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Abstract
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.
Glossary
- AAN=
- American Academy of Neurology;
- AD=
- Alzheimer disease;
- ALS=
- amyotrophic lateral sclerosis;
- CGRP=
- calcitonin gene-related peptide;
- DMT=
- disease-modifying therapy;
- FDA=
- Food and Drug Administration;
- ICER=
- Institute for Clinical and Economic Review;
- MS=
- multiple sclerosis;
- SMA=
- spinal muscular atrophy
Footnotes
Go to Neurology.org/N for full disclosures. Funding information and disclosures deemed relevant by the authors, if any, are provided at the end of the article.
See page 1120
- Received March 21, 2021.
- Accepted in final form September 29, 2021.
- © 2021 American Academy of Neurology
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