Translational obstacles with off-label drug use in acute traumatic brain injury

Author:

Mirzaei Sara1,Reinig Andrea M1,Berlau Daniel J1

Affiliation:

1. Rueckert-Hartman College for Health Professions, Regis University, 3333 Regis Blvd, H-28, Denver, CO 80221, USA

Abstract

Traumatic brain injury results in significant morbidity and mortality, and there is an urgent need for neuroprotective medications that can prevent the persisting symptoms and disabilities following injury. Several existing pharmacotherapies have been targeted for off-label benefit in traumatic brain injury, as these agents are well characterized and commercially available, easing the process of clinical trial development. Despite promising results in animal models, clinical trials have demonstrated minimal benefit. One possible reason for these failed translations could be that drug selection, characterization and dosing are not routinely established in the appropriate early phase trials before larger scale testing. Examining how recent trials may have bypassed these steps may help future trials to more definitively determine the efficacy of potential therapeutics.

Publisher

Future Medicine Ltd

Subject

Clinical Neurology,Neurology

Reference64 articles.

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2. Guidelines for the Management of Severe Traumatic Brain Injury, Fourth Edition

3. US Food and Drug Association. ‘Off-Label’ and Investigational Use of Marketed Drugs, Biologics, and Medical Devices – Information Sheet (2016). www.fda.gov/RegulatoryInformation/Guidances/ucm126486.htm.

4. Investigational New Drug Application, 21 C.F.R. § 312.2 (2016).www.ecfr.gov/cgi-bin/text-idx?SID=e68e797bc4fc55f298e2d4574fe27202&mc=true&node=se21.5.312_12&rgn=div8.

5. The neuropathology of traumatic brain injury

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