Clinical, Regulatory and Commercialization Strategies for Rare Diseases

Throughout the clinical development and commercialization lifecycle of products to treat rare diseases, there are a variety of clinical studies that can be conducted. Studies may be conducted for various reasons including product regulatory approval, patient access to a product for an unmet need, patient registries, or as a post-marketing requirement following product approval. The regulatory strategy employed often dictates the type of study that needs to be conducted to address an associated regulatory requirement.

This webinar will address the various clinical, regulatory (US & EU) and commercialization strategies that may be implemented in the development of products to treat rare diseases and orphan indications.

Key Learning Objectives:

• US Regulatory requirements and associated study design – Registries, pre-, post-, and peri-approval designs
• EU regulatory environment and Compassionate Use/Expanded Access Programs (EAP)
• Real World case examples and lessons learned

Speaker

William C. Maier, PhD, Chief Scientific Officer, REGISTRAT-MAPI

Dr. Maier has over 20 years experience in the bioPharmaceutical industry with specialty in the design, analysis, and interpretation for epidemiology and observational studies. He is the Founding Editor of the PharmacoEPI and Risk Management Newsletter quarterly publication (www.prmnewsletter.org). Dr. Maier is a frequent international speaker on Epidemiology, Health Technology Assessment, Drug Safety and Risk Management.

Previously, he served as the Senior Director of Epidemiology at GlaxoSmithKline and Elan Pharmaceuticals. He is an EMEA Working Group member focusing on the Network of Pharmacoepidemiology Centres in the EU EnCEPP Project. His research specialties include respiratory, endocrinology, gastrointestinal, autoimmune, vaccines, and oncology therapies

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