Diane Stephenson, Robert Alexander, Varun Aggarwal, Reham Badawy, Lisa Bain, Roopal Bhatnagar, Bastiaan R Bloem, Babak Boroojerdi, Jackson Burton, Jesse M Cedarbaum, Josh Cosman, David T Dexter, Marissa Dockendorf, E Ray Dorsey, Ariel V Dowling, Luc J W Evers, Katherine Fisher, Mark Frasier, Luis Garcia-Gancedo, Jennifer C Goldsack, Derek Hill, Janice Hitchcock, Michele T Hu, Michael P Lawton, Susan J Lee, Michael Lindemann, Ken Marek, Nitin Mehrotra, Marjan J Meinders, Michael Minchik, Lauren Oliva, Klaus Romero, George Roussos, Robert Rubens, Sakshi Sadar, Joseph Scheeren, Eiichi Sengoku, Tanya Simuni, Glenn Stebbins, Kirsten I Taylor, Beatrice Yang, Neta Zach
{"title":"建立竞争前共识,通过监管科学促进数字健康技术的使用,支持帕金森病药物开发。","authors":"Diane Stephenson, Robert Alexander, Varun Aggarwal, Reham Badawy, Lisa Bain, Roopal Bhatnagar, Bastiaan R Bloem, Babak Boroojerdi, Jackson Burton, Jesse M Cedarbaum, Josh Cosman, David T Dexter, Marissa Dockendorf, E Ray Dorsey, Ariel V Dowling, Luc J W Evers, Katherine Fisher, Mark Frasier, Luis Garcia-Gancedo, Jennifer C Goldsack, Derek Hill, Janice Hitchcock, Michele T Hu, Michael P Lawton, Susan J Lee, Michael Lindemann, Ken Marek, Nitin Mehrotra, Marjan J Meinders, Michael Minchik, Lauren Oliva, Klaus Romero, George Roussos, Robert Rubens, Sakshi Sadar, Joseph Scheeren, Eiichi Sengoku, Tanya Simuni, Glenn Stebbins, Kirsten I Taylor, Beatrice Yang, Neta Zach","doi":"10.1159/000512500","DOIUrl":null,"url":null,"abstract":"<p><p>Innovative tools are urgently needed to accelerate the evaluation and subsequent approval of novel treatments that may slow, halt, or reverse the relentless progression of Parkinson disease (PD). Therapies that intervene early in the disease continuum are a priority for the many candidates in the drug development pipeline. There is a paucity of sensitive and objective, yet clinically interpretable, measures that can capture meaningful aspects of the disease. This poses a major challenge for the development of new therapies and is compounded by the considerable heterogeneity in clinical manifestations across patients and the fluctuating nature of many signs and symptoms of PD. Digital health technologies (DHT), such as smartphone applications, wearable sensors, and digital diaries, have the potential to address many of these gaps by enabling the objective, remote, and frequent measurement of PD signs and symptoms in natural living environments. The current climate of the COVID-19 pandemic creates a heightened sense of urgency for effective implementation of such strategies. In order for these technologies to be adopted in drug development studies, a regulatory-aligned consensus on best practices in implementing appropriate technologies, including the collection, processing, and interpretation of digital sensor data, is required. A growing number of collaborative initiatives are being launched to identify effective ways to advance the use of DHT in PD clinical trials. The Critical Path for Parkinson's Consortium of the Critical Path Institute is highlighted as a case example where stakeholders collectively engaged regulatory agencies on the effective use of DHT in PD clinical trials. Global regulatory agencies, including the US Food and Drug Administration and the European Medicines Agency, are encouraging the efficiencies of data-driven engagements through multistakeholder consortia. To this end, we review how the advancement of DHT can be most effectively achieved by aligning knowledge, expertise, and data sharing in ways that maximize efficiencies.</p>","PeriodicalId":11242,"journal":{"name":"Digital Biomarkers","volume":"4 Suppl 1","pages":"28-49"},"PeriodicalIF":0.0000,"publicationDate":"2020-11-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7768153/pdf/dib-0004-0028.pdf","citationCount":"0","resultStr":"{\"title\":\"Precompetitive Consensus Building to Facilitate the Use of Digital Health Technologies to Support Parkinson Disease Drug Development through Regulatory Science.\",\"authors\":\"Diane Stephenson, Robert Alexander, Varun Aggarwal, Reham Badawy, Lisa Bain, Roopal Bhatnagar, Bastiaan R Bloem, Babak Boroojerdi, Jackson Burton, Jesse M Cedarbaum, Josh Cosman, David T Dexter, Marissa Dockendorf, E Ray Dorsey, Ariel V Dowling, Luc J W Evers, Katherine Fisher, Mark Frasier, Luis Garcia-Gancedo, Jennifer C Goldsack, Derek Hill, Janice Hitchcock, Michele T Hu, Michael P Lawton, Susan J Lee, Michael Lindemann, Ken Marek, Nitin Mehrotra, Marjan J Meinders, Michael Minchik, Lauren Oliva, Klaus Romero, George Roussos, Robert Rubens, Sakshi Sadar, Joseph Scheeren, Eiichi Sengoku, Tanya Simuni, Glenn Stebbins, Kirsten I Taylor, Beatrice Yang, Neta Zach\",\"doi\":\"10.1159/000512500\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Innovative tools are urgently needed to accelerate the evaluation and subsequent approval of novel treatments that may slow, halt, or reverse the relentless progression of Parkinson disease (PD). Therapies that intervene early in the disease continuum are a priority for the many candidates in the drug development pipeline. There is a paucity of sensitive and objective, yet clinically interpretable, measures that can capture meaningful aspects of the disease. This poses a major challenge for the development of new therapies and is compounded by the considerable heterogeneity in clinical manifestations across patients and the fluctuating nature of many signs and symptoms of PD. Digital health technologies (DHT), such as smartphone applications, wearable sensors, and digital diaries, have the potential to address many of these gaps by enabling the objective, remote, and frequent measurement of PD signs and symptoms in natural living environments. The current climate of the COVID-19 pandemic creates a heightened sense of urgency for effective implementation of such strategies. In order for these technologies to be adopted in drug development studies, a regulatory-aligned consensus on best practices in implementing appropriate technologies, including the collection, processing, and interpretation of digital sensor data, is required. A growing number of collaborative initiatives are being launched to identify effective ways to advance the use of DHT in PD clinical trials. The Critical Path for Parkinson's Consortium of the Critical Path Institute is highlighted as a case example where stakeholders collectively engaged regulatory agencies on the effective use of DHT in PD clinical trials. Global regulatory agencies, including the US Food and Drug Administration and the European Medicines Agency, are encouraging the efficiencies of data-driven engagements through multistakeholder consortia. To this end, we review how the advancement of DHT can be most effectively achieved by aligning knowledge, expertise, and data sharing in ways that maximize efficiencies.</p>\",\"PeriodicalId\":11242,\"journal\":{\"name\":\"Digital Biomarkers\",\"volume\":\"4 Suppl 1\",\"pages\":\"28-49\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2020-11-26\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7768153/pdf/dib-0004-0028.pdf\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Digital Biomarkers\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1159/000512500\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2020/1/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"Q1\",\"JCRName\":\"Computer Science\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Digital Biomarkers","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1159/000512500","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2020/1/1 0:00:00","PubModel":"eCollection","JCR":"Q1","JCRName":"Computer Science","Score":null,"Total":0}
Precompetitive Consensus Building to Facilitate the Use of Digital Health Technologies to Support Parkinson Disease Drug Development through Regulatory Science.
Innovative tools are urgently needed to accelerate the evaluation and subsequent approval of novel treatments that may slow, halt, or reverse the relentless progression of Parkinson disease (PD). Therapies that intervene early in the disease continuum are a priority for the many candidates in the drug development pipeline. There is a paucity of sensitive and objective, yet clinically interpretable, measures that can capture meaningful aspects of the disease. This poses a major challenge for the development of new therapies and is compounded by the considerable heterogeneity in clinical manifestations across patients and the fluctuating nature of many signs and symptoms of PD. Digital health technologies (DHT), such as smartphone applications, wearable sensors, and digital diaries, have the potential to address many of these gaps by enabling the objective, remote, and frequent measurement of PD signs and symptoms in natural living environments. The current climate of the COVID-19 pandemic creates a heightened sense of urgency for effective implementation of such strategies. In order for these technologies to be adopted in drug development studies, a regulatory-aligned consensus on best practices in implementing appropriate technologies, including the collection, processing, and interpretation of digital sensor data, is required. A growing number of collaborative initiatives are being launched to identify effective ways to advance the use of DHT in PD clinical trials. The Critical Path for Parkinson's Consortium of the Critical Path Institute is highlighted as a case example where stakeholders collectively engaged regulatory agencies on the effective use of DHT in PD clinical trials. Global regulatory agencies, including the US Food and Drug Administration and the European Medicines Agency, are encouraging the efficiencies of data-driven engagements through multistakeholder consortia. To this end, we review how the advancement of DHT can be most effectively achieved by aligning knowledge, expertise, and data sharing in ways that maximize efficiencies.