| Literature DB >> 32338719 |
Eric Hekler1,2,3, Jasmin A Tiro4,5, Christine M Hunter6, Camille Nebeker1,2,3.
Abstract
BACKGROUND: In 2015, Collins and Varmus articulated a vision for precision medicine emphasizing molecular characterization of illness to identify actionable biomarkers to support individualized treatment. Researchers have argued for a broader conceptualization, precision health. Precision health is an ambitious conceptualization of health, which includes dynamic linkages between research and practice as well as medicine, population health, and public health. The goal is a unified approach to match a full range of promotion, prevention, diagnostic, and treatment interventions to fundamental and actionable determinants of health; to not just address symptoms, but to directly target genetic, biological, environmental, and social and behavioral determinants of health.Entities:
Keywords: Implementation science; Precision health; Precision medicine; Research ethics; Research methods; Social and behavioral sciences
Year: 2020 PMID: 32338719 PMCID: PMC7646154 DOI: 10.1093/abm/kaaa018
Source DB: PubMed Journal: Ann Behav Med ISSN: 0883-6612
Fig. 1.A vision of precision health, with precision medicine as a referent. Collins and Varmus [1] provides an initial referent, which is then expanded to illustrate the potential future target of precision health. IRB Institutional Review Board; WHO World Health Organization.
Summary of four types of interventions to support tailoring
| Intervention classes | ||||
|---|---|---|---|---|
| Generic | Targeted | Adaptive | Continuous tuning | |
| Frequency of intervention selection | Single selection, based on inclusion/exclusion criteria or clinical expertise | Single selection, based on status of moderator variable | Repeated selection | Repeated selection |
| Relevant experimental designs | RCTs and FTs | RCTs and FTs with moderation hypotheses specified a priori | SMART, MRT, System ID | MRT + RL, COT |
| How are data from prior participants from prior studies used? | Intervention chosen based on main effect estimates of prior clinical trials; selection based on inclusion/exclusion criteria | Intervention chosen based on results from moderation tests from prior clinical trials | Intervention component type, frequency, and dosing and decision rules selected based on prior appropriate experimental designs | Insights from prior studies used as a “warm start” for identifying plausibly meaningful intervention component type, frequency, dosing, or adaptation algorithms |
| Targeted insights from studies conducted with prior individuals, to inform the intervention class | If the intervention produces a statistical and clinically meaningful effect compared with a meaningful control | If the intervention, for a subgroup as specified via a baseline variable as a moderator, produced a statistical and clinically meaningful effect among the subgroup | For SMART, if an intervention sequence is more favorable compared with other plausible treatment sequences. For MRT, if an intervention component type, frequency, or dose produces a statistical main effective or time-varying moderation. For System ID, if a dynamical model can be produced that is sufficiently stable and predictive of an individual’s future states, to inform plausible future dynamical models of individuals that can be selected at baseline as a “warm start” model | For MRT + RL, if an intervention option produces a meaningful effect within a particular state of the person, as defined by time-varying moderation variables; for System ID/COT, if a dynamical model can be produced that is sufficiently stable and predictive of an individual’s future states, to inform plausible future dynamical models of individuals that can be selected at baseline as a “warm start” model |
| How data from the current participant is used to select the intervention? | Data related to inclusion/ exclusion criteria are used to select the intervention | Baseline data previously shown as a reliable moderator are used to select, at one time point, one intervention over another | Via SMART, data from a participant defines selection of an appropriate intervention sequence/decision rule (e.g., what to offer to nonresponders). Via MRT, current repeated measures, define intervention type, frequency, or dose for that person at a given decision point. Via System ID, data are used to select and specify an initial dynamical model, as a “warm start” to guild predictions for each person | Via MRT + RL, same points as MRT described for adaptive plus current data on a person’s ongoing responsivity to an intervention is used to adjust the probability with which a person receives an intervention type, frequency, or dose; intervention type, frequency, or dose that a person responds more favorably to are selected more often; Via a COT, same as system ID, described in adaptation + intervention option selection is based on simulated predictions of future responses of a person based on a dynamical model generated for each person, coupled with a controller “closing the loop” to drive adaptive decision-making |
COT control optimization trial; FT factorial trial; MRT microrandomized trial; RCT randomized controlled trial; RL reinforcement learning; SMART sequential multiple assignment randomized trial.
Fig. 2.Two pathways to generalizable/transportable knowledge. The above visualizes two pathways to generalizable (labeled “transportable”) knowledge. The left pathway is the traditional research approach, which starts with generalizable knowledge as the target. The right pathway is an alternative strategy, which would enable better alignment of initial goals between research and practice. Image from Hekler et al. [94].
Recommendations for research on research ethics
| Principle | Application | Considerations for precision health research |
|---|---|---|
| Autonomy, respect for persons | The process of obtaining study information to facilitate decision-making Used to document voluntary participation Protect persons who have diminished capacity to make decisions | Are digital strategies for conveying study information appropriate? Do participants understand the granularity and volume of data collected? Is the consent content and process appropriate for people with limited technology and data literacy? What consent process is useful for an |
| Beneficence | Evaluation of the probability and magnitude of potential harms to participants Assessment of risks and mitigation strategies against potential benefits to the individual, persons represented by participants and society | What data management strategies (wireless transmission, encryption, etc.) are appropriate to ensure confidentiality of potentially sensitive digital data? What system-wide strategies are effective in capacity building? When data are obtained using commercial products, what terms of service and privacy protections are appropriate? Does the technology have sufficient evidence to support the use of the device/app? |
| Justice | Persons who participate should reflect those most likely to benefit from the study outcomes Considerations for vulnerable populations | What potential barriers to study access exist in digitally deployed studies? What methods increase participant representation and involvement as partners? Do preferences for privacy vary across lifespan or groups identified as underrepresented in research? |