Therapeutic development has accelerated rapidly in the past 5 years in many neurologic and neurodegenerative diseases. The therapeutic categories of development include small molecules, genetic therapies, and cell-based therapies. Current development has provided novel treatment approaches to disorders without available treatment. However, the regulatory procedures to allow for access to these therapies is challenging, as is the ability to provide wide access to increasingly expensive therapies. By 2035, these challenges are likely to have accelerated and have the potential to create bottlenecks in drug approval and reduced access to patients. Innovative regulatory and payer solutions are required. In addition, ethical considerations around genetic therapies should be considered in current and future development. These approaches will ensure that patients with neurologic disease have broad access to highly innovative therapies.
© 2021 American Academy of Neurology.