Digital Health Technologies and the Drug Development Life Cycle

Pain Points in the Drug R&D Lifecycle

The expanding frontier of biomedical science has led to increasing numbers of new drug candidates in the pipeline. However, progress from the laboratory bench to the patient’s bedside has been hampered at critical points in the drug lifecycle.

Challenges include:

• Recruitment and retention of study participants in clinical research Yvonne Yu-Feng Chan, Senior Director, Medical Affairs for Digital Medicine, Otsuka Pharmaceutical; Ritu Kapur, Head of Biomarkers, Verily Life Sciences; Sally Okun, Director of Policy and Ethics, UnitedHealth Group Research & Development; Edmondo Robinson, Chief Digital Innovation Officer, Moffitt Cancer Center
• Intermittent data collection across study sites Amy Abernethy, Principal Deputy Commissioner of Food and Drugs, U.S. Food and Drug Administration; Eric Perakslis, Rubenstein Fellow, Duke University; Larsson Omberg, Vice President of Systems Biology, Sage Bionetworks; Leonard Sacks, Associate Director for Clinical Methodology, Office of Medical Policy, Center for Drug Evaluation and Research, U.S. Food and Drug Administration
• Insufficient means of objective clinical event measurement Sean Khozin, Global Head of Data Strategy, Janssen Research & Development, LLC; Ritu Kapur, Head of Biomarkers, Verily Life Sciences; Leonard Sacks, Associate Director for Clinical Methodology, Office of Medical Policy, Center for Drug Evaluation and Research, U.S. Food and Drug Administration
• Data interoperability across data sources Luca Foschini, Chief Data Scientist and Co-Founder, Evidation Health; Alicia Staley, Senior Director of Patient Engagement, Medidata Solutions; Joseph Menetski, Associate Vice President of Research Partnerships, Foundation for the National Institutes of Health
• Post-market surveillance (establishing comparative effectiveness) Camille Nebeker, Director, Research Center for Optimal Digital Ethics (ReCODE), University of California San Diego; Sally Okun, Director of Policy and Ethics, UnitedHealth Group Research & Development; Ritu Kapur, Head of Biomarkers, Verily Life Sciences; Edmondo Robinson, Chief Digital Innovation Officer, Moffitt Cancer Center

Applications for DHTs in Drug R&D

Over time, incorporating DHTs may help transform current drug R&D and clinical trial paradigms by enabling continuous data collection, providing surrogate endpoints for efficacy, supporting participant engagement to improve adherence and retention, and broadening access to and increasing representation of clinical research.

DHTs have the potential to:

• Enable continuous data collection outside of the clinic and bridge gaps in clinical research data Amy Abernethy, Principal Deputy Commissioner of Food and Drugs, U.S. Food and Drug Administration; Chris Benko, Chief Executive Officer, Koneksa Health; Chris Lunt, Chief Technology Officer, All of Us Research Program
• Enable validated digital endpoints to be used as surrogates for efficacy as well as continuous monitoring to track changes in disease trajectory over time Christopher Leptak, Director, Regulatory Science Program, Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration
• Diversify data sources and expand the volume of data captured in clinical research Sean Khozin, Global Head of Data Strategy, Janssen Research & Development, LLC; Camille Nebeker, Director, Research Center for Optimal Digital Ethics (ReCODE), University of California San Diego; Larsson Omberg, Vice President of Systems Biology, Sage Bionetworks
• Support virtual/decentralized trials to broaden access for participants and enable real-world data collection Yvonne Yu-Feng Chan, Senior Director, Medical Affairs for Digital Medicine, Otsuka Pharmaceutical; Leonard Sacks, Associate Director for Clinical Methodology, Office of Medical Policy, Center for Drug Evaluation and Research, U.S. Food and Drug Administration; Sean Khozin, Global Head of Data Strategy, Janssen Research & Development, LLC
• Enhance patient-centricity in drug development by capturing information related to a patient’s lived experience Amy Abernethy, Principal Deputy Commissioner of Food and Drugs, U.S. Food and Drug Administration; Alicia Staley, Senior Director of Patient Engagement, Medidata Solutions; Sally Okun, Director of Policy and Ethics, UnitedHealth Group Research & Development

Challenges in the Selection, Verification & Validation, Standardization, and Implementation of DHTs for use in Drug R&D

Despite the promise of DHTs, challenges remain regarding the selection, evaluation, verification and validation, standardization, and implementation of these technologies. As DHTs have become more prominent tools in clinical research and care, challenges have emerged around interoperability and integration of various data types from multiple sources, the establishment of analytical and clinical validity for digital health measures with real-world clinical outcomes, buy-in from clinical providers, and reimbursement.

Challenges include:

• Lack of gold standard reference measurements and benchmarks to demonstrate that a DHT measures what it is intending to measure Chris Lunt, Chief Technology Officer, All of Us Research Program; Ritu Kapur, Head of Biomarkers, Verily Life Sciences; Leonard Sacks, Associate Director for Clinical Methodology, Office of Medical Policy, Center for Drug Evaluation and Research, U.S. Food and Drug Administration; Joseph Menetski, Associate Vice President of Research Partnerships, Foundation for the National Institutes of Health
• Lack of common methodologies and standards for verification and validation of DHTs Ritu Kapur, Head of Biomarkers, Verily Life Sciences
• Procedural and technical issues associated with the high-dimensional nature of data (data velocity, volume, variety, and veracity) emerging from DHTs Sean Khozin, Global Head of Data Strategy, Janssen Research & Development, LLC
• Data governance, privacy, and security protections for ethical, responsible, and optimal use of DHTs Camille Nebeker, Director, Research Center for Optimal Digital Ethics (ReCODE), University of California San Diego; Chris Lunt, Chief Technology Officer, All of Us Research Program; Eric Perakslis, Rubenstein Fellow, Duke University; Luca Foschini, Chief Data Scientist and Co-Founder, Evidation Health
• Lack of representative clinical trials due to the digital/technological divide Alicia Staley, Senior Director of Patient Engagement, Medidata Solutions; Luca Foschini, Chief Data Scientist and Co-Founder, Evidation Health

Opportunities and A Way Forward

Establishing standards and best practices would benefit from the collective work of stakeholders that seek to incorporate DHTs into the drug development lifecycle. Optimizing the role of DHTs in drug R&D may involve collaboration among a broad range of stakeholders from across sectors—including consumers, patients, clinicians, regulators, biopharmaceutical companies, academics, and technology developers—to share insights, experiences, and lessons learned.

Opportunities include:

• Developing standards for improving interoperability, enhancing data governance, and increasing population diversity in data collected Luca Foschini, Chief Data Scientist and Co-Founder, Evidation Health
• Collaborating on a common methodology for verifying and validating digital metrics Ritu Kapur, Head of Biomarkers, Verily Life Sciences
• Building trust and establishing channels of communication, as well as developing communities to foster connections among study participants, could help participants better understand how their data will be used and shared. Alicia Staley, Senior Director of Patient Engagement, Medidata Solutions; Yvonne Yu-Feng Chan, Senior Director, Medical Affairs for Digital Medicine, Otsuka Pharmaceutical; Camille Nebeker, Director, Research Center for Optimal Digital Ethics (ReCODE), University of California San Diego
• Establishing consent mechanisms that are less onerous to help participants better understand their role in a study Camille Nebeker, Director, Research Center for Optimal Digital Ethics (ReCODE), University of California San Diego
• Returning data to patients in a meaningful and actionable way Michelle Crouthamel, Director of Digital Health and Innovation, AbbVie Inc.; Camille Nebeker, Director, Research Center for Optimal Digital Ethics (ReCODE), University of California San Diego
• Considering access and usability, data management, privacy, and risk/benefit to ensure that DHTs are developed in a way that is effective and ethical Camille Nebeker, Director, Research Center for Optimal Digital Ethics (ReCODE), University of California San Diego

DHTs have the potential to help stakeholders build trusted relationships between developers, sponsors, researchers, clinicians, and patients. Establishing standards for evaluating DHTs could be addressed through a collective decision to accept the best-available standards—and work to refine them—rather than allowing “perfect to be the enemy of the good” and continuing to operate without any standards at all. Jennifer Goldsack, Executive Director, Digital Medicine Society; Joseph Menetski, Associate Vice President of Research Partnerships, Foundation for the National Institutes of Health; Ritu Kapur, Head of Biomarkers, Verily Life Sciences; Eric Perakslis, Rubenstein Fellow, Duke University