S.P.18 Clinical Evaluator training for the GSK DEMAND programme

Abstract High quality standardised training of Clinical Evaluators (CEs) in multi-centre clinical trials in Duchenne Muscular Dystrophy (DMD) is essential for reliable data. GSK is conducting clinical trials to evaluate the efficacy of an antisense oligonucleotide GSK2402968 in boys with DMD. The data by which interventions are measured are collected by CEs, primarily physiotherapists, but in some centres other professionals have been trained. Standardised, reliable and repeatable clinical evaluation is critical to the success of this clinical trial. Appropriate outcomes for ambulant boys including six minute walk distance, respiratory function tests, quantitative muscle testing, North Star Ambulatory Assessment and timed function tests were chosen. A ‘Master Physiotherapy’ team, consisting of eight experienced Neuromuscular Physiotherapists from the UK, Italy, Australia and the USA was established to provide a manual of operation, train in the standardised assessments and provide ongoing support to each CE and site. The team has developed a comprehensive package of training. Initial training included practical hands on sessions repeated over two days to facilitate learning, with the aim to standardise assessments, minimise inter and intra rater variability and reduce the potential for data drift. To ensure quality data collection throughout the duration of the trials and to support evaluators, sites submit video assessments at key points in the studies to Master Physiotherapists whom evaluate performance and provide feedback. Each CE receives follow up training via site visit or WebEx prior to the primary endpoint (efficacy) assessment. Since May 2010, the Master Physiotherapy team has trained 159 CEs in 65 sites across 24 countries, demonstrating that linguistic and cultural differences can be accommodated to give all sites the same opportunity to perform quality standardised assessments and work as a global team.