Patient-Centered Identification of Meaningful Regulatory Endpoints for Medical Devices to Treat Parkinson's Disease.

Autor:	Benz HL; US FDA Center for Devices and Radiological Health, Silver Spring, Maryland., Caldwell B; US FDA Center for Devices and Radiological Health, Silver Spring, Maryland., Ruiz JP; US FDA Center for Devices and Radiological Health, Silver Spring, Maryland., Saha A; US FDA Center for Devices and Radiological Health, Silver Spring, Maryland., Ho M; US FDA Center for Devices and Radiological Health, Silver Spring, Maryland., Christopher S; Medical Device Innovation Consortium, Arlington, Virginia., Bardot D; Medical Device Innovation Consortium, Arlington, Virginia., Sheehan M; The Michael J. Fox Foundation for Parkinson's Research, New York, New York., Donnelly A; The Michael J. Fox Foundation for Parkinson's Research, New York, New York., McLaughlin L; The Michael J. Fox Foundation for Parkinson's Research, New York, New York., Mange B; RTI Health Solutions, Research Triangle Park, North Carolina., Hauber AB; RTI Health Solutions, Research Triangle Park, North Carolina., Gwinn K; US FDA Center for Devices and Radiological Health, Silver Spring, Maryland., Heetderks WJ; US FDA Center for Devices and Radiological Health, Silver Spring, Maryland., Sheldon M; US FDA Center for Devices and Radiological Health, Silver Spring, Maryland.
Jazyk:	angličtina
Zdroj:	MDM policy & practice [MDM Policy Pract] 2021 Jul 02; Vol. 6 (1), pp. 23814683211021380. Date of Electronic Publication: 2021 Jul 02 (Print Publication: 2021).
DOI:	10.1177/23814683211021380
Abstrakt:	Introduction. A growing literature has developed on identifying outcomes that matter to patients. This study demonstrates an approach involving patient and regulatory perspectives to identify outcomes that are meaningful in the context of medical devices for Parkinson's disease (PD). Methods. A systematic process was used for specifying relevant regulatory endpoints by synthesizing inputs of various sources and stakeholders. First, a literature review was conducted to identify important benefits, risks, and other considerations for medical devices to treat PD; patient discussion groups ( n = 6) were conducted to refine the list of considerations, followed by a survey ( n = 29) to prioritize them; and patient and Food and Drug Administration (FDA) reviewers informed specification of the final endpoints. Two FDA clinicians gave clinical and regulatory perspectives at each step. Results. Movement symptoms were ranked as most important (ranked 1 or 2 by 72% of participants) and psychological and cognitive symptoms as the next most important (ranked 1 or 2 by 52% of participants). Within movement symptoms, falls, impaired movement, bradykinesia, resting tremor, stiffness, and rigidity were ranked highly. Overall, nine attributes were identified and prioritized as patient-centric for use in clinical trial design and quantitative patient preference studies. These attributes were benefits and risks related to therapeutics for PD as well as other considerations, including time until a medical device is available for patient use. Discussion. This prospective approach identified meaningful and relevant benefits, risks, and other considerations that may be used for clinical trial design and quantitative patient preference studies. Although PD was the focus of this study, the approach can be used to study patient perspectives about other disease or treatment areas. Competing Interests: The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: BC, AS, MH, M Sheldon, AD, LM, BM, AH, KG, WH, and M Sheehan have no conflicts of interest to report. HB is employed at Johnson and Johnson; during her contributions to the work, she was employed by the US Food and Drug Administration. JR is employed at Dataprise; during his contributions to the work, he was employed by the US Food and Drug Administration. SC is employed at Currently at National Organization for Rare Disorders (NORD); during her contributions to the work, she was employed by the nonprofit Medical Device Innovation Consortium. DB is employed by Abiomed; during her contributions to the work, she was employed by the nonprofit Medical Device Innovation Consortium BH is currently employed at Pfizer Inc.; during his contributions to the work, he was employed by RTI Health Solutions. (© The Author(s) 2021.)
Databáze:	MEDLINE
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