数字测量的评估、验收和鉴定：从概念验证到终点指标
Evaluation, Acceptance, and Qualification of Digital Measures: From Proof of Concept to Endpoint.
作者信息
Goldsack Jennifer C, Dowling Ariel V, Samuelson David, Patrick-Lake Bray, Clay Ieuan
机构信息
Digital Medicine Society (DiMe), Boston, Massachusetts, USA.
Takeda Pharmaceuticals Inc., Cambridge, Massachusetts, USA.
出版信息
Digit Biomark. 2021 Mar 23;5(1):53-64. doi: 10.1159/000514730. eCollection 2021 Jan-Apr.
Abstract
To support the successful adoption of digital measures into internal decision making and evidence generation for medical product development, we present a unified lexicon to aid communication throughout this process, and highlight key concepts including the critical role of participant engagement in development of digital measures. We detail the steps of bringing a successful proof of concept to scale, focusing on key decisions in the development of a new digital measure: asking the right question, optimized approaches to evaluating new measures, and whether and how to pursue qualification or acceptance. Building on the V3 framework for establishing verification and analytical and clinical validation, we discuss strategic and practical considerations for collecting this evidence, illustrated with concrete examples of trailblazing digital measures in the field.
摘要
为支持将数字措施成功应用于医疗产品开发的内部决策和证据生成，我们提出了一个统一的词汇表，以协助整个过程中的沟通，并强调关键概念，包括参与者参与数字措施开发的关键作用。我们详细介绍了将成功的概念验证扩大规模的步骤，重点关注新数字措施开发中的关键决策：提出正确的问题、评估新措施的优化方法，以及是否以及如何寻求认证或认可。在建立验证以及分析和临床验证的V3框架基础上，我们讨论收集此类证据的战略和实际考虑因素，并列举该领域开创性数字措施的具体示例加以说明。
相似文献
1
Evaluation, Acceptance, and Qualification of Digital Measures: From Proof of Concept to Endpoint.
Digit Biomark. 2021 Mar 23;5(1):53-64. doi: 10.1159/000514730. eCollection 2021 Jan-Apr.
2
From Meaningful Outcomes to Meaningful Change Thresholds: A Path to Progress for Establishing Digital Endpoints.
Ther Innov Regul Sci. 2023 Jul;57(4):629-645. doi: 10.1007/s43441-023-00502-8. Epub 2023 Apr 5.
3
Patient-centricity in digital measure development: co-evolution of best practice and regulatory guidance.
NPJ Digit Med. 2024 May 16;7(1):128. doi: 10.1038/s41746-024-01110-y.
4
Unlocking the full potential of digital endpoints for decision making: a novel modular evidence concept enabling re-use and advancing collaboration.
Expert Rev Pharmacoecon Outcomes Res. 2024 Jul;24(6):731-741. doi: 10.1080/14737167.2024.2334347. Epub 2024 May 15.
5
Why Language Matters in Digital Endpoint Development: Harmonized Terminology as a Key Prerequisite for Evidence Generation.
Digit Biomark. 2024 Jan 11;8(1):1-12. doi: 10.1159/000534954. eCollection 2024 Jan-Dec.
6
EVIDENCE Publication Checklist for Studies Evaluating Connected Sensor Technologies: Explanation and Elaboration.
Digit Biomark. 2021 May 18;5(2):127-147. doi: 10.1159/000515835. eCollection 2021 May-Aug.
7
Strategic approach to fit-for-purpose biomarkers in drug development.
Annu Rev Pharmacol Toxicol. 2008;48:631-51. doi: 10.1146/annurev.pharmtox.48.113006.094611.
8
Reverse Engineering of Digital Measures: Inviting Patients to the Conversation.
Digit Biomark. 2023 May 12;7(1):28-44. doi: 10.1159/000530413. eCollection 2023 Jan-Dec.
9
Evaluation of Speech-Based Digital Biomarkers: Review and Recommendations.
Digit Biomark. 2020 Oct 19;4(3):99-108. doi: 10.1159/000510820. eCollection 2020 Sep-Dec.
10
Developing a Novel Measurement of Sleep in Rheumatoid Arthritis: Study Proposal for Approach and Considerations.
Digit Biomark. 2021 Sep 2;5(3):191-205. doi: 10.1159/000518024. eCollection 2021 Sep-Dec.
引用本文的文献
1
Automatic speech analysis combined with machine learning reliably predicts the motor state in people with Parkinson's disease.
NPJ Parkinsons Dis. 2025 May 2;11(1):105. doi: 10.1038/s41531-025-00959-4.
2
V3+ extends the V3 framework to ensure user-centricity and scalability of sensor-based digital health technologies.
NPJ Digit Med. 2025 Jan 24;8(1):51. doi: 10.1038/s41746-024-01322-2.
3
An automatic measure for speech intelligibility in dysarthrias-validation across multiple languages and neurological disorders.
Front Digit Health. 2024 Jul 23;6:1440986. doi: 10.3389/fdgth.2024.1440986. eCollection 2024.
4
Digital endpoints in clinical trials: emerging themes from a multi-stakeholder Knowledge Exchange event.
Trials. 2024 Aug 3;25(1):521. doi: 10.1186/s13063-024-08356-7.
5
A coating machine for coating filaments with bioactive nanomaterials for extrusion 3D printing.
Heliyon. 2024 Jun 21;10(12):e33223. doi: 10.1016/j.heliyon.2024.e33223. eCollection 2024 Jun 30.
6
Do Measures of Real-World Physical Behavior Provide Insights Into the Well-Being and Physical Function of Cancer Survivors? Cross-Sectional Analysis.
JMIR Cancer. 2024 Jul 15;10:e53180. doi: 10.2196/53180.
7
From wearable sensor data to digital biomarker development: ten lessons learned and a framework proposal.
NPJ Digit Med. 2024 Jun 18;7(1):161. doi: 10.1038/s41746-024-01151-3.
8
Patient-centricity in digital measure development: co-evolution of best practice and regulatory guidance.
NPJ Digit Med. 2024 May 16;7(1):128. doi: 10.1038/s41746-024-01110-y.
9
Improved measurement of disease progression in people living with early Parkinson's disease using digital health technologies.
Commun Med (Lond). 2024 Mar 15;4(1):49. doi: 10.1038/s43856-024-00481-3.
10
Deep Learning-Based Psoriasis Assessment: Harnessing Clinical Trial Imaging for Accurate Psoriasis Area Severity Index Prediction.
Digit Biomark. 2024 Mar 4;8(1):13-21. doi: 10.1159/000536499. eCollection 2024 Jan-Dec.
本文引用的文献
1
Defining and Developing the Workforce Needed for Success in the Digital Era of Medicine.
Digit Biomark. 2020 Nov 26;4(Suppl 1):136-142. doi: 10.1159/000512382. eCollection 2020 Winter.
2
The Collaborative Aging Research Using Technology Initiative: An Open, Sharable, Technology-Agnostic Platform for the Research Community.
Digit Biomark. 2020 Nov 26;4(Suppl 1):100-118. doi: 10.1159/000512208. eCollection 2020 Winter.
3
Predicting Subjective Recovery from Lower Limb Surgery Using Consumer Wearables.
Digit Biomark. 2020 Nov 26;4(Suppl 1):73-86. doi: 10.1159/000511531. eCollection 2020 Winter.
4
Assessment of Fatigue Using Wearable Sensors: A Pilot Study.
Digit Biomark. 2020 Nov 26;4(Suppl 1):59-72. doi: 10.1159/000512166. eCollection 2020 Winter.
5
Precompetitive Consensus Building to Facilitate the Use of Digital Health Technologies to Support Parkinson Disease Drug Development through Regulatory Science.
Digit Biomark. 2020 Nov 26;4(Suppl 1):28-49. doi: 10.1159/000512500. eCollection 2020 Winter.
6
A Roadmap to Inform Development, Validation and Approval of Digital Mobility Outcomes: The Mobilise-D Approach.
Digit Biomark. 2020 Nov 26;4(Suppl 1):13-27. doi: 10.1159/000512513. eCollection 2020 Winter.
7
The Path Forward for Digital Measures: Suppressing the Desire to Compare Apples and Pineapples.
Digit Biomark. 2020 Nov 26;4(Suppl 1):3-12. doi: 10.1159/000511586. eCollection 2020 Winter.
8
Digital Measures That Matter to Patients: A Framework to Guide the Selection and Development of Digital Measures of Health.
Digit Biomark. 2020 Sep 15;4(3):69-77. doi: 10.1159/000509725. eCollection 2020 Sep-Dec.
9
Developing Smartphone-Based Objective Assessments of Physical Function in Rheumatoid Arthritis Patients: The PARADE Study.
Digit Biomark. 2020 Apr 30;4(1):26-43. doi: 10.1159/000506860. eCollection 2020 Jan-Apr.
10
A digital health industry cohort across the health continuum.
NPJ Digit Med. 2020 May 12;3:68. doi: 10.1038/s41746-020-0276-9. eCollection 2020.
Zotero 插件