Rachel Smith | Executive Director, Global Head of Rare Disease, CoE
Parexel
Rachel has more than 10 years of experience in all development phases of rare disease and cell and gene therapy clinical trials. Rachel is an ultra-rare disease subject matter expert with oversight of 30+ orphan programs in APAC, Europe and North America. She is a specialist in clinical development strategy, novel and adaptive trial design, decentralized trials and creative solutions for rare disease and products with a non-classical route to market. Prior to joining Parexel, Rachel was VP European Clinical Operations, Global Head of Cell & Gene Center of Excellence and Portfolio Director in Rare Disease at Veristat LLC
Appearances:
Day 1- World Orphan Drug Congress USA 2025 @ 09:45
Keynote: Vision 2030: Charting a bright future for rare medicine
Discussion to focus on immediate outcomes of Rare Disease Innovation Hub as a new model of collaboration between CDER and CBER. While the extent to which this initiative will translate into influencing policy or decision making is unknown, it has the potential significance on the scale of Patient Focused Drug Development 2.0 – representing an opportunity for a systematic approach to prioritize and streamline rare disease drug development. with. Discussion sub-topics:
- Vision for Rare Disease Innovation Hub and advice for the RD community
- Impacts to endpoint assessments, trial design, patient recruitment and development efficiency
Day 1- World Orphan Drug Congress USA 2025 @ 11:05
Panel: Where technology meets Human: Redefining patient personalisation
We will discuss what technology can bring to healthcare from three perspectives: patient, pharma and healthcare. How AI gives us capabilities to shine a new lens on understanding patients and the personalisation of care. Uncovering its impact on decision-making for all stakeholders.
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