No Opportunity Unturned: Optimizing Orphan Drug Development

Presented by

Angi Robinson, Lisa Pitt, and Juliet Moritz

About this talk

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

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Premier Research is a clinical research company, dedicated to helping biotech, specialty pharma, and device innovators transform life-changing ideas and breakthrough science into new medical treatments. As a global company, Premier specializes in the use of innovative technologies for smart study design and trial management to deliver clean, conclusive data to sponsors. Whether it’s developing product lifecycle strategies, reducing clinical development cycle times, securing access to patients, navigating global regulations, maximizing the impact of limited rare disease data, or providing expertise in specific therapeutic areas, Premier is committed to helping its customers answer the unmet needs of patients across a broad range of medical conditions.