Rare disease drug development is challenging due to limited information on patient distribution, change in disease progression and relevant outcomes to measure to define treatment benefits.
The use of disease natural history studies prior to initiation of clinical trials can help provide the information to design clinical studies and recruit patients into trials of new therapies. Due to small and pediatric patient populations, there is an imperative to maximize the number of patients on active therapy in a rare disease drug development program. As a result, external controls based on data from disease natural history studies are sometimes used instead of an active or placebo control arm to provide comparative information for drug regulatory agencies.
This webinar will provide guidance on the design and conduct of rare disease natural history studies. It will provide case studies and practical strategies for patient identification, recruitment and retention.
Register today to:
- Learn about natural history designs that are scientifically robust and address the unique challenges of rare disease
- Understand how to use disease natural history information in rare disease drug development
- Sources of rare disease natural history information
- Practical strategies for running rare disease natural history studies
Dr. William C. Maier, MPH, PhD – Epidemiology
Vice President, Rare Disease, Drug Development Sciences, ICON
Dr. Maier has over 25 years of experience in drug development and commercialisation at pharmaceutical companies in Europe, Canada, the United States and Asia. In his current role, he works with product development companies throughout the world to provide regulatory, strategic and scientific guidance on product development and commercialisation. He has experience in rare disease, respiratory, neurology, psychiatry, autoimmune, oncology, endocrinology, dermatology cardiovascular, cerebrovascular, urology, opioid dependency, vaccine and anti-infective therapy areas.
- Pharma and biotech professionals involved in rare disease development, including:
- Medical directors
- Therapy area directors
- Clinical trial directors and managers
- Research managers
- Patient advocacy groups looking to set up registries and clinical trials