On-Demand Webinar: Natural History Study for Ultra-Rare Diseases

Were you able to join Veristat’s live webinar- “Strategies for Solving the Challenges of an Extraordinary Natural History Study – Navigating the Patient Experience When Pivoting to a Virtual Model Mid-Study”? 

The webinar featured Rachel Smith and Kara Langer as they took a deep dive into a real-life case study of an ASPA Therapeutics clinical trial for Canavan disease. Ultra-rare disease research is an exceedingly difficult area of study given the small sample sizes, limited knowledge of disease progression, access to patients
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, and more. Factor in a pandemic and the need to switch to a virtual trial during the data gathering phase,  and the challenges grow exponentially. The webinar showcases how Veristat in partnership with ASPA was able to successfully address these hurdles with powerful solutions developed specifically for the virtual trial environment.
 

Learning points:

  • The need for and challenges of a Natural History study of Canavan Disease
  • Case Study: Strategies for addressing challenges of a unique study design
  • Pivoting to a virtual & in-person hybrid model during COVID-19

Click here to watch a replay of this webinar.


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Meet the Veristat team:

 

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Rachel Smith

Portfolio Director, Veristat

Rachel has worked in clinical research for over 10 years working across all clinical phases (I-IV) with a focus on complex advanced therapy trials. She’s worked closely with innovative biopharma across different therapeutic areas. Rachel is currently the Portfolio Director of global cell and gene therapy programs, and the lead for the Veristat Global Cell and Gene Center of Excellence. She has a BSc (Hons) Biochemistry from the University of Warwick, is a Registered Member of the Institute of Clinical Research and has been published in the European Journal of Drug Metabolism and Pharmacokinetics and Lambert Academic publishing.

 

Kara Langer_HeadshotKara Langer

Senior Project Manager, Veristat

Kara is a clinical operations professional with 13 years’ experience, specializing in rare disease, gene therapy, COVID-19, and Oncology. Prior to Veristat, Kara held several roles at Covance before becoming a Project Manager. Kara is currently a Senior Project Manager at Veristat, dedicated to promoting subject safety, generating high-quality data, and maximizing study efficiency. She has a Bachelor of Science from the University of Wisconsin-Madison in Natural Sciences-Genetics and Anthropology