Regulations supporting orphan drug research have been evolving for more than 20 years, providing incentives and guidance for development of potentially promising drugs to treat rare indications. More recently, with global attention and advocacy focused on improving treatments in these areas of unmet need, regulators and legislators have sought meaningful changes in the design and execution of rare disease drug development programs.
Understanding existing resources, financial aid, and incentives — and how to access them — is important for any orphan product developer. This webinar will describe support offerings available through global regulatory agencies and examine pending initiatives affecting orphan drug clinical development. These include:
Relevant guidance documents such as natural history studies and biomarkers.
Expedited review pathways such as FDA breakthrough therapy and EMA accelerated assessment designations.
Engaging regulators to advance development of priority medicines.
What lawmakers are discussing