Elsevier

Contemporary Clinical Trials

Volume 30, Issue 5, September 2009, Pages 436-445
Contemporary Clinical Trials

Marketing therapeutic precision: Potential facilitators and barriers to adoption of n-of-1 trials

https://doi.org/10.1016/j.cct.2009.04.001Get rights and content

Abstract

Background

N-of-1 trials may enhance therapeutic precision by predicting the long-term effectiveness of medical treatment on an individual basis. However, the n-of-1 approach has gained little traction with the clinical community. To learn why, we interviewed physicians and patients, focusing on the perceived benefits and drawbacks of n-of-1 trials and factors influencing these perceptions.

Methods

We convened focus groups and individual interviews with 21 physicians and 32 patients, most with chronic conditions. The study employed qualitative interview methods to explore and analyze subjects' views of n-of-1 trials. Analysis involved an iterative process of review and data abstraction after specific topics for coding, definitions of codes, and strategies for abstraction had been established. Previously defined domains and topics were then expanded and enriched, with key themes emerging during the analytic process.

Results

Physicians and patients remarked on 4 salient aspects of n-of-1 trials: scientific, relational, clinical, and logistical. Neither physicians nor patients were highly familiar with the n-of-1 concept, but both groups readily grasped the fundamental logic and appreciated the potential scientific benefits. Physicians saw n-of-1 trials as promoting an exciting but possibly threatening paradigm shift in the doctor–patient relationship, while patients viewed the relational consequences as modest. The best n-of-1 candidates were felt to be proactive, cognitively intact, reliable, motivated, and engaged in a trusting physician–patient relationship.

Conclusions

Researchers interested in expanding the appeal of n-of-1 trials will need to address these concerns by carefully explaining the approach, emphasizing the benefits, and minimizing the effort required of doctors and patients.

Section snippets

Methods

Our study was approved by the UC Davis and UCLA Institutional Review Boards. The study employed qualitative interview methods to explore and analyze subjects' views of the clinical research enterprise and n-of-1 trials. We recruited purposeful, non-random samples of physicians and patients, aiming to maximize diversity in terms of attitudes about and experience with clinical trials. Pre-established domains of inquiry and topics were used to systematically ask parallel (and in many cases

Participant characteristics

Twenty-one physician participants took part in one of three group interviews (n = 10) or an individual interview (n = 11). Group interviews involved 3–4 physicians and were held by teleconferencing. The 21 physician participants included 14 women and 7 men; 8 were non-white. The average age was 43. About half were general internists; 43% practiced within an academic medical center. Most reported spending at least 4 half-days per week in clinic (Table 1). Thirty-two patients participated in one of

Discussion

Though widely touted as a rigorous approach to individualization of therapy, n-of-1 trials have struggled to gain traction with physicians and patients [16]. We conducted this study to gain insight into perceived benefits and drawbacks of n-of-1 trials from the perspectives of physicians and the public. We learned that most physicians and patients readily grasp the logic of n-of-1 trials and appreciate their potential for personalizing care. However, both groups expressed reservations along

Acknowledgements

This work was funded by Pfizer but conducted, analyzed, and reported independently. Richard L. Kravitz had access to all of the data and takes responsibility for integrity and accuracy. The authors gratefully acknowledge Edward Niedzinski, PhD for his assistance in project management and interviewing, and Phil Raimondi, MD, Malathi Srinivasan, MD, and Andrew Hudnut, MD for help with physician recruiting.

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