Part 3: Rare Disease Clinical Development – Strategies for Ensuring Endpoint Integrity

Because clinical trial endpoints for rare diseases are often not well defined, consistency of endpoint assessments across study sites and over time are critical to ensuring data integrity. Having a cohesive training strategy that includes conducting pre-study evaluation and training, as well as ongoing review of clinical evaluator (CE) performance and adherence to standardised procedures is an important contributor to success.

In this free webinar, Medpace partners with Michelle Eagle of ATOM International, a provider of CE training for clinical trials across the world, to discuss approaches and steps that can be taken to ensure quality and integrity. For example, neuromuscular studies incorporate training of clinical evaluators at investigator meetings, support video-recording of patient assessments at study visits to review for quality control and perform periodic refresher training throughout the course of a study.

Topics will include:

• What does a comprehensive training program look like?
• How natural history data can inform endpoint selection (real-word examples)
• Rating assessment considerations and review (video QC and clinical assessment reliability)
• The role of data management – cleaning and coding of data to create structured output for review and reliability

To register for the other parts in this webinar series, please click below:

Speakers

Meredith James, Master Physiotherapist, ATOM International

Meredith James is a neuromuscular physiotherapist by training and a senior member of the ATOM International team. Working as part of a team of international trainers in the development, implementation and quality control of standardised endpoint study assessments, she trains Clinical Evaluators across the globe, leads Investigator meeting training, and works with pharmaceutical companies on protocol development and endpoint selection and analysis.

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Chad Leslie, Sr. Data Manager, Medpace

Chad Leslie is a Senior Data Manager with 12+ years of industry experience in data collection for drug development. Mr. Leslie specializes in EDC builds/endpoint data management of phase 1-3 oncology and rare disease studies. These studies include investigational products for Duchenne muscular dystrophy, Charcot-Marie-Tooth disease, Facioscapulohumeral Muscular Dystrophy, immunotherapy, targeted agents, viral vector, novel biologic agents, and radioactive agents. He holds an undergraduate degree in mathematics from the University of Cincinnati.

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Michelle Petersen, MS, Senior Director, Clinical Trial Management, Medpace

Michelle Petersen has 13 years’ experience in preclinical and clinical research which includes managing global studies in multiple phases of clinical development. She has managed multiple global rare/ultra-rare disease trials with a focus on neuromuscular and pediatric disorders. Ms. Petersen is a member of Medpace’s specialized Rare Disease Consortium and she is adept in developing strategies necessary to address the potential challenges of recruitment and logistical requirements for studies of this nature. Ms. Petersen holds a Master of Science degree in Physiology from the University of Cincinnati College of Medicine.

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