Engaging Stakeholders in Building Patient-Centered, N-of-1 Randomized and Other Controlled Trial Methods [Methods Study], New York, 2014-2019 (ICPSR 39585)

Version Date: Nov 11, 2025

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Principal Investigator(s):
Nathalie Moise, Columbia University Medical Center

https://doi.org/10.3886/ICPSR39585.v1

Version V1

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Summary

Medical studies usually focus on learning how a treatment works for most people. These studies have strict rules about who can take part. Patients with multiple health problems, such as high blood pressure and diabetes together, often can't take part because in such medical studies having multiple health problems can cause patients to respond differently to treatments than people with fewer health problems.

N-of-1 studies are a different way to design research. People with multiple health problems can take part in N-of-1 studies. An N-of-1 study has only one patient. Doctors try different treatments with that person and look for the treatment that works best. In this study, the research team wanted to know:

  • Whether patients and doctors thought N-of-1 studies could be useful
  • Which health problems patients thought N-of-1 studies could be helpful for
  • Which features, such as the length of the study or how much time it took to track treatment results, affected whether patients wanted to be in an N-of-1 study

Citation

Moise, Nathalie. Engaging Stakeholders in Building Patient-Centered, N-of-1 Randomized and Other Controlled Trial Methods [Methods Study], New York, 2014-2019. Inter-university Consortium for Political and Social Research [distributor], 2025-11-11. https://doi.org/10.3886/ICPSR39585.v1

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Funding

Patient-Centered Outcomes Research Institute (PCORI) (ME-1403-12304)

Subject Terms

Geographic Coverage

Distributor(s)

Inter-university Consortium for Political and Social Research
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Time Period(s)

2014 -- 2019
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Study Purpose

To determine patients' and clinicians' preferences for the clinical uses and design features of N-of-1 trials. Researchers aimed (1) to conduct in-depth focus groups with patients and clinicians to determine the circumstances under which n-of-1 trials are considered most valuable; (2) to survey a national sample of patients with multiple chronic conditions (MCCs) to elicit n-of-1 trial preferred medical conditions, symptoms, and outcomes; and (3) to survey a second national sample to determine preferences for an n-of-1 trial design. We surveyed patients with MCCs, who are often excluded from randomized controlled trials and are likely to have substantial HTEs.

Study Design

Standard clinical trials typically exclude patients with multiple chronic conditions (MCCs) because of higher variation in patients' responses to the same treatment, or heterogeneity of treatment effect (HTE). HTE can complicate the choice of effective treatments. N-of-1 trials include one patient and multiple-period crossover experiments that compare two or more treatments to detect the patient's response to treatment.

N-of-1 trials can test treatment response in patients with MCCs to help clinical decision making; however, clinicians and patients often do not understand or engage in these trials. In this study, the researchers wanted to determine clinicians' and patients' preferences for participation in N-of-1 trials and the conditions that influence those preferences. Researchers gathered input in three stages from patients with MCCs and clinicians who treat such patients:

  • Focus groups. Focus groups in English and Spanish included 54 patients and 24 clinicians recruited from two health centers in New York. Researchers used the findings to create two surveys.
  • Survey 1. To identify the preferred clinical conditions and symptoms for which patients would be willing to participate in an N-of-1 trial, researchers surveyed 501 patients with MCCs using a general population online poll panel. Of these, 89% were white, 8% were African American, and 1% were Asian; 4% were Hispanic. The average age was 56, and 57% were female.
  • Survey 2. Based on the focus group and initial survey findings, researchers created N-of-1 trial prototypes to describe hypothetical designs. In the second survey, patients identified their preferences among eight features of N-of-1 trial designs. A different group of 501 patients with MCCs participated. Of these, 79% were white, 13% were African American, and 5% were Asian; 15% were Hispanic. The average age was 57, and 56% were female. Patients with MCCs, clinicians, researchers, and web designers participated in planning the study

Universe

Clinicians and patients from two health centers in New York.

Data Source

Qualitative data from focus groups; surveys from 1,002 adult patients with MCCs

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Original Release Date

2025-11-11

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Notes

  • The public-use data files in this collection are available for access by the general public. Access does not require affiliation with an ICPSR member institution.

  • ICPSR usually offers files in multiple formats for researchers to be able to access data and documentation in formats that work well within their needs. If you have questions about the accessibility of materials distributed by ICPSR or require further assistance, please visit ICPSR’s Accessibility Center.

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This study is maintained and distributed by the Patient-Centered Outcomes Data Repository (PCODR). PCODR is the official data repository of the Patient-Centered Outcomes Research Initiative (PCORI).