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No Opportunity Unturned: Optimizing Orphan Drug Development

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
Recorded Apr 22 2020 59 mins
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Presented by
Angi Robinson, Lisa Pitt, and Juliet Moritz
Presentation preview: No Opportunity Unturned: Optimizing Orphan Drug Development

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    This webinar reviews the history of patient advocacy groups in advancing clinical research and examines the evolution of their role in light of recent and pending legislative and regulatory changes in the United States and European Union. Included is a discussion of how patient advocacy groups and industry can join to respond most effectively to these anticipated changes. Presenters are Susan Stein, MPH, a member of the Board of Directors of worldwide patient advocate umbrella organization Global Genes, and Juliet Moritz, MPH, Executive Director of Strategic Drug Development for Rare Diseases at Premier Research.
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    James Sacchetta and Jay Trepanier
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    However, manual processes have made forecasting of clinical trials challenging, leading to difficulties in reforecasting and cash management (and as a result, difficulties in maintaining site satisfaction) as well as hefty fines.

    Viewers will learn more about:
    - The consequences of poor forecasting
    - The difficulties of reforecasting with manual systems
    - The complexities that make forecasting so challenging
    - How technology that is purpose-built for clinical trials can help CROs and Sponsors forecast more effectively and accurately
Built for Biotech℠
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.

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  • Live at: Apr 22 2020 6:20 pm
  • Presented by: Angi Robinson, Lisa Pitt, and Juliet Moritz
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