Wearables, Sensors, and Apps Part 1 – Updates on FDA’s PDUFA VII Goals for the Use of Digital Health Technologies in Drug and Biological Product Development

August 24, 2023By Adrienne R. Lenz, Principal Medical Device Regulation Expert

As part of FDA’s Prescription Drug User Fee Act (PDUFA) VII goals published in PDUFA Reauthorization Performance Goals and Procedures Fiscal Years 2023 through 2027 (PDUFA Goal Letter), which we blogged about here, FDA included a goal for enhancing use of digital health technologies (DHT) to support drug development and review.  A DHT is considered “a system that uses computing platforms, connectivity, software, and sensors for healthcare and related uses.” DHTs have been used in clinical trials for decades in the context of measurements made when participants visit clinical trial sites.  More recently, use of wearable sensors and applications running on mobile devices (Apps) by study participants has introduced new questions on how to incorporate DHTs into the study, and what information is needed to verify and validate that they are fit for purpose in the study.

This blog provides an update on the DHT-related PDUFA VII goals that were targeted for completion in the first two quarters of FDA’s Fiscal Year (FY) 2023, including:

  • By the end of Q2 FY 2023, FDA will establish a DHT framework document to guide the use of DHT-derived data in regulatory decision-makings for drugs and biological products.
  • By the end of Q2 FY 2023, FDA will establish a committee including members from CDER and CBER to support implementation of the commitments in this section.
  • By the end of Q2 FY 2023, FDA will convene the first of a series of 5 public meetings or workshops with key stakeholders including patients, biopharmaceutical companies, DHT companies, and academia to gather input into issues related to the use of DHTs in regulatory decision-making.
  • By the end of Q1 FY 2023, FDA will publish draft, revised, or final guidance on the use of DHTs in traditional and decentralized clinical trials, addressing the validation of measurements made by DHTs, the development of novel endpoints using DHTs, the use of DHTs as new ways to measure existing endpoints, approaches to using the patients’ own DHTs such as cell phones or smart watches, usability considerations for patients, detection of safety signals during continuous data acquisition, and issues related to security and confidentiality of data.
  • By end of Q2 FY 2023, FDA will enhance its internal systems to support review of DHT-related submissions including capturing key information about clinical trials utilizing DHTs to support tracking the number and rate of change of DHT related submissions.

FDA has made good progress on these initial goals, providing updates on the first four on the Digital Health Technologies (DHTs) for Drug Development website.  Earlier this year, FDA published the Framework for the Use of Digital Health Technologies in Drug and Biological Product Development (DHT Framework).  As part of the DHT Framework, a DHT Steering Committee has also been established.  In March of this year, FDA convened the first public meeting, Understanding Priorities for the Use of Digital Health Technologies to Support Clinical Trials for Drug Development and Review.  FDA also released, in December 2021, draft guidance for Digital Health Technologies for Remote Data Acquisition in Clinical Investigations.

The DHT Framework describes activities being implemented to meet the goals established in the PDUFA Goal Letter.  Activities within FDA include the establishment of the steering committee, enhancing the agency’s technical expertise and training, promoting consistency of evaluations across review divisions, statistical considerations in the analysis of DHT-derived data, and enhancing IT capabilities to support the review of DHT-generated data.  External programs include FDA meetings with sponsors, drug development tool qualification, establishing guidance, public meetings, demonstration projects, and engagement with external organizations.

The DHT steering committee includes senior staff from CDER, CBER, CDRH, including the Digital Health Center of Excellence (DHCoE), the Oncology Center of Excellence, and the Office of Clinical Policy and Programs.  Given that the same DHT measurement may be used for development of multiple drug, biologic, or medical device products and may itself be considered a medical device, the cross-center membership of the steering committee is intended to lead to consistent expectations for use of DHTs in product development and use of DHT-derived data in regulatory decision-making across the Agency.

We’ll continue to monitor FDA’s progress against its goals related to DHTs and the various activities outlined in the DHT Framework.