Leveraging sensor-based functional outcomes to enhance understanding of the patient experience: challenges and opportunities

References

• Zvonareva O, Craveț C, Richards DP. Practices of patient engagement in drug development: a systematic scoping review. Res Involv Engagem. 2022 Jun 29;8(1):29. doi: 10.1186/s40900-022-00364-8
   PubMedGoogle Scholar
• Tegenge MA, Moncur MM, Sokolic R, et al. Advancing the science of patient input throughout the regulatory decision-making process. Learn Health Syst. 2017 Jul;1(3):e10032. doi: 10.1002/lrh2.10032
   PubMedGoogle Scholar
• Forsythe LP, Carman KL, Szydlowski V, et al. Patient engagement in research: early findings from the patient-centered outcomes research institute. Health Aff. 2019 Mar;38(3):359–367. doi: 10.1377/hlthaff.2018.05067
   PubMed Web of Science ®Google Scholar
• US Food and Drug Administration. Clinical outcome assessments (COAs) in medical device decision making [Internet]. 2023 Oct 3 [cited 2023 Nov 17]. Available from: https://www.fda.gov/about-fda/cdrh-patient-science-and-engagement-program/clinical-outcome-assessments-coas-medical-device-decision-making
   Google Scholar
• US Food and Drug Administration. Patient-focused drug development glossary [Internet]. 2018 Jun 8 [cited 2024 Feb 29]. Available from: https://www.fda.gov/drugs/development-approval-process-drugs/patient-focused-drug-development-glossary
   Google Scholar
• US Food and Drug Administration. Digital health technologies for remote data acquisition in clinical investigations [Internet]. 2023 Dec [cited 2024 Feb 4]. Available from: https://www.fda.gov/media/155022/download
   Google Scholar
• Walton MK, Cappelleri JC, Byrom B, et al. Considerations for development of an evidence dossier to support the use of mobile sensor technology for clinical outcome assessments in clinical trials. Contemp Clin Trials. 2020 Apr;91:105962. doi: 10.1016/j.cct.2020.105962
   PubMed Web of Science ®Google Scholar
• Izmailova ES, AbuAsal B, Hassan HE, et al. Digital technologies: innovations that transform the face of drug development. Clin Transl Sci. 2023 Aug;16(8):1323–1330. doi: 10.1111/cts.13533
   PubMed Web of Science ®Google Scholar
• European Medicines Agency. Qualification of digital technology-based methodologies to support approval of medicinal products. 2020 Jun 1 [cited 2023 Nov 17]. Available from: https://www.ema.europa.eu/en/documents/other/questions-answers-qualification-digital-technology-based-methodologies-support-approval-medicinal_en.pdf
   Google Scholar
• US Food and Drug Administration. Clinical outcome assessment (COA) qualification program [Internet]. 2023 Oct 27 [cited 2024 Jan 5]. Available from: https://www.fda.gov/drugs/drug-development-tool-ddt-qualification-programs/clinical-outcome-assessment-coa-qualification-program
   Google Scholar
• Critical Path Institute. 14th annual patient-reported outcome consortium workshop [Internet]. 2023 [cited 2024 Jan 5]. Available from: https://c-path.org/14th-annual-patient-reported-outcome-consortium-workshop
   Google Scholar
• US Food and Drug Administration; National Institutes of Health Biomarker Working Group. BEST (Biomarkers, Endpoints, and other Tools) resource [Internet]. 2021 Nov 29 [cited 2024 Jan 5]. Available from: https://www.ncbi.nlm.nih.gov/books/NBK326791
   Google Scholar
• Burrell A, Zrubka Z, Champion A, et al. How useful are digital health terms for outcomes research? An ISPOR special interest group report. Value Health. 2022 Sep;25(9):1469–1479. doi: 10.1016/j.jval.2022.04.1730
   PubMed Web of Science ®Google Scholar
• Leyens L, Northcott CA, Maloney L, et al. Why language matters in digital endpoint development: harmonized terminology as a key prerequisite for evidence generation. Digit Biomark. 2024;8(1):1–12. doi: 10.1159/000534954
   PubMedGoogle Scholar
• Campbell CM, Webster C, Parisi M, et al. An aligned framework of actively collected and passively monitored clinical outcome assessments (COAs) for measure selection. NPJ Digit Med. 2024 Mar;7(1):71. doi: 10.1038/s41746-024-01068-x
   PubMed Web of Science ®Google Scholar
• Digital Medicine Society. The playbook [Internet]. 2023 [cited 2023 Nov 22]. Available from: https://playbook.dimesociety.org/playbooks/the-playbook
   Google Scholar
• Studenski S, Perera S, Patel K, et al. Gait speed and survival in older adults. JAMA. 2011 Jan;305(1):50–58. doi: 10.1001/jama.2010.1923
   PubMed Web of Science ®Google Scholar
• US Food and Drug Administration. Accelerated approval [Internet]. 2023 Feb 24 [cited 2024 Feb 28]. Available from: https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/accelerated-approval
   Google Scholar
• Vasudevan S, Saha A, Tarver ME, et al. Digital biomarkers: convergence of digital health technologies and biomarkers. NPJ Digit Med. 2022 Mar;5(1):36. doi: 10.1038/s41746-022-00583-z
   PubMed Web of Science ®Google Scholar
• US Food and Drug Administration. Patient-focused drug development: collecting comprehensive and representative input [Internet]. 2020 Jun [cited 2023 Nov 17]. Available from: https://www.fda.gov/media/139088/download
   Google Scholar
• US Food and Drug Administration. Patient-focused drug development: methods to identify what is important to patients [Internet]. 2022 Feb [cited 2023 Nov 17]. Available from: https://www.fda.gov/media/131230/download
   Google Scholar
• US Food and Drug Administration. Patient-focused drug development: selecting, developing, or modifying fit-for-purpose clinical outcomes assessments [Internet]. 2022 Jun. Available from: https://www.fda.gov/media/159500/download
   Google Scholar
• US Food and Drug Administration. Patient-focused drug development: incorporating clinical outcome assessments into endpoints for regulatory decision-making [Internet]. 2023 Apr [cited 2023 Nov 30]. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-focused-drug-development-incorporating-clinical-outcome-assessments-endpoints-regulatory
   Google Scholar
• Byrom B, Watson C, Doll H, et al. Selection of and evidentiary considerations for wearable devices and their measurements for use in regulatory decision making: recommendations from the ePRO Consortium. Value Health. 2018;21(6):631–639. doi: 10.1016/j.jval.2017.09.012
   PubMed Web of Science ®Google Scholar
• Clinical Trials Transformation Initiative. Developing novel endpoints generated by digital health technology for use in clinical trials. 2017 [cited 2023 Nov 22]. Available from: https://ctti-clinicaltrials.org/wp-content/uploads/2022/03/CTTI-Digital-Health-Trials-Novel-Endpoint-Acceptance-Recommendations.pdf
   Google Scholar
• Clinical Trials Transformation Initiative. Considerations for advancing the use of digital technologies for data capture & improved clinical trials. 2018 [cited 2023 Nov 22]. Available from: https://ctti-clinicaltrials.org/wp-content/uploads/2021/06/CTTI_Digital_Health_Technologies_Recs.pdf
   Google Scholar
• Digital Medicine Society. Driving adoption of nocturnal scratch as a digital endpoint and improving patients’ lives[Internet]. 2023 [cited 2024 Feb 26]. Available from: https://datacc.dimesociety.org/digital-measures-nocturnal-scratch
   Google Scholar
• Cesnakova L, Meadows K, Avey S, et al. A patient-centred conceptual model of nocturnal scratch and its impact in atopic dermatitis: a mixed-methods study supporting the development of novel digital measurements. Skin Health Dis. 2023;3(5):e262. doi: 10.1002/ski2.262
   PubMedGoogle Scholar
• Müller M, Cosman J, Adams J, et al. A progress update on the critical path for Parkinson’s consortium’s pre-competitive 3DT initiative [abstract]. 2022 [cited 2024 Feb 28]. Available from: https://www.mdsabstracts.org/abstract/a-progress-update-on-the-critical-path-for-parkinsons-consortiums-pre-competitive-3dt-initiative
   Google Scholar
• Mammen JR, Speck RM, Stebbins GM, et al. Mapping relevance of digital measures to meaningful symptoms and impacts in early Parkinson’s disease. J Parkinsons Dis. 2023;13(4):589–607. doi: 10.3233/jpd-225122
   PubMedGoogle Scholar
• TransCelerate Biopharma Inc. Patient technology implementation framework [Internet]. 2023 [cited 2023 Nov 22]. Available from: https://www.transceleratebiopharmainc.com/assets/patient-technology-implementation-framework
   Google Scholar
• Gwaltney CM, Trudeau J, Amchin W, et al. Endpoint construction from activity monitor data: chronic heart failure [Internet]. 2019 [cited 2023 Nov 22]. Available from: https://c-path.org/wp-content/uploads/2019_session4_endpointconstwearables-chf_final.pdf
   Google Scholar
• Manta C, Patrick-Lake B, Goldsack JC. Digital measures that matter to patients: a framework to guide the selection and development of digital measures of health. Digit Biomark. 2020 Sep;4:69–77. doi: 10.1159/000509725
   PubMedGoogle Scholar