Using Incrementalism and Legitimacy to Ensure Ethical Use of Digital Health Data to Provide Accelerated Access to Medicines

Background: Provisional marketing approval and funding schemes allow medicines to be approved or funded on the basis of earlier data, with post-market data collection—including use of digital health data—to confirm their safety, effectiveness and cost-effectiveness. Ethical and responsible use of digital health data is necessary for both initiatives. Aims: To explore the beliefs of patients and physicians from Sydney, NSW regarding post-market data collection, including of digital health data, as a component of accelerated access. Methods: Semi-structured interviews were conducted with 18 Australian physicians and 13 patients and patient advocates; two focus groups were conducted with patients. Both patients and consumers were from medical specialties and disease areas in which accelerated access is most actively discussed. These were transcribed verbatim and analysed thematically. Results: There was significant diversity in stakeholder opinion. Although all acknowledged potential risks and benefits of increased reliance on post-market collection and use of digital health data, they disagreed on their magnitude and extent and how these should be balanced in both policy formation and clinical practice. Robust data collection, coupled with swift and decisive action by regulators and funders, were seen as necessary to address risks, but not all participants were confident this could be achieved. Conclusions: Stakeholder analysis shows a range of reasonable views on how to respond to calls for increased reliance on post-market collection and use of digital health data as part of accelerated access. Incremental policy change can maintain the protections of current systems while providing faster access to new medicines where this is needed most. However, not all substantive positions can be accommodated. A focus on fair procedures will increase legitimacy and acceptability of decisions about accelerated access to medicines. I offer a number of practical suggestions to achieve both legitimate and incremental policy making in regards use of post-market collection and use of digital health data in decisions about access to medicines