New Delhi: Parexel, one of the world’s largest clinical research organizations (CROs), has launched a new expert series, New Medicines, Novel Insights. The series will feature fresh insights from the company’s global, cross-functional experts analyzing trends impacting drug development and offering evidence-based guidance to the biopharmaceutical industry.
The inaugural report — Advancing Rare Disease Drug Development — explores the unique regulatory, scientific and market access challenges surrounding rare disease drug development and shares best practices to address them.
“Cutting-edge medicines are becoming more personalized and precise across the therapeutic landscape, while the process to develop those therapies is reaching new heights of complexity,” said Amy McKee, MD, Chief Medical Officer and Head of Oncology Center of Excellence. “Parexel’s New Medicines, Novel Insights research series offers expert-led guidance to deliver on the promise of patient-focused drug development and bring impactful treatments to patients more rapidly.”
Key takeaways from New Medicines, Novel Insights: Advancing Rare Disease Drug Development include strategies for:
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Keeping the promise of patient focus: Parexel leaders explain why understanding rare diseases from the perspective of patients and their caregivers and choosing patient-relevant endpoints are central to fulfilling the promise of patient-led drug development.
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Navigating regulatory pathways: Parexel regulatory luminaries — including former regulators from FDA, EMA and other major regulatory bodies — share insights on misconceptions about orphan drug designations, approaches to accelerate FDA Breakthrough Therapy designation, common pitfalls to authorization, and ways to meet patients’ needs earlier.
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Designing adaptive trials: Parexel’s scientific experts offer guidance on how to select optimal endpoints for rare diseases, how to successfully deploy complex innovative designs (CID) for rare disease trials, and how to collaborate with patient advocacy groups to design patient-focused trials.
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Clearing the way for patient access: Parexel’s market access experts share effective strategies for navigating reimbursement in the US and EU by understanding how payers and health technology assessment (HTA) agencies evaluate value.
