Bert Hartog

Background: The pharmaceutical industry faces unprecedented challenges, including declining drug approval rates, plummeting research and development returns, and escalating protocol complexity. Digital transformation represents the most promising end-to-end approach for addressing these challenges, yet there is limited understanding of industry-wide priorities and implementation barriers. Objective: This study aimed to identify digital transformation priorities across diverse pharmaceutical industry stakeholders and to explore the specific barriers preventing successful implementation through a collaborative, mixed methods approach. Methods: The study used a sequential mixed methods design with three phases: (1) workshop 1 in Copenhagen, Denmark, with 47 participants randomly divided into 3 working groups, all discussing three main theme areas and generating 110 digital transformation topics; (2) a follow-up online survey of workshop 1 participants (administered 4 wk after workshop 1); and (3) workshop 2 in Boston, United States, with expert keynote and panel discussions exploring implementation challenges. Participants were recruited via convenience sampling through industry networks and professional associations. The study was conducted between March 2023 and June 2025 across international workshop sites in Copenhagen and Boston. Data sources included workshop outputs, survey responses, and transcribed panel discussions. Analysis involved thematic coding, convergence analysis, and tension identification. Results: Workshop 1 identified 19 common categories across all working groups, despite the 3 diverse starting themes. Workshop 1 produced 7 consolidated categories for theme 1 (desired outcomes), 5 for theme 2 (pathways), and 7 for theme 3 (measuring success), totaling 19 consolidated categories. The survey achieved a 55.3% (26/47 participants) response rate and identified patient-centered outcomes, FAIR (Findable, Accessible, Interoperable, and Reusable)–aligned data practices, collaboration, access and equity, and the reduction of patient burden as leading priorities. Workshop 2 identified 5 key implementation tensions: innovation speed vs safety standards, collaboration vs competition, technology vs change management, patient preferences vs operational efficiency, and data democratization vs privacy protection. Panel discussions provided deep insights into implementation challenges and solutions. The results obtained support exploratory generalization across comparable settings and do not establish causal effects. Conclusions: The pharmaceutical industry demonstrates strong consensus on patient-centric digital transformation priorities across the value chain, with universal recognition of the need for precompetitive collaboration. However, significant implementation barriers exist beyond technology, requiring integrated approaches to organizational change, regulatory innovation, and industry-wide standards development. Panel discussions and expert insights provide a pathway for continued collaboration and knowledge sharing. An incremental approach to sharing industry-wide knowledge is further recommended.

BACKGROUND The pharmaceutical industry faces unprecedented challenges including declining drug approval rates, plummeting R&D returns [1, 2], and escalating protocol complexity. Digital transformation represents the most promising end-to-end approach for addressing these challenges, yet limited understanding exists regarding industry-wide priorities and implementation barriers. OBJECTIVE This study aimed to identify digital transformation priorities across diverse pharmaceutical industry stakeholders and explore the specific barriers preventing successful implementation through a collaborative, mixed-methods approach. METHODS We employed a sequential mixed-methods design with three phases: (1) WS1 (Denmark) with 47 participants randomly divided into 3 working groups, all discussing three main theme areas and generating 110 digital transformation topics; (2) Follow-up survey with 55.3% response rate (26/47 participants) validating topic priorities across all three theme areas; and (3) WS2 with expert keynote and panel discussions exploring implementation challenges. Data sources included workshop outputs, survey responses, and transcribed panel discussions. Analysis involved thematic coding, convergence analysis, and tension identification. RESULTS Analysis revealed 19 common categories across all working groups despite the 3 diverse starting themes. WS1 produced 7 consolidated categories for Theme 1 (desired outcomes), 5 for Theme 2 (pathways), and 7 for Theme 3 (measurement), totaling 19 consolidated categories. These were further refined into 17 survey topics (8+4+5) through post-workshop consolidation. The survey confirmed strong consensus with 55.3% response rate (26/47 participants). WS2 identified 5 key implementation tensions: innovation speed vs. safety standards, collaboration vs. competition, technology vs. change management, patient preferences vs. operational efficiency, and data democratization vs. privacy protection. Panel discussions provided deep insights into implementation challenges and solutions. Results obtained support exploratory generalization across comparable settings and do not establish causal effects. CONCLUSIONS The pharmaceutical industry demonstrates strong consensus on patient-centric digital transformation priorities as seen across the value chain, with universal recognition of the need for pre-competitive collaboration. However, significant implementation barriers exist beyond technology, requiring integrated approaches to organizational change, regulatory innovation, and industry-wide standards development. Panel discussions and expert insights provide a pathway for continued collaboration and knowledge sharing. Incremental approach to sharing of industry-wide knowledge is further recommended. CLINICALTRIAL This study is not a clinical trial.

We propose the addition of usability validation to the extended V3 framework, now “V3+”, and describe a pragmatic approach to ensuring that sensor-based digital health technologies can be used optimally at scale by diverse users. Alongside the original V3 components (verification; analytical validation; clinical validation), usability validation will ensure user-centricity of digital measurement tools, paving the way for more inclusive, reliable, and trustworthy digital measures within clinical research and clinical care.

In the last few decades, developers of new drugs, biologics, and devices have increasingly leveraged digital health technologies (DHTs) to assess clinical trial digital endpoints. To our knowledge, a comprehensive assessment of the financial net benefits of digital endpoints in clinical trials has not been conducted. We obtained data from the Digital Medicine Society (DiMe) Library of Digital Endpoints and the US clinical trials registry, ClinicalTrials.gov. The benefit metrics are changes in trial phase duration and enrollment associated with the use of digital endpoints. The cost metric was obtained from an industry survey of the costs of including digital endpoints in clinical trials. We developed an expected net present value (eNPV) model of the cash flows for new drug development and commercialization to assess financial value. The value measure is the increment in eNPV that occurs when digital endpoints are employed. We also calculated a return on investment (ROI) as the ratio of the estimated increment in eNPV to the mean digital endpoint implementation cost. For phase II trials, the increase in eNPV varied from $2.2 million to $3.3 million, with ROIs between 32% and 48% per indication. The net benefits were substantially higher for phase III trials, with the increase in eNPV varying from $27 million to $40 million, with ROIs that were four to six times the investment. The use of digital endpoints in clinical trials can provide substantial extra value to sponsors developing new drugs, with high ROIs.

Background In the last few decades developers of new drugs, biologics, and devices have increasingly leveraged digital health technologies (DHTs) to assess clinical trial digital endpoints. To our knowledge, a comprehensive assessment of the financial net benefits of digital endpoints in clinical trials has not been conducted. Data and Methods We obtained data from the Digital Medicine Society (DiMe) Library of Digital Endpoints and the U.S. clinical trials registry, ClinicalTrials.gov. The benefit metrics are changes in trial phase duration and enrollment associated with the use of digital endpoints. The cost metric was obtained from an industry survey of the costs of including digital endpoints in clinical trials. We developed an expected net present value (eNPV) model of the cash flows for new drug development and commercialization to assess financial value. The value measure is the increment in eNPV that occurs when digital endpoints are employed. We also calculated a return on investment (ROI) as the ratio of the estimated increment in eNPV to the mean digital endpoint implementation cost. Results For phase 2 trials, the increase in eNPV varied from $2.2 million to $3.3 million, with ROIs between 32% to 48% per indication. The net benefits were substantially higher for phase 3 trials, with the increase in eNPV varying from $27 million to $48 million, with ROIs that were four to seven times the investment. Conclusions The use of digital endpoints in clinical trials can provide substantial extra value to sponsors developing new drugs, with high ROIs.