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

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

Jess has 15 years of clinical research experience, with much of that time uniquely focused on rare and orphan diseases. Most recently, Jess oversaw clinical operations at the University of Pennsylvania’s Orphan Disease Center and Gene Therapy Program, both housed within the Perelman School of Medicine. While at Penn, Jess worked closely with biotech companies to implement strategic clinical development initiatives and with thought-leaders to foster collaboration within the rare disease community. She oversaw vendor partnerships to create patient registries and natural history studies, and, most impactfully, she worked hand-in-hand with patient advocacy groups, providing rare disease patients and their families with data-driven guidance while accelerating the clinical research process.

Prior to Penn, Jess spent eleven years in the CRO industry with Syneos Health, formerly INC Research, where she held director level positions growing their therapeutic business units, with a specific focus in global rare disease and pediatric trials. Jess earned bachelor’s degrees in both Molecular Biology and Psychology from Meredith College in Raleigh, NC.

Message Presenter