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W-19: Gene Therapy Clinical Trials in Rare Diseases: Considerations and Tools for Observing Delayed Adverse Events

Poster Presenter

      Amy Conroy Raymond

      • Clinical Scientist, Rare Diseases - Scientific Affairs
      • PRA Health Sciences
        United States


Patient retention and continued engagement throughout long-term follow up of gene therapy studies in rare disease indications is best supported through mobile health solutions.


A mobile health solution such as Parallel6 enables continued contact with the rare disease patient for the duration of safety surveillance, with any device and at any distance.


New technologies for gene editing and gene therapy are being brought to the clinic, offering exciting possibilities to treat and cure otherwise intractable diseases. The clinical research community is committed to supporting the FDA mission to protect the public health by ensuring the safety, efficacy, and security of these novel drugs and treatments, including complying with the FDA Guidance on Observing Subjects for Delayed Adverse Events in Gene Therapy Trials. While complying with this regulatory guidance is essential, it can also pose a barrier to enrolling and retaining study participants, especially for rare disease patient populations for whom access to the study sites can be a significant hurdle. A remote surveillance technique can prevent this hurdle from becoming insurmountable.


Just as new molecular technology drives these exciting regenerative medicine advanced therapy studies, new digital technologies can relieve some of the unintentional burdens imposed on participants and research sites, leading to a more robust clinical data and enhanced patient safety surveillance.

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