11 min read
Monthly FDA Guidance and Regulatory News Review - June 2026
This June 2026 regulatory update focuses on advancing innovative drug development through updated guidance on...
1 min read
Meet Veristat at the Orphan Drug Congress 2021 taking place on 15-18 November 2021, Booth #54
Whether you have a compound and need to get started on your IND, or want to recruit patients quickly to get your early phase trials running or just need medical writing or biostatistical support for your programs - Veristat can help. We provide impactful solutions on both a full-service or functional service basis.
Schedule A Meeting
Schedule a meeting by filling out the form on the right. Find out how we can help you advance your next clinical trial or regulatory submission to successful outcomes. Or learn more about our capabilities.
Veristat Talk at the Congress
Wednesday 17th November/ 11.40 a.m
" The Role of Natural History Studies in Ultra-Rare Disease Trials– Takeaways from a Canavan Disease Gene Therapy Trial on Their Challenges, Benefits and Successes"
Speakers:
John Balser, PhD, President and Chief Biostatistician, Veristat
Rachel Smith, Portfolio Director, Veristat
Abstract:
Completing a natural history study in conjunction with an interventional study for ultra-rare diseases can be critical to understanding the disease and its progression. The data from the natural history can be essential to informing the design and outcome selection of the interventional study. We explore the challenges of setting up a natural history study with both retrospective and prospective data to develop a new rating scale for disease progression in Canavan disease, a rare and relatively unknown childhood disease.
Our experts will share their experience, successes, and lessons learned on :
- Considerations for Setting Up Natural History Studies
- Utilizing Natural History Data to Support Interventional Study Outcomes
- Review of Special Considerations for Rare Gene Therapies
- Case Study with Canavan Disease Natural History Study Conducted
Virtual Conference Details
Website: https://www.terrapinn.com/conference/world-orphan-drug-congress/index.stm