RTI Health Solutions research experts will be participating in the schedule of events at #DIA2023. Their programs will cover post-market pregnancy requirements and safety in rare disease and gene therapy. Here are the details.
#314: Registries Aren’t Always the Answer: Novel Designs for Executing Post-Market Pregnancy Requirements
Wednesday, June 28 • 10:30am - 11:30am
Panelist: Andrea V Margulis, DrSc, MD, FISPE of RTI Health Solutions
In this forum, experts will provide a short overview on the challenges to executing traditional pregnancy registries, alternative observational study designs and methods. The experts will then come together to have a panel discussion on the current gaps in the regulatory guidance on post-market pregnancy studies.
#362: Assessing Safety in Rare Disease and Gene Therapy
Wednesday, June 28 • 4:00pm - 5:00pm
Chair: Alicia Gilsenan, PhD, MS, RPh, FISPE of RTI Health Solutions
Gene therapies offer unprecedented opportunities to address rare diseases, but these opportunities bring new safety challenges. Specific to gene therapy for example, in addition to organ toxicities, there is the potential for transmission of a viral vector to others, or integration of the payload gene into the patient's genome with possible formation of neoplasms. These novel concerns are raised in a setting of small patient populations and the need for decades-long follow up in some cases, and thus require creative solutions to monitor and assess safety. In this one-hour session 3 speakers will discuss
- an overview of the safety challenges that are specific to gene therapy development and surveillance
- an example of a specific method (e.g., historical control or synthetic control) to assess rare disease treatment safety
- advantages and example for setting up a multi-sponsor rare disease registry.
