TY - JOUR
T1 - Neurologic Therapeutics in 2035
T2 - The Neurology Future Forecasting Series
AU - Johnson, Nicholas E.
AU - Greene, Ericka
N1 - Funding Information:
N.E. Johnson has received grant funding from NINDS (R01NS104010), NCATS (21TR003184), CDC (U01DD001242-02), and the FDA (7R01FD006071-02); receives royalties from the CCMDHI and the CMTHI; receives research funds from Dyne, AveXis, CSL Behring, Vertex Pharmaceuticals, Fulcrum Therapeutics, ML Bio, Sarepta, and Acceleron Pharma; and has provided consultation for ML Bio, Arthex, AveXis, AMO Pharma, Fulcrum Therapeutics, Dyne, Avidity, and Vertex Pharmaceuticals. E. Greene has received funding from GBS/CIDP Foundation and Pfizer, Inc.; is a paid consultant for CSL Behring, Inc., Alexion Pharmaceuticals, and Argenx Inc.; and is a paid speaker for Alexion Inc. and CSL Behring, Inc., for FDA-approved therapies. Go to Neurology.org/N for full disclosures.
Publisher Copyright:
© American Academy of Neurology.
PY - 2021/12/14
Y1 - 2021/12/14
N2 - 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.
AB - 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.
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U2 - 10.1212/WNL.0000000000012976
DO - 10.1212/WNL.0000000000012976
M3 - Article
C2 - 34903613
AN - SCOPUS:85121912636
SN - 0028-3878
VL - 97
SP - 1121
EP - 1127
JO - Neurology
JF - Neurology
IS - 24
ER -