Transforming Performance of Clinical Trials in Pediatric Type 2 Diabetes: A Consortium Model.

Background: Completing phase 3 trials of new drugs for youth with type 2 diabetes is challenging. The Pediatric Diabetes Consortium (PDC) of U.S. pediatric treatment centers developed a Consortium model to improve the efficiency of successfully completing these trials. Aims and Innovations: An aim of the PDC model is to utilize the resources of the PDC Coordinating Center and Executive Committee to improve study protocols, centralize interactions with sponsors, and oversee the performance of PDC Clinical Centers. Key features include a Consulting Group to improve protocol design; Master Service Agreements between the Coordinating Center and Clinical Centers covering confidentiality agreements and contract language; negotiation of a standard Site Budget with Contract Research Organizations (CROs)/Sponsors that reflect actual Clinical Center costs; Weekly Conference Calls with CROs/sponsors to track progress of Clinical Center launches, Monthly Oversight Calls with investigators and study Coordinators to track Clinical Center performance, discuss enrollment strategies, and identify emerging problems. Successes and Challenges: The Consortium model played a key role in the completion of the pivotal trial of liraglutide for treatment of youth with type 2 diabetes. PDC centers also played a pivotal role in exceeding the projected number of randomized subjects needed by two ongoing studies that are nearing completion. Conclusions: While the Consortium model is still a work in progress, PDC has assisted in the successful launch of new type 2 diabetes studies, and negotiations are in underway for PDC participation in pediatric type 1 diabetes and other diabetes-related studies.

Effectiveness of paliperidone long-acting injection in clinical practice.

BACKGROUND: The efficacy of the long-acting injectable formulation of the antipsychotic paliperidone (paliperidone palmitate) has been investigated in randomized controlled trials. Due to the nature of study designs, these may not be representative of usual clinical practice. The aim of this study was to assess the clinical effectiveness of the long-acting injectable antipsychotic paliperidone palmitate using treatment continuation at 1 year as an outcome. METHODS: Patients were initiated on paliperidone palmitate prior to December 2014 in a single health board in Wales (UK). Demographic factors that may have influenced outcome, including diagnosis, age at initiation, sex, inpatient or outpatient status on initiation, were analysed to assess whether they influenced patient outcome. For patients completing 1 year of treatment, inpatient stay in the 12 months prior to and following paliperidone palmitate initiation was compared. RESULTS: Data were available for 64 patients; 41 had a diagnosis of schizophrenia and 7 had previously received clozapine. Continuation rates at 6 and 12 months were 69% and 63% respectively. Treatment continuation was not associated with demographic factors. For continuers, mean inpatient stay pre- and post-initiation was 83.2 ± 105.3 and 73.5 ± 103.3 days respectively (p = 0.61). The most common reason for discontinuation was lack of effect (n = 9). CONCLUSIONS: The proportion of patients remaining on treatment was comparable to that reported in other naturalistic studies. Prescribing for indications outside the product licence was relatively common, but did not appear to influence outcome, although the number of patients in each group was small. Treatment continuation at 6 months appeared to be a predictor of longer-term outcome.