Your Neurological Gene Therapy Trial's Secret Weapon: Caregiver Support

Gene therapies offer exciting possibilities in the treatment of devastating neurological indications. However, to successfully deliver these therapies to patients, drug sponsors must adequately prepare to accommodate the vulnerabilities of the trial participant population. The neurodegenerative and neurodevelopmental nature of these conditions presents the risk of participants losing the capacity to consent mid-trial due to rapid disease progression. To address this, sponsors must design rigorous informed consent protocols, including enlisting the support of caregivers and legally authorized representatives (LARs) who can step in to provide surrogate consent if needed.

A participant’s diminished capacity to consent and subsequent surrogate consent is an approach that should be planned for well in advance and built into a protocol. Without adequate preparation for this scenario, a study could be delayed or disrupted. Download the full article to gain insight into the different roles played by caregivers and LARs and how to prioritize compliance in your trial.