Nevin Manimala Statistics

Personalized (N-of-1) Trials for Patient-Centered Treatments of Multimorbidity

Harv Data Sci Rev. 2022;4(SI3). doi: 10.1162/99608f92.d99e6ff5. Epub 2022 Sep 8.


Treatment of patients who suffer from concurrent health conditions is not well served by (1) evidence-based clinical guidelines that mainly specify treatment of single conditions and (2) conventional randomized controlled trials (RCTs) that identify treatments as safe and effective on average. Clinical decision-making based on the average patient effect may be inappropriate for treatment of those with multimorbidity who experience burdens and obstacles that may be unique to their personal situation. We describe how the personalized (N-of-1) trials can be integrated with an automatic platform and virtual/remote technologies to improve patient-centered care for those living with multimorbidity. To illustrate, we present a hypothetical clinical scenario-survivors of both coronavirus disease 2019 (COVID-19) and cancer who chronically suffer from sleeplessness and fatigue. Then, we will describe how the four standard phases of conventional RCT development can be modified for personalized trials and applied to the multimorbidity clinical scenario, outline how personalized trials can be adapted and extended to compare the benefits of personalized trials versus between-subject trial design, and explain how personalized trials can address special problems associated with multimorbidity for which conventional trials are poorly suited.

PMID:38009131 | PMC:PMC10673634 | DOI:10.1162/99608f92.d99e6ff5

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