Translational Research: Preclinical to Healthy Volunteer to Patient

Tammara, Brinda; Raje, Sangeeta; McKeand, William; Korth‐Bradley, Joan M.

doi:10.1007/978-3-319-50042-3_11

Cited by 1 publication

(1 citation statement)

References 83 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Enrolled phase 1 subjects are generally healthy volunteers, or if they have some clinical pathology : : : their condition is relatively stable, so that participation in a short clinical study does not pose a serious health threat. 28 Fifty percent of non-neuro papers and 30% of neuro* papers discussed how determinations of readiness for translation into FIH trials was impacted by whether the goal of the trial was to simply establish the safety of the intervention, or if it was to determine the efficacy of the intervention for its intended clinical purpose. Dosing was described in 32% of non-neuro papers and 26% of neuro papers.…”

Section: Fih Trial Designmentioning

confidence: 99%

Readiness for First-In-Human Neuromodulatory Interventions

McCall

Minielly

Bethune

et al. 2020

Can. J. Neurol. Sci.

View full text Add to dashboard Cite

Background: Novel neurointerventions present innovative therapeutic approaches to a range of treatment-refractory disorders. We sought to characterize factors that inform and define translational readiness for first-in-human (FIH) neuromodulatory trials. Methods: We used a two-part methodology involving a scoping review of the biomedical literature on the readiness of FIH trials for both neurological and non-neurological applications, and semi-structured interviews with stakeholders about decision-making for neuromodulation using magnetic resonance-guided focused ultrasound as a case example. Results: One hundred and thirty factors relevant to FIH readiness were identified in the scoping review. Trial design, adequacy of preclinical evidence, and risk were ubiquitous across biotechnologies. Target organ, target function, and inadequacy of animal models were dominant in the neurointervention literature. Interview results on the relative importance of these factors reveal divergent values, priorities, and understandings both between patients and clinicians and between patients affected by different conditions. Conclusion: Readiness of neurotechnology for FIH trials is defined by a multitude of interacting factors that pertain to clinical and nonclinical priorities, perceptions, and values.

show abstract

Section: Fih Trial Designmentioning

confidence: 99%