in Boston, Massachusetts, in October 2020. Speakers from various areas of pediatric drug development addressed a variety of implications of including children in drug development programs, including implications for rare/orphan diseases. The speakers have written summaries of their talks. The session's lead Chair was Dr. Joan Busner, who wrote introductory and closing comments. Dr. Simon Day, regulatory consultant, outlined some of the past mistakes that have plagued trials that did not consult with patient groups in the early design phase. Dr. Atul Mahableshwarkar provided an industry perspective of a recent trial that benefited from the inclusion of patient input. Drs. Lucas Kempf and Maria Sheean provided regulatory input from the perspectives of the United States (US) Food and Drug Administration (FDA) and European Medicines Agency (EMA), respectively. Dr. Judith Dunn outlined a novel approach for assessing and rank ordering patient and clinician clinical meaningfulness and the disconnect that may occur. Dr. Busner provided closing comments, tied together the presented issues, and provided a synopsis of the lively discussion that followed the session. In addition to the speakers above, the discussion included two representatives from patient advocacy groups, as well as an additional speaker who described the challenges of conducting a pediatric trial in the US and European Union (EU), given the often competing regulatory requirements. This article should serve as an expert-informed reference to those interested and involved in CNS drug development programs that are aimed at children and rare diseases and seek to ensure a patient-centric approach.Copyright © 2023, Matrix Medical Communications. All rights reserved.
ABSTRACT
Background/Objective: This continuation study follows positive results of the Phase IIb study and aims to supplement findings with additional scale data and unblinded group membership. The goal is to further assess the potential impact of COVID-19 restrictions on symptom severity in additional domains captured by the Children's Depression Rating Scale-Revised (CDRS-R);Children's Yale-Brown Obsessive Compulsive Scale (CYBOCS);Swanson, Nolan, and Pelham (SNAP-IV);Pediatric Anxiety Scale (PARS);and Clinical Global Impression Scale-Severity (CGI-S). Design(s): Data was partitioned using the date of March 11, 2020. Baseline outcome measure data was analyzed. Additionally, individual item scores were considered across the two time periods, as were treatment group assignments. Descriptive statistics were computed using SPSS 27.0 and included mean, standard deviation, variance, and distribution measures. Result(s): YGTSS mean values under consideration did not appear to change significantly between the time periods before and after COVID-19 restrictions began, with total scores of 67.2 and 66.7, respectively. Mean baseline CY-BOCS scores of 7.45 (before) and 7.55 (after) also showed similar stability to PARS (11.7 before and 12 after) and CDRS (29 before and 27.5 after), with similar stability noted in the CGI-S. Conclusion(s): While clinically, it might be typical for symptoms of Tourette's syndrome to be susceptible to stressors, this was not demonstrated in relation to the COVID-19 pandemic by any significant differences in primary tic assessments or related/comorbid disorders. There did not appear to be significant differences in YGTSS, CY-BOCS, PARS, CDRS, or CGI-S domain scores or individual item scores before or after COVID-19 restrictions were in place.