Decoding Orphan Drug development: Bridging Boundaries and Thinking “Outside the Box”

This session will cover the development of Orphan Drugs and will highlight the opportunities and challenges in developing drugs for rare diseases. Most rare diseases are serious, life-altering and often fatal. It is estimated that worldwide the total number of persons afflicted by a rare disease is between 260 and 450 million, corresponding to about 4% to 6% of the world’s population. The session will discuss initiatives and regulatory frameworks aimed at attracting research and development efforts into rare diseases and orphan drugs, with a focus on Europe versus the United States. The advantages of early consultation with regulatory authorities will also be discussed, emphasizing areas where early agreements with Health Authorities are critical for the success of the development, authorization and commercialization of the product. An outline of a Global Regulatory Strategy Plan will be presented and discussed as well as implementation of an Expanded Access and Compassionate Use Programs. The regulatory strategy will discuss the advantages of the Orphan Drug Designation/Orphan Medicinal Product Designation by the FDA and EMA during drug development, and how to explore the synergies of parallel orphan designation applications. The session will also highlight case studies, including the use of innovative approaches for approval of Rylaze, a leukemia and lymphoma drug, as well as use of the FDA RTOR program and Project ORBIS, a multi-Health Authority program aimed at providing a framework for concurrent submission and review of oncology products amongst international partners. The session will also focus on key challenges that still hamper orphan drug development and the conduct of clinical trials in rare diseases.

* One lucky attendee will win a copy of RAPS Orphan Drug Development for Rare Diseases (e-book)