Patient-Centered Identification of Meaningful Regulatory Endpoints for Medical Devices to Treat Parkinson’s Disease
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 syst...
| Main Authors: | , , , , , , , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
SAGE Publishing
2021-06-01
|
| Series: | MDM Policy & Practice |
| Online Access: | https://doi.org/10.1177/23814683211021380 |
| _version_ | 1819136157962731520 |
|---|---|
| author | Heather L. Benz Brittany Caldwell John P. Ruiz Anindita Saha Martin Ho Stephanie Christopher Dawn Bardot Margaret Sheehan Anne Donnelly Lauren McLaughlin Brennan Mange A. Brett Hauber Katrina Gwinn William J. Heetderks Murray Sheldon |
| author_facet | Heather L. Benz Brittany Caldwell John P. Ruiz Anindita Saha Martin Ho Stephanie Christopher Dawn Bardot Margaret Sheehan Anne Donnelly Lauren McLaughlin Brennan Mange A. Brett Hauber Katrina Gwinn William J. Heetderks Murray Sheldon |
| author_sort | Heather L. Benz |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-12-22T10:30:31Z |
| format | Article |
| id | doaj.art-6a7002492e084b97aa87470ac6820e3a |
| institution | Directory Open Access Journal |
| issn | 2381-4683 |
| language | English |
| last_indexed | 2024-12-22T10:30:31Z |
| publishDate | 2021-06-01 |
| publisher | SAGE Publishing |
| record_format | Article |
| series | MDM Policy & Practice |
| spelling | doaj.art-6a7002492e084b97aa87470ac6820e3a2022-12-21T18:29:21ZengSAGE PublishingMDM Policy & Practice2381-46832021-06-01610.1177/23814683211021380Patient-Centered Identification of Meaningful Regulatory Endpoints for Medical Devices to Treat Parkinson’s DiseaseHeather L. BenzBrittany CaldwellJohn P. RuizAnindita SahaMartin HoStephanie ChristopherDawn BardotMargaret SheehanAnne DonnellyLauren McLaughlinBrennan MangeA. Brett HauberKatrina GwinnWilliam J. HeetderksMurray SheldonIntroduction. 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.https://doi.org/10.1177/23814683211021380 |
| spellingShingle | Heather L. Benz Brittany Caldwell John P. Ruiz Anindita Saha Martin Ho Stephanie Christopher Dawn Bardot Margaret Sheehan Anne Donnelly Lauren McLaughlin Brennan Mange A. Brett Hauber Katrina Gwinn William J. Heetderks Murray Sheldon Patient-Centered Identification of Meaningful Regulatory Endpoints for Medical Devices to Treat Parkinson’s Disease MDM Policy & Practice |
| title | Patient-Centered Identification of Meaningful Regulatory Endpoints for Medical Devices to Treat Parkinson’s Disease |
| title_full | Patient-Centered Identification of Meaningful Regulatory Endpoints for Medical Devices to Treat Parkinson’s Disease |
| title_fullStr | Patient-Centered Identification of Meaningful Regulatory Endpoints for Medical Devices to Treat Parkinson’s Disease |
| title_full_unstemmed | Patient-Centered Identification of Meaningful Regulatory Endpoints for Medical Devices to Treat Parkinson’s Disease |
| title_short | Patient-Centered Identification of Meaningful Regulatory Endpoints for Medical Devices to Treat Parkinson’s Disease |
| title_sort | patient centered identification of meaningful regulatory endpoints for medical devices to treat parkinson s disease |
| url | https://doi.org/10.1177/23814683211021380 |
| work_keys_str_mv | AT heatherlbenz patientcenteredidentificationofmeaningfulregulatoryendpointsformedicaldevicestotreatparkinsonsdisease AT brittanycaldwell patientcenteredidentificationofmeaningfulregulatoryendpointsformedicaldevicestotreatparkinsonsdisease AT johnpruiz patientcenteredidentificationofmeaningfulregulatoryendpointsformedicaldevicestotreatparkinsonsdisease AT aninditasaha patientcenteredidentificationofmeaningfulregulatoryendpointsformedicaldevicestotreatparkinsonsdisease AT martinho patientcenteredidentificationofmeaningfulregulatoryendpointsformedicaldevicestotreatparkinsonsdisease AT stephaniechristopher patientcenteredidentificationofmeaningfulregulatoryendpointsformedicaldevicestotreatparkinsonsdisease AT dawnbardot patientcenteredidentificationofmeaningfulregulatoryendpointsformedicaldevicestotreatparkinsonsdisease AT margaretsheehan patientcenteredidentificationofmeaningfulregulatoryendpointsformedicaldevicestotreatparkinsonsdisease AT annedonnelly patientcenteredidentificationofmeaningfulregulatoryendpointsformedicaldevicestotreatparkinsonsdisease AT laurenmclaughlin patientcenteredidentificationofmeaningfulregulatoryendpointsformedicaldevicestotreatparkinsonsdisease AT brennanmange patientcenteredidentificationofmeaningfulregulatoryendpointsformedicaldevicestotreatparkinsonsdisease AT abretthauber patientcenteredidentificationofmeaningfulregulatoryendpointsformedicaldevicestotreatparkinsonsdisease AT katrinagwinn patientcenteredidentificationofmeaningfulregulatoryendpointsformedicaldevicestotreatparkinsonsdisease AT williamjheetderks patientcenteredidentificationofmeaningfulregulatoryendpointsformedicaldevicestotreatparkinsonsdisease AT murraysheldon patientcenteredidentificationofmeaningfulregulatoryendpointsformedicaldevicestotreatparkinsonsdisease |