Webinar | The Evolving Development of Outcome Measures to Facilitate Clinical Trials for Rare Diseases
The development of clinically-relevant, robust and sensitive outcome measures is an essential component of designing meaningful clinical trials to test the effects of treatments for rare diseases. During this 60-minute webinar, our panel of speakers discuss the following topics:
- General principles for developing outcome measures based on their experiences in this field
- The challenges COVID-19 has posed to clinical trials in rare diseases, including how trials and natural studies already underway have adapted to develop remote outcome measures
- Learnings from developing remote outcome measures during the pandemic
- Examples of remote outcome measures that have been and are being developed for rare neuromuscular diseases
- Q&A session
Expert speakers include:
- Dr Michelle Eagle, Managing Director at ATOM International
- Meredith James, Clinical Specialist Neuromuscular Physiotherapist at John Walton Muscular Dystrophy Research Centre, Newcastle University, UK
- Nathan Peck, Founder and CEO of Cure VCP Disease Inc.
- Dr Lindsay Alfano – Assistant Professor at Nationwide Children’s Hospital, Columbus, Ohio, USA
You can watch the webinar recording below. We did our best to answer as many questions as possible during the session, but if you have any additional ones for us please submit them via email to firstname.lastname@example.org
About our speakers
Dr Michelle Eagle | Managing Director at ATOM International
Michelle has been a physiotherapist for nearly 40 years and has specialised in neuromuscular disorders for 30 years. Her PhD investigated the impact of nocturnal ventilation in DMD. From 1990 to 2016 Michelle was the Lead Physiotherapist at the John Walton Centre for Neuromuscular Diseases in Newcastle. She has been involved in the development of outcome measures and standards of care for many years and currently is the Managing Director of ATOM International Limited which is a company that specialises in consultancy, training and quality control for clinical trials in neuromuscular diseases.
Meredith James | Clinical Specialist Neuromuscular Physiotherapist at John Walton Muscular Dystrophy Research Centre, Newcastle University, UK
Meredith James is a clinical specialist neuromuscular physiotherapist at the John Walton Muscular Dystrophy Research Centre, Newcastle, UK, where she is involved in clinical and research activity for children and adults with neuromuscular disorders. After graduating from the University of Sydney, Australia, Meredith has worked in Australia and East Timor before moving to the UK and joining the Newcastle team.
Meredith has a special interest in outcome measures, orthoses, gait and research into these areas. In her clinical research capacity, Meredith is responsible for the clinical evaluation of children and adults involved in natural history and clinical trials, as well as the development of clinically meaningful, reliable and sensitive outcome measures for NMD. Meredith has recently led the international collaboration of Physiotherapists who have developed the North Star Assessment for limb girdle type muscular dystrophies.
Nathan Peck | Founder and CEO of Cure VCP Disease Inc.
Nathan Peck is the Founder and CEO of Cure VCP Disease, Inc., a patient advocacy organisation dedicated to driving a cure for VCP Disease, also known as IBMPFD. The disease is an adult-onset, hereditary, autosomal dominant disease caused by mutation of the valosin-containing protein gene on chromosome 9. It can affect any combination of a patient’s muscles, bones and brain. Nathan is a patient with the disease and was officially diagnosed in 2015. His mother and aunt passed away from the disease and he has two uncles still living with the disease. He is retired, on full disability and has quickly grown the awareness and influence of Cure VCP Disease among the research and patient community since 2018.
Dr Lindsay Alfano | Assistant Professor at Nationwide Children’s Hospital, Columbus, Ohio, USA
Dr Lindsay Alfano is a physical therapist with expertise in the care and evaluation of patients with neuromuscular disease, including outcome measure development, evaluation and administration. She has an integral role in planning and designing clinical trials, contributing to protocol development, outcome measure selection, statistical analysis and interpretation for ongoing clinical trials.
Lindsay serves on an international consortium of neuromuscular physical therapy experts who provide industry-standard training and reliability for multi-site international trials. In response to the COVID-19 pandemic, Lindsay and the physical therapy team at Nationwide Children’s Hospital have led initiatives to understand and validate remote testing for patients with neuromuscular diseases for clinical and research purposes.