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Natural History Data and the Path to Registration in Pediatric Rare Disease Research

Natural History Data and the Path to Registration in Pediatric Rare Disease Research

Date: Tuesday, March 30, 2021
Time: 11am EDT(NA) / 4pm BST (UK) / 5pm CEST (EU-Central)
Length: 60 minutes

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As the clinical research industry experiences a growing focus on rare diseases, particularly on gene therapy approaches to rare, genetic disorders, the need for access to or collection of natural history data becomes critical. The FDA and other global regulatory agencies recognize that while inclusion of a randomized, concurrent control group is ideal, this approach is not always possible, particularly in pediatric rare disease research. In studies requiring high-risk procedures and/or indications that are rapidly progressive and severe, it may not be considered practical or ethical to include a placebo arm. In these cases, the agencies recognize that an historical control may be appropriate.

What is the best way to access or collect these vital data in a vulnerable population? Are biopharma companies and academic researchers the only groups that can run this type of study? What if there is natural history data available for a disease, but the group collecting it isn’t willing to share it? For pediatric rare diseases, these questions are paramount and should be discussed very early in the clinical development planning process, ideally even before a potential therapy is developed. As is true for all rare disease research, there are logistical challenges to gathering natural history data, and a clinical program may hinge on the existence and robustness of this dataset.

Register for this webinar to join Jess Conicelli, Synteract’s Executive Director, Strategic Development, Rare, Orphan, and Pediatric Diseases on Tuesday, March 30, at 11:00 AM EDT as she discusses natural history studies and their place in the pediatric rare disease clinical development plan.

After the webinar, participants will have a better understanding of natural history data collection and how these datasets can be used in pediatric rare disease research.

Speaker:

  • Jess Conicelli, Executive Director, Strategic Development, Rare, Orphan, & Pediatric Diseases, Synteract


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Synteract General Medicince Clinical Trial Leadership

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