Last week, the Institute for Gene Therapies (IGT) sent a response to the Health Resources and Services Administration (HRSA) Request for Information (RFI) on Nomination and Evidence-Based Review Process of the Advisory Committee on Heritable Disorders in Newborns and Children.
“IGT supports efforts to realize the value of transformative therapies for patients, caregivers, the healthcare system, and society at large. More specifically, IGT advocates for efforts that mitigate unnecessary misdiagnoses, optimize outcomes, and accelerate new cures development, and the modernization of newborn screening (NBS) and genetic testing helps achieve those aims. Enhanced access is needed to screening tests that can facilitate diagnosis, monitoring, and treatment, which are all critical for patients with rare and serious diseases.
“Importantly, it is imperative for the Committee to recognize that diagnosis must keep pace with innovation in genomic medicine, as failure to do so will limit the ability to deliver these transformative treatments to patients in a timely manner to ensure maximum benefit.”