Document Type
Article
Department
Internal Medicine (East Africa)
Abstract
Background Over the past decade, neuropsychopharmacology has shifted from stagnation to momentum, with first-in-class mechanisms and biomarker-enabled trials spanning psychiatry and neurology.
Methods We narratively synthesized advances from 2013 to 2026 across central nervous system (CNS) discovery and development, including pivotal trials, regulatory actions, digital/real-world evidence, genetics, artificial intelligence (AI), and implementation/global-access themes that are endorsed by international societies.
Results Therapeutic gains include rapid-acting drugs for treatment-resistant depression (intranasal esketamine); psychedelic-assisted therapy for posttraumatic stress disorder and depression; neuroactive steroid γ-aminobutyric acid-A receptor positive allosteric modulators (brexanolone, zuranolone) for postpartum depression; non-dopaminergic muscarinic agonists (xanomeline–trospium) for schizophrenia; orexin receptor antagonists for insomnia; and anti-amyloid monoclonal antibodies (lecanemab, donanemab) for early Alzheimer’s disease. Persistent barriers include high mid-/late-stage attrition that is driven by placebo effects, subjective endpoints, and preclinical-to-clinical gaps; regulatory and economic headwinds; and limited generalizability from tightly run trials. Emerging enablers include adaptive/platform designs, digital health technologies, patient-reported outcomes, and clinical outcome assessments, real-world evidence (RWE), AI/machine learning (ML), genetics for target de-risking and biomarker-guided stratification, and publicly accessible large CNS relevant biological datasets.
Conclusions To convert momentum into durable progress, we recommend: (i) deeper academia–industry/stakeholder collaboration and sustained funding for high-risk/high-reward science from industry, governments and non-for profit foundations; (ii) modernized regulation (flexible evidentiary paths, novel endpoints, and clear guidance on adaptive/platform trials); (iii) data-driven development integrating RWE, AI/ML, and precision medicine; (iv) the adoption of Neuroscience-based Nomenclature (NbN); and (v) a global-access mandate with essential-medicine inclusion, equitable pricing/licensing, capacity building, tele-enabled mental health, and geographically diverse research. Aligning scientific innovation with implementation and equity can accelerate translation and ensure new treatments benefit patients worldwide.
Publication (Name of Journal)
The international journal of neuropsychopharmacology
DOI
https://doi.org/10.1093/ijnp/pyag023
Recommended Citation
Uchida, H.,
Gobbi, G.,
Zohar, J.,
Young, A.,
Rujescu, D.,
Huang, M.,
Sundram, S.,
Atwoli, L.,
Vukovic, J.,
Ikeda, K.
(2026). Global perspectives on CNS drug innovation: Achievements, barriers, and priorities for the next decade. The international journal of neuropsychopharmacology, 1-73.
Available at:
https://ecommons.aku.edu/eastafrica_fhs_mc_intern_med/598
Creative Commons License
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License