How Much Evidence Is Enough? Research Sponsor Experiences Seeking Regulatory Acceptance of Digital Health Technology-Derived Endpoints

Brian Perry; Lindsay Kehoe; Teresa Swezey; Quentin Le Masne; Jörg Goldhahn; Alicia Staley; Amy Corneli

doi:10.1159/000529878

How Much Evidence Is Enough? Research Sponsor Experiences Seeking Regulatory Acceptance of Digital Health Technology-Derived Endpoints

Digit Biomark. 2023 Jun 8;7(1):45-53. doi: 10.1159/000529878. eCollection 2023 Jan-Dec.

Authors

Brian Perry^{1

2}, Lindsay Kehoe^{1

3}, Teresa Swezey^{1

2}, Quentin Le Masne^{1

4}, Jörg Goldhahn^{1

5}, Alicia Staley^{1

6}, Amy Corneli^{1

2

3}

Affiliations

¹ Clinical Trials Transformation Initiative, Durham, NC, USA.
² Department of Population Health Sciences, Duke University School of Medicine, Durham, NC, USA.
³ Duke Clinical Research Institute, Duke University School of Medicine, Durham, NC, USA.
⁴ Global Digital Health, Merck KGaA, Darmstadt, Germany.
⁵ Institute for Translational Medicine, ETH Zurich, Zürich, Switzerland.
⁶ Medidata, a Dassault Systèmes Company, New York, NY, USA.

Abstract

Introduction: Digital health technologies (DHTs) provide opportunities for real-time data collection and assessment of patient function. However, use of DHT-derived endpoints in clinical trials to support medical product labelling claims is limited.

Methods: From November 2020 through March 2021, the Clinical Trials Transformation Initiative (CTTI) conducted a qualitative descriptive study using semi-structured interviews with sponsors of clinical trials that used DHT-derived endpoints. We aimed to learn about their experiences, including their interactions with regulators and the challenges they encountered. Using applied thematic analysis, we identified barriers to and recommendations for using DHT-derived endpoints in pivotal trials.

Results: Sponsors identified five key challenges to incorporating DHT-derived endpoints in clinical trials. These included (1) a need for additional regulatory clarity specific to DHT-derived endpoints, (2) the official clinical outcome assessment qualification process being impractical for the biopharmaceutical industry, (3) a lack of comparator clinical endpoints, (4) a lack of validated DHTs and algorithms for concepts of interest, and (5) a lack of operational support from DHT vendors.

Discussion/conclusion: CTTI shared the interview findings with the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and during a multi-stakeholder expert meeting. Based on these discussions, we provide several new and revised tools to aid sponsors in using DHT-derived endpoints in pivotal trials to support labelling claims.

Keywords: Clinical outcome assessment; Digital health technology; Endpoint; Regulatory trial.

Abstract

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