Centered on Sites: Committing to a Collaborative Approach for Rare Disease Clinical Trial Success

Investigative sites are fundamental to the success of clinical trials. In many rare disease trials, recruitment relies heavily on their existing patient population. As the epicenter of clinical trial execution, providing sites with complementary support enables them to execute the study at the highest level. COVID-19 has further amplified the need to work collaboratively as we continue to drive rare disease clinical research forward.

Sites are intimately connected to their patients. They have a deep understanding of the disease area and the challenges it presents to the patient and their caregivers. Tapping into their expertise and putting the site-patient relationships at the core of building a strategy can be an important first step toward success. Establishing the site as your partner early in the trial process, seeking opportunities to reduce site staff/patient burden, and providing appropriate support tools and personnel will aid enrollment. Implementing this upfront gets ahead of common enrolment issues, maintains motivation, and reduces lost time.

Additionally, reducing site visits, identifying the most critical collection points, and maximizing digital solutions can improve efficiency and reduce face-to-face interactions to ensure studies are conducted safely and effectively during the pandemic. Ensuring that sites are supported appropriately from a financial perspective instills confidence, particularly for rare disease studies that can continue for a number of years.

Speakers

Liz Moore, DNP, MSN, CPNP-AC, CPN, Advanced Clinical Practitioner Manager, Medpace

Dr. Moore is a board-certified acute care pediatric nurse practitioner with more than ten years of clinical experience in the acute care setting at Cincinnati Children’s Hospital. As a bedside nurse in a Level IV NICU, Ms. Moore managed the care of critically ill infants with infantile, surgical, and pediatric rare diseases. As a Nurse Practitioner, Ms. Moore was a provider in the CICU to patients within the full spectrum of cardiac indications, including both pediatric and adult congenial heart diseases, heart and lung transplant, and device. Since joining Medpace, her clinical trial experience has been focused on indications within cardiology, pediatrics, and rare disease including cardiomyopathy, arrhythmia, PAH, metabolic disease, and cardiac device. Ms. Moore leverages her clinical expertise to support both medical and operational aspects of clinical trial conduct, such as providing scientific background, disease education, CRA training, site/patient materials, and assistance with feasibility and recruitment activities.

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Kelly Millhaem, MHA, Senior Clinical Trial Manager, Medpace

Kelly Millhaem is an experienced Clinical Trial Manager with over twelve years of experience in clinical research. Ms. Millhaem has overseen multiple global trials and her experience includes management of trials in cardiovascular, neuromuscular, metabolic, hepatic and endocrine indications, with a special focus on rare/orphan diseases and pediatrics. Ms. Millhaem’s leadership and communication skills are reflected in her work with Sponsors, vendors, site staff, and study team members to ensure successful navigation of complex protocols, achieve recruitment and retention goals, meet study timelines, and ensure quality data in the overall conduct of trials. Ms. Millhaem’s past experience as a Global lead CRA in complex indications allows her special insight in implementing optimized, high quality monitoring strategies. Ms. Millhaem has an educational background in biology with a Master’s degree in Health Administration.

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Simon Jones, MBChB, MRCPCH Consultant in Paediatric inherited metabolic disease, Senior Lecturer at The University of Manchester

Dr. Jones is a consultant in paediatric inherited metabolic diseases at the Willink Unit at Saint Mary’s Hospital. His major research interest is therapy for lysosomal storage diseases (LSD’s). He received his medical training at the Edinburgh University Medical School, Edinburgh, UK, with a BSc in Neurosciences. He moved to London and trained in Paediatrics at Guy’s and St. Thomas’ Hospital, London, UK.  He has been working at the Willink Biochemical Genetics Unit in Manchester since September 2005. Since 2008, he has been a consultant in paediatric inherited metabolic diseases at the Willink Unit and is now the clinical lead for the LSD service. The Willink Unit is now part of the Manchester Centre for Genomic Medicine at Saint Mary’s Hospital. Dr. Jones has been actively involved in many phase I-IV international multicentre trials of novel therapies for LSD’s. He is currently the principal investigator in a number of LSD trials, and a senior lecturer at the University of Manchester. Dr Jones is now the medical director of the NIHR Manchester children’s clinical research facility. He is an author of over 200 peer-reviewed papers and 4 book chapters.

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