Characteristics and use of patient-reported outcomes of clinical trials for high-risk neurological medical devices that received FDA premarket approval from 2001 to 2022

Morgan E Ryan; Siddharth Srivastava; Lin Wan; Guang Yang; Bo Zhang

doi:10.1016/j.conctc.2024.101254

Characteristics and use of patient-reported outcomes of clinical trials for high-risk neurological medical devices that received FDA premarket approval from 2001 to 2022

Contemp Clin Trials Commun. 2024 Jan 2:37:101254. doi: 10.1016/j.conctc.2024.101254. eCollection 2024 Feb.

Authors

Morgan E Ryan¹, Siddharth Srivastava², Lin Wan^{3

4

5}, Guang Yang^{3

4

5

6}, Bo Zhang^{1

2}

Affiliations

¹ Biostatistics and Research Design Center, Institutional Centers for Clinical and Translational Research, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
² Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
³ Department of Pediatrics, The First Medical Center of Chinese PLA General Hospital, Beijing, China.
⁴ Senior Department of Pediatrics, The Seventh Medical Center of Chinese PLA General Hospital, Beijing, China.
⁵ Medical School of Chinese PLA, Beijing, China.
⁶ Second School of Clinical Medicine, Southern Medical University, Guangzhou, China.

Abstract

Neurological medical devices have revolutionized the management of neurological disorders, providing diagnostic, therapeutic, and monitoring solutions. High-risk neurological devices, such as deep brain stimulation and neurostimulators, offer groundbreaking treatments, emphasizing patient benefits while considering risks. To gain FDA approval, high-risk Class III devices necessitate premarket approval (PMA) applications with pivotal clinical trials, often assessing patient-reported outcomes (PROs). This article analyzes FDA-approved high-risk neurological devices from 2001 to 2022 via the PMA pathway. It explores device characteristics and pivotal clinical trials, and PRO incorporation. Of the 23 identified devices, pain neurology devices (30.4 %) predominated. All devices were therapeutic, with varying study designs. Pain neurology devices notably emphasized PRO endpoints as expected. This study underscores the significance of PROs in assessing device efficacy and safety, offering insights into regulatory processes and patient-centered care in neurological disorder management.

Keywords: FDA regulation; High-risk medical devices; Neurological disorders; Patient-reported outcomes (PROs); Pivotal clinical trials.