Biopharmaceuticals (includes Biologics)
A reflection on the progress achieved over the past decades is required in order to understand how to best address remaining challenges in the field of orphan medicines development. Industry, regulators and patient representatives will discuss the current trends happening worldwide in the area, and will provide their thought leadership on which is the best way forward to continuously and effectively tackle the remaining challenges of rare diseases. A discussion on multi-stakeholder collaboration so far, on where the key gaps exist and what changes need to happen to ensure no patient is left behind will require an active audience of regulatory professionals with an inspiring sense of purpose to fuel the debate. Content is expected to include discussions on EMA-FDA collaboration clusters, recent developments from global agencies and recent efforts from R&D industry in addressing R&D gaps.