About Leone Atkinson, MD, PhD

Dr. Leone Atkinson is a Senior Medical Director in Neuroscience and leads the Rare Disease Working Group. In this role, Dr. Atkinson is responsible for consolidating lessons learned, relationships and expertise for rare disease across the organization. She provides scientific and clinical support for a number of clinical trials in Rare Disorders, including Fragile X Syndrome, Mucopolysaccharidosis IV, Late-stage Pompe disease, Duchenne Muscular Dystrophy, Acute Intermittent Porphyria, and Progressive Supranuclear Palsy. Before joining Covance, Dr. Atkinson served as a Medical Consultant for biopharmaceutical companies and the National Institutes of Health. From 2007-2010, she served as Executive Director of Clinical Development at PTC Therapeutics where she was responsible for the global clinical development program for ataluren in Duchenne Muscular dystrophy and other rare genetic disorders. Prior to that, she served as a Clinical Research Director at Sanofi-Aventis, where she lead a large clinical program for the treatment of Multiple Sclerosis and served as Associate Medical Director of Neuroscience at Novartis Pharmaceuticals. Dr. Atkinson received her Ph.D. in Medical Genetics from the University of British Columbia in Vancouver, BC, Canada. She completed a residency in Neurology and a post-doctoral fellowship at Mount Sinai Medical Center in New York, NY.

Accelerating Rare Disease and Orphan Drug Development: Opportunities for Biomarkers, Diagnostics & Patient Engagement

Rare diseases affect more than 350 million people worldwide but patients often face limited Covance Labs Rare and Orphan Drug Development. Photo of DNA Strandoptions for approved therapies. As a result, many patients have joined advocacy groups first and foremost to connect with others struggling with their rare disease, but also to promote research, unite multiple stakeholders and stimulate new possibilities in the therapeutic pipeline. Research and orphan drug development efforts are starting to follow suit by increasingly incorporating patients’ needs and examining potential outcomes.

Addressing clinical challenges in rare disease and orphan drug development

With government-driven financial incentives, advances in genomic technology to identify promising targets for drug development, increasingly organized patient communities, and above average regulatory approval rates, drug developers are motivated to address rare diseases. While these trends are promising for patients with urgent unmet medical needs, orphan drug development still faces many challenges. The very nature of rare disease places pressure on identifying and accessing a sufficient number of patients for clinical trials.

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The Evolution of Open-Label Extensions and Rare Disease Studies

When running early development studies, sponsors must consider whether orCovance Employees in Lab Rare Disease Studies not to provide open-label, long-term treatment at the end of their randomized, placebo-controlled trial. In the past, sponsors have been hesitant to offer open-label extensions. But with our industry’s increasing focus on patient-centric care, sponsors are now weighing their options to incorporate more patient input into early development, especially when working with promising novel treatments in rare disease studies.

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