Stakeholders will need to draft a new set of first principles to inform the rules of the road for digital health, because many of the traditional elements of clinical development may not have meaning. Drug developers, regulators and payers will need to continually re-examine the foundations underlying validation, efficacy and outcomes.
Succeeding in digital health will mean being prepared to abandon some deeply embedded concepts, and creatively bridging from analog paradigms that have been the basis of drug development and reimbursement.
In clinical development, as elsewhere in digital health, the concept of continual change means that many routinely employed parameters may need to be redefined.
This means revisiting first principles -- how to validate a result, what is a relevant control, what statistical models to use -- and applying these methodologies to define endpoints, demonstrate efficacy and produce outcomes that payers will reimburse.
Fit for purpose: The first step to validation
The concept of “fit-for-purpose” data is emerging as a central issue in digital health, in particular for validation of biomarkers and endpoints.
The idea is that data must derive from the relevant population for the intended use. In standard therapeutics or diagnostics, that is an obvious constraint, ensured via eligibility criteria for clinical trials.
However, digital health is starting to incorporate measures from wearables and other consumer health products that were not designed with