Evaluation, Acceptance, and Qualification of Digital Measures: From Proof of Concept to Endpoint

Abstract

To support the successful adoption of digital measures into internal decision making and evidence generation for medical product development, we present a unified lexicon to aid communication throughout this process, and highlight key concepts including the critical role of participant engagement in development of digital measures. We detail the steps of bringing a successful proof of concept to scale, focusing on key decisions in the development of a new digital measure: asking the right question, optimized approaches to evaluating new measures, and whether and how to pursue qualification or acceptance. Building on the V3 framework for establishing verification and analytical and clinical validation, we discuss strategic and practical considerations for collecting this evidence, illustrated with concrete examples of trailblazing digital measures in the field.

Keywords: Acceptance; Digital endpoints; Digital measures; Patient centricity; Qualification; V3.

Fig. 1

From sensor to endpoint: the process of developing digital measures. Key phases of patient and stakeholder engagement, digital measure development and evaluation, and acceptance and qualification are outlined. Key practical differences are highlighted. Left: development and evaluation; participant engagement defines the key COI, sensor mapping defines how the COI might be captured, and this initial proof of concept is evaluated as a robust digital measure. This is typically done in a separate observational/cross-sectional study or directly as an exploratory aim in a clinical trial (or substudy). Right: acceptance and/or qualification: the developed measure is assessed to be fit for purpose, enabling it to be used as a basis for clinical decision making, e.g., for inclusion decisions, or promotion from exploratory to secondary endpoint. This process typically involves longitudinal clinical studies where the novel measure can be directly compared to the existing standard practice and/or gold standard measures. https://www.fda.gov/drugs/development-approval-process-drugs/drug-development-tools-ddts.